Q2 2026 Scholar Rock Holding Corp Earnings Call

August 6, 2026

Speaker #1: There, and Vikas Sinha, Chief Financial Officer. During today's call, David will provide introductory remarks in a business update, Akshay will review our R&D progress, Keith will provide an update on our commercial readiness activities and Vikas will provide a financial update.

Speaker #1: We will then open the call for questions. Before we begin, I'd like to remind you that during this call we will be making various statements about Scholar Rock's expectations, plans, and prospects, that constitute forward-looking statements for the purposes of Safe Harbor Provisions under the Private Securities Litigation Reform Act of 1995.

Speaker #1: Any forward-looking statements represent our views only as of today, and should not be relied upon as representing our views as of any future date.

Speaker #1: I encourage you to go to the investors in media section of our website for our most up-to-date SEC statements and filings. With that, I'd like to turn the call over to David.

Speaker #1: David?

Speaker #2: Thank you, Laura, and good morning. Thanks to everyone for joining our second quarter earnings call. Today, Scholar Rock is operating from a position of strength as we enter a defining period for our company and for the SMA community.

Speaker #2: Across the business, we are executing with focused discipline and urgency as we drive toward the U.S. launch of a pittogram map this quarter. Advance our epidogram map MAA in Europe to approval and prepare for launch in Germany.

Speaker #2: Build momentum across our world-leading anti-miostatin platform and maintain the financial strength to support our ambitions. Most importantly, we are on world's first muscle-targeted therapy to children and adults living with SMA as we advance epidogram map through the final stages of the FDA regulatory process.

Speaker #2: Let me now provide additional detail on the ongoing review of our epidogram map BLA in the U.S. As a reminder, the sole approveability issue for epidogram map noted in the complete response letter that we received on our priority review PDUFA date last September was related to observations identified at a routine general site inspection of the Catalin Indiana fill finish facility owned and operated by Novo Nordisk.

Speaker #2: Since our constructive and collaborative Type A meeting in November, the cadence of activities has reflected the shared understanding between us and the agency of the high unmet need in the SMA community and a shared sense of urgency to bring a pittogram map to children and adults with SMA as rapidly as possible.

Speaker #2: We are grateful for the agency's sustained level of engagement and for our ongoing dialogue including our March 3 Type C meeting where we discussed the accelerated progress that we had made at our second fill finish facility and the agreed-upon data package to facilitate the FDA review of the second fill finish facility.

Speaker #2: In alignment with FDA guidance from this discussion, we submitted the epidogram map BLA on March 30 with two fill finish facilities, both Catalin Indiana and our second fill finish facility.

Operator: Good morning, ladies and gentlemen, and welcome to Scholar Rock's Q2 2026 conference call. At this time, all lines are in listen-only mode. Following the presentation, we will conduct a question and answer session. To ask a question, please press star one one on your telephone. You will then hear an automated message advising that your hand is raised. If you would like to remove yourself from the queue, press star one one again. We also ask that you limit yourself to one question. This call is being recorded on Thursday, 7 August 2026. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Basma Radwan: Good morning, ladies and gentlemen, and welcome to Scholar Rock's Q2 2026 conference call. At this time, all lines are in listen-only mode. Following the presentation, we will conduct a question and answer session. To ask a question, please press star one one on your telephone. You will then hear an automated message advising that your hand is raised. If you would like to remove yourself from the queue, press star one one again. We also ask that you limit yourself to one question. This call is being recorded on Thursday, 7 August 2026. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Speaker #2: Our BLA was accepted in April with a PDUFA date of September 30. Agency review of our application is progressing well. Importantly, we have significant optionality with two independent paths to approval, either through Catalin Indiana or through our second fill finish facility or both, whichever is determined to be the most rapid.

Speaker #1: Following the presentation , we will conduct a question and answer session . To ask a question , please press star one one on your telephone .

Speaker #1: raised . If you would like to remove yourself from the queue , You star one again . We also ask that you limit yourself to one question .

Speaker #1: This call is being recorded on Thursday , August 6th , 2026 . I would now like to turn the conference over to Scholar Rock .

Speaker #2: As it relates to Catalin Indiana, the FDA inspection classification following the April 2026 general site inspection is pending. We continue to be very pleased by the progress made at our second fill finish facility.

Speaker #1: Please go ahead .

Laura Ekis: Good morning. I'm Laura Ekis, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Board Chair and Chief Executive Officer, Akshay Vaishnaw, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. During today's call, David will provide introductory remarks and a business update. Akshay will review our R&D progress. Keith will provide an update on our commercial readiness activities. Vikas will provide a financial update. We will then open the call for questions. Before we begin, I'd like to remind you that during this call, we will be making various statements about Scholar Rock's expectations, plans, and prospects that constitute forward-looking statements for the purposes of safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Laura Ekis: Good morning. I'm Laura Ekas, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Board Chair and Chief Executive Officer, Akshay Vaishnaw, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. During today's call, David will provide introductory remarks and a business update. Akshay will review our R&D progress. Keith will provide an update on our commercial readiness activities. Vikas will provide a financial update. We will then open the call for questions. Before we begin, I'd like to remind you that during this call, we will be making various statements about Scholar Rock's expectations, plans, and prospects that constitute forward-looking statements for the purposes of safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Speaker #2: Importantly, the data package for the FDA's review of the second facility has been submitted and the review is progressing. Underscoring our operational excellence at our second site, we now have more vials available from this facility than we do from Catalin Indiana.

Speaker #2: Notably, vials from both Catalin Indiana and our second facility are now on site at our third-party provider awaiting packaging and labeling upon approval. As we near the final step in the U.S.

Speaker #2: regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease. Turning now to our commercial readiness in the U.S., our team continues to advance our launch preparations across all key functions.

Laura Ekis: Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date. I encourage you to go to the Investors and Media section of our website for our most up-to-date SEC statements and filings. With that, I'd like to turn the call over to David. David?

Speaker #2: Ensuring we are prepared to support patients, caregivers, and prescribers from day one. Our team is ready to launch epidogram map at any time prior to and including our September 30 PDUFA date.

David Hallal: Thank you, Laura, and good morning. Thanks to everyone for joining our Q2 earnings call. Today, Scholar Rock is operating from a position of strength as we enter a defining period for our company and for the SMA community. Across the business, we are executing with focus, discipline, and urgency as we drive towards the US launch of apitegromab this quarter, advance our apitegromab MAA in Europe to approval and prepare for launch in Germany, build momentum across our world-leading anti-myostatin platform, and maintain the financial strength to support our ambitions. Most importantly, we are on the threshold of bringing the world's first muscle-targeted therapy to children and adults living with SMA as we advance apitegromab through the final stages of the FDA regulatory process. Let me now provide additional detail on the ongoing review of our apitegromab BLA in the US.

Speaker #2: Keith will discuss our commercial preparations and greater detail shortly. In addition to the U.S., we continue to look forward to serving children and adults living with SMA in Europe.

Speaker #2: I would now like to provide an update on where we stand in the European regulatory process. The epidogram map MAA includes only Catalin Indiana fill finish facility and the EMA is awaiting the FDA inspection classification for that facility.

Speaker #2: In parallel, we are engaging with European regulators with regards to potential inclusion of our second fill finish facility in the epidogram map application. Importantly, this facility has had recent inspections by the FDA and the EMA.

Speaker #2: We are grateful for the EMA's continued level of engagement, and and we look forward to providing updated guidance on the potential timing of a CHMP opinion upon alignment with the European regulators.

David Hallal: As a reminder, the sole approvability issue for apitegromab noted in the Complete Response Letter that we received on our priority review PDUFA date last September was related to observations identified at a routine general site inspection of the Catalent Indiana fill-finish facility owned and operated by Novo Nordisk. Since our constructive and collaborative Type A meeting in November, the cadence of activities has reflected the shared understanding between us and the agency of the high unmet need in the SMA community and a shared sense of urgency to bring apitegromab to children and adults with SMA as rapidly as possible.

Speaker #2: Turning to our launch preparations in Europe, we are executing our commercial playbook. Building momentum with launch readiness activities and engaging with the SMA community.

Speaker #2: We are planning for an initial launch in Germany with additional countries and regions to follow as we build out our planned 50-country operating platform.

Speaker #2: We know it is not a matter of if, but when epidogram map will be approved for children and adults with SMA in both the U.S.

Speaker #2: and Europe. And we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN-targeted therapy.

David Hallal: We are grateful for the agency's sustained level of engagement and for our ongoing dialogue, including our 3 March Type C meeting, where we discussed the accelerated progress that we had made at our second fill-finish facility and the agreed-upon data package to facilitate the FDA review of the second fill-finish facility. In alignment with FDA guidance from this discussion, we submitted the apitegromab BLA on 30 March with two fill-finish facilities, both Catalent Indiana and our second fill-finish facility. Our BLA was accepted in April with a PDUFA date of 30 September. Agency review of our application is progressing well. Importantly, we have significant optionality with two independent paths to approval, either through Catalent Indiana or through our second fill-finish facility, or both, whichever is determined to be the most rapid.

Speaker #2: Turning now to our world-leading anti-myostatin pipeline. We continue to make meaningful progress with our key clinical programs. We have robust enrollment in our Phase 2 OPAL study evaluating epidogram map in infants and toddlers with SMA.

Speaker #2: We initiated our randomized Phase 2 FORGE study in patients with FSHD. We are ready to engage with U.S. and European regulators on the development path of our high-concentration subcutaneous formulation of epidogram map, which we will do once we have regulatory approvals.

We are grateful for the agency, sustained level of Engagement, and for our ongoing dialogue included, our March 3rd, type c meeting where we discussed the accelerated progress that we had made at our second, Phil finish facility and the agreed upon data package to facilitate the FDA review of the second Bill finish facility.

Speaker #2: And enrollment and dosing is proceeding very well in our Phase 1 Healthy Volunteer study for SRK 439, our novel high-potency anti-myostatin antibody. Akshay will discuss these programs and greater detail shortly.

In alignment, with FDA guidance from this discussion. We submitted the epitome of bla on March 30th with 2, Phil finished facilities, both Catalin Indiana, and our second fill finish facility.

Our bla was accepted in April with a pupa date of September 30th.

Agency review of our application is progressing. Well,

Speaker #2: Turning now to the balance sheet. We were very pleased to have ended the second quarter of 2026 with $492 million in cash. Cash equivalents and marketable securities.

Importantly, we have significant optionality with 2 independent, paths to approval, either through Catalan, Indiana, or through our second fill finish facility, or both, whichever is determined to be the most rapid.

David Hallal: As it relates to Catalent Indiana, the FDA inspection classification following the April 2026 general site inspection is pending. We continue to be very pleased by the progress made at our second fill-finish facility. Importantly, the data package for the FDA's review of the second facility has been submitted and the review is progressing. Underscoring our operational excellence at our second site, we now have more vials available from this facility than we do from Catalent Indiana. Notably, vials from both Catalent Indiana and our second facility are now on site at our third-party provider, awaiting packaging and labeling upon approval. As we near the final step in the US regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease.

Speaker #2: This cash balance includes net proceeds of $63 million from our ATM program during the second quarter. Vikas will provide more detail later in the call.

As it relates to Catalan Indiana. The FDA inspection classification following the April 2026 General site. Inspection is pending.

Speaker #2: In June, we had the opportunity to be with the SMA Physician and Patient Community at the Cure SMA annual meeting in Orlando. I was able to sit down with several SMA treating physicians and with a number of patients and their families.

We continue to be very pleased by the progress made at our second fill finish facility.

Importantly, the data package for the fda's review of the second facility has been submitted, and the review is progressing.

Speaker #2: And during our time with them, we heard some very moving stories about the impact epidogram map has had on children and adults who are participating in our Onyx and EAP programs.

Underscoring, our operational excellence at our Second Sight.

We now have more vials available from this facility than we do from Catalan Indiana.

Speaker #2: Our team at by these patients and by their families and we look forward to ushering in the next phase of innovation for this community.

Notably vials from both Catalan, Indiana and our second facility are now on site.

At our third-party provider awaiting packaging and labeling upon approval.

Speaker #2: With that, I'll now turn the call over to Akshay for a closer look at our R&D initiatives. Akshay?

as we near the final step,

Speaker #3: Thank you, David, and good morning, everybody. As David shared, we're very pleased that the epidogram map BLA continues to progress through FDA review with two independent parts to approval, and we remain on track for decision by the September 30 PDUFA date.

First muscle, targeted therapy for this devastating disease.

David Hallal: Turning now to our commercial readiness in the US, our team continues to advance our launch preparations across all key functions, ensuring we are prepared to support patients, caregivers, and prescribers from day one. Our team is ready to launch apitegromab at any time prior to and including our 30 September PDUFA date. Keith will discuss our commercial preparations in greater detail shortly. In addition to the US, we continue to look forward to serving children and adults living with SMA in Europe. I would now like to provide an update on where we stand in the European regulatory process. The apitegromab MAA includes only Catalent Indiana fill-finish facility, and the EMA is awaiting the FDA inspection classification for that facility. In parallel, we are engaging with European regulators with regards to potential inclusion of our second fill-finish facility in the apitegromab application.

Speaker #3: The FDA inspection classification for Catalin Indiana is pending. We had anticipated the classification in late July, within 90 days following inspection completion based on the agency's guidelines.

Turning now to our commercial Readiness in the US, our team continues to advance our launch preparations across all key functions. Ensuring we are prepared to support patients, caregivers and prescribers from day 1.

Speaker #3: We remain engaged with the FDA and will provide updates as appropriate. As it relates to the second fill finish facility, we're very pleased to report that all necessary data have now been submitted to FDA and the agency's review of those data is progressing well.

Our team is ready to launch a pedigree map at any time prior to, and including our September 30th to do for date.

Keith will discuss our commercial preparations and greater details shortly.

In addition to the US, we continue to look forward to serving children and adults living with SMA in Europe.

Speaker #3: We're gratified by the agency's continued support since the CRL last September from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting.

I would now like to provide an update on where we stand in the European regulatory process.

Speaker #3: Throughout, the agency has appreciated the high met need in the SMA community and we look forward to the final steps in the process. Turning now to Europe, we're pleased with the EMA's review of the epidogram map marketing authorization application.

The epitome of M AAA includes only Catalan Indiana, fill finish facility and the EMA is awaiting, the FDA inspection classification for that facility.

Speaker #3: And with their continued level of engagement. As we have previously noted, approval in Europe is dependent on FDA clearance of the Catalin Indiana facility which is currently the sole fill finish site included in our MAA.

David Hallal: Importantly, this facility has had recent successful site inspections by the FDA and the EMA. We are grateful for the EMA's continued level of engagement, and we look forward to providing updated guidance on the potential timing of a CHMP opinion upon alignment with the European regulators. Turning to our launch preparations in Europe, we are executing our commercial playbook, building momentum with launch readiness activities and engaging with the SMA community. We are planning for an initial launch in Germany with additional countries and regions to follow as we build out our planned 50-country operating platform. We know it is not a matter of if but when apitegromab will be approved for children and adults with SMA in both the US and Europe, and we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN-targeted therapy.

In parallel, we are engaging with European Regulators with regards to potential inclusion of our second field finish facility in the epitome of application.

Importantly, this facility has had recent successful site inspections by the FDA and the EMA.

Speaker #3: The EMA continues to await the FDA's inspection classification for this facility. Additionally, we're engaging with the EMA regarding the process to include our second fill finish facility in our epidogram map application.

We are grateful for the EMA's continued level of Engagement and we look forward to providing updated guidance on the potential timing of a chmp opinion upon alignment with the European regulators.

Speaker #3: Importantly, this facility is in good standing with European regulators. We will provide an update on timing upon alignment with the EMA. Turning to our pipeline, let me start with the Phase 2 OPAL trial.

Turning to our launch preparations in Europe. We are executing our commercial Playbook.

Building momentum with launch Readiness activities and engaging with the SMA community.

Speaker #3: We continue to have robust enrollment in the study, which is evaluating epidogram map in infants and toddlers with SMA under the age of 2.

We are planning for an initial launch in Germany with additional countries and regions to follow. As we build out, our planned 50 country operating platform.

Speaker #3: As a reminder, this trial is enrolling participants who've been treated with an SMN-1-targeted gene therapy or who are receiving ongoing treatment with an SMN-2-targeted treatment.

We know it is not a matter of if, but when a pedigree map, will be approved for children and adults with SMA in both the US and Europe.

Speaker #3: This study is important because it is anticipated to expand the impact of epidogram map to the full spectrum of patients, including those treated with Zolgensma.

And we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN targeted therapy.

David Hallal: Turning now to our world-leading anti-myostatin pipeline. We continue to make meaningful progress with our key clinical programs. We have robust enrollment in our phase II OPAL study evaluating apitegromab in infants and toddlers with SMA. We initiated our randomized phase II FORGE study in patients with FSHD. We are ready to engage with US and European regulators on the development path of our high-concentration subcutaneous formulation of apitegromab, which we will do once we have regulatory approvals, and enrollment and dosing is proceeding very well in our phase I healthy volunteer study for SRK-439, our novel high-potency anti-myostatin antibody. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet.

Speaker #3: Notably, the rate at which the study is enrolling underscores the significant unmet need and the potential for epidogram map in the youngest of SMA patients.

Turning now to our world-leading.

We continue to make meaningful progress with our key clinical programs.

Speaker #3: Turning now to our next indication for epidogram map, fascioscapular humeral muscular dystrophy or FSHD. FSHD is a rare devastating neuromuscular disease. It is one of the most prevalent inherited muscular dystrophies and there are no approved therapies to date.

We have robust enrollment in our Phase 2 opal. Study evaluating a pedigree lab in infants and toddlers with SMA.

We initiated our randomized. Phase 2 Forge, study in patients, with fshd

Speaker #3: We've prioritized FSHD as the next indication for epidogram map for three key reasons. First, the significant unmet need since approximately 20% of patients become wheelchair dependent.

Speaker #3: Second, the compelling preclinical data from the gold standard FlexDux 4 mouse model that provides mechanistic rationale for epidogram map in FSHD. And finally, data from randomized studies in FSHD which suggest muscle mass can increase and has the capacity to show functional benefit.

We are ready to engage with us and European Regulators on the development path of our high concentration. Subcutaneous formulation of a paragraph which we will do 1. We have regulatory approvals and enrollment and dosing is proceeding very well in our Phase 1, healthy volunteer study,

For srk 439 are novel. High potency. Antimi antibody.

Oxy will discuss these programs in Greater detail shortly.

David Hallal: We were very pleased to have ended the Q2 2026 with $492 million in cash equivalents, and marketable securities. This cash balance includes net proceeds of $63 million from our ATM program during the Q2. Vikas will provide more detail later in the call. In June, we had the opportunity to be with the SMA physician and patient community at the Cure SMA annual meeting in Orlando. I was able to sit down with several SMA treating physicians and with a number of patients and their families. During our time with them, we heard some very moving stories about the impact apitegromab has had on children and adults who are participating in our ONYX and EAP programs.

Speaker #3: For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function.

Turning now, to the balance sheet, we were very pleased to have ended the second quarter of 2026, with 492 million in cash, cash, equivalents and marketable securities.

Speaker #3: These data suggest that epidogram map as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased to announce today that we've initiated our Phase 2 study called FORGE, which is a randomized double-blind placebo-controlled trial with a sample size of 60 patients.

This cash balance includes net proceeds of 63 million from our ATM program. During the second quarter,

The cost will provide more detail later in the call.

In June, we have the opportunity to be with the SMA physician and patient community at the Cure SMA annual meeting in Orlando.

Speaker #3: We're also advancing two additional therapeutic programs in our world-leading anti-myosin pipeline, a high-concentration subcutaneous formulation of epidogram map, and SRK439. In our subcutaneous epidogram map program, we showed some very exciting data in January from a Phase 1 study which demonstrated that subcutaneous epidogram map appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration.

I was able to sit down with several SMA treating Physicians and with a number of patients and their families.

David Hallal: Our team at Scholar Rock is so inspired by these patients and by their families, we look forward to ushering in the next phase of innovation for this community. With that, I will now turn the call over to Akshay for a closer look at our R&D initiatives. Akshay?

And during our time with them, we heard some very moving stories about the impact pigs had on children and adults who are participating in our ONYX and EAP programs.

Speaker #3: Additional development activities are ongoing and we continue to plan for engagement with US and European regulators later this year following approval of epidogram map.

Our team scholar rock is so inspired by these patients and by their families and we look forward to ushering in the next phase of innovation for this community.

Akshay Vaishnaw: Thank you, David, good morning, everybody. As David shared, we are very pleased that the apitegromab BLA continues to progress through FDA review with two independent paths to approval, we remain on track for a decision by the 30 September PDUFA date. The FDA inspection classification for Catalent Indiana is pending. We had anticipated the classification in late July, within 90 days following inspection completion based on the agency's guidelines. We remain engaged with the FDA and will provide updates as appropriate. As it relates to the second fill-finish facility, we are very pleased to report that all necessary data have now been submitted to FDA, the agency's review of those data is progressing well. We are gratified by the agency's continued support since the CRL last September from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting.

Speaker #3: Turning now to SRK439, our high-potency, high-affinity subcutaneously administered myostatic inhibitor. We're very excited about this program and dosing in our Phase 1 healthy volunteer study is progressing well.

Speaker #3: We expect to have top-line data from the study later this year. In closing, we're executing with urgency to bring epidogram map to children and adults with SMA whilst in parallel working to maximize our efforts maximize our impact for patients with our world-leading anti-myosin pipeline across a range of rare devastating neuromuscular diseases.

With that. I'll now turn the call over to a for a closer look at our R&D initiatives Ace. Thank you, David and good morning everybody. As David shared, we're very pleased that the epitome of De continues to progress through FTA FDA review. With 2 independent parts of approval and we remain on track for decision by the September 30th Paducah day

July within 90 days, following inspection completion, based on the agency's guidelines.

The remain engaged with the FDA and will provide updates as appropriate.

Speaker #3: I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Speaker #2: Thanks, Akshay. And good morning, everyone. As David noted, with the potential FDA approval of epidogram map for children and adults with SMA by September 30th, our US commercial organization is launch-ready across all key functions and we are prepared to support patients, caregivers, and prescribers from day one.

As it relates to the second fill finish facility, we're very pleased to report that all necessary data have now been submitted to FDA and the agencies review of those data is progressing. Well,

Akshay Vaishnaw: Throughout, the agency has appreciated the high met need in the SMA community, we look forward to the final steps in the process. Turning now to Europe. We are pleased with the EMA's review of the apitegromab marketing authorization application and with their continued level of engagement. As we have previously noted, approval in Europe is dependent on FDA clearance of the Catalent Indiana facility, which is currently the sole fill-finish site included in our MAA. The EMA continues to await the FDA's inspection classification for this facility. Additionally, we are engaging with the EMA regarding the process to include our second fill-finish facility in our apitegromab application. Importantly, this facility is in good standing with European regulators. We will provide an update on timing upon alignment with the EMA. Turning to our pipeline, let me start with the phase II OPAL trial.

We're gratified by the agency's continued support since the CRL last September, from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting.

Speaker #2: Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations to ensure that patients who can benefit from epidogram map will have broad and reliable access to epidogram map.

Throughout the agency is appreciated, the high Met need in the SMA community and we look forward to the final steps in the process.

Turning now to Europe we're pleased with the EMA's review of the epitome of marketing authorization application and with their continued level of Engagement.

Speaker #2: In the US, despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy.

As we have previously, noted approval in Europe is dependent on FDA clearance of the Catalan Indiana facility, which is currently the sole fill finish site included in our mmaa.

The EMA continues to await the fda's inspection classification for this facility.

Speaker #2: That limits both function and independence. As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated one-third of people living with SMA in the US have received two or more SMN-targeted treatments, either sequentially or in combination.

Additionally, we're engaging with the EMA regarding the process to include our second, fill finish facility in our pedigree App application.

Importantly, this facility is in good standing with European regulators.

We will provide an update on timing upon alignment with the EMA.

Akshay Vaishnaw: We continue to have robust enrollment in the study, which is evaluating apitegromab in infants and toddlers with SMA under the age of two. As a reminder, this trial is enrolling participants who have been treated with an SMN1-targeted gene therapy or who are receiving ongoing treatment with an SMN2-targeted treatment. This study is important because it is anticipated to expand the impact of apitegromab to the full spectrum of patients, including those treated with ZOLGENSMA. Notably, the rate at which the study is enrolling underscores the significant unmet need and the potential for apitegromab in the youngest of SMA patients. Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy or FSHD. FSHD is a rare, devastating neuromuscular disease. It is one of the most prevalent inherited muscular dystrophies, there are no approved therapies to date.

Turning to our pipeline, let me start with the Phase 2 OPAL trial.

Speaker #2: This data again underscores the significant opportunity we have with epidogram map, the world's first muscle-targeted therapy for children and adults with SMA. Since our last earnings call, our US field team continues to broaden their reach focusing on disease education and awareness around the unmet medical need while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle.

We continue to have robust enrollment in the study, which is evaluating apitegromab in infants and toddlers with SMA under the age of 2.

As a reminder, this trial is enrolling. Participants who've been treated with an smn1 targeted, gene therapy or who are receiving ongoing treatment within smn2 targeted treatment.

Speaker #2: The principal organ impacted by the disease. We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams.

This study is important because it is anticipated to expand the impact of a pyramid. To the full spectrum of patients, including those treatments with organisms.

Notably the rate at which the studies enrolling underscores, the significant unmet need, and the potential for a pedigree map in the youngest of our 7 patients.

Speaker #2: Through these engagements, our field team has established case flows on a center-by-center basis to ensure that upon approval, we are well-positioned to support the SMA treatment centers once epidogram map treatment decision has been made.

Setting. Now, to our next indication, for a pedigree map faccio scapular human muscular droopy or fshd

Akshay Vaishnaw: We've prioritized FSHD as the next indication for apitegromab for three key reasons. First, the significant unmet need, since approximately 20% of patients become wheelchair-dependent. Second, the compelling preclinical data from the gold standard FLExD/Oxy mouse model that provides mechanistic rationale for apitegromab in FSHD. Finally, data from randomized studies in FSHD which suggest muscle mass can increase and has the capacity to show functional benefit. For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function. These data suggest that apitegromab as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased to announce today that we've initiated our phase II study called FORGE, which is a randomized, double-blind, placebo-controlled trial with a sample size of 60 patients.

Fshd the rare devastating neuromuscular disease. It is 1 of the most prevalent inherited muscular drophy and there are no approved therapies today.

Speaker #2: This past quarter, we have also strengthened our Scholar Rock supports patient services program. The Scholar Rock supports team is fully trained and prepared to provide comprehensive individualized support to patients and caregivers at launch.

Speaker #2: Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and co-pay assistance, and navigate treatment logistics.

We've prioritized, fshd is the next indication for a pit of a map for 3 key reasons, first the significant unmet, need since approximately 20% of patients become wheelchair dependent second the compelling preclinical data from the gold standard Flex do for Mouse model. That provides mechanistic rationale for a period of MSA HD. And finally data from randomized studies in fshd which suggests muscle mass can increase and has the capacity to show functional benefit.

Speaker #2: Turning now to patient engagement, our connections with the SMA community remain strong. This past June, we had a significant presence at the Cure SMA annual meeting in Orlando.

For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function.

Speaker #2: Scholar Rock served as a presenting sponsor of the meeting and throughout the week, our teams engaged with healthcare professionals and members of the SMA patient community.

These data suggests that a pilgrim app, as a monotherapy may have the potential to bring important benefits to fshd patients.

Speaker #2: I was very pleased that the Scholar Rock symposium for healthcare professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy was one of the most attended expert sessions during the meeting.

Akshay Vaishnaw: We're also advancing two additional therapeutic programs in our world-leading anti-myostatin pipeline: a high-concentration subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data in January from a phase I study, which demonstrated that subcutaneous apitegromab appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration. We continue to plan for engagement with US and European regulators later this year following approval of apitegromab. Turning now to SRK-439, our high-potency, high-affinity, subcutaneously administered myostatin inhibitor. We're very excited about this program, and dosing in our phase I healthy volunteer study is progressing well. We expect to have top-line data from the study later this year.

We're very pleased to announce today that we've initiated our Phase 2. Study called Forge, which is a randomized, double blind, Placebo control trial with the sample size of 60 patients,

Speaker #2: Equally, our patient symposium muscle there is more to the story in SMA was attended by hundreds of SMA patients, caregivers, and families and during this session, we sought their perspective on needs and priorities for people living with SMA.

We're also advancing 2 additional therapeutic programs in our world-leading antimi, stand pipeline, a high concentration, subcutaneous formulation of the pyramid map and srk 439.

Speaker #2: Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families. Turning to US reimbursement, our market access team continues to advance discussions with national and key regional payers as well as Medicare and Medicaid.

In our subcutaneous epidural program, we showed some very exciting data in January, from a phase 1 Study, which demonstrated the subcutaneous epitome map appears to have favorable by availability and the pharmacodynamic profile comparable to IV Administration.

Additional development activities are ongoing and we continue to plan for engagement with us and European Regulators later this year, following approval of a pilgrim map.

Speaker #2: With the goal of achieving broad reimbursement for eligible patients after approval. Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well-positioned to successfully support epidogram map in the US immediately upon FDA approval.

Turning now to SRK-429, a high-potency, high-affinity, subcutaneously administered myostatin inhibitor.

We're very excited about this program, and dosing in our Phase 1 healthy volunteer studies is progressing well.

Akshay Vaishnaw: In closing, we're executing with urgency to bring apitegromab to children and adults with SMA, whilst in parallel working to maximize our efforts maximize our impact for patients with our world-leading anti-myostatin pipeline across a range of rare, devastating neuromuscular diseases. I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

We expect to have Topline data from the study later this year.

Speaker #2: Turning now to Europe, we are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application.

Speaker #2: Our team in Germany is using this additional time to execute the same launch readiness playbook that we have successfully deployed in the US over the last several months.

R. Keith Woods: Thanks, Akshay. Good morning, everyone. As David noted, with the potential FDA approval of apitegromab for children and adults with SMA by 30 September, our US commercial organization is launch-ready across all key functions. We are prepared to support patients, caregivers, and prescribers from day one. Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations to ensure that patients who can benefit from apitegromab will have broad and reliable access to apitegromab. In the US, despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy that limits both function and independence.

Keith Woods: Thanks, Akshay. Good morning, everyone. As David noted, with the potential FDA approval of apitegromab for children and adults with SMA by 30 September, our US commercial organization is launch-ready across all key functions. We are prepared to support patients, caregivers, and prescribers from day one. Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations to ensure that patients who can benefit from apitegromab will have broad and reliable access to apitegromab. In the US, despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy that limits both function and independence.

In closing, we're executing with, urgency to bring a p EP to children, and adults with SMA whilst in parallel, working to maximize, our efforts that maximize our impact for patients, with our world-leading anti-mine across a range of rare, devastating neuromuscular diseases. I'll now turn the call over to keys to discuss our commercial launch preparations. Keith

Speaker #2: This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers. In parallel, we are engaging with SMA advocates across Europe participating in educational programs at various congresses and symposia hosted by patient advocacy organizations.

Not say and good morning everyone as David noted with the potential FDA approval of a pedigree app for children and adults with SMA by September 30th. R Us commercial organization is launched ready across all key functions and we are prepared to support patients caregivers and prescribers from day 1.

Speaker #2: As it relates to reimbursement and patient access, following European Commission approval of epidogram map, we will be prepared to rapidly advance reimbursement submissions in Germany and other key markets.

Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations, to ensure that patients who can benefit from apitegromab will have broad and reliable access to apitegromab.

Speaker #2: In addition, we are advancing our distributor relationships to extend the commercial reach of epidogram map across multiple additional countries. In closing, we are fully prepared for a successful US launch immediately upon FDA approval.

R. Keith Woods: As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated one-third of people living with SMA in the US have received two or more SMN-targeted treatments, either sequentially or in combination. This data again underscores the significant opportunity we have with apitegromab, the world's first muscle-targeted therapy for children and adults with SMA. Since our last earnings call, our US field team continues to broaden their reach, focusing on disease education and awareness around the unmet medical need, while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle, the principal organ impacted by the disease. We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams.

Keith Woods: As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated one-third of people living with SMA in the US have received two or more SMN-targeted treatments, either sequentially or in combination. This data again underscores the significant opportunity we have with apitegromab, the world's first muscle-targeted therapy for children and adults with SMA. Since our last earnings call, our US field team continues to broaden their reach, focusing on disease education and awareness around the unmet medical need, while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle, the principal organ impacted by the disease. We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams.

Speaker #2: While advancing our launch preparations in Europe, and working to establish our 50-country operating platform with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy.

In the US, despite approximately 78% of children and adults, living with SMA, receiving an SMN, targeted therapy, 95% of patients. Continue to experience persistent and progressive muscle, atrophy that limits both function and Independence.

Speaker #2: We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver, and one family at a time.

As further evidence of the unmet medical need data shared with us by cure SMA, show that an estimated 1/3 of people living with SMA in the US have received 2 or more. SMN targeted treatments, either sequentially or in combination.

Speaker #2: With that, I'll turn the call over to Vikass for a review of our financial performance.

This data again, underscores the significant opportunity. We have with a pedigree map, the world's first muscle targeted therapy for children and adults with SMA.

Speaker #3: Vikass?

Speaker #4: Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial bill to deliver a strong epidogram map launch funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space.

Since our last earnings call our us field team continues to broaden their reach focusing on disease, education and awareness around the unmet. Medical need while also reinforcing a broader, understanding of SMA as a disease that consists of both the motor neuron, and the muscle, the principal organ impacted by the disease.

Speaker #4: And continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. Consistent with these priorities, I'd like to briefly review our second quarter financial results.

R. Keith Woods: Through these engagements, our field team is establishing case flows on a center-by-center basis to ensure that upon approval, we are well-positioned to support the SMA treatment centers once apitegromab treatment decision has been made. This past quarter, we have also strengthened our Scholar Rock Supports patient services program. The Scholar Rock Supports team is fully trained and prepared to provide comprehensive, individualized support to patients and caregivers at launch. Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and copay assistance, and navigate treatment logistics. Turning now to patient engagement. Our connections with the SMA community remain strong. This past June, we had a significant presence at the Cure SMA annual meeting in Orlando.

Keith Woods: Through these engagements, our field team is establishing case flows on a center-by-center basis to ensure that upon approval, we are well-positioned to support the SMA treatment centers once apitegromab treatment decision has been made. This past quarter, we have also strengthened our Scholar Rock Supports patient services program. The Scholar Rock Supports team is fully trained and prepared to provide comprehensive, individualized support to patients and caregivers at launch. Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and copay assistance, and navigate treatment logistics. Turning now to patient engagement. Our connections with the SMA community remain strong. This past June, we had a significant presence at the Cure SMA annual meeting in Orlando.

We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams.

Speaker #4: For the second quarter, we reported 108.9 million in operating expenses, which included 19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were 89.2 million.

Through these engagements. Our field team is establishing case flows On A Center by Center basis to ensure that upon approval. We are well, positioned to support the SMA treatment centers once a pilgrim, AB treatment decision has been made

Speaker #4: As we continue preparing for the anticipated launch of epidogram map, we have strategically increased our commercial investments while keeping our overall operating expenses at the levels generally consistent with the second quarter of 2025.

This past quarter, we have also strengthened our scholar Rock supports patient services program. The Scholar Rock support team is fully trained and prepared to provide comprehensive individualized support to patients and caregivers at launch.

Speaker #4: This disciplined approach to capital allocation has enabled us to advance launch readiness while continuing to invest in our key R&D programs and strengthening our global supply chain.

Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and co-pay assistance, and navigate treatment logistics.

Turning now to patient engagement, our connections with the SMA community remain strong.

R. Keith Woods: Scholar Rock served as a presenting sponsor of the meeting. Throughout the week, our teams engaged with healthcare professionals and members of the SMA patient community. I was very pleased that the Scholar Rock Symposium for Healthcare Professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy, was one of the most attended expert sessions during the meeting. Equally, our patient symposium, Muscle: There's More to the Story in SMA, was attended by hundreds of SMA patients, caregivers, and families. During this session, we sought their perspective on needs and priorities for people living with SMA. Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families. Turning to US reimbursement.

Keith Woods: Scholar Rock served as a presenting sponsor of the meeting. Throughout the week, our teams engaged with healthcare professionals and members of the SMA patient community. I was very pleased that the Scholar Rock Symposium for Healthcare Professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy, was one of the most attended expert sessions during the meeting. Equally, our patient symposium, Muscle: There's More to the Story in SMA, was attended by hundreds of SMA patients, caregivers, and families. During this session, we sought their perspective on needs and priorities for people living with SMA. Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families. Turning to US reimbursement.

This past June, we had a significant Presence at the Cure SMA annual meeting in Orlando.

Speaker #4: Turning to our balance sheet, we ended the second quarter with 492 million in cash, cash equivalents and marketable securities. During the quarter, we further strengthened our cash position with 63 million in net proceeds from our ATM program.

Speaker #4: Looking ahead, our FDA approval of epidogram map, we will have an option to draw down an additional 150 million from our existing debt facility and we plan to monetize a priority review voucher to further strengthen our balance sheet.

Scholar Rock served as a presenting sponsor of the meeting and throughout the week. Our teams engaged with Health Care Professionals and members of the SMA patient Community. I was very pleased that the scholar Rock Symposium for healthcare professionals. Entitled expert perspective on the evolving management of spinal muscular atrophy was 1 of the most attended expert sessions during the meeting.

Speaker #4: We continue to operate with a disciplined financial plan and our investment priorities remain focused on our epidogram map commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and anticipated global commercial demand for epidogram map over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call.

Equally our patient, Symposium muscle, there is more to the story in SMA was attended by hundreds of SMA, patients, caregivers and families. And during this session, we sought their perspective on needs and priorities for people living with SMA.

Every interaction we had during this meeting, reinforces our determination and further strengthens our commitment to serve patients and Families.

R. Keith Woods: Our market access team continues to advance discussions with national and key regional payers, as well as Medicare and Medicaid, with the goal of achieving broad reimbursement for eligible patients after approval. Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well-positioned to successfully support apitegromab in the US immediately upon FDA approval. Turning now to Europe. We are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application. Our team in Germany is using this additional time to execute the same launch readiness playbook that we have successfully deployed in the US over the last several months. This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers.

Keith Woods: Our market access team continues to advance discussions with national and key regional payers, as well as Medicare and Medicaid, with the goal of achieving broad reimbursement for eligible patients after approval. Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well-positioned to successfully support apitegromab in the US immediately upon FDA approval. Turning now to Europe. We are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application. Our team in Germany is using this additional time to execute the same launch readiness playbook that we have successfully deployed in the US over the last several months. This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers.

Speaker #4: With that, I'll turn the call back to David. David?

Turning to US reimbursement, our market access team continues to advance discussions with national and key regional payers, as well as Medicare and Medicaid, with the goal of achieving broad reimbursement for eligible patients after approval.

Speaker #1: Thanks, Vikass. As we look ahead, Scholar Rock is entering one of the most important chapters in our history from a position of strength. With 55 days until our PDUPA date, we have the team, the financial foundation, and the operational readiness to execute with confidence across the organization, our teams are prepared energized and focused on what comes next, bringing forward the world's first muscle-targeted therapy for children and adults with SMA.

Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well positioned to successfully support a pedigree MAB in the US immediately upon FDA approval.

Turning now to Europe, we are advancing our launch preparations, with a particular focus on Germany, as we work with the EMA on the next steps for our application.

Our team in Germany is using this additional time to execute the same launch Readiness Playbook that we have successfully deployed in the US over the last several months.

Speaker #1: We are moving into these final 55 days with urgency, discipline, and a deep sense of responsibility to the SMA community. We know what is at stake, we know what is possible, and we are ready to deliver.

R. Keith Woods: In parallel, we are engaging with SMA advocates across Europe, participating in educational programs at various congresses and symposia hosted by patient advocacy organizations. As it relates to reimbursement and patient access, following European Commission approval of apitegromab, we will be prepared to rapidly advance reimbursement submissions in Germany and other key markets. In addition, we are advancing our distributor relationships to extend the commercial reach of apitegromab across multiple additional countries. In closing, we are fully prepared for a successful US launch immediately upon FDA approval while advancing our launch preparations in Europe and working to establish our 50-country operating platform with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver, and one family at a time.

Keith Woods: In parallel, we are engaging with SMA advocates across Europe, participating in educational programs at various congresses and symposia hosted by patient advocacy organizations. As it relates to reimbursement and patient access, following European Commission approval of apitegromab, we will be prepared to rapidly advance reimbursement submissions in Germany and other key markets. In addition, we are advancing our distributor relationships to extend the commercial reach of apitegromab across multiple additional countries. In closing, we are fully prepared for a successful US launch immediately upon FDA approval while advancing our launch preparations in Europe and working to establish our 50-country operating platform with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver, and one family at a time.

This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers.

Speaker #1: As we close, we are mindful that August is SMA awareness month. This is an important opportunity to recognize the strength and resilience of the individuals living with SMA their families, and the advocacy organizations that work tirelessly on their behalf.

Programs at various congresses and symposia hosted by patient. Advocacy organizations.

As it relates to reimbursement and patient access following European commission approval of a pedigree. AB, we will be prepared to rapidly Advance. Reimbursement submissions in Germany and other key markets.

Speaker #1: We are grateful to the patients, caregivers, healthcare professionals, and advocates whose partnership continues to advance awareness, earlier diagnosis, and access to care. This month reinforces our commitment to the SMA community and our focus on delivering meaningful innovation that can make a lasting difference for people living with this rare and devastating disease.

In addition, we are advancing our distributor relationships to extend the commercial reach of a pedigree mab across multiple additional countries.

Speaker #1: We look forward to updating you on our continued progress and with that, we'll now open the line for questions. Operator?

Speaker #5: Thank you. As a reminder, if you would like to ask a question, please press star 11 on your telephone. You'll hear that automated message advising your hand is raised.

R. Keith Woods: With that, I'll turn the call over to Vikas for a review of our financial performance. Vikas?

Keith Woods: With that, I'll turn the call over to Vikas for a review of our financial performance. Vikas?

In closing, we are fully prepared for a successful U.S. launch immediately upon FDA approval, while advancing our launch preparations in Europe and working to establish our 50-country operating platform, with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA—one patient, one caregiver, and one family at a time.

Vikas Sinha: Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial build to deliver a strong apitegromab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space, and continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. Consistent with these priorities, I'd like to briefly review our Q2 financial results. For the Q2, we reported $108.9 million in operating expenses, which included $19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $89.2 million. As we continue preparing for the anticipated launch of apitegromab, we have strategically increased our commercial investments while keeping our overall operating expenses at the levels generally consistent with the Q2 2025.

Speaker #5: We also ask that you limit yourself to one question. One moment while we compile the Q&A roster and please wait for your name and company to be announced before proceeding with your question.

With that, I'll turn the call over to Vos for a review of our financial performance.

Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on.

Speaker #5: The first question will be coming from the line of Eric Schmidt of Cantor. Please go ahead.

Supporting our commercial build to deliver a strong epidemic launch.

Speaker #6: Well, thanks, team. Appreciate all the updates. My questions on epidogram maps second fill finish provider. How confident are you that this facility alone, independent of Catalent, can support approval by the PDUPA and can you share any kind of anecdotes from your FDA interactions to support that they're making progress in the review of the package you submitted?

Funding R&D activities to advance our Pipeline and expand our leadership in the myostatin and muscle space.

And continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value.

Consistent with these priorities. I'd like to briefly review a second quarter Financial results,

Speaker #6: Thank you.

Speaker #1: Thanks very much, Eric. Yeah, as we noted, really dating back to that March 3rd type C meeting, we were really gratified as we were updating the FDA on the rapid and meaningful progress we were making in our second facility that really together we agreed that resubmitting our BLA with two fill finish facilities and actually having a plan to enable an epidogram map approval with the fastest path, whether or not it be Catalent Indiana the second fill finish facility or both, we were gratified that there was a complete alignment between us and the agency that that was the best approach.

For the second quarter, we reported 108.9 million in operating expenses which included 19.7 million in non-cash stock based compensation.

Excluding stock-based compensation, operating expenses were $89.2 million.

as we continue preparing for the anticipated launch of appet map, we have strategically increased, our commercial Investments, while keeping our overall operating expenses at the levels, generally consistent with the second quarter of 2025,

Vikas Sinha: This disciplined approach to capital allocation has enabled us to advance launch readiness while continuing to invest in our key R&D programs and strengthening our global supply chain. Turning to our balance sheet, we ended the Q2 with $492 million in cash equivalents, and marketable securities. During the quarter, we further strengthened our cash position with $63 million in net proceeds from our ATM program. Looking ahead, our FDA approval of apitegromab, we will have an option to draw down an additional $150 million from our existing debt facility, and we plan to monetize a priority review voucher to further strengthen our balance sheet.

Speaker #1: As we've noted on the call, the team and the second the team here at Scholar Rock and the second facility were really proud of their efforts to now have more vials of epidogram map ready facility than we do from Catalent Indiana and frankly than we did from Catalent Indiana.

This disciplined approach to Capital. Allocation has enabled us to advance launch Readiness while continuing to invest in our key R&D programs and strengthening our Global Supply Chain.

Turning to our balance sheet, we entered the second quarter with 492 million in cash, cash, equivalents and marketable securities.

Speaker #1: At the time of our last PDUPA date, I think underscores how well we are working together. The review, I think, as both Akshay and I noted, is progressing well.

during the quarter, we further strengthen our cash position with 63 million in net proceeds, from our ATM program,

Speaker #1: The FDA has all of the data that we agreed upon during our type C meeting. That was going to enable the review and eventual approval from that second facility.

Looking ahead, our FDA approval of epigram app. We will have an option to draw down an additional 150 million from our existing debt facility and we plan to monetize a priority review voucher to further strengthen our balance sheet.

Vikas Sinha: We continue to operate with a disciplined financial plan, our investment priorities remain focused on our apitegromab commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and anticipated global commercial demand for apitegromab over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I'll turn the call back to David. David?

Speaker #1: So we're pleased with that and as we're noting, we're corresponding with the FDA on that. I would also note a couple of other facts that I think are important here.

We continue to operate with a disciplined financial plan and our investment priorities remain focused on.

Our epidural map commercial launch Readiness in the US and Europe.

Speaker #1: That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI and I think underscoring the performance of that facility during that time, the site underwent multiple routine GMP general site inspections by EMA and FDA.

Strengthening our supply chain to support our expanding Pipeline and anticipated. Global commercial demand for oppido map over time and advancing our highly Innovative clinical programs that axi discussed earlier in the call.

David Hallal: Thanks, Vikas. As we look ahead, Scholar Rock is entering one of the most important chapters in our history from a position of strength. With 55 days until our PDUFA date, we have the team, the financial foundation, and the operational readiness to execute with confidence. Across the organization, our teams are prepared, energized, and focused on what comes next, bringing forward the world's first muscle-targeted therapy for children and adults with SMA. We are moving into these final 55 days with urgency, discipline, and a deep sense of responsibility to the SMA community. We know what is at stake, we know what is possible, and we are ready to deliver. As we close, we are mindful that August is SMA Awareness Month.

With that, I'll turn the call back to David. David?

As we look ahead, Scholar Rock is entering one of the most important chapters in our history from a position of strength.

With 55 days until our Padua date.

We have the team.

Speaker #1: So we feel really good about the position that we are in. And then I might close by saying we contemplated the, well, what if we remove one of the facilities from the application?

The financial foundation and the operational Readiness to execute with confidence across the organization. Our teams are prepared energized and focused on what comes next.

Speaker #1: Could it impact our timeline at all? And that was contemplated and discussed between us and the agency and based upon our discussions with the agency, dating back to those March discussions, we do not expect that removing a facility from the application would have any impact on our ongoing review timeline at all.

Bringing forward the world’s first muscle-targeted therapy for children and adults with SMA.

We are moving into these final 55 days with urgency, discipline, and a deep sense of responsibility to the SMA community.

We know what is at stake.

We know what is possible, and we are ready to deliver.

Speaker #1: So Keith and team are ready to launch at any time between now and up to our September 30th PDUPA date I'm certainly gratified by Scholar Rock's technical operations and quality team they have stood up this second fill finish facility faster than almost any example we can find in the industry and to now have more epidogram map vials that will be commercially available from this second facility and they are at our labeling and packaging site awaiting approval.

David Hallal: This is an important opportunity to recognize the strength and resilience of the individuals living with SMA, their families, and the advocacy organizations that work tirelessly on their behalf. We are grateful to the patients, caregivers, healthcare professionals, and advocates whose partnership continues to advance awareness, earlier diagnosis, and access to care. This month reinforces our commitment to the SMA community and our focus on delivering meaningful innovation that can make a lasting difference for people living with this rare and devastating disease. We look forward to updating you on our continued progress. With that, we'll now open the line for questions. Operator?

As we close, we are mindful that August is SMA Awareness Month.

This is an important opportunity to recognize the strength and resilience of the individuals living with SMA.

And the advocacy organizations that work tirelessly on their behalf.

We are grateful to the patients, caregivers Health Care Professionals and Advocates, whose partnership continues to advance awareness earlier diagnosis and access to care.

Speaker #1: We're super excited. So we'll continue to keep you all updated over these last 55 days, but we feel like we're in a really good position.

This month, reinforces our commitment to the SMA community, and our focus on delivering meaningful Innovation that can make a lasting difference for people living with this rare and devastating disease.

Speaker #1: Thank you.

Speaker #6: Thank you.

Speaker #5: One moment for the next question. And the next question is coming from the line of Sripta Devarakonda. Of Truist Securities, please go ahead.

We look forward to updating you on our continued progress, and with that, we'll now open the line for questions. Operator?

Operator: Thank you. As a reminder, if you would like to ask a question, please press * one one on your telephone. You'll hear the automated message advising your hand is raised. We also ask that you limit yourself to one question. One moment while we compile the Q&A roster, please wait for your name and company to be announced before proceeding with your question. The first question will be coming from the line of Eric Schmidt of Cantor. Please go ahead.

Speaker #7: Hey guys, thank you so much for taking my question and congratulations on all the progress. I have a follow-up question on Eric's question regarding the second site.

Thank you as a reminder, if you would like to ask a question. Please press star, 1 1 1 on your telephone, you'll hear that automated message, advising your hand is raised. We also ask that you limit yourself to 1 question.

1 moment while we compile the Q&A roster and please wait for your name and Company to be announced before proceeding with your question.

Speaker #7: If the David, you just mentioned that you can drop one of the sites at any time and it won't delay, but I was wondering if Catalent remains classified as OAI, is there a deadline or a date for administratively withdrawing that site?

Eric Schmidt: Well, thanks, team. Appreciate all the updates. My question's on apitegromab's second fill finish provider. How confident are you that this facility alone, independent of Catalent, can support approval by the PDUFA? Can you share any kind of anecdotes from your FDA interactions to support that they're making progress in the review of the package you submitted? Thank you.

The first question will be coming from the line of Eric Smith of caner. Please go ahead.

Speaker #7: So it doesn't trigger any delay for your PDUPA? Thank you.

Oh, thanks team. Uh, appreciate all the updates, my questions on that particular, meds second, still finish provider. How confident are you that this facility, um, alone independent of Catalan can can support approval by the Padua? And can you share any kind of anecdotes from your FDA interactions to support that they're making progress in the review of the package you submitted? Thank you.

R. Keith Woods: Thanks very much, Eric. As we noted, really dating back to that March third Type C meeting, we were really gratified as we were updating the FDA on the rapid and meaningful progress we were making at our second facility that really together, we agreed that resubmitting our BLA with two fill finish facilities and actually having a plan to enable an apitegromab approval, with the fastest path, whether or not at the Catalent Indiana, the second fill finish facility or both. We were gratified that there was a complete alignment between us and the agency that that was the best approach.

Keith Woods: Thanks very much, Eric. As we noted, really dating back to that March third Type C meeting, we were really gratified as we were updating the FDA on the rapid and meaningful progress we were making at our second facility that really together, we agreed that resubmitting our BLA with two fill finish facilities and actually having a plan to enable an apitegromab approval, with the fastest path, whether or not at the Catalent Indiana, the second fill finish facility or both. We were gratified that there was a complete alignment between us and the agency that that was the best approach.

Speaker #1: It's a great question, Sripta. We are in correspondence with the agency. They are we're both well aware that the inspection classification is still pending from the April reinspection.

Uh, thanks very much Eric. Yeah, as as we noted, um, you know, really dating back to that.

Speaker #1: As Akshay noted, it's now drifted a bit beyond the 90-day guidance period, but it is only a guidance period from the FDA that they would classify an inspection.

March 3rd type c meeting. We were really gratified as we were updating the FDA on the rapid and meaningful progress. We were making in our second facility that uh, really together. We agreed, that resubmitting our bla with

Speaker #1: And look, we're very direct with one another about when we would reach that step. Should we need to reach that step? In your example that you provided, let's just say the inspection classification reveals that there's no change to the current OAI classification.

2, fill finish facilities and um actually having a plan to enable in a pedag approval uh with the fastest path whether or not it be.

David Hallal: As we've noted on the call, the team here at Scholar Rock and the second facility, we're really proud of their efforts to now have more vials of apitegromab ready for launch from this facility than we do from Catalent Indiana, and frankly, than we did from Catalent Indiana, at the time of our last PDUFA date, I think underscores how well we are working together. The review, I think, as both Akshay and I noted, is progressing well. The FDA has all of the data that we agreed upon during our Type C meeting that was going to enable the review and eventual approval from that second facility. We're pleased with that, and as we're noting, we're corresponding with the FDA on that.

Catalin Indiana. The second fil finish facility or both. We were we were gratified. Uh, that there was a

Speaker #1: We've discussed with the agency it would be a relatively simple step to notify them that on their signal that we are removing that site and the review would progress.

Complete alignment between us and the agency that, that was the the best approach. Um, as we've noted on the call that the team and the second, the team here at scholar Rock and the second facility, we're really proud of, you know, their efforts to now have

More vials of a pedigree map.

Speaker #1: With that second facility. So and again, with our alignment with the FDA, we would expect no impact to the ongoing timeline. So that's where we are.

Speaker #1: I think Akshay and I are both heartened by the fact that we have ongoing open correspondence with the FDA and the review is progressing well.

Uh, ready for launch, uh, from this facility. Then we do from Catalan. Indiana, and frankly, then we did from Catalan Indiana at the time of our, uh, last Padua date. Uh, I think underscores how well, we are working together.

Um, the review, I think, as both AI and I noted, is progressing well.

Speaker #7: Great. Thank you so much.

Speaker #5: Thank you. One moment for the next question. Our next question is coming from the line of Tessa Romero of JP Morgan. Please go ahead.

Speaker #8: Hi, David and team. Hope you are all well and thanks so much for taking our question. So just to double-click here on some of these earlier comments, what are the specific items procedurally from now to September 30th that still need to be ticked off to allow words final steps.

Uh, the FDA has all of the data that we agreed upon during our Type C meeting, uh, that was, um, going to enable the review and eventual approval, uh, from that second facility. So we're pleased with that. And

David Hallal: I would also note a couple of other facts that I think are important here. That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI. I think underscoring the performance of that facility during that time, the site underwent multiple routine GMP general site inspections by EMA and FDA. We feel really good about the position that we are in. I might close by saying, we contemplated the, well, what if we remove one of the facilities from the application? Could it impact our timeline at all?

As we're noting we're, uh, you know, we're, um, corresponding with the FDA, you know, on that.

Speaker #8: And just to set the record straight, is there any reason to believe that the FDA will not be able to complete this review by September 30th?

um I would also note a couple of other facts that I think are important here uh that second facility has had successful recent site inspections by FDA and EMA

uh none of the approvals from this facility in the last 12 months.

Speaker #8: Thank you.

Have required a pi or a pli.

Speaker #1: Yeah, I'll just hand it over to Akshay, but just underscore that last question. There really is no reason to believe with the work that's been done at the second facility and where we believe the FDA is in terms of reviewing the application, which as you recall, right, our resubmitted BLA really only included the new information from the second facility and the updated safety database.

And I think, underscoring the performance of that facility, during that time the site underwent multiple...

Uh, routine.

Speaker #1: There's no reason to believe the FDA can't get their work done in these next 55 days. Akshay?

David Hallal: That was contemplated and discussed between us and the agency. Based upon our discussions with the agency dating back to those March discussions, we do not expect that removing a facility from the application would have any impact on our ongoing review timeline at all. Keith and team are ready to launch at any time between now and up to our 30 September PDUFA date. I'm certainly gratified by Scholar Rock's technical operations and quality team. They have stood up this second fill finish facility faster than almost any example we can find in the industry. To now have more apitegromab vials that will be commercially available from this second facility, they are at our labeling and packaging site awaiting approval. We're super excited.

Speaker #6: Yeah, I would just reconfirm that Tess. So starting last November when we had a face-to-face meeting with the FDA with all relevant parties, and in March, the FDA guided that Catalent is the lead site in the MA, but the second site would be welcome because it gives them greater ability to help us get this drug approved in a compliant manner to patients in a high unmet need setting.

Uh, GMP General site inspections by, uh, EMA and FDA. So, um, you know, we feel really good about the position that we are in and then, you know, I might might close by saying, you know, we contemplated the well. What if we remove 1 of the facilities, uh, from the application, could it impact our timeline at all? Uh, and that was contemplated and discussed between us and the agency. And based upon our discussions with the agency dating,

Speaker #6: So at the March juncture, we said we're ready to submit a second fill finish site. They welcomed that. We've talked through the process of how to get to submission and then the finish line with the PDUPA date and we are exactly on track with all of that.

Back to, you know, those March discussions. Uh, we do not expect uh, that removing a facility from the application would have any, um, impact on our ongoing review, timeline at all. So, uh, Keith and team are ready to launch at any time between now, and up to our September 30th to do for date. I'm certainly gratified, uh, by scholar Rock's technical operations and quality team. Uh, they have stood up this, uh, second fill finish facility faster than, you know, almost any example, we can find in the industry and to now have more

Speaker #6: Their review of the second fill finish site is progressing well. And as David said, we see no reason why we can't get to the PDUPA date with this drug approved before or at that time.

David Hallal: We'll continue to keep you all updated over these last 55 days, but we feel like we're in a really good position. Thank you.

Speaker #6: And so it certainly feels on track. And we're very grateful to the agency's guidance and the expeditious manner in which they've been working with us.

Operator: Thank you.

Uh, approval. Um, we're super excited. So we'll continue to keep you all updated, uh, over these last 55 days. But um, uh, we feel like we're in a really good position. Thank you.

Thank you.

Operator: One moment for the next question. The next question is coming from the line of Kripa Devarakonda of Truist Securities. Please go ahead.

Speaker #6: And the true engagement and partnership.

1 moment for the next question.

Speaker #8: Thank you.

Speaker #5: One moment for the next question, please. Our next question is coming from the line of Michael Yee of UBS. Please go ahead.

And the next question is coming from the line of Crypto Deer, Wakanda of Truist Securities. Please go ahead.

Srikripa Devarakonda: Hey, guys. Thank you so much for taking my question and congratulations on all the progress. I have a follow-up question on Eric's question regarding the second site. David, you just mentioned that you can drop one of the sites at any time, and it won't delay, but I was wondering if Catalent remains classified as OAI, is there a deadline or a date for administratively withdrawing that site so it doesn't trigger any delay for your PDUFA? Thank you.

Kripa Devarakonda: Hey, guys. Thank you so much for taking my question and congratulations on all the progress. I have a follow-up question on Eric's question regarding the second site. David, you just mentioned that you can drop one of the sites at any time, and it won't delay, but I was wondering if Catalent remains classified as OAI, is there a deadline or a date for administratively withdrawing that site so it doesn't trigger any delay for your PDUFA? Thank you.

Speaker #7: Hi, this is Madeline on for Michael. Congrats on all the progress and thank you for the updates today. We were just wondering, do you have any color or any feedback from the FDA on that later than expected reclassification of the Catalent site given the decision was sort of expected by the end of July?

Speaker #7: If you just have any feedback, you could pass along.

Speaker #1: Yeah, the only thing that we would note is obviously the inspection report is public as are the 483 observations in that inspection report. We're well aware that Novo Nordisk provided a pretty robust response as they had the option to do within 15 days of that inspection.

Hey guys, thank you so much for taking my question and uh, congratulations on all the progress. Um, I have a follow-up question on Eric's question regarding the second site. Um, if if the David you just mentioned that you can drop 1 of the sites at any time and it won't delay, but I was wondering if Catelyn remains classified as oai. Um, is there a dead a deadline or a date for administratively, withdrawing that site. So, so it doesn't, um, trigger any delay for your producer. Thank you.

David Hallal: It's a great question, Kripa. We are in correspondence with the agency. We're both well aware that the inspection classification is still pending from the April re-inspection. As Akshay noted, it's now drifted a bit beyond the 90-day guidance period, but it is only a guidance period from the FDA that they would classify an inspection. Look, we're very direct with one another about when we would reach that step, should we need to reach that step. In your example that you provided, let's just say the inspection classification reveals that there's no change to the current OAI classification. We've discussed with the agency, it would be a relatively simple step to notify them that on their signal, that we are removing that site, and the review would progress with that second facility.

Uh, it's a great question. KPa, we um are in correspondence, uh, with the agency—they are, um,

we're both well aware that the um,

Inspection, classification is still pending from the

Speaker #1: So there was a lot there for the FDA to review. And I would just note that that guidance period is a guidance period where aware that sometimes the FDA does take a bit more time than that 90-day period and as we noted, we're awaiting that.

Speaker #1: I'm sure our friends at Novo Nordisk are awaiting that inspection classification. And while in parallel, the EMA is awaiting that classification. We're also engaging with our European regulators on the inclusion of our second fill finish facility.

Speaker #1: So there really is nothing more to it other than the fact that it does happen. The 90 days are not a statutory requirement. The FDA literally has provided that as guidance.

David Hallal: Again, with our alignment with the FDA, we would expect no impact to the ongoing timeline. That's where we are. I think Akshay and I are both heartened by the fact that we have ongoing open correspondence with the FDA, and the review is progressing well.

April reinspection as actually noted. It's, you know, now drifted, you know, a bit beyond the 90-day guidance period, but it is only a guidance period from the FDA that they would classify an inspection. Um, and and look, we're very direct with 1. Another about, you know, when we would reach that step, should we need to reach that step in, in, in your example, that you provided. Let's just say the inspection classification reveals that there's, there's no change, uh, to the current oai classification. Um, we've discussed with the agency. It would be a, a relatively simple step, um, to notify them that, you know, on their signal, that we are removing that site. And uh and the review would progress uh, you know, with that second facility. So uh, and again with, you know, our

Speaker #1: There's probably a lot there that the FDA is reviewing. And we await like everybody else the pending inspection classification. But I'd bring it back to this.

Alignment with the FDA, we would expect no impact to the ongoing timeline. So, that's, that's where we are. I think, um, actually, and I are both heartened by the fact that we have ongoing, um, open correspondence with the FDA. And, and the review is progressing. Well,

Srikripa Devarakonda: Thank you so much.

Kripa Devarakonda: Thank you so much.

Thank you so much.

Operator: Thank you. One moment for the next question. Our next question is coming from the line of Tessa Romero of J.P. Morgan. Please go ahead.

Operator: Thank you. One moment for the next question. Our next question is coming from the line of Tessa Romero of JPMorgan. Please go ahead.

Speaker #1: Both in US and Europe, we're continuing to move forward with this meaningful progress that we've made with our second fill finish facility. Really wanted to make sure that we had a belt and suspenders approach to serving children and adults living with SMA and their families.

Thank you. 1 moment for the next question.

Our next question is coming from the line of Tess from Morrow at JP Morgan. Please go ahead.

Tessa Romero: Hi, David and team. Hope you are all well, and thanks so much for taking our question. Just to double-click here on some of these earlier comments, what are the specific items procedurally from now to 30 September that still need to be ticked off to allow for an approval? I think Akshay used the words final steps. Just to set the record straight, is there any reason to believe that the FDA will not be able to complete this review by 30 September? Thank you.

Speaker #1: And we feel like we're in a position of strength as we move forward. And we await like everybody else that inspection classification.

Speaker #7: Makes sense. Thank you.

Speaker #1: Thank you.

Speaker #5: Thank you. One moment for the next question, please. The next question is coming from the line of Cory Kasimov. Ever Cory, please go ahead.

Hi David and team. Hope you are all. Well and thanks so much for taking our question. So just to double click here on some of these earlier comments, what are the specific items procedurally from now to September 30th that still need to be ticked off to allow for an approval? I think option, I use the words final steps and just to set the record straight. Is there any reason to believe that the FDA will not be able to complete this review by September 30th? Thank you.

David Hallal: Yeah, I'll just hand it over to Akshay, but just underscore that last question. There really is no reason to believe with the work that's been done at the second facility and where we believe the FDA is in terms of reviewing the application, which as you recall, our resubmitted BLA really only included the new information from the second facility and the updated safety database. There's no reason to believe the FDA can't get their work done in these next 55 days. Akshay?

Speaker #6: Good morning, guys. Thank you for taking the question. I guess I'll shift gears a little bit here and want to ask about your national and regional payer discussions.

Speaker #6: And curious how they're framing a pictogram in step edit terms. Are you seeing any payer signal that they'll impose time limits or require re-review of benefit after a shorter initial authorization?

Over to the a but just underscore that last question. There really is no reason to believe with the work that's been done, uh, at the second facility and where we believe the FDA is in terms of of reviewing the application uh which as you recall, right, our resubmitted bla really. Um,

Speaker #6: Thank you.

Speaker #1: Thanks, Cory. I'm going to hand that over to Keith for his comments. I would just note as a headline as Keith is prepared to answer that we do believe this robust clinical development program that we've been running for seven plus years and again, the fact that we met the highest bar, which was the Hammersmith Motor Function Scale in SMA, with a stat sig result from our pivotal sapphire trial, the robustness of that data sets up very, very well.

Akshay Vaishnaw: Yeah, I would just reconfirm that, Tess. Starting last November when we had a face-to-face meeting with the FDA, with all relevant parties, in March, the FDA guided that Catalent is the lead site in the MA, but a second site would be welcome because it gives them greater ability to help us get this drug approved in a compliant manner to patients in a high unmet need setting. At the March juncture, we said we're ready to submit a second fill-finish site. They welcomed that. We've talked through the process of how to get to submission and then the finish line with the PDUFA date, we are exactly on track with all of that. Their review of the second fill-finish site is progressing well.

Only included the new information from the second facility in the updated. Um, safety database, there's no reason to believe the FDA can't get their work done in these next 55 days option. Yeah, I would just, uh, reconfirm that test. So, starting last November, when we had a face-to-face meeting with the FDA with all relevant parties,

Speaker #1: As you know, and I'll just underscore for everybody listening in, I know you know it quite well, Cory, patients were randomized who were on ongoing SMN targeted therapy to receive either placebo or a pictogram.

Speaker #1: So they were on these ongoing therapies. And again, to hit that highest bar of the Hammersmith Motor Function Scale in SMA at stat sig with a 0.019 p-value, we feel with a very low number of patients, we think sets up very, very well for those national and regional payer discussions.

Akshay Vaishnaw: As David said, we see no reason why we can't get to the PDUFA date with this drug approved before or at that time. It certainly feels on track, and we're very grateful to the agency's guidance and the expeditious manner in which they've been working with us, and the true engagement and partnership.

Speaker #1: Keith?

Speaker #2: Yeah, thanks, David. Look, as I stated in the prepared remarks, the team has been working and really extending our not only our reach with these various payers, whether it's the commercial payers or government payers, but also the quality of the meetings by bringing in members from our medical team to discuss the robust clinical data package from our pictogram studies and ultimately with the goal of making a pictogram available for the broadest possible audience of children and adults living with SMA.

Tessa Romero: Thank you.

Thank you.

Operator: One moment for the next question, please. Our next question is coming from the line of Michael Yee of UBS. Please go ahead.

1 moment for the next question, please.

[Analyst] (UBS): Hi, this is Madeleine on for Michael. Congrats on all the progress and thank you for the updates today. We were just wondering, do you have any color or any feedback from the FDA on that later than expected reclassification of the Catalent site, given the decision was sort of expected by the end of July? If you just have any feedback you could pass along.

Our next question is coming from the line of Michael. Why of UBS? Please go ahead.

Speaker #2: So as I've stated before, the real goal with these meetings is to be able to create policies as rapid as possible and policies that we believe will align more with the potential label, the FDA label, and less with an inclusion exclusion criteria from our sapphire study.

Hi. This is Moline on for Michael, uh, congrats on all the progress, and thank you for the updates today. Uh, we were just wondering, do you have any color, or any feedback from the FDA on that later than expected? Reclassification of the Catalan site, given the decision was sort of expected by the end of July. If you just have any feedback, you could pass along,

David Hallal: Yeah, the only thing that we would note is obviously the inspection report is public as are the 483 observations in that inspection report. We're well aware that Novo Nordisk provided a pretty robust response as they had the option to do within 15 days of that inspection. There was a lot there for the FDA to review. I would just note that that guidance period is a guidance period. We're aware that sometimes the FDA does take a bit more time than that 90-day period. As we noted, we're awaiting that. I'm sure our friends at Novo Nordisk are awaiting that inspection classification. While in parallel, the EMA is awaiting that classification, we're also engaging with our European regulators on the inclusion of our second fill-finish facility. There really is nothing more to it other than the fact that it does happen.

Speaker #2: With all that being said, if you take a look at the data on treatment for SMA, so I'm talking about the three SMN targeted therapies that are available, almost 100% of them have a prior authorization.

yeah, the only thing that we would note is um, obviously the the

Speaker #2: So I fully expect that you'll see that a pictogram will have a prior authorization even when we have favorable policies that are constructed.

you know, the inspection report is is public as as as are the 4833, uh, observations in that inspection report, um, we are well aware that Novo Nordisk provided a

You know, a pretty robust response, uh, as they uh had. Um,

Speaker #6: Great. Thank you, guys. That's helpful.

Speaker #1: Thanks, Cory.

Speaker #5: Thank you. One moment for the next question. Next question is coming from the line of Amy Lee of Jeffries. Please go ahead.

Speaker #7: Awesome. Thanks so much. And congrats on the progress. I just wanted to put a finer point on the second fill finish facility. You mentioned that you submitted data that the FDA requested the type C, which sounds encouraging, but could you give us a sense of what was included in that data package?

You know, the option to do within 15 days uh, of that inspection. So there was a lot there for the fda's, um, to, to review and um, you know, I would just note that, um, that guidance period is a guidance period, where aware that sometimes the FDA does.

Um, you know, take a bit more time than that 90-day period. And, you know, as we noted, you know, we're awaiting that. I'm sure our friends at Novo Nordisk are waiting, uh, that uh, inspection classification. And um,

Speaker #7: Our stability runs in release testing for this facility fully complete, and you would consider the facility launch ready. And do you expect any additional clearance from the FDA?

David Hallal: The 90 days are not a statutory requirement. The FDA literally has provided that as guidance. There's probably a lot there that the FDA is reviewing. We await, like everybody else, the pending inspection classification. I'd bring it back to this, both in US and Europe, we're continuing to move forward with this meaningful progress that we've made with our second fill-finish facility. Really wanted to make sure that we had a belt and suspenders approach to serving children and adults living with SMA and their families. We feel like we're in a position of strength as we move forward, and we await, like everybody else, that inspection classification.

Speaker #7: Thanks so much.

Speaker #1: Thanks, Amy. Yeah. I mean, what was inclusive and what we agreed upon was obviously the data that we generated from engineering runs, PPQ runs, the FDA has the full package in hand where we know their review of that is progressing well.

While in parallel the F, you know, the EMA is awaiting that classification. We're also engaging with our uh, European uh, Regulators on the inclusion of our second, Bill finish facility. So there really is nothing more to it other than the fact that it does happen. Uh, the 90 days are not, you know, a statutory requirement. Uh, the fda's literally has provided that as guidance. Um, there's probably a lot there that the FDA is reviewing.

Speaker #1: We're in correspondence with them on that. Everything is straightforward and perfunctory. And there is, I guess, just to underscore the question, there is no more testing that is required for that for the product that has been vialed at that facility at all.

Speaker #1: And again, as noted, that product is at our third-party labeling and packaging facility awaiting approval to support the launch. And of course, in a belt and suspenders approach, we have vials from Catalan, Indiana, at the packaging facility as well.

And, um, you know, we await like everybody else, uh, the uh, the pending inspection, uh, classification. But I'd bring it back to this. Um, both in us and Europe, were continuing to move forward with this meaningful progress that we made with our second fill finish facility. Really wanted to make sure that we had a belt and suspenders approach, uh, to, uh, serving, uh, children and adults living with SMA, uh, and their families. And we, we feel like we're in a position of strength, uh, as we move forward. And we await like everybody else, uh, that inspection classification.

[Analyst] (UBS): Makes sense. Thank you.

David Hallal: Thank you.

Makes sense. Thank you.

Operator: Thank you. One moment for the next question, please. The next question is coming from the line of Cory Kasimov of Evercore. Please go ahead.

Thank you.

Speaker #1: So we're in a really good position. We're gratified to have reached an agreement with the FDA in March at the type C meeting on what was required.

Thank you. One moment for the next question, please.

Cory Kasimov: Good morning, guys. Thank you for taking the question. I guess I'll shift gears a little bit here and want to ask about your national and regional payer discussions. Curious how they're framing apitegromab in step edit terms. Are you seeing any payer signal that they'll impose time limits or require re-review of benefit after a shorter initial authorization? Thank you.

The next question is coming from the line of Corey, kasimo of ever core. Please go ahead.

Speaker #1: I would note that what was required was delivered to the FDA in a very timely fashion because you don't only agree on what is submitted, but when it would be submitted within your framework of your PDUPA date and we felt like we delivered that in a very, very, very timely fashion.

Speaker #1: And we're looking forward to these next 55 days to get through the final step and eventually launch a pictogram. So thank you very much for your question.

David Hallal: Thanks, Cory. I'm going to hand that over to Keith for his comments. I would just note as a headline as Keith is prepared to answer that we do believe this robust clinical development program that we've been running for 7+ years, and again, the fact that we met the highest bar, which was the Hammersmith Functional Motor Scale in SMA with a stat sig result from our pivotal SAPPHIRE trial. The robustness of that data sets up very well. As you know, I'll just underscore for everybody listening in, I know you know it quite well, Cory, patients were randomized who were on ongoing SMN targeted therapy to receive either placebo or apitegromab. They were on these ongoing therapies.

Good morning, guys. Uh, thank you for taking the question, I guess. I'll shift gears a little bit here and want to ask about your national and regional payer discussions, and I'm curious how they're framing a pedigree grab and step edit terms. Or are you seeing any payers signal that they'll impose time limits or require re-review of benefit after a shorter initial authorization? Thank you.

Thanks Corey. Uh, I'm going to hand that over to Keith for uh,

Speaker #7: Thank you.

Speaker #5: Thank you. One moment, please, for the next question. Our next question is coming from the line of Gary Machman of Canocard. Please go ahead.

Speaker #6: Thanks, and good morning. So as you've been preparing for a while now with the commercial team, any other initiatives you need to put in place between now and approval, or is it just really waiting for the final label?

Speaker #6: And what's the low-hanging fruit to go after with SMA patients where there could be a fair amount of pent-up demand for a pictogram? And how long do you think it'll take to get those patients on board?

David Hallal: Again, to hit that highest bar of the Hammersmith Functional Motor Scale in SMA at stat sig with a 0.019 p-value, we feel with a very low number of patients, we think sets up very well for those on national and regional payer discussions. Keith?

Speaker #6: Thanks.

For his comments. I would just notice a headline as as, as Keith, uh, is is is prepared to answer that. Um, we do believe this robust clinical development program that we've been running for, you know, 7 plus years. And you know, again the fact that we met, you know, the highest bar which was the Hammer Smith motor function scale in SMA uh with a stat Sig result from our pivotal Sapphire trial. Um the robustness of that data sets up very, very well as you know, and I'll just underscore for everybody listening in. I know, you know, it quite well, Corey patients were randomized, who are on ongoing, uh, SMN, targeted therapy to receive either Placebo or a pyramid. So they were on these ongoing therapies and again, to hit that highest bar of the hammer.

Speaker #1: Yeah. I'll start and Keith will get in, but I think as I mentioned during the call, Gary, we spend a lot of time with the community.

R. Keith Woods: Yeah, thanks, David. Look, as I stated in the prepared remarks, the team has been working and really extending not only our reach with these various payers, whether it is the commercial payers or government payers, but also the quality of the meetings by bringing in members from our medical team to discuss the robust clinical data package from our apitegromab studies. Ultimately with the goal of making apitegromab available for the broadest possible audience of children and adults living with SMA. As I have stated before, the real goal with these meetings is to be able to create policies as rapid as possible, and policies that we believe will align more with the potential label, the FDA label, and less with an inclusion/exclusion criteria from our SAPPHIRE study.

Keith Woods: Yeah, thanks, David. Look, as I stated in the prepared remarks, the team has been working and really extending not only our reach with these various payers, whether it is the commercial payers or government payers, but also the quality of the meetings by bringing in members from our medical team to discuss the robust clinical data package from our apitegromab studies. Ultimately with the goal of making apitegromab available for the broadest possible audience of children and adults living with SMA. As I have stated before, the real goal with these meetings is to be able to create policies as rapid as possible, and policies that we believe will align more with the potential label, the FDA label, and less with an inclusion/exclusion criteria from our SAPPHIRE study.

Speaker #1: Patients, their families, the advocacy groups, the healthcare providers, and our ambition is that any patient living with SMA that can benefit from a pictogram should have access to a pictogram.

Speaker #1: And so we really do look at this holistically across the 35,000 patients globally that have received an SMN targeted therapy. And we believe that we can really offer a meaningful benefits to them.

Speaker #1: So we do think about it very holistically. Keith can share with you some of the dynamics at launch, but I think our general view is it could vary patient by patient, family by family, physician by physician.

Speaker #1: In terms of their thinking about commencing treatment. Keith?

R. Keith Woods: With all that being said, if you take a look at the data on treatment for SMA, so I am talking about the three SMN targeted therapies that are available, almost 100% of them have a prior authorization. I fully expect that you will see that apitegromab will have a prior authorization even when we have favorable policies that are constructed.

Keith Woods: With all that being said, if you take a look at the data on treatment for SMA, so I am talking about the three SMN targeted therapies that are available, almost 100% of them have a prior authorization. I fully expect that you will see that apitegromab will have a prior authorization even when we have favorable policies that are constructed.

Speaker #2: Yeah. Thanks, David. I guess first of all, what else is there to do? The point I want to make really clear is we are ready if we were to get the call tomorrow.

Speaker #2: The team is kind of chomping at the bit to really get out there and launch this product with that being said, there's always additional work that we can do.

Speaker #2: One of the main things that's taking place that I mentioned is really working with the various centers so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our scholar rock supports program.

Goal with these meetings is to be able to create policies as rapid as possible. And policies that we believe will align more with the potential uh label the the FDA label and less with an inclusion exclusion criteria. Um from our from our Sapphire study, um with all that being said, if you take a look at the data on treatment for SMA. So I'm talking about the 3,

David Hallal: Great. Thank you, guys. That is helpful.

Cory Kasimov: Great. Thank you, guys. That is helpful.

Laura Ekis: Thanks, Cory.

David Hallal: Thanks, Cory.

Great. Thank you guys. That's helpful.

Thanks Corey.

Operator: Thank you. One moment for the next question. Next question is coming from the line of Amy Lee of Jefferies. Please go ahead.

Operator: Thank you. One moment for the next question. Next question is coming from the line of Amy Li of Jefferies. Please go ahead.

Thank you. 1 moment for the next question.

Speaker #2: We cannot begin to do any of this until after we have FDA approval. So we are doing a lot of dry run work here so that we can have a seamless and flawless execution as we take patients from being prescribed the product to ultimately being able to receive the product.

Next question is coming from the line of Amy, Lee of Jeffrey's, please go ahead.

Amy Lee: Awesome. Thanks so much, and congrats on the progress. I just wanted to put a finer point on the second fill-finish facility. You mentioned that you submitted data that the FDA requested, the Type C, which sounds encouraging, but could you give us a sense of what was included in that data package? Are stability runs and release testing for this facility fully complete, and you would consider the facility launch-ready? Do you expect any additional clearance from the FDA? Thanks so much.

Amy Li: Awesome. Thanks so much, and congrats on the progress. I just wanted to put a finer point on the second fill-finish facility. You mentioned that you submitted data that the FDA requested, the Type C, which sounds encouraging, but could you give us a sense of what was included in that data package? Are stability runs and release testing for this facility fully complete, and you would consider the facility launch-ready? Do you expect any additional clearance from the FDA? Thanks so much.

Speaker #2: Where do you think the stump pent-up demand is? I've shared before on previous calls, we do have an early access program. Those will be the first patients that we will be focused on converting from early access product over to commercial product.

David Hallal: Thanks, Amy. Yeah, what was inclusive and what we agreed upon was obviously the data that we generated from engineering runs, PPQ runs. The FDA has the full package in hand where we know their review of that is progressing well. We're in correspondence with them on that. Everything is straightforward and perfunctory, and there is, I guess, just to underscore the question there is no more testing that is required for the product that has been vials at that facility at all. Again, as noted that product is at our third-party labeling and packaging facility awaiting approval to support the launch. Of course, in a belts and suspenders approach, we have vials from Catalent Indiana at the packaging facility as well. We're in a really good position.

Awesome. Thanks so much and uh congrats on the progress. Um, I just wanted to put a finer point on the second sils, finish. Uh facility. Uh, you mentioned that, you know, you submitted data, uh, that the FDA requested the type-c which which uh, sounds sounds encouraging but um, could you give us a sense of what was included in that data package? Um, our stability runs and release testing for this facility fully complete and and you know, you would consider the facility launch ready. Um, and do you expect any additional clearance from the FDA? Thanks so much.

Speaker #2: That being said, we don't have full line of sight into what insurance coverage these early access patients have. So we're going to have to be going through that process and enrolling them in our program.

Speaker #2: The next will be our open label extension patients, our Onyx patients. I want to remind you that they will have to go to a close-out visit of that study before they can even convert over to commercial drug.

Thanks Amy. Um, yeah, I mean what was inclusive and what we agreed upon was, obviously the data that we generated from engineering runs. PPQ runs the FDA has the full package and hand where we, we know their review of that as progressing. Well, we're in correspondence with them on that. Everything is

Speaker #2: But we will begin to work them through the process of our scholar rock supports program. So when you ask how long will it take, it will take time, mostly because you're going to see a prior auth with all of our patients that are going to be prescribed a pictogram.

Speaker #2: The majority of them, you are most likely going to receive a denial for various reasons, whether it's a J code, or not a policy not created, or for some other reason.

straightforward and perun. And, uh, uh, there is, I guess just to underscore the question. Uh, there is no more testing, uh, that is required, uh, for that, uh, you know, for the, the product that has been viled at that, facility at all. And again, as noted, uh, that that product is, uh, is at our third party, uh, labeling and packaging facility, awaiting approval.

Speaker #2: That will then send us into an appeal process, which can sometimes take some time. So I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others.

David Hallal: We're gratified to have reached an agreement with the FDA in March at the Type C meeting on what was required. I would note that what was required was delivered to the FDA in a very timely fashion, because you don't only agree on what is submitted, but when it would be submitted within your framework of your PDUFA date. We felt like we delivered that in a very timely fashion. We're looking forward to these next 55 days to get through the final step and eventually launch apitegromab. Thank you very much for your question.

Speaker #2: But what I've typically seen in other launches is during this first six months of launch while we will be without a J code, the time the average time from prescription to a patient actually being able to get infused is greater than 60 days.

Speaker #6: Great. Thanks for all that.

Speaker #5: Thank you. One moment, please, for the next question. Next question is coming from the line of Mark Firm. Please go ahead.

Uh, to support the launch. Um, and of course, in a belt and suspenders approach. We have vials from Catalan, Indiana at the packaging facility, as well. So, we're in a, a really good position. We're gratified. Uh, you know, to have, uh, reached an agreement with the FDA in March, uh, at the type C meeting on what was required. Um, I would note that what was required was delivered to the FDA in a very timely fashion because you don't only agree on what is submitted, but when it would be submitted within your framework, uh, of your Padua date. Um, and we felt like we um, delivered that in a very, very, very timely fashion and uh, we're looking forward to these next 55 days to get uh, through the final step.

And eventually launched a paragraph. So thank you very much for your question.

Amy Lee: Thank you.

Amy Li: Thank you.

Thank you.

Operator: Thank you. One moment, please, for the next question. Our next question is coming from the line of Gary Nachman of Canaccord. Please go ahead.

Speaker #4: And thanks for taking my questions. Maybe just I mean, it seems like the medical review is kind of done on both sides of the Atlantic.

Thank you. One moment, please, for the next question.

Speaker #4: So maybe you can speak to kind of the labeling discussions and do you expect a largely identical label or do you think they're important maybe important differences between the US and European label?

Our next question is coming from the line of Gary Nachman of Canaccord. Please go ahead.

Gary Nachman: Thanks and good morning. As you've been preparing for a while now with the commercial team, any other initiatives you need to put in place between now and approval, or is it just really waiting for the final label? What's the low-hanging fruit to go after with SMA patients where there could be a fair amount of pent-up demand for apitegromab? How long do you think it'll take to get those patients on board? Thanks.

Speaker #4: And then I'll probably have a follow-up.

Speaker #1: Yeah. No, it's an important question. As you know, we Akshay and I both address that we felt like we were in a really good spot at the end of the last review period with the FDA, and we're kind of picking it up more we've picked it up in the BLA right where we last left off.

Thanks, and good morning. Um, so as you've been preparing for a while now with the commercial team, are there any other initiatives you need to put in place between now and approval, or is it just really waiting for the final label? And what's the low-hanging fruit to go after with SMA patients, where there could be a fair amount of pent-up demand, uh, for apitegromab? And how long do you think it'll take to get those patients on board? Thanks.

David Hallal: Yeah, I'll start and Keith will get in, but I think as I mentioned during the call, Gary, we spend a lot of time with the community, patients, their families, the advocacy groups, the healthcare providers. Our ambition is that any patient living with SMA that can benefit from apitegromab should have access to apitegromab. We really do look at this holistically across the 35,000 patients globally that have received an SMN targeted therapy, and we believe that we can really offer meaningful benefits to them. We do think about it very holistically. Keith can share with you some of the dynamics at launch, but I think our general view is it could vary patient by patient, family by family, physician by physician in terms of their thinking about commencing treatment. Keith?

Speaker #1: And then the comparisons between the two options.

yeah, I'll I'll start and and Keith will get in but I think the

Speaker #6: Yeah. I would just say we're very happy and comfortable with the way the dialogue has gone through the medical review. And obviously, it's not for us to comment on the final label.

As, as I mentioned during the call Gary, we, we, we spend a lot of time with the community.

Speaker #6: But we are certainly grateful for the engagement and very constructive conversation. So we look forward to getting this drug approved in the US and Europe and I think we'll be comfortable with how to get it to the right patients early.

um, patients, their their families, the advocacy groups, uh, the the health care providers and uh, our ambition is that, um,

Speaker #1: Yeah. And Mark, I think due to the great work by Akshay and the entire team, I think at the end of the day, we ask ourselves, are we going to have an opportunity to serve a meaningful number of patients living with SMA in the US, in Europe, and again, eventually our ambition is to reach patients in 50 countries around the world.

Speaker #1: And I think that Akshay has put us in a really good and the entire team Jing and the entire team has put us in a really good position to be able to do that.

R. Keith Woods: Yeah. Thanks, David. I guess first of all, what else is there to do? The point I want to make really clear is we are ready if we were to get the call tomorrow. The team is kind of chomping at the bit to really get out there and launch this product. With that being said, there is always additional work that we can do. One of the main things that is taking place that I mentioned is really working with the various centers so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our Scholar Rock Supports program. We cannot begin to do any of this until after we have FDA approval.

Keith Woods: Yeah. Thanks, David. I guess first of all, what else is there to do? The point I want to make really clear is we are ready if we were to get the call tomorrow. The team is kind of chomping at the bit to really get out there and launch this product. With that being said, there is always additional work that we can do. One of the main things that is taking place that I mentioned is really working with the various centers so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our Scholar Rock Supports program. We cannot begin to do any of this until after we have FDA approval.

Speaker #1: But Akshay is right. Until we have final USPIs and SMPCs, I think we'll comment on that when it's the right time. And hopefully that time is coming in the coming days.

Speaker #4: Okay. That's helpful. And then just on the CMC side, the MA clear seems to be essentially deferring to the FDA on the Catalan Indiana facility.

Speaker #4: Do you expect them to ultimately act kind of similarly with the second facility or is there something about the either that facility itself or the flexibility that the FDA is kind of granted you in terms of CMC requirements there that might lead the EMA to kind of be more proactive itself in making a decision?

R. Keith Woods: We are doing a lot of dry run work here so that we can have a seamless and flawless execution as we take patients from being prescribed a product to ultimately being able to receive the product. Where do you think some pent-up demand is? I have shared before on previous calls, we do have an early access program. Those will be the first patients that we will be focused on converting from early access product over to commercial product. That being said, we do not have full line of sight into what insurance coverage these early access patients have, so we are going to have to be going through that process and enrolling them in our program. The next will be our open label extension patients, our ONYX patients.

Keith Woods: We are doing a lot of dry run work here so that we can have a seamless and flawless execution as we take patients from being prescribed a product to ultimately being able to receive the product. Where do you think some pent-up demand is? I have shared before on previous calls, we do have an early access program. Those will be the first patients that we will be focused on converting from early access product over to commercial product. That being said, we do not have full line of sight into what insurance coverage these early access patients have, so we are going to have to be going through that process and enrolling them in our program. The next will be our open label extension patients, our ONYX patients.

Speaker #1: No, it's a very thoughtful question, Mark. Akshay?

Speaker #3: Yeah. I mean, in our prepared remarks, we emphasized that the second fill finished facilities in very good standing with regulators. And we're delighted that that's in progress with the FDA.

Speaker #3: Now, with respect to the EMA, obviously any approval for a pictogram map that involves the second facility will help. And we are right now heavily engaged with the CHMP to work on the progress of the MA to completion and how we incorporate the second fill finished facility if necessary.

First of all, what else is there to do? The the point I want to make really clear is we are ready. If we were to get the call tomorrow, uh, the team is kind of chomping at the bit to really get out there and launch this product. With that being said, there's always additional work that we can do 1 of the main things that, uh, that's taking place that I mentioned is, is really working with, uh, the the various centers, so that we can be prepared, uh, with these treatment centers on a case-by-case basis to work through the process of, uh, enrolling patients into our scholar, Rock supports program. Uh, we cannot begin to do any of this until after we have FDA approval. So we are doing a lot of dry run work here so that we can have uh, a seamless and Flawless execution. As we take patients, from being prescribed to product, to ultimately being able to receive the product. Um, where do you think the, uh, as some pent up demand is I've shared before on previous calls, you know?

Speaker #4: Okay. Thank you.

Speaker #5: One moment for the next question, please. Next question is coming from the line of Calpit Patel of Wolf Research. Please go ahead.

R. Keith Woods: I want to remind you that they will have to go to a closeout visit of that study before they can even convert over to commercial drug, but we will begin to work them through the process of our Scholar Rock Supports program. When you ask how long will it take, it will take time, mostly because you are going to see a prior auth with all of our patients that are going to be prescribed upadacitabine. The majority of them, you are most likely going to receive a denial for various reasons, whether it is a J-code or a policy not created or for some other reason. That will then send us into an appeal process, which can sometimes take some time. I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others.

Keith Woods: I want to remind you that they will have to go to a closeout visit of that study before they can even convert over to commercial drug, but we will begin to work them through the process of our Scholar Rock Supports program. When you ask how long will it take, it will take time, mostly because you are going to see a prior auth with all of our patients that are going to be prescribed upadacitabine. The majority of them, you are most likely going to receive a denial for various reasons, whether it is a J-code or a policy not created or for some other reason. That will then send us into an appeal process, which can sometimes take some time. I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others.

We do have an early Access program. Those will be the first patients that we will be focused on converting from Early Access product over to, uh, commercial product. Uh, that being said, we don't have full, um, line of sight into what insurance coverage these Early Access patients have. So we're going to have to be going through that process and enrolling them in our program. Uh, the next will be, our open label extension. Patients are onyx, patients? Um, I want to remind you that they will have to code.

Speaker #7: Yeah. Hey. Good morning and thanks for taking the question. When for Europe, if the FDA maintains the OAI classification for Catalent, can you walk us through your potential timeline to get the second fill finished facility into the MAA and your thoughts on the earliest projected timeline for European approval?

Speaker #7: Thank you.

Speaker #1: Yeah. No, thank you very much. And as we noted during our during the call, while EMA and we await the classification decision from the FDA in parallel, we're having the dialogue the EMA is very well aware of where we are with the second fill finished facility.

R. Keith Woods: What I have typically seen at other launches is during this first six months of launch, while we will be without a J-code, the average time from prescription to a patient actually being able to get infused is greater than 60 days.

Speaker #1: Akshay?

Keith Woods: What I have typically seen at other launches is during this first six months of launch, while we will be without a J-code, the average time from prescription to a patient actually being able to get infused is greater than 60 days.

Speaker #3: Yeah. And as that dialogue is ongoing, I don't want to second guess what the final advice will be, vis-à-vis necessity or the mechanism by which we incorporate the second fill finished facility.

Speaker #3: But suffice it to say that the engagement and flexibility that both FDA and CHMP have shown us is very gratifying to us. They appreciate the unmet need.

Go to a close out, visit of that study before they can even convert over to commercial drug, but we will begin to work them through the process of our scholar Rock supports program. So, when you ask how long will it take? Um, it it will take time mostly because you're going to see a prior off with, with all of our patients that are going to be prescribed to pedag the majority of them. You are most likely going to receive a denial for various reasons, whether it's a jode, um, or not a policy, not created or for some other reason that will then send us into an appeal process, uh, which can sometimes, you know, take some time. So, um, you know, I I, I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others. But what I've typically seen other launches is during this first 6 months of launch while we will be without a J Code. You know, it the time the average time from prescription to a patient, actually being able to get infused is greater than 60 days.

Gary Nachman: Great. Thanks for all that.

Great, thanks for all that.

Operator: Thank you. One moment please for the next question. Next question is coming from the line of Mark Ferm of J.P. Morgan. Please go ahead.

Speaker #3: And they're working with us. So we will be guided by them. There's an ongoing conversation. And hopefully soon in due course, we will update everything.

Thank you. 1 moment, please for the next question.

Next question, is coming from the line of Mark firm of Cohen. Please go ahead.

Mark Ferm: Thanks for taking my questions. It seems like the medical review is kind of done on both sides of the Atlantic. Maybe you can speak to kind of the labeling discussions, do you expect a largely identical label, or do you think there are maybe important differences between the US and European label? I'll probably have a follow-up.

Speaker #7: Okay. Thank you.

Speaker #1: I think just as a capper, I think what Akshay and I are really gratified. Just as we've experienced with the FDA, we're just gratified by the receptivity and the dialogue with EMA.

Speaker #1: But again, we'll await the specific approach pending the inspection classification. And of course, in parallel, our discussions on the second facility.

And thanks for uh, taking my my questions maybe just I mean, it seems like the medical review is kind of done on both sides of the Atlantic. So maybe you can speak to kind of the labeling discussions and do you expect the, you know, a largely identical label or you know, do you think there are important? Maybe important differences, um, between the US and and European label

David Hallal: Yeah, no, it's an important question. As you know, Akshay and I both addressed that we felt like we were in a really good spot at the end of the last review period with the FDA, we're going to be picking it up more. We picked it up in the BLA right where we last left off, the comparisons between the two. Akshay, any comment?

And that and then I'll probably have a follow-up.

Speaker #7: Great. Thank you.

Speaker #5: Thank you. One moment, please, for the next question. Our next question is coming from the line of Etzer. Please go ahead.

Akshay Vaishnaw: I would just say we're very happy and comfortable with the way the dialogue has gone through the medical review, and obviously it's not for us to comment on the final label. We are certainly grateful for the engagement and the very constructive conversation. We look forward to getting this drug approved in US and Europe, and I think we'll be comfortable with how to get it to the right patients quickly.

Yeah, no, it's an important question. Um, as you know, we actually—Akshay and I both addressed that we felt like we were in a really good spot at the end of the last review period with the FDA, and we're going to be picking it up more. We picked it up in the, uh, in the BLA, right? Where we last left off, and then the comparisons between the two officers on it.

Speaker #6: Hi. This is Luke Armstrong. Thanks for taking our question. For FSHD, on the clinical trial side, I know you guys mentioned that you're looking to focus in slightly less severe patients.

Speaker #6: And you're listing the participation requirement as a 1.5 to 3 on a Richie scale on a 0 to 5 scale. Just want to verify that you're using a modified scale there because I think the Richie scale is 0 to 10.

Speaker #6: And could you give some color as to what percentage of the FSHD population falls within that scale range?

David Hallal: Mark, I think due to the great work by Akshay and the entire team, I think at the end of the day, we ask ourselves, are we going to have an opportunity to serve a meaningful number of patients living with SMA in the US and Europe? Eventually, our ambition is to reach patients in 50 countries around the world, and I think that the entire team. Jing and the entire team has put us in a really good position to be able to do that. Akshay is right. Until we have final USPIs and SMPCs, I think we'll comment on that when it's the right time. Hopefully that time is coming in the coming days.

Yeah, I you know, I would just say we're very happy and comfortable with the way the dialogue is going through the medical review and obviously it's not for us to comment on the final label. Uh but uh, we are certainly grateful for the engagement and the Very constructive conversation. So we look forward to getting this drug approved in the US and Europe. And and I think uh we'll be comfortable with how to get it to the right patients. Perfect.

Yeah.

And and Mark, I think, you know, due to the great work.

Speaker #3: Yeah. We're using the same Richie scaling system or scoring system that the Roche folks are used. And I think just want to confirm what you said, that we're indeed focusing on patients with a Richie score of 1.5 to 3 because once you get beyond a score of 3, we know from an FSHD database, we have access to and I don't know whether Roche had it or not, but we know that by MRI, many muscle groups including the quads, which was their primary endpoint, begin to show a significant fat infiltration and fibrosis.

You know, by Ash in the entire team I think at the end of the day we ask ourselves, are we going to have an opportunity to to serve a meaningful number of patients living with SMA?

Speaker #3: So focusing on patients with the higher scores, I think is a tough ask. And so you need some muscle preserved so that the antimyostatic mechanism can act on it to boost muscle mass and hopefully function too.

In the US in Europe. And again, eventually, our ambition is to reach patients in 50, countries around the world. And I think that, uh, AI has put us in a, a really good. And in the entire team Jing and the entire team has placed a really good, uh, position to be able to do that. But AI is right until we have final, you know, uspis and smpp. So I think we'll comment on that. Um, you know, when when it when it's the right time and hopefully that time is coming, uh, in the coming days.

Mark Ferm: Okay. That's helpful. Then just on the CMC side, the EMA clearly seems to be essentially deferring to the FDA on the Catalent Indiana facility. Do you expect them to ultimately act kind of similarly with the second facility, or is there something about either that facility itself or the flexibility that the FDA has kind of granted you in terms of CMC requirements there that might lead the EMA to kind of be more proactive itself in making a decision?

Speaker #3: Also, in terms of functionality, you'll note that their inclusion criteria include 10-meter walk test between 4 to 12 seconds. So the upper bound is 12, whilst we've stipulated that the upper bound for our 10-meter walk is less than 5.

Catalin Indiana facility—do you expect them to ultimately act similarly with the second facility, or is there...

Speaker #3: So we're certainly in that mild to moderate group of patients, whilst they were in the moderate to severe number of patients, this is the commonest muscular dystrophy or there's at number of patients around the world to help.

David Hallal: No. It's a very thoughtful question, Mark. Akshay?

Something about either that facility itself or the flexibility that the FDA has kind of granted you in terms of CMC requirements there that might lead the EMA to be more proactive itself in making a decision.

Akshay Vaishnaw: Yeah. In our prepared remarks, we emphasized that the second fill finish facility is in very good standing with regulators. We're delighted that that's in progress with the FDA. Now, with respect to the EMA, obviously any approval for upadacitabine that involves the second facility will help. We are right now heavily engaged with the CHMP to work on the progress of the MAA to completion and how we incorporate the second fill finish facility if necessary.

Speaker #3: And I think we're comfortable that we will help many, many patients should this drug ultimately be approved in that space. So I'm not concerned about that.

Speaker #3: And of course, as we know in all these rare diseases, as soon as a therapeutic appears, the rate of diagnosis improves and the rate of access to therapies improves and patients start getting put on therapy earlier and earlier in the course of their disease.

No. I said it's a very thoughtful question mark oxen. Yeah. I mean in a prepared remarks we emphasize that the second floor finish facilities in very good standing with Regulators. Uh, and we're delighted that that's uh in progress with the FDA now with respect to the EMA. Obviously any approval for a pedigree map that involves the second facility will help. Um, and we are right now uh heavily engaged with the chmp to work on the progress of the M8 to completion. And how we uh incorporate the second pill fish finish facility. If necessary.

Mark Ferm: Okay. Thank you.

Okay, thank you.

Operator: One moment for the next question, please. Next question's coming from the line of Kal Patel of Wolfe Research. Please go ahead.

Speaker #3: So we're looking forward as we announce today to getting momentum going now in the study. The study is initiated. And we're excited to do this study where we have wonderful mouse data and where we think there's a robust rationale with our drug.

1 moment for the next question, please.

Next question is coming from the line of capet, Patel of wolf research, please go ahead.

Kal Patel: Yeah. Hey, good morning, thanks for taking the question. One for Europe. If the FDA maintains the OAI classification for Catalent, can you walk us through your potential timeline to get the second fill finish facility into the MAA and your thoughts on the earliest projected timeline for European approval? Thank you.

Speaker #6: Okay. Thank you.

Speaker #5: Thank you. One moment, please, for the next question. Next question is coming from the line of Basma Radwan. Of Lyrinc Partners, please go ahead.

Yeah. Hey, good morning, and thanks for taking the question. One for Europe. If the FDA maintains the OAI classification for Catalent, can you walk us through your potential timeline to get the second fill-finish facility into the MIAA and your thoughts on the...

Speaker #8: Good morning. Thank you for taking our question. Could you please share your perspective on a general recent update regarding the ILEA high-dose pre-fill syringe?

Earliest projected, uh, timeline for European approval. Thank you.

David Hallal: Yeah, no, thank you very much. As we noted during the call, while EMA and we await the classification decision from the FDA, in parallel, we're having the dialogue. The EMA is very well aware of where we are with the second fill-finish facility. Akshay.

Yeah, no, thank you very much. And, uh, as we noted during our—

um, you know, during the call

Speaker #8: Specifically, management indicated that it plans to add a third plant on their regulatory package to enhance the likelihood of approval. How do you interpret that decision, given that the situation is very similar to yours with regard to Catalent involvement?

While uh, EMA and we await the classification decision uh from the FDA and parallel, we're having the the dialogue. The the EMA is very well aware of where we are with the second.

Akshay Vaishnaw: Yeah. As that dialogue is ongoing, I don't want to second guess what the final advice will be vis-a-vis necessity or the mechanism by which we incorporate the second fill-finish facility. Suffice to say that the engagement and flexibility that both FDA and CHMP have shown us is very gratifying to us. They appreciate the unmet need, and they're working with us. We will be guided by them. They're the ongoing conversation. Hopefully soon, in due course, we will update everybody.

Speaker #8: Do you think it suggests that Catalent problems may be still outstanding? That's it for us. Thank you.

Speaker #1: Well, we know for first of all, I can't really comment on Regeneron other than to say I think we have a very different situation because our second fill finished facility is different than their second fill finished facility in this case.

Speaker #1: From our understanding, so that would just be a bright line. The similarity is that we both have Catalent Indiana in our applications. So I really can't comment on their commentary beyond the fact that I would note that our second fill finished facility is in good standing with the FDA and EMA.

Yeah. And and as that dialogue is ongoing, I don't want to second guess, you know what, the final advice will be. These are the necessity or the mechanism by which we incorporate uh the second Bill finish facility, but suffice it to say that the engagement uh and flexibility that both FDA and thmp have shown us uh is very gratifying to us, they appreciate the unmet need and they're working with us. So we we will be guided by them that the ongoing conversation and hopefully soon in due course we will update every week.

Kal Patel: Okay. Thank you.

David Hallal: Just as a capper, Akshay and I are really gratified. Just as we've experienced with the FDA, we're just gratified by the receptivity and the dialogue with EMA. Again, we'll await the specific approach pending the inspection classification and, of course, in parallel, our discussions on the second facility.

Okay, thank you. I think—I think it...

as a Capper.

Speaker #1: They've had several general site inspections by FDA and EMA in 2025 and 2026. Their last 12 months of approvals for products there, and they have nearly three dozen or more commercially available products from that facility.

I think what, you know, ache and I really gratify just as we've experienced with the the FDA. We're just, uh, gratified by the receptivity and the dialogue with Emma. But again, we'll we'll await the, you know, specific approach. Uh, you know, pending the inspection classification and of course in parallel our discussions uh on the second facility.

Speaker #1: But in the last 12 months, all approvals from that fill finished facility the PLI, PAIs have been waived by the agency. So I would just comment that yes, as we've noted, the Catalent Indiana inspection classification is still pending.

Kal Patel: Great. Thank you.

Operator: Thank you. One moment please for the next question. Our next question is coming from the line of Edgar Girod of Barclays. Please go ahead.

Operator: Thank you. One moment please for the next question. Our next question is coming from the line of Edgar Gerard of Barclays. Please go ahead.

Great. Thank you.

Thank you. One moment, please, for the next question. Our next question is coming from the line of Eder Abu, of Barclays. Please go ahead.

Luca Amore: Hi, this is Luca Amore. Thanks for taking our question. For FSHD, on the clinical trial site, I know you guys mentioned that you're looking to focus in slightly less severe patients, and you're listing the participation requirement is a 1.5 to 3 on a Ricci score on a 0 to 5 scale. Just want to verify that you're using a modified scale there because I think the Ricci score is 0 to 10. Could you give some color as to what percentage of the FSHD population falls within that scale range?

[Analyst] (Barclays): Hi, this is Luca Amore. Thanks for taking our question. For FSHD, on the clinical trial site, I know you guys mentioned that you're looking to focus in slightly less severe patients, and you're listing the participation requirement is a 1.5 to 3 on a Ricci score on a 0 to 5 scale. Just want to verify that you're using a modified scale there because I think the Ricci score is 0 to 10. Could you give some color as to what percentage of the FSHD population falls within that scale range?

Speaker #1: So that could be one thing that what you're referring to means. But they're very different situations in that we have a different second fill finished facility.

Speaker #8: Thank you.

Speaker #5: Thank you. One moment. And the last question will be coming from Jeff Mitchum. Of Citigroup, please go ahead.

Speaker #7: Great. Hey guys, thanks for the question. David, in line with your belt and suspenders comment, does a second facility provide a fully independent approval path or are there any elements of the filing that are still dependent on Catalent Indiana?

Hi. This is thanks for taking your question, um, for fshd on the, the clinical trial site. I know you guys mentioned that you're looking to focus in slightly less severe, patients, and you're listening. Uh the participation reporting is a 1.5 to 3 and reaching scale on a 0 to 5 scale. Just want to verify that you're using a modified scale there because I think the region scale 0 to 10 and could you give some color as to what percentage of the fshd population? Uh, Falls within that scale range?

Akshay Vaishnaw: Yeah. We're using the same Ricci scaling system or scoring system that the Roche folks have used. I think just want to confirm what you said, that we're indeed focusing on patients with a Ricci score of 1.5 to 3, because once you get beyond a score of three, we know from an FSHD database we have access to, and I don't know whether Roche had it or not, but we know that by MRI, many muscle groups, including the quads, which was their primary endpoint, begin to show a significant fat infiltration and fibrosis. Focusing on patients with the higher scores, I think is a tough ask. You need some muscle preserved so that the anti-myostatin mechanism can act on it, to boost muscle mass and hopefully function too.

Yeah, we using the same reachi scaling system or scoring system that the race folks are used. And I think just want to confirm what you said that.

Speaker #7: And then second question maybe for Akshay, what regulatory work will be required to get subcutaneous epididymomab into the next sort of pivotal development? I just wanted to maybe go over that.

Speaker #7: Thank you. Thank you guys.

Speaker #6: Akshay, you want to take call?

Speaker #3: Yeah. Thanks, Jeff. So on the first question, the second fill finished facility provides a fully independent path and the details of that were discussed in that March meeting we mentioned in the prepared remarks.

We indeed focusing on patients with a recent score of 1.5 to 3 because once you get beyond the score of 3 we know from an fshd database we have access to and I don't know whether the roach had it or not but we know that by MRI many muscle groups including the quads which which was their primary end point begin to show a significant fat infiltration and fibrosis. So you know focusing on patients with the um higher scores I think is a

Speaker #3: So we feel good about that and I think that sort of speaks to David's belt and suspenders comment. So that's great. And vis-à-vis the subcutaneous epididymomab, just to refresh folks, in January, we shared data showing the wonderful sort of PKPD profile of subQ epididymomab, which makes all this feasible.

Akshay Vaishnaw: In terms of functionality, you'll note that their inclusion criteria included a 10-meter walk test between 4 to 12 seconds. The upper bound is 12, while we've stipulated that the upper bound for our 10-meter walk is less than 5. We're certainly in that mild to moderate group of patients while they were in the moderate to severe. As to the exact numbers of patients, this is the commonest muscular dystrophy, or there's at least a very significant 5-digit number of patients around the world to help. I think we're comfortable that we will help many patients should this drug ultimately be approved in that space. I'm not concerned about that.

Speaker #3: And we have prepared a briefing document that we will be ready to send very soon after the approval of epididymomab so that we can engage with regulators and begin that next important phase of development to bring a subQ option for patients.

Speaker #3: Now, that involves a dialogue with the FDA because for different drugs, different parts have been adopted. There's one view of the world that says there can be a PKPD path matching the PKPD criteria seen with epididymomab IV.

Akshay Vaishnaw: Of course, as we know in all these rare diseases, as soon as a therapeutic appears, the rate of diagnosis improves, and the rate of access to therapies improves, and patients start getting put on therapy earlier and earlier in the course of their disease. We're looking forward, as we announced today, to getting momentum going now in the study. The study's initiated, and we're excited to do this study where we have wonderful mouse data and where we think there's a robust rationale with our drug.

Speaker #3: The level of myostatin suppression and saturation and so forth. And the other extreme is you need to do more substantive development work. You mentioned pivotal.

Speaker #3: We are now finalizing the briefing documents to present the path forward that we think is reasonable, but we obviously need to engage with regulators to get their guidance.

In that, um, you know, um, ball to moderate, uh, group of patients, whilst they were in the moderate to severe to severe as to the exact number numbers of patients. This is, you know, the commonest muscular drophy or there's at least a very significant 5 digit number of patients around the world to help. And I think we're comfortable that that we will help many many patients, should this drug ultimately be approved in that space so I'm not concerned about that. And of course, as we know in all these rare diseases, as soon as a therapeutic, appears the rate of diagnosis improves and the rate of access to therapies, improves and patients, start getting put on therapy early and earlier in the course of their disease. So um, we're looking forward, uh, as we announced today to getting momentum going now in the study, the studies initiated and uh, we're excited to do this study where we had wonderful Mouse data and where we think there's a robust rational with our drug.

Speaker #3: But as soon as we've done that, we will then provide an update in due course as to the path forwards because that will ultimately influence the timelines of getting subQ epididymomab to patients.

Luca Amore: Okay. Thank you.

[Analyst] (Barclays): Okay. Thank you.

Operator: Thank you. One moment please for the next question. Next question's coming from the line of Basma Radwan of B. Riley Securities. Please go ahead.

Operator: Thank you. One moment please for the next question. Next question's coming from the line of Basma Radwan of Leerink Partners. Please go ahead.

Thank you. 1 moment, please for the next question.

Next question is coming from the line of basma. Read 1 of Lee rink Partners. Please go ahead.

Speaker #7: Awesome. Thanks, guys.

Basma Radwan: Good morning. Thank you for taking our question. Could you please share your perspective on Regeneron recent updates regarding the EYLEA high-dose prefilled syringe? Specifically, management indicated that it plans to add a third plant on their regulatory package to enhance the likelihood of approval. How do you interpret that decision, given that the situation is very similar to yours with regard to Catalent involvement? Do you think it suggests that Catalent problems may be still outstanding? That's it for us. Thank you.

Speaker #6: Thank you.

Speaker #1: Thanks, Jeff.

David Hallal: Well, first of all, I can't really comment on Regeneron other than to say, I think we have a very different situation because our second fill-finish facility is different than their second fill-finish facility in this case, from our understanding. That would just be a bright line. The similarity is that we both have Catalent Indiana in our applications. I really can't comment on their commentary beyond the fact that I would note that our second fill-finish facility is in good standing with the FDA and EMA. They've had several general site inspections by FDA and EMA in 2025 and 2026. Their last 12 months of approvals for products there, and they have nearly three dozen or more commercially available products from that facility. In the last 12 months, all approvals from that fill-finish facility, the PLI, PAIs have been waived. By the agency.

Uh, good morning. Thank you for taking our question. Um, could you please share your perspective on regeneron recent update regarding the AA high, do prefill syringe uh, specifically management indicated that it plans to add a third plant, on their regulatory package to enhance the likelihood of approval. Um, how do you interpret that decision? Giving that the situation a very similar to yours with regard to Caroline involvement. Uh, do you think it suggests that uh, Catalan problems may be still outstanding? Um, that's it for. Thank you.

well, we, we know for first of all, I don't I can't really comment on regeneron on other than to say, I think we have a very different, um, situation and because, uh,

Our second still finish facility is, is different than their second fill finish facility in this case, uh, from our understanding. Um, so that would just, you know, be a bright line. Um, the similarity is that we both have Catalan in Indiana.

Uh, you know, in our application. So I really can't comment on uh, their commentary beyond the fact that, um, I would note that our second fill finish facility is in good standing with the sea and EMA. Um, they've had SE several General site inspections by FDA and EMA in 2025 and 2026, uh, their last 12 months of approvals, um, for products there and they have like nearly 3 dozen or more uh commercially available products from that facility. But in the last 12 months, all approvals from that, they'll finish facility.

David Hallal: I would just comment that, yes, as we've noted, the Catalent Indiana inspection classification is still pending. That could be one thing that what you're referring to means. They're very different situations in that we have a different second fill-finish facility.

The pli piss have been waived uh, by the agency. So I would just comment that. Um yes, as we've noted, the Catalan Indiana.

inspection classification is still pending so that, that that could be 1 thing that what you're referring to means, uh, but they're very different situations and that, um,

You know, we have a different second, still finished facility.

[Analyst] (B. Riley Securities): Thank you.

Basma Radwan: Thank you.

Thank you.

Operator: Thank you. One moment. The last question will be coming from Jeff Mitchum of Citigroup. Please go ahead.

Thank you. 1 more moment.

And the last question will be coming from JF Mitchum of City Group, please go ahead.

Jeff Mitchum: Great. Hey, guys. Thanks for the question. David, in line with your belts and suspenders comment, does a second facility provide a fully independent approval path, or are there any elements of the filing that are still dependent on Catalent Indiana? The second question may be for Akshay. What regulatory work will be required to get subcutaneous apitegromab into the next sort of pivotal development? I just wanted to maybe go over that. Thank you. Thank you, guys.

David Hallal: Akshay, you want to take both?

Great. Uh hey guys, thanks for the question. Uh, David in line with your belts and suspenders. Comment. Um there's a second facility provide a fully independent approval path. Or are there any elements of the filing that are still dependent on on Catalan Indiana? Uh, and then the second question, maybe for a, what regulatory work will be required to, to get, uh, subcutaneous of pit um, into, you know, in into the next sort of pivotal development. I just wanted to maybe go over that. Thank you. Thank you guys.

Akshay Vaishnaw: Well, thanks, Jeff. On the first question, the second fill-finish facility provides a fully independent path and the details of that were discussed in that March meeting we mentioned in the prepared remarks. We feel good about that and I think that sort of speaks to David's belt and suspenders comment. That's great. Vis-à-vis the subcutaneous apitegromab, just to refresh folks, in January we shared data showing the wonderful sort of PK/PD profile of subcu apitegromab which makes all this feasible. We have prepared a briefing document that we will be ready to send very soon after the approval of apitegromab so that we can engage with regulators and begin that next important phase of development to bring a subcu option for patients. Now, that involves a dialogue with the FDA because for different drugs, different paths have been adopted.

Oxy you want to take go? Yeah, I thanks Jeff. So, you know, on the first question, uh, the second film finish was provides a fully independent path and the, the, the details of that were discussed in that March meeting. We mentioned in the prepared remarks. So, uh, we feel good about that, and, and I think that sort of speaks to David's belt and suspenders comments. So, so that's great. And Visa V the subcutaneous of Pilgrim map, uh, just to refresh folks. In January, we shared data showing the wonderful sort of pkpd profile of subq, a bit of a map, which makes all this feasible, and we have prepared. Um,

A briefing document that we will be ready to send very soon after the approval of a bit of a map so that we can engage with the regulators and begin that next important phase of development to bring us up to your option for patients now. Um,

Akshay Vaishnaw: There's one view of the world that says there can be a PK/PD path matching the PK/PD criteria seen with apitegromab IV, the level of myostatin suppression and saturation and so forth. The other extreme is you need to do more substantive development work. You mentioned pivotal. We are now finalizing the briefing documents to present the path forward that we think is reasonable, we obviously need to engage with regulators to get their guidance. As soon as we've done that, we will then provide an update in due course as to the path forwards because that will ultimately influence the timelines of getting subcu apitegromab to patients.

Have been adopted. Uh there's 1 view view of the world that says it can be a pkpd path matching the um pkpd criteria seen with, you know, the level of my stance in suppression and saturation and so forth. Um and the other uh extreme is you, you know, need to do more substantive development work. Um, you mentioned pivotal, you know? Uh, we we are now, uh, finalizing briefing documents to present the path forward that we think is reasonable, but we obviously need to engage with Regulators to get their guidance. Uh but as soon as we've done, that we will then provide an update in due course, as to the platforms because that will you know, ultimately influence the timelines of getting subq app applicative amount to patients.

Jeff Mitchum: Awesome. Thanks, guys.

David Hallal: Thanks, Jeff.

Awesome. Thanks guys.

Thanks Jeff.

Operator: Thank you. This does conclude today's programming. Thank you so much for joining. You may now disconnect.

Thank you, and this does conclude today's programming. Thank you so much for joining. You may now disconnect

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