Q1 2026 Scholar Rock Holding Corp Earnings Call and Business Update

May 7, 2026

Operator: Good morning, ladies and gentlemen. Welcome to Scholar Rock's Q1 2026 conference call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question and answer session. This call is being recorded on Thursday, 7 May 2026. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Operator: Good morning, ladies and gentlemen. Welcome to Scholar Rock's Q1 2026 Conference Call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question and answer session. This call is being recorded on Thursday, 7 May 2026. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Speaker #1: Following the presentation, we will conduct a question-and-answer session. To ask a question during the session, you would need to press star 11 on your telephone and you will then hear an automated message advising your hand is raised and to withdraw your question, you may press star 11 again.

Speaker #1: This call is being recorded on Thursday, May 7th, the conference over to Scholar Rock. Please go ahead. Good morning. I am Laura Ekus, Vice President of Investor Relations at Scholar Rock.

Laura Ekus: Good morning. I am Laura Ekus, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Chairman and Chief Executive Officer, Akshay Vaishnaw, President of R&D, R. 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, and Vikas will provide a financial update. We will 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 the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Speaker #1: With me today are David Hallal, Chairman and Chief Executive Officer Akshay Vaishnaw, President of R&D; Keith Woods, Chief Operating Officer; and Vikas Sinha, Chief Financial Officer.

Speaker #1: 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.

Laura Ekus: Keith will provide an update on our commercial readiness activities, and Vikas will provide a financial update. We will 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 the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Speaker #1: And 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 the 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.

Laura Ekus: 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 #1: 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.

Speaker #1: David, thank you, Laura, and good morning. Thanks to everyone for joining our first quarter earnings call. Scholar Rock is positioned for a pivotal year ahead.

David Hallal: Thank you, Laura, and good morning. Thanks to everyone for joining our Q1 earnings call. Scholar Rock is positioned for a pivotal year ahead. To that end, today, I am very pleased to announce that the FDA has accepted for review our Biologics License Application for apitegromab for the treatment of children and adults living with SMA. The agency has assigned a PDUFA action date of 30 September. Importantly, the accepted BLA includes two fill/finish facilities, Catalent, Indiana, and a second US-based facility, providing Scholar Rock with two independent paths to apitegromab approval. As a reminder, the sole approvability issue for apitegromab noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalent, Indiana, fill/finish facility, which is owned and operated by Novo Nordisk.

David Hallal: Thank you, Laura, and good morning. Thanks to everyone for joining our Q1 Earnings Call. Scholar Rock is positioned for a pivotal year ahead. To that end, today, I am very pleased to announce that the FDA has accepted for review our Biologics License Application for apitegromab for the treatment of children and adults living with SMA. The agency has assigned a PDUFA action date of 30 September.

Speaker #1: To that end, today, I am very pleased to announce that the FDA has accepted four review, our biologics licensed application for a pittogram app for the treatment of children and adults living with SMA, the agency has assigned a PDUFA action date of September 30th.

David Hallal: Importantly, the accepted BLA includes two fill/finish facilities, Catalent, Indiana, and a second US-based facility, providing Scholar Rock with two independent paths to apitegromab approval. As a reminder, the sole approvability issue for apitegromab noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalent, Indiana, fill/finish facility, which is owned and operated by Novo Nordisk.

Speaker #1: Importantly, the accepted BLA includes two fill-finish facilities: Catalin, Indiana, and a second US-based facility. Providing Scholar Rock with two independent paths to a pittogram app approval.

Speaker #1: As a reminder, the sole approvability issue for a pittogram app noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalin, Indiana, fill-finish facility which is owned and operated by Novo Nordisk.

Speaker #1: Since our in-person Type A meeting with the FDA in Q4, we have continued to work constructively and collaboratively with the agency. And we have made steady and rapid progress.

David Hallal: Since our in-person Type A meeting with the FDA in Q4, we have continued to work constructively and collaboratively with the agency, and we have made steady and rapid progress. During Q1, we made meaningful advancements at Catalent, Indiana, and our second fill/finish facility. With our ongoing open communication with the agency, we resubmitted our apitegromab BLA in late March in complete alignment with the FDA to include both facilities. This approach underscores the shared understanding between the FDA and Scholar Rock of the unmet need in the SMA community and the shared urgency to bring apitegromab to children and adults in the US as quickly as possible. I would like to now provide an update on the status of each of these two sites.

Speaker #1: During the first quarter, we made meaningful advancements at Catalin, Indiana, and our second fill-finish facility. And with our ongoing open communication with the agency, we resubmitted our Pittogram app BLA in late March in complete alignment with the FDA to include both facilities.

Speaker #1: This approach underscores the shared understanding between the FDA and Scholar Rock of the unmet need in the SMA community and the shared urgency to bring a pittogram app to children and adults in the US as quickly as possible.

David Hallal: This approach underscores the shared understanding between the FDA and Scholar Rock of the unmet need in the SMA community and the shared urgency to bring apitegromab to children and adults in the US as quickly as possible. I would like to now provide an update on the status of each of these two sites.

Speaker #1: I would like to now provide an update on the status of each of these two sites. As it relates to Catalin, Indiana, we are pleased that following acceptance of our BLA, the FDA completed an unannounced reinspection of the facility this timing was in line with our expectations as the FDA had noted following multiple engagements with Novo in Q1 that they would conduct an unannounced inspection following routine manufacturing activities which resumed in late February.

David Hallal: As it relates to Catalent, Indiana, we are pleased that following acceptance of our BLA, the FDA completed an unannounced re-inspection of the facility. This timing was in line with our expectations, as the FDA had noted following multiple engagements with Novo in Q1 that they would conduct an unannounced inspection following routine manufacturing activities which resumed in late February. We are pleased that the inspection was completed in early Q2, and in accordance with FDA guidelines, the agency has up to 90 days to classify the facility. As it relates to the second fill/finish facility, we continue to be pleased with our ongoing meaningful progress. Importantly, the entirety of the apitegromab drug product required for FDA review and potential approval has been filed.

Speaker #1: We are pleased that the inspection was completed in early Q2, and in accordance with FDA guidelines, the agency has up to 90 days to classify the facility.

David Hallal: We are pleased that the inspection was completed in early Q2, and in accordance with FDA guidelines, the agency has up to 90 days to classify the facility. As it relates to the second fill/finish facility, we continue to be pleased with our ongoing meaningful progress. Importantly, the entirety of the apitegromab drug product required for FDA review and potential approval has been filed.

Speaker #1: As it relates to the second fill-finish facility, we continue to be pleased with our ongoing meaningful progress. Importantly, the entirety of the pittogram app drug product required for FDA review and potential approval has been vialed.

Speaker #1: From a commercial supply standpoint, we are well positioned as we expect to have ample commercial pittogram app available from the second facility in early Q3 well ahead of the September PDUFA date.

David Hallal: From a commercial supply standpoint, we are well-positioned as we expect to have ample commercial apitegromab available from the second facility in early Q3, well ahead of the September PDUFA date. We remain committed to the SMA community, and we are grateful that significant progress continues to be made at a rapid pace. Our US commercial team continues to advance the critical activities and capabilities required to deliver a seamless launch and support patients from day one. Importantly, the team stands ready to launch apitegromab immediately upon approval at any time prior to and including the September 30th PDUFA date. In addition to the US, we continue to look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well, and we expect a CHMP opinion near mid-year.

Speaker #1: We remain committed to the SMA community and we are grateful that significant progress continues to be made at a rapid pace. Our US commercial team continues to advance the critical activities and capabilities required to deliver a seamless launch and support patients from day one.

David Hallal: Importantly, the team stands ready to launch apitegromab immediately upon approval at any time prior to and including the September 30th PDUFA date. In addition to the US, we continue to look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well, and we expect a CHMP opinion near mid-year.

Speaker #1: Importantly, the team stands ready to launch a pittogram app immediately upon approval at any time prior to, and including, the September 30 PDUFA date.

Speaker #1: In addition to the US, we continue to look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well.

Speaker #1: And we expect a CHMP opinion near mid-year. We are building momentum with launch readiness activities and we continue to anticipate a launch in the second half of the year beginning with Germany.

David Hallal: We are building momentum with launch readiness activities, and we continue to anticipate a launch in H2, beginning with Germany. We know it is not a matter of if, but when apitegromab will be approved for children and adults with SMA, and Keith Woods will discuss the continued progress we are making with commercial preparations and our disease awareness initiatives shortly. We continue to advance our world-leading anti-myostatin pipeline with enrollment in our phase II OPAL study evaluating apitegromab in infants and toddlers with SMA, the anticipated initiation of our randomized phase II study in patients with FSHD, and progress with subcutaneous apitegromab and a novel high-potency anti-myostatin antibody, SRK-439, currently in phase I. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet.

Speaker #1: We know it is not a matter of if but when a pittogram app will be approved for children and adults with SMA and Keith will discuss the continued progress we are making with commercial preparations and our disease awareness initiatives shortly.

Speaker #1: We continue to advance our world-leading anti-myostatin pipeline with enrollment in our phase two OPAL study evaluating a pittogram app in infants and toddlers with SMA.

David Hallal: We continue to advance our world-leading anti-myostatin pipeline with enrollment in our phase II OPAL study evaluating apitegromab in infants and toddlers with SMA, the anticipated initiation of our randomized phase II study in patients with FSHD, and progress with subcutaneous apitegromab and a novel high-potency anti-myostatin antibody, SRK-439, currently in phase I. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet.

Speaker #1: The anticipated initiation of our randomized phase two study in patients with FSHD and progress with subcutaneous pittogram app and a novel high-potency anti-myostatin antibody SRK439 currently in phase one.

Speaker #1: Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet. We were pleased to have ended the first quarter of 2026 with $480 million in cash, cash equivalent, and marketable securities.

David Hallal: We were pleased to have ended Q1 2026 with $480 million in cash equivalents, and marketable securities. This cash balance includes the drawdown of an additional $100 million from our debt facility, which we took in March. Our cash balance also reflects net cash proceeds of $98 million from our ATM program during the quarter. Vikas will provide more details later in the call. We are building on a solid foundation for our company's growth, which we believe will be steady and consistent through the end of this decade and well into the next as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN-targeted therapy. Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases.

Speaker #1: This cash balance includes the drawdown of an additional $100 million from our debt facility which we took in March. Our cash balance also reflects net cash proceeds of $98 million from our ATM program during the quarter.

Speaker #1: The cost will provide more detail later in the call. We are building on a solid foundation for our company's growth which we believe will be steady and consistent through the end of this decade and well into the next, as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN targeted therapy.

David Hallal: We are building on a solid foundation for our company's growth, which we believe will be steady and consistent through the end of this decade and well into the next as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN-targeted therapy. Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases. With that, I'll now turn the call over to Akshay. Akshay?

Speaker #1: Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases. And with that, I'll now turn the call over to Akshay.

David Hallal: With that, I'll now turn the call over to Akshay. Akshay?

Speaker #1: Akshay, thanks, David, and good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during the first quarter. Turning first to a pittogram app for children and adults with SMA, we're delighted to share that the FDA has accepted the pittogram app BLA.

Akshay Vaishnaw: Thanks, David. Good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during Q1. Turning first to apitegromab for children and adults with SMA, we're delighted to share that the FDA has accepted the apitegromab BLA. As a reminder, the BLA was resubmitted in alignment with the agency to include both Catalent Indiana and a second US-based fill finish facility. The approach provides Scholar Rock with 2 independent paths to apitegromab approval by the PDUFA action date of 30 September. 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 and the current acceptance of the BLA.

Speaker #1: As a reminder, the BLA was resubmitted in alignment with the agency to include both Catalin, Indiana, and a second US-based fill-finish facility. The approach provides Scholar Rock with two independent paths to a pittogram app approval by the PDUFA action date of September 30th.

Akshay Vaishnaw: The approach provides Scholar Rock with 2 independent paths to apitegromab approval by the PDUFA action date of 30 September. 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 and the current acceptance of the BLA.

Speaker #1: 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 and the current acceptance of the BLA.

Speaker #1: Throughout the agency has appreciated the high unmet need in the SMA community and we now look forward to the final steps in the US regulatory process.

Akshay Vaishnaw: Throughout, the agency has appreciated the higher net need in the SMA community, and we now look forward to the final steps in the US regulatory process. Reflecting the agency's vigorous efforts, we were pleased most recently with the timing of the FDA's unannounced re-inspection of Catalent Indiana. Per FDA guidelines, the agency now has up to 90 days to classify the status of the facility. I'd now like to turn to our second fill finish facility, where we continue to make meaningful progress. As David noted, the apitegromab drug product required for FDA data review and potential approval has been filed, and we expect to have ample commercial apitegromab from the facility in early Q3 ahead of the September PDUFA date.

Speaker #1: Reflecting the agency's vigorous efforts, we were pleased most recently with the timing of the FDA's unannounced reinspection of Catalin, Indiana. Per FDA guidelines, the agency now has up to 90 days to classify the status of the facility.

Speaker #1: I'd now like to turn to our second fill-finish facility where we continue to make meaningful progress. As David noted, the pittogram app drug product required for FDA data review and potential approval has been vialed.

Akshay Vaishnaw: I'd now like to turn to our second fill finish facility, where we continue to make meaningful progress. As David noted, the apitegromab drug product required for FDA data review and potential approval has been filed, and we expect to have ample commercial apitegromab from the facility in early Q3 ahead of the September PDUFA date.

Speaker #1: And we expect to have ample commercial pittogram app from the facility in early Q3 ahead of the September PDUFA date. Based on the significant progress at both facilities, we anticipate approval of a pittogram app for children and adults with SMA which could be supported by either or both facilities by the end of the third quarter.

Akshay Vaishnaw: Based on the significant progress at both facilities, we anticipate approval of apitegromab for children and adults with SMA, which could be supported by either or both facilities by the end of Q3. Turning now to Europe, our MAA for apitegromab for treatment of children and adults with SMA continues to progress well through EMA review. As evidence of the progress, we had planned to be with the EMA recently for an oral explanation meeting. Because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary. As we highlighted previously, approval in Europe also requires FDA clearance for Catalent Indiana facility. Based on our discussions with the EMA, they're aware of the progress at Catalent Indiana and are comfortable with the review timeline that accounts for the FDA's classification of the site.

Speaker #1: Turning now to Europe, our MAA for a pittogram app for treatment of children and adults with SMA continues to progress well through EMA review.

Speaker #1: As evidence of the progress, we have planned to be with the EMA recently for an oral explanation meeting. However, because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary.

Akshay Vaishnaw: As evidence of the progress, we had planned to be with the EMA recently for an oral explanation meeting. Because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary. As we highlighted previously, approval in Europe also requires FDA clearance for Catalent Indiana facility.

Speaker #1: As we highlighted previously, approval in Europe also requires FDA clearance for Catalin, Indiana, facility. Based on our discussions with the MA, they're aware of the progress at Catalin, Indiana, and are comfortable with a review timeline that accounts for the FDA's classification for site.

Akshay Vaishnaw: Based on our discussions with the EMA, they're aware of the progress at Catalent Indiana and are comfortable with the review timeline that accounts for the FDA's classification of the site.

Speaker #1: We continue to be very pleased with how the review is progressing, and we anticipate a CHMP opinion near the middle of the year. Turning to our pipeline, let me start with the Phase 2 OPAL trial.

Akshay Vaishnaw: We continue to be very pleased with how the review is progressing. We anticipate a CHMP opinion near the middle of the year. Turning to our pipeline, let me start with the phase II OPAL trial. We continue to enroll and dose patients in this study, which is evaluating apitegromab in infants and toddlers under the age of 2. 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 therapy. 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. In addition, we believe early intervention with apitegromab could support muscle during a critical early development phase, potentially improving motor outcomes in the youngest of patients with SMA.

Speaker #1: We continue to enroll in those patients in this study which is evaluating a pittogram app in infants and toddlers under the age of two.

Speaker #1: 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 therapy.

Speaker #1: This study is important because it is anticipated to expand the impact of the pittogram app to the full spectrum of patients, including those treated with Zolgensa.

Akshay Vaishnaw: 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. In addition, we believe early intervention with apitegromab could support muscle during a critical early development phase, potentially improving motor outcomes in the youngest of patients with SMA.

Speaker #1: In addition, we believe early intervention with a pittogram app could support muscle during a critical early development phase, potentially improving motor outcomes in the youngest of patients with SMA.

Speaker #1: Turning now to our next indication for a pittogram app, fascia scapulohumeral muscular dystrophy or FSHD. FSHD is a rare, devastating neuromuscular disease with significant unmet need.

Akshay Vaishnaw: Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy, or FSHD. FSHD is a rare, devastating neuromuscular disease with significant unmet need. More than 30,000 patients are diagnosed in the US and Europe alone, and there are no approved therapies. We prioritized FSHD as the next indication for apitegromab for 3 key reasons. First, the significant unmet need. Second, the compelling preclinical data from the gold standard FLExD mouse or mouse model that provides mechanistic rationale for apitegromab in FSHD. Finally, as shown on slide 11, 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.

Speaker #1: More than 30,000 patients are diagnosed in the US in Europe alone and there are no approved therapies. We prioritized FSHD as the next indication for a pittogram app for three key reasons.

Speaker #1: First, the significant unmet need. Second, the compelling pre-clinical data from the gold standard FlexTest for mouse model that provides mechanistic rationale for a pittogram app in FSHD and finally, as shown on slide 11, data from randomized studies in FSHD which suggest muscle mass can increase and has the capacity to show functional benefit.

Akshay Vaishnaw: First, the significant unmet need. Second, the compelling preclinical data from the gold standard FLExD mouse or mouse model that provides mechanistic rationale for apitegromab in FSHD. Finally, as shown on slide 11, 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.

Speaker #1: 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 #1: These data suggest that a pittogram app as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased with the progress of activities to support the initiation of our phase two study called FORGE in the middle of this year.

Akshay Vaishnaw: These data suggest that apitegromab as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased with the progress of activities to support the initiation of our Phase II study called FORGE in the middle of this year. Enrollment will commence soon in this randomized, double-blind, placebo-controlled trial, which has a sample size of 60 patients. We're also advancing two additional programs in our world-leading anti-myostatin pipeline: a subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data from a Phase I study in January, which demonstrated that subcu apitegromab appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration.

Speaker #1: Enrollment will commence soon in this randomized double-blind placebo-controlled trial which has a sample size of 60 patients. We're also advancing two additional programs in our world-leading anti-myostatin pipeline.

Akshay Vaishnaw: We're also advancing two additional programs in our world-leading anti-myostatin pipeline: a subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data from a Phase I study in January, which demonstrated that subcu apitegromab appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration.

Speaker #1: A subcutaneous formulation of a pittogram app and SRK439. In our subcutaneous pittogram app program, we showed some very exciting data from a phase one study in January, which demonstrated that subcu pittogram app appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration.

Speaker #1: Additional development activities are ongoing and we continue to plan for engagements with US and European regulators later this year following approval of a pittogram app.

Akshay Vaishnaw: Additional development activities are ongoing, we continue to plan for engagements 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, which we discovered by leveraging our world-leading expertise. We're very excited about this program, dosing in our phase I healthy volunteer study is progressing well. We expect to have top-line data from this study later this year. In closing, we're executing with urgency to bring apitegromab to children and adults with SMA, while in parallel working to maximize our impact for patients with apitegromab and our world-leading anti-myostatin pipeline across a range of rare and devastating neuromuscular diseases. With that, I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Speaker #1: Turning now to SRK439, our high-potency, high-affinity subcutaneously administered myostatin inhibitor, which we discovered by leveraging our world-leading expertise. We're very excited about this program and dosing in our phase one healthy volunteer study is progressing well.

Speaker #1: We expect to have top-line data from the study later this year. In closing, we're executing with urgency to bring a pittogram app to children and adults with SMA whilst in parallel working to maximize our impact for patients with a pittogram app and our world-leading anti-myostatin pipeline across a range of rare, devastating neuromuscular diseases.

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

Speaker #1: With that, I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Speaker #2: Thanks, Akshay. And good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency, as we prepare for the launch of a pittogram app immediately upon approval, which may be granted at any time through September 30th, 2026.

R. Keith Woods: Thanks, Akshay. Good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency as we prepare for the launch of apitegromab immediately upon approval, which may be granted at any time through 30 September 2026. Nearly a decade after the introduction of SMN-targeted therapies, muscle strength and motor function remain the top unmet need, with 95% of patients continuing to experience persistent and progressive muscle atrophy that limits 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 2 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.

Keith Woods: Thanks, Akshay. Good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency as we prepare for the launch of apitegromab immediately upon approval, which may be granted at any time through 30 September 2026. Nearly a decade after the introduction of SMN-targeted therapies, muscle strength and motor function remain the top unmet need, with 95% of patients continuing to experience persistent and progressive muscle atrophy that limits function and independence.

Speaker #2: Nearly a decade after the introduction of SMN targeted therapies, muscle strength and motor function remain the top unmet need, with 95% of patients continuing to experience persistent and progressive muscle atrophy.

Speaker #2: That limits 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.

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 2 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.

Speaker #2: This data again underscores the significant opportunity we have with apitegromab, the world's first muscle-targeted therapy. Our US customer-facing team continues to make significant progress in the field, with disease education, awareness around the unmet medical need, and reinforcing a broader understanding of SMA as a disease which consists of both the motor neuron and the muscle, the principal organ impacted by the disease.

R. Keith Woods: Our US customer-facing team continues to make significant progress in the field with disease education, awareness around the unmet medical need, and reinforcing a broader understanding of SMA as a disease which consists of both the motor neuron and the muscle, the principal organ impacted by the disease. In the US, we have achieved significant reach across the approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams. Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well-positioned to support the SMA treatment centers once a treatment decision is made. This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers, and providers.

Keith Woods: Our US customer-facing team continues to make significant progress in the field with disease education, awareness around the unmet medical need, and reinforcing a broader understanding of SMA as a disease which consists of both the motor neuron and the muscle, the principal organ impacted by the disease. In the US, we have achieved significant reach across the approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams.

Speaker #2: In the US, we have achieved significant reach across the approximately 140 SMA treatment centers 2,600 prescribing physicians and their multidisciplinary care teams. Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well positioned to support the SMA treatment centers once a treatment decision is made.

Keith Woods: Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well-positioned to support the SMA treatment centers once a treatment decision is made. This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers, and providers.

Speaker #2: This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers, and providers.

Speaker #2: In the first quarter, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During the meeting, our team further engaged with healthcare professionals. As one example, we hosted a very well-attended industry forum called 'Going Beyond the Motor Neuron to the Muscle: Expanding the Focus of SMA Care.'

R. Keith Woods: In Q1, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with healthcare professionals. As one example, we hosted a very well-attended industry forum called Going Beyond the Motor Neuron to the Muscle: Expanding the Focus of SMA Care. We also remain highly focused on patients and community activation. We are building on our disease awareness campaign called Life Takes Muscle, and we continue to have numerous in-person patient and patient advocacy group engagements. Turning to US reimbursement, our market access team is advancing discussions with national and key regional payers, as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers. We are ready and well-positioned for a successful launch of apitegromab in the US immediately upon approval.

Keith Woods: In Q1, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with healthcare professionals. As one example, we hosted a very well-attended industry forum called Going Beyond the Motor Neuron to the Muscle: Expanding the Focus of SMA Care. We also remain highly focused on patients and community activation. We are building on our disease awareness campaign called Life Takes Muscle, and we continue to have numerous in-person patient and patient advocacy group engagements.

Speaker #2: We also remain highly focused on patients and community activation. We are building on our disease awareness campaign called Life Takes Muscle, and we continue to have numerous in-person patient and patient advocacy group engagements.

Speaker #2: Turning to US reimbursement, our market access team is advancing discussions with national and key regional payers as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers.

Keith Woods: Turning to US reimbursement, our market access team is advancing discussions with national and key regional payers, as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers. We are ready and well-positioned for a successful launch of apitegromab in the US immediately upon approval.

Speaker #2: We are ready and well positioned for a successful launch of a pittogram app in the US immediately upon approval. Scholar Rock is also making significant progress in Europe.

R. Keith Woods: Scholar Rock is also making significant progress in Europe. We have established our European headquarters in Switzerland. Also, in Germany, where we expect to launch apitegromab upon EMA approval, our local leadership is on board, we have hired our medical and commercial field teams, and we are actively enrolling patients in our compassionate use program. We are making meaningful progress with reimbursement planning to enable rapid patient access. In the broader region, we are advancing reimbursement dossiers in multiple countries, strengthening our distributor relationships, and building our EMEA infrastructure to support future commercialization. Additionally, we had a significant presence at the SMA Europe meeting in March in Budapest. Among other high-impact activities, we hosted an SMA disease education workshop and a healthcare professional symposium where the attendance reflected a high interest in further understanding SMA and the unmet needs in this disease.

Keith Woods: Scholar Rock is also making significant progress in Europe. We have established our European headquarters in Switzerland. Also, in Germany, where we expect to launch apitegromab upon EMA approval, our local leadership is on board, we have hired our medical and commercial field teams, and we are actively enrolling patients in our compassionate use program. We are making meaningful progress with reimbursement planning to enable rapid patient access.

Speaker #2: We have established our European headquarters in Switzerland, also in Germany, where we expect to launch a pittogram app upon EMA approval, our local leadership is on board, we have hired our medical and commercial field teams, and we are actively enrolling patients in our compassionate use program.

Speaker #2: We are making meaningful progress with reimbursement planning to enable rapid patient access. In the broader region, we are advancing reimbursement dossiers in multiple countries.

Keith Woods: In the broader region, we are advancing reimbursement dossiers in multiple countries, strengthening our distributor relationships, and building our EMEA infrastructure to support future commercialization. Additionally, we had a significant presence at the SMA Europe meeting in March in Budapest. Among other high-impact activities, we hosted an SMA disease education workshop and a healthcare professional symposium where the attendance reflected a high interest in further understanding SMA and the unmet needs in this disease.

Speaker #2: Strengthening our distributor relationships and building our EMEA infrastructure to support future commercialization. Additionally, we had a significant presence at the SMA Europe meeting in March in Budapest.

Speaker #2: Among other high-impact activities, we hosted an SMA disease education workshop and a healthcare professional symposium where the attendance reflected a high interest in further understanding SMA and the unmet needs in this disease.

Speaker #2: In closing, we are investing with discipline, to build the commercial foundation necessary to support a world-class launch and to achieve our long-term ambition to bring a pittogram app to the estimated 35,000 patients living with SMA around the world who have received at least one SMN targeted therapy.

R. Keith Woods: In closing, we are investing with discipline to build the commercial foundation necessary to support a world-class launch and to achieve our long-term ambition to bring apitegromab to the estimated 35,000 patients living with SMA around the world who have received at least one 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. With that, I'll turn the call over to Vikas. Vikas?

Keith Woods: In closing, we are investing with discipline to build the commercial foundation necessary to support a world-class launch and to achieve our long-term ambition to bring apitegromab to the estimated 35,000 patients living with SMA around the world who have received at least one 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. With that, I'll turn the call over to Vikas. Vikas?

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.

Speaker #2: With that, I'll turn the call over to Vikas. Vikas, thank you, Keith.

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 upadacimab launch.

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 upadacimab launch. Funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space. Continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I am pleased to provide our Q1 financial results.

Speaker #3: As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial bills to deliver a strong epiduromed launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space.

Vikas Sinha: Funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space. Continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I am pleased to provide our Q1 financial results. For Q1, we reported $102 million in operating expenses, which included $80 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $84 million. Turning to our balance sheet, we are very pleased to have ended Q1 with $480 million in cash equivalents, and marketable securities. During the quarter, we strengthened our cash position with the drawdown of an additional $100 million from our existing debt facility, which we took in March. We also had net cash proceeds of $98 million from our ATM program during Q1.

Speaker #3: And continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I'm pleased to provide our first quarter financial results.

Speaker #3: For the first quarter, we reported 102 million in operating expenses. Which included 18 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were 84 million dollars.

Vikas Sinha: For Q1, we reported $102 million in operating expenses, which included $80 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $84 million. Turning to our balance sheet, we are very pleased to have ended Q1 with $480 million in cash equivalents, and marketable securities. During the quarter, we strengthened our cash position with the drawdown of an additional $100 million from our existing debt facility, which we took in March. We also had net cash proceeds of $98 million from our ATM program during Q1.

Speaker #3: Turning to our balance sheet, we are very pleased to have ended the first quarter with 480 million in cash, cash equivalents, and marketable securities.

Speaker #3: During the quarter, we strengthened our cash position with the drawdown of an additional $100 million from our existing debt facility, which we took in March.

Speaker #3: We also had net cash receipts of 98 million from our ATM program during the first quarter. Looking ahead, upon FDA approval of epiduromed, 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: Looking ahead, upon FDA approval of apitegromab, we will have an option to draw down an additional $150 million from our existing debt facility. We plan to monetize our priority review voucher to further strengthen our balance sheet. We continue to operate with a tight financial plan. Our prioritized investments remain focused on our apitegromab commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and our anticipated growing global commercial demand for apitegromab over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I will turn the call back to David. David?

Speaker #3: We continue to operate with a tight financial plan and our prioritized investments remain focused on our epiduromed commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and our anticipated growing global commercial demand for epiduromed over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call.

Vikas Sinha: Our prioritized investments remain focused on our apitegromab commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and our anticipated growing global commercial demand for apitegromab over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I will turn the call back to David. David?

Speaker #3: With that, I will turn the call back to David. David?

Speaker #2: Thanks, Vikas. Scholar Rock is poised for a transformative year in 2026. Our priorities are clear, and we are executing with focus, discipline, and urgency as we seek to deliver the world's first muscle-targeted therapy to children and adults living with SMAs.

David Hallal: Thanks, Vikas. Scholar Rock is poised for a transformative year in 2026. Our priorities are clear, and we are executing with focus, discipline, and urgency as we seek to deliver the world's first muscle-targeted therapy to children and adults living with SMA, while also laying the foundation to realize our ambition to develop life-transforming therapies for patients with additional rare and severe neuromuscular diseases globally. We are ready, now more than ever, to usher in the next phase of innovation for the SMA community, and we look forward to updating you on our continued progress. With that, we will now open the line for questions. Operator?

Speaker #2: While also laying the foundation to realize our ambition to develop life-transforming therapies for patients with additional rare and severe neuromuscular diseases globally. We are ready.

David Hallal: We are ready, now more than ever, to usher in the next phase of innovation for the SMA community, and we look forward to updating you on our continued progress. With that, we will now open the line for questions. Operator?

Speaker #2: Now, more than ever, to usher in the next phase of innovation for the SMA community and we look forward to updating you on our continued progress.

Speaker #2: And with that, we'll now open the line for questions. Operator?

Speaker #4: Thank you. As a reminder to ask a question, please press star 11 on your telephone and wait for your name to be announced. And to withdraw your question, please press star 11 again.

Operator: Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. The first question comes from Eric Schmidt with Cantor Fitzgerald. Your line's open.

Speaker #4: And the first question comes from Eric Schmidt with Canner. Your line's open.

Eric Schmidt: Thank you, and congrats on all the progress. Maybe just a couple quick questions on apitegromab approval timelines in the US. Team, I know it's not your facility, the Catalent facility, but are you aware of any field notes that were provided to Novo following the re-inspection? I guess I'm also curious about the statement that you reiterated a couple times now that approval may come at any time. I know that probably reflects the shared understanding and communication you have with the FDA, but just curious about the intent of that statement. Thank you.

Speaker #5: Thank you, and congrats on all the progress. Maybe just a couple of quick questions on epiduromed approval timelines in the US. Team, I know it's not your facility—it's the Catalent facility—but are you aware of any field notes that were provided to Novo following the re-inspection? And then I guess I'm also curious about the statement that you've reiterated a couple of times now, that approval may come at any time.

Eric Schmidt: I guess I'm also curious about the statement that you reiterated a couple times now that approval may come at any time. I know that probably reflects the shared understanding and communication you have with the FDA, but just curious about the intent of that statement. Thank you.

Speaker #5: I know that probably reflects the shared understanding and communication you have with the FDA, but just curious about the intent of that statement. Thank you.

Speaker #2: Oh, thanks, Eric. I'll take both and look, we were obviously very pleased today to have announced that the FDA accepted the BLA with two fill-finish facilities and to be clear, it was a Class II resubmission with a PDUFA action date of September 30th, which is sort of per protocol for manufacturing-related issues.

David Hallal: Thanks, Eric. I'll take both. Look, we were obviously very pleased today to have announced that the FDA accepted the BLA with two fill finish facilities. To be clear, it was a Class 2 resubmission with a PDUFA action date of 30 September, which is, you know, sort of per protocol for manufacturing-related issues. We anticipated that. Of course, as a reminder, you know, we submitted that BLA in complete alignment with the FDA ahead of the re-inspection of Catalent Indiana commencing.

Speaker #2: So we anticipated that. And, of course, as a reminder, we submitted that BLA in complete alignment with the FDA ahead of the reinspection of Catalent Indiana commencing.

David Hallal: Look, like since that in-person Type A meeting that we had back in November, all the way through the Type C meeting that we had in early March, we have just been really pleased with the high level of engagement from the agency and the sort of the consistent pace and progress across this, you know, this period of time. Look, what I would note about the re-inspection is we were pleased with the timing. We think the FDA has done their job. We believe that Novo has done their job. Per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility.

Speaker #2: Look, since that in-person type A meeting that we had back in November, all the way through the type C meeting that we had in early March, we have just been really pleased with the high level of engagement from the agency and the sort of the consistent pace and progress across this period of time.

Speaker #2: So, look, what I would note about the reinspection is we were pleased with the timing. We think the FDA has done their job. We believe that Novo has done their job.

David Hallal: Look, what I would note about the re-inspection is we were pleased with the timing. We think the FDA has done their job. We believe that Novo has done their job. Per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility.

Speaker #2: And per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility.

Speaker #2: And I think, again, underscoring sort of the two paths to approval, I am gratified that our team has made massive amounts of progress with our second fill-finish facility as noted today.

David Hallal: I think, again, underscoring sort of the two paths to approval, I am gratified that our team has made massive amounts of progress with our 2nd fill finish facility. As noted today, all of the drug that is required for the FDA's review in this BLA at that 2nd facility has been filed. And that product would be available in early Q3. What you kind of see here, Eric, when we talk about, you know, we have to be ready at any time prior to and including 30 September, is that let's just do a little bit of math together. The FDA is now in a 90-day period of time to, you know, determine classification of Catalent Indiana. We have product that's going to be available in early Q3 from the 2nd fill finish.

Speaker #2: All of the drug that is required for the FDA's review in this BLA at that second facility has been vialed. And that product would be available in early Q3.

Speaker #2: So what you kind of see here, Eric, when we talk about we have to be ready at any time prior to and including September 30th is that let's just do a little bit of math together.

David Hallal: What you kind of see here, Eric, when we talk about, you know, we have to be ready at any time prior to and including 30 September, is that let's just do a little bit of math together. The FDA is now in a 90-day period of time to, you know, determine classification of Catalent Indiana. We have product that's going to be available in early Q3 from the 2nd fill finish.

Speaker #2: The FDA is now in a 90-day period of time to determine classification of Catalent Indiana. We have product that's going to be available in early Q3 from the second fill-finish.

Speaker #2: That sort of brings you to something that is well advanced from the September 30th PDUFA date. And so we just know that we need to be prepared because many times Class II resubmissions and action can be taken by the agency well ahead of that PDUFA date.

David Hallal: That sort of brings you to something that is well advanced from the 30 September PDUFA date. We just know that we need to be prepared because many times Class 2 resubmissions and action can be taken by the agency well ahead of that PDUFA date. That's really what we mean about at any time prior to. We'll continue to work with the FDA collaboratively, and we continue to be, you know, really excited, you know, with their level of engagement, again, as I noted from our Type A meeting right through this moment today. We'll keep you guys apprised on that progress.

Speaker #2: And that's really what we mean about at any time prior to. We'll continue to work with the FDA, collaboratively, and we continue to be really excited with their level of engagement, again, as I noted, from our type A meeting right through this moment today.

David Hallal: We'll continue to work with the FDA collaboratively, and we continue to be, you know, really excited, you know, with their level of engagement, again, as I noted from our Type A meeting right through this moment today. We'll keep you guys apprised on that progress.

Speaker #2: And we'll keep you guys apprised on that progress.

Speaker #5: Extremely helpful. Thank you.

Eric Schmidt: Extremely helpful. Thank you.

Operator: Thank you. We do ask if you can please limit to one question. The next question will come from Mani Foroohar with Leerink Partners. Your line is open.

Speaker #4: Thank you. And we do ask if you can please limit to one question in the next question. We'll come from Manny. So Roha with Lyric Partners, your line is open.

Lili Nsongo: Hi. Good morning. This is Lili Nsongo on for Mani. Thank you for taking my question, and congratulations on the progress. Now that the reinspection has occurred for the Catalent facility, how much risk or maybe I should say, how much confidence do you have in a successful non-MTR indicated classification for the facility? How do we think about the capacity split between the two facility inside the first year launch?

Lili Nsongo [VP, Equity Research: Hi. Good morning. This is Lili Nsongo on for Mani. Thank you for taking my question, and congratulations on the progress. Now that the reinspection has occurred for the Catalent facility, how much risk or maybe I should say, how much confidence do you have in a successful non-MTR indicated classification for the facility? How do we think about the capacity split between the two facility inside the first year launch?

Speaker #6: Hi. Good morning. This is Lirini Songo. I'm from Manny. Thank you for taking a question and congratulations on the progress. So now that the reinspection has occurred for the Catalent facility, how much risk or maybe I should say how much confidence do you have in a successful non-adjuvant-stated classification for the facility?

Speaker #6: And how should we think about the capacity split between the two facilities inside the first year of launch?

Speaker #2: I didn't get the second part of that question, Lily. The first part, as I noted to Eric, we feel like through this process, really, since the sole approvability issue with our initial file was the general site inspection, that the FDA had a Catalent Indiana we know that Novo has been working really hard on that site.

David Hallal: I didn't get the second part of that question, Lili. On the first part, as I noted to Eric, we feel through this process, really since the sole approvability issue with our initial file was the general site inspection that the FDA had at Catalent, Indiana. We know that Novo's been working really hard on that site with their initial remediation plan and then subsequently their follow-up remediation with the FDA. With a lot of engagement in Q1 with the FDA. As we previously noted, they had an early Q1 meeting that was then followed by a site visit and then subsequently in early Q2, the reinspection.

Speaker #2: With their initial remediation plan and then subsequently their follow-up remediation with the FDA, and with a lot of engagement in Q1 with the FDA as we previously noted, they had an early Q1 meeting that was then followed by a site visit.

David Hallal: With a lot of engagement in Q1 with the FDA. As we previously noted, they had an early Q1 meeting that was then followed by a site visit and then subsequently in early Q2, the reinspection.

Speaker #2: And then subsequently, in early Q2, the reinspection. So I think we just need to respect that the FDA has really worked diligently which we think is a rapid timeline given the situation at Catalent Indiana to reinspect that facility.

David Hallal: I think we just need to respect that, the FDA has, you know, really worked diligently, which we think is a rapid timeline given the situation at Catalent, Indiana, to reinspect that facility. They've done their work, Novo's done their work, and now we wanna be respectful of the time that the FDA will now take to make a classification decision. I think importantly, what Akshay and I were noting today is that we have a lot of drug vials from both facilities. And I think if any one of those two were to be the basis of the approval, Keith is gonna have plenty of product to launch with. I think that's great news.

Speaker #2: They've done their work. Novo has done their work. And now we want to be respectful of the time that the FDA will now take to make a classification decision.

Speaker #2: I think importantly, what Akshay and I were noting today is that we have a lot of drug vials from both facilities. And I think if any one of those two were to be the basis of the approval, Keith is going to have plenty of product to launch with.

David Hallal: I think importantly, what Akshay and I were noting today is that we have a lot of drug vials from both facilities. And I think if any one of those two were to be the basis of the approval, Keith is gonna have plenty of product to launch with. I think that's great news.

Speaker #2: So I think that's great news. I think one thing that maybe isn't lost on us is when you take a 90-day timeline for by up to a 90-day timeline for the FDA to reclassify the Catalent Indiana facility, and then you think about an early Q3 timing of having product available commercially from the second fill-finish, there's definitely an opportunity also that our file could be approved with both fill-finish facilities.

David Hallal: I think one thing that maybe isn't lost on us is when you take a 90-day timeline for, you know, by up to a 90-day timeline for the FDA to reclassify the Catalent, Indiana facility, and then you think about an early Q3 timing of having product available commercially from the second fill finish, there's definitely an opportunity also that our file could be approved with both fill finish facilities. I think that that was one of the things that Akshay and I wanted to communicate as well. A lot of optionality here, a lot of good news for patients, a lot of good news for the SMA community.

Speaker #2: And I think that that was one of the things that Akshay and I wanted to communicate as well. So, a lot of optionality here.

Speaker #2: A lot of good news for patients. A lot of good news for the SMA community. I am really grateful to our internal team at Scholar Rock for doing something pretty remarkable here with our second fill-finish facility but also grateful that the FDA and Novo for the continued progress at Catalent Indiana.

David Hallal: I am really grateful to our internal team at Scholar Rock for doing something pretty remarkable here with our second fill finish facility, but also grateful with the FDA and Novo for the continued progress at Catalent, Indiana. We will keep you guys apprised at the updates across the board on our application.

Speaker #2: And we will keep you guys apprised that the updates across the board on our application.

Speaker #6: Great. Thank you. The second part of my question was about commercial supply capacity split between the two facilities, which you also answered. So thank you.

Lili Nsongo: Great. Thank you. The second part of my question was about commercial supply capacity split between the 2 facilities, which you also answered, so thank you.

Lili Nsongo [VP, Equity Research: Great. Thank you. The second part of my question was about commercial supply capacity split between the 2 facilities, which you also answered, so thank you.

Speaker #2: Thank you, Lily.

David Hallal: Thank you, Lili.

Speaker #4: Thank you. And the next question will come from Tess Romero with JPMorgan. Your line is open.

Operator: Thank you. The next question will come from Tessa Romero with JP Morgan. Your line's open.

Tessa Romero: Hey, guys. Good morning. Thanks so much for taking our question. I actually wanted to ask a commercial question this morning. Now that Aduhelm is fully approved for ages older than 2 years old, how are you thinking about upadacitabine being able to be used in combination with that therapy if and when you are approved? Thank you.

Tess Romero [Senior Equity Research Analyst: Hey, guys. Good morning. Thanks so much for taking our question. I actually wanted to ask a commercial question this morning. Now that Aduhelm is fully approved for ages older than 2 years old, how are you thinking about upadacitabine being able to be used in combination with that therapy if and when you are approved? Thank you.

Speaker #7: Hey, guys. Good morning. Thanks so much for taking our question. I actually wanted to ask a commercial question this morning. Now that Advisma is fully approved for ages older than two years old, how are you thinking about a pentagram AB being able to be used in combination with that therapy if and when you are approved?

Speaker #7: Thank you.

Speaker #2: Yeah. Thank you, Tess. Keith can address how we're thinking about that opportunity as he noted today. Really important information from Cure SMA. In general, we are prepared to launch a pentagram ab at any time between now and up to September 30th.

David Hallal: Yeah. Thank you, Tess. I, you know, Keith can, you know, address how we're thinking about that opportunity. As you noted today, you know, really important information from Cure SMA. In general, we are prepared to launch upadacitabine at any time between, you know, now and up to 30 September. I think the incredible work that Akshay has done with, you know, with our team and engaging the FDA, there's going to be a very significant opportunity to serve patients with SMA. Keith, do you wanna comment on, you know, really more than anything else, the dynamics in the marketplace and your preparations for launch?

Speaker #2: And I think the incredible work that Akshay has done with our team and engaging the FDA that's going to be a very significant opportunity to serve patients with SMA.

David Hallal: I think the incredible work that Akshay has done with, you know, with our team and engaging the FDA, there's going to be a very significant opportunity to serve patients with SMA. Keith, do you wanna comment on, you know, really more than anything else, the dynamics in the marketplace and your preparations for launch?

Speaker #2: So Keith, do you want to comment on really more than anything else the dynamics in the marketplace and your preparations for launch?

Speaker #5: Sure. Thanks for the question, Tess. I guess what I'd say first of all is we believe that regardless of the therapy but any type of a therapy that an SMA patient can potentially benefit from in an SMN-targeted therapy, we're agnostic as to which one the treating physician chooses because we think that they go hand in hand along with our muscle-targeted therapy with a pentagram ab.

R. Keith Woods: Sure. Thanks for the question, Tess. What I'd say first of all is, you know, we believe that, regardless of the therapy, but any type of a therapy that an SMA patient can potentially benefit from in an SMN targeted therapy, we're agnostic as to which one the treating physician choose because we think that they go hand in hand along with our muscle-targeted therapy with apitegromab. Now, specifically, with Zolgensma, you know, in our SAPPHIRE study, we did not study patients that were previously on Zolgensma.

Keith Woods: Sure. Thanks for the question, Tess. What I'd say first of all is, you know, we believe that, regardless of the therapy, but any type of a therapy that an SMA patient can potentially benefit from in an SMN targeted therapy, we're agnostic as to which one the treating physician choose because we think that they go hand in hand along with our muscle-targeted therapy with apitegromab. Now, specifically, with Zolgensma, you know, in our SAPPHIRE study, we did not study patients that were previously on Zolgensma.

Speaker #5: Now, specifically, with Advisma, in our Sapphire study, we did not study patients that were previously on Xeljanzma. As Akshay has noted several times, we are studying them in our OPAL study.

David Hallal: As Akshay's noted several times, we are studying them in our OPAL study, and we've also shared with you that we do have post-Zolgensma patients in our EAP program. There's some experience out there with it. But as far as being able to utilize apitegromab with it, I think it's gonna depend upon the label and where the policies come out with the payers.

Keith Woods: As Akshay's noted several times, we are studying them in our OPAL study, and we've also shared with you that we do have post-Zolgensma patients in our EAP program. There's some experience out there with it. But as far as being able to utilize apitegromab with it, I think it's gonna depend upon the label and where the policies come out with the payers.

Speaker #5: And we've also shared with you that we do have post-Xeljanzma patients in our EAP program. So there's some experience out there with it. But as far as being able to utilize a pentagram ab with it, I think it's going to depend upon the label and where the policies come out with the payers.

Speaker #7: Thank you.

Tessa Romero: Thank you.

Tess Romero [Senior Equity Research Analyst: Thank you.

Speaker #4: Thank you. And our next question will come from Corey Kasimov with Evercore. Your line is open.

Operator: Thank you.

David Hallal: Thank you.

Operator: Our next question will come from Cory Kasimov with Evercore. Your line's open.

Cory Kasimov: Hey, good morning, guys. Thank you for taking the question. I wanted to ask you about the ongoing CHMP review. Coming out of the recent oral explanation, have the questions there been largely similar to what the FDA has inquired about during its review and now just really boils down to CMC? Or are there other non-manufacturing items that EU regulators are still trying to get their arms around? Thank you.

Speaker #8: Hey. Good morning, guys. Thank you for taking the question. I wanted to ask you about the ongoing CHMP review. Coming out of the recent oral explanation, have the questions there been largely similar to what the FDA has inquired about during its review and now just really boils down to CMC?

Speaker #8: Or are there other non-manufacturing items that EU regulators are still trying to get their arms around? Thank you.

Speaker #2: Thanks, Corey. Akshay?

David Hallal: Thanks, Cory. Akshay?

Speaker #5: Yeah. Thanks. So obviously, we don't get into the back and forth of regulatory reviews, FDA or EMA. The one thing I can say is that that oral explanation that was scheduled led to a very good dialogue in advance of the meeting.

Akshay Vaishnaw: Yeah, thanks. Obviously we don't get into the back and forth of regulatory reviews, FDA or EMA. The one thing I can say is that oral explanation that was scheduled led to a very good dialogue in advance of the meeting, and we were very happy with the pre-meeting alignment, which led to mutual agreement that there was no need for the meeting. In fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMA. You know, as to the remaining timeline, I commented in the formal remarks that the Catalent Indiana facility continues to support the application, and we look forward to a decision around mid-year. I think overall the progress has been excellent.

Speaker #5: And we were very happy with the pre-meeting alignment, which led to mutual agreement that there was no need for the meeting. And in fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMA.

Akshay Vaishnaw: In fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMA. You know, as to the remaining timeline, I commented in the formal remarks that the Catalent Indiana facility continues to support the application, and we look forward to a decision around mid-year. I think overall the progress has been excellent.

Speaker #5: As to the remaining timeline, I commented in the formal remarks that the Catalent Indiana facility continues to support the application. And we look forward to decision around mid-year.

Speaker #5: But I think overall, the progress has been excellent.

David Hallal: And, and then just tagging on-

David Hallal: And, and then just tagging on, Cory, just to tag on to Akshay, and Akshay has mentioned this multiple times. We've been having very good open dialogue with the European regulators about what's been happening here with the FDA and Catalent Indiana. It's been very collaborative. Like, everything going on here has, you know, you know, been a topic of discussion in Europe, and they're really been very flexible in working with us on timing.

Speaker #2: And then just taking on Corey, just to tag on to Akshay and Akshay has mentioned this multiple times. We've been having very good open dialogue with the European regulators about what's been happening here with the FDA and Catalent Indiana.

Cory Kasimov: Great, thank you.

Cory Kasimov: Cory, just to tag on to Akshay, and Akshay has mentioned this multiple times. We've been having very good open dialogue with the European regulators about what's been happening here with the FDA and Catalent Indiana. It's been very collaborative. Like, everything going on here has, you know, you know, been a topic of discussion in Europe, and they're really been very flexible in working with us on timing.

Speaker #2: So it's been very collaborative. Everything going on here is been a topic of discussion in Europe and they're really been very flexible in working with us on timing.

Speaker #8: It's very helpful. Appreciate it.

Cory Kasimov: It's very helpful. Appreciate it.

Speaker #4: Thank you. And the next question will come from Michael Yee with UBS. Your line is open.

Operator: Thank you. The next question will come from Michael Yee with UBS. Your line is open.

Speaker #9: Hey, guys. Thank you. Good morning. Two questions. Really quick, one is a follow-up just in terms of the fill-finish facility the second one. Can you remind me previously, I recall there was difference stability testings and things that had to be completed, but it sounds like this site had sort of been pulled very much forward.

Michael Yee: Hey, guys. Thank you. Good morning. Two questions. Really quick, one is a follow-up, just in terms of the fill finish facility, the second one. Can you remind me, previously, I recall there was different stability testings and things that had to be completed, but it sounds like this site had sort of been pulled very much forward and was filed earlier, which was fantastic. Is the understanding that either of these sites can support approval by 30 September, and that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site, and so I have that correct? The second question is regarding a potential approval and indications.

Speaker #9: And was filed earlier, which was fantastic. And so is the understanding that either of these sites can support approval by September 30th? And that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site?

Michael Yee: Is the understanding that either of these sites can support approval by 30 September, and that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site, and so I have that correct? The second question is regarding a potential approval and indications.

Speaker #9: And so I have that correct? And the second question is regarding a potential approval and indications. I know previously there has been some discussion around the broadness of the label type one versus type two and different age groups given the primary endpoint was on a certain age group definition.

Michael Yee: I know previously there has been some discussion around the broadness of the label, type 1 versus type 2, and different age groups, given the primary endpoint was on a certain age group definition. Can you just remind us about your confidence around general broadness of the label and how we should think about that? Thank you.

Speaker #9: Can you just remind us about your confidence around general broadness of the label and how we should think about that? Thank you.

David Hallal: Michael, great questions. I'll start on the, you know, the two fill finish facilities, and then Akshay will take up the label. Yeah, I mean, I guess at the end of the day, you know, we have an enormous amount of confidence in our BLA as the You know, the headline news of what's changed from late last year to this year is really the fact that we have two fill finish facilities in our BLA. One of those we expect reclassification within a 90-day window from the closeout of the inspection.

Speaker #2: Michael, great questions. I'll start on the two fill-finish facilities and then Akshay will take up the label. So yeah. I mean, I guess at the end of the day, we have an enormous amount of confidence in our BLA as the headline news of what's changed from late last year to this year is really the fact that we have two fill-finish facilities in our BLA.

Speaker #2: One of those, we expect reclassification within a 90-day window from the closeout of the inspection. And subsequently, in that second fill-finish facility, as you aptly noted, we have made massive amounts of progress in accelerating that where all of the drug that is required for the FDA's review and approval has been vialed.

David Hallal: Subsequently in that second fill finish facility, as you aptly noted, we have made massive amounts of progress in accelerating that, where all of the drug that is required for the FDA's review and approval has been filed, and that drug would be available commercially in early Q3. When you kinda take that 90-day window, the up to 90-day window per FDA guidelines for the Catalent Indiana facility, when you look at that window of commercial apitegromab being available in early Q3 from the second finish facility, we are very confident in this window that we're talking about within Q3 and up to the 30 September PDUFA.

Speaker #2: And that drug would be available commercially in early Q3. So when you kind of take that 90-day window, the up to 90-day window per FDA guidelines for the Catalent Indiana facility, when you look at that window of commercial a pentagram ab being available in early Q3 from the second-finish facility, we are very confident in this window that we're talking about within Q3 and up to the September 30th PDUFA and I think that what I'm most gratified about is we try to live here at Scholar Rock.

David Hallal: When you kinda take that 90-day window, the up to 90-day window per FDA guidelines for the Catalent Indiana facility, when you look at that window of commercial apitegromab being available in early Q3 from the second finish facility, we are very confident in this window that we're talking about within Q3 and up to the 30 September PDUFA.

David Hallal: I think that, you know, what I'm most gratified about is we try to live here at Scholar Rock, by a deep commitment to the patients and families, you know, that are impacted by SMA. I'm grateful to the team that we took it upon ourselves to say, Okay, let's do better this time than we did last time. Let's not rely on a single fill finish facility. Let's have multiple paths to get to that point where we can deliver the first-ever muscle-targeted therapy to patients, who are living with this disease and the families that are impacted by this disease. I think we've been able to do that.

Speaker #2: By a deep commitment to the patients and families that are impacted by SMA. And I'm grateful to the team that we took it upon ourselves to say, "Okay.

Speaker #2: Let's do better this time than we did last time. Let's not rely on a single fill-finish facility." Let's have multiple paths to get to that point where we can deliver the first-ever monthly targeted therapy to patients who are living with this disease and the families that are impacted by this disease.

David Hallal: Let's not rely on a single fill finish facility. Let's have multiple paths to get to that point where we can deliver the first-ever muscle-targeted therapy to patients, who are living with this disease and the families that are impacted by this disease. I think we've been able to do that.

Speaker #2: And I think we've been able to do that. And tying dovetailing nicely into that is the question that you had on the label and our opportunity to serve a meaningful percentage of the community that is impacted by this disease.

David Hallal: Tying, you know, dovetailing nicely into that is the question that you had on the label and our opportunity to serve a meaningful percentage of the community that is impacted by this disease, and I'll turn it over to Akshay to comment on that. Akshay?

Speaker #2: And I'll turn it over to Akshay to comment on that. Akshay?

Speaker #5: Yeah. Thanks. Mike, vis-à-vis the labels, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here and in Europe.

Akshay Vaishnaw: Yeah. Thanks. Michael, you know, vis-à-vis the label, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here and in Europe. What I would say is that generally speaking, the regulators have taken a very, very important approach to the labels for SMA products. They looked at the enrollment criteria of the pivotal studies, which exactly was the population. They looked at the plausibility of the mechanism across the spectrum of disease, they looked at the unmet need. I feel like, you know, they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, the draft label was completed. The one outstanding issue was the manufacturing issue.

Speaker #5: What I would say is that generally speaking, the regulators have taken a very important approach to the labels for SMA products. They look at the enrollment criteria of the pivotal studies.

Speaker #5: Which exactly was the population? They look at the plausibility of the mechanism across the spectrum of disease. And they look at the unmet need.

Akshay Vaishnaw: They looked at the plausibility of the mechanism across the spectrum of disease, they looked at the unmet need. I feel like, you know, they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, the draft label was completed. The one outstanding issue was the manufacturing issue.

Speaker #5: And so I feel like they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, the draft label was completed.

Speaker #5: The one outstanding issue was the manufacturing issue. And both the US and Europe all I can say is we've had constructive regulatory dialogues throughout the period last year and this year.

Akshay Vaishnaw: Both in US and Europe, all I can say is we've had constructive regulatory dialogues throughout the period last year into this year, and we look forward to launching this product for children and adults with SMA.

Speaker #5: And we look forward to launching this product for children and adults with SMA.

David Hallal: Michael, you know, Akshay Vaishnaw and I would just note that, as we previously had disclosed that, you know, where we were, you know, toward the tail end of our last, you know, BLA review, we were pleased. That's where we picked up this new application. It's exactly where we were at the tail end of the last one on the label, and we look forward to continuing to work with regulators to bring us to the point of approval and delivering upadacimab to the community.

Speaker #2: And Michael, Akshay and I would just note that because we previously had disclosed that where we were toward the tail end of our last BLA review, we were pleased.

Speaker #2: And that's where we picked up this new application. It's exactly where we were at the tail end of the last one on the label, and we look forward to continuing to work with regulators to bring us to the point of approval and delivering apitemgrmab to the community.

David Hallal: It's exactly where we were at the tail end of the last one on the label, and we look forward to continuing to work with regulators to bring us to the point of approval and delivering upadacimab to the community.

Speaker #9: Thank you.

Michael Yee: Thank you.

Speaker #4: Thank you. And the next question is going to come from Kazeen Ahmad with Bank of America. Your line's open.

Operator: Thank you. The next question is going to come from Tazeen Ahmad with Bank of America. Your line's open.

Speaker #10: Hi. Good morning. This is Wesley Hunter for Kazeen. Congrats to the team on all the progress. Really. I had a question sort of the game plan going forward now that you have a PDUFA date in hand.

Wesley: Hi. Good morning. This is Wesley on for Tazeen Ahmad. Congrats to the team on all the progress, really. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. Are there any sort of new types of discussions you can have with payers or other, like, commercial bodies now that apitegromab's officially under review? Is there any sort of, you know, new, I guess, strategies or ways that, you know, R. Keith Woods and the commercial team are sort of laying out the groundwork for potential approval? Or is it just kinda just chugging along and doing what's been done already? Thank you.

Wesley Yon [Equity Research: Hi. Good morning. This is Wesley on for Tazeen Ahmad. Congrats to the team on all the progress, really. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. Are there any sort of new types of discussions you can have with payers or other, like, commercial bodies now that apitegromab's officially under review?

Speaker #10: So are there any sort of new types of discussions you can have with payers or other commercial bodies now that a pentagram ab is officially under review?

Speaker #10: And is there any sort of new, I guess, strategies or ways that Keith and the commercial team are sort of laying out the groundwork for potential approval?

Wesley Yon [Equity Research: Is there any sort of, you know, new, I guess, strategies or ways that, you know, R. Keith Woods and the commercial team are sort of laying out the groundwork for potential approval? Or is it just kinda just chugging along and doing what's been done already? Thank you.

Speaker #10: Or is it just kind of just chugging along and doing what's been done already? Thank you.

David Hallal: Well, well, well Wesley, as I think you guys all know Keith very well, you would imagine, as disappointed as we were, to not launch late last year, we had to look at the opportunity that we had to prepare ourselves to be even better, to serve the SMA community. That was our obligation. One such piece of that under Akshay and under Lisa Wyman and team was to make sure that this application was even stronger than the last one, and that's inclusive of now the two fill finish facilities and two independent paths to approval. The other obligation that we made is to be better, you know, from a commercial perspective. How do you use that time to make sure that you can meet the moment for the SMA community?

Speaker #2: Well, Wesley, as I think you guys all know Keith very well. You would imagine as disappointed as we were to not launch late last year.

Speaker #2: We had to look at the opportunity that we had to prepare ourselves to be even better to serve the SMA community. That was our obligation.

Speaker #2: One such piece of that under Akshay and under Lisa Wyman and team was to make sure that this application was even stronger than the last one.

David Hallal: One such piece of that under Akshay and under Lisa Wyman and team was to make sure that this application was even stronger than the last one, and that's inclusive of now the two fill finish facilities and two independent paths to approval. The other obligation that we made is to be better, you know, from a commercial perspective. How do you use that time to make sure that you can meet the moment for the SMA community?

Speaker #2: And that's inclusive of now the two fill-finish facilities and two independent paths to approval. The other obligation that we made is to be better from a commercial perspective.

Speaker #2: How do you use that time to make sure that you can meet the moment for the SMA community? And I think your question is a good one now with the September 30th PDUFA, but yet being ready for an approval at any time.

David Hallal: I think your question is a good one now with the September 30th PDUFA, but yet being ready for an approval at any time. With that, I'll hand it over to Keith to talk about the things that he has been doing and what this means for him and the team. Keith?

Speaker #2: And with that, I'll hand it over to Keith to talk about the things that he has been doing and what this means for him and the team.

Speaker #2: Keith?

Speaker #5: Yeah. Thanks, David. And Wesley, thanks for the question. What I can tell you is that joining the company four months prior to the PDUFA date we were scrambling for that PDUFA date.

R. Keith Woods: Yeah. Thanks, David. Wesley, thanks for the question. What I can tell you is that joining the company 4 months prior to the PDUFA date, we were scrambling for that PDUFA date. We would've been able to launch successfully, but we have really been able to take advantage of the additional time that we had. Some specific examples that I've shared in the past, first of all, with payers. We are able to meet with the payers and with our medical, with our medical team to really discuss upadacimab and the data. Those discussions are ongoing. We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this.

Keith Woods: Yeah. Thanks, David. Wesley, thanks for the question. What I can tell you is that joining the company 4 months prior to the PDUFA date, we were scrambling for that PDUFA date. We would've been able to launch successfully, but we have really been able to take advantage of the additional time that we had.

Speaker #5: We would have been able to launch successfully, but we have really been able to take advantage of the additional time that we have. Some specific examples that I've shared in the past.

Keith Woods: Some specific examples that I've shared in the past, first of all, with payers. We are able to meet with the payers and with our medical, with our medical team to really discuss upadacimab and the data. Those discussions are ongoing. We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this.

Speaker #5: ongoing. We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this.

R. Keith Woods: Additionally, we built out how our site of care plans will be. I've shared with you before that we now, through our partners, have over 10,000 home infusion nurses available around the US that would be able to provide apitegromab to patients, shall they choose to go through home infusion. We've expanded our specialty pharmacy network so that no patient has to go to multiple specialty pharmacies to get their different meds that they may be on for SMA treatment, whether it's their SMN targeted therapy or that apitegromab. Just we continue to really move forward with patient engagement activities. That's through our program to really have patients demand better treatment for themselves with Life Takes Muscle.

Speaker #5: Additionally, we built out how our site of care plans will be. I've shared with you before that we now, through our partners, have over 10,000 home infusion nurses available around the US that would be able to provide a pentagram ab to patients, shall they choose to go through home infusion.

Keith Woods: Additionally, we built out how our site of care plans will be. I've shared with you before that we now, through our partners, have over 10,000 home infusion nurses available around the US that would be able to provide apitegromab to patients, shall they choose to go through home infusion.

Speaker #5: We've expanded our specialty pharmacy network so that no patient has to go to multiple specialty pharmacies to get their different meds that they may be on for SMA treatment, whether it's their SMN targeted therapy or that of a pentagram ab.

Keith Woods: We've expanded our specialty pharmacy network so that no patient has to go to multiple specialty pharmacies to get their different meds that they may be on for SMA treatment, whether it's their SMN targeted therapy or that apitegromab. Just we continue to really move forward with patient engagement activities. That's through our program to really have patients demand better treatment for themselves with Life Takes Muscle.

Speaker #5: And just we continue to really move forward with patient engagement activities. And that's through our program to really have patients demand better treatment for themselves with life takes muscle.

Speaker #5: And so I can tell you this. I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now.

R. Keith Woods: I can tell you this: I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now. Whatever that timeframe, that will be between now and September 30th, I want you to know that the team will be ready to be out there the next day. We will have supply in the channel very rapidly after approval. Thanks.

Keith Woods: I can tell you this: I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now. Whatever that timeframe, that will be between now and September 30th, I want you to know that the team will be ready to be out there the next day. We will have supply in the channel very rapidly after approval. Thanks.

Speaker #5: So whatever that time frame that will be between now and September 30th, I want you to know that the team will be ready to be out there the next day and we will have supply in the channel very rapidly after approval.

Speaker #5: Thanks.

Speaker #10: Got it. Thank you.

Wesley: Got it. Thank you.

Wesley Yon [Equity Research: Got it. Thank you.

Speaker #4: Thank you. And the next question will be coming from Mark Fromm with TD Cohen.

Operator: Thank you. The next question will be coming from Marc Frahm with TD Cowen. Thanks.

Marc Frahm: Hi. Thanks for taking my questions. A lot's been asked already on the PDUFA and upadacimab itself. Maybe just looking at the subQ version, I mean, you mentioned, you know, you have that data in hand, and once you get the approval for the IV formulation, you know, you'll look to meet with the FDA to discuss it. Just what are the kind of key issues you think you need answers from the FDA on and kind of what are the range of timelines for when you think you might be able to kind of launch that product, depending upon the outcome of those discussions?

Marc Frahm [Equity Research Analyst: Hi. Thanks for taking my questions. A lot's been asked already on the PDUFA and upadacimab itself. Maybe just looking at the subQ version, I mean, you mentioned, you know, you have that data in hand, and once you get the approval for the IV formulation, you know, you'll look to meet with the FDA to discuss it.

Speaker #11: Thanks for taking my questions. A lot's been asked already on the PDUFA and the pentagram itself. Maybe just looking at the sub-Q version, I mean, you mentioned you have that data in hand and once you get the approval for the IV formulation, you'll look to meet with the FDA to discuss it.

Speaker #11: Just what are the kind of key issues you think you need answers from the FDA on and kind of what are the range of timelines for when you think you might be able to kind of launch that product depending upon the outcome of those discussions?

Marc Frahm [Equity Research Analyst: Just what are the kind of key issues you think you need answers from the FDA on and kind of what are the range of timelines for when you think you might be able to kind of launch that product, depending upon the outcome of those discussions?

Speaker #2: Thanks, Mark. Akshay?

David Hallal: Thanks, Marc. Akshay?

Speaker #5: Yeah. Thanks, Mark. So I mean, I would say there are no issues as such. These things are a matter of just alignment with regulators as to what the optimum path forward to bring another innovation to SMA patients and in this case, it would be subcutaneous pentagram ab.

Akshay Vaishnaw: Yeah. Thanks, Marc. You know, I mean, I would say there are no issues as such. These things are a matter of just alignment with regulators as to what the optimum path forward to bring another innovation to SMA patients, and in this case, it would be subcutaneous apitegromab. The phase I data were excellent, showing they have very good bioavailability and pharmacodynamic overlap between the two routes of administration. What we have to do now is to share those data following the approval and align on the path forwards in terms of any further development that we needed. That could be PK/PD data and consideration of any additional safety or efficacy.

Akshay Vaishnaw: The phase I data were excellent, showing they have very good bioavailability and pharmacodynamic overlap between the two routes of administration. What we have to do now is to share those data following the approval and align on the path forwards in terms of any further development that we needed. That could be PK/PD data and consideration of any additional safety or efficacy.

Speaker #5: The phase one data were excellent showing very good bioavailability and pharmacodynamic overlap between the two routes of administration. And what we have to do now is to share those data following the approval and align on the path forwards in terms of any further development that we needed.

Speaker #5: So that could be PKPD data and consideration of any additional safety or efficacy. However, from a safety perspective, and obviously the exposure is maximized with IV a pentagram ab, and so with the very large database we have in hand, already from the studies we've done, we feel very good about safety via additional routes of administration.

Akshay Vaishnaw: However, from a safety perspective, obviously the exposure is maximized with IV epinephrin, with the very large database we have in hand already from the studies we've done, we feel very good about safety via additional routes of administration. We just want to get on and have those conversations and finalize the path. Once we've done that, obviously we'll guide you on the timelines. Premature to speak to that in advance of those conversations. Thanks.

Speaker #5: And so we just want to get on and have those conversations and finalize the path. Once we've done that, obviously we'll guide you on the timelines. Premature to speak to that in advance of those conversations.

Speaker #5: Thanks.

Speaker #11: Okay. Thank you. And if I can squeeze in also just on FORGE trial, just can you kind of walk through what's different about that trial or maybe the supporting data that a pentagram ab's been able to generate relative to the effort that Roche had in FSHD and which ultimately as of a few weeks ago, they disclosed did not lead to moving into pivotal development?

Marc Frahm: Okay. Thank you. If I can squeeze in also on FORGE trial, can you kind of walk through what's different about that trial or maybe the supporting data that apitegromab has been able to generate relative to, you know, the effort that Roche had in FSHD and, you know, which ultimately, as of a few weeks ago, you know, they disclosed did not lead to moving into pivotal development?

Marc Frahm [Equity Research Analyst: Okay. Thank you. If I can squeeze in also on FORGE trial, can you kind of walk through what's different about that trial or maybe the supporting data that apitegromab has been able to generate relative to, you know, the effort that Roche had in FSHD and, you know, which ultimately, as of a few weeks ago, you know, they disclosed did not lead to moving into pivotal development?

Speaker #5: Yeah. Three or four points here. Number one, we're obviously very proud of the innovations that have occurred at Scholar Rock with our leading anti-mice patent pipeline.

Akshay Vaishnaw: Yeah. You know, three or four points here. Number one, we're obviously very proud of the innovations that have occurred at Scholar Rock with our leading anti-myostatin pipeline. It still remains apitegromab, the only validated anti-myostatin antibody that's made it through phase II and has delivered the kind of risk and benefit profile that we saw in the phase III with the SAPPHIRE study in SMA. Now, whilst we await that approval, obviously many others are interested in this target. Roche and Chugai are, you know, world-leading company. It was sad to see that antibody drop out. We've never really seen any phase I data or the FLExD mouse model data from the Chugai Roche antibody. We don't quite know the nature of those data as we await to see how strong they were.

Speaker #5: It still remains a pentagram ab, the only validated anti-mice patent antibody that's made it through phase two and delivered the kind of risk and benefit profile that we saw in the phase three with the sapphire study in SMA.

Speaker #5: Now, whilst we await that approval, obviously many others are interested in this target. Roche and Shugai are world-leading companies. It was sad to see that antibody drop out.

Akshay Vaishnaw: Now, whilst we await that approval, obviously many others are interested in this target. Roche and Chugai are, you know, world-leading company. It was sad to see that antibody drop out. We've never really seen any phase I data or the FLExD mouse model data from the Chugai Roche antibody. We don't quite know the nature of those data as we await to see how strong they were.

Speaker #5: We've never really seen any phase one data or the flex ducts mouse model data from the Shugai Roche antibody so we don't quite know the nature of those data.

Speaker #5: And we await to see how strong they were. We know our data apart from the positive phase three study, of course, we have very nice data in the flex ducts for mouse model with an anti-mice patent approach showing increase in muscle mass and torque and additional function.

Akshay Vaishnaw: We know our data, apart from the positive phase III study, of course, we have very nice data in the FLExD mouse model with an anti-myostatin approach showing increase in muscle mass and torque and an additional function. We know that there are, within FSHD, normal fibers that can be boosted by means of an anti-myostatin approach. We know other clinical trials in FSHD that have shown increase in muscle mass and function. We're very encouraged by our data and our diligence. Finally, vis-a-vis the phase II design, it is different from the Roche study, specifically the inclusion/exclusion criteria and the severity of the disease that we're enrolling relative to what they enrolled, which appeared to be quite advanced.

Speaker #5: We know that there are within FSHD normal fibers that can be boosted by means of an anti-mice patent approach. We know other clinical trials in FSHD that have shown increased in muscle mass and function.

Speaker #5: So we're very encouraged by our data and our diligence. And finally, vis-à-vis the phase two design, it is different from the Roche study. Specifically, the inclusion-exclusion criteria and the severity of the disease that we're enrolling relative to what they enrolled, which appeared to be quite advanced.

Akshay Vaishnaw: We're very encouraged by our data and our diligence. Finally, vis-a-vis the phase II design, it is different from the Roche study, specifically the inclusion/exclusion criteria and the severity of the disease that we're enrolling relative to what they enrolled, which appeared to be quite advanced.

Speaker #5: And based on our diligence with the experts we decided because of input from them to go towards the milder end in terms of the reachy scores with patients with established disease where we felt we could still show benefit and so we remain confident with our validated asset going into that phase two study and look forward to kicking off very soon.

Akshay Vaishnaw: Based on our diligence with the experts, we decided, because of input from them, to go towards the milder end in terms of the Ricci score with patients with established disease where we felt we could still show benefit. You know, we remain confident with our validated asset, going into that phase II study and look forward to kicking off very soon.

Marc Frahm: All right. Thank you. Congrats on the progress.

Marc Frahm [Equity Research Analyst: All right. Thank you. Congrats on the progress.

Speaker #11: All right. Thank you. And congrats on all the progress.

Speaker #5: Thanks.

Akshay Vaishnaw: Thanks.

Speaker #4: Thank you. And the next question will come from Jeff Meacham with Citigroup. Your line's open.

Operator: Thank you. The next question will come from Geoff Meacham with Citigroup. Your line's open.

Speaker #12: Hey, guys. Thanks for the question. Morning. I had another commercial kind of reimbursement question. Just given the range of options in SMA, today, how are you guys thinking about incentivizing switches or maybe deploying a more novel outcome space pricing strategy just to help the early stages of the launch?

Geoff Meacham: Hey, guys. Thanks for the question. Morning. I had another commercial kind of reimbursement question. Just given the range of options in SMA, you know, today, how are you guys thinking about incentivizing switches or maybe de-deploying a more novel outcome space pricing strategy just to help, you know, the early stages of the launch? Are the strategies different when you look to the EU and the early launch in Germany versus the US launch? Thank you.

Geoff Meacham [Managing Director, Senior Analyst: Hey, guys. Thanks for the question. Morning. I had another commercial kind of reimbursement question. Just given the range of options in SMA, you know, today, how are you guys thinking about incentivizing switches or maybe de-deploying a more novel outcome space pricing strategy just to help, you know, the early stages of the launch? Are the strategies different when you look to the EU and the early launch in Germany versus the US launch? Thank you.

Speaker #12: And are the strategies different when you look to the EU and the early launch in Germany versus the US launch? Thank you.

Speaker #2: Thanks, Jeff. I think as Keith noted up, cornerstone of our sort of campaign thus far around the disease itself has been an acknowledgment. And we see that the community gets it, that this disease is the hallmark is not only the motor neuron but the resulting muscle atrophy.

David Hallal: Thanks, Geoff. As Keith noted, a cornerstone of our sort of campaign thus far around the disease itself has been, you know, an acknowledgement, and we see that the community gets it, that this disease is the hallmark is not only the motor neuron, but the resulting muscle atrophy. You know, all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. This has been needed innovation for the community. Yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. This will be the first and only muscle-targeted therapy that's approved. We don't necessarily really think about switches, Keith, right?

Speaker #2: And so all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. And this has been needed innovation for the community.

David Hallal: You know, all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. This has been needed innovation for the community. Yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. This will be the first and only muscle-targeted therapy that's approved. We don't necessarily really think about switches, Keith, right?

Speaker #2: And yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. And this will be the first and only muscle-targeted therapy that's approved.

Speaker #2: So we don't necessarily really think about switches, Keith, right? We really think about no matter what, you choose to do for motor neuron health, we applaud.

Akshay Vaishnaw: We really think about no matter what you choose to do for motor neuron health, we applaud, and we're gonna deliver something that addresses the organ that is, you know, the, you know, the principal organ affected by this disease is the muscle. That's what's been left behind over these 10 years of innovation that we're finally able to address. Putting that into practice, I know, Keith, that's been the cornerstone of what you guys have been talking about with the community, and I'll let you take it from here.

Speaker #2: And we're going to deliver something that addresses the organ that is the principal organ affected by this disease is the muscle. And that's what's been left behind over these 10 years of innovation.

Speaker #2: That we're finally able to address. And putting that into practice, I know, Keith, that's been the cornerstone of what you guys have been talking about with the community.

Speaker #2: And I'll let you take it from here.

Speaker #12: Yeah. Yeah. No. Jeff, we're really not going to be focused on any type of switches because what we've shared before is that in our own market research with treating physicians, we know that three-quarters of them have already said that they believe dual modality is the future standard of care for treatment in SMA.

R. Keith Woods: Yeah. Yeah. No, Geoff, we're really not going to be focused on any type of switches because

Keith Woods: Yeah. Yeah. No, Geoff, we're really not going to be focused on any type of switches because what we shared before is that, you know, in our own market research with treating physicians, we know that three-quarters of them have already said that they believe dual modality is the future, standard of care for treatment in SMA. That's directly targeting the motor neuron and directly targeting the muscle.

David Hallal: You know, what we shared before is that, you know, in our own market research with treating physicians, we know that three-quarters of them have already said that they believe dual modality is the future, standard of care for treatment in SMA. That's directly targeting the motor neuron and directly targeting the muscle. We believe that that will be how this is viewed. Additionally, you know, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks with roughly one-third of patients already receiving, you know, more than one SMN-targeted therapy. It just continues to drive home the unmet medical need that exists with these SMA patients.

Speaker #12: So that's directly targeting the motor neuron and directly targeting the muscle. So we believe that that will be how this is viewed. And then additionally, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks with roughly one-third of patients already one SMN-targeted therapy it just continues to drive home the unmet medical need that exists with these SMA patients.

Keith Woods: We believe that that will be how this is viewed. Additionally, you know, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks with roughly one-third of patients already receiving, you know, more than one SMN-targeted therapy. It just continues to drive home the unmet medical need that exists with these SMA patients. As David just referenced, the principal organ that's impacted in this disease is the muscle, and we look forward to bringing forward the world's first muscle-targeted therapy.

Speaker #12: But as David just referenced, the principal organ that's impacted in this disease is the muscle. And we look forward to bringing forward the world's first muscle-targeted therapy.

David Hallal: As David just referenced, the principal organ that's impacted in this disease is the muscle, and we look forward to bringing forward the world's first muscle-targeted therapy.

Operator: Thank you. The next question's gonna come from Ami Fadia with Jefferies & Company. Your line is open.

Speaker #4: Thank you. And the next question's going to come from Amy Lee with Jefferies & Company, your line is open.

Speaker #13: Awesome. Thanks so much for taking your question and big congrats on all the progress. Just wanted to get a sense of the next steps and timelines for the Catalent site.

Ami Fadia: Awesome. Thanks so much for taking our question and big congrats on all the progress. Just wanted to get a sense of the next steps and timelines for the Catalent site. Based on feedback from the FDA after the re-inspection and the Novo closeout meeting, do you expect a Form 483 related to re-inspection? Does the speed of your BLA filing acceptance, which was around 30 days compared to the standard 60 days, indicate any FDA urgency or prioritization? Finally, on the second manufacturing site, just wanted to clarify, are you maintaining it primarily as a hedge against Catalent, or is there a potential for approval of both sites? Thanks so much.

Amy Li [SVP Equity Research: Awesome. Thanks so much for taking our question and big congrats on all the progress. Just wanted to get a sense of the next steps and timelines for the Catalent site. Based on feedback from the FDA after the re-inspection and the Novo closeout meeting, do you expect a Form 483 related to re-inspection?

Speaker #13: Based on feedback from the FDA after the reinspection and the Novo closeout meeting, do you expect a form 483 related to reinspection? And does the speed of your BLA filing acceptance, which was around 30 days compared to the standard 60 days, indicate any FDA urgency or prioritization?

Amy Li [SVP Equity Research: Does the speed of your BLA filing acceptance, which was around 30 days compared to the standard 60 days, indicate any FDA urgency or prioritization? Finally, on the second manufacturing site, just wanted to clarify, are you maintaining it primarily as a hedge against Catalent, or is there a potential for approval of both sites? Thanks so much.

Speaker #13: And then finally, on the second manufacturing site, just wanted to clarify, are you maintaining it primarily as a hedge against Catalent, or is there a potential for approval of both sites?

Speaker #13: Thanks so much.

Speaker #2: Yeah. Thanks, Amy. I'll take that last point first. As we noted when Catalent Indiana was acquired by Novo, we knew that Novo was acquiring that facility really for its own internal purposes.

David Hallal: Thanks, Ami. I'll take that last point first. As we noted when Catalent Indiana was acquired by Novo, we knew that, you know, Novo was acquiring that facility really for its own internal purposes, and they would have this transition phase into, you know, moving, quote-unquote, "customers" out because they're not, they're not a CDMO. That's not their business model. You know, all along we've recognized that, you know, that we would want to have and would require to have an additional or more than one fill finish facilities that are outside of Catalent Indiana. All of that, right, was part of our plan, you know, even prior to, you know, the Form 483 observations that the FDA had in their general site inspection last year.

Speaker #2: And they would have this transition phase into moving 'customers' out because they're not a CDMO. That's not their business model. And so, all along, we've recognized that we would want to have, and would require to have, an additional or more than one fill-finish facility that is outside of Catalent Indiana.

David Hallal: You know, all along we've recognized that, you know, that we would want to have and would require to have an additional or more than one fill finish facilities that are outside of Catalent Indiana. All of that, right, was part of our plan, you know, even prior to, you know, the Form 483 observations that the FDA had in their general site inspection last year.

Speaker #2: So all of that, right, was part of our plan. Even prior to the form 483 observations that the FDA had in their general site inspection last year.

Speaker #2: So I think it's important to note that we see this second fill-finish facility as absolutely vital for all of our global demand. Now, we also see Catalent Indiana as important.

David Hallal: I think it's important to note that we see this second fill finish facility as absolutely vital for all of our global demand. Now, we also see Catalent Indiana as important. We have drug files there. We would anticipate that they would be part of our supply chain, and in due time, they're gonna phase, you know, we would phase them out as they're gonna be phasing us out. More than anything else, we see them both as being important, and yet we do think having two independent paths to an approval under this BLA is a very significant enhancement to our BLA in 2026 versus the one that we had last year in 2025. We also think timing is really good.

Speaker #2: We have drug vial there. We would anticipate that they would be part of our supply chain. And in due time, they're going to phase we would phase them out because they're going to be phasing us out.

Speaker #2: So more than anything else, we see them both as being important. And yet, we do think having two independent paths to an approval under this BLA is a very significant enhancement to our BLA in 2026 versus the one that we had last year in 2025.

David Hallal: More than anything else, we see them both as being important, and yet we do think having two independent paths to an approval under this BLA is a very significant enhancement to our BLA in 2026 versus the one that we had last year in 2025. We also think timing is really good.

Speaker #2: And we also think timing is really good. You note the FDA's urgency and how they've been working expeditiously with us. We do think that was really anchored by a very constructive in-person type A meeting in Q4 that Akshay led with our team down there.

David Hallal: You know, you note the FDA's urgency and how they've been working expeditiously with us. We do think that was really, you know, anchored by a very constructive in-person Type A meeting in Q4 that Akshay led with our team down there, and we are just grateful that the FDA has continued to show, you know, a sense of urgency understanding the needs of the community. More than anything else, we see a world in which apitegromab gets approved with one or the other or both, and we think that that's a wonderful spot to be in. We'll let the FDA do their work on the review of the second fill finish facility and the data that has been generated by us on that second fill finish facility with drugs becoming available in early Q3.

Speaker #2: And we are just grateful that the FDA has continued to show a sense of urgency understanding the needs of the community. So more than anything else, we see a world in which a pitigramab gets approved with one or the other or both.

David Hallal: More than anything else, we see a world in which apitegromab gets approved with one or the other or both, and we think that that's a wonderful spot to be in. We'll let the FDA do their work on the review of the second fill finish facility and the data that has been generated by us on that second fill finish facility with drugs becoming available in early Q3.

Speaker #2: And we think that that's a wonderful spot to be in. We'll let the FDA do their work on the review of the second fill-finish facility and the data that has been generated by us on that second fill-finish facility with drugs becoming available in early Q3.

Speaker #2: And we'll also let the FDA do their work expeditiously and thoroughly on their inspection as well as the inspectors concluded that reinspection recently. So we're excited for what the future brings.

David Hallal: We'll also let the FDA do their work expeditiously and thoroughly on their inspection as well, as the inspectors concluded that re-inspection recently. We're excited for what the future brings. More than anything else, I think you guys can see these timelines of the two facilities have really come, you know, pretty much together. I think that that's a key takeaway to recognize.

Speaker #2: And more than anything else, I think you guys can see these timelines of the two facilities have really come pretty much together. And I think that that's a key takeaway to recognize.

Speaker #13: Excellent. Thanks so much.

Ami Fadia: Excellent. Thanks so much.

Amy Li [SVP Equity Research: Excellent. Thanks so much.

Operator: Thank you. The next question will come from Gary Nachman with Canaccord. Your line is open.

Speaker #4: Thank you. And the next question will come from Gary Nachman with Can Accord, your line's open.

Gary Nachman: Thanks, and my congrats as well on all the progress. David, just to follow on the last point you were making there, if everything ends up being fine with Catalent with the classification, are you still considering pulling the second fill finish facility from the BLA to simplify it for the FDA? Or you'll just keep it in there regardless, to have that better supply chain, even if it would potentially delay the approval and push it out a little bit?

Speaker #14: Thanks. And my congrats as well on all the progress. So David, just to follow on the last point you were making there, if everything ends up being fine with Catalent, with the classification, are you still considering pulling the second fill-finish facility from the BLA to simplify it for the FDA?

Gary Nachman [Managing Director, Equity Research: Thanks, and my congrats as well on all the progress. David, just to follow on the last point you were making there, if everything ends up being fine with Catalent with the classification, are you still considering pulling the second fill finish facility from the BLA to simplify it for the FDA? Or you'll just keep it in there regardless, to have that better supply chain, even if it would potentially delay the approval and push it out a little bit?

Speaker #14: Or you'll just keep it in there regardless, to have that better supply chain, even if it would potentially delay the approval and push it out a little bit?

Gary Nachman: Yeah.

Gary Nachman: Then just a follow-up. Someone asked before on pricing, but just I guess to ask it a little differently, is there a strategy that would make more sense of launching first in Germany or in the US, or regardless, it would just be one global price and you're not anticipating any MFN issues? You know, pricing isn't really a consideration in terms of how you'll stagger the launches. Thanks.

Speaker #14: And then just a yeah. And then just a follow-up. Someone asked before on pricing, but just, I guess, to ask it a little differently.

Gary Nachman [Managing Director, Equity Research: Then just a follow-up. Someone asked before on pricing, but just I guess to ask it a little differently, is there a strategy that would make more sense of launching first in Germany or in the US, or regardless, it would just be one global price and you're not anticipating any MFN issues? You know, pricing isn't really a consideration in terms of how you'll stagger the launches. Thanks.

Speaker #14: Is there a strategy that would make more sense of launching first in Germany or in the US or regardless? It would just be one global price and you're not anticipating any MFN issues.

Speaker #14: So pricing isn't really a consideration in terms of how you'll stagger the launches. Thanks.

Speaker #2: Yeah. These are great, really great questions, Gary. I'll just make one comment and then hand it over to Akshay. When Akshay and I hosted a call late in Q1 on the resubmission of our BLA, we did actually talk about the alignment that we've had with the FDA, the dialogue that we had with the FDA throughout Q1 about the submission with both fill-finish plants and the optionality that that really provided us.

David Hallal: Yeah. These are great, really great questions, Gary. I'll make one comment and then hand it over to Akshay Vaishnaw. When Akshay Vaishnaw and I hosted a call late in Q1 on the resubmission of our BLA, we did actually talk about the alignment that we've had with the FDA, the dialogue that we had with the FDA throughout Q1 about the submission with both fill/finish plants and the optionality that that really provided us. Akshay Vaishnaw, do you wanna comment on that on, like if the, if there is a meaningful difference in timeline, the flexibility that we may or may not have here?

Speaker #2: And so Akshay do you want to comment on that? If there is a meaningful difference in timeline, the flexibility that we may or may not have here?

Speaker #15: Yeah. I mean, just repeating what you said, and I think this has been so important to all the progress that's occurred, that there's been very constructive collaborative approach between us and the FDA and indeed with the EMA throughout this whole period.

Akshay Vaishnaw: Yeah. I mean, just repeating what you said, I think this has been so important to all the progress that's occurred, that there's been very constructive collaborative approach between us and the FDA, and indeed with the EMA throughout this whole period. Based on that, we submitted with both facilities in the BLA with their full support, guidance, and alignment. You know, the most straightforward thing is Catalent, Indiana, is reclassified, is in compliance, and we can start getting drug out of there pending approval. The second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them.

Speaker #15: And based on that, we submitted with both facilities in the BLA with their full support, guidance, and alignment. And the most straightforward thing is Catalent Indiana is reclassified, is in compliance, and we can start getting drug out of there pending approval.

Akshay Vaishnaw: You know, the most straightforward thing is Catalent, Indiana, is reclassified, is in compliance, and we can start getting drug out of there pending approval. The second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them.

Speaker #15: And the second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them.

Speaker #15: And in the long run, we clearly want redundancy in the supply chain and so we look forward to clinging on an additional fill-finish site.

Akshay Vaishnaw: In the long run, we clearly want redundancy in the supply chain, and so we look forward to continuing on an additional fill-finish site. I think all the options are open for us. The really great position we're in now to serve patients is that by 30 September, we're gonna be approved by one or the other facility. In the long run, of course, we'll have redundancy in the supply chain.

Speaker #15: So I think all the options are open for us. And the really great position we're in now to serve patients is that by September 30th, we're going to be approved by one or the other facility.

Speaker #15: But in the long run, of course, we'll have redundancy in the supply chain.

Speaker #2: Yeah. So, Gary, let's just say the FDA has up to 90 days, but they make a decision faster than that, and they still need to review some information on the second fill-finish.

David Hallal: Yeah. Gary Nachman, let's just say the FDA has up to 90 days, they make a decision faster than that, they still need to review some information on the second fill-finish. As Akshay Vaishnaw had even described, you know, about a month ago, we would certainly have that flexibility of then just moving that second fill-finish to an sBLA, which was always an option that we had considered as well. Lots of flexibility and optionality there, it was a very good question. On sequencing and pricing, R. Keith Woods?

Speaker #2: As Akshay had even described, about a month ago, we would certainly have that flexibility of then just moving that second fill-finish to an SBLM, which was always an option that we had considered as well.

Speaker #2: So lots of flexibility and optionality there. And it was a very good question. On sequencing and pricing piece?

Speaker #15: Yeah. So first of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the US. But also, the team is built in Germany.

R. Keith Woods: Yeah. First of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the US, but also the team is built in Germany. If you think about, you know, is there a preference for one before the other? No. We wanna get this across the finish line both in the US and in Europe. You mentioned how does this overall affect pricing? We go out with our list price here in the US. We go out with our list price in Germany and in Europe. Remember Germany is the only place that we can proactively promote right after EMA approval.

Keith Woods: Yeah. First of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the US, but also the team is built in Germany. If you think about, you know, is there a preference for one before the other? No. We wanna get this across the finish line both in the US and in Europe.

Speaker #15: And so if you think about is there a preference for one before the other? No. We want to get this across the finish line both in the US and in Europe.

Speaker #15: You mentioned how does this overall affect pricing? We go out with our list price here in the US. We go out with our list price in Germany and in Europe.

Keith Woods: You mentioned how does this overall affect pricing? We go out with our list price here in the US. We go out with our list price in Germany and in Europe. Remember Germany is the only place that we can proactively promote right after EMA approval.

Speaker #15: Remember, Germany is the only place that we can proactively promote right after EMA approval. And so you're promoting and selling at your list price while you go through the AMNOD process and you go through the reimbursement and establishing that price.

R. Keith Woods: You're promoting and selling at your list price while you go through the AMNOG process and you go through the reimbursement and establishing that price. It really wouldn't have an impact. The bottom line is we're gonna be prepared to serve patients in whichever market comes first. There shouldn't be a substantial impact to our ability to price negotiate and an overall impact on most favored nations because we won't be at a point right away that we would even trip the clause of most favored nations.

Keith Woods: You're promoting and selling at your list price while you go through the AMNOG process and you go through the reimbursement and establishing that price. It really wouldn't have an impact. The bottom line is we're gonna be prepared to serve patients in whichever market comes first. There shouldn't be a substantial impact to our ability to price negotiate and an overall impact on most favored nations because we won't be at a point right away that we would even trip the clause of most favored nations.

Speaker #15: So it really wouldn't have an impact in the bottom line is we're going to be prepared to serve patients in whichever market comes first.

Speaker #15: And there shouldn't be a substantial impact to our ability to price negotiate in an overall impact on most favorite nations because we won't be at a point right away that we would even trip the clause of most favorite nations.

Speaker #14: Okay. That's very helpful. Thank you.

Gary Nachman: Okay. That's very helpful. Thank you.

Gary Nachman [Managing Director, Equity Research: Okay. That's very helpful. Thank you.

Operator: Thank you. Our next question will come from Srikripa Devarakonda with Truist. Your line's open.

Speaker #4: Thank you. And our next question will come from Kripa, Deborah Conda, with TruWis. Your line's open.

Alex Long: Hi. This is Alex Long for Kripa. Congrats on the great news today. We had one about Roche discontinuation of emugrobart in FSHD. Wanted to know if you've seen any uptick in investigator interest working Valeroc for your FSHD trial. Thanks.

Alex Xenakis [Equity Research: Hi. This is Alex Long for Kripa. Congrats on the great news today. We had one about Roche discontinuation of emugrobart in FSHD. Wanted to know if you've seen any uptick in investigator interest working Valeroc for your FSHD trial. Thanks.

Speaker #16: Hi. This is Alex Long for Kripa. Congrats on the great news today. We had one about the Roche discontinuation of Immigrabart and FSHD. Wanted to know if you've seen any uptick in investigator interest working in Colorado for your FSHD trial.

Speaker #16: Thanks.

Speaker #15: Yeah. Thanks for that, Alex. So the whole neuromuscular space is very excited about the epidural MAC program after our positive data in SMA. You're right.

Akshay Vaishnaw: Thanks for that, Alex. You know, the whole neuromuscular space is very excited about the apitegromab program after our positive data in SMA. You're right. The intensity of interest has increased. Obviously, everyone's looking forward to the approval in SMA. The neurology world looks with anticipation towards what a validated anti-myostatin approach like apitegromab can do, not just in FSHD but in a range of diseases. We're looking forward to the start of the study, which will be very soon now, phase II study in FSHD, and with additional indications to follow where we'll study this drug. You're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications.

Speaker #15: The intensive interest has increased. Obviously, everyone's looking forward to the approval in SMA. But the neurology world looks with anticipation towards what a validated antimyostatin approach like epidural MAC can do, not just in FSHD, but in a range of diseases.

Speaker #15: And so we're looking forward to the start of the study, which will be very soon now, the phase two study in FSHD. And with additional indications to follow where we'll study this drug.

Akshay Vaishnaw: We're looking forward to the start of the study, which will be very soon now, phase II study in FSHD, and with additional indications to follow where we'll study this drug. You're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications.

Speaker #15: But you're absolutely right. There is plenty of interest. And very constructive input as we think about triaging through these indications.

Speaker #16: Thanks.

Alex Long: Thanks. Appreciate it.

Alex Xenakis [Equity Research: Thanks. Appreciate it.

Operator: Thank you. This does conclude the question and answer session and also concludes today's conference call. Thank you for your participation, and you may now disconnect.

Speaker #4: Thank you. This does conclude the question and answer session. And also concludes today's conference call. Thank you for your participation. And you may now disconnect.