Q3 2025 Scholar Rock Holding Corp Earnings Call
Speaker #2: During today's call is outlined on slide two . David will provide introductory remarks in a business Akshay . update review R&D provide . Keith will update on our commercial readiness activities , and Vikas will provide a financial .
Speaker #2: then We will open the call update questions . Before we begin , I'd like to remind you that during this call , we will be various statements about Scholar Rock's plans , expectations , and prospects making forward that looking constitute statements for the purposes of the safe harbor provisions under the Private Securities Reform Litigation Act of 1995 .
Speaker #2: Any forward looking statements represent views only as of today our and should not be relied representing our views as of any date . I encourage you to go to the Media section of our future most website for our up to date SEC statements and Investors in filings .
Speaker #2: With that , I'd like to turn the call over to David . upon as David
Speaker #2: . Thank
Speaker #3: Laura , and you , good morning . Thanks to everyone for joining our third quarter earnings call today . April , when I was In appointed CEO after eight years in the board chairman role , and on the same day , we brought in Akshay , Keith and Vikas , confident that we were scholar Rock was to positioned next be the global biotech powerhouse .
Speaker #3: this on We based several factors . First , our conviction that the global ipilimumab and psma opportunity with offers the alone potential for many years of sustainable growth that company will the end of power our decade and into the next .
Speaker #3: Second , as leaders in myostatin biology , our ability to deliver transformative therapies to patients suffering with additional rare , severe and debilitating neuromuscular disorders , and third , leveraging our platform to advance our novel , subcutaneously administered myostatin inhibitor SRK 439 .
Speaker #3: When joined scholar we Rock , the most significant milestone ahead was the September 22nd . Date for a and , which Psma had been granted priority review Our .
Speaker #3: Bla was supported by robust data a demonstrating mab's and safety efficacy for children and adults living with SMA . Based upon our 188 patient prospective , randomized , double blind , placebo , multinational phase three trial , this trial showed a statistically significant and clinically meaningful controlled benefit and motor function as measured by the gold standard .
Speaker #3: Motor Function Scale for SMA . While we disappointed to receive complete response a letter on September 22nd , we were pleased that the strength of our phase three data was reflected in the FDA's review Bla our of , and that the soul Approvability issue referenced in the CRL the status of our third party finish facility in fill Indiana , which is owned by Novo Nordisk .
Speaker #3: We know it's not a matter of if, but when a Mab will be approved in the U.S. for patients living with SMA.
Speaker #3: We are emboldened by the commitment we have made to the more than 35,000 patients living globally with SMA who have received an SMN-targeted therapy.
Speaker #3: We are working expeditiously to deliver on our global ambition that SMA patients who can benefit from a Mab should have access to a Mab. And now, more than ever, they need a Mab.
Speaker #3: we are confident in the significant opportunity that we have ahead us to the SMA serve as we community of determination to bring this important medicine to children and work with adults .
Speaker #3: With SMA . This is indeed what we know well and what we do well . I like to now provide a regulatory update on a particular Mab .
Speaker #3: would our type A meeting FDA on with the We are grateful to the agency We had for their full participation , particularly in the context of a shutdown .
Speaker #3: The meeting was in and included the relevant leaders and decision from the makers including the person neurology Division and the Office of Compliance .
Speaker #3: team was joined by Kenneth Hobby Our SMA and representatives from Novo Nordisk . We were encouraged by the meeting . It was collaborative .
Speaker #3: It constructive and was that there is a , president of understanding of the high unmet need for shared the SMA community and a shared sense of urgency to bring a Mab to adults .
Speaker #3: With this disease, children and Novo Nordisk detailed the progress they have made in implementing their remediation plan at the facility and affirmed that they expect the facility to be ready for reinspection by the end of this year.
Speaker #3: We path the forward discussed and await the Bloomington final minutes of the meeting. We work closely with the FDA and anticipate resubmitting the BLA for the U.S. launch.
Speaker #3: Following approval of a Mab for children and adults with SMA and in 2026 . I'd like to now turn to adding redundancy to our supply chain .
Speaker #3: When Novo Nordisk purchased the Bloomington site in December of 2024 , they planned to plant internalize the for their own products . In light of that , scholar Rock implemented a plan to add an additional US based fill finish facility .
Speaker #3: Now , with the OAI classification , Rock has scholar accelerated our timelines for an additional Byler . We have selected a world class commercial proven facility that record and has successfully completed recent site inspections , including with the FDA and EMA .
Speaker #3: As you know , one of the bottlenecks to vilor is securing adding a new capacity . This can be a lengthy process . Importantly , we commercial have capacity commencing in the first quarter 2026 and tech transfer of is now underway .
Speaker #3: We anticipate submitting an Sbla for this facility later in 2026 . In summary , we will continue to work with urgency to bring this important medicine to the SMA community .
Speaker #3: We look forward to providing clarity on resubmission timelines as soon as we are able . In addition to the large opportunity we have to serve children and adults with SMA , we continue to strategically advance our pipeline .
In addition to the large opportunity, we have to serve children and adults with SMA. We continue to strategically Advance our pipeline. This includes the phase 2 opal study. Progressing a pyramid and a second rare. Debilitating neuromuscular disorder, as well as advancing srk 439 into the clinic.
AI will provide additional detail on these activities shortly.
Importantly, to reach our Ambitions, I am pleased the opportunistically, strengthened our balance sheet, during the third quarter, and we continue to operate with a tight financial plan, which vcos will discuss later in the call.
This plan is aligned to thoughtful, strategic investments to drive long-term value creation.
We remain confident in the strength of our strategy.
The grid of our team and the transformative potential of a pedigree map and our pipeline.
The regulatory challenges we face today are temporary, but the opportunities ahead to serve patients are extraordinary.
With that, I'll turn the call over to Auction to provide more detailed updates on our R&D progress. Auction.
Thank you, David and good morning everybody.
As David noted, we continue to work with urgency to bring a bit of a map to children and adults with SMA as quickly as possible.
SMA is a rare severe neuromuscular disease resulting in irreversible, loss of motor, neuron and progressive muscle wasting that diminishes, the independence of both children and adults.
map has the potential to reverse the trajectory of SMA from a loss of motor function to a gain of motor function as demonstrated in the phase 3, Sapphire study underscoring, the importance of the potential benefits of this therapeutic,
I'd now like to turn to Wednesday's Taipei meeting.
I was pleased to lead our team at that meeting for Thursday.
And David said, the meeting was in person and included the relevant leaders and decision makers from the agency including the neurology Division and the office of compliance.
Our team was joined by Kenneth Hobby, President of Pure SMA, and representatives from Nova and Nordis.
the meeting was constructed and collaborative.
We reviewed the comprehensive data set from the pit of amount development program, including the phase 2 topaz study, which demonstrated that delayed treatment results in suboptimal, suboptimal motor, function outcomes.
These data underscore the impact of delayed treatment and the urgency to make a political map available to the SMA community.
It was clear that the CRL we received on September 22nd was based solely on the need for the Bloomington facility to be in compliance with CGMP, or Current Good Manufacturing Practice regulations.
Robust remediation plan at the Bloomington facility.
Never know what it's all shared with the FDA. They did expect the facility to be ready for inspection by the end of the year.
We remain in close coordination with nivo nordis as we await the minutes from the type a meeting.
After Nova's, completion of remediation of the Bloomington facility in a site with inspection, by the FDA, we have to say, resubmission of the bla and US launched following approval of the program in 2026.
As part of our long-term growth plan to serve patients around the world, with the fit of the map, we're also accelerating timelines to bring a second feel finish with the online.
This process requires vigorous validation and regulatory approval to ensure the same quality, safety, and efficacy of the drug product.
Importantly, we've secured commercial capacity commencing in the first quarter of 2026 and anticipates submitting. An SBA for the second facility later in 2026.
Outside of the us we continue to expect a decision from the EMA on our a pilgrim man marketing authorization application or ma near the middle of next year.
Further to our commitment, to the broad SMA Community. We're now today that we've initiated dosing in our face to opal trial, evaluating a pedag app in infants and toddlers under the age of 2.
The trial is enrolled enrolling participants have been treated with an smn1 Target of gene therapy or who are receiving treatment with an approved SMN 2 times a therapy.
It is designed to investigate two different doses of the Pentagon lab for dourish for a duration of 48 weeks and will affect PK, PB, efficacy, and safety.
In the open study early, intervention with the pit could support muscle due to a critical early development phase, complimenting smile therapy that aims to preserve motor neurons.
By promoting muscle growth when most neurons and muscle were still forming, a significant opportunity has arisen to improve motor outcomes in young children with SMA.
Beyond SMA. We're on track to initiate clinical development activities for a bit of the map in a second neuromuscular disorder by year end.
We plan to provide additional information on the disease and the clinical development strategy in early 2026.
And finally, we continue to advance our world-leading anti-fat and platform beyond the bigger map.
The FTSE cleared the IMD for SRK-439, and we're on track to initiate a Phase 1 study in healthy volunteers before the end of this year.
This program Builds on the validated approach that delivered, a pedigree map.
Specifically, 49 was designed to be an Innovative, subcutaneously administered myat inhibitor binding to both Pro and late M with high affinity, and selectivity Based on preclinical data 439, has the potential to potentially inhibit mioton and increase muscle mass.
We expect to update from the sad portion of the phase 1 study in 2026.
In summary, our focus remains on bringing us a pilgrim and adding to patients.
And to patients and investing with financial discipline to deliver on the promise of our broader pipeline.
The strength of our data and the momentum across our programs. Gives us confidence in the impact, we can deliver.
now at this point, I'll turn the call over to Keith to discuss our commercial launch strategy and planning keep
Thanks Doc, Chey the SMA Community is demanding more, even with currently available treatments. They need a treatment that directly addresses progressive muscle wasting a pedigree mab, demonstrated that ability in our phase 3, Sapphire study, and we will be ready to deliver a pedigree app to the SMA Community upon approval. This is not a matter of if but when
Our understanding of the demand for a pedigree map and our confidence in its potential to address the unmet need for children and adults with SMA continues to strengthen.
As we look at SMA globally, nearly a decade following the launch of the first SMN. Targeted therapy. The demand for treatment, continues to grow.
After the first 3, quarters of 2025 annual revenue for current SMA treatments are trending to approximately 5 billion dollars globally with the continued growth of SMN. Targeted therapies the need for the world's first muscle. Targeted therapy is greater than it is ever been before.
Our small, lean, and highly experienced U.S. customer-facing team.
To enhance our engagement activities and to strengthen our performance against key pre-launch readiness metrics.
As a reminder, we are just under 4 months into our pre-commercial field deployment. Whereas most biotech companies typically benefit from a longer runway prior to approval.
Nationwide. There are approximately 140, SMA treatment centers and more than 2600 SMA. Prescribing Physicians with this additional time, we are working to both broaden and deepen. Our engagement with these potential. Prescribing Physicians,
However, an SMA patient is not just treated by one of these positions but by a broader cross-specialty SMA treatment team. This team can include Physical Therapy, pulmonology, orthopedics, and more.
This additional time is enabling us to better understand the patient journey and the roles of the SMA treatment team in each of these 140 treatment centers, and how they influence patient care.
Additionally, our Market Access team is expanding their focus beyond that of national payers to also include top regional payers.
this Builds on our ambition that any patient with SMA who can benefit from a pedigree map should have access to a pedigree map
Furthermore, our unwavering commitment to the SMA, patient Community continues by a partnership at a local and National events.
And to educate on the importance of targeting muscle.
We are deepening our collaboration with the advocacy groups. And we are also building lasting relationships. 1 patient, 1 caregiver, 1 family at a time.
In Europe, our efforts, continue to drive, SMA education, and awareness, laying the groundwork to ensure we reach patients efficiently across key markets.
Our opportunity to serve patients around the world. In SMA is significant
There are an estimated 35,000 people with SMA who have received an SMN-targeted therapy and who could be eligible for treatment with a pedigree map.
We are making strategic, disciplined investments in our launch infrastructure, and we will be ready to execute rapidly once a pit of remap is approved.
In short, we are ready. The strategy is clear, the team is in place and our commitment to the SMA Community has never been stronger. Now, I will turn the call over to vikas vikas.
Thank you, Keith.
Our overarching objectives are to fund our R&D activities, expand our leadership in the myostatin and muscle space to support a strong commercial launch, and extend our runway to meet our eventual timelines for epidural map approval.
In line with these objectives, I'm pleased to provide our third quarter financial results and to discuss our approach to managing our cash runway and investment prioritization moving forward.
turning first to our third quarter results, we ended the third quarter with 369.6 million in cash and cash equivalents
for the quarter. We reported 103 million in operating expenses which includes 18.3 million in non-cash stock based compensation.
Excluding stock based compensation operating expenses with 85.3 million which reflects ongoing investments in infrastructure, to support aidro map regulatory approval, commercial Readiness, and our clinical pipeline.
During the third quarter, we strengthened our balance sheet, adding 141.7 million. This cash came from 2 sources. First, we executed our ATM and sold approximately 2.8 million shares which resulted in net proceeds of 91.7 million. And second, we drew down $50 million from our existing debt facility.
As we await Aid to gab approval, we continue to operate with a tight financial plan focused on thoughtful Capital, allocation to advance our clinical Pipeline and strategic Investments to support commercial readiness.
Accordingly, we have adjusted our go-forward operating plan.
Certain R&D activities, including a third indication for oppido map and other discretionary spend.
now, I'll turn to
The 6 prior Investments. We are making.
The acceleration of a second field, finish facility for Aido app.
SMA commercial launch readiness, Onyx, the app extension study, the Phase 2 Opal study.
The second indicator app and the commencement of SRK-439 clinical development.
Turning to our balance sheet. Our current cash balance is 369.6 million. Which we expect to be augmented by approximately 60 million in cash from the exercise of outstanding common warrants by year end
with this, we expect our cash to be sufficient to fund operations into 2027
This cash runway has conservative assumptions and does not reflect any upside from the potential sale of the epidural map or a priority review voucher.
To further, strengthen our balance sheet, we intend to expand our credit facility while preserving our non-dialysis.
We will provide further clarity on this, as well as our anticipated operating expenses for 2026, during our fourth quarter earnings call.
Scholar Rock continues to operate from a position of financial strength, with a disciplined approach to capital allocation and a clear focus on supporting us today. Priorities with that. I've done the call back to David.
Thanks, Vikas. In closing, Scholar Rock remains focused on near-term execution while building with financial discipline for the future.
Our conviction in a pedigree map and in our broader strategy is stronger than ever, and we are moving with urgency and purpose to deliver meaningful impact for patients.
Our priorities are clear.
Execute with urgency to bring a pedigree map, the world's first and only muscle-targeted treatment.
That improves motor function for children and adults living with SMA as rapidly as possible.
Advanced, a pedigree map and activities, and a second rare debilitating neuromuscular disease, and that will be followed by additional indications where we can have a transformative impact for patients. We want to progress SRK-439 into the clinic.
And continue to invest in our future with discipline to support these high-value initiatives.
Before I close, I want to share my sincere appreciation for Cure SMA and the SMA patient community.
Over these past weeks, I have had the opportunity to meet with many individuals and families living with SMA.
And the words of support that have been shared with us, and with me directly, have been tremendously meaningful as we work harder, better, and faster to bring this impactful medicine to those who can benefit from it. We'll now open the line for questions. Operator?
Thank you as a reminder, to ask a question. Please press star 1, 1 1, 1 on your telephone and wait for your name, to be announced to withdraw your question. Please press star. 1, 1 1 again, we ask that you please limit yourselves to 1 question please. Stand by while we compile the Q&A roster,
Our first question comes from the line of Manny Fuhar from Ling Partners.
Hey guys, thanks for taking the question. Congrats on the progress through what's been, obviously a choppy, uh, period for everyone in the government. Uh,
I think a couple of quick questions. I know I'm violating the one-question rule. Um, in terms of thinking about further financing opportunities to top up the tank as necessary, how do you think about debt versus royalty equity? Like, how do you think about relative costs of capital and what's the most appropriate use? Um, once you get to a lodge, and then another commercial question in the early days of launch, it is probable that you will be transitioning from one facility to another. Um,
To what extent does that—does that introduce any operational risk going from products from 1 to 3 to another? And how can that be addressed ahead of time by you guys now?
Supply chain with Phil. Finish, because yeah, thank you, David. Hey Manny, our first objective here is to bridge the financing till the approval. And you know, the first path to go from the lowest cost to capital is.
to take um, additional extend our loan facility a little bit more. We're in discussions with that. That will be our first opportunity. Um, royalty. Probably comes next and you know, uh, we if, if, if uh, it goes
Too long. And then we, um,
Have to take a little bit of equity that will be the last and the most expensive 1, which we are trying to avoid at any cost and it's trying to get it as more more non-dilutive first.
Um, does that answer your question?
Yes. So, follow up—um, that would imply that, relatively speaking, we should expect you guys to wind down, slash use the ATM, much less going forward. Like, how did that fit into the strategy?
Yeah, you know, um obviously our first objective is to work with the loan facility and expand upon that, and um ATMs are put in place just to take um some small augmentation of the capital.
At at an opportunistic View and we did take uh take it down in the last quarter because we are only 50 million um, loan facility available. We're expanding that loan facility as we discussed and as soon as we have the new facility in place, we'll um we'll share it with all of you.
And then, and then Moni Monty regarding the, the second filer, um, you know, a couple of bottlenecks I've I've shared with you and others in the, in the past, 1 of the big ones I highlighted in the, in the call is obviously finding a commercial line. That is available. That is, uh, got the ideal configuration for for our V and, uh, our team under the leadership of, of Lisa, Lisa Wyman, our our chief Tech and quality officer, just did an extraordinary job in accelerating, uh, uh, uh, our second violer progress, uh, to secure commercial capacity, uh, in in q1 and commence Tech transfer and lightning speeds. Since that, um, middle of October timeline, when the oai hit, we thought that that was really important. Now, now to get there and to get there quickly, you want to change as little as possible in your second violer as in your primary violer whether or not it's vial. Configuration analytical testing like
So so that actually helps you with speed as well and those are the things that we'll be focused on. And then the impact in the marketplace, really should be almost seamless, uh, whether or not we are, um, you know, Distributing our API uh, from the Bloomington facility or the new second visor. It should really be quite seamless operationally uh, to the marketplace and we would expect it to be that way. Thank you.
All right. Thanks guys. I know, I squeezed 3 questions in there. Sorry. No, that's okay.
Thank you. 1 moment for our next question.
Our next question comes from the line of Eric Schmidt from caner.
Thanks for taking my question and congrats on the progress as well. Um, David and team for, for those of us who've kind of been reading the Gory play-byplay around the Catalan facility in in Bloomington, um, and and know, you know, some of the prior history and and all the past issues. How do you, how do you kind of provide confidence that, um, this remediation effort is is on good footing and, and that the inspection will will prove, uh, positive. And then, um, maybe secondarily to expect that to be a Class 1 or Class 2 uh acceptance for the resubmission. Thank you.
Uh, thanks, Eric. And and and and no doubt there is a history in the facility. We think the history is really anchored around.
you know, the quality system, the quality culture, and the facility, I think importantly it, uh, largely, uh, links back to um,
From the top of the organization, the changes in the staff that they are making the integration of the novo quality system into that facility. And then the substantial, um, progress that they've been making on a robust remediation plan, which, as they noted to the FDA, uh, on Wednesday. Uh, they feel like they're the, the facility is going to be reinspection ready, uh, by the end of this year, um, we don't think that Novo takes that lightly. Um, we think that they are going through a series of internal, um, exercises to make sure that they are reinspection ready, we would imagine that they'll continue to communicate with the FDA and gather feedback on what might be missing from their remediation plan that they would then need to tweak before any reinspection would take place, uh, but we are uh, surely uh, been pleased um, with the seriousness and the urgency that from the top.
Top of that organization right through that facility. They are taking the remediation plan.
Um, regarding Class 1 or Class 2, I’ll turn that over to Aha for his thoughts, as he was presiding over our team in person in Bethesda on Wednesday. The team just did a fantastic job. AA?
Yeah, thanks, David. I, you know, Eric, I just want to reiterate that it was a very constructive and collaborative meeting, and I think the agency, as you might expect, shares the need for urgency as we all work together to try and get a bit of an amount of patience. So, it's not for us, obviously, to second guess and say will it be Class 1 or Class 2? But we were very heartened by the comments they made and the approach they committed to in order to help protect them by getting the structured patient. So, um, you know, we need to work with Novo to get their work done. Let's, you know, wish them the best. Get the site reinstated, resubmit the BLA. Um, and I'm confident the agency is going to act with urgency and commitment to this community of patients, which they've always done when it comes to SMA.
Thank you very much.
Thank you. 1 moment for our next question.
Our next question comes from the line of tests Romero from JP Morgan.
Hey, David and team. Thanks so much for the question here. So to be clear the bla that you plan to submit in 2026 for a pedigree map will include Catalan as your primary sale finish and you plan to file the SBA for the additional sale, finish facility facility later in 2026, following the potential approval of the bla. Why is that the right path versus using an additional?
Still finish only and then follow up is just on the EMA review. How is that going with? Respect to manufacturing related items?
Thanks.
Thanks Tess. Yeah, I mean, I think given where we are given the tone and tenor of the meeting that aha presided over, uh, this week. And, um, you know, again, the progress that Novo was been been making, um, which really um,
You know, which really enabled them, uh, to communicate to the FDA that they are on track to, to be reinspection ready by the end of this year, uh, we just think that that is the absolute right path for us. Uh, we would expect, uh, that our, uh, bla would be resubmitted, uh, with Catalan as our primary filler. And we would expect the second filler to be added to our file. Which was, frankly, always going to be our plan anyway, given the fact that Novo wants that facility for internal purposes. Uh and so that is the path that we are following obviously everything that we are doing to accelerate. Our second filer is a great insurance policy for us. No matter what would happen and I was really gratified by our team's efforts over the course of just the past month uh with the major progress that they have made to secure a commercial capacity at a second Filer and already have Tech transfer under web.
And we'll, of course, be expecting the commencement of our commercial capacity to be leveraged beginning in Q1 of 2026.
Regarding the EMA. I'd love to have a comment on that uh obviously quality and compliance is important to all Regulators including them.
The tablet manufacturing status. And and the EMA, uh, there is a mutual recognition procedure. And so there is an interdependency. And I think we we obviously agree with that. Uh, though I would point out that everything we're doing for the 2026 resubmission, we Novo, uh, and all our collaborators from the 2026 resubmission of our pla here in the US launched, uh, following approval, uh, by the FDA is in line with supporting our MAA. So, uh, there's not much more I can say right now and we'll keep you updated. But um, let's say on track and as we proceed with the da, uh, we hope that supports the uh, EU approval as well.
Thank you.
Thank you. 1 moment for our next question.
Our next question comes from the line of tazeen Omar from Bank of America.
Hi guys. Good morning. Um, thanks for the details update.
So can I ask when is the latest that you can have this reinspection for Catalan be completed and given the the green light in order to meet your expectations for a 2026 launch and then just to play the scenarios for a second. If for whatever reason the the second inspection for Catalan, you know, doesn't resolve all issues. How quickly could you pivot to make it?
Any application, uh, you know, uh, with your second fill finish and how would that impact your timelines for 2026? Like, would you be able to switch that SBA filing to a bla filing and keep the timelines? The same as you just mentioned? Thanks.
Yeah. Thanks Cain it, it's again a good question. Um dating back to what or marketing back to Eric's point. You know, there's a history at the facility.
Uh, it was under prior ownership.
Um,
they've had a few.
uh, difficult inspections that have
Uh, that have that have led to form 483 and and in this case, some repeat observations. So I understand and we understand
That everybody could um share some level of concern and or skepticism that just getting a reinspection is not the objective. It's a successful reinspection.
To really put their own team through.
Related to, uh, your point about what if it doesn't resolve all issues? I, I think, you know, there's 2 ways to look at this to see.
Are there still observations in the facility? And and but yet to those observations warrant or not,
Um, sort of a reclassification of this facility, because that's really what we're playing for, a reclassification from OAI to either V or NYE.
And, um, for that, certainly that is what the objective is.
Um, I think regarding your timeline, I think a reinspection.
You know, could technically go pretty well into 2026 and we would still be within a frame of our guidance of um, you know, resubmitting our VA uh, and then and then the US launched upon approval. Um we're obviously um pleased.
that the, um,
The tone and tenor of our type. A meeting led by AI, with the FDA, with all the key decision-makers, all the key groups.
Um, I think it was constructive, it was collaborative, and there really was a shared understanding of the unmet need and a shared understanding that urgency is necessary to serve a very important patient population. So we're hoping that all of the steps that would be required to get us to a reinspection will be done in an expeditious way within the regulatory framework that exists, and that Novo will do their part. We don't think that they take lightly indicating that the
The FDA that they will be reinspection, ready by the end of this year. We don't think that that's a low bar. We think they're holding themselves given the commitment to Quality and compliance in the culture of Novo Nordisk. We think they say that with a pretty high hurdle in mind.
Um but back to your question about should like a meteorite hit that facility, in other words, should the inspection not go. Well,
Actually, our primary resubmission, uh, strategy. There are a number of ways.
That the FDA and we expect, they've done this in the past and given the shared urgency would understand.
Some level of potential Pathways to expedite adding a second filer, as your first filer, uh, in the form of a bla. And, um, everything that we're doing to expedite this process, we think will Aid us.
Uh, in case the impact of the inspection, uh, is not what we all expect it to be, which is a successful reinspection. And as we continue to work with that second buyer, we can provide further guidance, uh, to you, uh, as we progress from Tech transfer, which is now underway, uh, directly into, uh, uh, the the, the the, the, the the filling lines, that we will be executing in q1, and Q2, and we'll provide those updates over time.
Thank you.
Thank you. 1 moment for our next question.
Our next question comes from the line of krepa Deva rakanda from truist securities.
Hey, guys. Thank you so much for taking my question and congratulations on all the progress. Um, thanks for all the details. So, in terms of timelines for resubmission of the BLA, I feel like we're all asking the same question, but is the plan to wait for the reinspection and for the OAI to be resolved before you submit the BLA? And, um, I understand that Novo has said that they're going to be inspection-ready by year-end, but could you clarify more requests?
An inspection. And finally, you know, would you would you be able to address whether Novo hired any outside consultant to help with this process?
Thank you.
Yeah. Maybe related to the, the timelines on resubmission. Uh, I think that, uh, actually can can comment on our, on our thinking and recognizing that, uh, with the collaboration, with the sea, um, and the shared urgency, you know, it's a little Dynamic, we don't have our type, a meeting minutes yet, but actually can share at least our go forward plan with respect to that. Yeah, I mean I think base case could it safe to say that the inspector would have to account would submit after that but as David said that it is a dynamic situation and we'll do everything possible to resubmit the fashion to to expedite the approval of this work which patients need so badly. Uh, and we were hardened by the degree of support from the agency during outside taking into. Uh so you know a lot.
That happened in the coming weeks and and early part of next year. And we look forward to recently stealing
But for sure kPa given that again, it's reiterated our Sole and primary issue is the classification of this facility and their state of compliance. I think it's the safe assumption that we'd like to see that cleared, uh, and be ready to go immediately with a resubmission that's sort of our go forward plan at this point. And I think it's worth adding David that that resubmission is really competitively initial building. It's it's a very different asset. Uh, it's a much more contained effort around
just the safety update and the cncf space, the file
although it's very
You know, we're ready to to file that resubmission that very short notice.
Um, crypto related to the Novo request, uh, uh, reinspection. I think, in a way, they're signaling that they're ready. We would imagine that Novo and the agency still have some wood to chop, you know, just.
How do we feel about the remediation plan? Is there anything left that needs to be done before a reinspection? I would expect that to be happening, okay? That would be an expectation, but...
Um, in a way, they put themselves, um, you know, on notice with the FDA that they stand ready to be reinstated, uh, toward the end of this year. And we think that that is, you know, really really important. But as you know, this reinspection will not be uh, announced it would be like your typical unannounced inspection and so that you can put yourself on notice and communicate with the office of compliance that you're reinspection, ready. Um, as they notified at our, our type a meeting at the end of this year. Um but we would expect the FDA to um, when they do reinstate the facility, it would likely be an unannounced inspection and then your final um question about like third parties, I think.
Novo is really.
They are applying to this remediation plan, and we're thankful to them for that.
Thank you. One moment for our next question.
Our next question comes from the line of Ed sir, Dora from Barclays.
Thanks, uh, for taking the, the question. Uh, just, uh, you know, because investors have sort of been circling, this September 2026, uh, uh date, uh, in terms of sort of timing for a potential approval, David maybe if you could help understand what what could, you know, maybe the FDA minutes unveiled to you, on the type of of resubmission that you have to make, maybe their timelines around the decision. Once you have, um, um, filed we filed the, the bla. Thank you.
Yeah, thanks that sir. It's a, it's a great question. And again, something that I think AI, you know, wanted to have some robust conversation, you know, uh, you know, with the agency on it or type a meeting where obviously not, um,
We don't have meeting minutes in hand and we we certainly um want to allow the agency to do their work. But aha can comment on how we're thinking about the resubmission timing and and again whether or not it would be class 1 or Class 2. Yeah. I you know, as far as the minutes are concerned. I think it's always good to get the minutes in hand and we confirm the Impressions that we're conveying to you this morning that that documented in a minute. So the progress,
Uh, Nova has made, uh, of commitment that everyone is showing to the owners of America, uh, Nova's comment about being ready for reinfection, uh and you know everything we've discussed so far. So we we await those minutes and we're confident that they'll reflect uh what we're conveying this morning.
Now, you know, as far as the, uh, resubmission is concerned. We've just discussed that with the last question. And of course, we'll resubmit, as soon as the inspection is done or early, if possible. Um, but we'll be guided by the agency to all of that, and we'll also be guided on the review timeline. Now, the minutes we get won't, uh, spell out the, uh, nature of the review timelines for the resubmission that's not their package. Their weight resolution of the de before that. Um, but 1 thing I can tell you, is that David emphasized, the term antenna of the meeting that there was support to activate urgency to get on all parties. So including the agency, to get this drug to patients as soon as possible.
Great. Thank you. And thank you for all the updates today.
Thank you.
Thank you. One moment for our next question.
Our next question comes from the line of Mark from TD Cowen.
For taking my questions and all the, uh, detailed disclosures around this meeting. Maybe in that light, just as you moved forward and, Noah, hopefully, you know, is in fact in a position to be reinstated, it just—what do you expect to be able to disclose and kind of on what timeline? In particularly, given that it isn't even your facility directly, but it is a partner, um, you know, will you be able to disclose right when it gets inspected? Not until?
Maybe some 4,833, 4,333 are, um,
Received just what are the disclosure plans there?
Uh, thanks, Mark. I think our disclosure plans will really, you know, kind of look in the mirror and focus on us and the things that are, you know, material to us that we think are important to share. Obviously, you know, the reinspection timeline, when it happens, and the outcome of it is really important. So I think we want to be open to sharing the important information with you. Obviously, the way this would work is um,
You know, an inspection takes, you know, a week to a couple of weeks. Uh, there's generally a closeout meeting at that closeout meeting. There's um, you know, generally some kind of preliminary assessment, uh, when a a form 483 as we've noted in the past. I think again, it's hard to believe. Um, when we first disclosed these uh, form 483 observations
Was only last quarter because it feels like, for me, it's been a long time. But, uh, you know, as you know, a form 483, uh, usually travels 75% of the time with any inspection, um, but but of course, we wouldn't expect a form 483 to result in in an oai, most of the times and I think that's what startled all of us. Um, but I think that all along Novo has been approaching this, um, you know, very aggressively. So I think we'll just, you know, maintain as we have open lines of communication with you all. When we have important information to share, we'll certainly do that. And I think, what we've done in the past is even if it wasn't something for us,
Last month in October, you know, we tried to get out in front of that and disclose that, and and have some dialogue with you all on what that meant and we'll, we'll continue to, you know, make a commitment to do the same here. Uh, as we, uh, continue on this journey to an eventual resubmission and US launched upon approval,
Okay, that that's helpful. And maybe just on the idea of waiting for the reinspections to kind of happen in the reclassification before filing. But also, you know, when your prior answers, you noted, you know, this would be kind of like an unannounced reinspection once they've communicated that they really are, uh, in a position to be ready.
But kind of a forcing mechanism to that at some level, it could be a submission of a bla from anyone using the facility. So, you know, maybe is there some value of maybe filing a head to kind of try to force that the timeline on the inspection? Um, or is your expectation that there are just so many other products flowing through this facility that that's kind of going to happen on its own, without you guys being the fourth thing? Oh well, Mark, a couple of the things you said are really important. Um, one is that, um,
You know we've heard this too, right? I mean the thing that creates, you know, urgency are are pending applications. And right now, we don't have a pending application, we have a pending resubmission
Um, at the same time.
Uh, I I I would note that we were um generally pleased with how constructive and and, and collaborative the in-person type a meeting was and that there was this, you know, shared understanding of the unmet need and and shared urgency. So, while we're not on file, I would say of a lot of the things and you're absolutely right on your last point that there are other pending applications that that site and that can serve us. Well, we do think our type a meeting serves as a, a really good, um, central point of highlighting while we're not on file. There is real urgency here for a community that is desperately. Um, um, wanting to benefit from the world's first and only uh muscle directed uh therapy. And so we have to continue to work with the agency, the collaborative with them. Um find you know, everyone's right footing on what the right thing to do is and that is really our, our go forward plan.
And we think we've built sort of a foundation and framework with the agency. Frankly, all the way through the initial priority review period, up until September 22nd, and even through Wednesday, a really strong foundation for collaboration for us to work together to resolve this issue.
Yeah. Thanks Dave. And I just want to reiterate the importance of that collaborative approach and um, you know, you mentioned, you know, a forcing function. So to speak by resubmitting ahead of the reinspection. I, I don't know that. That's why we we want to be working closely with the fbn be guided by the map. As we said it's a dynamic situation and if you know if they invite us or they they they support any kind of resubmission in a in a particular time frame in and around the C in around the inspection. We will of course we are ready and we can be submit very efficiently. Uh but this is not about a forcing function. We have to collaborate with the FBI.
Okay, thank you.
Thank you. One moment for our next question.
Our next question comes from the line of Evans segerman from BMO Capital markets.
Hi, Michael Hoffman on for Evan. Thanks for taking our question regarding the delays for a pedigree M. Can you talk more about what your sales or market research team's efforts are to identify patients that have the launch? You had mentioned efforts to work with centers of excellence to understand the patient journey better, but do you feel you were developing a more robust number of patients which you could target for therapy following approval? Um, potentially leading to a little bit of a faster uptake than people were probably initially expecting. Thanks.
Yeah, it's a great question. Keith was highlighting it uh earlier in the call and I I'll turn it over to him for a further comments on uh, on uh, you know, launch prep.
Is that there's a clear understanding of the unmet medical need and the approach of attacking this disease. From a dual modality, no longer just the motor neuron but also directly targeting the muscle. And this is being well accepted. As as we get the opportunity to meet with more people in the community. Um, you add to the fact of the safety profile that a pedigree map has demonstrated in quite frankly, all of our studies, not just our SMA studies, but if you take a look at Embrace, that was also all adults, all treated with 10 milligram per kilogram and, you know, exceptional, safety results. Um, I guess I would end with the fact that at the end of the day we're offering the world's first muscle targeted therapy. And, you know,
In the event, if you have a choice to either be in a situation of, you know, having experienced muscle loss or the potential for muscle gain, why wouldn't you want to use a pedigree map?
Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.