Hi, everyone. I'm Gary Nachman, a senior biotech biopharma analyst at Canaccord Genuity. And we're very excited to have David Halal, CEO of Scalarock, with us to discuss all the recent developments at the company. David's been CEO since April of last year and has been chairman of the board since 2017. So it's been quite a journey. And we're getting so close to the finish line with a bidufidate for epitigramab and SMA coming on September 30th. So a really exciting time for you guys, and it's great to have you here, David. Thanks for being here.

Well, thanks, Gary, and it's wonderful to be with all of your clients here at the conference. And as I was noting, it's always fun when it's a home game for us, just across town from Cambridge to Boston. And as Gary noted, we will be celebrating our 15th anniversary as a company this year. And this is my 10th year as board chairman and second as CEO. And we certainly are excited to, again, be on the clock with the FDA with a PDUFA date of September 30th to usher in the next era of innovation in treating children and adults living with SMA. And that would be the world's first muscle-targeted therapy to be potentially approved by the FDA. So not that anyone is counting, but it is 49 days and counting. Anytime between now and September 30th, we're ready to go.

Well, I'm going to divulge a little secret that if you go into their offices, right when you walk in, there literally is a clock that's counting down and it's been there for a while. So it's like draft day, you know, you have more than three minutes to make your pick. Okay. So you started with giving a little bit of background, but just a little bit more on the selective anti-myostatin approach and why that's unique to Scalarock and how that enables you to look across a bunch of different indications potentially to improve muscle function.

Gary, you raise a really important point, and that is that myostatin is not a new target. It It was discovered at Johns Hopkins about 30 years ago in 1997 as a growth factor that is released from muscle, and it is the body's natural negative regulator of growing muscle. And since that very exciting discovery, nearly all pharmaceutical companies have actually They developed a strategy or a product candidate to develop to try to inhibit myostatin. In fact, Wyeth was the first company to put a myostatin inhibitor into the clinic. That was way back in 2004 before the acquisition by Pfizer. And since then, there's been somewhere between 10 to 15 approaches, as I said, from many household names in the pharmaceutical industry, and they've all failed. And so we thought when we started Scholarok, we knew the reason why they failed, and that is that myostatin, both the mature form of the growth factor of the protein as well as the receptor, resembles other TGF beta superfamily growth factors. And so if you develop either a trap or an antibody to target myostatin or the receptor, you're likely to have really unwanted off-target effects that could impact either safety or efficacy. And so we as a company had determined that when myostatin is released from the muscle, it's actually so potent that it's actually got a protective chemical cage. It's the latent form of myostatin. And the interesting thing is, and when the body needs it, the cage opens up and the mature myostatin is released. And we said, boy, there is nothing in the body that chemically resembles the pro-peptide or latent form of the disease. So let's develop an antibody to the latent form of myostatin. And we have exquisite selectivity to that and are able to completely inhibit myostatin in patients. So this was a product we brought into clinical development, healthy volunteers in 2018, brought into clinical development in SMA in 2019. So it's been a rigorous, long development program, and I'm really proud to say now seven, eight years later, epitagromab is the first and only myostatin inhibitor to ever have been proven to be successful in a phase three registration trial in any disease. And of course, in this case, we specifically chose SMA, spinal muscular atrophy.

So that background is helpful. And now, like we said, we're close to the finish line. The Pidoof is at the end of September. Focus has been on manufacturing. That's the gating factor. We've been talking about that for a while. So we had the latest update on Friday. Which, honestly, just clears the decks, right? And now we know what is happening going forward, that the Catalan facility got another OAI, but you were very smart and prescient in working with the FDA to have the optionality of the second facility. Just talk through the process now that we know Catalan is behind you and your data that you provided on the second facility and your confidence with the clock moving forward here to September 30th.

So maybe importantly, Gary, I'll just tell a little bit of recent history because then that actually guides us over these next 49 days. So as many of you know, we had our first meeting with the FDA after our complete response letter late last year. And that meeting happened on November 12th. And in that meeting, we obviously were joined by Cure SMA and Novo Nordis to discuss a path toward remediating the Catalan, Indiana facility that would then be the precursor to another inspection by the FDA of the facility to potentially clear them. At the same time, there was dialogue at that meeting about a parallel path and standing up an additional fill-finish facility. Remember, for our audience, It's important because we're in these last 49 days, but the sole approvability issue when we received our complete response letter after hours on our PDUFA date was the status of GMP practices that were observed in a general site inspection in Catalan, Indiana's facility in June, July of 2025. It was nothing related to epitogromab. It was just the status of compliance. And so it was important on our November 14th disclosure, we did share with the world that we had a constructive and collaborative meeting with the FDA, that NOVO was involved, that they said they would be re-inspection ready in the not-too-distant future, that Cure SMA was certainly sharing the very high unmet need that continued to exist. But we also noted on November 12th that we had reached an agreement with an additional fill finish facility and we were already securing reservations in the first half of 2026 to begin the qualification of that facility. So that probably was overlooked at the time, but as Gary noted, it's particularly important after the news of Friday, in which case the FDA classified their April 2026 inspection of that facility, once again, OAI. So again, while drug continues to be viled at Catalan, Indiana, and released commercially, it holds up any new drug approvals from that facility. The other bit of history that I want to cover is on March 3rd of 2026, we had a type C meeting with the FDA to update them on how much progress we were making at this alternate fill finish facility. And it was really during that meeting that, you know, the FDA and we agreed in complete alignment that we would resubmit our BLA, not just with Catalan, Indiana, but we had been making so much rapid progress with the second fill finish facility. It was agreed, let's resubmit the BLA with two independent paths to an approval, either through the faster path of if Catalan, Indiana had a successful inspection or re-inspection, or would it be the second fill-finished facility, or could it even be both? And so that was all predetermined back in March of 2026, and we agreed with the FDA. In essence, it's a horse race. Whichever one allows for the most expeditious path to an approval for epitigromab for children and adults living with SMA, that would be the one that we would take. As you might imagine, I would have been very happy when I learned on April 13th that FDA inspectors showed up at Catalan, Indiana, because you're thinking, you know, perhaps they are re-inspection ready, they have a clean inspection, and that could have been a faster path to an approval. Also, I would just tell you we did resubmit the BLA on March 30th of 2026, and in April of 2026, the FDA accepted that BLA with two fill-finished facilities and gave us our Class 2 resubmission date of September 30th, or six months. Okay, so now we know that the re-inspection did not go so well, that there were observations that seemed to be consistent with the unsuccessful inspection in 2025. And at the same time, we've been continuing to ramp up the second fill-finish facility. At that Type-C meeting on March 3rd, we agreed with the FDA on two key factors with the second facility. What data would be required for the qualification, for the review, qualification, and approval of that facility? And when within the PDUFA clock would it be due by where we would be staying on track for our PDUFA date?

Where it wouldn't be considered a major amendment. A major amendment.

And importantly, we met the exact data package that we agreed upon ahead of schedule. So the FDA had all of that information ahead of the agreed-upon timeline for that second year facility.

Is that in writing, the agreed-upon timeline for the data, or was it just a soft end in by then?

Between the Type-C meeting and then meeting minutes and then other correspondence, it is a pretty bright line on what was expected between the agency and us. So I'm really proud of two groups. One, the Scholarock technical operations and quality team that have done a wonderful job. I also just, while I'm not naming the second facility, I'm really proud of them. They really prioritized us. They had multiple lines available that were configured for our existing. They were validated for our vial configuration, and that enabled a faster path to qualifying that facility. So the FDA is in the review stage of all the information that we have provided, and we are extremely confident between now and September 30th, the FDA is going to be able to make a decision, and we're very pleased with the work that has been done at that second facility.

Okay. And just to put a finer point on the timeline, you're not anticipating an inspection at the second facility because of the track record where they've had inspections recently. I think that's a pretty important point.

Yeah. We made this point on our earnings call on August 6th on Thursday. A couple of factoids that actually point you in the direction that a pre-approval inspection or pre-license inspection would not be needed. The first is that in the last 12 months, this facility, with a long-term and recent positive track record of inspections by both the FDA and EMA, so multiple inspections between 2025 and 2026, so over the last 12 months, they've had multiple product approvals where the PLI or PAI was waived. That's an important fact. The other important fact I would note is that the FDA has had a recent successful general site inspection of that facility. And then maybe the third thing I would note is we would have wanted to make sure that the FDA knew the next time we were filling in that facility epitogramab, because unlike a general site inspection, which is unannounced, they usually like to schedule a PLI or a PAI. So they knew the next time we were filling epitogramab at the site just several weeks ago, and there was no scheduled inspection. So there's a lot of facts that sort of point in the direction that that is not likely to be needed in this case.

Okay. And given the stage that we're at right now, normally you would be in labeling discussions, but that happened already. So, and you're pretty confident in terms of where the label landed from the last go around. And you've said that the FDA, there's been a lot of good engagement and dialogue. There's urgency on their part. They want to get this through. Do you feel like there's still some back and forth on the label, on the indication, or does it feel like that's pretty settled? It's a great question.

Again, looking a little bit at recent history, what I've noted is there was never major distance between us and the FDA, even from the beginning on our labeling discussions. So that's good news. The second is why we thought we were, remember, this is all predating the OAI classification and the warning letter, why we thought we were on track for an approval on September 22nd of 2025 is we were getting into very late stage labeling discussions, so much so that on September 18th, the FDA had submitted their latest version of the label to us. They were expecting it back the next day, September 19th. That was a Friday. And the PDUFA was on Monday. We did get back to them on the 18th. And I would say that where we last left it, we were in a really good spot. We were very much in alignment with the label. So where we had left it, we were probably within 24 to 48 hours of being done. And I would imagine, and they point this out in the complete response letter, that's exactly the label that they wanted back was the last one that we had sent to them on the evening of september 8th so we would be very close on labeling and we were it'd be inappropriate to comment specifically on a label until it's approved but we were pleased with the alignment uh that we in the agency were at um the other thing i want to note gary because it may be on people's minds we've done so much work at that second fill finish facility I made note, and I thought it was important, on our earnings call on Thursday, August 6th, we now have more vials that are commercially filled from the second fill finish facility than we do from Catalan, Indiana, and more than we had from Catalan, Indiana on September 22nd of 2025. and those vials from the second facility are at our third-party provider awaiting approval for labeling and packaging so we can get on with the launch. So we're not just squeaking through with the second vialer. We feel like we're extremely well advanced with the inventory and the product that we have to, you know, support our launch. Awesome.

We'll talk more about commercial readiness in a second, but the last piece of the puzzle is the EU. And now that we know that Catalan is out of the picture for now, how quickly you could provide them, the EMA, with the data from the second facility and the level of dialogue there is just something that you could turn this around potentially by the end of the year, or you're going to give yourself a little bit more of time on that.

Yeah, Gary raises a very good point. And I was quick, if those of you look back on our disclosures about the BLA, I was quick in pointing out that, reminding everybody, just in the spirit of full disclosure, we never had the equivalent of a complete response letter in Europe. So to this point, our MAA, which was filed just a few months after our BLA back in 2025, only had Catalan, Indiana listed in it as our drug product or fill finish facility. We never had the opportunity of resubmitting or adding the second facility. We did have open dialogue with the European Medicines Agency that we'd like to submit that information. There just wasn't a vehicle for that. There wasn't an appetite for that. They said, can we just wait for the classification of the inspection that came down? And, of course, we just disclosed on Friday that we just learned that that classification past the 90-day period we expected came down as OAI. So what I'd like to say to you all is when we didn't have a classification decision by the June and the July CHMP meetings, we did issue a release and we shared with the world that we could no longer stand by our guidance of a CHMB decision near midyear, which would have been either just before midyear or just after midyear. When asked what is our new guidance, I said we can't really provide new guidance until we have a classification decision. We now have that. The next thing now we need to do is engage in the summertime, but engage with the European Medicines Agency. And I think once we understand their thoughts, they've been very flexible, they've been very open, but we are late in the cycle, which I just want to prepare everybody for. We did file this MAA back in Q1.

So they could come back and say that they're not accepting the application.

Or they could say something like the best vehicle is you withdraw and you resubmit, but we'll try to get on with this quickly, knowing that we were also done with everything, including a prospective SMPC. But we need to engage with the EMA. And once we understand both their preferred and recommended both process and timelines, we'll be fast to then let all of our stakeholders know, including the SMA community in Europe, know what that looks like. We have built our German team. That would have been the first country to launch in. And there's plenty for them to do. We have a playbook on what to do if there's a delay, as we've done in the United States. And we'll talk about commercial readiness in a moment. But we'll keep everybody apprised once we have more information from the European.

I mean, with all the data that you're submitting to the FDA, if it clears FDA, that's a big box to check for the EMA as well. Right. And there's got to be some reliance on that.

So Gary's bringing up a good point because there's mutual recognition, which is what's holding us up in Europe, is the FDA inspection of Catalan, Indiana. So we're hopeful with mutual recognition in FDA clearance of the second fill finish facility can hopefully, whatever path we take in Europe, could aid us with an expeditious path of some sort with European regulators.

Okay. So let's assume a positive outcome. You get the timely approval on a before the PDUFA in the U.S. You've been preparing for a year. You've talked extensively. You don't have to get into too much detail. But just, you know, the highlights internally of what it's been like to have the commercial team in place and ready to go. And, you know, all the different, I guess, touch points that you've been refining in terms of the advocacy groups with Cure SMA, the payers, and just making sure that's all on. You're going to have the supply, so no issues there. So day one, you're out of the gates.

So Gary knows our business very well, as you can tell. We have a small commercial team. It's an experienced team. The folks who lead it are the folks who have worked with me in the past at Alexion, but more importantly, both Keith Woods and Rebecca McLeod led the Vivgart launch globally and in the U.S., and they've been with us now for approximately a year, a little bit more than that. And so we think we've got a tremendous team. They are engaged with all the different constituents. Can I give you, you know, maybe one fact that I would provide? There was a survey that was done in 2025 at this time, and they asked about 30-some KOLs managing over 500 patients, what percentage of them would call all of their patients and offer epitogramab at approval to those patients. And it was about 23% of KOLs said they would. Now, a year later, about 42% are saying that. And I think it's because we've been using our time, obviously, to do what we can do, which is to educate that SMA is a disease of the motor unit, not just the motor neuron. For 10 years, we've had innovation focusing on motor neuron health by producing more SMN protein, either SMN1 or SMN2. But muscle is the principal organ impacted by the disease, and there's been no directed or targeted approach to the muscle. So just by educating on that, we think there's a greater appreciation that we now see about 75% of neurologists believe that addressing the muscle component of the disease as well as the motor neuron component is going to be important. And we know that about 90% of patients report that muscle atrophy, muscle weakness, motor function is their number one demand that they're looking for. So I think demand will be solid at approval. Gary, as you know, access is always an issue. And we want to be thoughtful. We've developed Scholarox supports, which is going to enroll each individual SMA patient and family into our system to help them with any number of access and reimbursement related issues. But we do expect at launch, payers need to develop medical policies. We do need to probably get at least six months behind us before we have a drug specific J code for an every four week infusion. And we also know that there may be formulary status issues, slow to approve and maybe fast to deny initial coverage. But this is a motivated community. This is a community that already has created access for themselves with the SMN-targeted therapies. So we don't think it's a matter of if, it's when epitigromab will be approved for a wide range of patients living with SMA. Okay.

And you think the payers are conditioned for the pricing? You haven't said specifically where that's going to land, but high level, it's probably going to be in a range of rare disease drugs typical to what we see on the SMN side.

Yeah. We're proud of the development plan.

I think anytime one thinks about pricing and reimbursement, you think about the rarity of the disease.

35,000 patients worldwide have received at least one SMN-targeted therapy, about 7,000 in the U.S. You think about severity of the disease, and that is that patients plateau and start to once again progress and lose motor function long-term on SMN-targeted therapies alone. And with our drug, there was a return to motor function. And thirdly is, do you have compelling clinical benefits? We have a robust 188-patient phase 3 trial. And what we saw in that trial, Gary, is when patients were randomized to either placebo or epitogramab, patients who got placebo were losing motor function. Patients who received epitogramab were gaining motor function, despite the use of standard background SMN targeted therapy. We think that combination sets up very, very well for long-term payer acceptance and coverage. Probably one would expect for sure in a rare disease category of pricing and reimbursement.

Okay. And one last very quick final question, because there is a platform here. There are other programs. I know what, if Akshay were here, what his answer would be. But, you know, starting a phase two in FSHD, your level of excitement on a scale of one to 10, just in terms of the data that you've seen, the opportunity that this is a program people should be paying more attention to. Thanks, Gary.

You know, I've said Akshay is an expert drug developer with a number of product approvals behind him. And he came here to do more than just bring epitogram out of the finish line for SMA. We look at a series of genetic and acquired neuropathies and myopathies where we think the platform of epitogramab, the first and only myostatin inhibitor to ever be proven to be successful in a phase three trial, sets up well for other muscular dystrophies, muscular atrophies. And the thing I would note, which is important, is we're two for two on animal models panning out in clinical development. One was SMA. And remember, the other was cardiometabolic and obesity. So it's always translated well, we call it SRK015, our murine version, our mouse version of epitogramab. And our FSHD preclinical data is pretty profound. We saw an improvement in a FLEX-DUX4 mouse model in muscle mass, muscle force and exercise endurance. And then when we look at sort of clinical series in FSHD, we saw that rigorous exercise programs in the form of physical therapy and or the use of anabolic agents, steroids, human growth hormone also showed efficacy in clinical development. And that bodes well for our 60-patient Phase II FORGE study and a moderate phenotype of FSHD as a monotherapy. So we'll have a look at that. This is a common rare neuromuscular disease, and it is devastating as well. A fifth of patients will end up in a wheelchair. But please note, we also hold out hope that in a more severe patient population, when one receives a corrector, like a DUX-4 targeted therapy, we feel like we're going to have a role there as well. So we are super excited to have announced that we've initiated that study, and we look forward to keeping you all updated over time.

Awesome. All right, we're going to leave it there. Thank you so much. Good luck with the countdown. Thank you, Gary. We're excited. Thanks, everybody.

Thanks for joining us.