Scholar Rock Holding Corporation Q2 FY2025 earnings call

• Core priorities include apitegromab regulatory approvals and U.S. launch, expanding apitegromab into other neuromuscular diseases, and disciplined capital allocation. • Ongoing regulatory progress: BLA accepted under priority review with target action date September 22, working collaboratively with FDA and EMA; EU MAA under review with potential approval mid-2026. • Commercial readiness: U.S. customer-facing team deployed, preparing for apitegromab launch. • R&D progress: Positive Phase II EMBRAZE study in obesity, plans to initiate OPAL Trial in Q3 2025 for infants with SMA, and expansion into other neuromuscular diseases like DMD and FSHD in mouse models.

No specific financial performance data for product segments provided in the transcript.

• BLA target action date is September 22. • Anticipate EU approval of apitegromab mid-2026. • Initiate OPAL Trial in children under 2 in Q3 2025. • File IND application for SRK-439 in the second half of 2025.

• FDA inspections at CDMOs resulted in observations; discussions ongoing with FDA regarding responses to these observations and potential impact on approval timeline. • General site inspection of fill/finish site for apitegromab at Catalent, Indiana, which was acquired by Novo Nordisk, with observations noted.

Q: Can you tell us a little bit more about the specific observation at those 2 CDMO sites, whether a reinspection is required prior to apitegromab approval and if the 2 facilities are currently able to release other pharmaceutical products?

A: David Hallal states GMP inspections are standard, observations were within review cycle for response, collaborative late cycle meeting indicates FDA working toward completing BLA review by September 22, and facilities continue to operate.

Q: Do you know whether the 2 facilities are releasing any product currently?

A: David Hallal notes the 2 facilities continue to operate, with Catalent, Indiana (now owned by Novo Nordisk) being active and the other facility manufacturing product.

Q: Further characterize any interactions with FDA on the apitegromab review outside of CDMO observations, feedback on overall confidence in receiving a broad label, and payer outreach?

A: Akshay Vaishnaw mentions constructive late cycle meeting with FDA, Keith Woods states payer outreach is progressing positively with durable response being impressive, and discussions on paying for another SMA therapy are going positive.

Q: What is the right way to think about the next step following CDMO observations given proximity to goal date and likelihood of positive review outcome; what does an ideal label look like?

A: David Hallal says working closely with CDMO partners on responses to observations, Akshay Vaishnaw and Keith Woods state ideal label would be broad to include all patients 2 years and older with SMA based on robust trial data.

Q: Talk through payer budget impact of concurrent SMA therapies and R&D new indication start later this year?

A: David Hallal mentions considering rarity of SMA, severity of disease, and clinical benefits when setting price; Akshay Vaishnaw states new indication studies will begin later this year with more to come.

Q: OPAL Trial enrollment timelines and significance of expansion opportunity with initial label for apitegromab in patients 2 years and older?

A: Akshay Vaishnaw states enrollment in OPAL Trial starts in Q3 2025, and the trial is important for helping babies and toddlers with SMA, with apitegromab potentially impacting across the entire age range.

Q: CDMO site inspections, need for another inspection, time lines for resolution?

A: Akshay Vaishnaw says working on robust responses to observations, effort placed on responses, and keeping stakeholders apprised of progress.

Q: Color on late cycle review meetings, interactions on dose efficacy difference, and financial modeling cadence?

A: Akshay Vaishnaw states constructive late cycle interaction, 10 and 20 mg doses have similar pharmacodynamic effects, and Vikas Sinha mentions thoughtful budget planning for 2026 with clinical expense drops and pipeline prioritization.