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    SRRK
    Earnings call· Dec 2025(Q4 FY25)

    Scholar Rock Holding Q4 FY25 earnings call SRRK

    Mar 3, 2026 Source

    Executive summary

    Scholar Rock Q4 FY25 — Apitegromab Regulatory Progress and Pipeline Advancement

    Scholar Rock is poised for a transformative 2026, driven by the anticipated BLA resubmission and US launch of apitegromab for SMA, alongside advancing its anti-myostatin pipeline. The company is actively preparing for commercialization in the US and Europe, while also strengthening its financial position through a new debt facility. Despite potential initial access challenges, the long-term opportunity for apitegromab is viewed as significant.

    Highlights

    5

    • Ended 2025 with $368 million in cash and cash equivalents, including $60.4 million from warrant exercises.

    • Secured a new debt facility for up to $550 million, enhancing financial flexibility.

    • FDA engagement and progress on Catalent Indiana facility remediation continues at a rapid pace, reaffirming 2026 BLA resubmission and US launch guidance.

    • Second fill-finish facility engineering runs underway, with supplemental BLA filing anticipated later in 2026 to strengthen supply chain.

    • EMA decision for apitegromab MAA expected mid-2026, with European launch preparations advancing.

    Concerns

    2

    • 95% of SMA patients continue to experience persistent and progressive muscle weakness despite SMN-targeted therapies.

    • Initial access speed bumps expected at launch due to payer reimbursement timelines and miscellaneous J-code period.

    Guidance & targets

    10
    CategoryTargetConfidence
    Apitegromab BLA resubmission and U.S. launch
    2026
    high materiality
    High
    Supplemental BLA for second fill-finish facility
    later this year
    medium materiality
    High
    EMA decision for apitegromab MAA
    mid-2026
    high materiality
    High
    European launch of apitegromab
    second half of the year, beginning with Germany
    high materiality
    High
    Initiate Phase II FORGE study for FSHD
    middle of this year
    medium materiality
    High
    Top line data from SRK-439 Phase I study
    second half of this year
    medium materiality
    High
    Debt facility draw down
    $100 million
    medium materiality
    High
    Debt facility draw down upon FDA approval
    up to $150 million
    medium materiality
    High
    Additional incremental debt facilities
    up to $200 million
    low materiality
    Medium
    Monetization of priority review voucher
    to further strengthen our balance sheet
    medium materiality
    High

    Operational metrics

    17
    Operating expenses (adjusted)
    $72.5 million
    Q4 FY25

    Reported operating expenses were $91.9 million.

    Operating expenses (adjusted)
    $309 million
    FY25

    Reported operating expenses were $384.6 million.

    Cash and cash equivalents
    $368 million
    end of 2025

    Includes $60.4 million from the exercise of warrants.

    Warrant exercise proceeds
    $60.4 million
    Q4 FY25

    From the exercise of warrants that were set to expire on December 31.

    New debt facility
    $550 million
    total

    Secured to strengthen the balance sheet and provide flexibility.

    Prior debt facility repaid
    $100 million
    Q1 FY26

    Repaid using the initial draw from the new Blue Owl Capital debt facility.

    SMA global annual sales
    $5 billion
    annual

    Market continues to grow nearly a decade after SMN-targeted therapies.

    SMA patients with persistent muscle weakness
    95%
    current

    Despite SMN-targeted therapies, muscle strength and motor function remain top unmet need.

    Neurologists believing in multiple modalities for SMA
    3/4
    current

    Underscores the significant opportunity for apitegromab.

    SMA treatment centers engaged
    approximately 140
    current

    US customer-facing team is active in the field.

    Prescribing physicians engaged
    2,600
    current

    US customer-facing team is active in the field.

    Home infusion network size
    more than 10,000
    current

    Established through patient access partners to enhance convenience.

    SRK-439 potency vs apitegromab
    10xmore potent
    preclinical

    SRK-439 is a subcutaneously administered myostatin inhibitor.

    SRK-439 lean mass change dose
    0.3 mg per kgas low as
    preclinical

    Can produce changes in whole body lean mass.

    FSHD patients diagnosed
    more than 30,000
    current

    FSHD is a rare, devastating neuromuscular disease with significant unmet need.

    FSHD patients wheelchair dependent
    20%
    current

    An estimated 20% of patients will become wheelchair dependent.

    SMA patients globally
    more than 35,000
    current

    Living with SMA who have received an SMN-targeted therapy.

    Deals & partnerships

    1
    Blue Owl CapitalNew debt facility for up to $550 million$550 million

    Consists of an immediate $100 million draw (used to repay prior $100 million debt facility with Oxford Finance), an additional $100 million available in Q1 2026, up to $150 million upon FDA approval of apitegromab, and an option for additional incremental facilities of up to $200 million.

    Risks & headwinds

    3
    Compliance status of Catalent Indiana facilityOngoing, impacting BLA resubmission timeline

    Sole approvability issue for apitegromab BLA

    Mitigation: Close collaboration with FDA and Novo Nordisk on remediation plan, FDA field team visit, anticipation of successful reinspection, and development of a second fill-finish facility.

    Payer access and reimbursement bottlenecks for apitegromab launchInitial launch period

    Initial access speed bumps expected

    Mitigation: Expanding specialty pharmacy network, establishing home infusion network (10,000+ nurses), launching 'Scholar Rock Supports' patient services program, and engaging with national/regional payers, Medicare, and Medicaid.

    Persistent muscle weakness in SMA patientsOngoing

    95% of patients continue to experience persistent and progressive muscle weakness

    Mitigation: Developing apitegromab as the world's first muscle-targeted therapy to address this unmet need, complementing SMN-targeted therapies.

    What to watch in Q1 FY26

    5

    FDA reinspection of Catalent Indiana facility

    next quarter
    CurrentFDA sent field team, stated intent to conduct reinspection following routine manufacturing activities (resumed late Feb)
    TargetSuccessful reinspection

    Why it matters

    A successful reinspection is the gating item for apitegromab BLA resubmission and subsequent US launch.

    At the conclusion of the visit, the FDA once again did not have any additional request to Novo's remediation plan and stated to Novo that it intends to conduct a site reinspection following routine manufacturing activities, which has since resumed in late February.

    Q&A highlights

    12

    Is Novo ready for reinspection, and what specific confidence is needed from the reinspection to trigger the BLA resubmission?

    Management is gratified by the FDA and Novo's urgency. The gating item is a reinspection following routine manufacturing activities, which have resumed. A successful reinspection will trigger a rapid BLA resubmission, and the company is confident given the progress.

    “We are at the ready to submit our BLA submission very, very quickly. But it really would be with some level of confidence that it was a successful reinspection.”

    asked by Eric Schmidt · answered by David Hallal

    2 min read4 chapters

    Detailed Narrative

    01

    Apitegromab Regulatory Progress and Supply Chain

    Scholar Rock is actively working towards the BLA resubmission for apitegromab, reaffirming its 2026 target for both resubmission and US launch. Following a Type A meeting in November, the FDA has shown continued engagement with Novo Nordisk regarding the Catalent Indiana facility remediation, including a field team visit and no additional requests to Novo's plan. The company is prepared to resubmit the BLA after a successful FDA reinspection. Additionally, a second fill-finish facility is progressing rapidly with engineering runs underway, and a supplemental BLA filing is expected later in 2026 to ensure supply chain redundancy.

    02

    Commercial Readiness and Market Opportunity

    The company is advancing commercial launch preparations for apitegromab in the US and Europe, with a planned launch in Germany in the second half of 2026. US customer-facing teams are educating prescribers and payers on the unmet need in SMA and the importance of muscle-targeted therapy. Despite SMN-targeted therapies, 95% of SMA patients still experience muscle weakness, and 75% of neurologists believe multiple modalities are necessary. Scholar Rock is expanding its specialty pharmacy network, establishing a home infusion network, and launching the 'Scholar Rock Supports' patient services program to mitigate access bottlenecks.

    03

    Anti-Myostatin Pipeline Advancement

    Scholar Rock's anti-myostatin pipeline is progressing with strong momentum. The Phase II OPAL study for apitegromab in infants and toddlers with SMA is enrolling and dosing participants, aiming to expand treatment to Zolgensma-treated patients. The IND for apitegromab in FSHD is cleared, with a robust, randomized, placebo-controlled Phase II FORGE study expected to initiate mid-2026. Promising Phase I data for subcutaneous apitegromab showed comparable pharmacodynamic profiles to IV administration, with regulatory engagements planned for later in the year. The highly innovative SRK-439 myostatin inhibitor, which is 10x more potent than apitegromab, is in Phase I, with top-line data expected in the second half of 2026.

    04

    Financial Position and Capital Structure

    Scholar Rock ended 2025 with $368 million in cash and cash equivalents, including $60.4 million from warrant exercises. The company secured a new debt facility for up to $550 million with Blue Owl Capital. This facility includes an immediate $100 million draw (used to repay a prior debt), an additional $100 million available in Q1 2026, up to $150 million upon FDA approval of apitegromab, and a further $200 million option. This strengthens the balance sheet and provides flexibility for commercial launch and pipeline investment, with plans to monetize a priority review voucher post-approval.

    AI-generated summary of the company’s earnings call. Not investment advice.