Skip to content
    SRRK
    Earnings call· Jun 2026(Q2 FY26)

    Scholar Rock Holding Q2 FY26 earnings call SRRK

    Aug 6, 2026 Source

    Executive summary

    Scholar Rock Q2 FY26 — Apitegromab BLA Progresses with Dual Fill/Finish Paths Towards September 30 PDUFA

    Scholar Rock is in a defining period, driving towards the U.S. launch of apitegromab this quarter, with its BLA progressing well through FDA review via two independent fill/finish facility paths. The company is also advancing its European MAA and expanding its anti-myostatin pipeline with new clinical studies in SMA and FSHD, supported by a strong financial position and robust commercial readiness.

    Highlights

    5

    • Ended Q2 FY26 with a strong cash position of $492 million in cash, cash equivalents, and marketable securities.

    • Apitegromab BLA review progressing well with two independent paths to approval (Catalent Indiana and second fill/finish facility) ahead of September 30 PDUFA date.

    • Initiated Phase II FORGE study for apitegromab in FSHD, addressing a significant unmet need.

    • Robust enrollment in Phase II OPAL study evaluating apitegromab in infants and toddlers with SMA.

    • Commercial organization is launch-ready in the U.S. and advancing preparations in Europe for apitegromab.

    Concerns

    3

    • FDA inspection classification for Catalent Indiana fill/finish facility is still pending, having drifted beyond the 90-day guidance period.

    • European MAA approval for apitegromab is dependent on FDA clearance of Catalent Indiana, with potential timing updates pending EMA alignment.

    • Anticipate prior authorization and potential initial denials for apitegromab reimbursement in the U.S., leading to an average time from prescription to infusion greater than 60 days during the first 6 months post-launch.

    Guidance & targets

    6
    CategoryTargetConfidence
    Apitegromab U.S. Launch
    Launch
    high materiality
    High
    Apitegromab BLA PDUFA Date
    September 30
    high materiality
    High
    SRK-439 Phase I Top-line Data
    Top-line data
    medium materiality
    High
    Subcutaneous Apitegromab Regulatory Engagement
    Engagement with U.S. and European regulators
    medium materiality
    High
    Debt Facility Drawdown Option
    $150 million
    medium materiality
    High
    Priority Review Voucher Monetization
    Monetize
    medium materiality
    High

    Operational metrics

    13
    Operating expenses
    $108.9 million
    Q2 FY26

    Total operating expenses for the second quarter.

    Operating expenses (excluding stock-based compensation)
    $89.2 millionconsistent with Q2 FY25
    Q2 FY26

    Operating expenses for the second quarter, excluding non-cash stock-based compensation.

    Stock-based compensation
    $19.7 million
    Q2 FY26

    Non-cash stock-based compensation included in operating expenses for the second quarter.

    Cash, cash equivalents and marketable securities
    $492 million
    Q2 FY26 end

    Balance sheet cash position at the end of the second quarter.

    ATM program net proceeds
    $63 million
    Q2 FY26

    Net proceeds generated from the At-The-Market (ATM) program during the second quarter.

    SMA patients worldwide (received SMN-targeted therapy)
    35,000
    Current

    Estimated number of people with SMA around the world who have received an SMN-targeted therapy.

    U.S. SMA patients receiving SMN-targeted therapy
    approximately 78%
    Current

    Percentage of children and adults living with SMA in the U.S. who are receiving an SMN-targeted therapy.

    U.S. SMA patients with persistent and progressive muscle atrophy
    95%
    Current

    Percentage of SMA patients in the U.S. who continue to experience persistent and progressive muscle atrophy despite receiving SMN-targeted therapy.

    U.S. SMA patients received 2 or more SMN-targeted treatments
    estimated 1/3
    Current

    Estimated proportion of people living with SMA in the U.S. who have received two or more SMN-targeted treatments.

    SMA treatment centers
    approximately 140
    Current

    Number of SMA treatment centers in the U.S. that the field team is expanding reach to.

    Prescribing physicians
    2,600
    Current

    Number of prescribing physicians and their multidisciplinary care teams in the U.S. that the field team is expanding reach to.

    Time from prescription to infusion (initial launch)
    greater than 60 days
    First 6 months post-launch

    Expected average time from prescription to a patient receiving infusion during the first six months of apitegromab launch, primarily due to prior authorization and appeal processes.

    FORGE study sample size
    60
    Phase II

    Sample size for the randomized, double-blind, placebo-controlled Phase II FORGE study in FSHD.

    Risks & headwinds

    3
    FDA inspection classification for Catalent Indiana fill/finish facility pendingOngoing

    drifted a bit beyond the 90-day guidance period

    Mitigation: Second fill/finish facility provides independent path to approval; ongoing dialogue with FDA to remove Catalent Indiana if needed without timeline impact.

    European MAA approval dependent on FDA clearance of Catalent IndianaPending

    N/A

    Mitigation: Engaging with EMA regarding inclusion of second fill/finish facility (which has successful recent site inspections by FDA/EMA).

    Reimbursement challenges for apitegromab launch in U.S.Initial 6 months post-launch

    average time from prescription to a patient actually being able to get infused is greater than 60 days during the first 6 months post-launch

    Mitigation: Strengthening Scholar Rock Supports patient services program, engaging with payers to create policies aligned with label, preparing for prior authorizations and appeals process.

    What to watch in Q3 FY26

    5

    Apitegromab BLA approval

    Q3 FY26 (by September 30)
    CurrentUnder FDA review with PDUFA date of September 30
    TargetApproval

    Why it matters

    Determines market entry for the company's lead product.

    Our BLA was accepted in April with a PDUFA date of September 30.

    Q&A highlights

    8

    How confident is Scholar Rock that the second fill/finish facility alone can support approval by the PDUFA date, and what FDA interactions support this progress?

    Management is highly confident, noting the second facility has more vials ready than Catalent Indiana had at the last PDUFA, has passed recent FDA/EMA inspections, and the FDA review of its data package is progressing well. They confirmed that removing Catalent Indiana from the application would not impact the timeline.

    “That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI.”

    asked by Eric Schmidt · answered by David Hallal

    2 min read5 chapters

    Detailed Narrative

    01

    Apitegromab Regulatory Progress

    Scholar Rock's BLA for apitegromab is progressing through FDA review with a PDUFA date of September 30. The company has two independent paths to approval, including the original Catalent Indiana fill/finish facility and a second facility. The FDA inspection classification for Catalent Indiana is pending, having exceeded the 90-day guidance period. However, the data package for the second facility has been submitted and its review is progressing well, with more vials available from this site than from Catalent Indiana. Management expressed confidence in the FDA's engagement and the ability to meet the PDUFA date.

    02

    European Regulatory and Commercial Strategy

    The apitegromab MAA in Europe is under review, but its approval is currently dependent on the FDA's inspection classification for the Catalent Indiana facility. Scholar Rock is actively engaging with European regulators to potentially include its second fill/finish facility, which has recently passed FDA and EMA inspections. Commercial preparations are advancing, with an initial launch planned for Germany, followed by expansion into other countries to build a 50-country operating platform aimed at reaching 35,000 SMA patients worldwide.

    03

    Pipeline Advancement

    The company is making meaningful progress across its anti-myostatin pipeline. The Phase II OPAL study, evaluating apitegromab in infants and toddlers with SMA, is experiencing robust enrollment, indicating significant unmet need. Scholar Rock also initiated the Phase II FORGE study for apitegromab in facioscapulohumeral muscular dystrophy (FSHD), a rare neuromuscular disease with no approved therapies. Additionally, development continues for a high-concentration subcutaneous formulation of apitegromab, with regulatory engagement planned later this year, and the Phase I healthy volunteer study for SRK-439 is progressing well, with top-line data expected later this year.

    04

    Commercial Readiness and Reimbursement

    Scholar Rock's U.S. commercial organization is fully launch-ready for apitegromab, prepared to support patients, caregivers, and prescribers immediately upon FDA approval. Efforts include disease education, expanding reach to ~140 SMA treatment centers and ~2,600 prescribing physicians, and strengthening the Scholar Rock Supports patient services program. While broad reimbursement is sought, the company anticipates prior authorizations and potential initial denials, expecting the average time from prescription to infusion to be greater than 60 days during the first six months post-launch.

    05

    Financial Strength and Capital Allocation

    The company ended Q2 FY26 with a strong cash position of $492 million, which includes $63 million in net proceeds from its ATM program during the quarter. Operating expenses for the quarter were $108.9 million, or $89.2 million excluding stock-based compensation. Scholar Rock maintains a disciplined financial plan, focusing investments on apitegromab commercial launch readiness, supply chain strengthening, and advancing its clinical pipeline. Upon FDA approval, the company has an option to draw an additional $150 million from its debt facility and plans to monetize a priority review voucher.

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