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    CAPR
    Earnings call· Jun 2026(Q2 FY26)

    CAPRICOR THERAPEUTICS Q2 FY26 earnings call CAPR

    Aug 13, 2026 Source

    Executive summary

    Capricor Therapeutics Q2 FY26 — Deramiocel BLA Amendment and PDUFA Extension Planned

    Capricor Therapeutics is navigating a complex regulatory path for its lead asset, Deramiocel, following a disappointing FDA Advisory Committee vote on its initial cardiomyopathy indication. The company plans to submit an amendment to its BLA, focusing on an upper limb skeletal muscle indication, which the FDA has indicated it will review, extending the PDUFA date. While commercial readiness continues at a slower pace and other pipeline programs are on hold, the company maintains a strong cash position and is pursuing arbitration in its dispute with NS Pharma.

    Highlights

    5

    • Planned BLA amendment to support a refined indication for Deramiocel focused on upper limb skeletal muscle, with FDA willingness to review and extend PDUFA date.

    • HOPE-3 clinical trial results published in The Lancet, highlighting efficacy and mechanism of action.

    • Deramiocel demonstrated statistically significant slowing of upper limb disease progression (PUL 2.0) with a mean difference of 4.55% (p=0.029).

    • Strong cash position with $237.9 million in cash, cash equivalents, and marketable securities as of June 30, 2026.

    • In-house GMP manufacturing facility is operational and expanding, positioned for initial commercial launch.

    Concerns

    4

    • FDA Advisory Committee voted 3 in favor, 9 against for Deramiocel's effectiveness for cardiomyopathy in DMD, based on the original BLA indication.

    • Net loss for Q2 FY26 increased to $40.7 million ($0.70 per share) from $25.9 million ($0.57 per share) in Q2 FY25.

    • Dispute with NS Pharma regarding the U.S. distribution agreement pricing structure remains unresolved, with arbitration estimated to begin this fall.

    • All pipeline work not directly related to Deramiocel is on hold until further regulatory clarity.

    Guidance & targets

    4
    CategoryTargetConfidence
    Deramiocel BLA PDUFA action date
    Extended accordingly
    high materiality
    High
    Expanded GMP manufacturing facility approval
    Full validation and FDA approval
    medium materiality
    Medium
    NS Pharma arbitration process start
    Begin this fall
    medium materiality
    High
    Clinical trial initiations for younger DMD and Becker muscular dystrophy
    Stage-gated by regulatory pathway for Deramiocel in U.S.A.
    low materiality
    Low

    Operational metrics

    11
    Cash, cash equivalents and marketable securities
    $237.9 million
    Q2 FY26

    Strong cash position.

    Total operating expenses
    $42.9 millionvs $27.7 million in Q2 FY25
    Q2 FY26

    Primarily driven by continued investment in clinical, regulatory and manufacturing activities as well as commercial infrastructure supporting the Duchenne program.

    Net loss
    $40.7 millionvs $25.9 million in Q2 FY25
    Q2 FY26
    Net loss per share
    $0.70vs $0.57 in Q2 FY25
    Q2 FY26
    Net loss (6 months YTD)
    $74.7 millionvs $50.3 million in YTD Q2 FY25
    YTD Q2 FY26
    Accumulated deficit
    $379.6 million
    Q2 FY26
    Intravenous infusions administered
    approximately 1,300
    Cumulative

    Across clinical program for Deramiocel.

    Patients treated with Deramiocel
    over 200
    Cumulative

    Across 3 separate clinical trials for DMD.

    Patients in open-label extension studies
    More than 80
    Current

    Some receiving continuous infusions for more than 5 years.

    Manufacturing facility expansion completion
    estimated to be in 2027
    Future

    Expansion to the second floor of the San Diego facility continues.

    FDA BIMO inspection observation
    1 observation
    Q2 FY26

    Responses submitted, awaiting feedback.

    Industry KPIs

    3
    MetricValueDetails
    Regulatory approvals filingsBLA amendment planned for Deramiocel
    Clinical trial efficacy safety dataPUL 2.0 mean difference of 4.55%%
    Cumulative patients uptake since launchOver 200 patientspatients

    Deals & partnerships

    1
    NS PharmaDispute over U.S. distribution agreement pricing structure, seeking rescission.

    Motion for preliminary injunction withdrawn without prejudice to allow for a more complete regulatory record for arbitration.

    Risks & headwinds

    5
    Negative FDA Advisory Committee vote for Deramiocel's cardiomyopathy indicationJuly 29, 2026

    3 in favor, 9 against

    Mitigation: Planning BLA amendment for refined indication (upper limb skeletal muscle) with FDA's willingness to review and extend PDUFA date.

    Uncertainty regarding Deramiocel's regulatory approval timeline and potential PDUFA delayNear-term

    Original PDUFA date August 22, 2026, will be extended

    Mitigation: Working closely with FDA on BLA amendment; pacing commercial expenditures to control cash.

    Ongoing contractual dispute with NS Pharma over U.S. distribution agreement pricingArbitration estimated to begin this fall

    Pricing structure fundamentally flawed, impeding patient access

    Mitigation: Withdrew preliminary injunction motion to pursue arbitration; position on merits unchanged.

    All pipeline work not directly related to Deramiocel is on holdUntil further regulatory clarity for Deramiocel

    All pipeline work on hold

    Mitigation: Focusing resources on Deramiocel's regulatory pathway.

    Increased net loss due to continued investmentQ2 FY26

    Net loss of $40.7 million in Q2 FY26 vs $25.9 million in Q2 FY25

    Mitigation: Pacing certain commercial expenditures and maintaining flexibility in capital deployment.

    What to watch in Q3 FY26

    5

    Deramiocel BLA amendment submission

    Timing to be finalized
    CurrentPlanned
    TargetSubmission of amendment

    Why it matters

    This submission is critical for advancing Deramiocel's regulatory pathway for a refined indication and securing a new PDUFA date.

    We are finalizing the timing of📎 that submission and will provide an update as appropriate.

    2 min read5 chapters

    Detailed Narrative

    01

    Regulatory Pathway for Deramiocel

    Following a 3-9 negative vote from the FDA Advisory Committee on the cardiomyopathy indication, Capricor plans to submit an amendment to its BLA for Deramiocel. This amendment will focus on an upper limb skeletal muscle indication, leveraging the primary efficacy endpoint data from the HOPE-3 study and 24-month open-label extension data. The FDA has expressed willingness to review this amendment and extend the PDUFA action date from August 22, 2026, accordingly.

    02

    HOPE-3 Data Publication and Statistical Clarification

    The results of the HOPE-3 clinical trial were published in The Lancet, an important milestone. The company clarified a statistical model issue impacting only the left ventricular ejection fraction endpoint, which was a key secondary endpoint. The primary endpoint, upper limb function (PUL 2.0), remains statistically significant with a p-value of 0.029, demonstrating a 4.55% mean difference in favor of Deramiocel.

    03

    Commercial and Manufacturing Readiness

    Capricor is continuing commercial readiness activities for Deramiocel, albeit at a slower pace due to regulatory uncertainty🌐, to control cash. The in-house GMP manufacturing facility in San Diego is operational for initial commercial launch, with an expansion to the second floor expected to be fully validated and FDA-approved in 2027. Michael Maurer has joined as Chief Commercial Officer to build out the launch organization.

    04

    Dispute with NS Pharma

    The company withdrew its motion for preliminary injunction against NS Pharma, opting for arbitration to resolve the contractual dispute regarding the U.S. distribution agreement's pricing structure. Arbitration is estimated to begin in Fall 2026. Capricor's position that the pricing structure is flawed and impedes patient access remains unchanged.

    05

    Pipeline and Life Cycle Management

    All pipeline work not directly related to Deramiocel is currently on hold pending regulatory clarity for the lead asset. However, life cycle management for Deramiocel includes initiated regulatory engagement in Europe and Japan, and expansion plans for younger DMD patients and Becker muscular dystrophy remain priorities, with clinical trial initiations stage-gated by the U.S. regulatory timeline.

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