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The call conveyed strong clinical and regulatory progress for deramiocel — statistically significant phase 3 efficacy (primary and cardiac secondary endpoints), robust long-term safety exposure, active BLA review with a clear PDUFA date, in-house manufacturing capabilities and a solid cash runway. Management also articulated a concrete commercialization plan and eligibility for a monetizable Priority Review Voucher. Offsetting these positives are notable near-term risks: an active lawsuit with NS Pharma that creates distribution uncertainty (and potential need to repay $50M), rising operating expenses and no current product revenue, manufacturing labeling constraints for an ultra-cold-chain therapy, and unresolved ASP/AMP payer complexities that could affect access. On balance, the material clinical/regulatory achievements and internal control of manufacturing and cash resources outweigh the legal and commercial execution risks, but the litigation and payer/pricing issues remain important near-term uncertainties.
Management said the deramiocel BLA resubmission (classified as a Class 2, accepted as complete) is under active FDA review with a PDUFA target action date of August 22, 2026 and near‑term labeling discussions expected; they highlighted HOPE‑3 Phase 3 results from 106 patients (1:1 randomization) showing ~54% reduction in disease severity on PUL v2.0 at 12 months (p=0.029), LVEF slowing of ~91% in all evaluable patients and ~120% in the cardiomyopathy subset (p=0.01), noted >800 IV infusions completed and ~90 open‑label extension patients with up to 5 years of dosing supporting safety, and said FDA information requests have been primarily CMC/clinical clarifications; commercial and manufacturing guidance included an in‑house PLI‑inspected facility able to support ~200–250 patients/year (≈1,000 doses/year at 4 doses/patient), a planned expansion to ~2,000–2,500 patients/year (~10,000 doses/year) with full validation targeted in 2027, plans to stockpile commercial doses once labeling is set (including an interim label solution for the ultracold product), upcoming EMA/PMDA engagements, potential receipt/monetization of a Priority Review Voucher upon approval, and financials of ~$279M cash (3/31/2026) with Q1 2026 operating expenses of ~$36.8M (vs ~$25M in Q1 2025) and a Q1 2026 net loss of ~$33.9M ($0.59/share), which management believes provides runway into 2027.
HOPE-3 (n=106) met its primary endpoint (PUL v2.0) with ~54% reduction in disease severity at 12 months (p=0.029). All type-1-error controlled secondary endpoints were also significant, supporting clinical benefit in Duchenne muscular dystrophy (DMD).
Left ventricular ejection fraction (LVEF) showed ~91% slowing of disease progression in all evaluable patients and ~120% slowing in the subset with confirmed cardiomyopathy (p=0.01), supporting deramiocel's potential to address DMD-associated cardiomyopathy.
Biologics License Application (BLA) resubmission accepted as a Class 2 filing; FDA active review with a PDUFA target action date of August 22, 2026. Multiple information requests have been addressed and labeling discussions are anticipated.
Deramiocel safety profile supported by >800 intravenous infusions across studies and ~90 patients in open-label extension (OLE) studies with some patients receiving continuous infusions up to 5 years, reinforcing long-term tolerability.
In-house GMP facility in San Diego completed FDA pre-license inspection with observations addressed; current capacity to support ~200–250 patients/year (≈1,000 doses/year) with staged expansion to support ~2,000–2,500 patients/year (≈10,000 doses/year). Full validation target: 2027.
Cash, cash equivalents and marketable securities of approximately $279 million as of 03/31/2026. Management believes this capital is sufficient to fund anticipated operating expenses and capital expenditures into 2027 (excludes potential product revenue or Priority Review Voucher monetization).
Company expects eligibility for a transferable Priority Review Voucher upon potential approval, representing a potentially meaningful, monetizable source of non-dilutive capital.
Ongoing work on exosome-based therapeutics (StealthX/HEK293 exosomes) and plans for lifecycle management of deramiocel including expansion to younger DMD patients and a planned clinical pathway for Becker muscular dystrophy; EMA and PMDA engagements expected later in the year.
Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics First Quarter 26 Conference Call. At this time, all participant lines are in listen only mode. Following the presentation, we will conduct a question and answer session. If at any time during this call you require immediate assistance, please press 0 for the operator. This call is being recorded on Tuesday, May 12, 2026. I would now like to turn the conference over to our CFO, AJ Bergmann, for the forward looking statements. Please go ahead.
Thank you, and good afternoon, everyone. Before we start, I would like to state that we will be making certain forward looking statements during today's presentation. These statements may include statements regarding among other things, the efficacy, safety and intended utilization of our product candidates potential regulatory developments involving our product candidates, our future R&D plans, our anticipated conduct and timing of preclinical and clinical studies, enrollment of patients in our clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, our financial position, our possible uses of existing cash investment resources, and statements regarding our litigation with Nippon Shinyaku and NS Pharma, Inc. Including the nature of the dispute, our expectations regarding any legal proceedings and our ability to commercialize deramiocel independent of our existing distribution agreeme...