Capricor Therapeutics (NASDAQ: CAPR) faced its Prescription Drug User Fee Act (PDUFA) target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived allogeneic cell therapy, not a peptide) in Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, with left ventricular ejection fraction (LVEF) endpoint outcomes appearing highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome, and CAPR stock slipped in advance of the decision on elevated volume as investors positioned for the anticipated regulatory rejection. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the DMD population. The DMD cardiomyopathy indication remains without an FDA-approved therapy. Capricor holds Rare Pediatric Disease Designation for deramiocel, which may qualify the company for a Priority Review Voucher upon eventual approval if it comes.
Capricor Therapeutics (NASDAQ: CAPR) faced its PDUFA target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived allogeneic cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, with left ventricular ejection fraction (LVEF) endpoint outcomes appearing highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the DMD population. The DMD cardiomyopathy indication remains without an FDA-approved therapy, which continues the substantial unmet need in the space. Capricor holds Rare Pediatric Disease Designation for deramiocel, which may qualify the company for a Priority Review Voucher upon eventual approval if it comes.
Capricor Therapeutics (NASDAQ: CAPR) faces its Prescription Drug User Fee Act (PDUFA) target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, saying that left ventricular ejection fraction (LVEF) endpoint outcomes appeared highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the population. The DMD cardiomyopathy indication remains without an FDA-approved therapy.
PepGen announced in May 2026 a strategic pivot away from Duchenne muscular dystrophy after the 10 mg/kg cohort of CONNECT1-EDO51 produced only 0.59% of normal dystrophin levels in four patients — well below the threshold of clinical meaningfulness. The company will discontinue DMD program development and focus on the FREEDOM2-DM1 Phase 2 program in myotonic dystrophy type 1, with 5 mg/kg cohort data anticipated. PepGen's Enhanced Delivery Oligonucleotide (EDO) platform conjugates peptide carriers to phosphorodiamidate morpholino oligomers (PMOs) for tissue-targeted delivery. The DM1 program competes with Vertex's VX-670 cyclic peptide-oligonucleotide conjugate (GALILEO Phase 1/2, H2 2026 readout) and Sarepta/Avidity Biosciences in the same indication.
Entrada Therapeutics announced May 7 positive topline results from Cohort 1 of the Phase 1/2 ELEVATE-44-201 study of ENTR-601-44, the first-in-class Endosomal Escape Vehicle (EEV) peptide-PMO conjugate for exon-44-amenable Duchenne muscular dystrophy. At 6 mg/kg, treated participants showed mean dystrophin increase of 2.36% from a 4.00% baseline, exon-skipping increase of 2.31% from a 2.66% baseline, and a statistically significant improvement in Time-to-Rise (TTR) velocity. No serious adverse events or treatment-driven discontinuations; the most common AE was headache. All eight Cohort 1 participants transitioned to the open-label portion. Cohort 2 (12 mg/kg) is now dosing, with year-end 2026 readouts planned for Cohort 1 OLE and Cohort 2 MAD; Cohort 3 (up to 18 mg/kg) follows.