Capricor Therapeutics (NASDAQ: CAPR) is developing Deramiocel (CAP-1002) as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 3 HOPE-3 trial has been completed, and topline data are expected to be reported in mid-Q4 2025 to support a BLA resubmission (1).
We believe CAP-1002 is unlikely to be effective in treating DMD; therefore, the upcoming Phase 3 readout will likely be negative, causing the company’s stock price to decline. Since this is the only drug in Capricor’s pipeline close to commercialization, and there are no other near-term assets within the platform, we believe the company’s valuation will fall toward its cash balance with marginal, if any, salvage value. Based on the latest Q2 2025 10-Q filing, we estimate that following the readout, the stock could trade around $1.62.
| Ticker | CAPR |
|---|---|
| MC | 346M |
| Price（2025-10-31） | $6.43 |
| Target Price | $2.16 |
| Downside | -66% |
Capricor develops cell- and exosome-based medicines for rare diseases. Its lead asset CAP-1002 is an allogeneic cardiosphere-derived cell (CDC) therapy for DMD that acts primarily through secreted exosomes with immunomodulatory and antifibrotic effects.
The company’s broader technology base includes StealthX™, a proprietary platform for engineering exosomes for targeted delivery and vaccinology (COVID-19). Note that these programs are still in preclinical or evaluation stages, with no near-term clinical catalysts or revenue potential.
The CDC technology originates from academic research by scientific founder Eduardo Marbán, who studied cardiac progenitor cells. The CEO of the company is Linda Marbán.
The team also includes Suzanne Hendrix of Pentara, who previously served as the statistician for Cassava Sciences.
NeurologyLive. (2021, December 13). The Duchenne muscular dystrophy (DMD) continuum: The case for early intervention and long-term treatment. Retrieved October 12, 2025, from https://www.neurologylive.com/view/the-duchenne-muscular-dystrophy-dmd-continuum-the-case-for-early-intervention-and-long-term-treatment
Disease progression:
Mutations in the DMD gene disrupt the production of the muscle protein dystrophin. These mutations often introduce premature stop codons, causing truncated, nonfunctional proteins.
The normal process of dystrophin synthesis proceeds as follows:
DMD gene (2.4 Mb, DNA) → transcription by RNA polymerase II → pre-mRNA (containing introns and exons) → splicing and processing → mature mRNA (exported to the cytoplasm) → translation by ribosomes → dystrophin protein (427 kDa).