Capricor’s positive HOPE-3 results prompted Shkreli to call his short thesis a “bad call” after the therapy showed statistically significant skeletal and cardiac benefits.
- Shkreli called his short thesis a “bad call” after Capricor reported statistically significant HOPE-3 results.
- The study showed meaningful improvements in both skeletal and cardiac function with a consistent safety profile.
- Capricor plans to use the data in its response to the FDA’s Complete Response Letter.
Capricor Therapeutics’ (CAPR) positive HOPE-3 results in Duchenne muscular dystrophy prompted former hedge fund manager and pharmaceutical executive Martin Shkreli on Wednesday to admit he made “a bad call” on the stock after previously urging investors to short the stock.
Shkreli Walks Back His Short Call
In a post on X, Shkreli wrote, “while I believe $CAPR is lying about their data, like they have before, this was a bad call and I apologize to anyone who followed me in it. onward and upward!”
His comment marked a notable shift from a month earlier, when he publicly announced a short position and asserted that Deramiocel would fail in the Phase 3 HOPE-3 trial.
Earlier Short Thesis
Shkreli had argued last month that Deramiocel will not yield positive results, calling it an allogeneic cell therapy lacking the ability to reach heart or skeletal muscle. He said most of the cells would become trapped in the lungs rather than reaching diseased tissue, making a clinical effect unlikely.
Shkreli also questioned the safety profile of donor-derived cells and dismissed the earlier HOPE-2 trial outcome as “not meaningful,” claiming the FDA disagreed with the company’s interpretation of that study. He reinforced these points in a 46-page report he co-authored with Anthony Staj, who also predicted the HOPE-3 readout would disappoint.
HOPE-3 Delivers Clear Statistical Wins
Capricor’s new data showed the trial met its primary endpoint on the PUL v2.0 score, an arm strength measure, and its key secondary cardiac endpoint measuring left ventricular ejection fraction, both achieving statistical significance. The company said results demonstrated clinically meaningful improvements in both skeletal and cardiac function, with safety and tolerability consistent with earlier experience.
Capricor said it plans to submit its response to the FDA’s Complete Response Letter incorporating the HOPE-3 data, noting prior alignment with the agency that the new results should be sufficient to support a regulatory filing seeking approval.
Analysts Lift Targets After HOPE-3 Surprise
H.C. Wainwright raised its price target on Capricor to $60 from $24 and reiterated a ‘Buy’ rating, saying the company recovered from its July 2025 Complete Response Letter with a “decisive clinical win.” The brokerage firm said it expects a conservative approval timeline of mid-2026 and believes the shares are likely to trade well into year-end.
Alliance Global also raised its price target, moving it to $48 from $16, while maintaining a ‘Buy’ rating. The firm said the HOPE-3 topline results were “unequivocally and wholly positive” across the 106-patient Duchenne study and removed its speculative designation on the stock.
Stocktwits Traders Celebrate Breakthrough
On Stocktwits, retail sentiment for Capricor was ‘extremely bullish’ amid ‘extremely high’ message volume.
One user rejoiced, “Can you imagine how the kids and parents are celebrating they now have a treatment and a chance to live a longer better life. HAPPY HOLIDAYS to all the families that are the real winners.”
Another user claimed, “20 years investing and 9 years trading. This is the reason I will never short! Ever!”
Capricor’s stock has more than doubled so far in 2025.
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