Shares of biotech company Capricor had their best day in a month. What inspired investors?

Shares of small-cap biotech company Capricor Therapeutics jumped 10.5% on September 30, marking its best performance in more than a month. Investors reacted to updated data from clinical trials of the company’s lead drug candidate, which is intended to treat Duchenne muscular dystrophy, a rare disease.

Capricor shares rose 10.5% on the Nasdaq on September 30, reaching $9.5. This marked their best performance in more than a month, according to the Stocktwits website.

At the World Society of Muscular Diseases Congress in Japan, the company presented updated data on its experimental drug, deramioxel, for Duchenne muscular dystrophy. This rare genetic disorder, which primarily affects boys, leads to gradual muscle weakness and death. Currently, there is only one drug available for its treatment—from Sarepta Therapeutics—but its use is severely limited.

According to Stocktwits, information about Deramiocel is currently being presented at a conference in the form of a poster that is being widely shared on the social media platform X. In particular, the photo was posted by Eduardo Marban, whose scientific work forms the basis for Capricor’s developments. He is also the husband of the company’s CEO, Linda Marban.

The image shows data from a two-year follow-up of patients who took Deramioxel. The study evaluates the function of their upper extremities.

The company is scheduled to present an oral report on deramiocel on October 3. And on November 22, the U.S. Food and Drug Administration (FDA) plans to issue a decision on the approval of deramiocel.

This is not the first time Capricor has attempted to convince the regulator of the need to approve deramioxel. In July 2025, the FDA rejected the initial application due to a lack of substantial evidence of efficacy. Earlier this year, Capricor resubmitted its application, but an advisory committee to the regulator voted against it in late July. The FDA was scheduled to make a final decision on August 22, but the company provided the agency with updated data from a two-year patient follow-up and asked officials to consider revising the labeling: Capricor’s first two applications emphasized the efficacy of deramiocel in cardiomyopathy (changes in the heart muscle), while the updated application focused on preserving upper limb function. The regulator deemed this data substantial and postponed the decision until November 22.

Against this backdrop, in early September, two Wall Street analysts—from Piper Sandler and B. Riley Securities— recommended that investors buy Capricor stock. At the same time, they raised their price targets for the company’s stock significantly: Piper Sandler raised its target from $2 to $25, and B. Riley Securities raised its target from $5 to $21.

However, Wall Street as a whole is taking a wait-and-see approach to the company’s stock for now: it has six “buy” recommendations from analysts, with four more advising investors to buy the stock. The average price target is $33.56, which implies upside potential of more than 253% relative to the most recent closing price.