Key Highlights
- Shares of CAPR jumped more than 100% during premarket hours on Friday following news that Capricor will file an amended BLA for its drug candidate Deramiocel
- Last month, an FDA advisory committee delivered a 9-3 vote against endorsing Deramiocel for cardiomyopathy associated with Duchenne muscular dystrophy
- The biotech company is shifting focus to an upper limb skeletal muscle indication, backed by 24-month open-label extension study results
- Federal regulators have indicated willingness to evaluate the amended submission and will postpone the PDUFA action date accordingly
- Legal action has been initiated against Capricor through a securities fraud class action, with September 28, 2026 set as the deadline for lead plaintiff applications
Shares of Capricor Therapeutics (CAPR) experienced explosive growth in Friday’s premarket session, rocketing upward by more than 100% following the company’s announcement regarding an amended Biologics License Application for Deramiocel, its primary drug candidate.
Capricor Therapeutics, Inc., CAPR
This dramatic rally follows a turbulent period for the biotechnology firm. Just weeks ago, on July 27, 2026, CAPR experienced a devastating 64.5% decline, plummeting from $19.70 to $7.00 per share. The collapse occurred after the Food and Drug Administration published briefing materials highlighting concerns regarding modifications to the company’s statistical analysis methodology prior to a scheduled advisory committee review.
The challenges intensified on July 29 when the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee delivered a disappointing 9-3 vote against endorsing Deramiocel as a treatment for cardiomyopathy in patients with Duchenne muscular dystrophy. The negative vote triggered an additional 36% stock decline, with shares falling from $6.57 to $4.19.
Currently, the BLA remains under regulatory review by the FDA, with an anticipated decision deadline of August 22.
During Capricor’s second quarter 2026 earnings conference call, CEO Linda Marbán provided critical context regarding the advisory committee’s evaluation. She emphasized that cardiomyopathy represented a secondary endpoint in the Phase 3 HOPE-3 clinical trial, rather than the study’s primary objective.
“The Advisory Committee was not asked to vote on whether they believe the data on the HOPE-3 primary efficacy endpoint could support approval of the product, nor whether the overall benefit-risk profile of Deramiocel was favorable,” Marbán said.
Strategic Pivot to Alternative Indication
Capricor has outlined its intention to seek regulatory approval for an upper limb skeletal muscle indication, which directly corresponds with the primary efficacy endpoint evaluated in the HOPE-3 trial. The amended BLA submission will incorporate 24-month data from open-label extension studies along with supplementary analyses drawn from the company’s comprehensive data repository.
Federal regulators have communicated their readiness to assess the amended application and confirmed they will adjust the PDUFA action date timeline once the submission is formally received.
Legal Challenges Emerge
Despite the positive market reaction, Capricor faces mounting legal challenges. The securities litigation firm Bleichmar Fonti & Auld has initiated a class action lawsuit targeting both the company and select senior management members.
The complaint contends that Capricor disseminated misleading information regarding Deramiocel and the scientific rigor of clinical evidence supporting the BLA. Additionally, the lawsuit claims the company concealed the fact that it implemented modifications to its pre-established statistical analysis framework without securing FDA concurrence prior to resubmitting the biologics license application.
The legal proceedings are underway in the U.S. District Court for the Southern District of California. Affected investors have until September 28, 2026 to petition for appointment as lead plaintiff in the action.
The existing PDUFA target decision date stands at August 22, though the forthcoming amended submission is expected to necessitate an extension of this timeline.
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