Latest news with #Deramiocel
Business Insider
19 hours ago
- Business
- Business Insider
Capricor Therapeutics Stock (CAPR) Plummets 40% on FDA Rejection
Capricor Therapeutics (CAPR) stock dove on Friday after the biotechnology company revealed a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA). This letter covered its Biologics License Application (BLA) for Deramiocel, a cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). That's a severe genetic disorder that causes progressive muscle weakness and degeneration. Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week. The big news here is that the FDA completed its review of the BLA for Deramiocel and has rejected the therapy in its current form. The FDA said it 'does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data.' The FDA is willing to restart its review with additional data and has granted Capricor Therapeutics a Type A meeting to discuss further action. Capricor Therapeutics CEO Linda Marbán noted that the CRL from the FDA was a surprise. She claimed that the company followed the FDA's guidance throughout the submission process and that no issues were revealed ahead of the CRL. She said the company will 'submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study.' Capricor Therapeutics Stock Movement Today CAPR stock was down 38.51% in pre-market trading on Friday, following a slight 0.18% dip yesterday. The shares were also down 17.39% year to date, but remained up 159.09% over the past 12 months. Today's news triggered a stock selloff, with some 4 million shares traded this morning, compared to a three-month daily average of about 2.59 million units. Is Capricor Therapeutics Stock a Buy, Sell, or Hold? Turning to Wall Street, the analysts' consensus rating for Capricor Therapeutics is Strong Buy, based on nine Buy and one Hold rating over the past three months. With that comes an average CAPR stock price target of $30.60, representing a potential 167.42% upside for the shares.
Business Wire
a day ago
- Business
- Business Wire
Capricor Therapeutics, Inc. (NASDAQ: CAPR) Securities Fraud Investigation; CAPR Investors Urged to Contact Award-Winning Firm, Gibbs Mura
BUSINESS WIRE)--Shares of Capricor Therapeutics, Inc. plunged over 30% in intraday trading on Friday, July 11, 2025, after the U.S. Food and Drug Administration rejected the company's Biologics License Application for its lead cell therapy candidate, Deramiocel, claiming that the application 'does not meet the statutory requirement for substantial evidence of effectiveness' and lacks 'certain outstanding items.' Gibbs Mura is investigating a potential Capricor Therapeutics, Inc. (NASDAQ: CAPR) Securities Class Action Lawsuit on behalf of shareholders who lost money in Capricor Therapeutics, Inc. What Should Capricor Therapeutics, Inc. Investors Do? If you invested in Capricor Therapeutics, Inc., visit our Capricor Therapeutics, Inc. Investigation webpage or call us at (888) 410-2925 to get more information about how you may be able to recover your losses. Our investigation concerns whether Capricor Therapeutics, Inc. has violated federal securities laws by providing false or misleading statements to investors. What is the Capricor Therapeutics, Inc. (CAPR) Lawsuit Investigation About? On July 11, 2025, Capricor Therapeutics announced that the FDA rejected the company's Biologics License Application (BLA) for its cell therapy candidate, Deramiocel, citing that the application 'does not meet the statutory requirement for substantial evidence of effectiveness' and lacks 'certain outstanding items.' The FDA's Complete Response Letter specified that the deficiencies were in the BLA's Chemistry, Manufacturing, and Controls section, and emphasized a need for additional clinical data. Following this news, shares of Capricor Therapeutics, Inc. plunged over 30% in intraday trading on July 11, 2025, causing harm to investors. Previously, on May 5, 2025, Capricor announced that the FDA 'confirmed its intent to hold an advisory committee meeting' following the completion of a mid-cycle review meeting for the company's BLA for Deramiocel. Following the announcement, Capricor's stock fell over 29% on May 6, 2025. Then, on June 20, 2025, STAT reported that the director of the FDA's Center for Biologics Evaluation and Research canceled the advisory committee meeting regarding Deramiocel, due to being 'skeptical of the treatment.' In the wake of the report, Capricor's stock fell over 30% on June 20, 2025. About Gibbs Mura Gibbs Mura represents investors nationwide in securities litigation. The firm has recovered over a billion dollars for its clients against some of the world's largest corporations, and our attorneys have received numerous honors for their work, including 'Best Lawyers in America,' 'Top Plaintiff Lawyers in California,' 'California Lawyer Attorney of the Year,' 'Class Action Practice Group of the Year,' 'Consumer Protection MVP,' and 'Top Women Lawyers in California.' This press release may constitute Attorney Advertising in some jurisdictions under the applicable law and ethical rules.
Yahoo
a day ago
- Business
- Yahoo
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial in Q3 2025 to continue pursuing the indication for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy FDA advised Capricor to request a meeting to determine next steps toward potential approval Conference call and webcast scheduled for today at 8:30 a.m. ET SAN DIEGO, July 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). In the CRL, the FDA stated that it had completed its review of the application but is unable to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. The CRL also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application, most of which Capricor believes it has addressed in prior communications to the FDA. However, these materials were not reviewed by the FDA due to the timing of the CRL issuance. The FDA confirmed that it will restart the review clock upon resubmission. In addition, the agency offered the company the opportunity to request a Type A meeting to discuss the path forward. Capricor plans to engage further with the FDA to determine the appropriate next steps. Capricor's BLA for Deramiocel was granted Priority Review in March 2025 and was supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets. 'We are surprised by this decision by the FDA. We have followed their guidance throughout the process. Prior to the CRL, the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review,' said Linda Marbán, Ph.D., CEO of Capricor. 'Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study. The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025. We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD. While this was an unexpected decision by the FDA, we remain committed to the DMD community to get Deramiocel through the approval process.'To participate in the conference call, please dial 1-800-717-1738 (Domestic) or 1-646-307-1865 (International) and reference the conference ID: 30613. Participants may dial in using the numbers above to speak with an operator or click the Call me™ link for instant access. To participate via webcast, click here to access the live stream. A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company's is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two cohorts evaluating the safety and efficacy of Deramiocel in participants with DMD. Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either Deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. Approximately 104 eligible study subjects have been enrolled in the dual-cohort study. For more information on this study, please visit (NCT05126758).Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown Deramiocel to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. Deramiocel is currently in late-stage development for the treatment of Duchenne Muscular Dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit and follow Capricor on Facebook, Instagram and in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words 'believes,' 'plans,' 'could,' 'anticipates,' 'expects,' 'estimates,' 'should,' 'target,' 'will,' 'would' and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 2025. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug (IND) and is not yet approved for any indications. Neither BMD nor any of Capricor's exosome-based candidates have been approved for clinical Media Contact:Raquel ConaKCSA Strategic Communications rcona@ Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@
Yahoo
25-06-2025
- Business
- Yahoo
No Ad Comm ‘not necessarily positive' for Capricor, says Roth Capital
Roth Capital analyst Boobalan Pachaiyappan tells investors in a research note that the FDA's intention not to require an Ad Comm 'at this time' for deramiocel for the treatment of cardiomyopathy associated with Duchenne Muscular Dystrophy 'may not necessarily be positive news,' and that it believes a promissory Ad Comm meeting would be positive, as a potential positive Ad Comm panel vote would put pressure on the new leadership to approve deramiocel in its current form. The firm reiterates a Buy rating and $31 price target on Capricor shares. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on CAPR: Disclaimer & DisclosureReport an Issue Capricor's deramiocel moves ahead without AdCom, says H.C. Wainwright HHS spokesperson tells Reuters FDA 'actively re-evaluating' Capricor AdCom Capricor Therapeutics provides regulatory update on Deramiocel BLA Roth says 'excessive' Capricor selloff due to 'investor overreaction' Capricor selloff brings buying opportunity, says H.C. Wainwright Sign in to access your portfolio
Yahoo
25-06-2025
- Business
- Yahoo
Capricor's deramiocel moves ahead without AdCom, says H.C. Wainwright
Capricor on Tuesday disclosed that the FDA has indicated that no AdCom is necessary for deramiocel's Biologics License Application, and an in-person late-cycle review meeting is scheduled for mid-July, H.C. Wainwright analyst Joseph Pantginis tells investors in a research note. The firm, which has a Buy rating and $77 price target on the shares, highlights that even in bear case scenarios with the FDA, Phase 3 HOPE-3 data readouts are 'waiting in the wings' to further strengthen deramiocel's overall clinical and regulatory package and thinks deramiocel's first-in-class therapeutic profile in tandem with strong manufacturing support will get the therapy over the finish line on expected timelines. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on CAPR: Disclaimer & DisclosureReport an Issue HHS spokesperson tells Reuters FDA 'actively re-evaluating' Capricor AdCom Capricor Therapeutics provides regulatory update on Deramiocel BLA Roth says 'excessive' Capricor selloff due to 'investor overreaction' Capricor selloff brings buying opportunity, says H.C. Wainwright Capricor Therapeutics price target lowered to $22 from $43 at Oppenheimer
