Welcome to our dedicated page for Capricor Therapeutics news (Ticker: CAPR), a resource for investors and traders seeking the latest updates and insights on Capricor Therapeutics stock.
Capricor Therapeutics develops cell and exosome-based therapeutics for rare diseases, with Deramiocel, an allogeneic cardiac-derived cell therapy, as its lead program for Duchenne muscular dystrophy. Company news commonly covers HOPE-3 clinical data, Biologics License Application communications with the FDA, regulatory designations for Deramiocel, and manufacturing activities tied to the DMD program.
Updates also include financial results, corporate presentations at neurology and muscular dystrophy medical meetings, collaboration revenue references tied to its U.S. Distribution Agreement with Nippon Shinyaku, and preclinical work using the StealthX exosome platform for vaccinology and delivery of oligonucleotides, proteins, and small-molecule therapeutics.
Capricor Therapeutics (CAPR) announced significant developments in Q4 2024 and FY2024. The FDA has accepted their Biologics License Application (BLA) for deramiocel, granting priority review with a PDUFA date of August 31, 2025. The therapy targets Duchenne muscular dystrophy (DMD) cardiomyopathy.
Key financial highlights include:
- Cash position of $151.5M as of December 31, 2024
- Q4 2024 revenues of $11.1M (vs $12.1M in Q4 2023)
- FY2024 revenues of $22.3M (vs $25.2M in 2023)
- Net loss of $7.1M in Q4 2024 ($0.16 per share)
- Current cash expected to support operations into 2027
Clinical data from the HOPE-2 open label extension trial showed a 52% slowing of disease progression over 3 years. The company received a $10M milestone payment from Nippon Shinyaku and expanded its manufacturing capacity in San Diego.
Capricor Therapeutics (NASDAQ: CAPR) announced positive long-term data from its HOPE-2 open label extension trial for deramiocel, showing significant efficacy in treating Duchenne muscular dystrophy (DMD). The data, presented at the 2025 MDA Conference, demonstrated a 52% slowdown in disease progression over three years.
Key findings show patients treated with deramiocel experienced an average decline of 3.46 points in Performance of the Upper Limb (PUL 2.0) total score, compared to 7.19 points in the control group (p=0.019). The treatment effect improved yearly, with mean annual PUL 2.0 declines of 1.8, 1.2, and 1.1 points in Years 1, 2, and 3 respectively.
The drug maintained a favorable safety profile with no new safety signals. The FDA has accepted Capricor's Biologics License Application for DMD-associated cardiomyopathy, with a PDUFA date set for August 31, 2025.
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, has scheduled its fourth quarter and full year 2024 financial results announcement for March 19, 2025, after market close.
The company will host a conference call and webcast at 4:30 p.m. ET on the same day to discuss the results. A replay of the webcast will be made available on the company's website following the live broadcast.
Capricor Therapeutics (NASDAQ: CAPR) has received FDA acceptance for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy targeting Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA has granted Priority Review with a PDUFA target action date of August 31, 2025.
NS Pharma, a subsidiary of Nippon Shinyaku, will be exclusively responsible for the U.S. commercialization and distribution of deramiocel, following their exclusive distribution agreement established in January 2022. The FDA has not identified any potential review issues at this time.
Capricor Therapeutics (NASDAQ: CAPR), a biotech company focused on developing cell and exosome-based therapeutics for rare diseases, has announced its participation in Leerink's Global Healthcare Conference 2025. The conference will take place from March 10-12, 2025, in Miami, Florida.
The company's management will engage in one-on-one investor meetings and deliver a corporate presentation on Wednesday, March 12, 2025, from 1:00-1:40 p.m. ET. During the presentation, Capricor will provide updates on their lead program deramiocel, which targets the treatment of Duchenne muscular dystrophy (DMD), along with other scientific and corporate developments.
Capricor Therapeutics (NASDAQ: CAPR) announced FDA acceptance and Priority Review of its Biologics License Application (BLA) for deramiocel, a cell therapy treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA set a PDUFA target action date of August 31, 2025.
If approved, deramiocel would be the first therapy specifically targeting DMD cardiomyopathy. The treatment would be administered quarterly as a lifelong therapy. The BLA submission is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared with FDA-funded natural history data.
The FDA has not identified any potential review issues and has not yet decided on the need for an Advisory Committee meeting. Deramiocel has received Orphan Drug Designation from both FDA and EMA, along with RMAT designation in the U.S. and ATMP Designation in Europe.
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, has announced its participation in three major upcoming investor conferences. The company will be presenting at:
• The Oppenheimer 35th Annual Healthcare Life Sciences Conference (Virtual) on February 12, 2025, from 12:00-12:30 p.m. ET
• The Barclays 27th Annual Global Healthcare Conference in Miami, FL (March 11-13, 2025)
• The 37th Annual ROTH Conference in Dana Point, CA (March 16-18, 2025)
During these conferences, Capricor's management will provide updates on their lead program, deramiocel, for treating Duchenne muscular dystrophy (DMD), along with general scientific and corporate updates.
Capricor Therapeutics (NASDAQ: CAPR) has completed its Biologics License Application (BLA) submission to the FDA for deramiocel, seeking full approval for treating Duchenne muscular dystrophy (DMD) cardiomyopathy. The submission triggers a $10 million milestone payment from Nippon Shinyaku.
The BLA is supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared with FDA-funded natural history data. Capricor has requested priority review, which could reduce the review timeline from 10 to 6 months.
Deramiocel has received Orphan Drug Designation from both FDA and EMA, RMAT Designation in the U.S., and ATMP Designation in Europe. If approved, it would be the first therapy for DMD cardiomyopathy, and Capricor would be eligible for a Priority Review Voucher based on its rare pediatric disease designation.
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on rare disease treatments through cell and exosome-based therapeutics, has announced its participation in two upcoming investor conferences. The company will present at the Piper Sandler 36th Annual Healthcare Conference on December 3, 2024, from 11:30-11:55 a.m. ET, featuring a fireside chat, industry panel, and one-on-one meetings. Additionally, Capricor will participate in the Oppenheimer Movers in Rare Disease Summit on December 12, 2024, from 10:00-10:50 a.m. ET. The Piper Sandler presentation will be available via webcast on Capricor's website.
Capricor Therapeutics (NASDAQ: CAPR) has received both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations from the European Medicines Agency for deramiocel, its lead asset targeting Duchenne muscular dystrophy (DMD). The Orphan Drug status grants 10-year market exclusivity upon approval and reduced regulatory fees, while ATMP designation provides regulatory support for cell-based therapy development. The company has also initiated a rolling Biologics License Application with the FDA, expecting completion by end of 2024, seeking full approval for deramiocel in treating DMD-cardiomyopathy.