Core Insights - Capricor Therapeutics is participating in one-on-one investor meetings and providing a corporate update at Leerink's Global Healthcare Conference 2025 from March 10-12, 2025 [1][2] - The company will present updates on its lead program, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD) [2][5] - Capricor is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel being a key product candidate in late-stage development [3] Company Overview - Capricor Therapeutics, Inc. is a biotechnology company listed on NASDAQ (CAPR) that aims to redefine treatment for rare diseases through innovative therapies [3] - The lead product candidate, deramiocel, is an allogeneic cardiac-derived cell therapy showing immunomodulatory, antifibrotic, and regenerative actions [3] - The company is also developing exosome technology using its proprietary StealthX™ platform for various therapeutic applications [3] Strategic Partnerships - Capricor has entered into an exclusive commercialization and distribution agreement for deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5]

Core Insights - The article discusses the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1 - The service offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - A promotional offer is available for new subscribers, including a two-week free trial and a discounted annual subscription price of $399, which represents a 33.50% discount compared to the monthly rate of $49 [1]. Group 2 - The author of the article has no current stock or derivative positions in any of the companies mentioned, nor plans to initiate any positions in the next 72 hours [3]. - The article emphasizes that past performance is not indicative of future results, and no specific investment recommendations are provided [4].

Core Points - The FDA has accepted Capricor Therapeutics Inc's Biologics License Application for deramiocel, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy [1] - The FDA granted Priority Review status with a target action date of August 31, 2025 [1] - No potential review issues have been identified by the FDA [2] Company and Industry Summary - The BLA submission is supported by data from Capricor's Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, which were compared to natural history data [3] - The FDA has not yet determined if an Advisory Committee meeting will be necessary for this application [3] - Deramiocel has received Orphan Drug Designation from both the FDA and the European Medicines Agency [4] - If approved by September 30, 2026, Capricor could receive a Priority Review Voucher due to its previous rare pediatric disease designation [4] - CAPR stock increased by 6.33% to $14.54 following the news [4]

Core Insights - The FDA has accepted Capricor Therapeutics' Biologics License Application (BLA) for deramiocel, targeting Duchenne muscular dystrophy (DMD) cardiomyopathy, with a PDUFA action date set for August 31, 2025 [1][2] - If approved, deramiocel would be the first therapy specifically for DMD cardiomyopathy, a condition currently lacking approved treatments [1][2] - The BLA is supported by data from Capricor's Phase 2 clinical trials, indicating the potential effectiveness of deramiocel in treating DMD cardiomyopathy [2][3] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, which is also recognized under the Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. [3] - Capricor has entered into an exclusive commercialization agreement for deramiocel with Nippon Shinyaku Co., Ltd. for the U.S. and Japan markets, pending regulatory approval [9] Product Details - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory, antifibrotic, and regenerative properties in clinical studies [4] - The therapy is designed to be administered quarterly and aims to provide a lifelong treatment option for patients with DMD cardiomyopathy [2][3] - Clinical trials have demonstrated deramiocel's ability to attenuate DMD cardiomyopathy, which is a leading cause of death in DMD patients [3] Disease Context - Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting approximately 15,000-20,000 individuals in the U.S., characterized by progressive muscle weakness and inflammation [5][6] - The median age of mortality for DMD patients is around 30 years, with heart failure being the leading cause of death due to the disease's impact on heart muscle cells [5][6]

Group 1 - Capricor Therapeutics submitted its Biologics License Application (BLA) for deramiocel, targeting patients with Duchenne muscular dystrophy (DMD) cardiomyopathy [1] - The company highlighted that deramiocel is slowing the progression of cardiac dysfunction in DMD patients [1]

SAN DIEGO, Feb. 10, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the Company is scheduled to participate in the following upcoming investor conferences. Event:Oppenheimer 35thAnnual Healthcare Life Sciences Conference (Virtual)Date:February 12, 2025 from 12:00-12:30 p.m. ETWebcast LinkClick here Event:Barclays 27thAnnual Global Healthcare Conferenc ...

Capricor Therapeutics (CAPR) closed the last trading session at $13.47, gaining 7.6% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $41.50 indicates a 208.1% upside potential.The mean estimate comprises six short-term price targets with a standard deviation of $18.52. While the lowest estimate of $25 indicates an 85.6% increase from the current price level, the most optimistic ana ...

Shares of Capricor Therapeutics (CAPR) gained 8.4% on Thursday following the completion of the submission of a biologics license application (BLA) to the FDA seeking full approval for deramiocel to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. CAPR’s lead product candidate and only clinical pipeline asset, deramiocel, is an investigational, allogeneic cardiac-derived cell therapy. A final decision of the FDA is anticipated in the second half of 2024.Capricor initiated its r ...

-If approved, deramiocel would be first approved therapy for Duchenne muscular dystrophy cardiomyopathy- -BLA submission triggers $10 million milestone payment to Capricor from Nippon Shinyaku- SAN DIEGO, Jan. 02, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and ...

Financial Performance - Capricor Therapeutics reported clinical development income of approximately $2.3 million for Q3 2024, down from $6.2 million in Q3 2023, and $11.1 million for the nine months ended September 30, 2024, compared to $13.1 million for the same period in 2023[148]. - The company has an accumulated deficit of approximately $192.7 million as of September 30, 2024, with net losses of approximately $12.6 million for Q3 2024 and $33.4 million for the nine months ended September 30, 2024[144]. - Cash used in operating activities for the nine months ended September 30, 2024, was approximately $25.2 million, an increase of $11.2 million compared to the same period in 2023[165]. - The company had cash flow provided by financing activities of approximately $71.7 million for the nine months ended September 30, 2024, compared to $2.4 million for the same period in 2023[167]. Research and Development - Research and Development (R&D) expenses for Q3 2024 increased by approximately $1.8 million, or 18%, compared to Q3 2023, driven by various clinical trial costs and personnel expenses[150]. - Total research and development expenses for the three months ended September 30, 2024, increased by $1,778,903, or 18%, compared to the same period in 2023, totaling $11,807,867[151]. - Compensation and other personnel expenses for the nine months ended September 30, 2024, rose by $3,278,017, or 44%, reaching $10,697,603[153]. - Exosomes platform research expenses increased by $951,089, or 61%, for the nine months ended September 30, 2024, totaling $2,518,052[153]. - The company expects to spend approximately $30.0 million to $35.0 million in 2024 on the deramiocel program for DMD, including clinical and manufacturing expenses[159]. - The company anticipates spending approximately $3.0 million to $5.0 million in 2024 on the development of its exosome platform, focusing on preclinical studies and manufacturing[160]. Product Development and Commercialization - Capricor plans to file a Biologics License Application (BLA) for deramiocel by the end of 2024, following a pre-BLA meeting with the FDA in August 2024[138]. - Deramiocel is designed to slow disease progression in Duchenne muscular dystrophy (DMD) through the actions of cardiosphere-derived cells (CDCs) and has shown statistically significant benefits in clinical trials[134][135]. - Capricor has entered into exclusive distribution agreements with Nippon Shinyaku for deramiocel in the U.S. and Japan, supporting its commercialization strategy[139]. - Capricor's StealthX™ vaccine candidate is part of Project NextGen, with plans for a Phase 1 clinical study expected to begin in Q1 2025[141][142]. Funding and Financial Agreements - Capricor received an upfront payment of $30.0 million from Nippon Shinyaku for the U.S. Distribution Agreement for deramiocel[171]. - The first milestone payment of $10.0 million was received upon completion of the interim futility analysis of the HOPE-3 trial[171]. - Capricor has potential milestones totaling up to $90.0 million leading up to BLA approval for deramiocel[171]. - In the Japan Distribution Agreement, Capricor received an upfront payment of $12.0 million and potential additional milestone payments of up to approximately $89.0 million[173]. - Capricor entered into a Binding Term Sheet with Nippon Shinyaku for the commercialization of deramiocel in Europe, with an upfront payment of $20.0 million and potential milestone payments of up to $715.0 million[175]. - In September 2023, Capricor raised approximately $23.0 million through a registered direct offering of 4,935,621 shares at $4.66 per share[176]. - The ATM Program generated approximately $75.0 million in gross proceeds from the sale of 9,228,383 shares at an average price of $8.13 per share[177]. - Capricor entered into a private placement with Nippon Shinyaku for 2,798,507 shares at a price of $5.36 per share, totaling approximately $15.0 million[178]. - In October 2024, Capricor completed a public offering of 5,073,800 shares at a price of $17.00 per share, generating approximately $86.3 million in gross proceeds[180]. Cash and Investment Management - The company has approximately $85.0 million in cash, cash equivalents, and marketable securities as of September 30, 2024, and raised approximately $86.3 million from a public offering post this date[143]. - Total cash, cash equivalents, and marketable securities as of September 30, 2024, were approximately $85.0 million, up from approximately $39.5 million as of December 31, 2023[164]. - As of September 30, 2024, the fair value of the company's cash, cash equivalents, and marketable securities was approximately $85.0 million[213]. - The company's investment portfolio primarily consists of money market funds, including short-term U.S. treasuries, bank savings, and checking accounts[213]. - The company aims to limit credit exposure by investing with highly rated credit issuers to improve the safety and preservation of invested funds[214]. - The company does not hedge interest rate exposure and believes that a hypothetical 100 basis point increase or decrease in interest rates would not significantly impact the fair value of its investment portfolio[214]. Accounting and Financial Reporting - The company recognizes grant income in the period in which the expense is incurred for reimbursable expenses[198]. - The company accounts for CIRM grant disbursements as long-term liabilities, recognizing them as a liability rather than income[200]. - Research and development expenses primarily consist of salaries, clinical trial costs, and other related expenses, which are expensed as incurred[201]. - The company estimates accrued expenses for clinical trials based on contracts with vendors and adjusts recognition if actual results differ from estimates[209]. - Stock-based compensation expense is recorded over the vesting period, with fair value determined using the Black-Scholes option-pricing model[206]. - The company is currently evaluating the impact of recently issued accounting standards on its financial statement disclosures[211].