Financial Performance - Revenues for Q4 2024 were approximately $11.1 million, a decrease from $12.1 million in Q4 2023; total revenues for the year were approximately $22.3 million, down from $25.2 million in 2023[10] - Total operating expenses for Q4 2024 were approximately $18.8 million, up from $13.4 million in Q4 2023; annual operating expenses increased to approximately $64.8 million from $49.3 million[11] - The net loss for Q4 2024 was approximately $7.1 million, or $0.16 per share, compared to a net loss of $0.8 million, or $0.02 per share, in Q4 2023; the annual net loss was approximately $40.5 million, or $1.15 per share, compared to $22.3 million, or $0.83 per share, in 2023[12] - Comprehensive loss for the year ended December 31, 2024, was $(39,676,044), compared to $(22,156,973) in 2023, indicating a worsening financial performance[26] - The company reported a net loss per share of $(0.16) for Q4 2024, compared to $(0.02) for Q4 2023, highlighting increased losses on a per-share basis[26] Cash and Liquidity - Capricor reported a cash position of approximately $151.5 million as of December 31, 2024, compared to $39.5 million at the end of 2023, reflecting a significant increase in liquidity[9] - Capricor received a $10 million milestone payment from Nippon Shinyaku, contributing to a cash balance expected to support operations into 2027[9] - Cash, cash equivalents, and marketable securities rose to $151,515,877 as of December 31, 2024, compared to $39,487,703 at the end of 2023, indicating improved liquidity[28] Assets and Liabilities - Total assets increased to $170,481,086 in 2024, up from $58,734,327 in 2023, reflecting strong growth in the company's financial position[28] - Total liabilities decreased to $25,018,750 in 2024 from $36,132,860 in 2023, showing a reduction in financial obligations[28] - Total stockholders' equity increased to $145,462,336 in 2024 from $22,601,467 in 2023, indicating a significant improvement in the company's equity position[28] Research and Development - Research and development expenses increased to $14,554,936 in Q4 2024, up 46% from $9,940,167 in Q4 2023[26] - The company is advancing its StealthX™ exosome-based vaccine for SARS-CoV-2, with plans for regulatory approval in Q2 2025[14] Regulatory Developments - The FDA accepted Capricor's Biologics License Application (BLA) for deramiocel, with a target action date set for August 31, 2025, and granted priority review status[5] - Positive data from the HOPE-2 trial indicated a 52% slowing of disease progression in patients treated with deramiocel over three years[5] - The European Medicines Agency granted deramiocel Orphan Drug and Advanced Therapy Medicinal Product designations, providing market exclusivity and regulatory benefits[6] - Capricor expanded its manufacturing capacity for deramiocel by amending its lease for additional GMP space in San Diego[6]

Core Insights - Capricor Therapeutics is advancing towards the market introduction of deramiocel, a cellular therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with a Biologics License Application (BLA) accepted by the FDA and a target action date set for August 31, 2025 [2][5][4] Financial Performance - As of December 31, 2024, the company reported cash, cash equivalents, and marketable securities totaling approximately $151.5 million, a significant increase from approximately $39.5 million at the end of 2023 [7][25] - Revenues for Q4 2024 were approximately $11.1 million, a decrease from $12.1 million in Q4 2023; total revenues for the year were approximately $22.3 million compared to $25.2 million in 2023 [8] - Total operating expenses for Q4 2024 were approximately $18.8 million, up from $13.4 million in Q4 2023; for the full year, operating expenses were approximately $64.8 million compared to $49.3 million in 2023 [9] - The net loss for Q4 2024 was approximately $7.1 million, or $0.16 per share, compared to a net loss of $0.8 million, or $0.02 per share, in Q4 2023; for the full year, the net loss was approximately $40.5 million, or $1.15 per share, compared to $22.3 million, or $0.83 per share, in 2023 [10][11] Clinical Developments - The FDA has granted priority review for the BLA of deramiocel, which is based on positive data from the HOPE-2 trial showing a 52% slowing of disease progression in treated patients [4][6] - The company has expanded its manufacturing capacity to support the production of deramiocel, with an amendment to its lease for additional GMP space in San Diego [5] - The European Medicines Agency has granted deramiocel Orphan Drug and Advanced Therapy Medicinal Product designations, which provide regulatory advantages and potential market exclusivity [5] Strategic Partnerships - Capricor has an exclusive commercialization and distribution agreement for deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., which includes milestone payments that have contributed to the company's cash position [20][7]

Core Insights - Capricor Therapeutics presented positive long-term data from its HOPE-2 clinical trial, showing that deramiocel can significantly slow disease progression in patients with Duchenne muscular dystrophy (DMD) [1][2] - The data indicates a 52% reduction in disease progression, with an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points over three years for treated patients compared to 7.19 points in the external comparator group [2] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead asset [1][8] - The company has received FDA acceptance for its Biologics License Application (BLA) for deramiocel, targeting cardiomyopathy associated with DMD, with a PDUFA action date set for August 31, 2025 [2][10] Treatment Efficacy - Patients treated with deramiocel showed a mean annual decline in PUL 2.0 scores of 1.8 points in Year 1, 1.2 points in Year 2, and 1.1 points in Year 3, indicating a year-over-year improvement in treatment effect [6] - During a one-year gap in treatment, patients originally on deramiocel experienced a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year) [6] Safety Profile - Deramiocel has demonstrated a favorable safety profile, being well tolerated with no new safety signals identified [6] Background on DMD - Duchenne muscular dystrophy is a severe genetic disorder leading to progressive muscle weakness and has a median mortality age of approximately 30 years, affecting about 15,000-20,000 individuals in the U.S. [5][7]

Core Viewpoint - Capricor Therapeutics is set to release its financial results for Q4 and the full year of 2024 on March 19, 2025, followed by a conference call to discuss the results and recent corporate updates [1][2]. Company Overview - Capricor Therapeutics, Inc. is a biotechnology company focused on developing cell and exosome-based therapeutics aimed at treating rare diseases [3]. - The company's lead product candidate, deramiocel, is an allogeneic cardiac-derived cell therapy currently in late-stage development for Duchenne muscular dystrophy [3]. - Capricor is also utilizing its proprietary StealthX™ platform for exosome technology in preclinical development, targeting areas such as vaccinology and targeted delivery of therapeutics [3]. Conference Call Details - The conference call will take place on March 19, 2025, at 4:30 p.m. ET, with toll-free and international dial-in options provided for participants [2]. - A replay of the webcast will be available on the company's website after the live broadcast [2]. Recent Developments - Capricor has entered into an exclusive commercialization and distribution agreement for deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [6].

Core Insights - NS Pharma, a subsidiary of Nippon Shinyaku, has received FDA acceptance for the Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with a target action date of August 31, 2025 [1][2] - The FDA has granted Priority Review status for deramiocel, indicating a potential expedited review process [1] - NS Pharma is exclusively responsible for the commercialization and distribution of deramiocel in the U.S. under an agreement with Capricor Therapeutics [2] About Deramiocel - Deramiocel is composed of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies [3] - CDCs function by secreting exosomes that target macrophages, promoting a healing phenotype rather than a pro-inflammatory one [3] - Over 100 peer-reviewed publications have focused on CDCs, which have been administered to more than 200 human subjects in various clinical trials [3] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy primarily affects males and leads to progressive weakness and loss of skeletal, cardiac, and respiratory muscles [4] - Early symptoms include delayed motor skills, with significant mobility loss often requiring wheelchair use by adolescence [4] - Cardiac and respiratory complications typically arise in the teenage years, posing serious health risks [4] About Capricor Therapeutics - Capricor Therapeutics is a biotechnology company focused on cell and exosome-based therapeutics for rare diseases [5] - The company is developing its proprietary StealthX™ platform for targeted delivery of therapeutics, including oligonucleotides and proteins [5] About NS Pharma - NS Pharma is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd., dedicated to advancing treatments for rare diseases [6]

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) along with other scientific and corporate updates [2] Company Overview - Capricor Therapeutics, Inc. is focused on developing cell and exosome-based therapeutics aimed at treating rare diseases, with deramiocel being its lead product candidate [3] - Deramiocel is an allogeneic cardiac-derived cell therapy that has shown immunomodulatory, antifibrotic, and regenerative actions, specifically for dystrophinopathies and heart disease [3] - The company is also utilizing its proprietary StealthX™ platform for preclinical development in areas such as vaccinology and targeted delivery of therapeutics [3] Commercialization Agreement - 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][6]

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]