Financial Data and Key Metrics Changes - As of December 31, 2024, the company's cash, cash equivalents, and marketable securities totaled approximately $151.5 million, with a pro forma total of approximately $161.5 million after receiving a $10 million milestone payment from Nippon Shinyaku [36][37] - Revenues for Q4 2024 were approximately $11.1 million, a decrease from approximately $12.1 million in Q4 2023 [37] - The net loss for Q4 2024 was approximately $7.1 million, compared to a net loss of approximately $800,000 for Q4 2023, while the net loss for the year ended December 31, 2024, was approximately $40.5 million, up from approximately $22.3 million in 2023 [38] Business Line Data and Key Metrics Changes - The primary source of revenue is the ratable recognition of a $40 million agreement with Nippon Shinyaku and a $10 million milestone payment triggered by the BLA submission [38] - Research and development expenses for Q4 2024 were approximately $13.6 million, compared to approximately $9.4 million in Q4 2023, while general and administrative expenses were approximately $3 million in Q4 2024, up from approximately $2.1 million in Q4 2023 [38] Market Data and Key Metrics Changes - The company anticipates that approximately 50% to 60% of the overall DMD population in the U.S., or around 7,500 boys and young men, would be eligible for treatment with deramiocel if approved [21] - The company is actively preparing for the launch of deramiocel in the U.S. with a commercial partner, NS Pharma, which has a dedicated team of approximately 125 employees focused on market access and reimbursement [20][21] Company Strategy and Development Direction - The company is transitioning from a translational medicines company to a potentially commercial stage company, focusing on the commercialization of deramiocel for DMD cardiomyopathy [11][27] - The company is expanding its manufacturing capacity to support anticipated demand, with plans to increase capacity to approximately 2,000 to 3,000 patients per year by mid-2026 [24][25] - The company is also exploring opportunities for label extension for other conditions, including Becker muscular dystrophy, and evaluating potential partnerships in Europe [27][28][112] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the BLA submission for deramiocel, highlighting its strong safety profile and statistically significant efficacy data [15][17] - The company is preparing for potential commercialization and is optimistic about reimbursement opportunities due to the lack of approved therapies for DMD cardiomyopathy [20][51] - Management emphasized the importance of early treatment for patients with DMD to preserve cardiac function and improve quality of life [125] Other Important Information - The company has a cash runway into 2027 with no additional cash infusions required, and potential non-dilutive cash infusions could total over $200 million in 2025 [25][26] - The company is developing its StealthX exosome platform technology as part of a next-generation drug delivery system, with a vaccine candidate in the manufacturing phase [29][32] Q&A Session Summary Question: What additional color can you tell us about the prep and division of labor between you and Nippon Shinyaku? - Management stated that NS Pharma is fully engaged in preparing for the commercial launch, with a focus on market access and reimbursement, while the company is responsible for shepherding deramiocel to market [41][43] Question: How are payers viewing the potential for patients on other premium-priced drugs for DMD? - Management received positive feedback from payers, emphasizing that deramiocel is the only therapeutic targeting DMD cardiomyopathy, which could reduce hospitalization and mortality [50][52] Question: Can you define the costs and timeframe for the new expanded facility? - Management indicated that the expansion is underway and is expected to be completed at a reasonable cost, similar to the original clean room built for under a couple of million dollars [62][64] Question: What are the outstanding questions regarding the potential signed NS partnership and EMA discussions? - Management is focused on getting deramiocel approved for Europe and is negotiating with NS Pharma while preparing for discussions with the EMA [75] Question: What is the ideal baseline ejection fraction for treatment impact? - Management believes that early treatment is crucial, with the greatest long-term benefits seen in patients with ejection fractions of 45% or greater [123][125]

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q4 2024 Earnings Conference Call March 19, 2025 4:30 PM ET Company Participants A.J. Bergmann - Chief Financial Officer Linda Marban - Chief Executive Officer Conference Call Participants Edward Tenthoff - Piper Sandler Leland Gershell - Oppenheimer Joseph Pantginis - H.C. Wainwright Kristen Kluska - Cantor Fitzgerald Catherine Novack - JonesTrading Aydin Huseynov - Ladenburg Madison El-Saadi - B. Riley Securities Operator Good afternoon, ladies and gentlemen, and w ...

Capricor Therapeutics (CAPR) came out with a quarterly loss of $0.16 per share versus the Zacks Consensus Estimate of a loss of $0.31. This compares to loss of $0.02 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 48.39%. A quarter ago, it was expected that this biotechnology company would post a loss of $0.34 per share when it actually produced a loss of $0.38, delivering a surprise of -11.76%.Over the last four quarters, the ...

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]

Company to Host Conference Call, March 19, 2025 at 4:30 p.m. ETSAN DIEGO, March 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, will release its financial results for the fourth quarter and full year ended December 31, 2024, after the market close on Wednesday, March 19, 2025. Management will then host a webcast and conference call at 4:30 p.m. ET on March 19. Title:Ca ...

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]