-Company remains on track for PDUFA target action date of August 31, 2025- Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment ...

Core Insights - Capricor Therapeutics is set to present at the 2025 Piper Sandler Virtual Cardio Day on April 2, 2025, focusing on its lead program, deramiocel, for Duchenne muscular dystrophy (DMD) [1][2] Company Overview - Capricor Therapeutics is a biotechnology company specializing in cell and exosome-based therapeutics aimed at treating rare diseases, with deramiocel being its lead product candidate [4] - Deramiocel is an allogeneic cardiac-derived cell therapy that has demonstrated significant immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function in conditions like DMD [4] - The company is also developing its proprietary StealthX™ platform for exosome technology, targeting various therapeutic areas including vaccinology and targeted delivery of therapeutics [4] Recent Developments - Capricor has entered into an exclusive commercialization and distribution agreement for deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd. [6] - The presentation at the Piper Sandler event will include discussions on regulatory, scientific, and corporate updates related to deramiocel [2][3]

Financial Performance - The company reported net losses of $40.5 million and $22.3 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $199.8 million as of December 31, 2024 [467]. - Cash flow used in operating activities was approximately $40.0 million for 2024, compared to $25.6 million for 2023, indicating a significant increase in cash outflow [491]. - The net loss for the year ended December 31, 2024, was approximately $40.5 million, driven by increased R&D expenses [490]. - Clinical development income for the years ended December 31, 2024 and 2023 was approximately $22.3 million and $25.2 million, respectively, reflecting a decrease of 11.5% [474]. - Total research and development expenses increased by approximately $13.5 million, or 37%, from $36.4 million in 2023 to $50.0 million in 2024 [476][477]. - General and administrative expenses rose by approximately $2.1 million, or 16%, from $12.8 million in 2023 to $14.9 million in 2024 [478]. Cash and Securities - As of December 31, 2024, Capricor had cash, cash equivalents, and marketable securities totaling approximately $151.5 million [466]. - Cash and cash equivalents decreased from $14.7 million in 2023 to $11.3 million in 2024, while marketable securities increased significantly from $24.8 million to $140.2 million [488]. - Total cash, cash equivalents, and marketable securities as of December 31, 2024, were approximately $151.5 million, a significant increase from $39.5 million in 2023 [490]. - The fair value of the company's cash, cash equivalents, and marketable securities was approximately $151.5 million as of December 31, 2024 [538]. Clinical Trials and Development - The ongoing Phase 3 HOPE-3 clinical trial has enrolled approximately 105 eligible study subjects, with a positive outcome of the futility analysis for Cohort A [462]. - The HOPE-2 trial met its primary efficacy endpoint with a p-value of 0.01 for the mid-level dimension of the Performance of the Upper Limb (PUL) v1.2 [460]. - Capricor plans to provide clinical data on skeletal muscle myopathy to support potential label expansion for DMD treatment [463]. - Capricor received a CIRM grant of approximately $3.4 million for the HOPE-Duchenne clinical trial, with a co-funding requirement of approximately $2.3 million [508]. Funding and Revenue Recognition - Capricor received an upfront payment of $30.0 million in 2022 under the U.S. Distribution Agreement for deramiocel [497]. - The first milestone payment of $10.0 million was received upon completion of the futility analysis of the HOPE-3 trial, with a second milestone payment of $10.0 million due upon BLA submission to the FDA in December 2024 [497]. - Potential sales-based milestones could reach up to $605.0 million based on annual net sales of deramiocel, with Capricor entitled to 30-50% of product revenue [497]. - An upfront payment of $12.0 million was received in Q1 2023 under the Japan Distribution Agreement, with additional milestone payments of up to approximately $89.0 million possible [499]. - Capricor entered into a Binding Term Sheet with Nippon Shinyaku for the European region, with a potential upfront payment of $20.0 million and additional milestone payments of up to $715.0 million [501]. - The company evaluates the probability of significant reversals in cumulative revenue recognized for milestones and shared revenue payments at the end of each reporting period [521]. - The company recognizes grant income in the period in which the expense is incurred for reimbursable expenses under the terms of the grant [525]. - The company uses the proportional performance method or straight-line basis for revenue recognition when performance obligations are satisfied over time [523]. Future Expenses and Investments - The company anticipates significant increases in expenses and operating losses as it continues to develop deramiocel and other product candidates [469]. - The company expects to spend approximately $40.0 million to $50.0 million in 2025 for the deramiocel program, primarily for CMC expansion and pre-commercial expenses [484]. - The company anticipates spending approximately $5.0 million to $7.5 million in 2025 on development expenses related to its exosome platform [485]. - The company expects to record additional non-cash compensation expense in the future related to stock-based compensation [532]. - The company aims to mitigate default risk by investing in high credit quality securities and does not hedge interest rate exposure [539]. Stock and Share Transactions - In 2024, Capricor sold 6,252,229 shares of common stock at an average price of approximately $9.34 per share, resulting in gross proceeds of $58.4 million [468]. - On October 16, 2024, Capricor completed a public offering of 5,073,800 shares at $17.00 per share, raising approximately $86.3 million before expenses [502]. - A private placement on September 16, 2024, resulted in the sale of 2,798,507 shares at $5.36 per share for approximately $15.0 million [503]. - The ATM Program raised approximately $75.0 million from the sale of 9,228,383 shares at an average price of $8.13 per share, which was closed on October 1, 2024 [507]. Exosome Platform Development - The company is developing a precision-engineered exosome platform technology for therapeutic applications, including a vaccine for COVID-19 [465]. - The company performs assessments to determine if a significant financing component exists in its distribution agreements, typically finding that it does not [522]. - Research and development expenses primarily consist of salaries, clinical trial costs, and other related expenses, which are expensed as incurred [526]. - The company is currently evaluating the impact of ASU No. 2024-03 on its financial statement disclosures, effective after December 15, 2026 [536].

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]

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 - Capricor 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 orphan cardiomyopathies for future pipeline expansion [112][115] 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 [12][15] - 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 higher ejection fractions to preserve cardiac function and improve quality of life [123] Other Important Information - The company has a cash balance of approximately $150 million, providing a runway into 2027 without additional cash infusions [25] - If FDA approval is received, the company expects to receive an additional $80 million milestone payment and a priority review voucher, potentially totaling over $200 million in non-dilutive cash infusions [26] Q&A Session Summary Question: What additional color can you tell us about the commercial preparation for deramiocel and the division of labor with Nippon Shinyaku? - Management stated that NS Pharma is fully engaged in preparing for the commercial launch, with a focus on market access, reimbursement, and working with physicians and infusion centers [41][43] Question: How are payers viewing the potential for patients on other premium-priced drugs for DMD? - Management reported 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 construction [62][64] Question: What is the status of discussions with the FDA regarding the need for an AdCom? - Management is awaiting confirmation from the FDA regarding an AdCom and believes the data stands strong on its own [66][68] Question: What are the outstanding questions regarding the potential NS partnership and EMA discussions? - Management is focused on getting deramiocel approved in Europe and is negotiating with NS Pharma while preparing for discussions with the EMA [72][74] 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]

Core Insights - Capricor Therapeutics reported a quarterly loss of $0.16 per share, which was better than the Zacks Consensus Estimate of a loss of $0.31, representing an earnings surprise of 48.39% [1] - The company posted revenues of $11.13 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 248.28%, although this was a decrease from $12.09 million in the same quarter last year [2] - Capricor shares have declined approximately 15.9% year-to-date, compared to a 4.5% decline in the S&P 500 [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.21 on revenues of $8.25 million, and for the current fiscal year, it is -$0.70 on revenues of $20.78 million [7] - The estimate revisions trend for Capricor is currently favorable, leading to a Zacks Rank 2 (Buy) for the stock, indicating expected outperformance in the near future [6] Industry Context - The Medical - Products industry, to which Capricor belongs, is currently ranked in the bottom 44% of over 250 Zacks industries, suggesting potential challenges ahead [8] - Another company in the same industry, MacroGenics, is expected to report a quarterly loss of $0.23 per share, with revenues projected to be $45.26 million, reflecting a significant year-over-year increase of 322.2% [9][10]

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].