Financial Performance - Revenues for Q1 2025 were $0, a decrease from $4.9 million in Q1 2024, primarily due to the recognition of previous milestone payments[10] - Total operating expenses for Q1 2025 were approximately $25 million, compared to $15.2 million in Q1 2024, reflecting an increase in R&D and administrative costs[11] - The net loss for Q1 2025 was approximately $24.4 million, or $0.53 per share, compared to a net loss of $9.8 million, or $0.31 per share, in Q1 2024[11] - Cash, cash equivalents, and marketable securities decreased to $144.78 million from $151.52 million, a decline of approximately 4.8%[27] - Total assets decreased to $153.77 million from $170.48 million, representing a decrease of about 9.8%[27] - Total liabilities increased to $26.12 million from $25.02 million, an increase of approximately 4.4%[27] - Total stockholders' equity remained relatively stable at $45.68 million compared to $45.58 million, showing a slight increase of about 0.2%[27] - The number of common shares issued and outstanding decreased to 127.65 million from 145.46 million, a reduction of approximately 12.2%[27] Regulatory and Clinical Developments - The FDA has set a PDUFA target action date for the BLA of deramiocel for Duchenne muscular dystrophy on August 31, 2025[5] - Capricor's StealthX™ vaccine program is on track for a Phase 1 clinical trial initiation in Q3 2025, pending regulatory clearance from NIAID[3] - The company completed a mid-cycle review meeting with the FDA with no significant deficiencies identified, and a late cycle meeting is scheduled for June 2025[5] - Capricor's BLA for deramiocel is supported by data from the Phase 2 HOPE-2 trial, demonstrating significant improvements in cardiac function[6] Management and Operational Updates - Capricor ended Q1 2025 with a cash position of approximately $145 million, expected to support operations into 2027[3] - Capricor appointed Dr. Michael Binks as Chief Medical Officer, bringing over 25 years of experience in clinical development[8] - The company has expanded its internal manufacturing capacity to support increased commercial manufacturing[6]

Core Insights - Capricor Therapeutics is making significant progress towards delivering the first approved therapy for Duchenne cardiomyopathy, with an FDA target action date of August 31, 2025 for their Biologics License Application (BLA) [2][4][5] Financial Results - As of March 31, 2025, Capricor reported a cash position of approximately $145 million, down from $151.5 million at the end of 2024 [6][9] - Revenues for the first quarter of 2025 were $0, a decrease from $4.9 million in the same quarter of 2024, primarily due to the recognition of previous milestone payments [7] - Total operating expenses for Q1 2025 were approximately $25 million, compared to $15.2 million in Q1 2024, leading to a net loss of approximately $24.4 million, or $0.53 per share, compared to a net loss of $9.8 million, or $0.31 per share, in Q1 2024 [8][20] Corporate Developments - The FDA has accepted Capricor's BLA for deramiocel, which is under priority review, with no significant deficiencies identified in the recent mid-cycle review [4][5] - Capricor appointed Dr. Michael Binks as Chief Medical Officer, who brings over 25 years of experience in clinical development [5] - The National Institute of Allergy and Infectious Diseases (NIAID) plans to initiate a Phase 1 clinical trial of Capricor's StealthX™ exosome vaccine in Q3 2025, pending regulatory approval [5][12] Upcoming Milestones - Capricor is preparing for an FDA advisory committee meeting and a pre-approval inspection as part of the BLA process [2][4] - The company expects its cash reserves to support operations into 2027, excluding additional potential milestone payments [9]

Core Viewpoint - Pomerantz LLP is investigating claims of potential securities fraud or unlawful business practices involving Capricor Therapeutics, Inc. and its officers or directors [1] Group 1: Company Developments - On May 5, 2025, Capricor announced that the FDA confirmed its intent to hold an advisory committee meeting regarding the Biologics License Application for deramiocel, a treatment for Duchenne muscular dystrophy cardiomyopathy [2] - Following this announcement, Capricor's stock price dropped by $3.00 per share, representing a 29.13% decline, closing at $7.30 per share on May 6, 2025 [3] Group 2: Legal Context - Pomerantz LLP is recognized for its expertise in corporate, securities, and antitrust class litigation, having a history of recovering significant damages for victims of securities fraud and corporate misconduct [4]

NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of  Capricor Therapeutics, Inc. (“Capricor” or the “Company”) (NASDAQ: CAPR).  Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Capricor and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] On ...

Company to Host Conference Call, May 13, 2025 at 4:30 p.m. ETSAN DIEGO, May 06, 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 first quarter ended March 31, 2025, after the market close on Tuesday, May 13, 2025. Management will then host a webcast and conference call at 4:30 p.m. ET on May 13. Title:Capricor Therapeutics First Qu ...

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]

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 ...

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]