Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $144.8 million [23] - Revenues for Q1 2025 were zero, compared to approximately $4.9 million for Q1 2024, with the previous revenue being from a $40 million distribution agreement fully recognized by the end of 2024 [23][24] - Operating expenses for Q1 2025 were approximately $16.2 million for R&D, up from $10.1 million in Q1 2024, and general and administrative expenses were approximately $3.1 million, compared to $1.8 million in Q1 2024 [24] - The net loss for Q1 2025 was approximately $24.4 million, compared to a net loss of approximately $9.8 million for Q1 2024 [25] Business Line Data and Key Metrics Changes - The company is focused on the BLA for daramycin, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy, with a strong emphasis on the safety and efficacy data supporting the application [5][8] - The company has been providing daramycin to all open-label extension patients for over three years, with nearly all HOPE-three patients now in open-label extension [15] Market Data and Key Metrics Changes - The company is negotiating with Nippon Shinyaku for the distribution of deramycin in Europe, with the negotiation period extended through the end of Q2 2025 [18] - The company is also exploring opportunities for its technology in other global markets [18] Company Strategy and Development Direction - The company aims to transition from a translational medicine company to a commercial stage entity, actively working with NS Pharma on launch readiness in the U.S. [12] - The company plans to have over 100 patients transition from clinical to commercial product following potential BLA approval [14] - The company is enhancing its medical leadership to guide physicians through the prescribing process for daramycin [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of the data supporting the BLA and the unmet need in treating DMD cardiomyopathy, which could lead to FDA approval [5][6] - Management noted that the FDA has not indicated any substantive issues with the application, providing confidence in the ongoing review process [30] - The company has a cash balance of approximately $145 million, with a runway extending into 2027 without additional cash infusions [20] Other Important Information - The company is developing its Stealth Exosome Platform technology as part of a next-generation drug delivery platform, although this program has taken a backseat to daramycin [19] - The company is also working on a vaccine candidate for COVID-19 prevention under Project NextGen, with Phase 1 trials set to start in Q3 2025 [20] Q&A Session Summary Question: Has the site inspection in San Diego occurred? - The site inspection is scheduled for the upcoming quarter, and management feels prepared for it [28] Question: What is the status of negotiations with Nippon Shinyaku for Europe? - The company is actively negotiating and evaluating opportunities for commercialization in Europe, while also preparing to work directly with European authorities [37] Question: What are the key drivers of proof for the efficacy data? - The statistical significance of cardiac MRI data is a key driver, showing very little chance that the data is due to chance [46] Question: What is the plan if the FDA issues a CRL for efficacy? - The company would submit data from the HOPE-three trial for skeletal muscle dysfunction if a CRL is issued [55] Question: What is the plan for the PRV voucher? - The current plan is to sell the PRV voucher to strengthen the balance sheet [72]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $144.8 million [22] - Revenues for Q1 2025 were zero, compared to approximately $4.9 million for Q1 2024, with the previous revenue being recognized from a $40 million distribution agreement [22][23] - Operating expenses for Q1 2025 were approximately $16.2 million for R&D, up from approximately $10.1 million in Q1 2024, and general and administrative expenses were approximately $3.1 million, compared to $1.8 million in Q1 2024 [24][25] - The net loss for Q1 2025 was approximately $24.4 million, compared to a net loss of approximately $9.8 million for Q1 2024 [25] Business Line Data and Key Metrics Changes - The company is focused on the BLA for daramycin, a therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with ongoing preparations for an FDA advisory committee meeting [4][5] - The company has been providing daramycin to open label extension patients for over three years, with plans for over 100 patients to transition to commercial products following potential BLA approval [12][13] Market Data and Key Metrics Changes - The company is negotiating with Nippon Shinyaku for the distribution of daramycin in Europe, with an extended negotiation period through the end of Q2 [17] - The company is also exploring opportunities for its technology in other global markets [17] Company Strategy and Development Direction - The company aims to transition from a translational medicine company to a commercial stage entity, actively working with NS Pharma on launch readiness in the U.S. [11] - The strategy includes enhancing medical leadership and preparing physicians for prescribing daramycin, which targets inflammation and fibrosis associated with DMD [14][39] - The company is also developing its Stealth Exosome Platform technology for next-generation drug delivery [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the FDA's review process and the strength of the data supporting daramycin's efficacy and safety [4][30] - The company has a cash balance of approximately $145 million, with a runway extending into 2027 without additional cash infusions [19] - If FDA approval is received, the company expects to receive an $80 million milestone payment and a priority review voucher, potentially totaling over $200 million in non-dilutive cash [20] Other Important Information - The company has appointed Dr. Michael Binks as the new Chief Medical Officer, bringing over 25 years of experience in clinical development [12] - The San Diego GMP manufacturing facility is fully operational and producing doses of daramycin, with plans for expansion to meet potential demand [15][16] Q&A Session Summary Question: Has the site inspection in San Diego occurred? - The site inspection is scheduled for this quarter, and preparations are underway [28] Question: What is the status of negotiations with Nippon Shinyaku? - The company is actively negotiating with Nippon Shinyaku and evaluating opportunities for launching in Europe independently [34][37] Question: What are the key drivers of proof for the efficacy of daramycin? - The statistical significance of cardiac MRI data is a key driver, indicating that the treatment effects are unlikely due to chance [44][59] Question: What is the plan if the FDA issues a CRL for efficacy? - The company would submit data from the HOPE-three trial for skeletal muscle dysfunction if a CRL is issued [53][54] Question: What is the company's plan for the priority review voucher? - The current plan is to sell the priority review voucher to strengthen the balance sheet [69]

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 Insights - Capricor Therapeutics has appointed Dr. Michael Binks as Chief Medical Officer, effective immediately, to lead the advancement of deramiocel for Duchenne muscular dystrophy (DMD) and future pipeline opportunities [1][2] - Dr. Binks has 25 years of experience in clinical development and has held senior roles at Pfizer and GlaxoSmithKline, contributing to the advancement of several first-in-class therapies [2] - Capricor's lead product candidate, deramiocel, is in late-stage development and has shown potent immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in dystrophiopathies [3] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with a commitment to innovative treatments [3] - The company has entered into an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization and distribution of deramiocel for DMD in the United States and Japan, pending regulatory approval [6]

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 ...

Core Viewpoint - Capricor Therapeutics is set to release its financial results for the first quarter of 2025 on May 13, 2025, followed by a conference call to discuss the results and recent corporate updates [1][2]. Company Overview - Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, with its lead product candidate being deramiocel, a cardiac-derived cell therapy [3]. - Deramiocel is currently in late-stage development for the treatment of Duchenne muscular dystrophy (DMD) and has shown significant immunomodulatory and anti-fibrotic effects in preclinical and clinical studies [3]. - The company is also utilizing its proprietary StealthX™ platform for the preclinical development of exosome technology aimed at various therapeutic applications, including vaccinology and targeted delivery of therapeutics [3]. 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., pending regulatory approval [6].

Core Insights - Capricor Therapeutics is progressing towards the FDA's PDUFA target action date of August 31, 2025, for its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy [1][2] - The FDA's mid-cycle review meeting indicated no significant deficiencies in the BLA submission, and an advisory committee meeting will be scheduled [1][2] - DMD is a severe genetic disorder affecting approximately 15,000-20,000 patients in the U.S., characterized by progressive muscle weakness and heart failure [3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in clinical studies [4][7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, along with RMAT and ATMP designations to support its regulatory pathway [5] Clinical Development - The BLA submission for deramiocel is backed by data from Phase 2 trials (HOPE-2 and HOPE-2 OLE) and comparisons to FDA-funded datasets on DMD-cardiomyopathy [2] - Efficacy data from the ongoing HOPE-3 study is not included in the current BLA submission [2] - Capricor has entered an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization of deramiocel in the U.S. and Japan, pending regulatory approval [9]

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]