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].

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]

Announced the U.S. FDA has accepted our Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathyBLA granted priority review with a Prescription Drug User Fee Act (PDUFA) target action date set for August 31, 2025 Reported positive data from HOPE-2 open label extension (OLE) trial at 2025 MDA Conference showing preservation of skeletal muscle function over 3 years resulting in 52% slowing of disease Received $10 million mile ...

--Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the 2025 Muscular Dystrophy Association (MDA) Conference-- SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive long-term data from its ongoing HOPE-2 open label extension (“OLE”) clinical trial, demonstrating the ...

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]