Core Insights - Capricor Therapeutics announced positive four-year safety and efficacy results for Deramiocel, its lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD) [1][3] - The findings will be presented at the PPMD 2025 Annual Conference, highlighting the importance of addressing both cardiac and skeletal muscle functions in DMD treatment [1][4] Efficacy Results - After four years of treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline, indicating clinical benefit, especially in patients with baseline LVEF >45% [2] - The treatment also slowed skeletal muscle disease progression, with a smaller average decline in Performance of the Upper Limb (PUL v2.0) in the fourth year (0.6 points) compared to the first year (1.8 points) [3][7] Safety Profile - Deramiocel maintained a favorable safety profile throughout the study, reinforcing its potential as a therapeutic option for DMD [3][7] Regulatory Progress - Capricor is in the process of obtaining regulatory approval for Deramiocel, with its Biologics License Application (BLA) under priority review and no evidence of delays in discussions with the FDA [4][10] Study Background - The HOPE-2 study was a randomized, double-blind, placebo-controlled Phase 2 trial, with patients receiving intravenous infusions of Deramiocel (150 million cells) every three months [5] - Following the study, all patients entered a treatment gap phase before enrolling in the HOPE-2 Open-Label Extension (OLE) study, which continues to monitor safety and efficacy [5] About Duchenne Muscular Dystrophy - DMD is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., characterized by progressive muscle degeneration and leading to cardiomyopathy, which is a major cause of mortality [6][9] About Deramiocel - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) known for their immunomodulatory and anti-fibrotic properties, which may help preserve cardiac and skeletal muscle function in DMD [8][10] - The therapy has received multiple designations from regulatory agencies, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation [9]

Core Viewpoint - Capricor Therapeutics has received Orphan Drug Designation from the FDA for its lead cell therapy candidate, Deramiocel, for the treatment of Becker Muscular Dystrophy, enhancing its strategic position in the neuromuscular disease market [1][3][7] Company Overview - Capricor Therapeutics is a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead product candidate [9] - The company is advancing a fully integrated platform targeting cardiac and skeletal complications associated with muscular dystrophy [1][9] Product Details - Deramiocel (CAP-1002) is an allogeneic cell therapy derived from cardiosphere-derived cells (CDCs), which have shown immunomodulatory and anti-fibrotic effects in preserving muscle function in dystrophies [6][9] - The therapy is currently in late-stage development for Duchenne Muscular Dystrophy (DMD) and has received multiple designations from regulatory bodies, including Orphan Drug Designation for both DMD and Becker Muscular Dystrophy [8][11] Disease Context - Becker Muscular Dystrophy (BMD) is a progressive neuromuscular disorder affecting approximately 5,000 individuals in the U.S., characterized by slower disease progression compared to Duchenne Muscular Dystrophy (DMD) [5] - Both BMD and DMD are caused by mutations in the dystrophin gene, leading to significant muscle deterioration and serious cardiac complications [2][4][5] Regulatory Milestones - Capricor has successfully completed its Pre-License Inspection, a key regulatory milestone for its Biologics License Application (BLA) for DMD, with a target action date set for August 31, 2025 [3][7] - The company is preparing for the potential commercial launch of Deramiocel, aiming to deliver meaningful benefits to patients with both DMD and BMD [3][7]

Core Insights - Capricor Therapeutics, Inc. (NASDAQ: CAPR) is set to have its Biologics License Application (BLA) for deramiocel, aimed at treating Duchenne Muscular Dystrophy (DMD), reviewed by the FDA with a Priority Review Prescription Drug User Fee Act (PDUFA) date approaching [2] Company Overview - Capricor Therapeutics focuses on developing innovative therapies for rare diseases, particularly in the field of muscular dystrophy [2] Market Context - The FDA's Priority Review designation indicates that the agency recognizes the potential significance of deramiocel in addressing unmet medical needs in DMD patients, which could lead to expedited review and approval processes [2]

Core Insights - Capricor Therapeutics has successfully completed the FDA Pre-License Inspection for its manufacturing facility in San Diego for Deramiocel, its lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD) [1][5] - The inspection resulted in a Form 483 with several observations related to quality systems and documentation practices, but no material changes to the cGMP process were required [1][5] - The FDA Advisory Committee meeting is scheduled for July 30, 2025, with a PDUFA action date for the Biologics License Application (BLA) set for August 31, 2025 [2][5] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel being a key product in late-stage development for DMD [7] - The company has received Orphan Drug Designation from both the FDA and EMA for Deramiocel, which is supported by RMAT and ATMP designations [5][6] - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [9] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in preserving muscle function in DMD [4][7] - DMD is a severe genetic disorder affecting approximately 15,000-20,000 individuals in the U.S., characterized by progressive muscle weakness and inflammation [3][4] - The pathophysiology of DMD involves impaired dystrophin production, leading to muscle cell damage and heart failure, which is the leading cause of death in DMD patients [3]

Core Viewpoint - Pomerantz LLP is investigating 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 [3] - Following this announcement, Capricor's stock price dropped by $3.00 per share, representing a decline of 29.13%, closing at $7.30 per share on May 6, 2025 [4] Group 2: Legal Investigation - Pomerantz LLP is reaching out to investors of Capricor to discuss claims related to potential securities fraud [1] - The firm has a long history in corporate, securities, and antitrust class litigation, having recovered significant damages for victims of securities fraud and corporate misconduct [5]

Group 1 - Capricor Therapeutics, Inc. (CAPR) is a biotechnology company focused on two platforms: cardiosphere-derived cell therapy (Deramiocel) and StealthX exosome engineering [1] - Deramiocel is considered the flagship candidate of the company and has received FDA approval [1]

Group 1 - The article discusses the analytical approach of Stephen, who combines clinical insight with valuation methods to analyze healthcare and tech stocks [1] - Stephen specializes in scenario-based DCF modeling, sensitivity analysis, and Monte Carlo simulations to identify asymmetric risk-reward opportunities [1] - The focus is on translating complex scientific and market dynamics into actionable investment theses [1] Group 2 - The article emphasizes the importance of independent verification of information and conducting thorough research before making investment decisions [3] - It highlights that past performance is not indicative of future results and that no specific investment recommendations are provided [4]

Core Viewpoint - The Schall Law Firm is investigating Capricor Therapeutics, Inc. for potential violations of securities laws related to misleading statements and undisclosed information regarding its Biologics License Application for deramiocel, a treatment for Duchenne muscular dystrophy cardiomyopathy [1][2]. Group 1 - The investigation is centered on whether Capricor issued false or misleading statements or failed to disclose important information to investors [2]. - On May 5, 2025, Capricor announced that the FDA confirmed its intent to hold an advisory committee meeting regarding the Company's BLA for deramiocel [2].

Product Development - Capricor Therapeutics is focused on developing deramiocel for treating Duchenne muscular dystrophy (DMD), a significant market opportunity due to the lack of approved treatments [147][150]. - The company completed a Biologics License Application (BLA) submission in December 2024, with the FDA granting Priority Review and a target action date of August 31, 2025 [151]. - Clinical trials for deramiocel have shown promising results, with the Phase II trial HOPE-2 meeting its primary efficacy endpoint (p=0.01) and demonstrating improvements in cardiac function (p=0.002) [152]. - The ongoing Phase 3 trial (HOPE-3) has enrolled approximately 105 subjects, with a positive futility analysis outcome allowing the trial to continue as planned [154]. - Capricor is developing an exosome-based platform technology for various therapeutic applications, including a recombinant protein-based vaccine for COVID-19 [158]. Financial Performance - The company reported net losses of $24.4 million for the three months ended March 31, 2025, and an accumulated deficit of $224.2 million [160]. - Clinical development income for Q1 2025 was $0, compared to approximately $4.9 million in Q1 2024, with a total of $50.0 million in milestone payments recognized from Nippon Shinyaku [164]. - Total research and development (R&D) expenses for Q1 2025 were $18.9 million, a 70% increase from $11.1 million in Q1 2024, driven by significant increases in personnel and program-related expenses [166]. - General and administrative (G&A) expenses for Q1 2025 totaled $6.1 million, a 49% increase from $4.1 million in Q1 2024, primarily due to increased headcount and stock-based compensation [170]. - The company expects to spend approximately $40.0 million to $50.0 million in 2025 on the deramiocel program, focusing on CMC expansion and pre-commercial expenses [173]. Cash and Securities - As of March 31, 2025, Capricor had cash, cash equivalents, and marketable securities totaling approximately $144.8 million, following a $10 million milestone payment received in January 2025 [159]. - Cash and cash equivalents as of March 31, 2025, were $28.8 million, up from $11.3 million as of December 31, 2024 [179]. - Marketable securities decreased to $116.0 million as of March 31, 2025, from $140.2 million as of December 31, 2024 [179]. - As of March 31, 2025, the fair value of the company's cash, cash equivalents, and marketable securities was approximately $144.8 million [227]. Funding and Partnerships - The company is exploring partnerships for the development of its product candidates in the U.S. and other territories, while maintaining distribution agreements with Nippon Shinyaku [162]. - The U.S. Distribution Agreement with Nippon Shinyaku includes potential milestone payments totaling $80.0 million upon marketing approval and sales-based milestones of up to $605.0 million [187]. - Capricor received an upfront payment of $12.0 million under the Japan Distribution Agreement and may receive additional milestone payments of up to approximately $89.0 million [190]. - Capricor entered into a Binding Term Sheet with Nippon Shinyaku for the commercialization of deramiocel in Europe, with an upfront payment of $20.0 million and potential milestone payments of up to $715.0 million [191]. - The Company completed an underwritten public offering of 5,073,800 shares at a price of $17.00 per share, generating total gross proceeds of approximately $86.3 million [192]. - In a private placement, Capricor issued 2,798,507 shares at $5.36 per share, raising approximately $15.0 million [193]. - The Company sold an aggregate of 9,228,383 shares under the ATM Program at an average price of approximately $8.13 per share, resulting in gross proceeds of approximately $75.0 million [197]. - Capricor received a CIRM Award of approximately $3.4 million to fund its Phase I/II clinical trial, with a co-funding requirement of approximately $2.3 million [198]. - Capricor notified CIRM of its election to convert the CIRM Award into a loan, which could result in accrued interest of up to approximately $7.1 million [201]. Expense Management - Capricor anticipates significant increases in expenses as it develops deramiocel and other product candidates, necessitating substantial additional funding [161]. - The company anticipates additional non-cash compensation expenses in the future, which may be significant [163]. - Stock-based compensation expense in R&D increased by 177% to $2.7 million in Q1 2025 from $1.0 million in Q1 2024 [166]. - The company has issued stock options and restricted stock awards under its 2020 and 2021 Equity Incentive Plans, with stock-based compensation expense expected to be significant in the future [221][223]. Revenue Recognition - The Company has not achieved commercial sales of its drug candidates to date, but applies ASU 606 for revenue recognition in its distribution agreements [209]. - The revenue standard requires the Company to assess whether a significant financing component exists, which typically does not apply due to upfront payments from customers [213]. - The company recognizes grant income in the period expenses are incurred, with the transaction price varying based on incurred expenses [216]. - Research and development (R&D) expenses primarily consist of salaries, clinical trial costs, and other related expenses, which are expensed as incurred [217]. - The company accrues clinical trial expenses based on estimates of services received and efforts expended, with adjustments made if actual results differ from estimates [224]. - The company’s revenue recognition policy requires significant management judgment in determining the level of effort required under contracts [215].

Core Viewpoint - Capricor Therapeutics is conducting its Q1 2025 earnings conference call, indicating ongoing engagement with investors and stakeholders regarding its financial performance and future plans [1]. Group 1: Company Overview - The conference call features key participants including the Chief Financial Officer A.J. Bergmann and Chief Executive Officer Linda Marban, highlighting the leadership's involvement in communicating with investors [1]. - The call is structured to include a presentation followed by a question-and-answer session, suggesting a transparent approach to investor relations [1]. Group 2: Forward-Looking Statements - The CFO, A.J. Bergmann, emphasizes that forward-looking statements will be made regarding various aspects such as product efficacy, safety, R&D plans, clinical study timelines, and regulatory developments [3]. - These statements are based on current information and assumptions, which are subject to change due to risks and uncertainties that could lead to actual results differing from projections [4].