This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensureAdvisory Committee meeting scheduled for July 30, 2025Mid-cycle meeting recently completed with no significant issues or major deficiencies; late-cycle meeting planned for mid-JulyBiologics License Application remains under priority review with PDUFA target action date of August 31, 2025 SAN DIEGO, June 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnol ...

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].

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]