SAN DIEGO, June 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Additionally, Deramiocel has been selecte ...

Capricor Therapeutics (CAPR) , a biotechnology firm focused on innovative therapies for rare diseases, has announced favorable three-year results from the ongoing HOPE-2 open-label extension (OLE) study on CAP1002 for Duchenne muscular dystrophy (DMD). Patients treated with CAP-1002 showed sustained improvements in upper limb function and cardiac performance compared to an external dataset from Cincinnati Children's Hospital Medical Center, indicating potential long-term benefits. Statistical Significance i ...

--Results in Performance of the Upper Limb (PUL 2.0) Continue to Show Benefits in Skeletal Muscle Function After 3 Years of CAP-1002 Treatment (p< 0.001)-- --Stabilization in Left Ventricular Ejection Fraction (LVEF) Suggests Preservation of Cardiac Function-- --Results Recently Shared with FDA at Type-B Meeting Held in May 2024-- SAN DIEGO, June 04, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the tre ...

Financial Position - As of March 31, 2024, Capricor Therapeutics had cash, cash equivalents, and marketable securities totaling approximately $39.9 million[396]. - The investment portfolio as of March 31, 2024, consisted primarily of money market funds and short-term U.S. treasuries[87]. - Capricor's current cash balance is expected to fund operating expenses and capital expenditures into Q1 2025, excluding potential milestone payments[396]. Revenue Generation - Capricor has not generated any revenues from the commercial sale of products and will not be able to do so until FDA approval is obtained[396]. Research and Development - R&D expenses are expected to increase due to additional non-cash compensation expenses in the future[401]. - There was a $0.5 million increase in stock-based compensation expense primarily due to an increase in the risk-free rate and stock price[405]. - The company is collaborating with an undisclosed pharmaceutical company to investigate the therapeutic application of its StealthX™ exosome platform[399]. - Data presented at the ASGCT 27th Annual Meeting highlighted a potential exosome-based approach for treating arginase-1 deficiency[399]. Milestones and Analysis - In Q4 2023, a positive outcome of the interim futility analysis for the HOPE-3 trial was announced, leading to a milestone payment of $10.0 million received in January 2024[396]. Internal Controls - There has been no change in internal control over financial reporting that materially affected the company during the quarter ended March 31, 2024[89].

Financial Data and Key Metrics Changes - As of March 31, 2024, the company's cash, cash equivalents, and marketable securities totaled approximately $39.9 million, compared to approximately $39.5 million on December 31, 2023 [36] - The net loss for the first quarter of 2024 was approximately $9.8 million, compared to a net loss of approximately $7.8 million for the first quarter of 2023 [60] Business Line Data and Key Metrics Changes - Research and development expenses were approximately $10.1 million in Q1 2024, up from approximately $7.2 million in Q1 2023, primarily due to increased clinical and manufacturing costs associated with the Phase 3 HOPE-3 clinical trial [60] - General and administrative expenses remained stable at approximately $1.8 million for both Q1 2024 and Q1 2023 [60] Market Data and Key Metrics Changes - The company is focused on the commercialization of CAP-1002 for Duchenne muscular dystrophy (DMD), with a potential addressable patient population of approximately 7,500 to 10,000 in the United States [53] - The company is actively preparing for potential commercialization in the U.S. and Japan, with increased meetings with Nippon Shinyaku and its U.S. subsidiary [29] Company Strategy and Development Direction - The company is focused on four main areas: clinical, manufacturing, BLA readiness, and commercial preparations for CAP-1002 [24] - The company plans to leverage its manufacturing processes for potential expansion into other indications, including Becker muscular dystrophy [32][92] - The company is also developing an exosome-based antisense oligonucleotide program in collaboration with an undisclosed pharma partner [57] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong clinical data and the potential for CAP-1002 to be a preferred treatment option for DMD patients [40] - The company is optimistic about the upcoming Type B meeting with the FDA, which is critical for aligning on the BLA submission [50] - Management noted that the rapid enrollment in Cohort B reflects the urgent need for effective treatments among patients with DMD [90] Other Important Information - The company received a $10 million milestone payment from Nippon Shinyaku in Q1 2024 following a positive interim analysis of the HOPE-3 trial [48] - The company is exploring nondilutive capital opportunities to support its exosome platform and CAP-1002 development [35] Q&A Session Summary Question: Can you provide updates on possible preclinical work related to Becker muscular dystrophy? - Management indicated that they are evaluating opportunities for CAP-1002 in Becker muscular dystrophy and have the freedom to operate in this area [92] Question: What are the expected costs of goods sold (COGS) for CAP-1002? - Management stated that they are working to reduce COGS and believe that costs will be minimal compared to expected reimbursement [42] Question: What is the company's strategy for global expansion? - Management confirmed ongoing discussions for potential partnerships in Europe and emphasized the strong demand for DMD treatments in that market [62] Question: How does the company view the potential for CAP-1002 in treating cardiomyopathy associated with DMD? - Management expressed optimism about CAP-1002's role in treating cardiomyopathy and the importance of preserving cardiac function in DMD patients [68]

Over the last four quarters, the company has surpassed consensus EPS estimates three times. Capricor, which belongs to the Zacks Medical - Products industry, posted revenues of $4.91 million for the quarter ended March 2024, missing the Zacks Consensus Estimate by 50.93%. This compares to year-ago revenues of $2.99 million. The company has topped consensus revenue estimates two times over the last four quarters. Capricor Therapeutics (CAPR) came out with a quarterly loss of $0.31 per share versus the Zacks ...

-Phase 3, HOPE-3 Trial (Cohort A) of CAP-1002 in Duchenne Muscular Dystrophy Fully Enrolled; On Track to Report Top-Line Data in Q4 2024- -Recent FDA Feedback Supports Requests for a Pre-BLA Meeting and Subsequent Rolling BLA Submission at Upcoming Type-B Clinical FDA Meeting in May 2024- SAN DIEGO, May 13, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced it ...

-Preclinical findings show exosome-based therapeutic approach for the treatment of arginase-1 deficiency (ARG1-D)- -Presentation to be featured in an oral session being held on May 9, 2024- SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ ...

-Company Aligned with FDA on Demonstration of Non-Clinical Comparability; Allowing for Immediate Use of San Diego Manufacturing Facility- -FDA Feedback Supports Requests for a Pre-BLA Meeting and Subsequent Rolling BLA Submission Following Upcoming Q2 Type-B FDA Meeting- -Company Granted Subsequent Type-B Clinical Meeting with FDA in Second Quarter to Continue to Discuss Pathway to BLA- -Capricor Management to Host Virtual Investor Webcast to Discuss Latest Program Updates on Monday, April 29 at 8:30 a.m. E ...

Shares of Capricor Therapeutics (CAPR) have gained 11.9% over the past four weeks to close the last trading session at $6.60, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $21 indicates a potential upside of 218.2%.The average comprises four short-term price targets ranging from a low of $8 to a high of $40, with a standard deviation of $14.38. While the lowest estimate indica ...