-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
The FDA has affirmed alignment with
Pre-BLA Meeting and Rolling BLA Submission
- The FDA advised
Capricor to include discussion for a pre-BLA meeting and rolling BLA schedule in the upcoming Type-B meeting.- Based on this feedback,
Capricor has already been granted a subsequent Type-B meeting to be held in the second quarter of 2024 to discuss these topics, with the results of those discussions to potentially lead to an accelerated BLA filing. Capricor plans to share with FDA its HOPE-2 open label extension (“OLE”) 3-year safety and efficacy data which is expected to be available in the second quarter of 2024 as part of Capricor’s ISS and ISE strategy.
- Based on this feedback,
Establishment of Non-Clinical Comparability
- The FDA agreed that comparability between drug product manufactured at our two different facilities (
Los Angeles andSan Diego ) has been demonstrated using the provided analytical comparability data.- This allows for the use of CAP-1002 drug product manufactured at our
San Diego manufacturing facility upon potential product approval. - Data from Cohort B of the HOPE-3 clinical trial will not be necessary for FDA approval of the product.
- This allows for the use of CAP-1002 drug product manufactured at our
"I am extremely pleased with our recent FDA interactions as we continue to work collaboratively with the agency to align on the most expeditious path towards registration of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Capricor has generated extensive safety and efficacy data in multiple clinical trials and we are very encouraged by the FDA’s agreement that we have successfully demonstrated product comparability which allows for a seamless transition to our
CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if
Virtual Investor Webcast and Conference Call
About
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended
For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
Source:
2024 GlobeNewswire, Inc., source