Exhibit 99.1
Capricor Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Corporate Update
-Enrollment Continues to Progress in HOPE-3, the Phase 3 Clinical Trial of CAP-1002 in Duchenne Muscular Dystrophy (DMD); Plan to Report on Interim Analysis in Q4 2023-
-Held Type-B CMC Meeting with U.S. Food and Drug Administration (FDA) Regarding Pathway Towards Biologics License Application (BLA) for CAP-1002 in DMD-
-Expanded Partnership with Nippon Shinyaku to Japan to Leverage Commercial DMD Franchise with $12 Million Upfront and Additional Potential Milestone Payments of up to $89 Million-
-To Host Conference Call and Webcast Today at 4:30 p.m. ET-
SAN DIEGO, Calif., March 15, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced today its financial results for the fourth quarter and full year ended December 31, 2022 and provided a corporate update.
“In 2022, we continued to advance our clinical development program for CAP-1002 in patients with Duchenne Muscular Dystrophy (DMD), highlighted by the initiation of our HOPE-3 Phase 3 trial and are well positioned to deliver on multiple value-driving milestones throughout 2023,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “As CAP-1002 continues to advance towards potential commercialization, we recently met with the FDA in a Type-B CMC meeting and discussed our manufacturing plans in anticipation of a Biologics License Application (BLA). We are pleased that the FDA continues to work with us under our RMAT designation and we will provide further updates on our plans as they become available. Furthermore, enrollment for HOPE-3 continues to progress and with patient dosing underway, we plan to report the results of our interim analysis in the fourth quarter of 2023. This trial builds on the recently presented statistically significant 18-month results from our ongoing HOPE-2 open label extension study (OLE), which further positions CAP-1002 as a potential anchor therapy for DMD. These results suggest that patients accumulate benefit over time with steady preservation of skeletal muscle functions, highlighting the potential disease modifying effect and long-term benefit of CAP-1002. They also contribute to the safety profile of the therapy. Further, we continue to explore opportunities for additional strategic partnerships outside of the United States and Japan to maximize the value of our asset and ensure rapid therapy availability to the global patient community.”
Dr. Marbán continued, “In parallel, we are pleased with the steady progress across our exosome program, designed to support the advancement of next generation vaccines and innovative therapeutics. We recently published preclinical data from our proprietary StealthXTM platform, which generated two vaccine candidates that induced a strong, long-lasting immune response against two SARS-CoV-2 proteins, spike and nucleocapsid. We look forward to exploring the potential therapeutic utility of these vaccine candidates, and more broadly, expanding our pipeline and partnership opportunities with this platform.”
Fourth Quarter 2022 and Recent Operational Developments
| ● | HOPE-3, our Phase 3 clinical trial of CAP-1002 in DMD continues to progress well. The multi-center, randomized, double-blind, placebo-controlled study (NCT05126758) is designed to treat up to 68 patients in the United States. At this time, we have treated over 30% of the patients in the currently designed study with 11 active sites. |
| ● | Met with the FDA in a Type-B CMC meeting to discuss our commercial planning in anticipation of a potential BLA submission. The meeting was very constructive and provides us with increased visibility on the expectations the FDA has for the HOPE-3 trial and commercial scale manufacturing of CAP-1002 in support of a future BLA. |