Capricor Therapeutics Announces Q1 2024 Financial Results and Corporate Update

Capricor Therapeutics, a biotechnology company focused on cell and exosome-based treatments for rare diseases, has released its financial results for Q1 2024 and provided a comprehensive corporate update. The company has made considerable progress in its CAP-1002 cell therapy program for Duchenne Muscular Dystrophy (DMD), marked by the completion of enrollment for Cohort A of its Phase 3 pivotal trial. This trial, known as HOPE-3, is examining the safety and efficacy of CAP-1002 in patients with DMD, a severe muscle-wasting disease.

A significant milestone was achieved with the FDA's alignment on key chemistry, manufacturing, and controls (CMC) deliverables, essential for filing a Biologics License Application (BLA). This includes establishing non-clinical comparability, a major step toward regulatory clearance. Capricor is also collaborating closely with its partner, Nippon Shinyaku, preparing for the potential market launch of CAP-1002.

CAP-1002 is designed to slow disease progression in DMD patients through immunomodulatory, anti-inflammatory, and anti-fibrotic actions, potentially improving skeletal and cardiac muscle function. The HOPE-3 trial is a multi-center, randomized, double-blind, placebo-controlled study conducted in the United States, involving 102 participants divided into two cohorts. The trial has received Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Additionally, Capricor is eligible for a Priority Review Voucher (PRV) upon FDA approval, due to its rare pediatric disease designation.

In Q1 2024, Capricor received a $10 million milestone payment following a positive interim analysis of Cohort A. The company also had a successful Type-B CMC meeting with the FDA, which confirmed the comparability of CAP-1002 products manufactured at different facilities. This agreement permits the use of products produced at Capricor’s new San Diego facility, pending regulatory approval.

Capricor plans to enroll 44 more U.S.-based patients by the end of Q2 2024 in Cohort B of the HOPE-3 trial. The company is considering expanding Cohort B to include European patients and is scaling up manufacturing capacity at its San Diego facility, which is now operational and producing clinical doses.

Beyond DMD, Capricor is advancing its StealthX™ exosome platform, aiming to utilize exosomes for vaccine development and therapeutic applications. This includes a collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) to develop an exosome-based multivalent vaccine for SARS-CoV-2, with NIAID conducting and funding a Phase 1 clinical trial. Capricor is also exploring therapeutic uses of its exosome technology in partnership with an undisclosed pharmaceutical company.

Financially, Capricor reported a net loss of approximately $9.8 million for Q1 2024, a slight increase from the $7.8 million loss in Q1 2023. Revenues rose to approximately $4.9 million, up from $3.0 million in the same period last year, primarily driven by milestone payments from Nippon Shinyaku. The company's cash, cash equivalents, and marketable securities totaled around $39.9 million as of March 31, 2024, slightly up from $39.5 million at the end of 2023.

Looking ahead, Capricor aims to meet with the FDA in May 2024 to discuss a pre-BLA meeting and rolling BLA submission, with plans to report 3-year data from the HOPE-2 open-label extension (OLE) trial in Q2 2024. The company also anticipates reporting top-line data from Cohort A of the HOPE-3 trial by Q4 2024.

Overall, Capricor is making significant strides in developing CAP-1002 for DMD and leveraging its exosome platform for broader therapeutic applications, supported by strategic partnerships and financial milestones.