Capricor Therapeutics’ Earnings Call Highlights Progress and Challenges

Capricor Therapeutics, Inc. ((CAPR)) has held its Q4 earnings call. Read on for the main highlights of the call.

Capricor Therapeutics, Inc. recently held its fourth-quarter 2024 earnings call, highlighting significant strides towards the FDA approval and commercialization of Deramiocel. The sentiment during the call was optimistic, underscored by strong safety and efficacy data, a robust manufacturing strategy, and a solid financial position. However, the company faces financial challenges with increased operating expenses and a larger net loss. Additionally, the pending European agreement remains a point of uncertainty.

FDA Priority Review for Deramiocel

The Biologics License Application (BLA) for Deramiocel, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy, has been accepted by the FDA for priority review. The Prescription Drug User Fee Act (PDUFA) date is set for August 31, 2025, marking a significant milestone in Capricor’s journey towards bringing this treatment to market.

Strong Safety and Efficacy Data

Deramiocel has demonstrated a strong safety profile, having been administered to over 250 human subjects. The treatment has shown clinical and statistically significant efficacy in addressing DMD cardiomyopathy, reinforcing confidence in its potential impact on patient health outcomes.

Robust Manufacturing and Expansion Plans

Capricor’s manufacturing facility in San Diego currently supports 250-500 patients annually, with plans to expand capacity to accommodate 2,000-3,000 patients by mid-2026. This expansion is crucial to meet the anticipated demand following FDA approval.

Solid Financial Position

Capricor reported a cash balance of approximately $151.5 million, providing a financial runway into 2027. The company anticipates potential non-dilutive cash infusions exceeding $200 million upon FDA approval, bolstering its financial stability.

Positive Market Access and Reimbursement Outlook

Surveys conducted with the top five US payers have shown favorable responses regarding reimbursement for Deramiocel, suggesting a promising market access landscape post-approval.

Increased Operating Expenses

The company reported an increase in research and development expenses from $9.4 million in Q4 2023 to $13.6 million in Q4 2024. General and administrative expenses also rose from $2.1 million to $3 million in the same period, reflecting the financial pressures of advancing Deramiocel.

Net Loss Increase

Capricor’s net loss for the fourth quarter of 2024 was approximately $7.1 million, a significant increase from a net loss of $800,000 in the same quarter of 2023. This highlights the financial challenges the company faces as it progresses towards commercialization.

Pending Finalization of European Agreement

Negotiations with Nippon Shinyaku for European distribution of Deramiocel have yet to result in a definitive agreement, adding an element of uncertainty to Capricor’s international expansion plans.

Forward-Looking Guidance

During the earnings call, Capricor provided substantial guidance on Deramiocel’s future. The company is preparing for a potential Advisory Committee meeting and a pre-licensing inspection of their manufacturing facility in the second quarter of 2025. They anticipate launching Deramiocel commercially in the U.S., with approximately 100 patients transitioning from clinical trials. Capricor projects that 50-60% of the U.S. DMD population could be eligible for treatment. Financially, they expect significant non-dilutive cash infusions, including an $80 million milestone payment upon FDA approval.

In conclusion, Capricor Therapeutics is on a promising path towards the FDA approval and commercialization of Deramiocel, supported by strong clinical data and a solid financial position. However, the company must navigate increased expenses and finalize its European distribution strategy to fully capitalize on Deramiocel’s potential. Investors and stakeholders will be closely monitoring these developments as Capricor moves forward.