Release Date: March 19, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Capricor Therapeutics Inc (CAPR, Financial) has received FDA acceptance for priority review of its Biologic License Application (BLA) for deramiocel to treat DMD cardiomyopathy.
- The company has a strong safety profile for deramiocel, with over 700 infusions showing safety and efficacy in attenuating disease progression.
- Capricor Therapeutics Inc (CAPR) has a robust cash position of approximately $150 million, with a runway into 2027 without additional cash infusions.
- The company is actively preparing for the commercial launch of deramiocel in collaboration with NS Pharma, with a focus on market access and reimbursement.
- Capricor Therapeutics Inc (CAPR) is expanding its manufacturing capacity to support anticipated demand, with plans to increase capacity to 2,000 to 3,000 patients per year by mid-2026.
Negative Points
- The company reported a net loss of approximately $40.5 million for the year ended December 31, 2024, compared to a net loss of approximately $22.3 million for the previous year.
- Revenues for the fourth quarter of 2024 decreased to approximately $11.1 million from $12.1 million in the fourth quarter of 2023.
- There is uncertainty regarding whether an FDA Advisory Committee (ADCOM) meeting will be required for the approval process of deramiocel.
- The company is still negotiating final terms for a definitive agreement with Nippon Shinyaku for the European market.
- Capricor Therapeutics Inc (CAPR) faces potential challenges in expanding its exosome platform technology, which is still in the early stages of development.
Q & A Highlights
Q: Can you provide more details on the commercial preparation for deramiocel and the division of labor between Capricor and Nippon Shinyaku?
A: Linda Marban, CEO, explained that Nippon Shinyaku has about 125 employees in the US dedicated to the Duchenne space, focusing on getting ready for commercialization. Capricor's role is to shepherd deramiocel to market, while Nippon Shinyaku will handle sales and distribution. They are working together on market penetration modeling, launch activities, and market access.
Q: How are payers viewing the potential use of deramiocel alongside other premium-priced drugs for DMD?
A: Linda Marban noted that payers have responded positively, emphasizing that deramiocel is the only treatment targeting Duchenne cardiomyopathy. The therapy is expected to provide therapeutic benefits in an area largely unaddressed, potentially reducing hospitalization and mortality.
Q: What are Capricor's plans for using its cash reserves, especially with potential FDA approval and additional funds?
A: Linda Marban stated that Capricor is exploring opportunities for label expansion and focusing on their exosome pipeline. They are evaluating various opportunities to move the company forward, including potential in-licensing and expanding their pipeline.
Q: Can you provide details on the new expanded manufacturing facility and its cost?
A: Anthony Bergmann, CFO, mentioned that the expansion is within their current San Diego footprint. The original clean room was built for under a couple of million dollars, and they expect to expand at a reasonable cost. More details will be provided as they progress.
Q: What is the status of discussions with the FDA regarding an ADCOM, and how might it affect the HOPE-3 data?
A: Linda Marban stated that they are waiting to hear from the FDA about an ADCOM. They are prepared for one if needed, but the FDA has indicated that HOPE-3 data is not required for the current BLA. The focus is on HOPE-2 data and natural history comparisons.
Q: How does Capricor plan to address the European market and the potential partnership with Nippon Shinyaku?
A: Linda Marban explained that they are working with the EMA and consultants to build a program for Europe. They are negotiating with Nippon Shinyaku for a definitive agreement and are optimistic about their commitment to deramiocel.
Q: What is the potential for deramiocel in treating other heart diseases beyond DMD and BMD?
A: Linda Marban mentioned that Capricor is evaluating orphan cardiomyopathies and other opportunities to expand the use of deramiocel. They are considering areas where the therapy's immunomodulatory and antifibrotic properties could be beneficial.
Q: What is the ideal baseline ejection fraction for deramiocel's impact, and how does this affect the eligible patient population?
A: Linda Marban stated that early treatment is better, with the greatest long-term stabilization seen in patients with ejection fractions of 45% or greater. They are working with cardiologists and neurologists to integrate deramiocel into the treatment paradigm for DMD cardiomyopathy.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
