Capricor Therapeutics Announces Q2 2024 Financial Results and Corporate Update

Capricor Therapeutics, a biotechnology company focused on cell and exosome-based therapeutics for rare diseases, has announced its financial results for Q2 2024, alongside a corporate update. The company, listed on NASDAQ under the ticker CAPR, is advancing its lead asset, deramiocel, for treating Duchenne muscular dystrophy (DMD).

CEO Linda Marbán confirmed significant progress towards filing a Biologics License Application (BLA) for deramiocel, also known as CAP-1002. A positive pre-BLA meeting with the U.S. FDA discussed rolling submissions, potential label expansions, and commercial manufacturing plans. Further details will be shared once meeting minutes are finalized.

Recent data from the HOPE-2 open-label extension (OLE) trial demonstrated positive 3-year results, bolstering confidence in deramiocel's safety and efficacy. Capricor is also seeking a European partnership to strengthen its financial position.

The company's proprietary StealthX™ platform, focusing on exosome technology, continues to progress. Exosomes are extracellular vesicles that regulate various cell functions and hold promise for treating multiple diseases. Partnership opportunities and non-dilutive funding sources are being explored to advance this platform.

Operational highlights for the second quarter of 2024 include:

1. Deramiocel DMD Program:
- Regulatory Advancements: A pre-BLA meeting with the FDA supported the rolling BLA submission schedule and commercial manufacturing plans. Final outcomes will be communicated once meeting minutes are available.
- Clinical Trials:
- HOPE-2 OLE Trial: 36-month data showed a significant improvement in the PUL v2.0 score and various cardiac measures.
- HOPE-3 Phase 3 Trial: Enrollment is complete with 105 subjects. Top-line data from Cohort A is expected in Q4 2024.
- FDA Meetings: Positive outcomes from Type-B clinical and CMC meetings affirmed comparability between products manufactured at different facilities, allowing future use of products from the San Diego facility.

2. StealthX™ Exosome Platform:
- SARS-CoV-2 Vaccine: Selected for Project NextGen by the U.S. Department of Health and Human Services. The investigational product is in the manufacturing phase with delivery to NIAID planned by the end of 2024.
- Preclinical Data Presentations: Highlighted potential treatments for arginase-1 deficiency and targeted cargo delivery to mice's lower limbs.

3. Corporate Updates:
- Index Inclusions: Capricor has been added to the Russell 2000 and 3000 indexes.
- Conference Presentations: Presented at various industry conferences, including the Parent Project Muscular Dystrophy (PPMD) Annual Conference and the Cure Duchenne Futures National Conference.

Financially, Capricor reported a cash position of $29.5 million as of June 30, 2024, down from $39.5 million at the end of 2023. The company raised $2.1 million in net proceeds through common stock issuances during Q2 2024.

Revenue for Q2 2024 was approximately $4 million, slightly up from $3.9 million in Q2 2023. For the first half of 2024, revenue totaled $8.9 million compared to $6.9 million in the same period last year. The primary revenue source was the commercialization agreement with Nippon Shinyaku.

Operating expenses increased to $15.6 million in Q2 2024 from $11.7 million in Q2 2023, primarily due to higher research and development costs. The net loss for the quarter was $11 million, compared to $7.4 million in Q2 2023. For the first half of 2024, the net loss was $20.8 million, up from $15.1 million in the first half of 2023.

Capricor anticipates that its current financial resources will cover expenses into Q1 2025, excluding potential milestone payments from its Nippon Shinyaku agreement and any strategic capital uses.

Upcoming events for Capricor include participation in several industry conferences from September to October 2024, such as the H.C. Wainwright Global Investment Conference and the World Muscle Society Annual Congress.

Capricor Therapeutics continues to make strides in developing treatments for rare diseases, leveraging its innovative cell and exosome-based platforms. The company's focus on regulatory approvals, strategic partnerships, and advancing clinical trials positions it for future growth and success in the biotechnology sector.

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