Capricor Therapeutics(US:CAPR)2025-11-10 22:30Financial Data and Key Metrics Changes - As of September 30, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $98.6 million, which is expected to cover anticipated expenses into the fourth quarter of 2026 [17] - Revenue for Q3 2025 was zero, compared to approximately $2.3 million for Q3 2024, and revenue for the first three quarters of 2025 was zero, compared to approximately $11.1 million for the same period in 2024 [18] - The net loss for Q3 2025 was approximately $24.6 million, compared to a net loss of approximately $12.6 million for Q3 2024, and the net loss for the first three quarters of 2025 was approximately $74.9 million, compared to approximately $33.4 million for the same period in 2024 [19] Business Line Data and Key Metrics Changes - The company has administered over 800 infusions to approximately 150 boys and young men with Duchenne muscular dystrophy, demonstrating a strong and consistent safety profile for deramiocel [8] - The HOPE-3 phase 3 clinical study focuses on non-ambulant individuals, a patient population that has historically had limited clinical research dedicated to it [4] Market Data and Key Metrics Changes - The company is preparing for potential global expansion while closely monitoring evolving U.S. and international pricing policies [15] - The FDA has classified the resubmission of the BLA as Type 2, which means the review period can be up to six months, but there is precedent for faster review times [11] Company Strategy and Development Direction - The company aims to bring forward the first therapy that directly addresses Duchenne muscular dystrophy-associated cardiomyopathy, emphasizing the life-limiting cardiovascular impact of the disease [8] - The company is engaging both neurology and cardiology specialists to ensure an integrated approach to patient care should deramiocel receive approval [15] - The company is advancing its StealthX program under Project NextGen to develop next-generation vaccines for COVID-19 and other infectious threats [16] Management's Comments on Operating Environment and Future Outlook - Management remains highly motivated to achieve approval for deramiocel as early as possible in 2026, well ahead of the September 30, 2026 deadline for priority review voucher eligibility [11] - Management expressed confidence in the potential of deramiocel to stabilize disease progression and preserve both muscle and heart function [12] Other Important Information - The company has published a peer-reviewed paper detailing new mechanistic insights into deramiocel's mechanism of action, reinforcing the biologic rationale for its development program [12] - The San Diego commercial facility is fully operational and preparing for GMP production activities, addressing all CMC-related items cited in the CRL [14] Q&A Session Summary Question: What to expect from the upcoming HOPE-3 data? - The company will release top-line data including primary and key secondary endpoints as soon as available and will host a conference call to explain the implications of the data [22] Question: Are there any statistical changes in the study regarding left ventricular ejection fraction? - The study was always powered to measure ejection fraction, and the results from previous studies provide strong support for this analysis [23] Question: How will the statistical analysis plan handle the primary and secondary endpoints? - The primary endpoint remains the performance of the upper limb, and the analysis will focus on both combined cohorts and specifically on Cohort B [25] Question: What is the FDA's view on cohort B? - The focus on cohort B is due to its importance in addressing manufacturing concerns and ensuring the efficacy data is tied to the approved facility [41] Question: What is the potential for label expansion upon approval? - The company will discuss potential label expansion during conversations with the FDA, contingent on the data outcomes [50]