Capricor Therapeutics(CAPR) - 2022 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of June 30, 2022, the company's cash, cash equivalents, and marketable securities totaled approximately $51.4 million, an increase from approximately $34.9 million on December 31, 2021 [28] - For the first half of 2022, net cash provided by operating activities was approximately $17.5 million, primarily driven by a $30 million upfront payment from Nippon Shinyaku [29] - The net loss for the first half of 2022 was approximately $14.9 million, compared to a net loss of approximately $9.9 million for the first half of 2021 [30] Business Line Data and Key Metrics Changes - The CAP-1002 program for Duchenne muscular dystrophy (DMD) has completed two successful clinical trials, demonstrating safety and tolerability in over 200 patients [6] - The ongoing Phase III pivotal study, HOPE-3, aims to enroll 70 patients across 15 to 20 investigative sites in the U.S., with seven patients already enrolled as of the call [7][8] Market Data and Key Metrics Changes - The target patient group for CAP-1002 comprises about 10,000 boys and young men in the U.S. who are largely non-ambulant and in the later stages of DMD [6] Company Strategy and Development Direction - The company is focused on executing the HOPE-3 clinical trial as a pivotal trial and preparing for potential commercial launch, including scaling up manufacturing [17][21] - A distribution and commercial agreement with Nippon Shinyaku includes a $30 million upfront payment and potential milestone payments up to $705 million [20] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency of bringing CAP-1002 to market quickly, highlighting the importance of the open label extension data in demonstrating disease-modifying activity [34][35] - The company plans to meet with the FDA to present open label extension data and discuss the path forward for regulatory approval [15][37] Other Important Information - The company is developing an exosome platform technology for drug delivery, with potential applications in vaccines and RNA delivery [22][24] - The current cash position is expected to cover anticipated expenses into the second quarter of 2024, allowing the company to focus on core programs [28][46] Q&A Session Summary Question: What is the goal for the upcoming FDA meeting regarding the open label extension data? - The ultimate goal is to get CAP-1002 to market as quickly as possible, with a focus on demonstrating the data's significance to the FDA [33][35] Question: Is there a potential for CAP-1002 to be on the market before the HOPE-3 readout? - The company is preparing for commercialization and will evaluate the best path forward after discussions with the FDA [37] Question: What are the plans for manufacturing capacity beyond the current setup? - The company is working on a plan to scale up manufacturing and will assess needs as they become apparent [40] Question: Can you provide details on the exosome vaccine candidate? - The exosome-based vaccine candidate is a twist on a previous COVID candidate, with potential applications for various infectious diseases [42][44] Question: How will the company manage its cash and potential pipeline expansion? - The company is prioritizing CAP-1002 and the exosome pipeline while being judicious with cash spending [46]