Capricor Therapeutics(US:CAPR)2021-08-13 03:14Financial Data and Key Metrics Changes - As of June 30, 2021, the company's cash and cash equivalents totaled approximately $38.1 million, an increase from approximately $32.7 million on December 31, 2020, indicating a positive cash flow situation [21] - The net cash used in operating activities for the first half of 2021 was approximately $7.8 million, with a net loss of approximately $9.9 million compared to a net loss of approximately $5.6 million for the first half of 2020 [22] Business Line Data and Key Metrics Changes - Research and development expenses for Q2 2021 were approximately $3.4 million, up from approximately $1.8 million in Q2 2020, reflecting increased investment in clinical programs [22] - General and administrative expenses for Q2 2021 were approximately $1.2 million, compared to approximately $1 million in Q2 2020, indicating a slight increase in operational costs [22] Market Data and Key Metrics Changes - The company is planning to expand its footprint to San Diego, California, to enhance its research capabilities and attract talent, which is expected to support its clinical programs [5][19] - The company is engaging with payers regarding the reimbursement potential for CAP-1002, with projected price targets in line with other cell and gene therapies for rare diseases [10] Company Strategy and Development Direction - The company plans to proceed with a Phase 3 pivotal trial for CAP-1002 in Duchenne muscular dystrophy (DMD) after a productive end-of-phase meeting with the FDA [7] - The company is also advancing its engineered exosome platform technology, with plans to develop a multivalent COVID-19 vaccine and explore other therapeutic indications [14][19] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential impact of CAP-1002 in treating severe COVID-19, citing positive data from previous studies and ongoing enrollment in the INSPIRE trial [13][12] - The company is committed to securing a partner for the commercialization of CAP-1002 and is actively discussing potential partnerships [10][36] Other Important Information - The company has recruited a team of experienced scientists for its exosome platform technology, which is expected to drive innovation in vaccine development [15] - The company anticipates submitting an IND for its COVID-19 vaccine in the fourth quarter of 2021, despite delays due to supply chain challenges [18] Q&A Session Summary Question: Details on trial endpoints for CAP-1002 - The primary efficacy endpoint for the Phase 3 trial will be the PUL 2.0, with secondary endpoints including cardiac measures [24] Question: Eligibility of HOPE-2 patients for HOPE-3 - Patients from HOPE-2 will not roll directly into HOPE-3 but will continue in an open-label extension study [25] Question: Estimated cost of HOPE-3 study - The estimated cost for the HOPE-3 study is around $15 million, which is considered low for a Phase 3 trial [28] Question: Clinical development of the booster vaccine - The company is actively exploring the booster vaccine strategy and has received supportive feedback from the U.S. government [29] Question: Route of administration for the booster vaccine - The booster vaccine will be administered via intramuscular injection, similar to existing vaccines [32] Question: Plans for EUA submission if COVID-19 trial data is positive - The company is open to all possibilities regarding EUA submission based on the trial data [33] Question: Epigenetic modification profile of CAP-1002 exosomes - Preclinical work has shown that CAP-1002 may suppress certain cytokines, but further data from the current study is being analyzed [34] Question: Focus of the INSPIRE trial - The company is currently focused on severe COVID-19 but may explore broader applications for respiratory distress in the future [42] Question: Manufacturing costs and scalability of exosomes - The company is confident in solving manufacturing challenges for engineered exosomes and is in scale-up mode [44]