Capricor Therapeutics(CAPR) - 2020 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of December 31, 2020, the company's cash, cash equivalents, and marketable securities totaled approximately $32.7 million, compared to approximately $9.9 million on December 31, 2019, indicating significant growth in liquidity [47] - The net loss for the fourth quarter of 2020 was approximately $4.2 million, compared to a net loss of approximately $1.5 million for the fourth quarter of 2019, reflecting increased operational costs [49] - For the full year of 2020, the net loss was approximately $13.7 million, compared to a net loss of approximately $7.6 million for the full year 2019, highlighting a trend of increasing losses [49] Business Line Data and Key Metrics Changes - The research and development expense for Q4 2020 was approximately $2.7 million, up from approximately $800,000 in Q4 2019, indicating a substantial increase in investment in R&D [48] - General and administrative expenses also rose to approximately $1.1 million in Q4 2020 from approximately $800,000 in Q4 2019, reflecting higher operational costs [48] Market Data and Key Metrics Changes - The company is focused on becoming a leading exosome RNA company, with plans to develop engineered exosomes and synthetic mRNAs for therapeutic applications [13][35] - The exosome platform is positioned to address a broad spectrum of diseases, leveraging the success of mRNA vaccines as a market opportunity [9][10] Company Strategy and Development Direction - The company is pivoting from traditional cell therapy towards an exosome platform, which is expected to provide significant opportunities in vaccine and therapeutic development [72] - The strategy includes developing a multi-antigen exosome-mRNA vaccine, with plans to initiate a Phase 1 clinical trial in Q3 of 2021, pending FDA approval [37] - The company aims to optimize its exosome platform for both vaccine and therapeutic applications, with a focus on monogenic diseases and other infectious diseases [35][38] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in 2020 and the potential of the exosome platform to revolutionize therapeutic delivery [5][45] - The company is awaiting FDA feedback on its pre-IND package and is prepared to move into clinical trials based on that feedback [37][57] - Management emphasized the importance of CAP-1002 for Duchenne muscular dystrophy and the ongoing discussions with the FDA regarding its approval pathway [41][42] Other Important Information - The company has entered into a Sponsored Research Agreement with Johns Hopkins University to support its exosome program, enhancing its research capabilities [6] - The company is exploring partnerships for CAP-1002, targeting corporate partners with existing products in the Duchenne muscular dystrophy space [61] Q&A Session Summary Question: How should Capricor be viewed going forward? - Management sees Capricor positioned between traditional cell therapy companies and those focused on engineered exosomes, emphasizing the shift towards RNA medicines in exosomes [52] Question: Will the mRNA strategy focus on internal development or partnerships? - The plan includes both internal programs and attracting partners for collaborative development, aiming for a plug-and-play model [54] Question: What is the expected cold chain requirement for the exosome-based mRNA vaccine? - Current expectations are for similar cold chain requirements as existing mRNA vaccines, with potential for a single-dose option being explored [55] Question: What work is still required for entering the clinic with the vaccine? - The company is ready to proceed into the clinic based on FDA feedback, leveraging its experience with regulatory processes [57] Question: What is the regulatory pathway for exosome-based therapeutics? - Management believes the regulatory process will be relatively straightforward, drawing on previous experiences with biologics [59] Question: What is the ideal partner for CAP-1002 development? - An ideal partner would have activity or commercial products in the Duchenne muscular dystrophy space, with ongoing discussions with multiple parties [61] Question: Plans for label expansion on post-COVID patients in the INSPIRE trial? - The company is open to label expansion based on trial data and the evolving landscape of COVID-19 treatment [63] Question: What additional studies are being conducted with CAP-1002? - The company is working with consultants and statisticians to analyze the impact of CAP-1002 in Duchenne muscular dystrophy [65] Question: Insights on the exosome platform compared to lipid nanoparticles? - The exosome platform shows promise in safety, repeat dosing, and targeting capabilities, with ongoing research to optimize these aspects [68][70]