Capricor Therapeutics(CAPR) - 2022 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of March 31, 2022, the company's cash and cash equivalents totaled approximately $58.3 million, compared to approximately $34.9 million on December 31, 2021, indicating a significant increase in liquidity [21] - The net cash provided by operating activities in Q1 2022 was approximately $24 million, primarily driven by a $30 million upfront payment from Nippon Shinyaku [21] - The net loss for Q1 2022 was approximately $7.8 million, compared to a net loss of approximately $5.2 million for Q1 2021 [22] Business Line Data and Key Metrics Changes - The CAP-1002 program for Duchenne muscular dystrophy (DMD) remains the primary focus, with a commercial partnership secured with Nippon Shinyaku, bringing in $30 million in upfront cash to fund the Phase III program [5][12] - Research and development expenses for Q1 2022 were approximately $4.9 million, up from approximately $3.2 million in Q1 2021, indicating increased investment in R&D [22] - General and administrative expenses were approximately $1.9 million in Q1 2022, compared to approximately $1.3 million in Q1 2021, reflecting a rise in operational costs [22] Market Data and Key Metrics Changes - The HOPE-3 Phase III clinical trial is designed to enroll approximately 70 patients in the U.S., targeting a patient population that comprises over half of the DMD market, or about 10,000 boys and young men per year [10][11] - The enthusiasm for the HOPE-3 trial is strong within the patient community, supported by positive anecdotal reports and published data from the Phase II HOPE-2 trial [12] Company Strategy and Development Direction - The company aims to partner with commercially talented companies to prepare for global expansion of the DMD program, while also advancing its bioengineered exosome platform [7][8] - The exosome platform is seen as a significant opportunity for drug delivery, with ongoing advancements in targeting capabilities and scalable manufacturing processes [15][16] - The company maintains a strong balance sheet with no near-term need for equity financing, providing at least two years of runway to execute its strategies [9] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, highlighting the successful partnership with Nippon Shinyaku and the positive reception of the HOPE-2 trial results [5][19] - The company is focused on advancing both the DMD program and the exosome platform, with plans to present at various conferences and provide updates on progress [19][20] Other Important Information - The agreement with Nippon Shinyaku includes potential milestone payments of up to $705 million, which could be realized during the course of the HOPE-3 trial [12] - The company retains rights to a rare pediatric voucher coupon upon FDA approval, which has historically been a valuable asset for companies with approved products [13] Q&A Session Summary Question: Interest in funding or accelerating younger patient trials by Nippon - Management has not discussed younger patients specifically with Nippon Shinyaku but is excited about opportunities for both non-ambulant and ambulant patients [24] Question: Enrollment speed for HOPE-3 trial - Management expects the trial to enroll well due to strong community feedback and the positive perception of CAP-1002's efficacy [25][26] Question: Advancements in exosome platform - Management is optimistic about the exosome platform's future and is making significant progress in core technologies and identifying indications for development [27][28] Question: Accounting for the $30 million payment - The $30 million payment was received at the end of Q1 2022 and will be recognized ratably over the duration of the clinical development program [29]