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Capricor Therapeutics(CAPR) - 2025 Q1 - Earnings Call Transcript
CAPRCapricor Therapeutics(CAPR)2025-05-13 21:32
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Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately 144.8million[23]−RevenuesforQ12025werezero,comparedtoapproximately144.8 million [23] - Revenues for Q1 2025 were zero, compared to approximately 4.9 million for Q1 2024, with the previous revenue being from a 40milliondistributionagreementfullyrecognizedbytheendof2024[23][24]−OperatingexpensesforQ12025wereapproximately40 million distribution agreement fully recognized by the end of 2024 [23][24] - Operating expenses for Q1 2025 were approximately 16.2 million for R&D, up from 10.1millioninQ12024,andgeneralandadministrativeexpenseswereapproximately10.1 million in Q1 2024, and general and administrative expenses were approximately 3.1 million, compared to 1.8millioninQ12024[24]−ThenetlossforQ12025wasapproximately1.8 million in Q1 2024 [24] - The net loss for Q1 2025 was approximately 24.4 million, compared to a net loss of approximately 9.8millionforQ12024[25]BusinessLineDataandKeyMetricsChanges−ThecompanyisfocusedontheBLAfordaramycin,aimedattreatingDuchennemusculardystrophy(DMD)cardiomyopathy,withastrongemphasisonthesafetyandefficacydatasupportingtheapplication[5][8]−Thecompanyhasbeenprovidingdaramycintoallopen−labelextensionpatientsforoverthreeyears,withnearlyallHOPE−threepatientsnowinopen−labelextension[15]MarketDataandKeyMetricsChanges−ThecompanyisnegotiatingwithNipponShinyakuforthedistributionofderamycininEurope,withthenegotiationperiodextendedthroughtheendofQ22025[18]−Thecompanyisalsoexploringopportunitiesforitstechnologyinotherglobalmarkets[18]CompanyStrategyandDevelopmentDirection−Thecompanyaimstotransitionfromatranslationalmedicinecompanytoacommercialstageentity,activelyworkingwithNSPharmaonlaunchreadinessintheU.S.[12]−Thecompanyplanstohaveover100patientstransitionfromclinicaltocommercialproductfollowingpotentialBLAapproval[14]−Thecompanyisenhancingitsmedicalleadershiptoguidephysiciansthroughtheprescribingprocessfordaramycin[15]Management′sCommentsonOperatingEnvironmentandFutureOutlook−ManagementexpressedconfidenceinthestrengthofthedatasupportingtheBLAandtheunmetneedintreatingDMDcardiomyopathy,whichcouldleadtoFDAapproval[5][6]−ManagementnotedthattheFDAhasnotindicatedanysubstantiveissueswiththeapplication,providingconfidenceintheongoingreviewprocess[30]−Thecompanyhasacashbalanceofapproximately9.8 million for Q1 2024 [25] Business Line Data and Key Metrics Changes - The company is focused on the BLA for daramycin, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy, with a strong emphasis on the safety and efficacy data supporting the application [5][8] - The company has been providing daramycin to all open-label extension patients for over three years, with nearly all HOPE-three patients now in open-label extension [15] Market Data and Key Metrics Changes - The company is negotiating with Nippon Shinyaku for the distribution of deramycin in Europe, with the negotiation period extended through the end of Q2 2025 [18] - The company is also exploring opportunities for its technology in other global markets [18] Company Strategy and Development Direction - The company aims to transition from a translational medicine company to a commercial stage entity, actively working with NS Pharma on launch readiness in the U.S. [12] - The company plans to have over 100 patients transition from clinical to commercial product following potential BLA approval [14] - The company is enhancing its medical leadership to guide physicians through the prescribing process for daramycin [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of the data supporting the BLA and the unmet need in treating DMD cardiomyopathy, which could lead to FDA approval [5][6] - Management noted that the FDA has not indicated any substantive issues with the application, providing confidence in the ongoing review process [30] - The company has a cash balance of approximately 145 million, with a runway extending into 2027 without additional cash infusions [20] Other Important Information - The company is developing its Stealth Exosome Platform technology as part of a next-generation drug delivery platform, although this program has taken a backseat to daramycin [19] - The company is also working on a vaccine candidate for COVID-19 prevention under Project NextGen, with Phase 1 trials set to start in Q3 2025 [20] Q&A Session Summary Question: Has the site inspection in San Diego occurred? - The site inspection is scheduled for the upcoming quarter, and management feels prepared for it [28] Question: What is the status of negotiations with Nippon Shinyaku for Europe? - The company is actively negotiating and evaluating opportunities for commercialization in Europe, while also preparing to work directly with European authorities [37] Question: What are the key drivers of proof for the efficacy data? - The statistical significance of cardiac MRI data is a key driver, showing very little chance that the data is due to chance [46] Question: What is the plan if the FDA issues a CRL for efficacy? - The company would submit data from the HOPE-three trial for skeletal muscle dysfunction if a CRL is issued [55] Question: What is the plan for the PRV voucher? - The current plan is to sell the PRV voucher to strengthen the balance sheet [72]