Financial Data and Key Metrics Changes - Revenues for Q1 2025 were $400,000, a decrease of $100,000 or 30% compared to $500,000 in Q1 2024, primarily due to decreased participant demand for the Bahamas registry trial [23][24] - Net loss for the quarter increased to approximately $5,000,000 from $4,000,000 for the same period in 2024 [25] - Cash and cash equivalents as of March 31, 2025, were $14,300,000, expected to fund operations into Q3 2025 [25][26] Business Line Data and Key Metrics Changes - Clinical trial revenue from the Bahamas registry trial decreased to $300,000 in Q1 2025 from $500,000 in Q1 2024 [24] - Contract manufacturing revenue increased to $100,000 in Q1 2025 from $33,000 in Q1 2024, reflecting increased activity [24] Market Data and Key Metrics Changes - The U.S. market opportunity for HLHS is approximately $1 billion, with an estimated 1,000 babies born with HLHS each year [9][35] - The company anticipates a market penetration of about 65%, representing a potential revenue opportunity of over $500 million in the U.S. alone [35] Company Strategy and Development Direction - The company is focused on advancing stem cell therapy with its lead product, Laromestrocel, targeting multiple chronic indications [4][5] - Key strategic priorities include completing the Phase 2b study for HLHS, preparing for BLA submission, and pursuing strategic collaborations for the Alzheimer's disease program [9][10] - The company aims for a potential BLA submission in 2026 if the current HLHS trial is successful [25][26] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the upcoming transformational milestones over the next 12 to 18 months, including potential BLA submissions and partnerships [6][14] - The management team emphasized the importance of rigorous trial design and the supportive relationship with the FDA [39][40] Other Important Information - The company has received multiple FDA designations for its development programs, including orphan drug and fast track designations for HLHS [16] - The company is ramping up BLA enabling activities and anticipates increased operating expenses and capital requirements throughout 2025 [25][26] Q&A Session Summary Question: Clarification on HLHS market and enrollment challenges - Management noted that they have successfully enrolled approximately 95% of the trial population, which is a significant achievement for an orphan disease [32][33] Question: Impact of FDA leadership changes on BLA filing - Management stated that recent interactions with the FDA have been supportive and collaborative, with no indications that leadership changes would impact the BLA [39][40] Question: Current manufacturing capacity and plans for scaling operations - The company believes its current capacity can produce at least 1,500 doses of Laromestrocel, with plans to enhance GMP systems and potentially partner with a CDMO for commercial capabilities [43][44] Question: Value-based pricing applicability for Laromestrocel - Management indicated that they are considering value-based pricing and referenced a similar product priced at approximately $1.5 million for treatment, suggesting a potential for substantial revenue generation [52][53] Question: Salesforce requirements for HLHS and commercial strategy - Management highlighted that the 12 centers involved in the clinical trials conduct nearly 80% of the surgeries, indicating a focused commercial strategy with a small salesforce [60][61]

Financial Data and Key Metrics Changes - Revenues for Q1 2025 were $400,000, a decrease of $100,000 or 30% compared to $500,000 in Q1 2024, primarily due to decreased participant demand for the Bahamas registry trial [24][25] - Net loss for the quarter increased to approximately $5,000,000 from $4,000,000 for the same period in 2024 [26] - Cash and cash equivalents as of March 31, 2025, were $14,300,000, expected to fund operating expenses into Q3 2025 [26] Business Line Data and Key Metrics Changes - Clinical trial revenue from the Bahamas registry trial decreased to $300,000 in Q1 2025 from $500,000 in Q1 2024 [25] - Contract manufacturing revenue increased to $100,000 in Q1 2025 from $33,000 in Q1 2024, reflecting increased activity [25] Market Data and Key Metrics Changes - The U.S. market opportunity for HLHS is approximately $1 billion, with an estimated 1,000 babies born with HLHS each year [10][37] - The company anticipates a market penetration of about 65% for laromastrocel, representing a potential revenue of over $500 million in the U.S. alone [39] Company Strategy and Development Direction - The strategic plan focuses on the efficient execution of the development program for laromastrocel, with key priorities including HLHS BLA preparedness and seeking partnerships for the Alzheimer's disease program [10][11] - The company aims for a potential BLA submission in 2026, contingent on positive results from ongoing clinical trials [26][27] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming 12 to 18 months, highlighting multiple critical milestones that could be transformational for the company [6][15] - The management team emphasized the importance of rigorous trial design and the supportive relationship with the FDA, indicating confidence in the regulatory pathway [41][42] Other Important Information - The company has received five FDA designations for its lead product, laromastrocel, including orphan drug and fast track designations [17] - The enrollment for the Phase 2b clinical trial for HLHS is nearing completion, with approximately 95% enrollment expected to finish in Q2 2025 [11][20] Q&A Session Summary Question: Clarification on HLHS market and laromastrocel eligibility - Management confirmed that the majority of children who undergo stage two surgery will be considered for laromastrocel treatment once approved, with minimal exclusion criteria [34][36] Question: Impact of FDA leadership changes on BLA - Management stated that recent interactions with the FDA have been supportive and collaborative, with no indications that leadership changes would impact the BLA submission [41][42] Question: Manufacturing capacity and scaling plans - The company believes its current manufacturing capacity can produce at least 1,500 doses of laromastrocel, with plans to ramp up GMP systems and potentially partner with a CDMO for commercial capabilities [45][46] Question: Value-based pricing applicability - Management indicated that value-based pricing is applicable, with potential benchmarks from recent drug launches in the rare disease space, suggesting a premium pricing strategy for laromastrocel [51][56] Question: Salesforce requirements for HLHS - Management noted that the 12 centers involved in the ELPIS two study conduct the majority of surgeries, indicating a focused sales strategy with a small team [61][62] Question: Adaptive protocol for Alzheimer's study - The adaptive design will allow for a seamless transition from Phase 2 to Phase 3, with an interim analysis planned to assess efficacy before full approval [68][70]