Financial Data and Key Metrics Changes - As of June 30, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $122.8 million [23] - Revenues for Q2 2025 were zero, compared to approximately $4 million for Q2 2024, and total revenues for 2025 were zero compared to approximately $8.9 million for 2024 [23][24] - Research and development expenses for Q2 2025 were approximately $20.1 million, up from approximately $11.7 million in Q2 2024, and total R&D expenses for 2025 were approximately $36.3 million compared to approximately $21.8 million for 2024 [25][26] - General and administrative expenses for Q2 2025 were approximately $4 million, compared to approximately $1.8 million in Q2 2024, and total G&A expenses for 2025 were approximately $7 million compared to approximately $3.6 million for 2024 [26] - The net loss for Q2 2025 was approximately $25.9 million, compared to a net loss of approximately $11 million for Q2 2024, and the total net loss for 2025 was approximately $50.3 million compared to approximately $20.8 million for 2024 [26] Business Line Data and Key Metrics Changes - The company is focused on advancing daramycin for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, which is a significant unmet need in the DMD community [5][6] - The HOPE-three trial is fully enrolled with 104 patients, and the primary efficacy endpoint has been amended to Left Ventricular Ejection Fraction (LVEF) [9][10] Market Data and Key Metrics Changes - The company is actively working with the FDA to clarify the path forward for daramycin following a complete response letter received in July 2025 [6][12] - The company is also involved in Project NextGen, focusing on advancing next-generation vaccines for COVID-19 and other infectious diseases through its Stealth X vaccine [17][18] Company Strategy and Development Direction - The company aims to deliver transformative therapies for rare diseases, with a strong commitment to the DMD community [5] - The strategy includes working constructively with the FDA to define a clear path for daramycin's approval and preparing for potential commercial launch activities [8][16] - The company is also exploring opportunities in vaccine development through its exosome platform, which could lead to partnerships and expansion into other therapeutic areas [20][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of their data and the potential for daramycin to stabilize cardiac function in DMD patients [14][29] - The company remains focused on regulatory clarity and is optimistic about the upcoming Type A meeting with the FDA [35][64] - Management emphasized the importance of LVEF as a primary efficacy endpoint and the need for innovative thinking from the FDA regarding rare disease approvals [82][87] Other Important Information - The company has received formal acceptance of all items from its pre-license inspection, validating its quality systems and manufacturing capabilities [14][15] - The company is well-positioned financially, with over $120 million in cash, allowing it to support operations until late 2026 [28] Q&A Session Summary Question: What is the plan for unblinding the HOPE-three trial? - The company is waiting for FDA adjudication on requirements for HOPE-three before proceeding with unblinding [33] Question: What incremental data could be submitted to the FDA? - The company is considering submitting supplemental open-label extension data to support safety and efficacy [41] Question: How has the FDA review team changed? - The company is uncertain about changes in the review team but is looking forward to working with the FDA [111] Question: What are the key takeaways expected from the upcoming Type A meeting? - The company aims to clarify what is required for approval and the primary endpoint for HOPE-three [64] Question: What accounts for the increase in R&D expenses? - The increase is due to ongoing clinical studies and preparations for CMC endeavors [105]