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]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $144.8 million [23] - Revenues for Q1 2025 were zero, compared to approximately $4.9 million for Q1 2024, with the previous revenue being from a $40 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 $10.1 million in Q1 2024, and general and administrative expenses were approximately $3.1 million, compared to $1.8 million in Q1 2024 [24] - The net loss for Q1 2025 was approximately $24.4 million, compared to a net loss of approximately $9.8 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]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $144.8 million [22] - Revenues for Q1 2025 were zero, compared to approximately $4.9 million for Q1 2024, with the previous revenue being recognized from a $40 million distribution agreement [22][23] - Operating expenses for Q1 2025 were approximately $16.2 million for R&D, up from approximately $10.1 million in Q1 2024, and general and administrative expenses were approximately $3.1 million, compared to $1.8 million in Q1 2024 [24][25] - The net loss for Q1 2025 was approximately $24.4 million, compared to a net loss of approximately $9.8 million for Q1 2024 [25] Business Line Data and Key Metrics Changes - The company is focused on the BLA for daramycin, a therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with ongoing preparations for an FDA advisory committee meeting [4][5] - The company has been providing daramycin to open label extension patients for over three years, with plans for over 100 patients to transition to commercial products following potential BLA approval [12][13] Market Data and Key Metrics Changes - The company is negotiating with Nippon Shinyaku for the distribution of daramycin in Europe, with an extended negotiation period through the end of Q2 [17] - The company is also exploring opportunities for its technology in other global markets [17] 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. [11] - The strategy includes enhancing medical leadership and preparing physicians for prescribing daramycin, which targets inflammation and fibrosis associated with DMD [14][39] - The company is also developing its Stealth Exosome Platform technology for next-generation drug delivery [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the FDA's review process and the strength of the data supporting daramycin's efficacy and safety [4][30] - The company has a cash balance of approximately $145 million, with a runway extending into 2027 without additional cash infusions [19] - If FDA approval is received, the company expects to receive an $80 million milestone payment and a priority review voucher, potentially totaling over $200 million in non-dilutive cash [20] Other Important Information - The company has appointed Dr. Michael Binks as the new Chief Medical Officer, bringing over 25 years of experience in clinical development [12] - The San Diego GMP manufacturing facility is fully operational and producing doses of daramycin, with plans for expansion to meet potential demand [15][16] Q&A Session Summary Question: Has the site inspection in San Diego occurred? - The site inspection is scheduled for this quarter, and preparations are underway [28] Question: What is the status of negotiations with Nippon Shinyaku? - The company is actively negotiating with Nippon Shinyaku and evaluating opportunities for launching in Europe independently [34][37] Question: What are the key drivers of proof for the efficacy of daramycin? - The statistical significance of cardiac MRI data is a key driver, indicating that the treatment effects are unlikely due to chance [44][59] 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 [53][54] Question: What is the company's plan for the priority review voucher? - The current plan is to sell the priority review voucher to strengthen the balance sheet [69]