Financial Data and Key Metrics Changes - The company ended the quarter with $295 million in cash, with an additional $50 million available under a debt facility for the year [29] - The company anticipates receiving approximately $16 million from the exercise of outstanding common warrants by year-end, extending its financial runway into 2027 [30] Business Line Data and Key Metrics Changes - The company is preparing for the U.S. launch of epitigramab, pending regulatory approval, with a target action date of September 22 [7][9] - Epitigramab has shown statistically significant improvements in motor function in clinical trials, with a pivotal Phase III trial demonstrating a threefold higher chance of improvement compared to placebo [17] Market Data and Key Metrics Changes - Approximately 10,000 patients in the U.S. are currently living with SMA, with about two-thirds having received SMN-targeted therapies [26] - Globally, around 35,000 patients have received SMN-targeted therapies, indicating a significant market opportunity for epitigramab [27] Company Strategy and Development Direction - The company aims to become a global biotech powerhouse by focusing on regulatory approvals for epitigramab, expanding its use in additional neuromuscular diseases, and maintaining disciplined capital allocation [6][31] - The first European launch of epitigramab is planned for Germany in 2026, with ambitions to reach patients across Europe, Asia Pacific, and Latin America [10][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing dialogue with the FDA, indicating a collaborative approach towards the review of the BLA [9][35] - The company is committed to ensuring that no patient with SMA is left behind, emphasizing the urgency of delivering epitigramab to the SMA community [32] Other Important Information - The company is preparing for a global launch of epitigramab, with a focus on addressing the unmet needs in muscle strength and motor function for SMA patients [12][24] - The EMBRAZE study demonstrated that epitigramab increased lean mass preservation by over 54% compared to tirzepatide alone, highlighting its potential in treating obesity [13] Q&A Session Summary Question: Can you tell us more about the specific observations at the two sites? - Management acknowledged the observations were not surprising and emphasized their commitment to addressing them within the review cycle [34][35] Question: What feedback have you received from the FDA regarding the review of epitigramab? - Management reported a constructive late cycle meeting with the FDA, expressing optimism about the review process and potential labeling [41][44] Question: How are discussions with U.S. payers progressing? - The payer outreach has been positive, with discussions focusing on the durability of response and the unmet medical need for dual modality therapies [45][46] Question: What is the expected timeline for the OPAL trial enrollment? - Enrollment for the OPAL trial is set to begin in Q3, with further guidance on completion timelines to follow [80] Question: How does the company plan to address the budget impact of dual SMA therapies? - Management indicated that the rarity and severity of SMA, along with the compelling clinical benefits of epitigramab, will be considered in pricing discussions [68][70]