Financial Data and Key Metrics Changes - As of June 30, 2025, the company's cash, cash equivalents, and marketable securities were approximately $630.5 million, down from $861.7 million as of December 31, 2024 [27] - Collaboration revenue increased to $14.2 million in Q2 2025 from $6.9 million in the prior year quarter, driven by cost reimbursements related to collaboration with Regeneron Pharmaceuticals [28] - R&D expenses decreased to $97 million in Q2 2025 from $114.2 million in the prior year quarter, primarily due to lower employee-related expenses and stock-based compensation [28] - G&A expenses decreased to $27.2 million in Q2 2025 from $31.8 million in the prior year quarter, mainly due to lower stock-based compensation [29] Business Line Data and Key Metrics Changes - The company is increasing enrollment in the Phase 3 study evaluating Nexi in ATTR cardiomyopathy to approximately 1,200 patients, up from the previous target of 765 patients [7][20] - Enrollment in the HALO study for LONVOZIE in HAE has exceeded expectations, with a notable screening of 41 patients in April alone [14][15] - The company anticipates completing enrollment in the HAE and ATTR polyneuropathy programs earlier than previously thought [6] Market Data and Key Metrics Changes - The company noted strong interest from both patients and physicians in its clinical trials, which has led to faster-than-expected enrollment [6][10] - The competitive landscape for TTR therapies is evolving, with the company positioning Nexi as a formidable competitor in a growing market [20][105] Company Strategy and Development Direction - The company is focused on transforming the treatment landscape for HAE and ATTR amyloidosis, with plans to launch LONVOZIE for HAE by 2027 [5][30] - The company is building its commercial and medical teams to prepare for a successful product launch, having filled key leadership positions [12] - The company aims to demonstrate a significant clinical benefit over existing therapies, particularly in the context of combining Nexi with stabilizers [62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's plans and execution, highlighting the strong demand for its clinical trials and the potential for significant clinical benefits from its therapies [12][30] - The company is committed to maintaining a solid cash runway to support its operating plans into 2027, despite the increased enrollment in clinical trials [29][40] Other Important Information - The Chief Medical Officer announced plans for retirement in August 2026, with a search for a successor already underway [13] - The company is focused on ensuring a seamless transition in leadership while continuing to advance its clinical pipeline [13] Q&A Session Summary Question: What is the target proportion of patients on stabilizers in the ATTR study? - The company estimates that approximately 70% of patients will be on stabilizers, which is higher than the initial estimate of 50-60% [35] Question: How will cash runway be affected by the trial expansion? - The company indicated that the increase in enrollment to 1,200 patients will not significantly impact cash runway or net cash burn guidance [40] Question: What is the latest regarding Phase 3 events accrual rate for Nexe? - The company is not disclosing specific event rates but is optimistic about the potential for robust outcomes based on emerging data [42] Question: How does the enrollment pace compare to competitors? - The company noted that despite the availability of new therapies, enrollment has increased, indicating strong interest from patients and physicians [49] Question: Will the study be powered to show a statistically significant difference in the subset on stabilizer background? - Yes, the study is now powered to show a statistically significant difference in that subset [76] Question: What are the qualities of patients enrolling in gene editing studies? - Patients are motivated to find permanent solutions for their diseases, as evidenced by their willingness to wash out from standard therapies to participate in trials [80] Question: How does the company view the competitive landscape for Nexi? - The company believes Nexi will be a formidable competitor in the market, particularly due to its unique profile and the unmet needs in the treatment of ATTR amyloidosis [105]