Stoke Therapeutics(STOK) - 2025 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended Q3 2025 with $328.6 million in cash, cash equivalents, and marketable securities, and raised an additional $48.7 million post-quarter through an ATM [20][21] - Total revenue for the quarter was $10.6 million, up from $4.9 million in Q3 2024, primarily due to a $6.7 million increase from collaboration with Biogen [20][21] - Net loss for Q3 was $38.3 million, or $0.65 per share, compared to a net loss of $26.4 million, or $0.47 per share, in the prior year, driven by increased operating expenses [20][21] Business Line Data and Key Metrics Changes - The company has made significant progress in the clinical development of zorevunersen, with over 200 patients identified for the phase 3 study, an increase from 150 previously reported [5][18] - R&D expenses rose to $37.7 million from $22.2 million a year ago, reflecting spending on the phase 3 study and early-stage pipeline advancements [20][21] - SG&A expenses increased to $16 million from $12.7 million, indicating investment in commercial capabilities [20][21] Market Data and Key Metrics Changes - The company is preparing for a meeting with the FDA to discuss expedited regulatory pathways for zorevunersen, which has received breakthrough therapy designation [6][19] - The market opportunity for zorevunersen is growing due to increased awareness of Dravet syndrome and the potential disease-modifying effects of the drug [6][19] Company Strategy and Development Direction - The company aims to rapidly scale its business to reach as many patients as possible with zorevunersen, focusing on medical affairs and commercial leadership [7][8] - There is a strategic emphasis on developing additional disease-modifying medicines, particularly in haploinsufficient disease areas [8][9] - The company is also launching a disease awareness campaign to improve diagnosis rates of Dravet syndrome through genetic testing [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the company's growth trajectory and the potential impact of zorevunersen on patients with Dravet syndrome [3][22] - The company is committed to educating the medical community and increasing awareness of Dravet syndrome [22] - Management highlighted the importance of the upcoming FDA meeting and the potential for expedited pathways to bring zorevunersen to market [19][22] Other Important Information - The company is actively recruiting for the phase 3 Emperor study, with 30 sites in the U.S., U.K., and Japan [17][18] - The first patient was randomized in August, and over 20 patients have been randomized so far [18] - The company anticipates completing enrollment in the second half of 2026, with data expected in late 2027 [39] Q&A Session Summary Question: What potential scenarios might be anticipated based on the PDD meeting with the FDA? - The company is preparing to present extensive data, including four-year safety data, to the FDA, which could lead to expedited pathways or a request to continue with the phase 3 study [26][27] Question: How clinically meaningful are the cognitive and behavioral improvements on the VABS-III data? - A change of one to three points on the VABS-III scale is considered clinically meaningful, with significant improvements observed in communication skills [31][33] Question: Can the company pursue early approval based solely on seizure reduction? - Yes, the company could seek approval based on seizure reduction, but it intends to pursue a label that reflects the disease-modifying potential of zorevunersen [37] Question: What are the common reasons for screen failure in the Emperor study? - Common reasons include not meeting the minimum seizure count required for enrollment, illness during the screening period, or family commitments [78] Question: How does the company plan to handle the differences in dosing schedules between phase 1/2 and phase 3 studies? - The company acknowledges the differences but believes the data from phase 1/2 and the open-label extension supports the dosing regimen planned for phase 3 [66]