Stoke Therapeutics, Inc.

Stoke did not provide revenue or EPS on this call, but it said quarter-end cash, cash equivalents and marketable securities were $354.3 million. Shortly after quarter-end, the company raised $65.7 million in net proceeds through its ATM program by selling approximately 2.1 million shares, bringing pro forma cash to approximately $420 million. Management said that, together with Biogen reimbursements and a potential development milestone payment, this should fund operations through a potential U.S. launch in early 2028. On guidance, the company reiterated a Phase III EMPEROR readout in the third quarter of 2027, a pre-NDA meeting later this year, a rolling NDA beginning in Q1 2027, and clinical module completion in Q3 2027.

Ian Smith framed the quarter as evidence that execution is becoming the focus, highlighting completion of EMPEROR enrollment and the absence of discontinuations as signs of confidence in zorevunersen’s tolerability and potential. He stressed that the company believes it is within 1 year of the Phase III readout and is preparing for a rolling NDA and commercial launch activities. He also positioned the broader platform as a growth story beyond Dravet, citing ADOA, SYNGAP1, and new CNS haploinsufficiency targets.

Thomas Leggett emphasized balance-sheet strength rather than operating-line details, saying the company ended the quarter with $354.3 million in cash, cash equivalents and marketable securities. He added that the post-quarter ATM raise generated $65.7 million in net proceeds from about 2.1 million shares, lifting pro forma cash to approximately $420 million. He said that, alongside Biogen reimbursements and a potential development milestone payment, the company expects its cash to support operations through a potential U.S. launch in early 2028.

Analysts pressed on what the EMPEROR milestones imply for readout confidence and on the pre-NDA meeting’s focus; management said it wants FDA alignment on rolling submission timing, the statistical analysis plan for secondary endpoints, and how to incorporate 5-year OLE data. The team also reiterated that the Phase III study is overpowered relative to initial assumptions because 162 patients enrolled versus 150 targeted and there have been 0 discontinuations, while Barry Ticho said the Vineland subdomains are each individually powered and the study was designed for a 2 to 3 point delta. Questions on pricing and label value were answered with market-research feedback: providers and payers reportedly view the long-term OLE data, published in NEJM, as the most compelling evidence, and management believes the product could support rare-disease, genetically targeted disease-modifying pricing. Analysts also asked about commercial site capacity, adult and infant/toddler studies, and ADOA timelines; management said top Dravet centers already run intrathecal programs, an adult study will begin later this year, and ADOA early safety/efficacy data are expected in the first half of next year.