Stoke CEO: FDA Never Raised An Advisory Committee, And Five-Year Data Is A 'Review Issue'
Stoke plans to feed data to the FDA through the first half of 2027, with $358.8 million in cash expected to last into early 2028.
Stoke Therapeutics does not expect an FDA advisory committee for its Dravet syndrome drug, CEO Ian Smith told analysts on the company's third-quarter earnings call Thursday, saying there was "absolutely no discussion relating to an adcom" at an FDA meeting about a month ago. The company ended the quarter with $358.8 million in cash and securities.
A rolling submission, if the FDA agrees
Smith said Stoke plans to submit data to the FDA throughout the first half of 2027 and finish the application in the second half. The aim is to let the agency start reviewing early, though he said there is no guarantee of that until the FDA confirms a rolling review.
He also said the company reiterated a data readout in the third quarter of 2027, with a potential US approval in early 2028.
Stoke, which develops RNA-based medicines for severe genetic diseases, is running a Phase 3 study of zorevunersen in Dravet syndrome, a severe genetic epilepsy. Smith said about 110 of 162 enrolled patients have completed the Week 28 visit, where the main goal is measured, and no one has discontinued treatment.
What "review issue" means for the label
Asked by a Leerink Partners analyst what the FDA meant, Smith said Stoke asked whether five years of open-label extension data could go on the label. The FDA does not say yes at this stage, he said, but called it a review issue, meaning the totality of data will count.
Smith said the FDA also agreed to accept a multi-component assessment of Vineland-3 scores, which measure cognition and behavior, for the key secondary endpoint. A miss on the main goal was not discussed, he said, though close misses on a p-value can still be argued given the full data set.
In Europe, he said, individual Vineland subdomains still apply, and Biogen controls that filing. When a Jefferies analyst asked about Biogen's comments on pricing, Smith said he did not mean to speak for Biogen.
Cash, safety studies and the eye program
Chief Financial Officer Thomas Leggett said the quarter's net loss was $73.6 million, including $11 million of non-cash stock-based compensation. With Biogen reimbursements and a potential development milestone payment, he said Stoke expects its cash to fund operations into early 2028.
Chief Patient Officer Jason Hott said Stoke aims to start two more safety studies of the drug, in adults and in infants and toddlers, by the end of this year. The company also expects initial safety and efficacy data in the first half of 2027 from its Phase 1 study of STK-002 for autosomal dominant optic atrophy.
Stoke's next marker is the Week 52 data readout it has targeted for the third quarter of 2027.
Sources
- Stoke Therapeutics, Inc. (STOK) Reports Q3 Loss, Tops Revenue Estimates — Yahoo Finance
- Stoke Therapeutics GAAP EPS of -$1.08 misses by $0.16, revenue of $11.26M beats by $3.08M (STOK:NASDAQ) | Seeking Alpha — seekingalpha.com
- Stoke Therapeutics Announces Third Quarter 2026 Financial Results and Provides Business Updates – Company Announcement - FT.com — Financial Times
