Stoke Therapeutics Announces Q3 Financial Results and Business Updates

Stoke Therapeutics, Inc., a biotechnology company focused on RNA medicine to restore protein expression, has announced its financial results for the third quarter of 2024. The company, listed on Nasdaq under the symbol STOK, is working on zorevunersen, an antisense oligonucleotide (ASO) designed to tackle the genetic cause of Dravet syndrome.

Edward M. Kaye, M.D., CEO of Stoke Therapeutics, highlighted the company's busy schedule as they gear up to present new data pertinent to their Phase 3 dosing regimen and finalize discussions with regulatory bodies on the study design. The assessments of behavior and cognition in Dravet syndrome patients have shown promising results, with significant effects observed within the first year of treatment, raising confidence in the potential of zorevunersen to address more than just seizure management.

Stoke Therapeutics has submitted abstracts for presentation at the American Epilepsy Society meeting in December. They plan to present new data from patients treated with an initial 70mg dose followed by 45mg maintenance doses. The proposed Phase 3 regimen includes multiple loading doses of 70mg followed by 45mg maintenance doses, which are currently under discussion with global regulatory agencies. The company has decided to delay the Phase 1 study of STK-002, aimed at treating autosomal dominant optic atrophy (ADOA), to concentrate on zorevunersen.

Key recent updates include the FDA lifting the Partial Clinical Hold on zorevunersen in August and the presentation of Phase 1/2a and open-label extension (OLE) study data at the European Epilepsy Congress in September. The company will also present at the Jefferies London Healthcare Conference in November, with a live webcast available on their website.

Financially, as of September 30, 2024, Stoke Therapeutics reported $269.2 million in cash, cash equivalents, and marketable securities. Revenue from the License and Collaboration Agreement with Acadia Pharmaceuticals was $4.9 million for the third quarter, up from $3.3 million in the same period in 2023. The net loss for the quarter was $26.4 million, or $0.47 per share, compared to $24.5 million, or $0.55 per share, the previous year. Research and development expenses rose to $22.2 million from $20.3 million, while general and administrative expenses increased to $12.7 million from $10.3 million.

For the nine months ending September 30, 2024, revenue from the same agreement was $13.9 million, compared to $6.0 million in 2023. The net loss for this period was $78.5 million, or $1.48 per share, compared to $77.7 million, or $1.78 per share, in the previous year. Research and development expenses were $65.7 million, up from $60.5 million, and general and administrative costs were $36.0 million, up from $30.7 million in 2023. The increase in operating expenses is primarily due to higher personnel costs, third-party contracts, consulting, facilities, and other costs associated with developing zorevunersen and STK-002, as well as research on additional therapeutics and expanding the company.

Dravet syndrome, a severe genetic epilepsy beginning in the first year of life, presents significant challenges, including frequent, prolonged seizures, intellectual disability, developmental delays, and a higher risk of sudden unexpected death in epilepsy (SUDEP). There are currently no approved disease-modifying therapies for Dravet syndrome, which affects about one in 16,000 infants globally.

Zorevunersen (STK-001) is an investigational medicine for Dravet syndrome that aims to restore normal levels of the NaV1.1 protein by using the non-mutant copy of the SCN1A gene. This approach seeks to reduce both seizures and associated co-morbidities. Zorevunersen has received orphan drug designation from both the FDA and EMA, and rare pediatric disease designation from the FDA.

Stoke Therapeutics, headquartered in Bedford, Massachusetts, is leveraging its TANGO (Targeted Augmentation of Nuclear Gene Output) technology to develop ASOs for restoring protein levels. Their first compound, zorevunersen, is undergoing clinical testing for Dravet syndrome. The company is also developing STK-002 for ADOA. Stoke’s primary focus is on haploinsufficiencies and central nervous system and eye diseases, although their approach shows potential for other organs and systems.