Stoke Therapeutics Q2 Financial Results and Business Updates

Stoke Therapeutics, Inc., a biotechnology company focused on RNA medicine to restore protein expression, has disclosed its financial results for the second quarter of 2024, alongside key business updates, including progress on its lead drug candidate, zorevunersen (STK-001). This investigational antisense oligonucleotide (ASO) aims to treat Dravet syndrome, a severe genetic epilepsy.

Chief Executive Officer Edward M. Kaye, M.D., highlighted the significance of their earlier data, which support zorevunersen as a potential first disease-modifying treatment for Dravet syndrome by reducing seizures and enhancing cognitive and behavioral functions. He expressed gratitude to the FDA for lifting the Partial Clinical Hold on zorevunersen and indicated ongoing discussions with global regulatory bodies, aiming to finalize a Phase 3 study design by the end of the year.

Key updates from the company include the announcement that the FDA has lifted the Partial Clinical Hold on zorevunersen. Stoke intends to present positive data from its Phase 1/2a and open-label extension studies at the European Epilepsy Congress (EEC) in Rome, Italy, from September 7-11, 2024. They are also on track to provide updates on Phase 3 plans in the latter half of 2024 and to initiate a Phase 1 study of STK-002 for Autosomal Dominant Optic Atrophy (ADOA) this year.

Financially, as of June 30, 2024, Stoke Therapeutics reported $282.0 million in cash, cash equivalents, and marketable securities. Revenue for the second quarter included $4.8 million recognized from a License and Collaboration Agreement with Acadia Pharmaceuticals. The net loss for the same period was $25.7 million, or $0.46 per share, compared to a $30.7 million net loss, or $0.69 per share, the previous year. Research and development expenses saw a slight increase to $21.1 million, while general and administrative expenses rose to $13.0 million.

For the first six months of 2024, revenue was $9.0 million, with a net loss of $52.1 million, or $1.02 per share. Research and development expenses amounted to $43.5 million, and general and administrative expenses were $23.3 million. The rise in operating expenses was attributed to increased personnel costs, third-party contracts, consulting, and other development-related activities.

Dravet syndrome is a genetic epilepsy marked by frequent and prolonged seizures starting within the first year of life, often leading to a poor quality of life and a high risk of sudden unexpected death in epilepsy (SUDEP). There are no approved disease-modifying therapies for Dravet syndrome, which affects about one in 16,000 babies globally.

Zorevunersen (STK-001), currently under clinical trials, is designed to treat Dravet syndrome by targeting the SCN1A gene, which is responsible for the NaV1.1 protein. This investigational ASO aims to restore normal protein levels and reduce both seizures and non-seizure-related symptoms. It has received orphan drug designation from both the FDA and EMA, and rare pediatric disease designation from the FDA.

Stoke Therapeutics is also developing STK-002 for ADOA, a common inherited optic nerve disorder causing progressive vision loss. The company believes STK-002 can become the first disease-modifying treatment for ADOA by enhancing OPA1 protein expression, potentially stopping or slowing vision loss. Preclinical data have shown promising results, and STK-002 has been granted orphan drug designation by the FDA and a CTA authorization from the MHRA.

Overall, Stoke Therapeutics continues to advance its pipeline with a focus on genetic diseases caused by protein insufficiencies, leveraging its proprietary RNA medicine platform to develop potentially transformative therapies.