Stoke Therapeutics (STOK)

Executive Summary

Stoke Therapeutics is a clinical-stage RNA medicine company pioneering a novel approach to treat severe genetic disorders by upregulating functional protein expression from healthy genes. Its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform uses antisense oligonucleotides to increase the production of proteins that are under-expressed due to genetic haploinsufficiency. The lead program, zorevunersen (STK-001), is in Phase 3 development for Dravet syndrome, a devastating genetic epilepsy with high unmet need. The company has demonstrated promising Phase 1/2 data showing durable reductions in seizure frequency and improvements in cognitive function, supporting a disease-modifying potential. With a strong cash position and a focused strategy, Stoke aims to transform the treatment landscape for Dravet syndrome and expand its pipeline into other CNS disorders. The successful development of zorevunersen could validate the TANGO platform and unlock significant value for the company. Stoke is well-positioned to execute on its upcoming milestones. The Phase 3 EMPEROR trial (Dravet syndrome) is actively recruiting, and top-line data are anticipated in the second half of 2026. Additionally, the company expects to engage with regulatory authorities for potential accelerated approval pathways, building on encouraging long-term data from its Phase 2 study. Stoke's ability to deliver on these catalysts will be critical for near-term value creation. The company's focus on a validated target and robust clinical data de-risk the program, making it an attractive opportunity in the RNA therapy space. However, execution risk remains as the Phase 3 trial enrollment and data quality are key to regulatory success.

Upcoming Catalysts (preview)

• H2 2026Phase 3 EMPEROR Trial Top-Line Data Readout65% success
• H2 2026FDA/EMA Regulatory Interaction for Accelerated Approval40% success
• TBDPipeline Expansion Announcement (New Indication or Platform Update)50% success