Stoke Therapeutics Q1 2024 Financial Results and Business Updates

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company committed to addressing severe diseases by enhancing protein expression through RNA-based medicines, has reported its financial results for the first quarter of 2024, alongside updates on its ongoing projects, particularly STK-001. This investigational antisense oligonucleotide (ASO) is being developed as a pioneering treatment for Dravet syndrome, a severe genetic epilepsy.

Edward M. Kaye, M.D., CEO of Stoke Therapeutics, emphasized the significant progress made in advancing STK-001. Recent data have shown notable and sustained reductions in seizure frequency and improvements in cognition and behavior among patients, indicating that STK-001 could be a disease-modifying treatment for Dravet syndrome. The company is preparing to discuss a randomized, controlled registrational study design with regulatory agencies, with updates expected in the latter half of 2024.

Recent milestones include positive data from Phase 1/2a and open-label extension (OLE) studies involving 81 children and adolescents with Dravet syndrome. These studies indicated substantial and durable reductions in seizure frequency and improvements in cognitive and behavioral measures, supporting the potential for STK-001 as a disease-modifying therapy. The drug has generally been well-tolerated in doses up to 70mg. Stoke Therapeutics plans to present these findings at upcoming conferences in May 2024.

The company also announced plans to initiate the Phase 1 study (OSPREY) of STK-002 for Autosomal Dominant Optic Atrophy (ADOA) within 2024. Initial data from the FALCON natural history study of ADOA will be presented at a conference in May 2024. Additionally, Stoke's collaboration with Acadia Pharmaceuticals continues, focusing on RNA-based treatments for rare genetic neurodevelopmental diseases, including Rett syndrome and SYNGAP1.

Financially, as of March 31, 2024, Stoke Therapeutics held $178.6 million in cash and cash equivalents. In April, they bolstered their financial position with $120.3 million in net proceeds from a public follow-on offering. The company’s revenue for the first quarter of 2024, primarily from a collaboration with Acadia Pharmaceuticals, was $4.2 million, a slight decrease from the same period in 2023. Research and development expenses rose to $22.4 million, reflecting increased costs related to personnel, third-party contracts, and consulting.

Dravet syndrome is a severe genetic epilepsy characterized by frequent, prolonged, and refractory seizures that begin within the first year of life. It is difficult to treat and has a poor long-term prognosis, often accompanied by intellectual disability, developmental delays, and other complications. Currently, there are no approved disease-modifying therapies for Dravet syndrome.

STK-001 is designed to address the genetic cause of Dravet syndrome by upregulating NaV1.1 protein expression. This approach leverages the non-mutant copy of the SCN1A gene to restore normal NaV1.1 levels, potentially reducing seizures and other comorbidities. The FDA and EMA have granted orphan drug designation to STK-001, and the FDA has also given it a rare pediatric disease designation.

Autosomal Dominant Optic Atrophy (ADOA) is the most common inherited optic nerve disorder, leading to progressive and irreversible vision loss. There is no approved treatment for ADOA, which affects approximately one in 30,000 people globally. STK-002 aims to be the first disease-modifying therapy for ADOA by upregulating OPA1 protein expression, potentially halting or slowing vision loss.

Stoke Therapeutics uses its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to develop ASOs that selectively restore protein levels. The company's mission is to address haploinsufficiencies and diseases of the central nervous system and the eye. Stoke Therapeutics is headquartered in Bedford, Massachusetts, with additional offices in Cambridge, Massachusetts.