• The data was presented at the annual meeting of American Epilepsy Society (AES) held in Baltimore
   
• Stokes will apply for investigational new drug (IND) to the U.S. FDA in the first half of 2020

Massachusetts headquartered Stoke Therapeutics has reportedly presented new preclinical data on STK-001, a potential medicine used in the treatment of Dravet syndrome. 

Reportedly, the biotechnology company is involved in the development of novel therapies to treat the fundamental cause of various genetic diseases through upregulating protein expression.

The study revealed STK-001 showed distribution across the brain, achieving target engagement leading to an increase in Na_v1.1 protein expression throughout the cortex after dosing with a single intrathecal injection.

According to Edward M. Kaye, M.D., CEO at Stoke Therapeutics, the impact of Dravet syndrome extends beyond seizures and often leads to cognitive regression, speech impairment, ataxia, sleep disturbances or development stagnation. The condition is likely to affect multiple areas of the brain, with the cerebral cortex performing a vital role, he further added.

Additionally, the new data demonstrates the ability of STK-001 to largely distribute in the brain and stimulate target engagement and increased Na_v1.1 across the cortex.

For the record, Dravet syndrome is a serious and progressive type of genetic epilepsy that affects nearly 35,000 people in Canada, Japan, Germany, the United Kingdom, the United States, and France. Roughly 85% of cases of Dravet Syndrome are caused due to impulsive, heterozygous mutations in the SCN1A gene, leading to 50% of normal Na_v1.1 protein expression.

Stoke Therapeutics chose two STK-001 dose levels for carrying out the non-GLP study in order to assess safety, target engagement, brain biodistribution, and Na_v1.1 protein expression. 

The company aims to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) during the first quarter of 2020. Further, subject to acceptance of the IND, the company is planning to start the Phase 1/2 single-ascending dose study in adolescents and children diagnosed with Dravet syndrome in early 2020.

Source Credits: https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-presents-preclinical-data-biodistribution