Stoke Therapeutics Announces FDA Orphan Drug Designation for STK-001 in Dravet Syndrome

On August 7, 2019, VCBeat (WeChat ID: vcbeat) learned via Business Wire that biotechnology and pharmaceutical company Stoke Therapeutics (“Stoke”) announced that its investigational therapy for Dravet syndrome, STK-001, had received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

Dravet syndrome is a genetic epilepsy that occurs in infancy, affecting 8% of children with epilepsy under the age of three. This rare disease is characterized by frequent seizures, a prolonged course, and resistance to cure, often accompanied by symptoms such as developmental stagnation, cognitive decline, ataxia, speech disorders, and sleep disturbances. Compared to individuals with general epilepsy, those with Dravet syndrome have a higher mortality rate. Currently, there are approximately 35,000 patients with Dravet syndrome worldwide, but there is no effective treatment for this condition.

In patients with protein-deficiency genetic disorders such as Dravet syndrome, only one allele of a disease-associated gene pair is expressed normally, which is insufficient to maintain normal cellular function. Therefore, Stoke’s R&D team is dedicated to precisely regulating gene expression levels, pioneering new therapies for rare genetic diseases.

Stoke Therapeutics, founded in 2014 and headquartered in Massachusetts, USA, is a leading biotechnology and pharmaceutical company. The company provides new treatment options for patients with severe genetic diseases by building advanced medical technology platforms. Stoke went public on the NASDAQ Stock Exchange in the United States in June 2019, under the ticker symbol STOK.

STK-001 is an antisense oligonucleotide therapeutic developed by Stoke Therapeutics, primarily indicated for the treatment of Dravet syndrome. This drug upregulates the expression of the SCN1A allele, restoring protein levels to normal physiological ranges and thereby reducing seizure frequency. Currently, STK-001 is undergoing preclinical trials and holds promise as the first innovative therapy to elevate protein levels in patients with Dravet syndrome.

Based on the mechanism of upregulating protein levels, Stoke Therapeutics’ antisense oligonucleotide drugs are not only effective in treating hereditary epilepsy but also show certain efficacy against severe monogenic genetic disorders affecting the central nervous system, liver, and kidneys.

The FDA established the Office of Orphan Products Development primarily to support the development of drugs for rare diseases, providing safe and effective methods for prevention, diagnosis, and treatment to patients with rare diseases worldwide. In addition to FDA orphan drug designation, STK-001 will also receive a series of incentives, including tax credits for qualified clinical testing, waivers of FDA application fees, and seven years of market exclusivity in the United States.

Dr. Barry S. Ticho, Chief Medical Officer at Stoke Therapeutics, stated, “There is a critical need for the development of treatments for Dravet syndrome, and our drug undoubtedly brings new hope to families affected by this rare genetic disorder. Stoke Therapeutics aims to alleviate symptoms in patients with Dravet syndrome through STK-001, and even potentially cure the disease. We initiated preclinical trials for this drug in the first half of this year and plan to submit an Investigational New Drug (IND) application to the FDA for clinical trials of STK-001 in humans in early 2020.”

(Compiled by Xu Xiaoxue)