Stoke Therapeutics Files for $86 Million IPO to Advance Antisense Oligonucleotide Therapy STK-001 for Dravet Syndrome

VCBeat (WeChat ID: vcbeat) has learned that biopharmaceutical company Stoke Therapeutics recently announced its upcoming initial public offering on the Nasdaq Stock Market under the ticker symbol STOK. The company aims to raise $86 million through the IPO, with the proceeds designated for the research and development of therapies for rare genetic diseases.

Stoke Therapeutics, founded in Massachusetts, USA, in 2014, is a biopharmaceutical company established by Dr. Adrian Krainer, an expert in the field of RNA splicing. The company is developing antisense oligonucleotide drugs that target RNA splicing to increase gene expression for the treatment of genetic disorders caused by protein deficiency due to single-gene mutations.

According to Crunchbase, Stoke Therapeutics has completed two rounds of financing since its inception, raising a total of $130 million. In January 2018, the company closed a $40 million Series A financing round led by Apple Tree Partners. In October 2018, it completed a $90 million Series B financing round co-led by Apple Tree Partners and RTW Investments. Reportedly, Apple Tree Partners is the company’s largest shareholder, holding a 65.3% stake.

Stoke Therapeutics’ lead candidate is STK-001, a therapy for Dravet syndrome. This antisense oligonucleotide drug is administered via intrathecal injection every four months. By modulating RNA splicing, it induces cells to produce more mRNA, restoring target protein levels to near-normal ranges and thereby achieving therapeutic efficacy.

Stoke Therapeutics stated that its antisense oligonucleotide technology is not dependent on specific gene mutations. This means that a single drug from the company can be used to treat multiple genetic disorders. The company has identified that STK-001 has the potential to treat approximately 2,900 monogenic diseases.

Dravet syndrome is a severe epileptic disorder that can lead to frequent and prolonged seizures. The non-profit Dravet Syndrome Foundation estimates that one in every 15,700 newborns is affected by Dravet syndrome. Epidiolex, a drug under GW Pharmaceuticals, has been approved by the FDA for the treatment of Dravet syndrome. However, this medication can only reduce the frequency of seizures in patients and does not increase their protein levels. Stoke Therapeutics states that STK-001 aims not only to alleviate the symptoms of Dravet syndrome but also to fundamentally cure the disease and prevent potential sequelae.

Currently, STK-001 remains in the preclinical research phase. Stoke Therapeutics plans to submit an application to regulatory authorities in early 2020 to initiate first-in-human trials. If the company successfully obtains approval in the first half of 2020 to conduct a Phase 1/2 study in children and adolescents with Dravet syndrome, preliminary data will be released in 2021.

(Compiled by Jiao Yanli)