Ed Kaye’s Stoke Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The preclinical-stage biotech wants the cash to take an antisense oligonucleotide treatment for Dravet syndrome through to phase 3.
Stoke’s pitch to investors rests on the potential for its platform to generate drugs that upregulate the expression of proteins. While two companies, including Kaye’s former employer Sarepta Therapeutics, have won approvals for antisense oligonucleotides that upregulate protein expression, these are exceptions enabled by certain biological features. Neither drug is underpinned by a broadly applicable approach.
By interfering with the synthesis of productive and non-productive mRNA, Stoke thinks it can boost levels of a wider range of proteins, starting with Nav1.1. The loss of Nav1.1 channels is responsible for Dravet, a form of genetic epilepsy that affects around five people in 100,000 worldwide.
Stoke raised about $120 million last year to develop a drug, STK-001, designed to increase Nav.1.1 levels, leading to studies in mice linking single injections to the restoration of near-wild-type levels of the protein. Buoyed by the data, Stoke plans to start a phase 1/2 trial in the first half of next year and then move swiftly through clinical development and hit phase 3 before the IPO haul runs dry.
The plan requires investors to buy into the prospects of a biotech that is yet to generate clinical data. In its favor, Stoke has a drug that could radically improve the treatment of Dravet and a platform that may generate similarly promising candidates.
As it stands, the only antiepileptic drugs approved by the FDA in Dravet are GW Pharmaceuticals’ Epidiolex and Biocodex’s Diacomit. These drugs help many patients, but the complete eradication of seizures is rare. Also, the drugs only address the occurrence of seizures, not the symptoms.
Stoke hopes to improve on the seizure control rates and potentially address the intellectual and developmental disabilities associated with Dravet. To do so, it wants investors to bet on its preclinical program.