Genetic Epilepsy Drug Boosts Stoke Stock

Stoke Therapeutics recently announced promising results from its Phase I/IIa clinical trial for STK-001, a drug candidate for the treatment of Dravet syndrome, a rare and severe form of epilepsy. The positive data has led to a significant surge in the company's stock value, with a 98% increase during Tuesday's morning trading session.

The study revealed that patients who received a 70-mg dose of STK-001 experienced a median 85% reduction in convulsive seizure frequency after three months, when combined with their existing anti-seizure medication. An extension study demonstrated that continued administration of lower doses, 30 mg and 45 mg, sustained seizure reduction and led to significant improvements in cognitive and behavioral aspects over a period of 12 months.

These improvements are notably different from the outcomes of a natural history study, which indicated that most patients with Dravet syndrome did not see a substantial decrease in seizures and experienced a decline in cognitive and behavioral abilities, even with anti-seizure medication.

The FDA has authorized the continuation of the treatment plan, allowing patients to receive three 70 mg doses followed by ongoing dosing at 45 mg. Stoke is planning to consult with the FDA about a potential registrational study based on this treatment strategy.

Dravet syndrome is a genetic disorder that emerges in early childhood and is marked by frequent, prolonged seizures that are often resistant to conventional treatments. It has a higher mortality rate compared to other epilepsy types, with a significant proportion of affected children not surviving into adulthood.

Affected children frequently suffer from intellectual disabilities and developmental delays, among other complications. Current treatments are unable to provide adequate seizure control, and there is no cure for the syndrome.

STK-001 represents a breakthrough as it could be the first therapy to address the genetic root of Dravet syndrome. Administered via a spinal tap, the antisense oligonucleotide therapy utilizes the non-mutated copy of the patient's gene to restore the function of the deficient sodium channel protein. This approach is fundamentally different from existing anti-seizure medications, which primarily aim to reduce abnormal brain electrical activity.

Another company, Encoded Therapeutics, received FDA approval for its Dravet syndrome gene therapy candidate in February 2024. The treatment is intended to be a one-time gene regulation therapy targeting the SCN1A gene, with a Phase I/II trial set to commence in the first half of this year.

Dravet syndrome remains a significant challenge for the medical community, and the development of novel therapies like STK-001 and Encoded Therapeutics' gene therapy candidate offers hope for improved treatment options and potentially better outcomes for patients.