Stoke’s Early-Stage Data for Genetic Epilepsy Drug Sends Stock Skyrocketing

2024-03-26

临床结果临床研究寡核苷酸基因疗法

Pictured: A hand pointing to an upward stock chart on a screen/iStock, tadamichi Stoke Therapeutics on Monday reported positive Phase I/IIa data for its Dravet syndrome candidate and now hopes to leapfrog straight into a registrational study. The news sent the company’s stock skyrocketing 98% in Tuesday morning trading. Patients receiving a 70-mg dose of STK-001 achieved median reductions of 85% in convulsive seizure frequency at three months, on top of their current regimen of anti-seizure medicines. An open label extension study showed continued dosing of 30 mg and 45 mg, respectively, achieved durable seizure reduction and “clinically meaningful” improvements in cognition and behavior over 12 months. Stoke reported the improvements are in “stark contrast” to a natural history study that showed most patients not only had no meaningful reduction of seizures, but also had widening gaps in cognition and behavior despite treatment with anti-seizure medications. The FDA has now cleared patients to receive three doses at 70 mg followed by continued dosing at 45 mg. Stoke announced plans to meet with the agency regarding a registrational study of that treatment plan. Dravet syndrome is a rare, severe genetic epilepsy that begins in the first treatment of life. It’s characterized by prolonged, repeated and sometimes febrile seizures that are treatment resistant. It carries a higher mortality rate than other epileptic conditions, with up to 20% of children never reaching adulthood. Most children with the syndrome also suffer from intellectual disability and development delays among other comorbidities. There is no cure and current anti-seizure medicines often do not offer the seizure control needed. Stoke’s candidate has the potential to be the first disease-modifying therapy targeting the genetic cause of Dravet syndrome. Administered by spinal tap, the antisense oligonucleotide leverages the non-mutated copy of the patient’s gene to restore function of the lacking sodium channel protein. This is a vastly different approach than current anti-seizure medications which work by reducing abnormal electrical activity in the brain. Encoded Therapeutics received IND clearance from the FDA in February 2024 for its Dravet syndrome gene therapy candidate. The treatment aims to be a one-time gene regulation therapy targeting the SCN1A gene. The two-part, Phase I/II trial will begin in the first half of this year. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

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