Stoke Therapeutics, Inc. (STOK) has announced positive data from its Phase I/IIa and open-label extension (OLE) studies of STK-001 in children and adolescents (ages 2-18) with Dravet syndrome, a severe form of epilepsy. The investigational antisense oligonucleotide aims to address the underlying genetic cause of Dravet syndrome, offering a potential disease-modifying approach compared to current symptomatic treatments.
Clinically Meaningful Seizure Reduction and Cognitive Improvements
The data revealed that STK-001 treatment led to clinically significant outcomes, notably substantial and sustained reductions in convulsive seizure frequency. Furthermore, improvements were observed across multiple measures of cognition and behavior, indicating the potential for disease modification. These benefits were seen in patients with highly refractory Dravet syndrome, who were already on optimized anti-seizure medications. The treatment was also generally well-tolerated, with an acceptable safety profile.
In the cohort receiving a single 70mg dose of STK-001, a median reduction of 43% in convulsive seizure frequency from baseline was observed at three months post-dose, increasing to 57% at six months. Patients receiving two or three 70mg doses of STK-001 experienced even greater reductions, with median decreases of 85% and 74% at three and six months, respectively.
Durable Effects in Open-Label Extension Studies
Patients who continued treatment with STK-001 in the OLE studies maintained durable reductions in convulsive seizure frequency. The OLE studies also corroborated the improvements seen in cognition and behavior, reinforcing the potential for long-term benefits.
Regulatory Update and Path Forward
The FDA has granted Stoke Therapeutics permission to treat patients with an initial regimen of three 70mg doses of STK-001, followed by continued dosing at 45mg. Stoke is now planning to engage with regulatory agencies to discuss a registrational study utilizing this dosing schedule. An update on these discussions is expected later in 2024.
Financials
Stoke Therapeutics reported a loss of 60 cents per share in the fourth quarter of 2023, compared to a Zacks Consensus Estimate of a loss of 62 cents. Revenues for the quarter were $3 million, slightly below the Zacks Consensus Estimate of $4 million. Research and development expenses were $21.8 million, a marginal increase from $21.1 million in the same quarter of the previous year. General and administrative expenses rose to $10.6 million, up 13% year-over-year.
