Clinical-stage biotech Stoke Therapeutics ( NASDAQ: STOK ) shed ~28% after reporting interim data from two ongoing Phase 1/2a trials for STK-001, a candidate targeted at the genetic epilepsy disorder Dravet syndrome.
More than 90% of patients with seizures linked to Dravet syndrome do not respond to anti-seizure medicines.
Safety data of today’s readout based on 55 patients who received single or multiple doses of STK-001 and were followed up for three months after their last dose indicated that the 45mg dose level was well tolerated, the company said.
However, 27% (15/55) of patients experienced mild to moderate drug-related treatment-emergent adverse events (TEAE) none of which led to withdrawals of the study drug.
Based on 27 patients, efficacy data indicate a 55% of median reduction in convulsive seizure frequency from baseline in those who received three doses of STK-001 at 45mg.
The plasma levels of STK-001 were found to be dose-dependent, with 30mg and 45mg demonstrating a bigger increase compared to 20mg and 30mg.
“Based on these data, we believe we have entered the therapeutic range, which is translating to a clinical benefit for patients,” Chief Medical Officer of Stoke ( STOK ) Barry Ticho noted, adding more data from the trials are expected next year.
However, BTIG analyst Thomas Shrader with a Buy rating on STOK cited a safety finding that could slow the development of the treatment.
A “safety signal of increased protein in the [cerebrospinal fluid] that may correlate with the monthly CSF dosing and with epilepsy in general” will “likely slow development modestly and the company has agreed to limit dosing in two of the extension trials.” Shrader wrote.
Stoke ( STOK ) is a favorite on Wall Street with 11 Buy ratings and no Sell or Hold ratings.
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Stoke Therapeutics drops 27% after early data for anti-seizure medicine