August 13, 2025

Stoke Therapeutics release new data on Dravet syndrome drug

From my colleague Matt Herper: Stoke Therapeutics, which is developing an antisense drug aimed at treating Dravet's syndrome, a severe form of epilepsy that also leads to developmental delays and cognitive issues, released follow-up data late yesterday from its Phase 1/2 study out to three years.

Patients who received the highest doses showed a more than 75% reduction in seizures from baseline as well as continuing improvements in expressive communication, interpersonal relationships, and motor skills.

If those results are confirmed by a Phase 3 study now being run with Biogen, they could represent a substantial market for the treatment, called zorevunersen.

In a note to investors last month, analyst Andrew Tsai at Jeffries said a physician consultant said he would prescribe the drug to all of his patients with Dravet, even if the drug only had a benefit on seizures. That, Tsai wrote, could mean that sales could be greater than his $1.5 billion peak estimate should the medicine reach the market. Stoke has a market capitalization of $738 million.

CHRONIC DISEASE

Insmed wins approval for lung disease drug

Insmed yesterday received FDA clearance for the first treatment for bronchiectasis, a chronic inflammatory lung disease.

The company estimates the daily pill, called Brinsupri, can deliver peak sales of $5 billion, making it a growth driver that could help propel it into becoming a major biotech like Vertex Pharma.

The biotech is also studying the drug, a DPP1 inhibitor, in other diseases like chronic rhinosinusitis.

biotech

Expedition licenses immune drug from China's Fosun Pharma

Coincidentally, U.S.-based Expedition Therapeutics said it's licensing a DPP1 inhibitor from Chinese company Fosun Pharma in a deal worth up to $645 million. (That's the same type of drug as Insmed's that was just approved, as discussed above.)

The drug, called XH-S004, is in Phase 2 trials in China for bronchiectasis and in Phase 1 studies for chronic obstructive pulmonary disease (COPD).

Expedition, which was founded with the focus of licensing promising candidates from Asian companies, recently received seed financing from high-profile investors such as Venrock and BVF Partners, STAT previously reported. The biotech said at the time that it's particularly interested in treatments for immune and inflammatory diseases, as well as bispecific antibody therapies.

More around STAT