- Takeda, Japan's largest drugmaker,on Wednesday said that it had stopped two mid-stage clinical trials of an experimental sleep disorder treatment as an "immediate precautionary measure" after observing a "safety signal."
- While offering few other details, Takeda said dosing of patients in the trials was suspended so it could better assess the potential risks and determine next steps for the research program.
- The drug, called TAK-994, is in development for two types of narcolepsy and another sleep disorder called idiopathic hypersomnia. Takeda is developing two other treatments that are designed to work similarly by turning on a cellular switch in the brain.
TAK-994 is one of about a dozen compounds in what Takeda dubs the "first wave" in a planned revitalization of its research and development in cancer, uncommon disorders and neurological diseases. Takeda's turnaround plan was accelerated in 2019, when the company closed its $62 billion acquisition of Shire, a rare disease drugmaker.
Early study data for a related compound to TAK-994 around the same time appeared to strengthen Takeda's confidence in targeting what are known as orexin neurons, the loss of which is thought to drive disorders like narcolepsy.
TAK-994, as well as the related compound TAK-925, act as orexin "agonists," activating a receptor in the brain and potentially restoring neurotransmitter activity.
Takeda's orexin agonist treatments, alongside a would-be therapy for rare epilepsies, make up the neuroscience portion of the "first wave" pipeline, and are forecast by the company to be potential billion-dollar sales opportunities.
For now, however, testing of TKA-994 is on hold as Takeda parses the safety data it's collected to date.
"We are working to quickly assess the totality of available data to inform the further development of TAK-994," said Sarah Sheikh, head of the company's neuroscience unit, in a statement.
The two studies Takeda suspended appear to be Phase 2 clinical trials in adults with two types of narcolepsy. The first began in September 2019, while the second got underway this March.
Results are meant to inform design of a Phase 3 study and help support advancement of the drug to regulators by 2024.
If successful, Takeda's narcolepsy treatment would compete with three products marketed by Jazz Pharmaceuticals. Meanwhile, the biotech Alkermes is preparing to begin testing an orexin agonist in narcolepsy.