Originally at: http://julieflygare.com/orexin-hypocretin-agonists-are-coming-part-i-reporting-back-from-world-sleep-2019/
Room 116: A Glimpse of the Future
It was a basic, boring conference room, but there was nothing basic or boring about Room 116 at the Vancouver Convention Center on Wednesday, Sept. 25, 2019 at 4:30 p.m. As the final session of the final of five days of the World Sleep Congress including World Narcolepsy Day, I felt wobbly and cataplectic, but I couldn’t go home, not yet. At exactly 4:30 p.m., I eagerly stepped into Room 116 to join narcolepsy experts, drug developers and other patient advocates for a look into the future.
This final session, simply titled “Narcolepsy” – included researchers sharing important new results related to novel and upcoming narcolepsy therapies. In July 2018, I reported here on the unprecedented amount of drug development underway for narcolepsy. Since then, the FDA has made three important approvals and more drug development continues on, full-steam ahead!
So it’s time for an update. Part I will catch you up to speed on orexin/hypocretin agonists, and Part II will share many more exciting developments.
(Please note: I am not a scientist or doctor. You should always speak with your narcolepsy specialist about treatment options and whether a clinical trial would be a good option for you. However, I hope this post helps provide access to information, because when navigating a complex healthcare system with a serious condition like narcolepsy, information is power.)
Advancing Orexin-Related Therapies
Since 1999, we’ve known that type 1 narcolepsy with cataplexy is caused by a selective loss of neurons producing the neuropeptide orexin (or hypocretin), which plays a central role in maintaining wakefulness. However, finding compounds able to cross the blood-brain barrier and mimic the function of orexin has been scientifically challenging. Over the past 20 years, a few approaches have been explored, but promising research started coming out of Japan a few years ago with Takeda Pharmaceuticals.
So when Dr. Rebecca Evans took the stage in Room 116 to present Takeda’s first-in-human clinical trial findings for the orexin 2 receptor selective agonist, TAK-925, the excitement was palpable. I felt honored to be in the room as these outcomes were shared publicly for the first time here.
- Background: In 2017, a patent for orexin 2 receptor selective agonists, including the clinical candidate TAK-925 was claimed. In April 2018, the orexin 2 receptor-selective agonist, TAK-925, was found to significantly increase wakefulness and recover cataplexy-like episodes in mice with a type of narcolepsy.
- A second publication reported on the pharmacological and electrophysiological characterization of TAK-925 from in vitro (petri dish-type studies) and in vivo (studies in wild-type mice, common marmosets, and cynomolgus