Actelion’s successor Idorsia reported its candidate for insomnia met the primary endpoint in Phase II and will advance to Phase III.

In its first report from the clinic last Friday, Idorsia announced positive results from a Phase II trial for its insomnia candidate ACT-541468 (DORA), which it inherited from its illustrious parent, Actelion. The drug showed “the desired effect on sleep maintenance and onset,” as well as a favorable safety profile, and based on these results, it will advance to Phase III to confirm its effects.

Martine Clozel, who continues to serve as CSO after her stint as such at Actelion before its acquisition, explained the drug’s history, “more than 20,000 compounds were synthesized and studied before ACT-541468 was selected. Its discovery is the result of an extensive effort to combine two important characteristics of a sleep medication: maintaining efficacy throughout the night, and a low potential for a next-day residual effect.”

The drug is a dual orexin receptor antagonist (hence DORA), whose ligand promotes wakefulness. Since it was only discovered in 1998, the peptide’s effects are still under investigation but its major role is thought to be the integration of metabolism, circadian rhythms and sleep debt to determine wakefulness.

idorsia-actelion-phase-ii-insomnia

Originally launched by Actelion, the program compared the effect of inhibiting the system with ACT-541468 to those of the benzodiazepine zolpidem, the first-line treatment for insomnia better known as Ambien, for reference. The 418 patients generally tolerated the investigational drug well, took less time to fall asleep and spent less time awake after falling asleep. No serious adverse events were reported.

“We couldn’t have asked for a better start for Idorsia,” commented Jean-Paul Clozel, CEO of Idorsia previously of Actelion. The report bumped the company’s stock up by 6% on Friday, and the share price continued to climb over the weekend. The market cap now stands at 2.16B CHF on the Swiss Exchange.

With these positive results in hand, Idorsia is planning to start a Phase III trial “as quickly as possible,” Guy Braunstein, Head of Global Clinical Development for the company, said in a statement. In the meantime, the specifics of the Phase II program are due to be presented and published in peer-reviewed publications in the near future.

Not many companies are taking on insomnia, though insufficient sleep is held to be a public health issueErgomed and Ferrer, in partnership, stand alone on the European radar. No doubt Idorsia will encounter competition as it develops a broad range of other programs in resistant hypertension, lupus and Fabry disease to name a few. But the “fledgling” company is not to be trifled with: with deep pockets and a hefty pipeline, Idorsia has earned itself a place on our now-outdated list of Europe’s largest biotechs.


Images from Kashin, Art Malivanov / shutterstock.com

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