Takeda Pharmaceutical＊s experimental narcolepsy drug has significantly improved outcome for patients in late-stage clinical trials, and could become the first treatment to target the root cause of the chronic sleep disorder.

Patients who took the drug, called oveporexton, for 12 weeks experienced increased daytime alertness, fewer episodes of sudden muscle tone loss, and an overall improvement in quality of life compared to those given a placebo. The findings were part of studies published at World Sleep Congress held in Singapore, which runs through Sept. 10.

While no serious side effects were reported, the most common adverse events included insomnia as well as increased urinary urgency and frequency, according to the studies.

The data is ※transformative,§ Takeda＊s head of research and development Andy Plump said in an interview. ※It potentially offers a functional cure for many patients with narcolepsy. We are confident it＊s first and potentially best in class.§

Takeda is advancing a new class of therapies aimed at orexin receptor 2, a biological mechanism linked to narcolepsy, which affects about 3 million people worldwide. Existing treatments only partially address the disorder＊s symptom, leaving a significant gap in treatment.

A potential competitor, ALKS 2680 from Alkermes, delivered mixed results in midstage trials in July.

Takeda tested twice-daily doses of 1 milligram and 2 mg of its narcolepsy drug over the trial period, enrolling 273 patients across 19 countries. The company plans to file for regulatory approvals globally by March and is moving ※as quickly as possible,§ Plump said.

The treatment is expected to serve as the foundation of a broader franchise Takeda is developing to address sleep disorders and related conditions.

※In my long tenure in the industry, I＊ve never seen data as compelling as what we＊ve seen in this orexin trial,§ Plump said.

Oveporexton could generate peak sales of $3 billion for narcolepsy type 1, while Takeda＊s broader orexin agonist portfolio may generate twice or more across a wider range of indication, Stephen Barker, a healthcare analyst at Jefferies Japan, said in July.

Symptoms of narcolepsy include excessive daytime sleepiness, sudden ※sleep attacks§ called cataplexy, a loss of muscle control that can lead to a full-body collapse as well as sleep paralysis, hallucinations and disrupted nighttime rest. Type 1 refers to cases with cataplexy, while type 2 occurs without it, according to Narcolepsy Network.