TAK-861, the drug that wakes up patients with narcolepsy type 1

Existing treatments for type 1 narcolepsy are based on symptom relief, with moderate success. Now, an international study involving Spanish centers, published in The New England Journal of Medicine , presents a new drug, TAK-86, which has demonstrated its efficacy in a Phase 2 clinical trial.

Narcolepsy type 1 is a sleep disorder characterized by excessive daytime sleepiness, cataplexy, and other symptoms related to hypocretinergic signaling, which is deficient in these patients. The study, which included 112 participants from diverse regions of the world, demonstrated that TAK-861, TAK-861 (Oveporexton), a selective orexin receptor 2 agonist, significantly improves the ability to stay awake, reduces daytime sleepiness, and decreases the frequency of cataplexy episodes.

According to data from the Spanish Society of Neurology, narcolepsy type 1 is a rare disease that affects between 20 and 60 people per 100,000 inhabitants. It is estimated that approximately 25,000 people in Spain suffer from it.

"The results of this trial are promising and suggest that TAK-861 could become an effective therapeutic option for patients with narcolepsy type 1. The improvement in the ability to stay awake and the reduction in daytime sleepiness, as well as the control of cataplexy, have a very significant impact on the quality of life of these patients," explains Rafael del Río Villegas, from the CEU San Pablo University and Vithas Foundation , and one of the authors of the study.

The randomized, placebo-controlled Phase 2 clinical trial showed that participants receiving TAK-861 experienced significant improvements in sleep latency on the Maintenance of Wakefulness Test (MWT) and the Epworth Sleepiness Scale (ESS). Additionally, a significant reduction in the weekly frequency of cataplexy episodes was observed.

The study also evaluated the safety and tolerability of TAK-861. The most common adverse events were insomnia, urinary urgency, and frequency , most of which resolved within a week. No cases of hepatotoxicity were reported, highlighting the drug's safety compared to other agents used previously.

For del Río Villegas, the results are very promising : "We are excited by the findings of this study and hope that TAK-861 (Oveporexton) may offer new hope for patients suffering from narcolepsy type 1. We will continue with further research to confirm these results and explore the potential of this treatment in phase 3 studies," he notes.

For Carles Gaig, a neurologist at the Hospital Clínic in Barcelona , ​​the clinical trial is "excellent news" and a potential turning point in treatment: "It seems like it will be a turning point."

Speaking to SMC, Gaig comments that the study is "very well designed and published in a leading journal," and that, unlike a previous trial halted due to liver toxicity, this new drug "does not appear to be hepatotoxic." However, he clarifies: "This is an initial Phase 2 study with only 90 patients included."