Takeda to Launch Phase 3 Narcolepsy Type 1 Trials for TAK-861 in H1 FY2024

Takeda Pharmaceutical Company has reported positive results from a Phase 2b clinical trial of TAK-861, a drug designed to treat narcolepsy type 1 (NT1). The trial, which was a randomized, double-blind, placebo-controlled study involving 112 patients, showed significant improvements in measures of wakefulness in patients who received TAK-861 compared to those on a placebo. The drug, an oral orexin receptor 2 (OX2R) agonist, targets the underlying cause of NT1, which is characterized by a loss of orexin neurons leading to a deficiency in orexin, a neurotransmitter that promotes wakefulness.

The primary endpoint of the study, the Maintenance of Wakefulness Test (MWT), demonstrated a highly significant result (p < 0.001) in favor of TAK-861. Additionally, key secondary endpoints, including the Epworth Sleepiness Scale (ESS) and Weekly Cataplexy Rate (WCR), also showed significant improvements. The drug was found to be safe and well-tolerated, with no serious adverse events related to the treatment reported.

Following the successful trial, Takeda plans to commence global Phase 3 trials for TAK-861 in the first half of the fiscal year 2024, in consultation with global health authorities. However, the company does not intend to proceed with the drug's development for narcolepsy type 2 (NT2), where orexin levels are normal. Instead, Takeda will focus on analyzing the data further to determine future steps for orexin agonists in NT2 and other conditions where orexin biology plays a role.

Takeda's commitment to advancing treatments for NT1 was underscored by Sarah Sheikh, Head of the Neuroscience Therapeutic Area Unit and Global Development at Takeda, who expressed enthusiasm for the potential of TAK-861 to address the root cause of the disease. Sheikh also acknowledged the contributions of patients, caregivers, and investigators involved in the trials and emphasized Takeda's ongoing efforts to deepen its understanding of orexin biology to develop transformative treatments.

The results of the Phase 2b trials are not expected to impact Takeda's full-year consolidated forecast for the fiscal year ending March 31, 2024. Takeda, a leading biopharmaceutical company headquartered in Japan, is dedicated to developing life-transforming treatments across various therapeutic areas, including rare diseases like NT1. The company's global presence and commitment to innovation are reflected in its diverse pipeline and its employees' dedication to improving patient experiences worldwide.

The upcoming presentation of the trial results at a scientific congress will provide further insights into the drug's efficacy and safety profile. Takeda's announcement highlights the importance of continued research and development in the field of neuroscience, particularly for conditions with significant unmet medical needs like NT1.