Takeda Plans Prompt Commencement of Global Phase 3 Trials for TAK-861, an Oral Orexin Agonist, in Narcolepsy Type 1 by First Half of Fiscal Year 2024

Takeda (TSE:4502/NYSE:TAK) has unveiled positive topline findings from a randomized, double-blind, placebo-controlled, multiple dose Phase 2b trial assessing TAK-861, an oral orexin receptor 2 (OX2R) agonist, in patients diagnosed with narcolepsy type 1.

Narcolepsy, a chronic and rare neurological disorder characterized by central hypersomnolence, presents a significant unmet medical need despite existing therapies. It is categorized into two types: narcolepsy type 1 (NT1), stemming from a notable loss of orexin neurons resulting in orexin deficiency, and narcolepsy type 2 (NT2), where orexin levels typically remain normal. Activation of the orexin receptor 2 in NT1 patients addresses the fundamental pathophysiology of the disorder, aiming to restore orexin signaling. Two distinct Phase 2b studies were conducted, one for NT1 (NCT05687903) and another for NT2 (NCT05687916).

The NT1 trial, TAK-861-2001, involving 112 patients, demonstrated statistically significant and clinically meaningful enhancements in both objective and subjective measures of wakefulness compared to placebo at week 8, notably on the primary endpoint Maintenance of Wakefulness Test (MWT) (p < 0.001). Improvements in key secondary endpoints, including the Epworth Sleepiness Scale (ESS) and Weekly Cataplexy Rate (WCR), were also statistically significant and clinically relevant, in line with the primary endpoint. A majority of trial completers entered a long-term extension study. Based on these outcomes, and after consultation with global health authorities, Takeda plans to promptly initiate global Phase 3 trials of TAK-861 in NT1 during the first half of its fiscal year 2024.

Presently, Takeda does not intend to progress TAK-861 in NT2. Data are being further evaluated to determine the subsequent actions in populations with normal orexin levels. Takeda is advancing multiple orexin agonists in patient groups where orexin biology is implicated, including those with normal orexin levels such as NT2 and other relevant indications.

TAK-861 demonstrated a generally favorable safety and tolerability profile across both trials. No treatment-related serious adverse events were reported. Moreover, neither hepatotoxicity nor visual disturbances were observed in the Phase 2b trials or in the ongoing long-term extension trial of TAK-861.

Sarah Sheikh M.Sc., B.M., B.Ch, MRCP, Head of Neuroscience Therapeutic Area Unit and Head of Global Development at Takeda, expressed enthusiasm, stating, “We are delighted to announce these compelling results from the TAK-861 trial in narcolepsy type 1, enabling us to swiftly commence Phase 3 trials this year as we strive to provide patients with a treatment that could address the underlying pathophysiology of the disease.” Sheikh extended gratitude to the patients, caregivers, and investigators involved in the orexin agonist trials, emphasizing Takeda’s commitment to leveraging its deep understanding of orexin biology to develop and deliver transformative therapies for various indications where this mechanism holds promise.

The results from both trials will be presented at an upcoming scientific congress.

It’s noted that the results from the Phase 2b trials have no bearing on the full-year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

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