Takeda Advances Narcolepsy Drug to Phase III, Halts Another

Takeda, a leading pharmaceutical company, has declared its intention to initiate global Phase III trials for its oral ORX2 agonist, TAK-861, which is being developed to treat narcolepsy type 1 (NT1). The trials are scheduled to commence in the first half of the fiscal year 2024. This decision follows the release of encouraging results from a Phase IIb trial, where TAK-861 demonstrated significant improvements in both objective and subjective measures of wakefulness when compared to a placebo.

The Phase IIb study highlighted that after a period of eight weeks, patients who received TAK-861 exhibited markedly enhanced results in the Maintenance of Wakefulness Test, which served as the primary endpoint of the trial. The outcomes were statistically significant with a p-value far below 0.001. Additionally, TAK-861 proved superior to the placebo in secondary endpoints such as the Epworth Sleepiness Scale and the Weekly Cataplexy Rate.

In terms of safety, the trial reported no serious adverse events related to the treatment. Notably, there were no cases of hepatotoxicity or visual disturbances, both in the mid-stage trial and in the ongoing long-term extension trial. The majority of participants chose to continue into the long-term extension phase of the study.

Sarah Sheikh, the head of global development at Takeda, expressed excitement over the results and stated that they will facilitate the swift initiation of Phase III trials. However, Takeda has decided not to proceed with the development of TAK-861 for narcolepsy type 2, although the specific reasons for this decision have not been disclosed.

The company is committed to further analyzing the data from both trials to chart the course for its narcolepsy programs, especially focusing on populations with normal orexin levels. Takeda also intends to present the findings at an upcoming scientific conference.

The advancement of TAK-861 is significant for Takeda as it represents a potential rebound from a previously halted narcolepsy program. In October 2021, Takeda had to discontinue its Phase II oral narcolepsy drug, TAK-994, due to safety concerns. The specific issues were not disclosed, but the drug was subsequently removed from the company's pipeline. Despite this setback, Takeda had backup programs in place, and the team from TAK-994 transitioned to work on TAK-861.

The development of TAK-861 is a testament to Takeda's resilience and commitment to finding effective treatments for narcolepsy. With the positive results from the Phase IIb trial and the upcoming Phase III trials, Takeda is poised to contribute significantly to the field of narcolepsy treatment.