Takeda intends to file a new drug application with the FDA in fiscal year 2025.
Summary: Takeda’s investigational orexin-2 receptor agonist oveporexton met all primary and secondary endpoints in two phase 3 trials for narcolepsy type 1, showing significant symptom improvements and supporting plans for global regulatory submissions in 2025.
Key takeaways:
- Takeda’s narcolepsy type 1 drug candidate met all endpoints in both phase 3 studies, demonstrating statistically significant improvements across symptoms at all doses.
- Oveporexton was generally well-tolerated in the phase 3 safety profile.
- Takeda is rapidly advancing regulatory submissions and launch preparedness.
All primary and secondary endpoints were met in two phase 3 randomized, double-blind, placebo-controlled studies of oveporexton (TAK-861), Takeda’s potential first-in-class investigational oral orexin receptor 2-selective agonist, in narcolepsy type 1 (NT1).
For the first time, a drug candidate that addresses the underlying orexin deficiency in narcolepsy type 1 human trials has been validated in phase 3 studies, which demonstrate significant improvement across a broad range of symptoms.
“We are thrilled to reach this pivotal milestone for the oveporexton program. Oveporexton is a testament to Takeda’s strength in discovering and developing a potential new class of medicines for difficult-to-treat diseases such as narcolepsy type 1,” says Christophe Weber, president and CEO at Takeda, in a release. “Our leadership in orexin biology and building a multi-asset orexin franchise with transformative potential will position Takeda for long-term future growth.”
Endpoints Analyzed for Oveporexton
The phase 3 FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) studies were conducted in 19 countries. Both studies achieved statistically significant improvement compared to placebo for all primary and secondary endpoints across all doses at week 12.
The endpoints measuring objective and patient-reported improvements demonstrate statistically significant and clinically meaningful improvements, achieving near normal ranges across the broad range of symptoms investigated, including improvements in:
- wakefulness,
- excessive daytime sleepiness,
- cataplexy,
- ability to maintain attention,
- overall quality of life, and
- daily life functions.
Safety Profile
Oveporexton was generally well-tolerated with a safety profile from the phase 3 studies overall consistent with oveporexton studies to date, including the phase 2b study.
No serious treatment-related adverse events were reported.
The most common adverse events were:
- insomnia,
- urinary urgency,
- and frequency.
More than 95% of the participants who completed the studies enrolled in the ongoing long-term extension study.
“We are grateful to the patients who took part in these clinical studies and to their families, the investigators, and clinical staff. The studies were accelerated at an unprecedented pace with the aim to bring this potential treatment to people living with narcolepsy type 1 as quickly as possible,” says Andy Plump, MD, PhD, president of R&D at Takeda, in a release. “The comprehensive assessments from our phase 3 studies build on the transformative results we saw with our phase 2b study with most participants reaching normative ranges and reporting clinically meaningful improvement across a broad range of symptoms at the end of the 12-week treatment period. The positive results also reinforce the continued momentum for our late-stage pipeline, which we believe will deliver value to the patients we serve around the world.”
Takeda intends to present the results at upcoming medical congresses and plans to submit a New Drug Application with the United States Food and Drug Administration and additional global regulatory authorities in fiscal year 2025.
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