OSAKA, Japan & CAMBRIDGE, Mass. - Monday, 03. June 2024 AETOSWire
–
Phase 2b Trial Demonstrated Statistically Significant and Clinically
Meaningful Improvements Across Primary and all Secondary Endpoints up to
8 Weeks
– TAK-861 is the First Oral Orexin Receptor 2 Agonist to Potentially Address the Underlying Pathophysiology of NT1
– Safety Results Indicated TAK-861 is Generally Safe and Well Tolerated
– Phase 3 Trials of TAK-861 to be Initiated in 1H FY2024
(BUSINESS
WIRE)--Takeda (TSE: 4502/NYSE:TAK) will present today positive results
from its Phase 2b trial of TAK-861 in narcolepsy type 1 (NT1) as
late-breaking data presentations at SLEEP 2024, the 38th annual meeting
of the American Academy of Sleep Medicine and the Sleep Research
Society. TAK-861 is an investigational oral orexin receptor 2 (OX2R)
agonist and, based on the results, has the potential to provide
transformative efficacy in addressing the overall disease burden in
people with NT1. The randomized, double-blind, placebo-controlled,
multiple dose trial, TAK-861-2001 (NCT05687903), in 112 patients with
NT1 demonstrated statistically significant and clinically meaningful
improvements across primary and secondary endpoints, with efficacy
sustained over 8 weeks of treatment.*
NT1 is a chronic, rare
neurological central disorder of hypersomnolence caused by a significant
loss of orexin neurons, resulting in low levels of orexin neuropeptides
in the brain and cerebrospinal fluid. No currently approved treatments
target the underlying pathophysiology of NT1. People with NT1 suffer
from excessive daytime sleepiness (EDS), cataplexy (sudden loss of
muscle tone), disrupted nighttime sleep, hypnagogic and hypnopompic
hallucinations and sleep paralysis. These debilitating symptoms lead to a
markedly reduced quality of life and can severely impact job
performance, academic achievement and personal relationships. TAK-861 is
designed to address the orexin deficiency in NT1 by selectively
stimulating the orexin receptor 2.
The presentation highlights results from the Phase 2b trial including:
The primary endpoint demonstrated statistically significant and
clinically meaningful increased sleep latency on the Maintenance of
Wakefulness Test (MWT) versus placebo across all doses (LS mean
difference versus placebo all p ≤0.001). Improvements were sustained
over 8 weeks.
Consistent results were achieved in the key
secondary endpoints including the Epworth Sleepiness Scale (ESS) and
Weekly Cataplexy Rate (WCR), demonstrating significantly improved
subjective measures of sleepiness and cataplexy (sudden loss of muscle
tone) frequency versus placebo that were also sustained over 8 weeks.
The majority of NT1 patients in the trial were found to be within
normative ranges for MWT and ESS by the end of the 8-week treatment
period as a result of these sustained improvements.
The
majority of the participants who completed the trial enrolled in the
long-term extension (LTE) study with some patients reaching one year of
treatment.
The trial also included additional exploratory
endpoints that showed meaningful improvements in narcolepsy symptoms and
functioning according to most participants. These data will also be
presented in poster presentations at SLEEP and at future scientific
congresses.
The dataset showed that TAK-861 was generally
safe and well tolerated during the study, with no treatment-related
serious treatment-emergent adverse events (TEAEs) or discontinuations
due to TEAEs.
No cases of hepatotoxicity or visual
disturbances were reported in the Phase 2b trial or in the ongoing LTE
study. The most common TEAEs were insomnia, urinary urgency and
frequency, and salivary hypersecretion. Most TEAEs were mild to moderate
in severity, and most started within 1-2 days of treatment and were
transient.
“In this trial, TAK-861's profile balanced efficacy
and safety with the potential to establish a new standard of care for
people with NT1,” said Sarah Sheikh, M.D., M.Sc., B.M., B.Ch., MRCP,
Head, Neuroscience Therapeutic Area Unit and Head, Global Development at
Takeda. “We are dedicated to investigating the full potential of orexin
biology and advancing TAK-861 to late-stage clinical trials, with the
ultimate goal of delivering a potential first-in-class treatment that
can make a meaningful difference for patients.”
Based on these
results, and in consultation with global health authorities, Takeda
plans to initiate global Phase 3 trials of TAK-861 in NT1 in the first
half of its fiscal year 2024. The Phase 2b data also supported the
recent Breakthrough Therapy designation for TAK-861 for the treatment of
EDS in NT1 from the U.S. Food and Drug Administration (FDA).
Breakthrough Therapy designation is a process designed to expedite the
development and review of a drug that is intended to treat a serious or
life-threatening condition, for which preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement over
available therapies on at least one clinically significant endpoint.
Takeda
will be hosting a call to discuss these data this evening, June 3, at
7:30 p.m. CT for investors and analysts. Presentation slides and a
virtual meeting link will be available here.
Additional
presentations on TAK-861 will be shared during the SLEEP 2024 poster
presentation session on Tuesday, June 4, from 10:00 to 11:45 a.m. CT,
assessing function and health-related quality of life in individuals
with NT1, as well as patient satisfaction with TAK-861 treatment.
There
is no change in Takeda’s full year consolidated forecast for the fiscal
year ending March 31, 2025 (FY2024), announced on May 9, 2024.
About Takeda’s Orexin Franchise
Takeda
is advancing the field of orexin therapeutics with a multi-asset
franchise offering tailored treatments to unlock the full potential of
orexin. Orexin is a key regulator of the sleep-wake cycle and is
involved in other essential functions, including respiration and
metabolism. TAK-861 is the leading program in this franchise. The
company is also progressing multiple orexin agonists in patient
populations with normal levels of orexin neuropeptides and other
indications where orexin biology is implicated. This includes TAK-360,
an oral OX2R agonist being investigated for narcolepsy type 2 and
idiopathic hypersomnia, which recently initiated a Phase 1 trial and
received Fast Track designation from the U.S. FDA, and danavorexton
(TAK-925), an intravenously administered OX2R agonist being investigated
in a Phase 2 trial in patients with moderate to severe obstructive
sleep apnea undergoing general anesthesia.
About Takeda
Takeda
is focused on creating better health for people and a brighter future
for the world. We aim to discover and deliver life-transforming
treatments in our core therapeutic and business areas, including
gastrointestinal and inflammation, rare diseases, plasma-derived
therapies, oncology, neuroscience and vaccines. Together with our
partners, we aim to improve the patient experience and advance a new
frontier of treatment options through our dynamic and diverse pipeline.
As a leading values-based, R&D-driven biopharmaceutical company
headquartered in Japan, we are guided by our commitment to patients, our
people and the planet. Our employees in approximately 80 countries and
regions are driven by our purpose and are grounded in the values that
have defined us for more than two centuries. For more information, visit
www.takeda.com.
* The topline results were announced on February
8, 2024, via a press release, “Takeda Intends to Rapidly Initiate the
First Global Phase 3 Trials of TAK-861, an Oral Orexin Agonist, in
Narcolepsy Type 1 in First Half of Fiscal Year 2024.”
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Contacts
Media Contacts:
Japanese Media
Yuko Yoneyama
Yuko.yoneyama@takeda.com
+81 70-2610-6609
U.S. and International Media
Rachel Wallace
Rachel.wallace2@takeda.com
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