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Takeda’s TAK-861 Phase 2b Late-Breaking Data Presentations at SLEEP 2024 Demonstrate Clinically Meaningful Impact of Oral Orexin Agonist in Narcolepsy Type 1 Compared to Placebo

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Takeda presented positive Phase 2b trial results for TAK-861, an oral orexin receptor 2 agonist, in treating narcolepsy type 1 (NT1) at SLEEP 2024. The trial showed statistically significant and clinically meaningful improvements across primary and secondary endpoints over 8 weeks, including increased sleep latency and reduced cataplexy. TAK-861 was generally safe and well tolerated, with no serious adverse events. The Phase 3 trials are set to commence in the first half of FY 2024. The FDA has granted Breakthrough Therapy designation to TAK-861 for excessive daytime sleepiness in NT1.

Positive
  • Statistically significant and clinically meaningful improvements in sleep latency and cataplexy.
  • Sustained efficacy over an 8-week period.
  • TAK-861 was generally safe and well tolerated.
  • No serious treatment-emergent adverse events reported.
  • Majority of patients reached normative ranges for MWT and ESS by 8 weeks.
  • Participants continued with long-term extension study, some reaching one year of treatment.
  • FDA granted Breakthrough Therapy designation for EDS in NT1.
Negative
  • Potential side effects included insomnia, urinary urgency, and salivary hypersecretion.
  • Most common TEAEs were mild to moderate but could affect patient comfort.
  • No change in Takeda’s financial forecast for FY2024, indicating immediate financial impact.

Insights

The breakthrough data from Takeda's Phase 2b trial of TAK-861 represents a significant advancement in the treatment of Narcolepsy Type 1 (NT1). The trial demonstrated statistically significant and clinically meaningful improvements across both primary and secondary endpoints. Importantly, these improvements were sustained over the 8-week period of the trial. Given that existing NT1 therapies primarily manage symptoms without addressing the underlying cause, TAK-861's mechanism of action as an orexin receptor 2 agonist is promising. By selectively stimulating the orexin receptor 2, TAK-861 aims to compensate for the orexin deficiency that is central to NT1. This could lead to a paradigm shift in treating NT1, offering patients not only symptom relief but potentially improving their overall neurological health.

Additionally, the safety profile of TAK-861 is reassuring, with no serious treatment-emergent adverse events reported. The mild to moderate TEAEs, such as insomnia and urinary urgency, were transient, which bodes well for long-term patient adherence and acceptance. The fact that many patients opted to continue into the long-term extension study further supports the tolerability and perceived benefit of the treatment. The upcoming Phase 3 trials are important to solidify these findings and move towards potential regulatory approval.

A Breakthrough Therapy designation by the FDA further underscores the potential of TAK-861, as this designation is intended to expedite the development of drugs that demonstrate substantial improvement over existing therapies for serious conditions. This designation not only accelerates the drug's development but also reflects strong preliminary evidence supporting its efficacy.

From a financial perspective, the positive results from the Phase 2b trial of TAK-861 significantly bolster Takeda's pipeline and its potential market value. Narcolepsy Type 1 is a niche but critical market and the absence of treatments targeting the underlying pathophysiology presents an opportunity for TAK-861 to potentially capture substantial market share. The sustained improvements in both primary and secondary endpoints, coupled with a favorable safety profile, enhance TAK-861’s commercial viability.

The FDA's Breakthrough Therapy designation is particularly noteworthy as it implies an expedited regulatory review process, reducing the time to market for TAK-861. This can translate into earlier revenue generation, which is beneficial for Takeda's financial health. Furthermore, the decision to initiate global Phase 3 trials in the first half of FY2024 aligns with strategic timelines to potentially introduce TAK-861 to the market within a few years.

Investors should also note that there is no change in Takeda’s full-year consolidated forecast for FY2024. This indicates that while the Phase 2b results are promising, they are already factored into the company’s financial outlook. The successful completion and subsequent approval of Phase 3 trials would be the next catalyst for more substantial financial impact.

The NT1 treatment landscape is currently underserved, with existing therapies focusing primarily on symptom management rather than addressing the root cause. TAK-861’s novel mechanism of action as an orexin receptor 2 agonist addresses this unmet need, potentially offering a more effective treatment option. The Phase 2b trial data showing statistically significant and clinically meaningful improvements in sleep latency and cataplexy frequency are promising indicators of TAK-861’s market potential.

Given the debilitating nature of NT1, the improvement in patients' quality of life over the 8-week treatment period is a strong selling point. The long-term extension study will provide further insights into the sustainability of these benefits, which is important for market acceptance. The favorable safety profile, coupled with the lack of serious adverse events, enhances TAK-861’s appeal to both patients and healthcare providers.

The upcoming Phase 3 trials will be critical in confirming these findings and could pave the way for eventual market entry. The potential of TAK-861 to establish a new standard of care in NT1 could position Takeda as a leader in this niche market, potentially driving significant revenue growth in the long term.

– 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

OSAKA, Japan & CAMBRIDGE, Mass.--(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.”

Important Notice

For the purposes of this notice, “press release” means this document, any oral presentation, any question-and-answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws. The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could”, “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/sec-filings-and-security-reports/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.

Medical Information

This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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

Source: Takeda Pharmaceutical Company Limited

FAQ

What were the results of Takeda's Phase 2b trial for TAK-861 in NT1?

The trial showed statistically significant and clinically meaningful improvements in sleep latency and cataplexy, sustained over 8 weeks.

Is TAK-861 safe to use for NT1?

Yes, TAK-861 was generally safe and well tolerated, with no serious treatment-emergent adverse events reported.

What are the potential side effects of TAK-861?

The most common side effects were insomnia, urinary urgency, and salivary hypersecretion, mostly mild to moderate in severity.

When will Takeda start Phase 3 trials for TAK-861?

Takeda plans to initiate Phase 3 trials for TAK-861 in NT1 in the first half of FY 2024.

Has TAK-861 received any special designations from the FDA?

Yes, the FDA has granted Breakthrough Therapy designation to TAK-861 for treating excessive daytime sleepiness in NT1.

Will the Phase 2b results of TAK-861 impact Takeda’s financial forecast?

There is no change in Takeda’s full year consolidated forecast for FY2024.

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