Latest news with #narcolepsy
Yahoo
a day ago
- Business
- Yahoo
Takeda's oral narcolepsy drug shines in two Phase III trials
Takeda's oral narcolepsy drug, oveporexton, has met the primary and secondary endpoints in two Phase III trials. The FirstLight (NCT06470828) and RadiantLight (NCT06505031) trials both met all their endpoints in patients with narcolepsy type 1 (NT1) after 12 weeks of treatment. The primary and secondary endpoints measured changes in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, overall quality of life and daily life functions. These were all statistically significant and clinically meaningful improvements, achieving near normal ranges across the broad range of symptoms investigated. The drug also remained generally well-tolerated with a safety profile consistent with other oveporexton studies, with the most common adverse events (AEs) being insomnia, urinary urgency and frequency. No serious treatment-related adverse events were reported. Based on the study results, Takeda plans to submit a new drug application (NDA) to the US Food and Drug Administration (FDA) and other global regulators during the 2025 fiscal year. Oveporexton (TAK-861) is an investigational oral orexin receptor 2 (OX2R)-selective agonist. It is designed to address the core cause of NT1 by compensating for orexin deficiency, aiming to improve wakefulness and restore normal sleep-wake patterns. Orexin is a natural brain chemical that plays a crucial role in maintaining wakefulness and regulating the sleep-wake cycle. Takeda president Christophe Weber said: '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.' Of the patients across the two Phase III studies, more than 95% enrolled in the ongoing long-term extension (LTE) study. In 2020, Jazz Pharmaceuticals' Xywav (calcium, magnesium, potassium, and sodium oxybates) was approved by the FDA and was the first approved new treatment option indicated for both cataplexy and excessive daytime sleepiness in people living with narcolepsy in more than 15 years. Other treatment options for NT1 include trying to manage a regular sleeping pattern, as well as therapies, including stimulants such as Provigil (modafinil), Nuvigil (armodafinil), and Ritalin (methylphenidate) to promote wakefulness. Patients may also receive solriamfetol or pitolisant. To help with cataplexy attacks, either sodium oxybate or some antidepressants such as SSRIs [selective serotonin reuptake inhibitors] and SNRIs [serotonin-norepinephrine reuptake inhibitors] can be used to manage symptoms. If approved, GlobalData predicts sales of oveporexton will reach $1bn in 2030. GlobalData is the parent company of Clinical Trials Arena. "Takeda's oral narcolepsy drug shines in two Phase III trials" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
National Post
2 days ago
- Business
- National Post
Takeda Announces Positive Results from Two Pivotal Phase 3 Studies of Oveporexton (TAK-861) in Narcolepsy Type 1
Article content – Both Phase 3 Studies Met all Primary and Secondary Endpoints Demonstrating Statistically Significant Improvements Across Symptoms at All Doses, Building Upon Phase 2b Results – Oveporexton was Generally Well-Tolerated in Phase 3 Safety Profile – Takeda is Rapidly Advancing Regulatory Submissions and Launch Preparedness with the Aim to Bring Oveporexton to People Living with Narcolepsy Type 1 as Quickly as Possible – These Results Mark a Major Advancement Toward Transforming the Standard of Care by Addressing the Underlying Cause of Narcolepsy Type 1 OSAKA, Japan & CAMBRIDGE, Mass. — Takeda ( TSE:4502/NYSE:TAK) today announced that all primary and secondary endpoints were met in two Phase 3 randomized, double-blind, placebo-controlled studies of oveporexton (TAK-861), a potential first-in-class investigational oral orexin receptor 2 (OX2R)-selective agonist, in narcolepsy type 1 (NT1). NT1 is caused by the loss of orexin-producing neurons in the brain. Orexin agonists are designed to address this underlying orexin deficiency. For the first time, this mechanism of action has been validated in Phase 3 studies demonstrating significant improvement across a broad range of symptoms. These results reinforce the potential of oveporexton to transform the standard of care. Article content '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,' said Christophe Weber, president and chief executive officer at Takeda. 'Our leadership in orexin biology and building a multi-asset orexin franchise with transformative potential will position Takeda for long-term future growth.' Article content The FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) studies were two large, global Phase 3 studies conducted in 19 countries. Both studies achieved statistically significant improvement compared to placebo with p-values of <0.001 for all primary and secondary endpoints across all doses at week 12. The primary and secondary endpoints measuring objective and patient reported improvements in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, overall quality of life and daily life functions demonstrate statistically significant and clinically meaningful improvements achieving near normal ranges across the broad range of symptoms investigated. Article content 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 percent of the participants who completed the studies enrolled in the ongoing long-term extension (LTE) study. Article content '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,' said Andy Plump, M.D., Ph.D., president of R&D at Takeda. '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.' Article content 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. Article content Results from the Phase 3 studies have no significant impact on the full year consolidated forecast for the fiscal year ending March 31, 2026. Article content About Narcolepsy Type 1 (NT1) and Orexin Science Article content NT1 is a chronic, rare neurological disease that results in a range of debilitating symptoms including excessive daytime sleepiness (EDS), cataplexy, disrupted nighttime sleep, sleep paralysis and hallucinations upon falling asleep or waking. Additionally, individuals living with NT1 often report cognitive symptoms, including difficulty thinking clearly, remembering, concentrating and paying attention. NT1 is caused by loss of the orexin-producing neurons in the brain, which regulate wakefulness and sleep, and is also believed to be essential to other functions such as attention through activation of orexin receptors. Currently, the standard of care is limited to symptomatic therapies that may only partially address some of the symptoms people face. Article content About Oveporexton (TAK-861) Article content Oveporexton (TAK-861) is an investigational orexin receptor 2 (OX2R)-selective agonist, which selectively stimulates the OX2R to restore signaling and address the underlying orexin deficiency that causes narcolepsy type 1 (NT1). By activating OX2Rs, oveporexton is designed to promote wakefulness and reduce abnormal rapid eye movement (REM)-sleep like phenomena, including cataplexy, to address the broad spectrum of daytime and nighttime symptoms. Article content FirstLight (TAK-861-3001; NCT06470828) and RadiantLight (TAK-861-3002; NCT06505031) are global, multicenter, placebo-controlled studies to evaluate the efficacy, safety and tolerability of oveporexton compared to placebo in patients with narcolepsy type 1 (NT1) over 12 weeks. The studies were conducted in 19 countries with enrollment completed within six months. The FirstLight study enrolled 168 participants randomized to one of three dosing arms (high dose, low dose and placebo). The RadiantLight study enrolled 105 participants randomized to two dosing arms (high dose and placebo). The primary endpoint in both studies was improvement in excessive daytime sleepiness (EDS) as measured by the Maintenance of Wakefulness Test (MWT), a standard measure of wakefulness. Key secondary endpoints included improvement in EDS as measured by the Epworth Sleepiness Scale (ESS) and in the Weekly Cataplexy Rate (WCR), a measure evaluating cataplexy. The studies also evaluated the effect of oveporexton on participants' ability to maintain attention, participants' overall quality of life, the spectrum of narcolepsy symptoms and daily life functions, as well as the safety and tolerability of oveporexton. Article content About Takeda's Orexin Agonists for Sleep-Wake Disorders Article content Takeda is leading the field of orexin science with a multi-asset franchise. Orexin is a key regulator of sleep and wake patterns and contributes to other essential functions including attention, mood, metabolism and respiration. Oveporexton (TAK-861) is the lead investigational orexin receptor 2 (OX2R) agonist asset in Takeda's orexin franchise and received Breakthrough Therapy designation for the treatment of excessive daytime sleepiness in narcolepsy type 1 (NT1) from the U.S. Food and Drug Administration and the Center for Drug Evaluation of China's National Medical Products Administration. The company is also investigating other orexin agonists in populations with orexin levels in the normal range, including TAK-360, an oral OX2R agonist initially being investigated for the treatment of narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and other potential indications where orexin signaling is implicated. Article content About Takeda Article content 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 Article content Important Notice Article content 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. Article content 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. Article content Forward-Looking Statements Article content 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', 'forecasts', 'outlook' 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 and with respect to international trade relations; competitive pressures and developments; changes to applicable laws and regulations, including tax, tariff and other trade-related rules; 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; the success of our environmental sustainability efforts, in enabling us to reduce our greenhouse gas emissions or meet our other environmental goals; the extent to which our efforts to increase efficiency, productivity or cost-savings, such as the integration of digital technologies, including artificial intelligence, in our business or other initiatives to restructure our operations will lead to the expected benefits; 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: or at 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. Article content Medical Information Article content Article content Article content Article content Article content Contacts
Yahoo
3 days ago
- Health
- Yahoo
World Champion Cheerleader with Narcolepsy Reveals How She 'Tricks' Her Brain Before Performing (Exclusive)
Rylie Shaw is a championship-winning athlete who started competing in gymnastics at 11, later transitioning to cheerleading The now 20-year-old was diagnosed with narcolepsy as a teen and uses her platform to inform people about her experience Despite her condition, she just won her second World Championship titleRylie Shaw is a two-time world champion cheerleader whose life took an unexpected turn when she was diagnosed with narcolepsy in high school. With no family history of the condition, navigating the challenges of sudden sleep attacks and chronic fatigue was a big learning curve. Despite the hurdles, the 20-year-old from Wildomar, Calif., continues to compete at the highest level, balancing the demands of elite athletics with the realities of her diagnosis. 'I remember constantly telling my mom, 'I don't feel good,'' Shaw tells PEOPLE exclusively. 'I would frequently doze off in my classes and had a few instances of falling asleep at the wheel, when I realized my tiredness was not normal.' In high school, she discussed her concerns with her doctor and was referred to a sleep specialist who enrolled her in an overnight study at a dedicated facility. After her sleep patterns were monitored and evaluated, the results ultimately led to a narcolepsy diagnosis. 'I was the first in my family to be diagnosed, so navigating that was difficult as I didn't have someone that knew what it was like,' Shaw admits. As a young woman with a limited 'battery,' spending time with friends and family was tiresome, and even more 'isolating.' 'When I'm really tired, it's overwhelming and often results in me shutting down and having a difficult time holding a conversation,' she explains. 'To others, this can appear rude, and they often don't understand.' Throughout college, Shaw felt limited compared to her peers, realizing she couldn't stay out or socialize as long as others her age. Working through it meant accepting her limits and learning to prioritize her health without guilt. Given that Shaw is also an athlete, managing her unpredictable exhaustion can be extremely difficult despite years of experience in gymnastics and cheerleading. 'Sometimes during practice, I would hit a wall of tiredness and my skills would suffer,' she says. 'With a high-energy sport like cheerleading, this can be really difficult when a sleep attack hits.' Shaw has been an All-Star cheerleader since age 15. In April 2025, the California Allstars Rangers won their third World Championship title, making Shaw a two-time, back-to-back champion. However, given the high demands of being a decorated cheerleader, Shaw is extremely cautious when it comes to cataplexy – a symptom of narcolepsy that can cause sudden loss of muscle control or even consciousness, often triggered by strong emotions. Since being diagnosed, she has taken nightly medication to help regulate her sleep cycles, which is essential for preventing cataplexy attacks. 'I learned this trick that if I convince myself I'm not anxious or excited and prevent those emotions from growing, I can prevent cataplexy from being triggered,' she explains. 'This typically works during competition, and I've never had a cataplexy attack on stage. However, the emotion of relief when it's all over is a big trigger I typically can't prevent.' The most difficult part for Shaw isn't even competing itself – adrenaline helps her power through events – but rather the toll it takes afterward. 'I typically spend the next few days crashed out and recovering by sleeping it off,' she reveals. 'Not to mention, I'm an emotional wreck. It's not fun, but definitely worth the thrills of competing with my team.' Due to her limited energy, Shaw makes sure to prioritize rest and recovery. As an athlete, she recognizes that training demands much of her energy, often requiring her to turn down other opportunities or commitments. Given the intense travel that comes with competing around the country, Shaw has had to coordinate with her team's schedule as best she can. 'I have to be very strategic with scheduling the best flights that disrupt my sleep schedule the least amount because missing sleep means I can't take my medication and makes me more prone to cataplexy,' Shaw says. Never miss a story — sign up for to stay up-to-date on the best of what PEOPLE has to offer, from celebrity news to compelling human interest stories. However, this season, several of Shaw's teammates were also dealing with their own medical challenges, creating a supportive environment of mutual understanding. 'Along with that, I had teammates see me struggle and in response gave me support and prayer, which meant so much to me,' she shares. Despite her condition, Shaw has come to embrace being different from others her age. She uses her social media platforms to shed light on how narcolepsy shapes her everyday life and career, offering insight, honesty, and inspiration. When it comes to being a young athlete managing a chronic illness, Shaw says she has no regrets. 'Even if you fail, the experience will be worth it, so go for it!' Read the original article on People
Telegraph
4 days ago
- Health
- Telegraph
Drivers with sleeping disorders can lease cars under benefits scheme
Drivers with sleeping disorders are able to lease cars under the Motability benefits scheme. More than 400 people who list obstructive sleep apnoea (OSA) as a primary medical condition accessed a vehicle through the taxpayer-subsidised Motability scheme in 2024, according to official government figures. People with OSA experience their breathing stopping and starting when they sleep, which can lead to waking several times during the night and loud snoring, according to the NHS. The health service says that daytime symptoms include tiredness, mood swings and headaches. It also says that the Driver and Vehicle Licensing Agency must be informed if someone receives a diagnosis of OSA, and that a sufferer must not drive until symptoms are under control. The leasing scheme allows successful claimants to exchange part of their personal independence payments (Pip) for a car, and in some instances requires a downpayment. Those judged to be on the highest rate of mobility Pip, designed to help disabled people cover the costs of getting around, are able to access the scheme. Carers and parents are also able to drive the vehicle on behalf of the disabled person. A total of 420 people whose 'primary condition' was OSA made claims on the Motability scheme last year, and a further 190 claimants suffered from narcolepsy. Narcolepsy is a chronic neurological disorder which leads to excessive daytime sleepiness as well as other symptoms including hallucinations and poor memory. There were a total of 589,550 people claiming on the Motability scheme in April of this year, and cost the Government £600 million last year. More than 320 people with dyslexia have accessed a car, as have 420 who are diagnosed with dyspraxia. Twenty drivers with food intolerances listed as their primary conditions also made claims, as well as 190 people with social phobia. The scheme has come under renewed scrutiny in recent weeks after the Government battled with its backbenchers to try and force through reforms to the welfare system. Rachel Reeves and Sir Keir Starmer were forced to back down on plans to slash the benefits bill, in part by raising the threshold for Pip eligibility. The proposed changes to assessments were watered down, and the changes will only affect new claimants, while those already on the benefit will continue to receive it. Kemi Badenoch, the Conservative Party leader, said in a speech on Thursday: 'We are going to have to draw a line in the sand about which conditions the state gives out support for.' Earlier this week, the Centre for Social Justice called on the Government to withdraw Pip and Universal Credit from those with milder anxiety, depression or ADHD (attention deficit hyperactivity disorder). They estimated that such changes would save £7.4 billion by 2029-30, and that £1 billion of it should be redirected towards reinvestment in front-line mental health services.
Health Line
4 days ago
- Health
- Health Line
Recognizing and Managing the Symptoms of Narcolepsy
Key takeaways Narcolepsy causes extreme daytime sleepiness, which can lead to dozing off at inappropriate times and difficulty concentrating. Type 1 narcolepsy involves cataplexy, or sudden muscle weakness, that can be triggered by strong emotions, while both types can cause sleep paralysis and hallucinations as you fall asleep or wake up. Lifestyle changes like short naps, regular sleep schedules, and avoiding caffeine, along with medications such as stimulants and SNRIs, can help manage narcolepsy symptoms. Narcolepsy is a rare neurological condition that causes extreme daytime drowsiness and overwhelming urges to sleep. Narcolepsy symptoms usually begin when people are in their teens. However, most diagnoses occur in people 20–40 years old. There are two types of narcolepsy. Type 1 features a sudden loss of muscle tone, known as cataplexy. Type 2 does not have cataplexy as a symptom. What causes narcolepsy is unknown, but people with type 1 usually have lower amounts of hypocretin, a brain protein that regulates sleep-wake cycles. This may be due to a gene mutation. People with a parent or sibling with narcolepsy are up to 40 times more likely to have it. Keep reading to learn more about narcolepsy symptoms and how to manage them. Excessive daytime sleepiness People with type 1 and type 2 narcolepsy experience excessive daytime sleepiness. You may doze off at inappropriate times during the day. Because of this, you may lack energy and have difficulty concentrating. Cataplexy People with type 1 narcolepsy experience cataplexy. This sudden muscle weakness usually occurs in your face, neck, and knees. Cataplexy can be mild, where your head drops, or it can be severe, causing you to collapse. It may look like you're having a seizure, but you're not. Strong emotions like fear or anger, or when you're laughing, may trigger cataplexy. The frequency of cataplexy varies by person. You may experience cataplexy several times a day or only once a year. Medications such as sodium oxybate (Xyrem) and venlafaxine (Effexor) can help prevent cataplexy. Sleep paralysis Narcolepsy may cause you to lose muscle function while falling asleep or waking up. Sleep paralysis is the inability to move or talk during these times. Episodes of sleep paralysis may last from a few seconds to a few minutes. People who don't have narcolepsy may also experience sleep paralysis. According to the American Academy of Sleep Medicine, 5–40% of people experience it. Hallucinations As you fall asleep or wake up, you may hallucinate, seeing vivid images that aren't real. They may seem so realistic that they may scare you, and you may jump out of bed. Most hallucinations are visual. They may also affect your other senses, such as touch, smell, and sound. These visions that occur when you fall asleep are called hypnagogic hallucinations. About one-third of all people have these hallucinations at some point. If you have narcolepsy, you may experience these hallucinations during the day. Other symptoms Some other symptoms associated with narcolepsy include: Fragmented sleep: You may have difficulty falling asleep or staying asleep at night. Automatic behaviors: If you fall asleep while eating, driving, or doing another activity, you may continue to do that activity without consciously knowing it. Immediate rapid eye movement (REM) sleep: People usually begin the REM stage of sleep, during which dreaming occurs, about 60–90 minutes after falling asleep. With narcolepsy, you may enter REM sleep in under 15 minutes after falling asleep and at any time during the day. How can I manage narcolepsy symptoms? Although there is no cure for narcolepsy, you can manage its symptoms with lifestyle strategies and medications. Some lifestyle strategies that may help include the following: Take a few 15- to 20-minute naps during the day if possible. Try to go to bed and wake up at the same times every day. Let your teachers or supervisors know you have narcolepsy and may fall asleep. Get regular physical activity during the week. Avoid heavy meals, caffeine, and alcohol before bedtime. Medications a doctor may prescribe to treat narcolepsy include: stimulants to help reduce excessive daytime sleepiness, such as: armodafinil (Nuvigil) modafinil (Provigil) methylphenidate (Ritalin) serotonin-norepinephrine reuptake inhibitors (SNRIs) to hold off REM sleep and prevent symptoms like cataplexy and hallucinations, such as venlafaxine (Effexor) pitolisant (Wakix), which reduces daytime sleepiness by releasing histamines in your brain When to contact a doctor If your daytime sleepiness affects your quality of life, or if you often fall asleep during the day, contact a doctor to determine the cause. They can make a diagnosis and prescribe treatments to help you feel better. Takeaway The symptoms of narcolepsy, especially excessive sleepiness, may make it difficult to do your daily activities. A treatment plan that includes lifestyle strategies and medications can help you manage this condition and improve your quality of life.
