Oct 21, 2024
Aculys Pharma Delivers Positive Results in Domestic Phase III Clinical Trial of Pitolisant for Narcolepsy Patients
- Aculys completed the double-blind portion of a Phase 3 trial in Japan for the use of pitolisant in narcolepsy patients.
- In the study, the primary efficacy endpoint was achieved, and the study confirmed that the safety and tolerability of pitolisant were comparable to those observed in overseas clinical trials.
- Based on the study results, Aculys will file an NDA for narcolepsy with and without cataplexy with the PMDA.
Tokyo, Japan, October 21, 2024 - Aculys Pharma, Inc. (“Aculys”), a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological conditions, announced that pitolisant (compound development code: BF2.649), a histamine H3 receptor antagonist/inverse agonist, has achieved its primary endpoint in a Phase III clinical trial targeting Japanese patients with narcolepsy.
In this trial, pitolisant was administered orally to narcolepsy patients for eight weeks. The trial demonstrated the drug’s effectiveness regarding its primary endpoint, which was improvement in excessive daytime sleepiness compared to a placebo group using the Epworth Sleepiness Scale (ESS). Statistically significant difference in ESS was observed between the two groups. Furthermore, the secondary endpoint regarding the frequency of cataplexy attacks showed a suppression effect comparable to that observed in overseas trials. No serious adverse effects were noted, and the safety and tolerability results were consistent with those from clinical trials conducted abroad.
Hidemasa Tanigaki, Chief Executive Officer of Aculys, stated, "Patients with narcolepsy face serious challenges in their daily lives due to symptoms such as unexpectedly falling asleep at school or work and experiencing cataplexy attacks triggered by strong emotions. Moreover, there has been no new treatment option for narcolepsy in Japan for approximately 17 years. We are committed to delivering this drug as soon as possible so that narcolepsy patients can feel more secure and lead positive daily lives."
Based on the data obtained from this trial, Aculys aims to submit the New Drug Application for pitolisant to help create a world where narcolepsy patients and their families can live with greater peace of mind and authenticity.
View details of the clinical trial at: https://jrct.niph.go.jp/en-latest-detail/jRCT2031220432
About pitolisant
Pitolisant is an antagonist/inverse agonist that selectively binds to the histamine H3 receptor, an autoreceptor located in the presynaptic region of the histamine-containing neurons in the human brain that plays a critical role in regulating sleep and wake rhythm. The drug was discovered by Paris-based Bioprojet and was approved by the European Medicines Agency (EMA) for the treatment of narcolepsy with or without cataplexy in 2016 and for the treatment of excessive daytime sleepiness associated with obstructive sleep apnea syndrome (OSAS) in 2021. In the United States, the drug was approved by the Food and Drug Administration (FDA) for the treatment of excessive daytime sleepiness associated with narcolepsy in 2019 and cataplexy associated with narcolepsy in 2020 and is already used in the clinical setting. As of the end of 2023, pitolisant has obtained regulatory approval in 38 countries including US and EU for narcolepsy, and in 30 countries in EU for OSAS.
About narcolepsy
Patients with narcolepsy often start suffering from symptoms in childhood, adolescence, or young adulthood, but are not diagnosed until much later due to misdiagnosis and low awareness of the condition’s symptoms, posing a global challenge.1 The causes and pathophysiology of narcolepsy are largely unknown. In healthy people, neurotransmitters in the brain, such as orexin and histamine, help maintain the regular cycle of daytime wakefulness and nighttime sleep (REM and non-REM sleep), while in patients with narcolepsy, this sleep-wake cycle becomes irregular.2 Due to this irregular sleep-wake cycle, patients with narcolepsy suddenly, and against their will, fall asleep or have difficulty maintaining concentration while engaged in school or work. Some patients also have cataplexy attacks, which are a sudden loss of muscle tone of the whole body or certain voluntary muscles that are triggered by strong emotions, hypnagogic hallucinations (a hallucination that occurs right before falling asleep), and sleep paralysis (“old hag” syndrome). Different patients experience different types and severity of symptoms, but these symptoms significantly affect the patients’ lifestyles, including challenges in education or employment.3
About Aculys Pharma, Inc.
Aculys Pharma is a clinical stage biopharmaceutical company that is pioneering ways to eliminate drug lag/drug loss in Japan, and is working to resolve social issues related to neurological and psychiatry diseases. Its corporate name was created from the philosophy of serving as a "Catalyst to Access." Aiming to act as a bridge for innovative medical care in the field of neuropsychiatry, Aculys Pharma develops and commercializes novel pharmaceuticals and provides innovations for better medical care to patients, their families, healthcare professionals, and society.
Company name: Aculys Pharma, Inc.
Address: 2-14-4 KitaAoyama, Minato-ku, Tokyo, Japan
Representative: Hidemasa Tanigaki
Establishment date: January 2021
URL: https://www.aculys.com
Information concerning pharmaceutical products (compound under development) contained herein is not intended as advertising or as medical advice but for disclosure of management information.
References:
- Thorpy MJ, Krieger AC. Delayed diagnosis of narcolepsy: characterization and impact. 2014 May;15(5):502-7.
- Scammell TE. Narcolepsy. NEJM. 2015;373:2654-2662.
- Scammell TE, Jackson AC, Franks NP, Wisden W, Dauvilliers Y. Histamine: neural circuits and new medications. Sleep. 2019 Jan 1;42(1):zsy183.
