- Aculys has completed the double-blind portion of a Phase 3 trial evaluating the use of pitolisant in Japanese OSAS patients experiencing excessive daytime sleepiness (EDS) currently on Continuous Positive Airway Pressure (CPAP) therapy.
- In the study, the primary efficacy endpoint was statistically superior to placebo (p=0.007). Further the study confirmed that the safety and tolerability of pitolisant were comparable to those observed in overseas clinical trials.
- Based on these results, Aculys intends to file a New Drug Application (NDA) for OSAS with PMDA.
Tokyo, Japan, January 9, 2025 – Aculys Pharma, Inc. (“Aculys”), a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological and psychiatric disorders, announced that its Phase III clinical trial of pitolisant (development code: BF2.649), a histamine H3 receptor antagonist/inverse agonist, has met its primary efficacy endpoint. The trial evaluated the effect of pitolisant in Japanese patients with OSAS who were experiencing residual EDS despite treatment with CPAP therapy.
At the end of the 12 week treatment period, patients receiving pitolisant scored lower on the Epworth Sleepiness Scale used to measure EDS compared to those in the placebo group and this difference was statistically significant (p=0.007). Additionally, safety and tolerability were consistent with results from overseas clinical studies, with no serious adverse events observed.
Hidemasa Tanigaki, Chief Executive Officer of Aculys, stated, “EDS associated with OSAS has been reported to lead to a decline in quality of life, reduced work productivity, and an increased risk of traffic accidents, significantly impacting the daily lives of OSAS patients. There have been no new drug options introduced for the treatment of residual sleepiness in OSAS patients in Japan for approximately 15 years, highlighting the need for new treatment options. We are committed to delivering this medication to patients in need as quickly as possible.”
Based on data obtained from this trial, Aculys plans to submit a NDA for pitolisant. The company aims to bring the medication to patients with OSAS near-term, contributing to a society where individuals can live with greater confidence and independence.
The clinical trial details can be viewed here: https://jrct.niph.go.jp/en-latest-detail/jRCT2031220501
About OSAS
OSAS is characterized by an interruption of proper breathing due to the narrowing or obstruction of the upper airway during sleep. Typical symptoms include loud snoring, daytime sleepiness, insomnia, sense of insufficient deep sleep, and systemic fatigue. If OSAS is not properly treated, in medium- to long-term, patients may develop complications such as diabetes, depression, hypertension, arteriosclerosis, and other cardiovascular conditions1,2.
CPAP is considered the standard treatment in OSAS and is used to mitigate the narrowing and obstruction of the upper airway and is known to contribute to improving various OSAS symptoms and patients’ quality of life. However, some patients reportedly experience persistent EDS despite appropriate basic treatment3. EDS is considered a risk factor for labor productivity, quality of life, and accidents such as traffic accidents4. Therefore new, effective treatment options that are well tolerated and have low potential for abuse are needed.
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 OSAS in 2021. In the United States, pitolisant 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 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 introduced from the US and European countries and provides innovations for better medical care to patients, their families, healthcare professionals, and society. Aculys Pharma is based in Tokyo, Japan, and was founded by Catalys Pacific.
Company name: Aculys Pharma, Inc.
Address: 2-14-4 KitaAoyama, Minato-ku, Tokyo, Japan
Representative: Hidemasa Tanigaki
Established: January 2021
URL: https://aculys.com
Information contained herein relating to pharmaceutical products and compounds under development is not intended as advertising or as medical advice but for disclosure of management information only.
References:
- The Japanese Circulation Society. JCS Joint Working Groups for Guidelines for Diagnosis and Treatment of Cardiovascular Diseases (2008-2009 joint research team report) Guidelines for Diagnosis and Treatment of Sleep Disordered Breathing in Cardiovascular Disease
- Clinical Practice Guidelines for Sleep Apnea Syndrome (SAS) 2020 (edited by committee preparing Clinical Practice Guidelines for Sleep Apnea Syndrome (SAS)
- Update on Persistent Excessive Daytime Sleepiness in OSA; Chest . 2020 Aug;158(2):776-786.
- The economic and societal burden of excessive daytime sleepiness in patients with obstructive sleep apnea. Sleep Med Rev. 2020 Jun;51:101275.