Aculys Pharma, Inc., a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological conditions, today announced that it has achieved its primary efficacy endpoint in a pre-specified interim analysis of its Phase 3 clinical study of a diazepam nasal spray (compound development code: NRL-1), an antiepileptic drug for the treatment of status epilepticus or epileptic seizures that may lead to status epilepticus targeting Japanese patients aged 6 to 17 years.
The study was designed to evaluate the clinical efficacy of diazepam nasal spray based on comprehensive evidence from efficacy, pharmacokinetic, safety, and tolerability data using multiple clinically relevant endpoints. Diazepam nasal spray is Japan’s first nasally administered antiepileptic that has demonstrated efficacy and safety in Phase 3 clinical trials in Japan for the treatment of status epilepticus or epileptic seizures that may lead to status epilepticus.
The study demonstrated the drug’s effectiveness regarding its primary endpoint, which was the proportion of patients who achieved resolution of clinically relevant seizures within 10 minutes after administration of a single dose and who remained free from seizures or convulsions for 30 minutes after a single dose. Additionally, no adverse events leading to medication discontinuation or serious adverse events causally related to the study drug were observed, and no adverse events related to respiratory depression were observed.
Based on the data obtained from the interim analysis of the Phase 3 clinical trial, Aculys Pharma intends to submit a New Drug Application for diazepam nasal spray as the first intranasally administered antiepileptic drug in Japan to meet the urgent treatment needs of patients with recurrent epileptic seizures. Aculys Pharma also plans to present the results of this interim analysis at an upcoming Japanese medical conference.
“Many epilepsy patients are able to lead normal social lives with appropriate treatment, but those who have difficulty controlling their seizures with medication are living with the anxiety of seizures that could occur anywhere and at any time.” said Kazunari Tsunaba, President and Representative Director of Aculys Pharma. “We believe that access to emergency treatment that is convenient and easy for non-medical personnel to use appropriately in the event of a seizure, in addition to being effective and safe, will help reduce the emotional burden on patients and their caregivers, as well as the risk of sequelae due to prolonged seizures. Intranasal anticonvulsants, which are easy for families and caregivers to administer to patients with epileptic seizures, have been approved in the U.S. since 2020 and are widely used there. In our efforts to eliminate the problems of drug lag and drug loss, we will continue to work with medical professionals and regulatory authorities to promote our business in order to bring innovative treatments and hope to patients with recurrent seizures and their caregivers as soon as possible.”
In addition to developing innovative drugs, Aculys Pharma is also working with external partners to identify issues and challenges for people with epilepsy in Japan. The data obtained through such surveys will help to improve the healthcare delivery system for epilepsy seizures in the future, as well as promoting the creation of an ecosystem to address epileptic seizures in the community as a whole. Aculys Pharma will continue to contribute to Japanese society by identifying medical care issues from a social perspective, utilizing the latest digital technologies to solve these issues, and proactively utilizing external partnerships to provide new means of medical care.
About diazepam and diazepam nasal spray
Diazepam has been used in Japanese medical practice for over 60 years as a treatment for epileptic seizures in the form of injections and other forms. It is also administered as a suppository by non-medical personnel, such as patients and caregivers, outside medical institutions.
Diazepam nasal spray was developed by the U.S. pharmaceutical company Neurelis, Inc. Aculys Pharma holds an exclusive license to develop and commercialize this treatment in Japan and the Asia-Pacific region*(excluding Greater China and Singapore). In 2020, the FDA approved Neurelis’ VALTOCO® (diazepam nasal spray) as acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 6 years of age and older. In June 2023, China’s National Medical Products Administration (NMPA) approved diazepam nasal spray as effective for the same indication.
(*Asia-Pacific region: Australia, Brunei, Cambodia, Indonesia, South Korea, Laos, Malaysia, Myanmar, New Zealand, Philippines, Thailand, Vietnam)