Nov 10, 2022

Aculys Pharma initiates a phase 3 clinical study of a diazepam nasal spray: an antiepileptic drug for the treatment of epileptic seizures

Aculys Pharma, Inc. (“Aculys Pharma” Headquarters: Minato-ku, Tokyo; Representative Director and CEO: Kazunari Tsunaba), a company focused on the development and commercialization of new innovative drugs in the fields of neurology and psychiatry, announced that it has initiated a 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.

This phase 3 study, conducted in Japan, is a multi-center, open-label study in children with status epilepticus and those at risk for status epilepticus to assess the efficacy and safety of NRL-1. Please visit the following site for detailed information on the study.


NRL-1 was developed by the U.S. pharmaceutical company Neurelis, Inc. Aculys Pharma has obtained an exclusive license to develop and commercialize it in Japan and the Asia-Pacific region (excluding Greater China and Singapore) and is currently developing it in Japan. In 2020, Neurelis obtained approval from the U.S. Food and Drug Administration for its diazepam nasal spray (trade name in the U.S.: VALTOCO®) as an acute treatment for intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters or acute repetitive seizures) that are distinct from an individuals’ usual seizure pattern in adult and pediatric patients 6 years of age and older.

The cause, symptoms, and severity of epileptic seizures vary widely among people with epilepsy, and epileptic seizures themselves exhibit diverse characteristics in each person. Among the diverse seizure types, some people experience seizures that recur many times a day or that do not terminate after a certain period of time (status epilepticus). These patients are at increased risk of brain damage and reduced life expectancy.1 When repetitive seizures occur, people with epilepsy must seek emergency treatment by healthcare professionals. On average, it takes 40 minutes from the time an emergency call is placed until a person is transferred to a hospital for treatment.1 An extensive, online, U.S. survey of patients, their families, and physicians has reported that recurrent epileptic seizures cause significant emotional, social, and financial burdens on patients and their families.2

To improve quality of life for people with recurrent epileptic seizures and their families/care partners, Aculys Pharma aims to bring this product to Japan as soon as possible.

Aculys Pharma also is collaborating with external partners to conduct surveys in Japan to identify challenges for people with epilepsy. The data will help guide development of a medical care plan for people with acute seizures, enabling more rapid responses to these episodes. As part of its mission, Aculys will continue to contribute to Japanese society by identifying healthcare issues from a social point of view and finding solutions through new technologies and developing external partnerships.

(*In the Asia Pacific region, Aculys has exclusive rights to Australia, Brunei, Cambodia, Indonesia, South Korea, Laos, Malaysia, Myanmar, New Zealand, the Philippines, Thailand, and Vietnam)

About Diazepam nasal spray

Diazepam has been used in Japan for about 60 years as a therapeutic agent, for the treatment of epileptic seizures, in various forms, including injections. It also has a history of use as a suppository by non-medical personnel, such as patients and caregivers, outside of medical institutions.

About epilepsy

Epilepsy is a chronic brain disorder that causes loss of consciousness and/or convulsions (epileptic seizures) as the result of excessive electrical excitation of nerve cells (neurons) in the brain. An estimated 600,000 to 1,000,000, or 5 to 8 in 1,000 people in Japan have epilepsy. As treatment has advanced, many people with epilepsy are able to control their seizures with appropriate diagnoses and antiepileptic medications; however, about 30% of people continue to experience frequent, repeated seizures f that require immediate treatment.3,4

About Neurelis

Neurelis, Inc. is a commercial-stage neuroscience company focused on the development and commercialization of therapeutics for the treatment of epilepsy and orphan neurologic disorders characterized by high unmet medical need. In 2020, the FDA approved Neurelis’ VALTOCO® (diazepam nasal spray) as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from an individual’s usual seizure pattern in adult and pediatric patients six years of age and older. VALTOCO is a proprietary formulation of diazepam incorporating the science of INTRAVAIL®. INTRAVAIL’s transmucosal absorption enhancement technology enables the noninvasive delivery of a broad range of protein, peptide and small-molecule drugs. In its approval of VALTOCO, the U.S. Food and Drug Administration also granted Neurelis Orphan Drug Exclusivity and recognized VALTOCO’s intranasal route of administration as a clinically superior contribution to patient care over the previously approved standard-of-care treatment (a rectal gel formulation of diazepam). For more information on VALTOCO, please visit


1 The Japanese Society of Child Neurology. The guidelines for the treatment of status epilepticus in children 2017. [In Japanese]

2 Penovich PE, Buelow J, Steinberg, et al. Burden of seizure clusters on patients with epilepsy and caregivers survey of patient, caregiver, and clinician perspectives. The Neurologist. 2017;22:207–214.

3 Epidemiological survey on the prevalence, diagnosis, and treatment of epilepsy and issues to be solved to establish a healthcare system for the disease, MHLW grant survey 201317043B, 2013. [Japanese Only]

4 Kwan P, MJ Brodie. Early identification of refractory epilepsy. N Engl J Med. 2000 Feb 3;342(5):314–9.

About Aculys Pharma, Inc.

Aculys Pharma is a biopharmaceutical company focused on the development and commercialization of innovations in the fields of neurology and psychiatry. 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: Kazunari Tsunaba
Established: Jan 2021

Information concerning pharmaceutical product (compound under development) contained herein is not intended as advertising or as medical advice, but intended for disclosure of management information.