Kissei Initiates Additional Phase III Clinical Trial for Rovatirelin (KPS-0373) for the Treatment of Spinocerebellar Degeneration

Kissei Pharmaceutical Co., Ltd. (Head Office: Matsumoto, Nagano; Chairman and CEO: Mutsuo Kanzawa; "Kissei") announced that Kissei initiates additional Phase III clinical trial for Rovatirelin (KPS-0373) for the treatment of spinocerebellar degeneration.

Regarding Rovatirelin, Kissei has conducted two Phase III clinical trials and a combined analysis conducted to date, we determined that Rovatirelin was the first drug which has been verified to be effective to improve ataxia in patients with spinocerebellar degeneration assessed with international SARA score. Kissei submitted an application for marketing approval on December 22, 2021^１. However, the Pharmaceuticals and Medical Devices Agency (PMDA) indicated that approval based on the current clinical trial data would be difficult, leading Kissei to withdraw the approval application on July 19, 2023^２. Kissei then assessed the feasibility of conducting additional clinical trials and, upon the acceptance of their clinical trial plan in discussions with the PMDA, decided to commence an additional Phase III clinical trial.

In the upcoming clinical trial, the efficacy of this drug in improving ataxia in patients with spinal cerebellar degeneration will be evaluated through a double-blind method to verify its superiority over a placebo when administered orally.

Kissei is committed to focusing on the development of this drug to provide a new treatment option for spinal cerebellar degeneration.

¹ News Release: December 22, 2021
Submission of New Drug Application for Rovatirelin (KPS-0373) for the treatment of Spinocerebellar Degeneration

² News Release: July 19, 2023
Kissei Announces Withdrawal of Application for Marketing Approval for Rovatirelin (KPS-0373) for the Treatment of Spinocerebellar Degeneration in Japan


《 Reference 》

About Rovatirelin (development number: KPS-0373)
Rovatirelin is a derivative of thyrotropin-releasing hormone (TRH) discovered by Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi) It activates the nervous system by promoting the release of acetylcholine and monoamine neurotransmitters such as dopamine after binding to TRH receptors distributed in the central nervous system, and is expected to improve the ataxias in patients with spinocerebellar degeneration.

About spinocerebellar degeneration
Spinocerebellar degeneration is a nerve degeneration disorder of unknown etiology in which symptoms such as ataxia appear owing to the degeneration of the cerebellum or spinal cord. It is designated as an intractable disease by the Ministry of Health, Labour and Welfare, and there are approximately 37,000 recognized patients in Japan^※. Although the pathogenesis has been investigated by recent basic research, many aspects remain unclear and there is no definitive treatment. Therefore, symptomatic treatment is currently performed. The "Survey of Medical Needs on Neurologic Diseases" conducted in cooperation with the Japan Health Sciences Foundation and the Japan Society of Neurological Therapeutics reported that spinocerebellar degeneration is a "disease for which development of new therapies is urgent" for the following reasons: low treatment satisfaction, low availability of drugs, and the absence of effective treatment.

※ As of the end of fiscal year 2023, the number of holders of the Medical Expense Recipient Certificate for Designated Intractable Diseases, according to the Rare Disease Information Center.

About SARA (Scale for the Assessment and Rating of Ataxia)
It is a semi-quantitative method for the assessment of ataxia in spinocerebellar degeneration and other similar conditions; it consists of eight categories (gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide) and is used internationally. The scores from the eight categories are summed, with a maximum score of 40; 0 indicates no symptoms and 40 indicates the most severe symptoms.