Takeda Wins Breakthrough Award from Pharmaceutical Society of Japan for Discovery of Soticlestat (TAK-935), a Cholesterol 24-hydroxylase (CH24H) Inhibitor

- Award recognizes Takeda’s contribution to the advancement of drug discovery and unique approach to discovering soticlestat, an innovative therapy with a novel chemical structure developed at the company’s Shonan research center

Osaka, Japan, November 25, 2022 – Takeda today announced that its research for the discovery of soticlestat (TAK-935), a cholesterol 24-hydroxylase (CH24H) inhibitor, has won a Breakthrough Award from the Division of Medicinal Chemistry, Pharmaceutical Society of Japan (PSJ). The award ceremony was held online today.

_{From left to right (back): Masato Yoshikawa, Maki Miyamoto.
From left to right (front): Toshiya Nishi, Tatsuki Koike, Haruhi Ando.}

Soticlestat is a novel, first-in-class therapy designed and developed as a selective CH24H inhibitor with the potential to reduce seizure susceptibility and improve seizure control¹.   In February 2022, the Minister of Health, Labour and Welfare granted an orphan drug designation for soticlestat as a potential treatment for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), both of which are developmental epileptic encephalopathies (DEEs), designated as intractable diseases in Japan. The therapy is currently in phase 3 studies for patients with DS and LGS. The award recognizes Takeda’s research as contributing to the advancement of drug discovery research through the unique approaches taken to developing an innovative drug with a novel chemical structure. Takeda won this award for the first time in 9 years, marking its sixth win.

The Division of Medicinal Chemistry is the PSJ’s first working group, launched in 1990 to contribute to the further development of the PSJ and pharmaceutical industry. It seeks to build an environment where members who are conducting basic or applied research into the creation of innovative new drug entities can present their research findings, exchange knowledge, communicate with other members, and collaborate with other organizations in and outside Japan, as well as support the advancement of pharmaceutical chemistry and strengthen the basis of new drug discovery research. The Division of Medicinal Chemistry’s Breakthrough Awards were launched in 2000 to recognize excellent findings in the field of drug discovery research. 

Tatsuki Koike, Leader of the Soticlestat Research Project and Director of the Neuroscience Drug Discovery Unit at Takeda said, “Soticlestat is a new drug candidate designed and created at Takeda’s Shonan research center, which is currently undergoing global Phase 3 clinical trials. We are delighted to receive this award in recognition of our contribution to the advancement of drug development research through the pursuit of innovative science.”

Award Winners

Head researcher:     Tatsuki Koike, Director, Neuroscience Drug Discovery Unit, Takeda
Co-researchers:      Masato Yoshikawa, Associate Director, Neuroscience Drug Discovery Unit, Takeda
                             Maki Miyamoto, Associate Director, Neuroscience Drug Discovery Unit, Takeda
                             Toshiya Nishi, former Takeda member
                             Haruhi Ando, former Takeda member

About Soticlestat

Soticlestat is a highly selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), with the potential to reduce seizure susceptibility and improve seizure control¹. CH24H is predominantly expressed in the brain, where it converts cholesterol into 24S-hydroxycholesterol (24HC) to adjust the homeostatic balance of brain cholesterol. 24HC is a positive allosteric modulator of the NMDA receptor and modulates glutamatergic signaling associated with epilepsy. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates that 24HC is involved in the over-activation of the glutamatergic pathway through modulation of the NMDA channel and that increased expression of CH24H can disrupt the reuptake of glutamate by astrocytes, resulting in epileptogenesis and neurotoxicity². Inhibition of CH24H by soticlestat reduces the neuronal levels of 24HC and may improve the excitatory/inhibitory balance of NMDA channel activity.

About Takeda

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities and partnerships to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com/jp/.

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

Media Contacts:
Japanese Media
Yuko Yoneyama
[email protected]
+81 (0) 70-2610-6609

^{1　J. Med. Chem. 2021 Aug 26;64(16):12228-12244. Doi: 10.1021/acs.jmedchem.1c00864. Epub 2021 Aug 13.
2　J. Neurochem. 2021 May;157(4):899-918. Doi: 10.1111/jnc.15228. Epub 2020 Nov 18.}

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