GEXVal Selected for AMED’s ‘Strengthening Program for Pharmaceutical Startup Ecosystem’

株式会社GEXVal
2024年12月16日

We are delighted to announce that GEXVal Inc.'s planned global Phase 2a Proof of Concept (POC) clinical trial of GXV-001⁽¹⁾ for Fragile X Syndrome (FXS) ⁽²⁾ has been selected for the Japan Agency for Medical Research and Development (AMED⁽³⁾) "Strengthening Program for Pharmaceutical Startup Ecosystem⁽⁴⁾" for the fiscal year 2024.

The program supports drug development initiatives by pharmaceutical venture companies, supported by investment from AMED⁽³⁾-Registered Venture Capital firms.

Moving forward, GEXVal will be eligible to receive non-dilutive funding from AMED⁽³⁾ equivalent to twice the investment amount from Registered VCs, with a total project value of up to approximately 3 billion Japanese yen (JPY).

With this support, GEXVal will accelerate its global Proof of Concept (POC) trial to evaluate the efficacy of GXV-001⁽¹⁾ in patients with Fragile X Syndrome (FXS)⁽²⁾.

Juran Kato, PhD, President and CEO of GEXVal Inc. (Fujisawa, Kanagawa, Japan) says, whilst there are currently no established therapeutic drugs for FXS⁽²⁾, we are committed to advancing our development efforts to provide patients and their families with a new treatment option as soon as possible.

Through our innovative pharmacoinformatics technology, GEXVal is dedicated to drug discovery and clinical development, leveraging our unique technology to address unmet medical needs, with a focus on rare diseases, while ensuring that no patient is left without access to treatment opportunities.

１）About GXV-001: GXV-001 is currently under clinical development for Fragile X Syndrome (FXS). With support from FRAXA Research Foundation (FRAXA), a US non-profit organization, the compound has been evaluated through the FRAXA Drug Validation Initiative (FRAXA-DVI). Through our wholly-owned subsidiary, GEXVal AU Pty Ltd, we have completed Phase I clinical trials, confirming its safety, tolerability, and pharmacokinetic profile. Furthermore, we have verified the Proof of Mechanism (POM) using multiple biomarkers, demonstrating the drug exhibits its pharmacological effects on the intended target molecule and induces physiological responses. We are pursuing clinical development of GXV-001 as a novel therapeutic candidate for neurodevelopmental disorders classified as autism spectrum disorders including FXS, rare neurological diseases such as Rett syndrome (RTT), and cognitive disorders including delirium.
Reference (February 28, 2023 FRAXA news release): https://www.fraxa.org/coming-together-for-rare-disease-day-2023/

２）About Fragile X Syndrome: Fragile X Syndrome (FXS) is a developmental disorder classified as an Autism Spectrum Disorder (ASD). In Japan, it is designated as intractable disease No. 206 under Japan’s Intractable Disease Program. According to FRAXA Research Foundation, FXS is considered the most common inherited cause of autism and intellectual disabilities.
For more information:
・Japan Intractable Diseases Information Center: https://www.nanbyou.or.jp/entry/4615(information available in Japanese only)
・NORD: https://rarediseases.org/rare-diseases/fragile-x-syndrome/
・Orphanet: https://www.orpha.net/en/disease/detail/908
・FRAXA Research Foundation: https://www.fraxa.org

３）About AMED: The Japan Agency for Medical Research and Development (AMED) is an organization that supports research and development essential for medical advancement. AMED provides comprehensive support from basic research to practical application in the development grants including the Strengthening Program for Pharmaceutical Startup Ecosystem.
For more information:
https://www.amed.go.jp/en/program/list/19/02/005.html

４）About the Strengthening Program for Pharmaceutical Startup Ecosystem: For information about the selected projects in the 6^th round of the “Strengthening Program for Pharmaceutical Startup Ecosystem” for fiscal year 2024, please visit:
https://www.amed.go.jp/koubo/19/02/1902C_00056.html (information available in Japanese only)

有关本事宜请咨询:
Head of Corporate office
Atsushi Sugizaki
info@gexval.com