GEXVal Receives EMA’s CHMP Scientific Advice for Lead Pipeline GXV-001

株式會社GEXVal
May 21, 2026

GEXVal Receives Scientific Advice from EMA’s CHMP for
Lead Pipeline GXV-001 in Fragile X Syndrome

GEXVal Inc. (President & CEO: Juran Kato, PhD; Fujisawa, Kanagawa, Japan; hereinafter “GEXVal”) is pleased to announce that it has received Scientific Advice (SA) from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for its lead pipeline GXV-001.

About GXV-001’s Target Indication
Fragile X Syndrome (FXS) is a rare, congenital disorder caused by mutations in the FMR1 gene on the X chromosome, resulting in loss of FMRP protein function. In Japan, FXS is designated as Specified Intractable Disease No. 206¹⁾ and is classified as a developmental disorder. According to the FRAXA Research Foundation, FXS is the most common known genetic cause of autism and intellectual disability, occurring in approximately 1 in 2,400 to 6,000 births. While diagnosis is more common in Europe and North America, FXS remains largely underdiagnosed in Japan despite similar prevalence rates. Currently, there are no approved treatments for FXS, representing a significant unmet medical need for patients and their families.

Characteristics: FXS is classified within autism spectrum disorders (ASD) and can present with a wide range of symptoms, including attention-deficit/hyperactivity, anxiety, sensory sensitivities, epilepsy, sleep disturbances, and intellectual disability.

About GXV-001
In the FRAXA Drug Validation Initiative (FRAXA-DVI) program, pharmacological activity across multiple behavioral parameters was observed for GXV-001 in FXS animal models.* Based on these and other findings, GXV-001 has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In addition, Phase I trials conducted through GEXVal's Australian subsidiary in healthy adult and elderly participants confirmed safety, tolerability, pharmacokinetics, and target engagement. GEXVal is now preparing to initiate a Phase IIa clinical trial in Europe.

*: For details, please refer to our press releases dated July 1,2024, and November 19, 2025, and February 28, 2026.

About the EMA’s Scientific Advice
Through this SA procedure, GEXVal received formal advice on its clinical development plan in Europe, covering the design of the planned Phase IIa clinical trial and the overall quality, non-clinical, and clinical data package. This advice does not guarantee the outcome of any future regulatory review. GEXVal will carefully review the advice and reflect it appropriately in its future development plans.

1) Japan's Specified Intractable Disease designation is granted under the Act on Medical Care for Patients with Intractable Diseases (enforced January 2015), which built on a national rare-disease policy framework dating back to 1972. To qualify, a disease must have an unknown cause, no established treatment, require long-term care, and affect no more than approximately 0.1% of Japan's population, among other criteria. As of April 2026, 348 diseases were designated. Designated patients are eligible for subsidized medical costs, including a monthly out-of-pocket cap based on income.

For more information (Japanese only), see the Japan Intractable Disease Information Center: https://www.nanbyou.or.jp/entry/4141

Additional Resources:
Japan Intractable Disease Information Center: https://www.nanbyou.or.jp/entry/4612
National Organization for Rare Disorders (NORD): https://rarediseases.org/rare-diseases/fragile-x-syndrome/
Orphanet: https://www.orpha.net/en/disease/detail/908?name=fxs&mode=name
FRAXA Research Foundation: https://www.fraxa.org

[About GEXVal]
GEXVal strives to create and develop innovative pharmaceuticals for unmet medical needs, ensuring Treatment Reaches the Unreached with focus on rare diseases and underserved medical conditions. By leveraging our proprietary AI-powered pharmacoinformatics technology, we illuminate paths to breakthrough therapies, identifying hidden potential in drug candidates to deliver life-changing medicines that bring new hope to patients and their families.

This press release contains forward-looking statements, including statements regarding GEXVal's planned clinical trials, regulatory strategy, and future development plans for GXV-001. These statements are based on current expectations and assumptions and are subject to risks and uncertainties, including those related to clinical trial results, the regulatory approval process, and the availability of funding, among others. Actual results may differ materially from those expressed or implied by these forward-looking statements. GEXVal undertakes no obligation to update these statements except as required by law.

有關本事宜請諮詢:
Head of Corporate Office
Atsushi Sugizaki
info@gexval.com