Oryzon's Vafidemstat Gains Key Japan Patent for CNS Disorders

MADRID, Spain – February 02, 2026 – Spanish biopharmaceutical company Oryzon Genomics has secured a major intellectual property victory in Asia, receiving a “Decision to grant” a new patent in Japan for its flagship central nervous system (CNS) drug candidate, vafidemstat. The patent specifically protects the drug's use in treating aggression and social withdrawal, debilitating behavioral symptoms common across several psychiatric disorders.

This move provides a critical shield for Oryzon’s commercial ambitions in one of the world's most valuable pharmaceutical markets and reinforces the company's strategy of building a global bulwark of intellectual property around its innovative epigenetic therapies. The announcement comes as vafidemstat advances toward late-stage clinical trials for conditions like Borderline Personality Disorder (BPD) and Autism Spectrum Disorder (ASD), where effective treatments for aggression remain a significant unmet medical need.

A Strategic Foothold in a Crucial Market

The Japanese patent is more than a legal formality; it's a strategic keystone for Oryzon's expansion. Japan's CNS therapeutic market, valued at over $5.8 billion in 2024, is projected to surge to over $10 billion by 2030. This growth is fueled by two powerful demographic and social trends: a rapidly aging population leading to a higher prevalence of neurodegenerative diseases and a growing societal awareness and diagnosis of mental health conditions.

For a European biotech like Oryzon, establishing a strong patent position in Japan is essential for long-term commercial viability. The country’s Pharmaceuticals and Medical Devices Agency (PMDA) has significantly modernized its regulatory process, with approval times for new drugs now among the fastest in the world. This efficiency, combined with Japan's increasing participation in multi-regional clinical trials, has made the market both attractive and intensely competitive.

The newly allowed claims, stemming from patent application JP2023-136283, will protect vafidemstat’s use for aggression and social withdrawal until at least 2038, excluding potential extensions. This secures a long runway for potential market exclusivity should the drug gain regulatory approval.

“This patent allowance in Japan further expands our global patent portfolio and strengthens Oryzon’s IP position for vafidemstat, protecting key therapeutic indications under clinical development and supporting its long-term commercial potential,” said Neus Virgili, Oryzon’s Chief IP Officer, in a statement.

Vafidemstat: Targeting the Unmet Needs of Brain Disorders

Vafidemstat is an oral, brain-penetrant molecule at the forefront of a new class of psychiatric drugs known as LSD1 inhibitors. Its development program is squarely aimed at behavioral symptoms that are poorly addressed by current medications. Following a series of promising mid-stage trials, Oryzon is now preparing for the next critical phase of clinical evaluation.

The company is gearing up for a pivotal Phase III trial to assess vafidemstat’s efficacy in treating aggression in patients with Borderline Personality Disorder. This follows the completion of the global Phase IIb PORTICO trial, which provided the foundation for this late-stage advancement. Furthermore, a Phase II trial is being prepared to investigate its effects on aggression in individuals with Autism Spectrum Disorder, and a separate Phase IIb study in schizophrenia, called EVOLUTION, is already underway.

Confidence in this development path is supported by earlier Phase IIa studies. The REIMAGINE trial, which included patients with BPD, ASD, and ADHD, demonstrated a significant reduction in aggression. Similarly, the REIMAGINE-AD trial showed positive results in reducing agitation and aggression in patients with moderate to severe Alzheimer's disease. Across these studies, vafidemstat has consistently shown a favorable safety and tolerability profile, a crucial factor for drugs intended for long-term use in psychiatric care.

The Promise of Epigenetics in Psychiatry

Oryzon's approach with vafidemstat represents a paradigm shift in treating brain disorders. The drug is part of an emerging field called epigenetics, which focuses on modifying gene expression without altering the underlying DNA code itself. Essentially, epigenetic therapies aim to correct the cellular “software” that can go awry in complex diseases.

Vafidemstat works by inhibiting an enzyme called LSD1 (Lysine-specific demethylase 1), a key regulator of gene activity. In several CNS disorders, the function of LSD1 is dysregulated, contributing to the disease's pathology. Preclinical research has suggested that by inhibiting this enzyme, vafidemstat can help restore normal gene expression patterns, thereby reducing neuroinflammation, providing neuroprotective effects, and mitigating behavioral symptoms like aggression.

This novel mechanism offers a departure from traditional psychiatric drugs that primarily target neurotransmitter systems like serotonin or dopamine. While CNS drug development is notoriously challenging, with high costs and failure rates, the scientific community has shown renewed interest in the field, driven by innovative approaches like epigenetics that promise more precise and effective treatments for conditions that have long resisted therapeutic breakthroughs.

Building a Global Fortress of Intellectual Property

The Japanese patent is the latest addition to Oryzon's extensive and meticulously constructed global IP portfolio for vafidemstat. This single patent does not stand alone but is part of a coordinated global strategy to ensure long-term market exclusivity across key territories. Corresponding patents covering the treatment of aggression and social withdrawal have already been granted or allowed in Europe, Australia, Canada, South Korea, and Russia, among other nations.

Beyond this, Oryzon holds a separate patent family specifically directed at the treatment of BPD, which has already been granted in major markets including Europe and Japan. These patents are expected to remain in force until at least 2040, providing an even longer horizon of protection for one of the drug's primary indications.

This multi-layered IP strategy is designed to protect the company's significant investment in research and development and secure its commercial future. By creating a global fortress of patents, Oryzon ensures that if vafidemstat successfully navigates the final stages of clinical trials and regulatory review, the company will be well-positioned to capitalize on its pioneering work.

With a strong scientific rationale and a fortified patent portfolio, the focus now shifts entirely to clinical execution. The upcoming Phase III and Phase II trials will be the ultimate crucible, determining whether vafidemstat can fulfill its potential and deliver a much-needed new therapeutic option for patients and families grappling with the profound challenges of severe behavioral disturbances.