ORYZON Granted Two Key Patents for Vafidemstat in Japan

Oryzon Genomics, S.A., a clinical-stage biopharmaceutical firm specializing in epigenetics, has announced that the Japanese Patent Office (JPO) has granted approval for two patent applications related to its LSD1 inhibitor, vafidemstat. This drug is currently in clinical development for treating psychiatric conditions like Borderline Personality Disorder (BPD) and Attention Deficit Hyperactivity Disorder (ADHD).

The two newly approved patent applications are JP2021-556616, which focuses on methods for treating BPD, and JP2021-556629, which pertains to using KDM1A inhibitors like vafidemstat for ADHD treatment. These patents will remain valid until at least 2040, with potential extensions available. Similar applications are pending in other major pharmaceutical markets.

Oryzon has recently received similar approvals from the European and Korean patent offices for vafidemstat, specifically for treating aggression and social withdrawal, with a corresponding patent already granted in Russia. Neus Virgili, Oryzon's Chief IP Officer, expressed satisfaction with the approval, emphasizing that it strengthens the company's protective measures around vafidemstat. Dr. Carlos Buesa, the CEO, highlighted that their expanding intellectual property portfolio enhances the drug's unique value in significant market potential areas such as BPD and ADHD.

Founded in Barcelona in 2000, Oryzon is a leader in epigenetics and focuses on personalized medicine in central nervous system (CNS) disorders and oncology. The company has a skilled team in its offices in Barcelona, Boston, and San Diego. Oryzon's clinical portfolio includes two LSD1 inhibitors, vafidemstat for CNS disorders and iadademstat for oncology, both in various Phase II trials. They also have other pipeline assets targeting epigenetic factors like HDAC-6, with ORY-4001 being developed for Charcot-Marie-Tooth disease (CMT) and Amyotrophic Lateral Sclerosis (ALS).

Vafidemstat, also known as ORY-2001, is an oral LSD1 inhibitor optimized for CNS use. It has demonstrated the ability to reduce cognitive impairment, memory loss, and neuroinflammation, while also providing neuroprotective effects. In animal models, vafidemstat has restored memory and reduced aggression to normal levels. It has also shown efficacy in improving sociability and reducing social avoidance in mice. Additionally, the drug has proven effective in various preclinical models of multiple sclerosis (MS).

Oryzon has conducted several Phase IIa trials with vafidemstat, including the REIMAGINE trial for patients with different psychiatric disorders and REIMAGINE-AD for aggressive/agitated Alzheimer's Disease patients, both showing positive results. The ETHERAL trial in mild to moderate Alzheimer's patients showed a significant reduction in the inflammatory biomarker YKL40. The SATEEN trial in MS patients also indicated anti-inflammatory benefits. Moreover, vafidemstat has been tested in severe COVID-19 patients in the ESCAPE trial, where it demonstrated significant anti-inflammatory effects.

Currently, vafidemstat is under investigation in two double-blind, randomized, placebo-controlled Phase IIb trials: EVOLUTION for schizophrenia and PORTICO for Borderline Personality Disorder. The PORTICO trial has concluded, and Oryzon has requested an End-of-Phase II meeting with the FDA to discuss options for a Phase III trial in BPD. The company is also exploring vafidemstat's use in genetically-defined subpopulations of CNS disorders and preparing for a clinical trial in patients with Kabuki Syndrome. Further investigations into other neurodevelopmental syndromes are also underway.