Spanish biopharmaceutical firm Oryzon Genomics has reported that vafidemstat led to improvements in aggressive behaviour of autism spectrum disorder (ASD) patients enrolled in the Phase IIa REIMAGINE study.

Vafidemstat is a central nervous system (CNS) epigenetic drug designed to specifically inhibit lysine-specific histone demethylase 1 (LSD1) and monoamine oxidase B (MAOB).

The drug is said to mitigate cognitive impairment, including memory loss and neuroinflammation, and also has neuroprotective effects.

REIMAGINE is intended to assess the safety, tolerability and efficacy of the drug to treat aggression in adults with ASD, borderline personality disorder (BPD) and attention deficit hyperactivity disorder (ADHD).

A total of 30 patients have been recruited for the eight-week, single-arm, open-label, basket study.

In the autism cohort of the trial, vafidemstat met the primary goal following two months of therapy.

Oryzon noted that significant global improvements were achieved on the Clinical Global Impression (CGI) of Severity (CGI-S) and Improvement (CGI-I) scales that measure aggressive behaviour.

In addition, the drug showed significant global improvement on the Neuropsychiatric Inventory (NPI) total score and specific improvement on a subscale for agitation/aggression.

The improvement in the NPI is believed to indicate a broad psychiatric effect with potential for the treatment beyond aggressiveness.

The drug was also found to be safe and well-tolerated and did not show any significant adverse events in ASD patients.

Oryzon Genomics Clinical and Product Development vice-president Dr Michael Ropacki said: “The REIMAGINE trial of vafidemstat has now produced positive results in three debilitating psychiatric disorders with high-unmet medical need: ADHD, autism spectrum disorder, and borderline personality disorder.

“Vafidemstat shows promise as a safe, well-tolerated and differentiated therapeutic option for treating agitation and aggression, and for treating non-aggressive features of three distinct psychiatric diseases.”

The drug is being developed for the treatment of multiple CNS conditions, including multiple sclerosis, Alzheimer’s disease and aggressiveness in psychiatric disorder patients.