Newron Pharmaceuticals (NWRN) 38th Annual Roth Conference summary

Clinical program overview

• Three pivotal phase III studies are underway globally, enrolling over 1,300 patients, with one study led by a Japanese partner and two by the company itself.

• The clinical program targets treatment-resistant and poorly responding schizophrenia patients, a population with significant unmet medical need.

• The studies are designed as add-ons to existing antipsychotics, including clozapine, with durations of 12 weeks and one year to capture both short- and long-term efficacy.

• The Japanese partnership with Eisai covers 7% of the global market and values the drug at EUR 1.5 billion for global rights.

Unmet medical need and mechanism of action

• Over 70% of schizophrenia patients do not benefit sufficiently from current dopamine-targeting drugs, leading to frequent medication switches and significant side effects.

• Evenamide is positioned as the first add-on therapy with a novel mechanism, targeting hyperexcitability in the hippocampus and reducing excessive glutamate release.

• The drug acts only on hyperactive neurons, sparing normal brain function and minimizing side effects.

• Clinical results show sustained improvement up to one year, including remission in treatment-resistant patients.

Key clinical results and study design

• Phase II and III studies demonstrated increasing efficacy over time, with responder rates tripling from six weeks to one year.

• In a four-week double-blind, placebo-controlled study, the drug doubled responder rates versus standard of care and showed significant improvement even in clozapine-resistant patients.

• The pivotal phase III studies include rigorous patient selection, blood level monitoring, and rater training to ensure data quality and minimize placebo response.

• The primary endpoint is PANSS Total, with a 3-4 point improvement over placebo considered sufficient for regulatory approval.