Newron Pharmaceuticals (NWRN) 38th Annual Roth Conference summary

Clinical program overview

• Three pivotal global studies are underway, enrolling over 1,300 patients, including a major phase III trial in Japan with a partner, Eisai.

• The clinical focus is on treatment-resistant and poorly responding schizophrenia patients, a group with significant unmet needs.

• The drug, evenamide, is positioned as the first add-on therapy with a novel mechanism, targeting hyperexcitability in the hippocampus rather than dopamine pathways.

• Clinical results show sustained improvement up to one year, with responder rates increasing over time and notable remission rates in treatment-resistant patients.

• The pivotal phase III studies are designed for both short-term (12 weeks) and long-term (one year) efficacy, with rigorous patient selection and monitoring to ensure data quality.

Mechanism of action and differentiation

• Evenamide uniquely targets hyperexcitable neurons in the hippocampus, reducing excessive glutamate release without affecting basal glutamate levels.

• This mechanism addresses both positive and negative symptoms, as well as cognitive decline, unlike current antipsychotics.

• The drug acts only on abnormally firing neurons, minimizing side effects and offering a "magic switch" effect.

• No other drugs in development or on the market target this mechanism or patient population as an add-on.

• Competitive therapies have failed in similar settings, often due to dopaminergic overlap or lack of efficacy in add-on trials.

Clinical results and regulatory expectations

• Phase II and III studies demonstrated statistically significant improvements in PANSS Total scores, with responder rates doubling or tripling over time.

• The drug outperformed standard of care, including clozapine and olanzapine, in key subpopulations.

• Placebo response rates were minimized through strict patient screening and compliance checks.

• Regulatory approval is expected with a 2–4 point delta in PANSS Total, with the NDA submission targeted within 12–15 months and potential approval by end of 2027.

• The drug's value is reinforced by a EUR 100 million partnership deal and a EUR 1.5 billion intrinsic valuation.