Newron Pharmaceuticals (NWRN) Investor Day 2024 summary

Strategic focus and future plans

• Development is centered on evenamide as an add-on therapy for treatment-resistant schizophrenia, with regulatory alignment for a single pivotal phase III study to support approval in the US and Europe.

• The pivotal phase III trial will enroll more severe patients, with no upper limit on PANSS scores, and will use rigorous adherence monitoring.

• The study will assess efficacy at 12, 26, and 52 weeks, with continuous dosing throughout the year.

• Commercial strategy includes seeking global or regional partnerships, with a focus on retaining US and Japan rights until after pivotal results.

• Preparations for the pivotal trial are advanced, with plans to initiate within months and explore additional indications such as bipolar disorder and dementia.

Financial guidance and business developments

• The company has invested several million euros in preparatory work for the pivotal trial and is actively seeking licensing or partnership deals to fund further development.

• The commercial approach aims to maximize shareholder value by potentially licensing non-strategic territories while retaining key markets.

• The pivotal trial is expected to provide the data required for global registration, with a target of 1,500 patients for approval.

• The company anticipates significant value inflection upon successful completion of the pivotal study.

• The company is exploring partnering options to complete Phase III development, aiming for potential blockbuster status if approved.

Clinical and scientific highlights

• Evenamide, a glutamate release modulator, has shown significant efficacy in placebo-controlled and long-term studies for patients with inadequate response or resistance to antipsychotics.

• The drug is well-tolerated, with a high completion rate, minimal side effects, and 25% of TRS patients achieving remission.

• Mechanistically, evenamide targets hippocampal hyperactivity, blocks voltage-gated sodium channels, and normalizes glutamate release, addressing both positive and negative symptoms.

• The pivotal trial will use international TRRIP criteria for treatment resistance and aims to confirm efficacy and safety in a broader, more severe population.

• Preclinical models show evenamide reverses abnormal neuronal activity, normalizes dopamine function, and may induce neuronal repair.