Newron Initiates U.S. Phase II Trial in Patients with Schizophrenia

MILAN--()--Newron Pharmaceuticals S.p.A. ("Newron"), a research and development company focused on novel CNS and pain therapies, announced today the initiation of a U.S. Phase II study with its novel sodium channel blocker, NW-3509, in patients with schizophrenia. NW-3509 is an orally available new chemical entity that specifically targets voltage-gated sodium channels, acting with a unique mechanism of action.

The Phase II trial is designed to investigate safety and preliminary evidence of efficacy in a double-blind, placebo-controlled randomized trial. NW-3509 will be administered as add-on treatment in schizophrenic patients experiencing break-through psychotic symptoms while on stable and adequate doses of their current atypical antipsychotics. The 4-week study will be performed in a minimum of 60 patients in two U.S. study centers. Patients will be treated twice daily with 5-25 mg/day of NW-3509, or placebo. The doses to be tested in this study in patients were found to be well-tolerated in healthy subjects and were associated with plasma levels that overlapped with exposure shown to be effective in preclinical models of psychosis. Doses and study design were finalized after receiving FDA input and guidance. Study results are expected to be available in Q4 2016.

The potential benefits of NW-3509 have been demonstrated in numerous animal models predictive of efficacy in psychiatric diseases, including models of psychosis such as amphetamine-induced hyperactivity, sensorimotor gating and information processing deficits (pre-pulse inhibition impairment induced by different stimuli), mania and depression.

Ravi Anand, M.D., Ph.D., Newron's Chief Medical Officer, stated: "The addition of NW-3509 in patients not responding to their current antipsychotic treatment may improve positive symptoms. New preclinical results also indicate that NW-3509 improves social withdrawal due to Phencyclidine or PCP ("angel dust"), an important predictor of negative symptom improvement."

About Schizophrenia

Schizophrenia is a long-term mental health condition that causes a range of different psychological symptoms. It is one of the most common serious mental health conditions. About 1 in 100 people will experience schizophrenia in their lifetime, with many continuing to lead normal lives. Schizophrenia is most often diagnosed between the ages of 15 and 35. Men and women are affected equally. There is no single test for schizophrenia. It is most often diagnosed after an assessment by a mental health care professional, such as a psychiatrist. It is important that schizophrenia is diagnosed as early as possible, as the chances of recovery improve the earlier it is treated. Schizophrenia is often described in terms of positive and negative (or deficit) symptoms. Positive symptoms are those that most individuals do not normally experience but are present in people with schizophrenia. They can include delusions, disordered thoughts and speech, and tactileauditoryvisualolfactory andgustatory hallucinations, typically regarded as manifestations of psychosis. Hallucinations are also typically related to the content of the delusional theme. Positive symptoms generally respond well to medication. Negative symptoms are deficits of normal emotional responses or of other thought processes, and are less responsive to medication. They commonly include flat expressions or little emotionpoverty of speechinability to experience pleasurelack of desire to form relationships, and lack of motivation. Negative symptoms appear to contribute more to poor quality of life, functional ability, and the burden on others than do positive symptoms. People with greater negative symptoms often have a history of poor adjustment before the onset of illness, and response to medication is often limited.

About NW-3509

NW-3509 is an orally available new chemical entity that specifically targets voltage-gated sodium channels. The compound modulates sustained repetitive firing, without inducing impairment of normal neuronal excitability. NW-3509 normalizes glutamate release induced by aberrant sodium channel activity. The potential benefits of NW-3509 have been demonstrated in numerous preclinical models predictive of efficacy in psychiatric diseases, including models of psychosis such as amphetamine-induced hyperactivity, sensorimotor gating and information processing deficits (pre-pulse inhibition impairment induced by different stimuli), mania and depression. Efficacy of NW-3509 has also been demonstrated in models of aggression and compulsive behavior, as well as in short- and long-term memory tests. Sub-threshold doses of NW-3509 increased the activity of inactive doses of both typical and atypical antipsychotics in models of schizophrenia, psychosis and mania. Moreover, given its neuronal stabilization properties, NW-3509 may reduce relapses and prevent or treat episodes of psychosis due to established super-sensitivity psychosis (SSP) induced by antipsychotics. As it is devoid of the risk of drug-induced movement disorders or weight gain, NW-3509 can be given in combination for extended periods of time.

About Newron Pharmaceuticals

Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain. The Company is headquartered in Bresso near Milan, Italy. Marketing authorization in the EU for Xadago® (safinamide) for the treatment of Parkinson's disease was granted by the EU Commission in February 2015, followed by the launch by Zambon in the first key EU country - Germany - in May 2015. The New Drug Application (NDA) has been accepted for review by the FDA, as reported in March 2015. In November 2015, Swissmedic has granted marketing authorization to Zambon, Newron's partner. Zambon has the rights to develop and commercialize safinamide globally, excluding Japan and other key Asian territories, where Meiji Seika has the rights to develop and commercialize the compound. Newron's additional projects are based on highly promising treatments for rare disease patients and are at various stages of clinical development. They include sarizotan for patients with Rett syndrome, for which Newron received Orphan Drug Designation in both the US and the EU, ralfinamide for patients with specific rare pain indications, and NW-3509 as potentially the first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. For additional information, please visit http://www.newron.com.

Suggested Articles

Novartis is ending early discovery at its R&D site in Shanghai to “rebalance” its discovery and early development efforts.

Californian RNA biotech Arrowhead will lose its COO and R&D head from next year but is hiring a new CMO and CSO to help steady its research exec team.

The biotech began testing the small molecule in a phase 3 trial of heavily pretreated small cell lung cancer patients late last year.