PRESS RELEASE : Newron announces results of explanatory studies with evenamide in healthy volunteers and patients with schizophrenia

DGAP-News: Newron Pharmaceuticals S.p.A. / Key word(s): Study results 
Newron announces results of explanatory studies with evenamide in healthy volunteers and patients with schizophrenia 
2021-04-01 / 07:00 
The issuer is solely responsible for the content of this announcement. 
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Newron announces results of explanatory studies with evenamide in healthy volunteers and patients with schizophrenia 
  . Primary objective of safety of evenamide met on all safety variables for study 010 in healthy volunteers and study 
    008 in patients with schizophrenia 
  . Additional safety and efficacy study 008A in therapeutic dose (30 mg BID) to start in April 2021 
Milan, Italy and Morristown, NJ, USA, April 1, 2021 - Newron Pharmaceuticals S.p.A. ("Newron") (SIX: NWRN, XETRA: NP5), 
a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and 
peripheral nervous system, today announced initial results from two short-term explanatory studies in evenamide: study 
010 in healthy volunteers and study 008 in patients with schizophrenia. 
Results from study 010, a four-week, single dose, cross-over Thorough QT (TQT) study in 56 healthy volunteers, designed 
to evaluate the effects of evenamide (30 mg and 60 mg) compared with placebo and moxifloxacin 400 mg on the QT segment 
specifically, and on the electrocardiogram (ECG) generally, was requested by the US Food and Drug Administration (FDA) 
and under the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use 
(ICH). The results indicate that evenamide was devoid of any QTcF prolongation compared to placebo (indicating lack of 
any increased risk of arrythmia), while moxifloxacin was associated with a 17.3 ms median maximum increase suggestive 
of clinically significant risk of arrhythmia. These results strongly suggest that evenamide does not increase a 
patient's risk of QTc prolongation and arrhythmias, a risk generally associated with antipsychotics. 
Study 008, a four-week, randomized, double-blind placebo-controlled study was designed to primarily evaluate the 
safety, tolerability, and electroencephalogram (EEG) effects of two fixed doses of evenamide (7.5 mg and 15 mg BID). 
The study was requested by the FDA to address questions which arose from a study of evenamide in rats, and central 
nervous system (CNS) events observed following high-dose administration of evenamide in dogs. The study was performed 
in 138 outpatients with chronic schizophrenia, receiving treatment with a second-generation atypical antipsychotic at 
study centers in the United States and India. 
Over 95% of the patients completed study 008. No patient on evenamide discontinued from the study due to adverse 
events, and there were no significant adverse events relating to evenamide. No symptoms were observed suggestive of 
severe CNS events, symptoms/signs of seizures, EEG diagnosis of seizure like activity, or cardiac events in patients 
with evenamide. There were no differences in laboratory, ECG or vital signs abnormalities between evenamide and 
placebo-treated patients. The most frequent adverse events observed were related to CNS, gastrointestinal disorders, 
psychiatric disorders, metabolism and nutrition disorders and laboratory investigations. The most frequent adverse 
events reported (greater than 5%) were headache and somnolence, which were equally distributed between evenamide and 
placebo. 
Study 008 was designed to primarily assess safety and was not powered to demonstrate efficacy. The results, as 
expected, indicated that the 7.5 mg BID was a "no-effect" dose, which will not be investigated further. The 15 mg BID 
dose produced a higher magnitude response on the total PANSS than 7.5 mg BID, but this was not statistically 
significant when compared to placebo. The safety of the 30 mg BID (designated as the therapeutic dose) will be assessed 
in a planned additional study 008A in patients with schizophrenia, which is required in order to fully comply with the 
FDA's original request prior to starting the planned phase III program. Study 008A will be initiated in the next days, 
with results expected in the second half of 2021. 
Ravi Anand, MD, Newron's CMO, commented: "The results of study 010 are of far-reaching importance as they indicate that 
evenamide, even at doses of 60 mg (twice the therapeutic dose), is devoid of any arrhythmic effect and thus can be 
safely added to any other antipsychotic. Furthermore, the safety data, specifically, the lack of any systemic pattern 
of adverse effects relating to the CNS (including EEG) indicate that the drug is safe at the doses investigated. We 
will now evaluate the safety of the 30 mg (BID) dose, the expected therapeutic dose, in patients with schizophrenia, in 
study 008A and plan for the initiating of our phase III program shortly after." 
The proposed phase III clinical trial program with evenamide targets patients with schizophrenia experiencing worsening 
of psychosis on atypical antipsychotics, and treatment-resistant patients not responding to clozapine. Clozapine is the 
only antipsychotic approved worldwide for treatment-resistant schizophrenia. The program will commence once study 008A 
results are available. 
Newron is currently evaluating potential options for partnering/co-developing the further development of evenamide. 
About evenamide 
Evenamide has the potential to be first add-on therapy for the treatment of patients with positive symptoms of 
schizophrenia. The compound is an orally available New Chemical Entity that specifically targets voltage-gated sodium 
channels for the treatment of schizophrenia. Evenamide originates from Newron's ion channel program and has a unique 
mechanism of action: glutamate modulation and voltage-gated sodium channel blockade. Evenamide modulates sustained 
repetitive firing, without inducing impairment of normal neuronal excitability. It normalizes glutamate release induced 
by aberrant sodium channel activity. In a Phase IIa clinical study, Newron demonstrated evenamide's evidence of 
efficacy in significantly improving symptoms of psychosis compared with placebo when added to two of the most commonly 
prescribed atypical antipsychotics in patients with chronic schizophrenia. The study also indicated that evenamide is 
devoid of an effect on any of the over 130 neurotransmitters, enzymes, or transporters targeted by most antipsychotics. 
About Newron Pharmaceuticals 
Newron (SIX: NWRN, XETRA: NP5) is a biopharmaceutical company focused on the development of novel therapies for 
patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, 
Italy. Xadago^(R)/safinamide has received marketing authorization for the treatment of Parkinson's disease in the 
European Union, Switzerland, the USA, Australia, Canada, Brazil, Colombia, Israel, the United Arab Emirates, Japan and 
South Korea, and is commercialized by Newron's Partner Zambon. Supernus Pharmaceuticals holds the commercialization 
rights in the USA. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian 
territories. Newron is also developing evenamide as the potential first add-on therapy for the treatment of patients 
with positive symptoms of schizophrenia. For more information, please visit: www.newron.com 
For more information 
Newron 
Stefan Weber - CEO 
[phone] 
[e-mail] 
UK/Europe 
Simon Conway/ Natalie Garland-Collins, FTI Consulting 
[e-mail] 
Switzerland 
Valentin Handschin, IRF Reputation 
[phone] 
[e-mail] 
Germany/Europe 
Anne Hennecke/Caroline Bergmann, MC Services 
[phone] 
[e-mail] 
USA 
Paul Sagan, LaVoieHealthScience 
[phone], Ext. 112 
[e-mail] 
Important Notices 
This document contains forward-looking statements, including (without limitation) about (1) Newron's ability to develop 
and expand its business, successfully complete development of its current product candidates, the timing of 
commencement of various clinical trials and receipt of data and current and future collaborations for the development 
and commercialization of its product candidates, (2) the market for drugs to treat CNS diseases and pain conditions, 
(3) Newron's financial resources, and (4) assumptions underlying any such statements. In some cases, these statements 
and assumptions can be identified by the fact that they use words such as "will", "anticipate", "estimate", "expect", 
"project", "intend", "plan", "believe", "target", and other words and terms of similar meaning. All statements, other 
than historical facts, contained herein regarding Newron's strategy, goals, plans, future financial position, projected 
revenues and costs and prospects are forward-looking statements. By their very nature, such statements and assumptions 
involve inherent risks and uncertainties, both general and specific, and risks exist that predictions, forecasts, 
projections and other outcomes described, assumed or implied therein will not be achieved. Future events and actual 
results could differ materially from those set out in, contemplated by or underlying the forward-looking statements due 
to a number of important factors. These factors include (without limitation) (1) uncertainties in the discovery, 
development or marketing of products, including without limitation difficulties in enrolling clinical trials, negative 
results of clinical trials or research projects or unexpected side effects, (2) delay or inability in obtaining 
regulatory approvals or bringing products to market, (3) future market acceptance of products, (4) loss of or inability 
to obtain adequate protection for intellectual property rights, (5) inability to raise additional funds, (6) success of 
existing and entry into future collaborations and licensing agreements, (7) litigation, (8) loss of key executive or 

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