Sarizotan Hydrochloride

“We are very disappointed in the topline results in the STARS study that did not meet the study endpoints,” said Ravi Anand, Newron’s chief medical officer. Newron Pharmaceuticals , based in Milan, Italy and Morristown, New Jersey, announced topline results from its STARS clinical trial of sarizotan in Rett syndrome. The drug failed to hit the primary or secondary efficacy endpoints. As a result, the company is terminating the program. Rett syndrome is a severe neurodevelopment disorder. It mostly affects females, about one in 10,000. There are no approved treatments. The disease is marked by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, causing loss of ability to conduct daily life activities, walk or communicate. More than 95% of Rett patients have a random mutation in the MeCP2 gene. About 70% of patients have apnea, hyperventilation and disordered breathing. It is believed that about 25% of the deaths from the disease are related to multiple cardiorespiratory dysrhythmias. “We are very disappointed in the topline results in the STARS study that did not meet the study endpoints,” said Ravi Anand, Newron’s chief medical officer. “The results of this well designed and executed study, based on highly promising data from a genetic model of Rett syndrome in mice, indicate the difficulties inherent in translating effects in animal models to human clinical studies. We are currently awaiting results of additional explanatory analyses and will continue to analyze the full data set from the study to understand more about the results.” Sarizotan is a highly selective modulator of specific serotonin or dopamine receptors in the brain. The drug was originally developed by Merck KGaG, Darmstadt, Germany. hbspt.cta.load(4413123, '4e87abe9-779b-4c65-98dd-ea451779faa9', {}); STARS, which stands for Sarizotan for the Treatment of Apneas in Rett Syndrome, enrolled 129 Rett syndrome patients in 14 centers throughout the U.S., Europe, Asia and Australia. The trial lasted six months. Patients received daily doses of 10 mg and 20 mg of sarizotan or placebo. The primary endpoint was a percentage decrease in episodes of apnea during the waking time compared with placebo. No specific details were released yet, and the company indicates it plans to work with the Rett research community and families to share results from the trial as well as from the Rett Syndrome International Burden of Illness Survey. “We sincerely want to thank the patients, caregivers and their families who participated in this landmark study,” said Stefan Weber, Newron’s chief executive officer. “This was an extremely challenging study and the enthusiasm of the patients and caregivers indicates their need to find a treatment for this serious, underserved disease. Each participant has the gratitude of the study investigators, Newron’s employees, advisors and vendors who have worked diligently on this study over the last five years.” The company’s Xadago/safinamide has been approved for Parkinson’s disease in Europe, Switzerland, the U.S., Australia, Canada, Brazil, Colombia, the United Arab Emirates and Japan. The company plans to prioritize its Phase III program of evenamide in schizophrenia and evaluate additional pipeline candidates. Newron delayed the launch of a Phase II/III schizophrenia trial in evenamide last summer at the request of the U.S. Food and Drug Administration (FDA). The delay was requested after reviewing preclinical animal studies. However, evenamide, an oral selective glutamate inhibitor, passed through Phase IIa clinical trial in 89 people in 2017 without any safety issues. There were apparently some central nervous system issues observed in dogs receiving the higher doses of the drug. The company and the FDA have since “agreed on the design and conduct of explanatory studies with evenamide required to address previously announced potential safety issues raised by the FDA including a four-week explanatory study in patients with schizophrenia; initial results in rats and humans are expected in Q3 2020.”

- Funding Set to Accelerate Clinical Development of Novel Medicines for Rare Central Nervous System Disorders - MILAN--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”), a research and development company focused on novel CNS and pain therapies, announces that it has completed a capital increase raising gross proceeds of CHF24.3 million/US$ 25.5 million, following the subscription by institutional investors of 843,072 newly issued shares. The capital increase was approved at the Company’s Extraordinary Shareholder’s Meeting on March 24, 2015. Once issued, the new shares will be immediately and freely tradable. “The funds raised will enable us to accelerate the development of sarizotan in Rett Syndrome and of NW-3509, a highly innovative add-on therapy for schizophrenia, for which we plan to start a Phase II study in Q3 2015.” The fundraising was supported by current institutional shareholders and institutional investors from Europe and the U.S., including Aviva, J.P. Morgan Asset Management, Investor AB, Sphera Global HealthCare Fund and Nyenburgh. The subscription price was set at CHF 28.80 per share, slightly below the closing price of Newron’s shares on April 27, 2015, of CHF29.60. The net proceeds from the fundraising will be used to accelerate the development of Newron’s pipeline of innovative CNS therapeutics, primarily sarizotan for patients with Rett Syndrome, as well as NW-3509, a novel antipsychotic for patients with positive symptoms of schizophrenia. Additional compounds in development include sNN0031 for patients with Parkinson’s disease who no longer respond to oral therapy and sNN0029 for patients with Amyotrophic Lateral Sclerosis (ALS). Stefan Weber, CEO of Newron, commented: “With Xadago® (safinamide) approved in the EU, where it will shortly be launched in the first member states by our partner Zambon, and with the U.S. NDA under FDA review for marketing authorization, Newron’s focus is now on expediting development of its highly promising CNS orphan compounds for which it holds the commercial rights. We thank existing and new shareholders for their support of Newron’s strategy for sustainable growth.” Roberto Galli, Newron’s VP Finance, added: “The funds raised will enable us to accelerate the development of sarizotan in Rett Syndrome and of NW-3509, a highly innovative add-on therapy for schizophrenia, for which we plan to start a Phase II study in Q3 2015.” Closing of the transaction will be subject to customary Italian and Swiss regulatory requirements. The new shares will be listed and traded on the SIX Swiss Exchange under the same ISIN as the Company’s existing shares (ISIN: IT0004147952) on or around May 6, 2015. Bank am Bellevue and WG Partners acted as advisors to Newron in connection with the transaction. About Newron PharmaceuticalsNewron (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) was granted by the EU Commission in February 2015, following the recommendation by the Committee for Medicinal Products for Human Use (CHMP) to approve the compound in the EU on Dec. 19, 2014. The New Drug Application NDA to the U.S. FDA, as informed early March, has been accepted for filing, after being re-submitted by Newron on Dec. 26, 2014. In March 2014, Zambon, a partner of Newron, submitted a MAA to Swissmedic. 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, including sarizotan for patients with Rett syndrome, sNN0031 for patients with Parkinson’s disease, non-responsive to oral drug treatments, sNN0029 for patients with ALS and ralfinamide for patients with specific rare pain indications. Newron is also developing NW-3509 as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. Important NoticesThis 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 and current and future collaborations for the development and commercialisation of its product candidates and reduce costs (including staff costs), (2) the market for drugs to treat CNS diseases and pain conditions, (3) Newron’s anticipated future revenues, capital expenditures and 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 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 other employees, (9) adverse publicity and news coverage, and (10) competition, regulatory, legislative and judicial developments or changes in market and/or overall economic conditions. Newron may not actually achieve the plans, intentions or expectations disclosed in forward-looking statements and assumptions underlying any such statements may prove wrong. Investors should therefore not place undue reliance on them. There can be no assurance that actual results of Newron's research programmes, development activities, commercialisation plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions. Newron does not undertake any obligation to publicly up-date or revise forward looking statements except as may be required by applicable regulations of the SIX Swiss Exchange where the shares of Newron are listed. This document does not contain or constitute an offer or invitation to purchase or subscribe for any securities of Newron and no part of it shall form the basis of or be relied upon in connection with any contract or commitment whatsoever. Contacts MediaNewron PharmaceuticalsStefan Weber, CEO, +39 02 6103 46 26pr@newron.comorUK/EuropeFTI ConsultingJulia Phillips, +44 (0)20 3727 1000orSwitzerlandIRF CommunicationsMartin Meier-Pfister, +41 43 244 81 40orU.S.LaVoieHealthScienceDavid Connolly, +1 617-374-8800, Ext. 108dconnolly@lavoiehealthscience.comorInvestors and analystsNewron PharmaceuticalsStefan Weber, CEO, +39 02 6103 46 30ir@newron.comorU.S.LaVoieHealthScienceKristina Coppola, +1 617-374-8800, Ext. 105kcoppola@lavoiehealthscience.com Help employers find you! Check out all the jobs and post your resume.