CNS

Roeselare, Belgium
Roeselare, Belgium
SEARCH FILTERS
Time filter
Source Type

Patent
Cns | Date: 2015-08-21

Remote collaboration systems and methods may involve a server in communication with a network and a plurality of remote computers in communication with the network. The server may connect to the plurality of remote computers via the network. The server may generate a collaboration environment for each of the remote computers. The server may assign an access level from a plurality of access levels to each of the remote computers, the plurality of access levels including a view only access level and an edit access level. The server may provide secure access to a file to each of the remote computers at the access level assigned to each of the remote computers via the collaboration environment. The file can be remotely viewed by any of the remote computers having a view only access level and remotely viewed and remotely edited by any of the remote computers having an edit access level via the collaboration environment.


FREMONT, Calif., May 09, 2017 (GLOBE NEWSWIRE) -- Zosano Pharma Corporation (NASDAQ:ZSAN), a CNS-focused company with a lead product, M207, that recently established a differentiated safety and efficacy profile in a pivotal trial as an acute treatment for migraine, today reported financial results for the first quarter ended March 31, 2017. In addition, John P. Walker, Chairman of the company’s Board of Directors, has been named Interim Chief Executive Officer to replace Konstantinos Alataris who has resigned as CEO and director of the company. Georgia Erbez, our Chief Business Officer and Interim Chief Financial Officer, has assumed full responsibility for both functions. “The first quarter saw our lead product candidate meet both co-primary endpoints in ZOTRIP, our pivotal efficacy study of M207 as an acute treatment for migraine. In addition, the company completed a follow-on offering that resulted in $29.3 million in gross proceeds earmarked for advancing M207 towards FDA approval. These two important accomplishments are a result of the commitment and capabilities of Zosano’s management team and gives me great confidence in our ability to continue to meet the strategic milestones established by the company.” “The pivotal study results importantly validate our technology platform, and, if approved by the FDA, point to M207’s positioning as an acute treatment for migraine sufferers that is differentiated from what is currently available.  I look forward to working with the team at Zosano and to bringing this exciting new drug to market,” commented John P. Walker, Interim Chief Executive Officer. "On behalf of the Board of Directors, I want to thank Konstantinos Alataris for his efforts and commitment to the company over the past two years. We wish him well in his future endeavors,” added Walker. In February, the Company announced statistically significant results from the ZOTRIP trial, which demonstrated that the 3.8mg dose of M207 met both co-primary endpoints, achieving pain freedom and most bothersome symptom freedom at 2 hours. The 3.8mg dose achieved a p value of <0.05  in the secondary endpoints of pain freedom at 45 minutes and 1 hour, and showed durability of effect on pain freedom to 24 and 48 hours. These results demonstrated that M207 not only provided fast onset but also a durability of effect, up to 2 days and hence freedom from recurrence of migraine. Additionally, M207 demonstrated a similar safety profile as other triptans and no Serious Adverse Events (SAEs) were reported in the trial. The FDA has indicated that a single, positive, pivotal efficacy study, in addition to a safety study of M207, will be sufficient to file for approval under a 505(b)(2) pathway. The Company plans to initiate the safety study in the second half of 2017. Financial Results for the First Quarter Ended March 31, 2017 Walker brings to Zosano more than 40 years of experience as a Board Chairman, Chief Executive Officer and interim CEO at life science companies. In these roles, he has been involved with Vitaphore, which was sold to Union Carbide Chemicals and Plastics; Arris/Axys, which was sold to Celera Genomics; Centaur, which was sold to Renovis; Signal Pharmaceuticals, which was sold to Celgene; Kai Pharmaceuticals, which was sold to Amgen; Guava Technologies, which was sold to Millipore; and iPierian, which was sold to Bristol Myers Squibb as well as in the mergers of Novacea and Transcept and of Renovis and Evotec. He is currently serving as Executive Chairman of Vizuri Life Sciences LLC and has been a director on the Boards of other life science companies, including Geron, Evotec and Affymax. In addition, he currently serves on the Board of Directors of the Lucille Packard Children’s Hospital at Stanford University and Random Acts of Flowers, a non-profit that repurposes flowers for ill patients. He began his early business career at American Hospital Supply Corporation where he became President of the Hospital Company. Migraine is the leading cause of disability among neurological disorders in the United States according to the American Migraine Foundation. Migraine symptoms can include moderate to severe headache pain combined with nausea and vomiting, or abnormal sensitivity to light and sound.  According to the Migraine Research Foundation, migraine affects 30 million men, women and children in the United States. Most migraines last between four and 24 hours, but some last as long as three days. According to published studies, 63% of migraine patients experience between one and four migraines per month. According to Decision Resources, prescription drug sales for migraine in the top seven countries were estimated to be $3.3 billion in 2015, and are expected to grow to $4.4 billion in 2020. Triptans, a family of tryptamine-based drugs first sold in the 1990s, account for almost 75% of anti-migraine therapies prescribed at office visits. M207 is our proprietary formulation of zolmitriptan coated onto our patented intracutaneous microneedle patch, which is then applied with our proprietary applicator to ensure uniform and consistent application. In February 2017, the Company announced statistically significant results from the ZOTRIP trial, which demonstrated that the 3.8mg dose of M207 met both co-primary endpoints, achieving pain freedom and most bothersome symptom freedom at 2 hours.  In a Phase 1 trial, M207 demonstrated markedly faster absorption kinetics compared to oral zolmitriptan. The Company presented these results at the 2016 annual meeting of the American Headache Society. Zosano Pharma Corporation is an emerging CNS company focusing on providing rapid symptom relief to patients using known therapeutics and altering their delivery profile using the Company’s proprietary intracutaneous delivery system. The Company’s goal is to make intracutaneous drug delivery a standard of care for delivering drugs requiring fast onset of action. Zosano Pharma has developed its proprietary intracutaneous delivery system to administer proprietary formulations of existing drugs through the skin for the treatment of a variety of indications.  The Company believes that its intracutaneous delivery system offers rapid and consistent drug delivery combined with ease of use. The Company is focused on developing products that deliver established molecules with known safety and efficacy profiles for markets where patients remain underserved by existing therapies. Zosano Pharma anticipates that many of its current and future development programs may enable the Company to utilize a regulatory pathway that would streamline clinical development and accelerate the path towards commercialization. Learn more at www.zosanopharma.com. This press release contains forward-looking statements regarding the timing of expected clinical development milestones, sufficiency of our capital resources and need for future funding and other future events and expectations. Readers are urged to consider statements that include the words "may," "will," "would," "could," "should," "might," "believes," "estimates," "projects," "potential," "expects," "plans," "anticipates," "intends," "continues," "forecast," "designed," "goal," "unaudited," "approximately" or the negative of those words or other comparable words to be uncertain and forward-looking. These statements are subject to risks and uncertainties that are difficult to predict and actual outcomes may differ materially. These include risks and uncertainties, without limitation, associated with the process of discovering, developing and commercializing products that are safe and effective for use as human therapeutics, risks inherent in the effort to build a business around such products and other risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent annual report on Form 10-K.. Although we believe that the expectations reflected in these forward-looking statements are reasonable, we cannot in any way guarantee that the future results, level of activity, performance or events and circumstances reflected in forward-looking statements will be achieved or occur. All forward-looking statements are based on information currently available to Zosano and Zosano assumes no obligation to update any such forward-looking statements.


FREMONT, Calif., May 09, 2017 (GLOBE NEWSWIRE) -- Zosano Pharma Corporation (NASDAQ:ZSAN), a CNS-focused company with a lead product, M207, that recently established a differentiated safety and efficacy profile in a pivotal trial as an acute treatment for migraine, today reported financial results for the first quarter ended March 31, 2017. In addition, John P. Walker, Chairman of the company’s Board of Directors, has been named Interim Chief Executive Officer to replace Konstantinos Alataris who has resigned as CEO and director of the company. Georgia Erbez, our Chief Business Officer and Interim Chief Financial Officer, has assumed full responsibility for both functions. “The first quarter saw our lead product candidate meet both co-primary endpoints in ZOTRIP, our pivotal efficacy study of M207 as an acute treatment for migraine. In addition, the company completed a follow-on offering that resulted in $29.3 million in gross proceeds earmarked for advancing M207 towards FDA approval. These two important accomplishments are a result of the commitment and capabilities of Zosano’s management team and gives me great confidence in our ability to continue to meet the strategic milestones established by the company.” “The pivotal study results importantly validate our technology platform, and, if approved by the FDA, point to M207’s positioning as an acute treatment for migraine sufferers that is differentiated from what is currently available.  I look forward to working with the team at Zosano and to bringing this exciting new drug to market,” commented John P. Walker, Interim Chief Executive Officer. "On behalf of the Board of Directors, I want to thank Konstantinos Alataris for his efforts and commitment to the company over the past two years. We wish him well in his future endeavors,” added Walker. In February, the Company announced statistically significant results from the ZOTRIP trial, which demonstrated that the 3.8mg dose of M207 met both co-primary endpoints, achieving pain freedom and most bothersome symptom freedom at 2 hours. The 3.8mg dose achieved a p value of <0.05  in the secondary endpoints of pain freedom at 45 minutes and 1 hour, and showed durability of effect on pain freedom to 24 and 48 hours. These results demonstrated that M207 not only provided fast onset but also a durability of effect, up to 2 days and hence freedom from recurrence of migraine. Additionally, M207 demonstrated a similar safety profile as other triptans and no Serious Adverse Events (SAEs) were reported in the trial. The FDA has indicated that a single, positive, pivotal efficacy study, in addition to a safety study of M207, will be sufficient to file for approval under a 505(b)(2) pathway. The Company plans to initiate the safety study in the second half of 2017. Financial Results for the First Quarter Ended March 31, 2017 Walker brings to Zosano more than 40 years of experience as a Board Chairman, Chief Executive Officer and interim CEO at life science companies. In these roles, he has been involved with Vitaphore, which was sold to Union Carbide Chemicals and Plastics; Arris/Axys, which was sold to Celera Genomics; Centaur, which was sold to Renovis; Signal Pharmaceuticals, which was sold to Celgene; Kai Pharmaceuticals, which was sold to Amgen; Guava Technologies, which was sold to Millipore; and iPierian, which was sold to Bristol Myers Squibb as well as in the mergers of Novacea and Transcept and of Renovis and Evotec. He is currently serving as Executive Chairman of Vizuri Life Sciences LLC and has been a director on the Boards of other life science companies, including Geron, Evotec and Affymax. In addition, he currently serves on the Board of Directors of the Lucille Packard Children’s Hospital at Stanford University and Random Acts of Flowers, a non-profit that repurposes flowers for ill patients. He began his early business career at American Hospital Supply Corporation where he became President of the Hospital Company. Migraine is the leading cause of disability among neurological disorders in the United States according to the American Migraine Foundation. Migraine symptoms can include moderate to severe headache pain combined with nausea and vomiting, or abnormal sensitivity to light and sound.  According to the Migraine Research Foundation, migraine affects 30 million men, women and children in the United States. Most migraines last between four and 24 hours, but some last as long as three days. According to published studies, 63% of migraine patients experience between one and four migraines per month. According to Decision Resources, prescription drug sales for migraine in the top seven countries were estimated to be $3.3 billion in 2015, and are expected to grow to $4.4 billion in 2020. Triptans, a family of tryptamine-based drugs first sold in the 1990s, account for almost 75% of anti-migraine therapies prescribed at office visits. M207 is our proprietary formulation of zolmitriptan coated onto our patented intracutaneous microneedle patch, which is then applied with our proprietary applicator to ensure uniform and consistent application. In February 2017, the Company announced statistically significant results from the ZOTRIP trial, which demonstrated that the 3.8mg dose of M207 met both co-primary endpoints, achieving pain freedom and most bothersome symptom freedom at 2 hours.  In a Phase 1 trial, M207 demonstrated markedly faster absorption kinetics compared to oral zolmitriptan. The Company presented these results at the 2016 annual meeting of the American Headache Society. Zosano Pharma Corporation is an emerging CNS company focusing on providing rapid symptom relief to patients using known therapeutics and altering their delivery profile using the Company’s proprietary intracutaneous delivery system. The Company’s goal is to make intracutaneous drug delivery a standard of care for delivering drugs requiring fast onset of action. Zosano Pharma has developed its proprietary intracutaneous delivery system to administer proprietary formulations of existing drugs through the skin for the treatment of a variety of indications.  The Company believes that its intracutaneous delivery system offers rapid and consistent drug delivery combined with ease of use. The Company is focused on developing products that deliver established molecules with known safety and efficacy profiles for markets where patients remain underserved by existing therapies. Zosano Pharma anticipates that many of its current and future development programs may enable the Company to utilize a regulatory pathway that would streamline clinical development and accelerate the path towards commercialization. Learn more at www.zosanopharma.com. This press release contains forward-looking statements regarding the timing of expected clinical development milestones, sufficiency of our capital resources and need for future funding and other future events and expectations. Readers are urged to consider statements that include the words "may," "will," "would," "could," "should," "might," "believes," "estimates," "projects," "potential," "expects," "plans," "anticipates," "intends," "continues," "forecast," "designed," "goal," "unaudited," "approximately" or the negative of those words or other comparable words to be uncertain and forward-looking. These statements are subject to risks and uncertainties that are difficult to predict and actual outcomes may differ materially. These include risks and uncertainties, without limitation, associated with the process of discovering, developing and commercializing products that are safe and effective for use as human therapeutics, risks inherent in the effort to build a business around such products and other risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent annual report on Form 10-K.. Although we believe that the expectations reflected in these forward-looking statements are reasonable, we cannot in any way guarantee that the future results, level of activity, performance or events and circumstances reflected in forward-looking statements will be achieved or occur. All forward-looking statements are based on information currently available to Zosano and Zosano assumes no obligation to update any such forward-looking statements.


News Article | May 11, 2017
Site: globenewswire.com

REHOVOT, Israel, May 11, 2017 (GLOBE NEWSWIRE) -- NeuroDerm Ltd. (Nasdaq:NDRM), a clinical stage pharmaceutical company developing drug-device combinations for central nervous system (CNS) disorders, today announced financial results for the first quarter ended March 31, 2017. “We are pleased with our continued clinical progress for our lead product candidates, and remain on track to achieve our anticipated milestones for ND0612 and ND0701,” stated Oded Lieberman, PhD, CEO of NeuroDerm. “Our recently announced preclinical, life cycle management product candidate ND0901 demonstrates our long term commitment to the treatment of Parkinson's Disease and to continuous levodopa delivery, and we are excited to be developing another potential treatment option for patients suffering from this debilitating disease. We had the opportunity to meet with many patients during the Parkinson’s Unity Walk, which took place in New York City last month, and their support of our efforts was both gratifying and an important reminder of the urgency with which new treatments are needed.” Announced ND0901, a continuous, subcutaneous, follow-on life cycle management product for the treatment of Parkinson’s disease At its first Investor Day this past April, the Company announced a new drug-device candidate for continuous subcutaneous LD/CD delivery. This pre-clinical drug candidate comprises a novel, concentrated aqueous LD prodrug, and will potentially be developed for treating Parkinson's disease motor complications. Clinical development of ND0901 is anticipated to start by early 2018. The currently anticipated timelines for the ongoing and upcoming clinical trials are as follows: Financial Results for the Quarter Ended March 31, 2017 Research and development expenses were $10.1 million in the three months ended March 31, 2017 compared to $4.1 million in the same period in 2016.  The increase was primarily due to an increase in subcontractors and materials mainly from ongoing clinical trials and pre-clinical studies, and due to an increase in payroll and related expenses, including share-based compensation expenses, as a result of an increase in the number of employees. General and administrative expenses were $3.3 million in the three months ended March 31, 2017 compared to $1.6 million in the same period in 2016.  The increase was primarily due to an increase in share-based compensation expenses. The company reported a net loss of $13.1 million in the three months ended March 31, 2017 compared to $5.4 million for the same period in 2016.  The increase in net loss was primarily due to the increase in research and development expenses. As of March 31, 2017, the company had cash and cash equivalents and short-term bank deposits totaling $142.3 million. NeuroDerm is a clinical-stage pharmaceutical company with a limited operating history, has not yet generated revenue from its operations and continues to incur significant research and development and other expenses related to its ongoing operations. Accordingly, there is no assurance that NeuroDerm’s business will generate positive cash flow.  As of March 31, 2017, NeuroDerm had an accumulated deficit of $193.8 million and its activities have been funded through public and private offerings of the company’s securities and issuance of convertible loans and warrants. NeuroDerm hosted its first Investor Day on April 21, 2017 and provided a thorough review of its product candidates and timelines. Therefore, the company will not host a conference call surrounding first quarter, 2017 financial results, but plans to resume quarterly updates surrounding the release of second quarter financial results. Individuals interested in listening to the Investor Day presentation may do so by clicking here. About NeuroDerm NeuroDerm is a clinical-stage pharmaceutical company developing central nervous system (CNS) product candidates that are designed to overcome major deficiencies of current treatments and achieve enhanced clinical efficacy through continuous, controlled administration.  The company has three product candidates in different stages of development which offer a solution for almost every Parkinson’s disease patient from the moderate to the very severe stage of the disease.  The company has developed a line of levodopa and carbidopa (LD/CD) product candidates administered through small belt pumps that deliver a continuous, controlled dose of LD/CD.  The LD/CD product candidates are ND0612L and ND0612H, which are used for treatment of moderate and advanced Parkinson’s disease patients, respectively, and which are delivered subcutaneously.  In addition, NeuroDerm is developing ND0701, a novel subcutaneously delivered apomorphine formulation for patients who suffer from moderate to severe Parkinson’s disease and who do not respond well to LD/CD.  NeuroDerm is headquartered in the Weizmann Science Park in Rehovot, Israel. Forward-Looking Statements This press release contains forward-looking statements, within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended that involve risks and uncertainties. Such forward-looking statements may include projections regarding our future performance and may be identified by words like "anticipate," "assume," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "future," "will," "seek" and similar terms or phrases. The forward-looking statements contained in this press release are based on management's current expectations and projections about future events. There are important factors that could cause our actual results, levels of activity, performance or achievements to differ materially from the results, levels of activity, performance or achievements expressed or implied by the forward-looking statements. In particular, you should consider the risks provided under "Risk Factors" in our annual report on Form 20-F for the year ended December 31, 2016 filed with the Securities and Exchange Commission. Any forward-looking statement made by us in this press release speaks only as of the date hereof. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.


News Article | May 9, 2017
Site: www.prnewswire.com

"We are pleased to welcome the dedicated, knowledgeable, and highly skilled teams of MedAvante and ProPhase and delighted to add Dr. Jeff Litwin as the CEO of this important addition to our organization," said Donald A. Deieso, PhD, chairman and chief executive officer of WCG.  "To accelerate the development of life-saving therapies for the patients who need them is our priority.  We seek partners who share our vision of increasing efficiencies in the clinical trial process through the intelligent application of innovation, clinical expertise, and technology." He added, "MedAvante and ProPhase have a proven track record and long-standing history in the CNS space-- pioneering ingenious solutions to some of pharma's most intractable problems with the best and most current thinking in this field." "My co-founder, Amy Ellis, and I launched MedAvante to conceive and globalize innovations to improve the lives of patients," says Paul M Gilbert, co-founder & chief executive officer. "We're thrilled to become part of WCG, which has the resources and capabilities to help us achieve the next level of our vision." "We are excited to be joining WCG and combining forces with another market leader to transform how novel treatments are brought to market and improve treatment options and outcomes for all stakeholders," said principals Sofija Jovic, PhD, MBA, chief executive officer and Mark Opler, PhD, MPH, chief scientific officer and founder at ProPhase. "Combining the strengths of MedAvante and ProPhase with the expansive clinical research expertise and global reach of WCG will provide best-of-class expertise and services for those conducting clinical trials in the challenging fields of CNS and mental health," said Jeffrey Litwin, MD, CEO of MedAvante and ProPhase. "The collective tools and scientific expertise of both companies – bolstered by the formidable resources of WCG – will have a tremendous impact on the success of clinical trials, contributing to improving the lives of patients suffering from debilitating and life-threatening diseases." Clients of both MedAvante and ProPhase can look forward to the same scientific excellence, technological advances, and dedication to quality customer service they have come to expect. WCG will provide corporate support to these two organizations as they continue to expand, with access to capital, and complementary clinical and regulatory expertise. Financial details about the transaction were not disclosed. About WIRB-Copernicus Group WIRB-Copernicus Group (WCG) is the world's leading providers of solutions that measurably improve the quality and efficiency of clinical research. The industry's first clinical services organization (CSO), WCG enables biopharmaceutical companies, CROs and institutions to accelerate the delivery of new treatments and therapies to patients, while maintaining the highest standards of human subject protections. For more information, please visit www.wcgclinical.com or follow us on Twitter @WCGClinical. About MedAvante MedAvante is a clinical science-driven data services company dedicated to maximizing signal detection in global clinical trials. With Virgil™, its electronic clinical outcome assessment (eCOA) platform, clinical research sponsors are able to maximize outcome data quality and increase operational efficiency for clinical trials across multiple therapeutic areas. MedAvante built the first electronic source (eSource) data platform and has conducted more than 687,000 assessments of 56,000 clinical trial subjects across 233,000 study visits. MedAvante's eSource data has been accepted by both the Food and Drug Administration and the European Medicines Agency in successful regulatory submissions. About ProPhase ProPhase is a global provider of measurement-related specialty solutions in clinical trials. For over a decade, ProPhase has focused on applying innovation to optimize the selection, use, and analysis of behavioral endpoints to mitigate trial risk and maximize the likelihood of study success.  ProPhase specializes in the development of new outcome measures, calibration of real world outcomes, placebo response mitigation, and prediction of treatment adherence.  These approaches have led to successful trials in indications across the CNS spectrum, as well as multiple rare and orphan diseases. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/wirb-copernicus-group-wcg-acquires-medavante-and-prophase-300453748.html


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sage Therapeutics, Inc. (NASDAQ:SAGE), a clinical-stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders, today reported business highlights and financial results for the first quarter ended March 31, 2017. “We continue to make good progress in building Sage into a leading CNS company with the potential to deliver differentiated medicines for a variety of central nervous system disorders. We are focused on closing the innovation gap in areas of brain disorders where more breakthroughs for patients are needed,” said Jeff Jonas, M.D., Chief Executive Officer of Sage. “We are now nearing completion of enrollment in our lead Phase 3 program in super-refractory status epilepticus (SRSE), and remain focused on completing Phase 3 clinical development of brexanolone in both SRSE and postpartum depression (PPD) in 2017. Further, we continue to advance our growing pipeline of novel product candidates, including our lead oral compound, SAGE-217, in four clinical programs in both mood and movement disorders.” Sage is nearing completion of enrollment in the Phase 3 STATUS Trial, the first ever global, randomized, double-blind, placebo-controlled trial in SRSE. The Company expects to report top-line results from the STATUS Trial in the third quarter of 2017 after enrollment of an anticipated 126 evaluable patients and completion of all study follow-up periods and data analysis. Sage expects top-line results to include the primary endpoint, safety and tolerability, and select secondary endpoints, including open-label retreatment arm response and the Clinical Global Impression scale. Top-Line Results from Part A of SAGE-217 Phase 2 in Parkinson’s Disease Sage today announced top-line results from the Part A open-label portion of a Phase 2 clinical trial of SAGE-217 in Parkinson's disease: Sage is advancing a portfolio of novel central nervous system (CNS) product candidates targeting the GABA and NMDA receptor systems. Dysfunction in these systems is known to be at the core of numerous psychiatric and neurological disorders. Sage is pursuing a data-driven approach to CNS drug development by employing efficient human proof-of-concept studies both to uncover activity signals and to help understand future trial methodology, before investing in larger clinical programs. Financial Results for the First Quarter of 2017 Sage will host a conference call and webcast today at 4:30 PM ET to discuss its first quarter financial results and recent corporate updates. The live webcast can be accessed on the investor page of Sage's website at investor.sagerx.com. The conference call can be accessed by dialing 1-866-450-8683 (toll-free domestic) or 1-281-542-4847 (international) and using the conference ID 12287678. A replay of the webcast will be available on Sage’s website approximately two hours after the completion of the event and will be archived for up to 30 days. Sage Therapeutics is a clinical-stage biopharmaceutical company committed to developing novel medicines to transform the lives of patients with life-altering central nervous system (CNS) disorders. Sage has a portfolio of novel product candidates targeting critical CNS receptor systems, GABA and NMDA. Sage's lead program, brexanolone (SAGE-547), is in Phase 3 clinical development for super-refractory status epilepticus, a rare and severe seizure disorder, and for postpartum depression. Sage is developing its next generation modulators, including SAGE-217 and SAGE-718, in various CNS disorders. For more information, please visit www.sagerx.com. Various statements in this release concern Sage's future expectations, plans and prospects, including without limitation: our expectations regarding development of our product candidates and their potential in the treatment of various CNS disorders; the expected timing of initiation and completion of clinical trials; the anticipated availability and announcement of data and results from clinical trials of our product candidates; our plans for evaluation of new indications and new compounds; our expectations regarding the regulatory pathway for brexanolone (SAGE-547) in the treatment of SRSE in the EU, and our belief that the results of the current development program for brexanolone in SRSE, if successful, will be sufficient for an MAA filing in the EU; our expectations regarding a potential future new drug application and MAA filing and commercial launch of brexanolone, if successfully developed and approved; and our expectations with respect to future cash use and cash needs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: we may continue to experience slower than expected enrollment and randomization of evaluable patients in the STATUS trial or slower than expected clinical site initiation and enrollment in our other clinical trials, or the potential need for additional analysis or data or the need to enroll additional patients, leading to possible delays in completion of trials or in the availability of data; we may not be able to generate supportive non-clinical data or to successfully demonstrate the efficacy and safety of our product candidates at each stage of clinical development; success in our non-clinical studies or in earlier stage clinical trials may not be repeated or observed in ongoing or future studies involving the same compound or other product candidates, and ongoing and future pre-clinical and clinical results may not support further development of product candidates or be sufficient to gain regulatory approval to launch and commercialize any product; decisions or actions of regulatory agencies may affect the initiation, timing, progress and cost of clinical trials, and our ability to proceed with further clinical studies of a product candidate or to obtain marketing approval or may result in restrictions in an approved indication or the need for additional clinical trials, including the risk that the EMA may, despite scientific advice, decide that the data from our Phase 3 trial in SRSE are not sufficient to support approval; the internal and external costs required for our activities, and to build our organization in connection with such activities, and the resulting use of cash, may be higher than expected, or we may conduct additional clinical trials or pre-clinical studies, or engage in new activities, requiring additional expenditures and using cash more quickly than anticipated; and we may encounter technical and other unexpected hurdles in the development and manufacture of our products which may delay our timing or increase our expenses and use of cash, as well as those risks more fully discussed in the section entitled "Risk Factors" in our most recent Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today, and should not be relied upon as representing our views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.


News Article | May 12, 2017
Site: www.businesswire.com

MILAN--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”, SIX: NWRN), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, today announced the receipt of milestone payments linked to the approval of its lead compound Xadago® (safinamide) for the treatment of Parkinson’s disease by the US Food and Drug Administration. The payments were made by Newron’s partner Zambon, who holds the rights to develop and commercialize Xadago® globally, excluding Japan and other key territories where Meiji Seika has the rights to develop and commercialize the compound, and relate to the 2012 “strategic collaboration and license agreement for safinamide” between Newron and Zambon. Payments totaled EUR11.3 million which under the applicable IFRS rules will be recognized as immediate revenue. Newron is also eligible to receive royalty payments on sales as part of the 2012 agreement. 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. Xadago® (safinamide) has received marketing authorization for the treatment of Parkinson’s disease in the European Union, Switzerland and the USA, and is commercialized by Newron’s Partner Zambon. US WorldMeds 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. In addition to Xadago® for Parkinson’s disease, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. www.newron.com 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 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.


LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 09, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company developing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2017 and highlighted recent progress in advancing its pipeline.    “In early 2017, we continued to make progress across our strategic priorities, including our gene therapy programs in hemophilia B, Huntington’s disease and congestive heart failure,” stated Matthew Kapusta, chief executive officer of uniQure. “Over the past few months, AMT-060 received Breakthrough Therapy and PRIME designation based on the promising data from our ongoing Phase I/II study, and we look forward to presenting updated data from the study in July at ISTH.  We also initiated discussions with the FDA regarding planning for a pivotal study, and expect additional interactions with regulators in 2017 as we prepare for late-stage development.” “In addition, we recently presented promising, new preclinical data in Huntington’s disease from a large animal model, and remain on track to initiate an IND-enabling toxicology study in the second half of 2017,” added Mr. Kapusta.  “We also continue to advance, in collaboration with BMS, our preclinical studies of S100A1 in heart failure, as well as other early stage product candidates targeting CNS and liver-directed diseases, and we look forward to providing an update on our progress this year.” Cash Position:  As of March 31, 2017, the Company held cash and cash equivalents of $120.3 million, compared with $132.5 million as of December 31, 2016. The decrease in cash was primarily related to the advancement of its clinical and preclinical gene therapy targets, general corporate activities and capital expenditures related to its facilities in Lexington, MA and Amsterdam. The Company intends to significantly reduce capital expenditures in 2017 and 2018 and realize operational cost savings from the strategic restructuring initiated in November 2016 and the withdrawal of Glybera in October 2017.  As a result of these initiatives, the Company expects its cash on hand will be sufficient to fund operations into 2019. Revenues:  Revenue for the first quarter of 2017 was $3.3 million compared with $4.3 million in the first quarter of 2016. The reduction in the current year period was driven by a decrease in collaboration revenue compared to the prior year period.  The reduction was primarily due to the timing of various reimbursable activities, including the production of preclinical material for the Company’s S100A1 gene therapy that occurred in the prior year period. R&D Expenses:  Research and development expenses for the first quarter of 2017 were $17.0 million compared with $16.7 million in the first quarter of 2016.  The increase was primarily related to higher operating expenses and depreciation related to our Amsterdam facilities. SG&A Expenses:  Selling, general and administrative expenses for the first quarter of 2017 were $6.4 million compared with $7.3 million in the first quarter of 2016.  The decrease was primarily related to one-time costs related to our CEO transition incurred in the prior year period. Net Loss:  The net loss for the first quarter of 2017 was $20.3 million, or $0.80 per share, compared with $22.3 million, or $0.90 per share, for the first quarter of 2016. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with liver/metabolic, central nervous system and cardiovascular diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the implementation and effects of the Company’s new strategic and organizational changes, the development of our gene therapy product candidates, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with corporate restructuring, collaboration arrangements, our and our collaborators’ clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s 2016 Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 15, 2017. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.


NEW YORK, May 12, 2017 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc., (NASDAQ:OVID) a biopharmaceutical company committed to developing medicines for patients with rare neurological diseases, today announced that it will present preclinical data on TAK-935/OV935, a potent and highly selective cholesterol 24-hydroxylase inhibitor in development for the treatment of rare epileptic encephalopathies, at the Antiepileptic Drug and Device (AEDD) Trials XIV Conference taking place May 17-19, 2017, in Aventura (North Miami Beach), Fla. This will be the first public presentation of TAK-935 data. Details of the oral presentation are listed below. Presentation Session: Session XI: Drug Pipeline Title: TAK935/OV935, a Novel NMDA Modulator Under Development for Epileptic Encephalopathies Presenter: Matthew During, M.D., D.Sc., Ovid Therapeutics Time: May 19, 2017, 2:10 pm ET Location: Turnberry Isle Miami Hotel About TAK-935 TAK-935, which is being studied in rare pediatric epilepsies, is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). CH24H is predominantly expressed in the brain, where it plays a central role in cholesterol homeostasis. CH24H converts cholesterol to 24-S-hydroxycholesterol (24HC), which then exits the brain into the blood plasma circulation.i Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity.ii Recent literature indicates CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel,iii implying its potential role in CNS diseases such as epilepsy. To our knowledge, TAK-935 is the only molecule with this mechanism of action in clinical development. TAK-935 has been tested in preclinical models to provide data to support the advancement of the drug into human clinical studies in patients suffering from rare epilepsy syndromes. A novel proprietary PET ligand, developed by Takeda and Molecular Neuroimaging, LLC (MNI), has been used to determine target occupancy of TAK-935 in the brain.iv In addition, TAK-935’s effect on CH24H enzyme activity in the brain has been assessed by following measurable reductions in the plasma concentration of 24HC. TAK-935 has completed four Phase 1 clinical studies,v,vi,vii,viii which have assessed tolerability and target engagement at doses which are believed to be therapeutically relevant. About Ovid Therapeutics Ovid Therapeutics is a New York-based biopharmaceutical company developing therapies for patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of symptoms of Angelman syndrome and Fragile X syndrome. Ovid is also developing TAK-935/OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare epileptic encephalopathies. For more information on Ovid, please visit http://www.ovidrx.com/. This press release includes certain disclosures which contain “forward-looking statements,” including, without limitation, statements regarding the clinical development and scope of clinical trials, the timing of reporting clinical data for Ovid’s product candidates, and the potential use of TAK-935/OV935 to treat epileptic encephalopathies. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission, including its registration statement on Form S-1, as amended, under the caption “Risk Factors.” Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. i Russell DW, Halford RW, Ramirez DMO, Shah R, Kotti T. Cholesterol 24-hydroxylase: An enzyme of cholesterol turnover in the brain. Annu Rev Biochem. 2009;78:1017-1040. ii Mehta A, Prabhakar M, Kumar P, Deshmukh R, Sharma PL. Excitotoxicity: bridge to various triggers in neurodegenerative disorders. Eur J Pharmacol. 2013;698(1-3):6-18. iii Paul SM, Doherty JJ, Robichaud AJ, Belfort GM, Chow BY, Hammond RS, et al. The major brain cholesterol metabolite 24(S)-hydroxycholesterol is a potent allosteric modulator of N-methyl-D-aspartate receptors. J Neurosci. 2013;33(44):17290-300.


VY-AADC01 interim Phase 1b data recently presented at American Academy of Neurology and American Association of Neurological Surgeons meetings; program on track to report 6-month data from Cohort 3 and longer-term data from Cohorts 1 and 2 during the third quarter of 2017 VY-SOD101 lead clinical candidate selected and progressing towards IND filing; preclinical pipeline programs targeting devastating neurological diseases progressing towards lead clinical candidate selection CAMBRIDGE, Mass., May 09, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe neurological diseases, today reported its first quarter of 2017 financial results and corporate highlights. “The first quarter of 2017 reflected solid progress across the entire organization with our lead clinical program for advanced Parkinson’s disease, our pipeline of preclinical gene therapy programs, our product engine and our manufacturing platform,” said Steven Paul, M.D., president and chief executive officer of Voyager Therapeutics.  “For the first time, our investigators presented interim Phase 1b results for VY-AADC01 for advanced Parkinson’s disease at two major scientific conferences targeting key physician audiences for this program, and we remain on track to provide additional data from this trial during the third quarter.  The pipeline continues to advance towards IND filing for VY-SOD101 for the treatment of ALS and towards lead clinical candidate selections for both our Huntington’s disease and Friedreich’s ataxia programs.  All of this progress is based, in part, on our ability to optimally deliver these therapeutics to the brain or spinal cord, as well as to manufacture high quality GMP vectors at scale.”                  Recent Program Highlights As part of an oral presentation at the recent American Academy of Neurology meeting, investigators presented VY-AADC01 motor symptom data from Cohorts 1 and 2 and new functional and quality of life data as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS) Part II and Part IV and the patient-reported 39-item Parkinson's Disease Questionnaire (PDQ-39).  Along with improving motor symptoms, VY-AADC01 improved patients’ function and quality of life, which are key indicators of the potential clinical impact of this one-time treatment for advanced Parkinson’s disease.  The company remains on track to report data from Cohorts 1-3 during the third quarter of this year.  These data as well as data from the posterior (i.e., back of the head) infusion trajectory study will help inform the design of the double-blind, placebo-controlled (sham surgery) trial planned to begin during the fourth quarter of 2017. At the recent American Association of Neurological Surgeons Annual Scientific Meeting, also during an oral presentation, investigators reviewed the evolution of the real-time MRI-guided surgical procedure with VY-AADC01 that resulted in increased coverage of the putamen, a region of the brain associated with motor function in Parkinson’s disease.  These developments helped to increase the average coverage of the putamen from 21% in Cohort 1, to 34% in Cohort 2, and 42% in Cohort 3, as previously reported. Voyager continues to advance multiple preclinical programs towards clinical trials, with the goal of filing three IND applications within the next 24-months for the VY-SOD101, VY-HTT01, and VY-FXN01 programs.  During the first quarter of 2017, Voyager selected VY-SOD101 as a clinical candidate for the treatment of ALS caused by mutations in the superoxide dismutase 1 gene (SOD1).  With a single intrathecal (IT) injection, VY-SOD101 has the potential to durably reduce the levels of toxic mutant SOD1 protein in the CNS to slow the progression of disease.  Preclinical pharmacology and toxicology studies are now underway to support the filing of an investigational new drug (IND) application for VY-SOD101 during late 2017 or early 2018.  This timing update reflects the time required to complete the preclinical toxicology testing and to produce good manufacturing process (GMP) material to support the clinical trial.  During the quarter, the company also progressed its preclinical candidates towards selection of lead clinical candidates for VY-HTT01 for Huntington’s disease and VY-FXN01 for Friedreich’s ataxia. Voyager’s management team members will participate in the following investor conference: Live-streaming webcasts of this presentation can be accessed through the Investors & Media section of Voyager’s website at www.voyagertherapeutics.com.  The webcast will be archived for 30 days after the live event concludes. First Quarter 2017 Financial Results and 2017 Guidance Voyager reported a GAAP net loss of $16.6 million, or $0.65 per share, for the first quarter ended March 31, 2017, compared to a GAAP net loss of $7.2 million, or $0.29 per share, for the same period in 2016. Collaboration revenues of $1.5 million for the first quarter of 2017 compared to collaboration revenues of $4.8 million for the first quarter of 2016.  Collaboration revenues reflect recognition of payment for research and development services provided by Voyager for various programs under the Sanofi-Genzyme collaboration agreement.  The decrease in collaboration revenues for the first quarter of 2017 compared to the same period in 2016 reflects ongoing reassessments of performance periods for individual programs under the collaboration, as well as previously announced deprioritized development of VY-SMN101 for spinal muscular atrophy. Research and development (R&D) expenses of $14.1 million for the first quarter ended March 31, 2017 compared to $8.7 million for the same period in 2016.  The increase in R&D expenses was largely due to expenditures associated with the development of Voyager’s pipeline, and increased personnel and facility costs to support the advancement of the pipeline programs. General and administrative (G&A) expenses were $4.9 million for the first quarter ended March 31, 2017 compared to $3.6 million for the same period in 2016.  The increase in G&A expenses was primarily due to personnel costs to support Voyager’s growth and facility costs. Cash, cash equivalents, and marketable debt securities as of March 31, 2017 were $157.7 million.  Based on the company’s current operating plan, Voyager continues to expect to end 2017 with cash, cash equivalents, and marketable debt securities of approximately $90 million to $100 million and continues to project that its existing cash, cash equivalents, and marketable debt securities will be sufficient to fund operating expenses and capital expenditure requirements into 2019.             Conference Call Information Voyager will host a conference call and webcast today at 5:30 p.m. EDT.  The live call may be accessed by dialing (877) 851-3834 for domestic callers or +1 (631) 291-4595 for international callers, and referencing conference ID number 18530851.  A live audio webcast of the conference call will be available online from the Investors & Media section of Voyager’s website at www.voyagertherapeutics.com.  The webcast will be archived for 30 days. Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately 700,000 people in the U.S.1 and seven to 10 million people worldwide2.  It is estimated that up to 15% of the prevalent population with Parkinson’s disease, or approximately 100,000 patients in the U.S., have motor fluctuations that are refractory, or not well-controlled, with levodopa.  While the underlying cause of Parkinson's disease in most patients is unknown, the motor symptoms of the disease arise from a loss of neurons in the midbrain that produce the neurotransmitter dopamine.  Declining levels of dopamine in this particular region of the brain leads to the motor symptoms associated with Parkinson’s disease including tremors, slow movement or loss of movement, rigidity, and postural instability.  Motor symptoms during the advanced stages of the disease include falling, gait freezing, and difficulty with speech and swallowing, with patients often requiring the daily assistance of a caregiver. There are currently no therapies that effectively slow or reverse the progression of Parkinson’s disease.  Levodopa remains the standard of care treatment, with its beneficial effects on symptom control having been discovered over 40 years ago3.  Patients are generally well-controlled with oral levodopa in the early stages of the disease, but become less responsive to treatment as the disease progresses.  Patients experience longer periods of reduced mobility and stiffness termed off-time, or the time when medication is no longer providing benefit, and shorter periods of on-time when their medication is effective. The progressive motor symptoms of Parkinson’s disease are largely due to the death of dopamine neurons in the substantia nigra, a part of the midbrain that converts levodopa to dopamine, in a single step catalyzed by the enzyme AADC.  Neurons in the substantia nigra release dopamine into the putamen where the receptors for dopamine reside.  In advanced Parkinson’s disease, neurons in the substantia nigra degenerate and the enzyme AADC is markedly reduced in the putamen, which limits the brain’s ability to convert oral levodopa to dopamine4.  The intrinsic neurons in the putamen, however, do not degenerate in Parkinson’s disease5,6.  VY-AADC01, comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression, is designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located, bypassing the substantia nigra neurons and enabling the neurons of the putamen to express the AADC enzyme to convert levodopa into dopamine.  The approach with VY-AADC01, therefore, has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration. Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe neurological diseases.  Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing and dosing and delivery techniques.  The company’s pipeline focuses on severe neurological diseases in need of effective new therapies, including advanced Parkinson’s disease, a monogenic form of ALS, Huntington’s disease, Friedreich’s ataxia, frontotemporal dementia, Alzheimer’s disease and severe, chronic pain.  Voyager has broad strategic collaborations with Sanofi Genzyme, the specialty care global business unit of Sanofi, and the University of Massachusetts Medical School.  Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager Therapeutics is headquartered in Cambridge, Massachusetts.  For more information, please visit www.voyagertherapeutics.com.  Follow Voyager on LinkedIn. This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities law. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements.  For example, all statements Voyager makes regarding the initiation, timing, progress and reporting of results of its preclinical programs and clinical trials and its research and development programs, its ability to advance its AAV-based gene therapies into, and successfully complete, clinical trials, its ability to continue to develop its product engine, its ability to add new programs to its pipeline, ability to enter into new partnerships or collaborations, its expected cash, cash equivalents and marketable debt securities at the end of a fiscal year and anticipation for how long expected cash, cash equivalents and marketable debt securities will last, and the timing or likelihood of its regulatory filings and approvals, are forward looking.  All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes to be reasonable, are inherently uncertain.  All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.  These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its future filings with the Securities and Exchange Commission.  Any forward-looking statement speaks only as of the date on which it was made.  Voyager undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. 1 Willis et al, Neuroepidemiology.2010;34:143–151  2 www.pdf.org/en/parkinson_statistics  3 Poewe W, et al, Clinical Interventions in Aging.2010;5:229-238.  4 Lloyd, J Pharmacol Exp Ther. 1975;195:453-464, Nagatsu, J Neural Transm Suppl.2007  5 Cold Spring Harb Perspect Med 2012;2:a009258  6 Braak et al, Cell Tissue Res.2004;318:121-134

Loading CNS collaborators
Loading CNS collaborators