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GAITHERSBURG, Md.--(BUSINESS WIRE)--Sigma-Tau Pharmaceuticals, Inc., a leader in the development and commercialization of medicines for patients with rare diseases, today announced that the company has changed its name to Leadiant Biosciences, Inc. reaffirming the company’s continued strong commitment to the patient communities it serves. The announcement coincides with Rare Disease Day 2017, a global campaign to raise awareness of rare diseases and improve access to available treatments and medical representation for people, and their caregivers, whose lives are impacted by these conditions. Now in its 10th year, Rare Disease Day is an annual celebration organized by the European Organization for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD). This year’s theme, With Research, Possibilities Are Limitless, underscores the importance of collaborative research in the drug-development process and recognizes the contributions of patients and families in advocating for increased investment in rare disease research. “Rare Disease Day is the perfect time to unveil our new name and reaffirm our commitment to the study of rare diseases, which has been an integral part of our heritage dating back to 1984 when we became only the fourth company in the world to receive an Orphan Drug Designation in the U.S.,” said Michael Minarich, chief executive officer, Leadiant Biosciences, Inc. “In 2017, Leadiant Biosciences will realize several important and exciting milestones in our product pipeline, as well as continuing multiple ongoing clinical trials.” For more information about the vision, mission and work of Leadiant Biosciences, Inc. visit www.leadiant.com. For more information about Rare Disease Day 2017, visit www.rarediseaseday.org or www.rarediseaseday.us. Leadiant Biosciences, Inc. (formerly Sigma-Tau Pharmaceuticals, Inc.) is a U.S.-based, wholly owned subsidiary of Leadiant Biosciences S.p.A., a research-based pharmaceutical company dedicated to the development and commercialization of medicines for patients with rare diseases. Based in Gaithersburg, Maryland, Leadiant Biosciences, Inc. dedicates considerable scientific and financial resources to the research, development, and distribution of novel and effective therapies that address patient needs and improve quality of life. For more information, visit www.leadiant.com.


News Article | February 28, 2017
Site: globenewswire.com

OXFORD, United Kingdom, Feb. 28, 2017 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, recognises the tenth annual Rare Disease Day taking place today, 28 February 2017. The Rare Disease Day 2017 theme, ‘with research, possibilities are limitless,’ emphasises the importance of scientific research in helping to understand, diagnose and treat rare diseases that affect millions of people and their families worldwide. Summit seeks to remain at the forefront of utrophin modulation research through its strategic alliance with the University of Oxford, under the guidance of Professor Kay Davies. The collaboration is focussed on developing future-generation utrophin modulators for the potential treatment of all patients with the progressive muscle wasting disorder, DMD. To date, the research team has identified two series of novel utrophin modulators, one of which has a mechanism of action potentially distinct from ezutromid, the Company’s lead utrophin modulator that is in a Phase 2 clinical trial in DMD patients. “In our quest to bring a potentially disease-modifying treatment to all patients with DMD, we have collaborated with the preeminent expert in utrophin modulation biology, Professor Kay Davies, and her research team at the University of Oxford,” said Glyn Edwards, Chief Executive Officer of Summit. “We applaud EURORDIS, the organisation representing rare disease patients in Europe, for bringing an annual spotlight to the plight of millions of people affected by rare diseases and in this year, recognising the immense impact that research is having and will continue to have for those living with rare diseases.” In the European Union a rare disease is defined as one that affects fewer than 5 in 10,000 of the general population, while in the United States, it is defined as a disease that affects fewer than 200,000 people. There are between 6,000 and 8,000 known rare diseases with around five new rare diseases described in the literature each week. Rare diseases are often chronic and life threatening and include rare conditions, such as childhood cancers, and some other well-known conditions including cystic fibrosis and DMD. Rare Disease Day takes place on the last day of February each year, and its objective is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was launched in Europe in 2008 by EURORDIS. It is now observed in more than 80 nations, and is sponsored in the US by the National Organization for Rare Disorders (NORD). For more information, please visit www.rarediseaseday.org. About Utrophin Modulation in DMD DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward-looking Statements Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


News Article | February 28, 2017
Site: globenewswire.com

ROCKVILLE, Md., Feb. 28, 2017 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (Nasdaq:RGNX), a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV® Technology Platform, joins the global rare disease community today in honoring Rare Disease Day® 2017, dedicated to raising awareness of rare diseases and their impact on the lives of patients and their families. This year’s theme, “Research,” recognizes the importance that scientific research plays in identifying and increasing understanding of unknown diseases, enabling doctors to make correct diagnoses and generating the development of treatments and potentially of cures. “REGENXBIO is proud to continue our support of Rare Disease Day to raise awareness of the importance of research and therapeutic development in rare diseases,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “REGENXBIO is dedicated to advancing a broad pipeline of NAV-based gene therapies to address a wide range of rare diseases with high unmet need. There are currently over 20 development programs based on NAV Technology including our internal development efforts and the work of our NAV Technology Licensees.” In recognition of Rare Disease Day, REGENXBIO will participate in activities in Washington, D.C., and will host a company-wide discussion at its Rockville, Maryland, headquarters with people affected by Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II), two rare diseases for which NAV-based gene therapies are in development. “Partnering with the communities that we serve is crucial to the development of innovative gene therapies that address the needs of individuals and their families,” said Vivian Fernandez, Director of Patient Advocacy at REGENXBIO. “We are actively engaged with a number of patient advocacy organizations including the FH Foundation and the National MPS Society, and we look forward to continuing to work closely with them as our lead gene therapy programs advance toward the clinic.”                                        REGENXBIO has received orphan drug designations from the United States Food and Drug Administration (FDA) for: The FDA has also granted rare pediatric disease designation to the RGX-111 and RGX-121 programs. For more information on REGENXBIO’s rare disease programs, please visit REGENXBIO’s website at www.regenxbio.com. To sign up for updates, please visit the Patients & Families section of REGENXBIO’s website. Rare Disease Day takes place every year on the last day of February to raise awareness among the general public and decision-makers about rare diseases and their impact on patients’ lives. Each year, organizations around the world hold a variety of activities that highlight rare and genetic diseases. Rare Disease Day was established in Europe in 2008 by the European Organization for Rare Diseases (EURORDIS), the organization representing the rare disease community in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the United States by the National Organization for Rare Disorders (NORD), a leading independent, non-profit organization committed to the identification, treatment and cure of rare diseases. For additional information on Rare Disease Day in the United States, please visit: www.rarediseaseday.us. For global activities, please visit: www.rarediseaseday.org. REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO’s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO’s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third-party NAV Technology Platform Licensees. REGENXBIO and its licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIO’s research, development and regulatory plans for RGX-111, RGX-121, RGX-501 and other gene therapies. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of REGENXBIO’s development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Meaningful factors which could cause actual results to differ include, but are not limited to, the timing of enrollment, commencement and completion of REGENXBIO’s clinical trials; the timing and success of preclinical studies and clinical trials conducted by REGENXBIO and its development partners; the ability to obtain and maintain regulatory approval to conduct clinical trials and to commercialize REGENXBIO’s product candidates, the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIO’s product candidates; competitive products; REGENXBIO’s ability to obtain and maintain intellectual property protection for REGENXBIO’s product candidates and technology; trends and challenges in REGENXBIO’s business and the markets in which REGENXBIO operates; REGENXBIO’s ability to attract or retain key personnel; the size and growth of the potential markets for REGENXBIO’s product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of REGENXBIO’s product candidates; REGENXBIO’s ability to establish and maintain development partnerships; REGENXBIO’s expenses and revenue; the sufficiency of REGENXBIO’s cash resources and needs for additional financing; regulatory developments in the United States and foreign countries; as well as other factors discussed in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2015 and Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which are available on the SEC’s website at www.sec.gov. Additional factors may be set forth in those sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2016, to be filed in the first quarter of 2017. In addition to the risks described above and in REGENXBIO’s filings with the SEC, other unknown or unpredictable factors also could affect REGENXBIO’s results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). REGENXBIO undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.


News Article | February 21, 2017
Site: www.businesswire.com

MILAN & MORRISTOWN, N.J.--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, today announced its support of this year’s Rare Disease Day® - which takes place worldwide on the last day of February each year – and the Rett community to raise awareness of the importance of research for rare diseases. United together, patients and caregivers, researchers and advocacy groups are working in common to elevate understanding and improve the lives of those impacted by rare diseases. “We are proud to continue our support of NORD (The National Organization for Rare Disorders in the USA) and EURORDIS (the European Organization for Rare Diseases) as they work to increase awareness for rare diseases and the unique challenges of those affected,” said Dennis Dionne, Vice President Commercial Affairs, Newron. “We are particularly honored to support groups such as Rettsyndrome.org which is dedicated to improving the lives of girls living with Rett syndrome.” Burden of Disease Study As part of its commitment to the rare disease patient community, Newron is partnering with the global Rett community to work on the first Burden of Disease (BOD) study. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. These learnings can help identify improved intervention programs and services designed to complement the Rett care pathway. STARS Study Newron has also initiated the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study, a potentially pivotal clinical study to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms. Among the core symptoms of Rett, breathing disturbances may affect the whole person body; they can have a marked effect on biochemistry, influence emotions, circulation and digestive function as well as musculoskeletal structures in the respiratory process. About Rare Disease Day® Rare Disease Day takes place on the last day of February each year, thus on February 28, 2017. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives. The campaign targets primarily the general public and also seeks to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases. The campaign started as a European event and has progressively become a world phenomenon, with the USA joining in 2009 and participants in over 80 countries throughout the world in 2016. In the United States, the National Organization for Rare Disorders (NORD) promotes and celebrates Rare Disease Day. For more information about Rare Disease Day in the U.S., please visit http://www.rarediseaseday.us. For information about global activities, please visit www.rarediseaseday.org. About Rare Diseases The European Union considers a disease as rare when it affects fewer than 5 in 10,000 citizens. According to EURORDIS, over 6,000 different rare diseases have been identified to date, affecting over 60 million people in Europe and the USA alone. Due to the low prevalence of each disease, medical expertise is rare, knowledge is scarce, care offering inadequate and research limited. Despite their great overall number, rare disease patients are the orphans of health systems, often denied diagnosis, treatment and the benefits of research. About Rett Syndrome Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence ranging from one in 10,000 to one in 20,000 females. There are no approved treatments available. Rett syndrome is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which leads to loss of ability to conduct daily life activities, walk or communicate. Rett syndrome also is associated with a reduced life expectancy. Approximately 25 percent of the deaths in patients with Rett syndrome are possibly related to multiple cardio-respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. More than 95 percent of these patients have a random mutation in the MECP2 gene. Episodes of apnea, hyperventilation and disordered breathing are found in approximately 70 percent of patients with Rett syndrome at some stage of their life. For more information on Rett Syndrome, visit www.rettsyndrome.org. 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 and Switzerland and is commercialized by Newron’s partner Zambon. US WorldMeds holds the commercialization rights in the US. 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 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 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 update 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.


CORAL GABLES, Fla., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), (Catalyst) a biopharmaceutical company focused on  developing  and  commercializing  innovative  therapies  for  people  with  rare  debilitating diseases, today announced that  it  has  joined  forces  with  30  million  Americans  and  health  care  advocates around the world for Rare Disease Day® on February 28. Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges they present to patients and families. Catalyst continues to support patient organizations that provide disease awareness for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS). These are rare diseases in which patients are often misdiagnosed with other diseases prior to getting a definitive diagnosis, thus educating the patient and physicians at medical congresses is an important outreach to the rare disease community to facilitate proper and prompt diagnoses. "Completing one phase 3 clinical study and enrolling a second phase 3 clinical study for LEMS indicates our commitment to the LEMS community for patients to have access to an FDA approved treatment" says Patrick J. McEnany, Chairman and CEO of Catalyst. "In addition, we have expanded our CMS phase 3 clinical study to include adults in recognition of the need to provide access to a potential treatment across the entire CMS population. We are pleased to continue to support the efforts of the rare disease community to find ways to broaden the awareness of rare diseases and improve access to treatments.” According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases and one which Catalyst actively supports.  Additionally, Catalyst supports Global Genes in their efforts to continue to get patients with rare diseases to take an active role in raising awareness about their disease. For more information about Rare Disease Day in the U.S., go to http://www.rarediseaseday.us.  For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website www.rarediseases.org and Global Gene’s website https://globalgenes.org/world-rare-disease-day. Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), infantile spasms, and Tourette's Disorder. Firdapse® has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of LEMS and Orphan Drug designation for LEMS, CMS and myasthenia gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS. Catalyst is also developing CPP-115 to treat infantile spasms, epilepsy and other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette's Disorder. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission.  In addition, Catalyst is developing a generic version of Sabril® (vigabatrin). This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA or the likelihood that the product will be found to be safe and effective, the timing on Catalyst's second trial evaluating Firdapse for the treatment of LEMS and whether the trial will be successful, whether Catalyst's assumptions in its updated business plan will be accurate and the impact of unanticipated events or delays in projected activities on Catalyst's cash requirements and on Catalyst's ability to get to an accepted NDA submission for Firdapse without the need for additional funding, what clinical trials and studies will be required before Catalyst can resubmit an NDA for Firdapse for the treatment of CMS and whether any such required clinical trials and studies will be successful, whether the investigator-sponsored study evaluating Firdapse for the treatment of MuSK-MG will be successful, whether any NDA for Firdapse resubmitted to the FDA will ever be accepted for filing,  the timing of any such NDA filing or acceptance, whether, if an NDA for Firdapse is accepted for filing, such NDA will be given a priority review by the FDA, whether Firdapse will ever be approved for commercialization, whether Catalyst will be the first company to receive approval for amifampridine (3,4-DAP), giving it 7-year marketing exclusivity for its product, whether CPP-115 will be determined to be safe for humans, what additional testing will be required before CPP-115 is "Phase 2 ready", whether CPP-115 will be determined to be effective for the treatment of infantile spasms, post-traumatic stress disorder, Tourette's Disorder or any other indications, whether Catalyst can successfully design and complete a bioequivalence study of its version of vigabatrin compared to Sabril that is acceptable to the FDA, whether any such bioequivalence study the design of which is acceptable to the FDA will be successful, whether any ANDA that Catalyst submits for a generic version of Sabril will be accepted for filing, whether any ANDA for Sabril accepted for filing by the FDA will be approved (and the timing of any such approval), whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2015 and its other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc., (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases, today joins forces with 30 million health care advocates around the world for the 10th annual Rare Disease Day®. Rare Disease Day, which is always the last day of February, is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges people living with rare diseases face. This year’s focus is on research and the global slogan for 2017 is “With Research, Possibilities are Limitless.” “A decade into its existence, the annual Rare Disease Day has achieved a noteworthy milestone in its efforts calling attention to the public health issues associated with rare diseases, a mission Ra Pharma is proud to support,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “As we prepare to initiate two Phase 2 clinical trials of our lead compound RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and refractory generalized myasthenia gravis (rMG), as well as a Phase 1b clinical trial supporting development in lupus nephritis (LN), we are confident in our ability to address the unmet need for people living with rare diseases where there are limited or no treatment options.” According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website, www.rarediseases.org. Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, subcutaneous self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement C5 with subnanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. RA101495 also directly binds to C5b, disrupting the interaction between C5b and C6 and preventing assembly of the membrane attack complex (MAC). This activity defines an additional and novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity. Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform allows us to produce synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. The platform generates highly specific and stable peptide-like molecules with the potential for greatly increased bioavailability, improved cell permeability, and the opportunity to address protein-protein interactions including previously undruggable targets. It can produce libraries of 10 to 100 trillion members, allowing for the rapid discovery of highly potent candidate molecules. The platform is being leveraged for our emerging pipeline projects in Factor D and C1. Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com. This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the safety, efficacy and regulatory and clinical progress of our product candidates, including RA101495. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Ra Pharma’s product candidates, including RA101495, will not successfully be developed or commercialized; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Quarterly Report on Form 10-Q, as well as other risks detailed in Ra Pharma’s subsequent filings with the Securities and Exchange Commission. There can be no assurance that the actual results or developments anticipated by Ra Pharma will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Ra Pharma. All information in this press release is as of the date of the release, and Ra Pharma undertakes no duty to update this information unless required by law.


News Article | February 28, 2017
Site: globenewswire.com

VANCOUVER, British Columbia and CAMBRIDGE, Mass., Feb. 28, 2017 (GLOBE NEWSWIRE) -- Novelion Therapeutics Inc. (NASDAQ:NVLN) (TSX:NVLN), a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, today announced today its alliance with the National Organization for Rare Disorders (NORD), Rare Diseases Europe (EURORDIS), Canadian Organization of Rare Disorders (CORD), and The Global Genes Project in observance of the annual Rare Disease Day. Chief Executive Officer Mary Szela commented, “Today we join with others in the community to raise awareness of rare diseases. We are mindful of the challenges these patients, their families and caregivers face daily, and we are motivated by a passion to support advocacy and the development of treatments for these rare diseases.” In the United States, a rare disease is defined as one that affects fewer than 200,000 persons. According to the National Institutes of Health (NIH), there are nearly 7,000 rare diseases affecting nearly 30 million Americans. Rare Disease Day was established by EURORDIS and was first observed in Europe in 2008. In 2009, EURORDIS partnered with NORD for this initiative in the U.S. For more information about Rare Disease Day, visit www.rarediseaseday.us or www.rarediseaseday.org. About Novelion Therapeutics Novelion Therapeutics is a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases. The company seeks to advance its portfolio of rare disease therapies by investing in science and clinical development. Novelion has a diversified commercial portfolio through its indirect subsidiary, Aegerion Pharmaceuticals, Inc., which includes MYALEPT® and JUXTAPID®, and is also developing zuretinol acetate for the potential treatment of inherited retinal disease caused by underlying mutations in RPE65 or LRAT genes.


News Article | February 28, 2017
Site: globenewswire.com

CAMBRIDGE, Mass., Feb. 28, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, announced the company has joined forces with 30 million Americans and health care advocates around the world today for Rare Disease Day®. Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases. This year’s theme focuses on research and the critical role it plays in identifying, diagnosing, and ultimately treating rare diseases. “At Editas Medicine, we have the bold vision to unlock the potential of CRISPR to design and develop genome editing therapies for patients suffering from genetically-defined and genetically-treatable diseases, including rare diseases,” said Katrine Bosley, President and Chief Executive Officer of Editas Medicine. “It’s a privilege to join organizations like EURORDIS and NORD, as well as our colleagues within the industry, to raise awareness of the importance of research, funding, and partnerships for the millions of people living with rare diseases across the world and their families.” CRISPR is a dynamic, versatile tool that can be programmed to target specific stretches of genetic code and edit DNA at precise locations in the human genome. The technology allows researchers to permanently modify genes and has the potential to create medicines with a durable treatment effect following a single dose. Editas Medicine is currently focused on using its CRISPR technology to treat diseases for which there are few or no available treatments, including a rare inherited eye disease called Leber congenital amaurosis type 10, or LCA10, that appears at birth or in the first few months of life and leads to significant vision loss. According to the National Institutes of Health (NIH), nearly one in 10 Americans lives with a rare disease—collectively affecting 30 million people—and nearly half of these patients are children. There are approximately 6,000 rare diseases, 95 percent of which have no FDA-approved therapies. Rare Disease Day was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. About Editas Medicine Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients. Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption "Risk Factors" included in the Company's most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


News Article | February 27, 2017
Site: globenewswire.com

LEIDEN, the Netherlands, Feb. 27, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases including cystic fibrosis (CF), Leber's congenital amaurosis Type 10 (LCA 10) and dystrophic epidermolysis bullosa (DEB), today announced its support of Rare Disease Day, joining rare disease patients and families, patient organizations and health care providers to raise awareness of rare diseases. Together with members of the rare disease community, ProQR is proud to participate at a Rare Disease Day Lunch Symposium, organized by Leiden Bio Science Park Foundation (NL) to highlight the importance of research in rare diseases and recognize the crucial role that patients play in it. Every last day of February, Rare Disease Day is an international awareness campaign led by the patient organization EURORDIS and supported by hundreds of other patient organizations around the world. February 28, 2017 marks the tenth Rare Disease Day and this year’s slogan 'With research, possibilities are limitless’ underlines the hope that research brings to the millions of people living with a rare disease around the world and their families. “It is our mission to make a meaningful impact on the lives of patients that suffer from severe genetic rare diseases and therefore we are honored to be part of this event on Rare Disease Day to raise awareness and emphasize the importance of research,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Together with our partners at the Leiden Bio Science Park and many others around the world we are inspired by the stories of people affected by rare diseases and hope many more will join us in our quest to find treatments for all of them.” CF is the most common fatal inherited disease in the Western world and affects an estimated 65,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick, buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 27 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 70% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation. Leber’s congenital amaurosis is the most common cause of blindness in children and consists of a group of diseases of which LCA Type 10 (LCA 10) is one of the more severe forms. LCA 10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is most common. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA 10 because of this mutation. Dystrophic epidermolysis bullosa (DEB) is a rare blistering disorder of the skin and mucosal membranes and is characterized by extremely fragile skin that blisters and tears from minor friction or trauma leaving poorly healing wounds. DEB is painful and debilitating and is associated with very low quality of life and limited life expectancy. The disease is caused by mutations in the COL7A1 gene that lead to a very weak connection between the dermis (inner layer) and the epidermis (outer layer) in the skin. Approximately 2,000 patients have DEB because of mutations in exon 73 of the COL7A1 gene. There is currently no treatment available. 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 Rare Disease Day and our mission to pursue treatments for rare diseases. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. 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.


News Article | February 28, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sanofi Genzyme, the specialty care global business unit of Sanofi, today announced its support for Rare Disease Day with events and activities around the world. Observed annually on the last day of February, Rare Disease Day is an international awareness campaign established by the European Organisation for Rare Diseases (EURORDIS) in 2008, and supported by hundreds of other patient organizations. Rare Disease Day provides an important opportunity to shed light on the impact that these frequently debilitating diseases have on patients and their families. Given their rarity, these conditions are often difficult to diagnose and treat. Patients can endure a long odyssey involving many specialty doctors, tests, and numerous misdiagnoses. “ Patients with rare diseases often go years or even decades without a correct diagnosis,” said North America Head of Rare Diseases, Yann Mazabraud. “ Sanofi Genzyme is dedicated to working together with the patient, physician and research community to shorten the journey to diagnosis, through support of focused education, testing and diagnostic initiatives. Our vision is that by 2025, all patients with the rare diseases that we serve will have access to an early diagnosis and appropriate treatment.” This year, Sanofi Genzyme launched a global initiative called Pledge 4 Rare, aimed at bringing together the global rare disease community to raise awareness of the more than 7,000 different rare diseases collectively affecting more than 350 million people around the world. The campaign encourages people to pledge to run or walk a mile or more on Rare Disease Day in support of the rare disease community, and to share photos and videos on social media using the hashtag #Pledge4Rare. Follow the conversation on Sanofi Genzyme’s Twitter and Facebook channels. In addition to the global initiative, Rare Disease Day 2017 is being recognized with a program of events at Sanofi Genzyme sites around the world including: About Sanofi Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organized into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Consumer Healthcare. Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families. Sanofi Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi's ability to benefit from external growth opportunities and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic conditions, the impact of cost containment initiatives and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2015. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

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