SOURCE: Horizon Pharma plc Phase 2 Clinical Study in Friedreich's Ataxia Patients Shows Strong Efficacy Signal; Management Estimates Peak Net Sales Potential for ACTIMMUNE of $500 Million With FA Approval KING OF PRUSSIA, PA--(Marketwired - Oct 13, 2014) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, is hosting analysts and investors this evening to discuss the opportunities for ACTIMMUNE (interferon gamma-1b).ACTIMMUNE is currently approved by the U.S. Food and Drug Administration (FDA) to treat two ultra-rare orphan diseases, Chronic Granulomatous Disease (CDG) and severe, malignant osteopetrosis (SMO). The meeting is being conducted in King of Prussia, PA to coincide with the Children's Hospital of Philadelphia (CHOP) 7th Annual Friedreich's Ataxia (FA) Symposium.The results from the Phase 2 clinical trial, sponsored by Friedreich's Ataxia Research Alliance (FARA) and conducted by the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA), were presented at the CHOP event and are being discussed at the Horizon analyst day. Highlights from the Horizon Pharma meeting include: "We are committed to helping patients and physicians identify the early signs and symptoms of CGD and SMO to ensure patients receive more effective treatment … Continue reading →

Published 14 October 2014 Concert Pharmaceuticals has announced Phase 1 data of CTP-354, a novel, potentially first-in-class, non-sedating, once-daily oral treatment for spasticity. In this multiple ascending dose trial, no sedation or ataxia was observed with CTP-354 and the drug was generally well tolerated across all dose cohorts. The Company will be presenting these findings today during a poster session at the American Neurological Association's Annual Meeting in Baltimore, Maryland. Concert expects to initiate a Phase 2 clinical trial evaluating CTP-354 in patients with spasticity associated with spinal cord injury by the end of 2014. "We are very pleased to have completed our clinical evaluation of CTP-354 in this Phase 1 trial. We remain on track to advance the program into Phase 2 testing later this year, initially targeting spasticity in patients with spinal cord injury followed by the start of a Phase 2 trial in multiple sclerosis patients in early 2015," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. Dr. Tung added, "CTP-354 was designed to avoid dose-limiting sedation and ataxia, which currently limit the broader use of other GABAA receptor modulators such as benzodiazepines. This trial provides evidence that CTP-354 can achieve high plasma … Continue reading →

Health care stocks were posting moderate gains, with the NYSE Health Care Sector Index ahead about 0.7% and shares of health care companies in the S&P 500 rising 0.5% as a group. In company news, Horizon Pharma ( HZNP ) was narrowly lower Tuesday afternoon despite announcing late Monday positive results from Phase II testing of its Actimmune interferon gamma-1b medication in children with Friedreich's ataxia, finding the drug was well tolerated with no serious adverse events. HZNP shares recently were down about 0.6% at $11.90 apiece, giving back a 4.6% rise to $11.46 a share early in Tuesday's trading. The stock has traded within a 52-week range of $3.69 to $18.30 a share, rising slightly more than 192% over the past 12 months through Monday's close. The disease is a debilitating, degenerative neuro-muscular disorder affecting around 4,000 to 5,000 children and adults in the U.S. and another 10,000 individuals elsewhere around the globe, according to the Friedreich's Ataxia Research Alliance. There currently is no approved FDA treatment for the disease. The trial was conducted at Children's Hospital of Philadelphia starting in August 2013 with 12 children ages five to 17 years old and with release of the results timed … Continue reading →

Top Healthcare Stocks JNJ -0.15% PFE +0.79% ABT +2.37% MRK +1.21% AMGN +0.34% Health care stocks were posting moderate gains, with the NYSE Health Care Sector Index ahead about 0.7% and shares of health care companies in the S&P 500 rising 0.5% as a group. In company news, Horizon Pharma ( HZNP ) was narrowly lower Tuesday afternoon despite announcing late Monday positive results from Phase II testing of its Actimmune interferon gamma-1b medication in children with Friedreich's ataxia, finding the drug was well tolerated with no serious adverse events. HZNP shares recently were down about 0.3% at $11.93 apiece, giving back a 4.6% rise to $11.46 a share early in Tuesday's trading. The stock has traded within a 52-week range of $3.69 to $18.30 a share, rising about slightly more than 192% over the past 12 months through Monday's close. The disease is a debilitating, degenerative neuro-muscular disorder affecting around 4,000 to 5,000 children and adults in the U.S. and another 10,000 individuals elsewhere around the globe, according to the Friedreich's Ataxia Research Alliance. There currently is no approved FDA treatment for the disease. Read more: Sector Update: Smaller Rise for Health Care Stocks Today; Horizon Pharma Turns Lower … Continue reading →

PUBLIC RELEASE DATE: 14-Oct-2014 Contact: Jim Fessenden james.fessenden@umassmed.edu 508-856-2688 University of Massachusetts Medical School @UMassMedNow WORCESTER, MA A team of scientists led by researchers at the University of Massachusetts Medical School (UMMS) and the University of Miami Miller School of Medicine (UMMSM) have identified what is likely a key genetic pathway underlying bipolar (manic depressive) disorder, a breakthrough that could lead to better drugs for treating bipolar affective disorder, as well as depression and other related mood disorders. The new findings, published online this week in Nature Molecular Psychiatry, show that a rare genetic dwarfism called Ellis van-Creveld (EvC) syndrome protects against bipolar affective disorder. The discovery was made thanks to decades of translational research in a few Old Order Amish families of Pennsylvania with a high incidence of both diseases. Forty years of documented research across multiple generations showed that no person with EvC has been reported with bipolar disorder. "No one doubts that bipolar affective disorder has an important, disease causing genetic component," said neurologist and geneticist Edward I. Ginns, MD, PhD, professor of psychiatry at UMMS and lead author of the study. "In our search for the causes of bipolar affective disorder, this is a paradigm changing … Continue reading →

PUBLIC RELEASE DATE: 13-Oct-2014 Contact: Rachel Seroka rseroka@aan.com 612-928-6129 American Academy of Neurology @GreenJournal MINNEAPOLIS A new guideline from the American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) recommends guidance on how doctors should evaluate the full picturefrom symptoms, family history and ethnicity to a physical exam and certain lab test resultsin order to determine what genetic tests may best diagnose a person's subtype of limb-girdle or distal muscular dystrophy. The guideline is published in the October 14, 2014, print issue of Neurology, the medical journal of the American Academy of Neurology. To develop the guideline, researchers reviewed all of the available studies on the disorders, which cause muscles to waste away. "These are rare muscle diseases that can be difficult to diagnose," said guideline lead author Pushpa Narayanaswami, MD, of Harvard Medical School in Boston and a Fellow of the AAN and AANEM. "With an accurate diagnosis, unnecessary tests or treatments may be avoided. Knowing the specific subtype is important for getting the best possible care." "Limb girdle" refers to the hip and shoulder areas, where the limbs attach to the body. Limb-girdle muscular dystrophy most affects muscles close to the center … Continue reading →

New technologies, and a little help from the U.S. Supreme Court, have made it possible for large numbers of women to find out whether they carry genetic mutations that increase their risk of breast cancer - a development warmly welcomed by experts in the field. But the availability and relative affordability of multigene-panel tests can also lead to anxiety and confusion about what course of action to choose, because the risk associated with many of those genes remains unknown. "Genetic testing holds a lot of potential and a whole lot of uncertainty," said Beth Peshkin, a professor of oncology and senior genetic counselor at Georgetown Universitys Lombardi Comprehensive Cancer Center in Washington. "The more genes we test, the more variants were likely to find," explained Peshkin. "A recent study found that about 40 percent of people who underwent panel testing had variants, or genetic changes, that we dont know how to interpret." In 2013 the Supreme Court invalidated Myriad Genetics patents on the two major genes that predispose women to breast and ovarian cancer, ruling that human genes cannot be patented. Since then, several companies have begun testing for mutations in those genes, BRCA1 and BRCA2, which are responsible for … Continue reading →