NOVATO, Calif., Sep 26, 2013 (GLOBE NEWSWIRE via COMTEX) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that three abstracts related to UX001 Sialic Acid for Hereditary Inclusion Body Myopathy, also known as GNE Myopathy, were accepted for poster presentation at the 18th Annual World Muscle Society Congress, October 1-5, 2013 in Asilomar, California.
Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress
Lumena Pharmaceuticals Receives Orphan Drug Designation from US Food & Drug Administration for LUM001 in Four Rare Liver Diseases
Company Pursuing Orphan Path to Development in Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis, Primary Biliary Cirrhosis and Primary Sclerosing Cholangitis
SAN DIEGO – September 26, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development for LUM001, the company’s lead drug candidate. LUM001 received Orphan Designation in four rare cholestatic liver diseases including Alagille syndrome (ALGS); progressive familial intrahepatic cholestasis (PFIC); primary biliary cirrhosis (PBC); and primary sclerosing cholangitis (PSC).
Lumena Pharmaceuticals Launches Global Clinical Program to Evaluate Safety and Efficacy of LUM001 in Children with Alagille Syndrome
First patient dosed in U.K. in Phase II IMAGO Study; Phase II ITCH Study to start in U.S.
Cooperative Research and Development Agreement established with the National Institutes of Health and the Childhood Liver Disease Research and Education Network (ChiLDREN)
SAN DIEGO – September 26, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome (ALGS). The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S.
Chimerix Presents Brincidofovir (CMX001) Adenovirus Phase 2 Results
Positive Phase 2 Study Results Presented as Late Breaker at ICAAC
DURHAM, NC, September 11, 2013 – Chimerix, Inc. (NASDAQ: CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced the results from its exploratory Phase 2 Study 202 evaluating brincidofovir (CMX001) in hematopoietic cell transplant (HCT) recipients with early adenovirus (AdV) infection. Study 202 was the first trial of an antiviral agent in AdV infection. Brincidofovir (CMX001) is an investigational oral nucleotide analog lipid-conjugate that has demonstrated activity against all pathogenic families of double-stranded DNA (dsDNA) viruses, including herpesviruses, adenoviruses, and polyomaviruses.
Liquidia Technologies Announces Extension of Collaboration with PATH to Develop Next Generation Pneumococcal Vaccine
RESEARCH TRIANGLE PARK, NC– May 21, 2013 – Liquidia Technologies today announced that its collaborative agreement with PATH, a global health nonprofit organization, has been extended. The extension of this agreement allows for the continuation of preclinical proof-of-concept studies on a next generation pneumococcal vaccine. If successful, the vaccine could potentially allow for broadened efficacy and manufacturing efficiencies that translate to greater access to the vaccine by the global population. Pneumonia kills approximately 1.3 million children under the age of five years every year—more than any other illness. Nearlyhalf of these deaths are caused by Streptococcus pneumoniae, a bacterium that has many variations globally and is becoming increasingly resistant to antibiotics.
“We are exceptionally proud of the work we have done with PATH and excited about the opportunity to continue this work with them,” said Neal Fowler, Chief Executive Officer at Liquidia Technologies. “Our shared goal to improve vaccine performance and reduce manufacturing costs using our transformative technology remains unchanged as does our confidence that this collaboration has the potential to significantly impact the spread of pneumonia in the developing world.”
Art Pappas Elected to NVCA Board of Directors
RESEARCH TRIANGLE PARK, N.C., May 15, 2013 — The National Venture Capital Association (NVCA) today announced the election of Arthur M. Pappas, managing partner of Pappas Ventures, to its board of directors. He will serve a four-year term. The appointment was made official at the association’s annual meeting – VentureScape 2013 – in San Francisco.
Mr. Pappas has over 30 years of operating experience as a pharmaceutical and biotechnology industry executive and venture capital investor in life science companies. He founded Pappas Ventures in 1994, and works with a team that has invested in numerous life science companies that have created novel innovative products addressing unmet medical needs. Prior to forming Pappas Ventures, he held senior level leadership positions at several multinational pharmaceutical companies, for which he led operations and was responsible for the development, licensing and launch of a number of global products.
The NVCA serves as the venture capital community’s trade association, representing more than 400 members by advocating for policies that encourage innovation and reward long term investment.
Independent Study Shows Significant Decline in Cardiac Device Infections with TYRX® Antibacterial Envelope
New Data in High Risk Patients Presented at Heart Rhythm Society
Shows a 70% Reduction in CIED Infections
Denver, CO (May 10, 2013): Use of TYRX, Inc.’s AIGISRx® Antibacterial Envelope significantly reduced device infection rates in patients at high risk for device infection who received cardiovascular implantable electronic device (CIED) implants, according to a study presented today at Heart Rhythm 2013, the Heart Rhythm Society’s 34th Annual Scientific Sessions.
Ultragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders
NOVATO, CA –January 10, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, announced that it has in-licensed rights for triheptanoin, a promising treatment for long-chain fatty acid oxidation disorders (FAOD), from Baylor Research Institute in Dallas, TX. Triheptanoin is intended as a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD and potentially other genetic metabolic disorders.
LIPOSCIENCE COLLABORATES WITH A LEADING ACADEMIC MEDICAL CENTER TO IDENTIFY RISK FACTORS AIMED AT IMPROVING MANAGEMENT OF ACUTE CORONARY SYNDROME
RALEIGH, N.C. – January 09, 2013 – LipoScience, Inc., an in vitro diagnostic company committed to advancing patient care in cardiovascular, metabolic and other diseases, today announced that it has entered into a research agreement with Duke University School of Medicine. The research will be aimed at identifying cardiovascular risk factors that can be used alone or in combination with other clinical
measures to improve stratification and management of acute coronary syndrome. The agreement encompasses studies to be performed with Duke’s CATHGEN (CATHeterizationGENetics) and other patient registries.
Ultragenyx Raises $75 Million in Oversubscribed Crossover Financing
NOVATO, CA – December 20, 2012 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced the successful completion of a $75 million Series B round financing led by Adage Capital Partners, LP. Joining Adage as new investors in this financing are mutual funds and separate account clients advised by T. Rowe Price Associates, Inc., Jennison Associates LLC (on behalf of clients), funds and accounts under management by subsidiaries of BlackRock, Inc., Sanofi-Genzyme BioVentures 1, Shire plc and additional blue chip public market funds. Existing investors, TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction.
