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.
Ultragenyx selected as 2013 Fierce 15 Company
FierceBiotech selected Ultragenyx Pharmaceutical as one of the 2013 Fierce 15 Companies.
What makes Ultragenyx fierce: The most recent addition to the pipeline is KRN23, an antibody designed to treat rare cases of X-linked hypophosphatemia that is now wrapping a Phase I/II study at Kyowa Hakko Kirin. At the time KRN23 showed up on Ultragenyx's radar, it was the only rare-disease program at the Japanese pharma company, says CEO Emil Kakkis. When the deal was done just days ago, it was Ultragenyx's fourth midstage therapy.
Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Treatment with triheptanoin appears to reduce the frequency and length of hospitalizations associated with LC-FAOD disease.
NOVATO, CA –September 16, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of positive data from a retrospective protocol-driven medical record review oftriheptanoin treatment of patients with long-chain fatty acid oxidation disorders(LC-FAOD). LC-FAODpatients have severe morbidity and mortality with frequent complications and hospitalizations. The study evaluated the impact of triheptanoin treatment on the rate and extent of hospitalizationsin 20 of 24 patients who have been treated with triheptanoin for up to 13 years as part of a compassionate use protocol and consented to be part of the retrospective study. The study compared the incident rate for the major medical events before and after triheptanoin treatment, including the total number of hospitalizations and hospital days per year due to all causes,muscle rupture (rhabdomyolysis), hypoglycemia, or cardiomyopathy.
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.
Chimerix Initiates Phase 3 SUPPRESS Trial of Brincidofovir (CMX001) for Prevention of CMV in HCT Recipients
DURHAM, N.C., Sept. 9, 2013 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced initiation of dosing in the Phase 3 SUPPRESS trial (ClinicalTrials.gov ID: NCT01769170). SUPPRESS is evaluating brincidofovir (CMX001) for the prevention of cytomegalovirus (CMV) infection, the most significant infectious disease in hematopoietic cell transplant (HCT) recipients. Brincidofovir is an investigational oral nucleotide analog lipid-conjugate that has demonstrated activity against all pathogenic double-stranded DNA (dsDNA) viruses, including herpesviruses, adenoviruses, and polyomaviruses.
LipoScience Announces New Data Supporting High-Density Lipoprotein Particle (HDL-P) Number As Marker Of Cardiovascular Risk
JUPITER study data published in Circulation, suggest HDL-P may be a better marker of residual risk than HDL cholesterol or apolipoprotein A-1 in patients on statin therapy
RALEIGH, N.C., Sept. 4, 2013 /PRNewswire/ -- LipoScience, Inc., (NASDAQ: LPDX) a diagnostic company pioneering a new field of personalized nuclear magnetic resonance (NMR) diagnostics to advance the quality of patient care in cardiovascular, metabolic and other diseases, today announced the publication of data supporting the measurement of high-density lipoprotein particle (HDL-P) number as a marker of cardiovascular risk in patients on cholesterol-lowering statin therapy. The JUPITER data analysis appeared September 3, 2013 in the online version of the journal Circulation in advance of the September 10, 2013 print issue.
Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia
Exclusive partnership will advance global clinical development of
KRN23, an antibody for a rare bone disease
Novato, CA – September 3, 2013--Ultragenyx Pharmaceutical Inc.today announced it has entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK),to develop and commercialize KRN23. KRN23 is a recombinant fully human monoclonal IgG1 antibody intended to treat X-linked hypophosphatemia (XLH). KHK is currently completing a Phase 1/2 study in adults with XLH in the US and Canada. The two companies plan to initiate a pediatric XLH program in 2014.
Syndax Pharmaceuticals Secures $26.6 Million Series B Financing for Advancement of Epigenetic Therapies for Treatment-Resistant Cancers
Company Prepares for Pivotal Phase 3 Study of Entinostat, Most Advanced HDAC Inhibitor in Development for ER+ Metastatic Breast Cancer
WALTHAM, Mass., Aug. 27, 2013 — Syndax Pharmaceuticals Inc., which is developing epigenetic therapies for treatment-resistant cancers, today announced it has secured a $26.6 million Series B financing. Participating investors included Domain Associates, MPM Capital, Forward Ventures and RusnanoMedInvest (RMI).
