KRN23 increased phosphate levels and was safe and well tolerated
NOVATO, CA – October 6, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of data from a first-in-human, randomized, double-blind, placebo-controlled, single dose study of a human monoclonal anti-FGF23 antibody (KRN23) in X-linked hypophosphatemia in adults. X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease characterized by short stature and skeletal deformities. A Phase I study (US-02) was conducted by KHK with KRN23 to assess its safety and tolerability and to measure changes in biochemical markers in adult patients with XLH. Thirty-eight adults with XLH were randomized to receive single doses of KRN23 or placebo via intravenous (IV) (0.003 to 0.3 mg/kg) or subcutaneous (SC) (0.1 to 1.0 mg/kg) routes.
Ultragenyx Announces the Presentation of Data from a Single Dose Phase 1 Study, Conducted by Kyowa Hakko Kirin Co. Ltd. (KHK), of KRN23 in X-linked Hypophosphatemia (XLH) in Adults
Chimerix Announces Publication of Positive Phase 2 Results of Brincidofovir (CMX001) in the New England Journal of Medicine
Phase 2 Trial Evaluated Brincidofovir for the Prevention of Cytomegalovirus (CMV) Infection in Hematopoietic Cell Transplant Recipients
DURHAM, N.C., Sept. 26, 2013 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced the publication of results from its Phase 2 Study CMX001-201 evaluating brincidofovir (CMX001) for the prevention of cytomegalovirus (CMV) infection in hematopoietic cell transplant (HCT) recipients. The article, entitled "CMX001 to Prevent Cytomegalovirus Disease in Hematopoietic-Cell Transplantation," appears in the September 26th issue of the New England Journal of Medicine (N Engl J Med 369:1227-36).
Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress
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.
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.
