NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.
Ultragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
BrainCells Inc. Announces the Successful Completion of the Single Ascending Dose Study With BCI-838 and the Initiation of the Multiple Ascending Dose Study
SAN DIEGO, Feb. 29, 2012 /PRNewswire/ -- BrainCells Inc., a leading biotechnology company developing novel compounds for the treatment of central nervous system (CNS) diseases, announced today that the Phase 1 single ascending dose (SAD) study of BCI-838 has been successfully completed and that the company has initiated the Phase 1 multiple ascending dose (MAD) study. The SAD study evaluated BCI-838 for safety, tolerability, pharmacokinetics and food effect in healthy male subjects. Single oral doses of BCI-838 up to 900 mg were administered and the drug was well tolerated. No serious adverse events were reported and all adverse events were mild in intensity, transient, and resolved without sequelae.
ACH-1625 Receives Fast Track Designation From the FDA for the Treatment of Chronic Hepatitis C
NEW HAVEN, Conn., Jan. 4, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a leader in the discovery and development of small molecule drugs to combat the most challenging infectious diseases, announced today the receipt of a Fast Track designation from the U.S. Food and Drug Administration (FDA) for ACH-1625 for the treatment of chronic hepatitis C virus (HCV). ACH-1625 is a once-daily protease inhibitor with broad genotypic coverage against HCV that was discovered by Achillion and is currently being evaluated in a Phase 2 clinical trial.
Marina Biotech, Inc. (Formerly Known as MDRNA, Inc.) and Mirna Therapeutics, Inc. Announce License Agreement for Up to $63 Million for the Development
BOTHELL, WA and AUSTIN, TX--(Marketwire - December 23, 2011) - Marina Biotech, Inc. (NASDAQ: MRNA), a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc. (Mirna), a privately-held biotechnology company pioneering microRNA (miRNA) replacement therapy for cancer, announced today that they have entered into a license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna's proprietary microRNAs and Marina Biotech's novel SMARTICLES liposomal delivery technology. Mirna will have full responsibility for the development and commercialization of any products arising under the Agreement and Marina Biotech will support pre-clinical and process development efforts. Under terms of the Agreement, Marina Biotech could receive up to $63 million in total upfront, clinical and commercialization milestone payments, as well as royalties on sales, based on the successful outcome of the collaboration. Further terms of the Agreement were not disclosed.
Anthera Pharmaceuticals Completes Interim B-Cell Analysis of PEARL-SC Study
HAYWARD, Calif., Dec. 1, 2011 /PRNewswire/ -- Anthera Pharmaceuticals, Inc. (Nasdaq: ANTH), a biopharmaceutical company developing drugs to treat serious diseases associated with inflammation, today announced positive interim biomarker data from the PEARL-SC phase 2b clinical study in patients with systemic lupus erythematosus.
After analysis by an independent statistician, data from the on-going PEARL-SC study indicate that weekly and monthly subcutaneous doses of blisibimod resulted in statistically significant reductions of B-cells. Elevations in these B-cells have been associated with an increased risk of disease activity in lupus patients. These findings are consistent with data from previous clinical studies of blisibimod. The company remains blinded to primary efficacy data.
Anthera Pharmaceuticals, Inc. (ANTH) Announces Last Patient Enrolled in PEARL-SC Study
HAYWARD, Calif., Oct. 25, 2011 /PRNewswire/ -- Anthera Pharmaceuticals, Inc. (NASDAQ: ANTH), a biopharmaceutical company developing drugs to treat serious diseases associated with inflammation, today announced it has completed enrollment in the Phase 2b PEARL-SC study. PEARL-SC (A randomized, double-blind Phase 2b study to evaluate the efficacy, safety, and tolerability of blisibimod administration in subjects with systemic lupus erythematosus) is examining the therapeutic benefit of weekly and monthly subcutaneous injections of blisibimod in patients with active and antibody positive systemic lupus erythematosus (SLE). The targeted number of five hundred and forty (540) patients has been randomized in 11 countries and 72 clinical sites worldwide.
Chimerix Announces Late-Breaker Presentation At 51st Interscience Conference On Antimicrobial Agents And Chemotherapy (ICAAC) Annual Meeting
DURHAM, N.C., Sept. 16, 2011 /PRNewswire/ -- Chimerix, Inc., a biotechnology company developing orally-available antiviral therapeutics, announced today that investigators will present preliminary data for CMX001 in a late-breaker presentation at the 51st Annual Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC, Chicago – September 17-20).
Syndax Pharmaceutical's Positive Phase 2 Data Supports Potential For Entinostat In Advanced Breast Cancer
WALTHAM, Mass., Sept. 6, 2011 /PRNewswire/ -- Syndax Pharmaceuticals, Inc., a clinical-stage epigenetics oncology company, announced today that ENCORE 301, a randomized, placebo-controlled phase 2 study of exemestane with and without entinostat hit its primary endpoint of an improvement in progression-free survival (PFS). The study showed that patients who received entinostat, a novel, oral small molecule inhibitor of class I histone deacetylases, with the hormone therapy exemestane, lived longer without their disease getting worse than people who received exemestane alone. Safety and efficacy results from the trial will be presented in a poster and an oral presentation at the American Society of Clinical Oncology (ASCO) Breast Cancer Symposium 2011 in San Francisco, CA this week.
CoLucid Pharmaceuticals Raises $7.5 Million of Planned $9.5 Million
DURHAM, N.C., Aug. 30, 2011 /PRNewswire-iReach/ -- CoLucid Pharmaceuticals, Inc., a privately held biopharmaceutical company, announced today that it has raised $7.5 million of a planned $9.5 million convertible note financing from its existing investors to support further late stage development of lasmiditan. Participating in the financing were Pappas Ventures, Domain Associates, Care Capital, Pearl Street Venture Funds and Triathlon Medical Ventures.
Achillion Initiates 12-Week Dosing in Phase 2 Trial of ACH-1625 for the Treatment of Chronic Hepatitis C
NEW HAVEN, Conn., June 22, 2011 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced that the Company has initiated patient dosing in segment 2 of its Phase 2 clinical trial of ACH-1625 for the treatment of hepatitis C virus (HCV) for genotype 1 treatment naïve HCV-infected patients. ACH-1625, discovered and advanced by Achillion, is a potent small molecule inhibitor of HCV protease, an enzyme necessary for viral replication.
