Genzyme: FDA will decide Lumizyme’s fate in June

The FDA has told beleaguered Genzyme that a new decision on Lumizyme, the company's drug for Pompe disease, will come in June--more than six months after the agency rejected the product for the second time. The agency rejected the drug last March and then again in November. The second rejection was related to problems with the Allston-based manufacturing plant that produced it. Because of the production problems, Genzyme shifted production of the drug to a Belgium plant, Dow Jones reports....

PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy

SOUTH PLAINFIELD, N.J., Jan. 19 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to...

TD2 and Critical Outcome Technologies develop novel anti-cancer drug

TGen Drug Development (TD2) and Critical Outcome Technologies Inc. (COTI) announced today that they will work together to obtain approval of clinical trials for a promising new anti-cancer drug called...

Spherix Completes Enrollment in NEET, a Phase 3 Clinical Trial Evaluating an Oral Drug for Type 2 Diabetes

BETHESDA, Md., Jan. 12 /PRNewswire-FirstCall/ -- Spherix Inc. (Nasdaq SPEX), an innovator in biotechnology for diabetes therapy, and a provider of technical and regulatory consulting services to food, supplement, biotechnology and pharmaceutical companies, today announced the completion of patient...

Targacept gets $200M payment from AstraZeneca

Targacept has received a hefty $200 million milestone payment from AstraZeneca. The money is part of a deal signed in December that could be worth up to $1.2 billion. Targacept is developing TC-5214, a drug for major depressive disorder (MDD), which is headed into Phase III trials this year. The milestone payment was triggered by the early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Targacept and AstraZeneca hope to file an NDA for TC-5214 as a...

Galapagos, Roche enter $588.7M COPD alliance

Galapagos and Roche have entered a global alliance to develop new COPD therapies in a deal worth almost $590 million (€406 million). Galapagos will use its discovery platform to identify potential COPD targets and develop small molecule candidates against the targets. Roche will select the candidates it wants to develop after either clinical candidate selection or completion of Phase I trials. The company will then be fully responsible for further development of the drugs it licenses....

Sanofi touts dengue fever vaccine data

In a study published in the Journal of Infectious Diseases, Sanofi-Aventis reports that all 66 subjects enrolled in a trial of its dengue fever vaccine were protected from the disease after receiving three injections over 15 months. And many were protected in just two shots--even patients whose first injection was a placebo. Sanofi says the results "paved the way for ongoing and future large-scale efficacy trials of our...

Quark Pharmaceuticals Poised to Advance Clinical Studies of QPI-1002

Quark Pharmaceuticals, Inc., the world leader in clinical development of RNAi-based therapeutics, announced today that the independent Data Safety Monitoring Board ("DSMB") recommended that QPI-1002 (I5NP) continue on to the next phase of clinical testing. This followed review of available safety data from two clinical trials for Quark's siRNA QPI-1002, at doses up to 10 mg/kg, the highest evaluated to date. QPI-1002 is designed to temporarily inhibit expression of the stress-response gene,...

CLINICAL RESEARCH TRENDS AND TECHNOLOGY FORUM: For the Patient’s Sake, Standardize your Research Processes

NEW YORK, Jan. 4 /PRNewswire/ -- The Clinical Research Trends and Technology Forum (CRT&T) presents a unique approach to solving the issues presented to pharmaceutical executives. CRT&T blends educational keynotes and roundtables focusing on standards of care, quality processes and workflow with exposure to the newest and most innovative technologies on the market through one-on-one business meetings and networking...

Pearl Therapeutics Announces Positive Results From Phase 2a Trial of Formoterol Inhalation Aerosol in Chronic Obstructive Pulmonary Disease

Pearl Therapeutics Inc., a company developing high-quality combination therapies for the treatment of highly prevalent chronic respiratory diseases, today announced positive results from its first clinical trial in patients with chronic obstructive pulmonary disease (COPD). Results from a Phase 2a dose-ranging study of PT005, the company's formoterol fumarate hydrofluoroalkane metered dose inhaler (HFA-MDI) formulation, showed that it was well tolerated with bronchodilator efficacy and safety...