Long-term follow-up of 16 boys treated with gene therapy to correct the immune deficiencies ADA-SCID and X-SCID in a British study has shown that despite some heartbreaking setbacks along the way, overall, the procedure can be considered a success. Read More
WASHINGTON – Orphan drug rules intended to encourage the development of treatments for rare diseases are creating an uneven playing field for follow-on biologics and personalized medicine. Read More
The FDA granted Pharmasset Inc., of Princeton, N.J., fast-track designation for PSI-938 for the treatment of chronic hepatitis C virus (HCV) infection. PSI-938 is an oral guanosine nucleotide analogue polymerase inhibitor of HCV. Pharmasset plans to initiate an interferon-free combination trial with PSI-938 and PSI-7977, an uracil nucleotide analogue, in the third quarter. In an earlier study, PSI-938 demonstrated potent antiviral activity, both as monotherapy and in combination with PSI-7977. Read More
Zogenix Inc., of San Diego, filed a registration statement to raise funding through the public offering of 12 million shares – 13.8 million if underwriters exercise their full overallotment option. Based on Monday's closing price of $3.40 per share, the company anticipates net proceeds of about $37.7 million – or $43.5 million with overallotments – to support submission of a new drug application for Zohydro, other clinical and commercialization activities and for general corporate purposes. Read More
Peregrine Pharmaceuticals Inc., of Tustin, Calif., reported that bavituximab in combination with docetaxel in patients with locally advanced or metastatic breast cancer demonstrated a 20.7 month median overall survival (OS) in a single-arm Phase II trial. Read More
Investors of United Therapeutics Corp. experienced a bit of déjà vu Wednesday as shares took a tumble on disappointing data from a Phase III study testing oral treprostinil on top of background therapy in patients with pulmonary arterial hypertension (PAH). Read More
LONDON – The scientists behind KuDOS Pharmaceuticals Ltd., which was sold to London-based AstraZeneca plc in December 2005 for £121.5 million (then US$210 million), have left the company to form another start-up, Mission Therapeutics Ltd. Read More
