The requests Sarepta Therapeutics (SRPT) has gotten from the FDA represent a sign of FDA seriousness towards bringing the firm’s drug eteplirsen for Duchenne muscular dystrophy as soon as possible to the children suffering from the debilitating, fast deteriorating disease. The FDA did not wait for the firm to file for early approval, it rather asked Sarepta to bring in more data that would legitimize granting early approval of the first drug ever to claim treating the disabling disease at its root-origin. Investors who should have been optimistic about the FDA initiative sold their shares instead, causing them to lose around 10% of their value. More...
News has come from Northwest Biotherapeutics (NWBO) on its DCVax® personalized immune therapies for solid tumor cancers. The information is an announcement that Phase 1/2 DCVax-Direct clinical trial for all inoperable solid tumor cancers, which is planned to begin within approximately the next sixty days, will not be blinded. This means that the results will be seen as the trial proceeds. Knowing that an efficacy endpoint is tumor shrinkage or elimination), this trial is expected to yield meaningful ongoing results by the second half of 2013. More...
Forgetting about Idera Pharmaceuticals (IDRA) seems to make its value sink, but failed to make it disappear. With a stock that sells at less than a three quarter of a dollar and a market of $20 million, lower than a new comer biotech having drugs still in preclinical lab testing, the firm was capable of drawing investors’ attention, riding new data about an attractive possibility of beating all existing approaches to treating psoriasis. The surprising data, which bring hopes in treating other autoimmune diseases, in addition to psoriasis were presented by James G. Krueger, M.D., PhD, of The Rockefeller University, at the Late-Breaking Research Symposium on March 2nd, 2013, during the American Academy of Dermatology Annual Meeting. More...
XOMA (XOMA) is addictive and its relationship to investors is typical of that of married couples who have a love/hate alternating history, which is interrupted with divorces and remarrying. Around a week ago, the firm announced interim data from a phase 2 proof-of-concept (POC) study on gevokizumab, which is being developed for the treatment of inflammatory facial lesions in patients suffering from moderate to severe acne vulgaris. More...
Using scientific skills towards building pipelines of promising products does not guarantee a happy ending for a small biotech firm. Pipelines’ breakthroughs need managements’ capabilities and swiftness of actions, which would walk them through the safest and fastest road towards approvals. We believe Theravance (THRX) has benefited from both its scientists and management. The firm has come nearer than ever to reaching a happy beginning of substantial growth, or perhaps, a happy ending through acquisition, probably, by its partner GlaxoSmithKline (GSK). More...