Phase I
An independent Data Safety Monitoring Board (DSMB) recommended that Titan Pharmaceuticals’ Phase I/II trial of its ropinirole implant for Parkinson’s disease continue with a second cohort of patients. However, the company has decided to temporarily halt the study and focus on another asset.
Ebola cases, as well as cases of other significantly lethal viral outbreaks that have occurred has spurred researchers to focus on the development of antivirals that can treat the illnesses.
Miracles, it seems, have a high price tag. At least, if those miracles are miracle drugs. There’s no doubt that trends in gene therapy and immuno-oncology are producing drugs that are as close to miraculous as we’re likely to get, doing a great job, generally, in beating back diseases that to this point were untreatable or didn’t respond well to other therapies.
The biopharma industry generally views vaccines to be low-margin investments. From a public health perspective, however, vaccines can be one of the best investment dollars.
This morning two companies announced deals that will yield potential benefits to multiple patient populations.
Shares of Incyte Corporation are up slightly this morning after the company started off its annual R&D day with positive news from its pivotal REACH1 study of Jakafi (ruxolitinib) in steroid-refractory acute graft-versus-host disease (GVHD) and sets up the filing of a supplemental New Drug Application.
Sarepta Therapeutics shares rocketed by as much as 80 percent yesterday after the company released interim data on its experimental gene therapy AAVrh74.MHCK7.micro-Dystrophin for Duchenne muscular dystrophy (DMD).
Chinese companies and investors continue to flex their financial muscles with a series of deals this week that highlight the continued growing impact that country is having on the global pharmaceutical and biotech landscape.
PTC Therapeutics presented updated interim clinical data from Part 1 of its FIREFISH clinical trial of risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy.
The U.S. Food and Drug Administration (FDA) placed a clinical hold on Boston-based Ziopharm Oncology’s Phase I trial of its Sleeping Beauty CAR-T therapy.
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