The tone was solemn on Summit Therapeutics plc's conference call early Wednesday as CEO Glyn Edwards briefly restated the Oxford, U.K., firm's terse press release acknowledging failure of ezutromid to meet primary or secondary endpoints following 48 weeks of treatment in the phase II PhaseOut DMD study in individuals with Duchenne muscular dystrophy (DMD). The findings were an unexpected turnabout from interim data suggesting the small-molecule utrophin modulator seemed to help reduce muscle fiber damage and to increase levels of the protein utrophin, linked to the restoration and maintenance of healthy muscle function. Read More
South San Francisco-based Global Blood Therapeutics Inc. (GBT) is discussing pursuit of an FDA accelerated approval for its experimental sickle cell therapy, voxelotor, after meeting the primary endpoint in the first part of its pivotal phase III study, called HOPE. GBT reported statistically significant increases in hemoglobin vs. baseline using two different doses of the drug, a sickle hemoglobin shape modulator. Read More
In May, when Aquinox Pharmaceuticals Inc. signed its potential $155 million deal with Astellas Pharma Inc. for once-daily, oral rosiptor (AQX-1125) for the treatment of interstitial cystitis/bladder pain syndrome (IC/BPS), hopes ran high for the first-in-class SHIP1 activator, which seemed on track to become the first such therapy to reach the market in 20 years. Read More
As widely expected, Array Biopharma Inc.'s combo regimen of an oral, small-molecule BRAF kinase inhibitor and an oral, small-molecule MEK inhibitor cleared the FDA for use in patients with unresectable or metastatic melanoma with BRAFV600E or BRAFV600K mutations, a first approval that could prove only the tip of the iceberg for the Boulder, Colo.-based firm's development of two- and three-drug combos to follow. Read More
While lawmakers and the administration have spent a lot of time over the past few years complaining about the pain of high drug prices in the U.S., those prices are just one symptom of what ails the nation's health care system, members of the Senate Health, Education, Labor and Pensions (HELP) Committee were told Wednesday. Read More
Scientists at the Johns Hopkins Medical School have demonstrated that the experimental glucagon-like peptide 1 (GLP-1) receptor agonist NLY-01 (Neuraly Inc.) was protective against Parkinson's disease (PD) in two separate mouse models. Read More
Is a fourth complete response letter (CRL) in the works for embattled pain drug Remoxy ER? Wall Street seemed to think so, sending shares of developer Pain Therapeutics Inc. (NASDAQ:PTIE) down 71.5 percent to close Wednesday at $2.44, the day after a joint meeting of the FDA's Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee voted 14-3, recommending against approval of the abuse-deterrent, extended-release, oral formulation of opioid drug oxycodone. Read More
Cel-Sci Corp., of Vienna, Va., said, since April 1, it has received about $2.1 million through the exercise of warrants by investors. The number of shares outstanding is 19.5 million. Read More
The House Energy and Commerce Subcommittee on Health voted Wednesday to advance the House version of the Pandemic and All-Hazards Preparedness Reauthorization Act to the full committee. Read More
Lumicks BV, of Amsterdam, the Netherlands, has partnered with Astrazeneca plc, of Cambridge, U.K., and the University of Cambridge biochemistry department to form the Center of Excellence for Dynamic Single-Molecule Analysis for Accelerated Drug Discovery and Biology, the first of its kind worldwide. Read More
