The Burrill Report


Targacept shares fell 35 percent after reporting top-line results from a mid-stage study of TC-5619 as an augmentation therapy for the treatment of negative symptoms of schizophrenia. The North Carolina-based biotech said the drug failed to hit both the primary and secondary endpoints of the study. Based on these results, Targacept said it would discontinue its development as a treatment for schizophrenia or Alzheimer’s disease. The company said it will focus its efforts on its other ongoing mid-stage programs, TC-5214 for overactive bladder and TC-1734 for Alzheimer's disease, and will also go forward with the planned development of TC-6499 for diabetic gastroparesis.

Onconova Therapeutics fell 16 percent after it said it stopped a late-stage study of its lead experimental cancer drug rigosertib plus gemcitabine in front-line metastatic pancreatic cancer. The decision came after a planned interim analysis in which the Data Safety Monitoring Board determined that the combination of rigosertib and gemcitabine was unlikely to demonstrate a statistically significant improvement in overall survival compared to gemcitabine alone. The late-stage study used an adaptive design that allowed for an interim review of the data to assess the rationale for continuing the study. While Onconova was disappointed by the interim results, it will conduct a detailed analysis of the efficacy and safety results, which will be presented at a future scientific meeting. Onconova’s rigosertib is small molecule inhibitor of phosphoinositide 3-kinase, or PI3K, and polo-like kinase, or PLK, two important cellular signaling pathways that frequently overactive in cancer cells.

Vertex Pharmaceuticals reported disappointing results from a late-stage study of ivacaftor to treat cystic fibrosis in people who have the R117H mutation. The study of 69 people 6 years of age and older did not meet its primary endpoint of the absolute change from baseline in percent predicted forced expiratory volume in one second throughout the treatment period for ivacaftor compared to placebo across all patients. However, a subset analysis of 50 patients 18 years of age and older showed statistically significant improvements in lung function and other key secondary endpoints. Vertex believes that the results show a clinical benefit for patients age 18 and older with the R117H mutation. The company plans to meet with the U.S. Food and Drug Administration in early 2014 to discuss these data and the potential submission of a supplemental New Drug Application for people with the R117H mutation. Ivacaftor was approved by the FDA in January 2012 under the trade name Kalydeco to treat the underlying cause of cystic fibrosis in people with the G551D mutation in the CFTR gene.

December 19, 2013
http://www.burrillreport.com/article-targacept_ends_another_program_shares_tumble.html