Questions about the clinical meaningfulness of the primary endpoint and the potential for long-term neurodevelopmental issues will frame some of the discussion Thursday as the FDA's Pediatric Advisory Committee and Gastrointestinal Drugs Advisory Committee consider the risk-benefit profile of Infacare Pharmaceutical Corp.'s stannsoporfin injection. Read More
Icagen Inc., of Tucson, Ariz., received an up to $11 million award from the Cystic Fibrosis Foundation for a project focused on the discovery of therapeutics aimed at treating cystic fibrosis caused by nonsense mutations. Read More
Incyte Corp., of Wilmington, Del., reported first-quarter GAAP net product revenue totaling $314 million for JAK inhibitor Jakafi (ruxolitinib), marking 25 percent growth over 2017. GAAP net product revenue for leukemia drug Iclusig (ponatinib) totaled $21 million vs. $14 million for the same period last year. Read More
Azurrx Biopharma Inc., of Brooklyn, N.Y., is selling 4.16 million shares of its common stock at $2.50 per share, grossing the company $10.4 million. Azurrx plans to use the capital to continue clinical development of MS-1819, its recombinant lipase for exocrine pancreatic insufficiency, advancing its preclinical programs for AZX-1103 and for other general corporate purposes and capital expenditures. Read More
Early Tuesday, Karyopharm Therapeutics Inc. officials shook off the previous disappointment of lead agent selinexor in acute myeloid leukemia and turned to top-line findings from the phase IIb STORM (Selinexor Treatment of Refractory Myeloma) study. Tested in heavily pretreated patients with refractory multiple myeloma (MM), the oral selective inhibitor of nuclear export (SINE) hit its primary endpoint, achieving a 25.4 percent overall response rate (ORR), including two complete responses (CRs) and 29 partial (PRs) or very good partial responses (VGPRs) in patients with penta-refractory myeloma. Read More
LONDON – Spending on drugs is outstripping growth in the total National Health Service (NHS) budget and now threatens efforts to balance the competing priorities of patient access, incentivizing pharma innovation and affordability, according to a new report from the U.K's leading independent health research group. Read More
Directors at Ironwood Pharmaceuticals Inc. have green-lit plans to spin off the company's R&D programs into a new publicly traded company, a move that could help sales of Linzess (linaclotide) and other approved treatments carry the commercial business to profitability in 2019 while compartmentalizing risks of its rare disease-focused soluble guanylate cyclase (sGC) stimulator programs. Read More
The latest chapter in Siga Technologies Inc.'s arduous history with Tpoxx (tecovirimat) closed happily with the FDA's Antimicrobial Drugs Advisory Committee (AMDAC) voting 17-0 to recommend approval of the smallpox vaccine caused by variola virus in adult and pediatric patients. Read More
DUBLIN – Cellectis SA is supplying its transcription activator-like effector nuclease (Talen) genome editing technology to what has been billed as the first "grand-scale community-wide project" of the Genome Project Write (GP-Write) initiative, an effort to develop a cell line that is resistant to naturally occurring viruses. Read More
