Roche Holding AG's interleukin-6 antagonist, Actemra (tocilizumab), gained a supplemental approval Monday, becoming the first FDA-approved treatment for giant cell arteritis (GCA), a chronic form of vasculitis for which the only current treatment option involves steroids.
Though the debut of its stock was marred by the U.S. market's overall decline Wednesday, G1 Therapeutics Inc. priced an IPO to raise $105 million to advance lead candidates aimed at inhibiting cyclin-dependent kinases (CDKs).
While last year's positive readout from the first part of Array Biopharma Inc.'s phase III COLUMBUS trial testing binimetinib and encorafenib in BRAF-mutant advanced, unresectable or metastatic melanoma cleared the way for regulatory filings, supplementary results from the study's second part, released late Tuesday, could bolster the combination's prospects for approval and market penetration.
The FDA has joined Japanese and South Korean regulators in approving edaravone, an intravenously administered antioxidant free radical scavenger, for treating amyotrophic lateral sclerosis (ALS), with its late Friday approval coming more than a month ahead of the PDUFA date.
Astrazeneca plc won FDA accelerated approval for durvalumab, its breakthrough-designated PD-L1 inhibitor, for treating locally advanced or metastatic urothelial carcinoma, a market in which it will face Roche Holding AG’s Tecentriq (atezolizumab), approved in May 2016.
Companies ignoring the advice of their independent data monitoring committees (IDMCs) do so at their own peril, but Argos Therapeutics Inc. might have a more compelling case than most for opting to continue the phase III ADAPT study testing rocapuldencel-T in combination with Sutent (sunitinib, Pfizer Inc.) in metastatic renal cell carcinoma (mRCC), despite a disappointing interim analysis in February, in which the IDMC recommended the study be stopped for futility.
Ultragenyx Pharmaceutical Inc. said it believes data from a phase III study testing burosumab (KRN23), a recombinant fully human monoclonal IgG1 antibody, are sufficient to support a BLA filing this year in X-linked hypophosphatemia (XLH), a bone disease characterized by phosphate wasting due to excess activity of FGF23.
When going after macrophages, "in my view, you play directly with the immune system," said Maxime Ranger, president and CEO of 2016 startup Mperia Therapeutics Inc., which is advancing macrophage-targeting immunotherapies based on the CD36 scavenger receptor for diseases involving chronic inflammation such as dry age-related macular degeneration (AMD) and atherosclerosis.
Wave Life Sciences Ltd. boasted a balance sheet of $150.3 million as of Dec. 31, funds the company said during its fourth-quarter earnings should get the firm into 2019. But as always in the field of drug development, a little extra padding never hurts, and Cambridge, Mass.-based Wave Life priced a $100 million public offering Wednesday as it gets ready to move its first three candidates into the clinic targeting CNS and neurological disorders.
Vertex Pharmaceuticals Inc. is targeting third quarter filings in the U.S. and Europe after reporting positive data from two phase III studies testing oral CFTR corrector tezacaftor (VX-661) plus approved CFTR potentiator Kalydeco (ivacaftor) in cystic fibrosis (CF), a combination expected to broaden the swath of patients eligible for therapy.
