The week in pharma: action, reaction and insight – week to February 4, 2022

Among last week’s notable news, Pfizer and Ionis pulled the plug on the development of their cholesterol candidate vupanorsen, with rights to the drug returned to Ionis. US biotech Biogen last week exercised its option to co-develop cancer candidate mosunetuzumab, paying Roche a one-time fee of $30 million to do so. Separately, Biogen also released financial results for 2021, along with business development plans as it tries to overcome the disappointment of its Alzheimer’s treatment Aduhelm. Also, cell therapy developer Orchard Therapeutics gained clearance in England and Wales for its gene therapy Libmeldy for a rare disease affecting babies, which at a list price of around $3.8 million is seen as the most expensive drug in the world, although this has been heavily discounted.

Vupanorsen discontinuation impact for Ionis' pipeline, raises questions on ANGPTL3

Last Monday, Pfizer announced discontinuation of vupanorsen after review of Phase IIB results, returning the asset to Ionis. Commenting on the news, SVB Leerink Research analyst Dr Mani Foroohar noted that the Phase IIb trial of the ANGPTL3-targeting ASO in dyslipidemia patients demonstrated statistically-significant reductions in ANGPTL3, triglycerides, and non-HDL-C late last year, though at the cost of dose-dependent AST/ALT elevations, and observed increases in liver fat. Pfizer concluded that the magnitude of non-HDL-C and triglyceride reduction falling below the desired range, combined with the dose-dependent safety signals, made continuation of the program untenable (a decision that seems reasonable to us).