Randy Osborne

What PTC Therapeutics Inc.'s latest data with votoplam might mean in the Huntington's disease (HD) landscape became grist for Wall Street after the firm unveiled top-line results from the phase II Pivot-HD study, sharing data from the 24-month interim analysis of the long-term extension effort.

The memory of Merck & Co. Inc.'s $10 billion takeover last year of inhaled respiratory drug specialist Verona Pharma plc may not have been far from

Wall Street pundits were divided about the likely fate of ivonescimab, Summit Therapeutics Inc.'s bispecific antibody partnered with Akeso Pharmaceuticals Inc., of Hong Kong, and undergoing phase III testing in first-line squamous non-small-cell lung cancer (NSCLC).

Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).

Thanks to a trial design that, according to Evercore ISI analyst Cory Kasimov, “played out exactly as drawn up,” Cytokinetics Inc. hit both of the phase III co-primary endpoints with aficamten in symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

Climb Bio Inc. outlined May 5 an enticing data spill ahead this year with Fc-enhanced monoclonal antibody budoprutug (budo) in autoimmune diseases. Mizuho analyst Joseph Catanzaro appreciated in his report Wellesley Hills, Mass.-based Climb's “conviction that CD19 represents a best-in-class pan-B-cell depletion target across antibody-mediated autoimmune diseases – a relative white space for conventional mAbs.”

How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.

Upbeat phase II news lately from Spyre Therapeutics Inc. in inflammatory bowel disease (IBD) perked not only the company's stock but also wider interest in the buzzing space, where developers big and small are trying to tackle such chronic bedevilments as Crohn's disease and ulcerative colitis (UC).

The “deep dive” April 8 by Ovid Therapeutics Inc. into potassium-chloride co-transporter 2 (KCC2) research included company officials and independent experts in the space, where a handful of drug developers are known to be working.

Payoff for the November 2023 buyout of Immunogen Inc. came for Abbvie Inc. in the form of U.S. FDA clearance for the CD123-targeting antibody-drug conjugate Decnupaz (pivekimab sunirine) to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare, aggressive and quick-killing hematologic malignancy.