Strategy

Highlights from day one of the BIO convention include advice for firms hoping to go public, a call for companies to push the US Congress on rare disease priority review vouchers, and updates on next-generation gene therapies.

With two deaths in non-ambulatory DMD patients, Sarepta is attempting damage control and will ask the US FDA to advise, setting up a possible confrontation with CBER director Prasad.

Supernus agreed to pay $561m upfront plus a contingent value right that could add $234m to buy Sage, topping a previously rejected offer from Biogen.

First results for a first-in-class mutCALR-targeted therapy in essential thrombocythemia presented at EHA point to a lucrative future for the early-stage product.

UroGen’s Zusduri was approved despite an advisory committee vote recommending against it in May. It is the first approved drug for a type of recurring bladder cancer.

The Belgian drugmaker is boosting its biologics capacity over the pond.

Deal Snapshot: Including its internal efforts with bimagrumab, Lilly has been seeking a muscle-sparing therapy to complement GLP-1 obesity drugs, such as Zepbound.

Plus deals involving BioAge/JiKang, Sanyou/Medicovestor, AriBio/Acino, Intragrand/Transpire, Kaken/Twocells, Sumitomo/Novo Nordisk, NIBEC/Undisclosed, Formosa/Adalvo, Drug Farm/Amoytop, Lupin/SteinCares and Lupin/Honeywell.

The oral BTK inhibitor impresses in a Phase II trial for IgG4-related disease.

Will Zydus’s acquisition and licensing deal with Agenus turn out to be a strategic move towards building an oncology portfolio or an opportunistic one driven by a venture capital mindset?

The first Phase III data for the CD38-targeting antibody is expected in 2027 in antibody-mediated rejection in kidney transplant patients, followed by other indications.

Novo Nordisk will partner with privately held Deep Apple to study a novel, non-incretin target that could be better suited than GLP-1s for long-term obesity treatment.

The big pharma CEO with the highest-valued compensation in 2024 was David Ricks of Eli Lilly, while Pfizer and J&J executives slipped into third and fourth place behind AbbVie's now retired chief Richard Gonzalez. European firms brought up the rear.

Series B cash will be used to advance its Stargardt disease gene therapy.

Insmed’s inhaled treprostinil exceeded goals for reducing pulmonary vascular resistance and improving six-minute walk test distance in Phase IIb data.

The US FDA approved Merck’s Enflonsia (clesrovimab) to prevent RSV in newborns and infants on the same day Sanofi assured that it would ship Beyfortus in time for the 2025-2026 RSV season.

Intended as obesity monotherapy and for combination with the firm’s GLP-1 analog, MET-0233i showed best-in-class efficacy with placebo-like tolerability at low doses.

The company announced positive data from two Phase III trials testing the cholesterol-lowering medicine.

The French company will start shipping its blockbuster shortly to meet expected strong demand in the 2025–26 RSV season.

Otsuka bested Vera on Phase III immunoglobulin A nephropathy data for proteinuria, but analysts wonder if the difference is especially meaningful.