October saw big pharma placing bets in different areas through notable acquisitions.
The highest value deal of the month came early on when Bristol Myers Squibb (BMS) agreed to acquire cardiovascular company MyoKardia for $3.1 billion through an all-cash deal. The acquisition was largely driven by mavacamten, MyoKardia’s promising first-in-class therapeutic for the treatment of a type of heart disease known as hypertrophic cardiomyopathy. BMS will also gain a number of other cardiovascular candidates through the purchase.
Later in the month, Bayer agreed to acquire gene therapy company Asklepios BioPharmaceutical, also known as AskBio, for $2 billion upfront and a potential further $2 billion in success-based milestones. Already an experienced partner, AskBio has gene therapy capabilities that include a proprietary cell line manufacturing process and an adeno-associated viruses (AAV) capsid and promoter library, both of which are already being used in various company technology platforms.
According to the feature Next-generation therapeutics sustain momentum earlier this year, only 5 of the top 20 companies in the field of cell, gene and nucleic acid therapies—based on 2024 sales forecasts from Evaluate Pharma—are large biopharma: Novartis, BMS (following the acquisition of Celgene), Biogen, Gilead and Roche.
Novartis currently leads the top 20 companies, and further strengthened its position by signing a similar gene therapy based-deal to Bayer with its acquisition of Vedere Bio for $280 million, which includes $150 million upfront and $130 in milestone payments. Novartis will gain access to Vedere Bio’s AAV gene therapy platform, as well as an optogenetics program indicated for the treatment of vision loss and blindness.
And finally also investing in AAV gene therapy, rare disease company Ultragenyx Pharmaceutical teamed up with Solid Biosciences to collaborate on the development and commercialization of new gene therapies for Duchenne muscular dystrophy (DMD), which is caused by a lack of functional dystrophin. The deal will see Solid combine its synthetic gene microdystrophin construct with AAV8 variants and Ultragenyx’s HeLa producer cell line manufacturing platform to produce an AAV therapy that delivers the microdystrophin gene. Solid will receive $40 million upfront and up to $255 million in milestones plus royalty payments.
