Novartis pays $12B for Avidity and its muscle-targeting RNA platform
Acquisition aims to accelerate 3 late-stage MD treatments toward approval
In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop several promising experimental therapies for various types of muscular dystrophy.
The core of the acquisition is Avidity’s antibody-oligonucleotide conjugate (AOC) platform. This innovative technology attaches a small piece of RNA (an oligonucleotide) to an antibody. Essentially, the antibody targets specific cells to deliver the oligonucleotide, which then modulates the genetic activity of the cell.
“Avidity’s pioneering AOC platform for RNA therapeutics and its late-stage assets bolster our commitment to delivering innovative, targeted and potentially first-in-class medicines to treat devastating, progressive neuromuscular diseases,” Vas Narasimhan, MD, CEO of Novartis, said in a company press release. “The Avidity team has built robust programs with industry-leading delivery of RNA therapeutics to muscle tissue. We look forward to developing these programs to meaningfully change the trajectory of diseases for patients.”
Developing treatments for muscular dystrophy
Avidity is currently developing treatments for three types of muscular dystrophy. One, delpacibart etedesiran (del-desiran), previously called AOC 1001, is for myotonic dystrophy type 1 (DM1). Early clinical testing suggested it is generally safe, may improve muscle strength, and eases the inability of muscle to relax in DM1 patients. A Phase 3 clinical trial called HARBOR (NCT06411288) is ongoing.
Another Avidity therapy currently in late-stage development is delpacibart braxlosiran (del-brax) for facioscapulohumeral muscular dystrophy (FSHD). That therapy has also shown promise in clinical testing and is now being assessed in a Phase 3 trial, called FORTITUDE-3 (NCT07038200), which is recruiting FSHD patients ages 16 to 70 at sites in the U.S.
Assuming that the HARBOR and FORTITUDE-3 trials have positive results, Avidity had previously announced plans to submit applications seeking approval of del-desiran and del-brax.
Avidity is also developing delpacibart zotadirsen (del-zota), an exon-skipping therapy for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. Recent one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730) and its extension study (NCT06244082) indicated del-zota works as intended and that it can reduce muscle damage and improve limb function in DMD patients.
Avidity previously announced plans for a Phase 3 trial to further test del-zota. It also said it planned to use the Phase 1/2 results as a basis to ask the U.S. Food and Drug Administration to grant the therapy accelerated approval, which is a type of conditional approval allowing a therapy to be sold based on early evidence that it is likely to help patients, while requiring developers to run additional testing to confirm clinical benefit.
The merger between Novartis and Avidity is expected to be completed in early 2026, pursuant to conditions including the formation of a spin-off company to develop Avidity’s investigational therapies for heart disease, which were not included in the Novartis deal. Until the deal closes, Novartis and Avidity will continue to operate as separate and independent companies.
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