Rare diseases
Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this disease space alongside Avidity Biosciences, Lupin and AMO Pharma.
Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
The Boston-area company’s previous raises were also aimed at getting its investigational antibody treatment for inclusion body myositis through clinical trials. Now, Abcuro is eyeing a regulatory submission and potential launch.
Vertex expects to make the newly approved non-opioid pain medicine Journavx available by the end of February.
AceLink is advancing a small-molecule treatment option for Fabry disease that could provide a more convenient alternative to the current enzyme replacement therapy standard.
Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to compete in the hemophilia A space with Roche’s blockbuster Hemlibra.
Despite the discontinuations, CEO Pascal Soriot said AstraZeneca’s December 2020 acquisition of Alexion was a “fantastic” deal for the pharma.
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space dominated by Vertex.
PRESS RELEASES