Drug Development
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.
FEATURED STORIES
In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline.
Pharmaceutical companies are combining antibodies with radioisotopes in a bid to more precisely deliver radiation to cancers and tumors.
Artificial intelligence won’t replace people in biopharma, but it is infiltrating every step of drug development, including in some ways that aren’t so obvious.
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GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
The overall survival edge over J&J’s Darzalex will help GSK strengthen its case as it plots the market comeback of Blenrep, which was pulled after a failed confirmatory study.
Anito-cel has shown no signs of delayed neurotoxicity at around 9 months of follow-up, hinting at a safety profile that could set it apart from J&J and Legend’s Carvykti.
According to Jake Van Naarden, president of Lilly Oncology, the excess deaths could be due to the high rate of crossover in BRUIN CLL-321.
Relmada Therapeutics will halt two Phase III trials of a major depressive disorder drug after a futility assessment and explore strategic alternatives including a potential sale to maximize shareholder value.
Tavapadon improved motor and daily living complications at week 26. The news comes nearly one month to the day after AbbVie announced the Phase II failure of another key Cerevel asset, emraclidine.
Analysts called the data “very competitive” but raised questions about safety. Merck gained ownership of the ADC when it acquired VelosBio in November 2020 for $2.75 billion.
The discontinuation of STRIDES is a rare stumble for the next-generation obesity field and comes just weeks after Amgen announced underwhelming mid-stage data for MariTide.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.