News

Can Pfizer’s GDF15-blocking, appetite-boosting antibody ponsegromab address a cancer-induced wasting syndrome — and help patients live longer in the process?

Two dual-payload antibody–drug conjugates are now in the clinic for cancer, with many more on the way.

Insmed’s DPP1 inhibitor brensocatib could become the first drug to secure an approval for bronchiectasis, and is in trials for other inflammatory diseases.

Drug developers are re-advancing amylin analogues for obesity, two decades since the FDA approved the first of these hormone mimetics for diabetes.

Johnson & Johnson has discontinued development of aticaprant for depression with anhedonia, a blow to hopes for drugs for ‘domains’ of psychiatric disorders.

Halda Therapeutics has started a clinical trial of its first-in-modality RIPTAC drug HLD-0915 in prostate cancer, to see if bifunctional drugs can provide tissue-specific activity.

Scholar Rock’s myostatin blocker has shown its efficacy in spinal muscular atrophy, and the 28-year-old myostatin field is now looking to muscle in on obesity.

The FDA approved 50 new therapeutics in 2024, with green lights for a novel schizophrenia drug, a first NASH medicine and much more.

Vertex’s first-in-class suzetrigine could soon usher in a new class of analgesic.

Clinic-ready molecular glues and heterobifunctional PROTAC drugs are taking targeted protein degradation into uncharted territory.

Drug developers want to reprogramme immune cells directly in the body, opening up new gene therapy frontiers in cancer and autoimmunity.

Karuna and Bristol Myers Squibb’s muscarinic agonist KarXT looks set to offer the first new drug mechanism for patients with schizophrenia in over 70 years.

Small molecules that block a key protein–protein interaction could change the treatment landscape for many patients with acute leukaemias.

Biohaven’s BHV-1300 lowered levels of immunoglobulin G in the blood of healthy volunteers — a first clinical demonstration of the potential of a small-molecule extracellular protein degrader.

‘Pre-mRNA trans-splicing’ drugs could offer an alternative to gene replacement therapies for genetic diseases — starting with Stargardt disease.

ADAR-based editors that can change the mRNA code offer new opportunities in both rare genetic diseases and common complex ones.

Madrigal’s resmetirom has scored a landmark FDA win, bolstering hopes for a diverse pipeline of drugs for non-alcoholic steatohepatitis.

The CACHE hit-finding competition highlights the potential of AI to identify small molecules that bind to hard-to-drug targets — and the long road ahead for these computational screening approaches.

The FDA approved 55 novel therapeutics in 2023, the second highest count in the past 30 years.

The FDA could soon approve Iovance’s lifileucel for advanced melanoma, a much-anticipated milestone for the 35-year-old T cell therapy modality.