An experimental chimeric antigen receptor (CAR) T cell therapy intended for the treatment of multiple sclerosis received a fast track designation from the US Food and Drug Administration on 19 January, indicating its potential to address a major medical need. The therapy is being developed by Kyverna Therapeutics Inc, a biotech company incorporated in 2018 and located in Emeryville, California, US.
Sanofi has stopped a global programme evaluating the clinical effectiveness of tusamitamab ravtansine, an antibody-drug conjugate (ADC), after a Phase 3 trial of the drug failed to improve progression-free survival in patients with a type of non-small cell lung cancer. Tusamitamab ravtansine was targeting a transmembrane cell adhesion molecule – carcinoembryonic antigen-related cell adhesion molecule 5 – which is associated with a number of cancers.
argenx SE reported the second failure of a trial involving its antibody fragment efgartigimod alfa on 20 December following unexpectedly positive results from the placebo arm of the study. Called ADDRESS, the Phase 3 trial was investigating a subcutaneous formulation of efgartigimod (efgartigimod alfa and hyaluronidase) in adults with two types of pemphigus, an autoimmune disease that affects the skin and mucous membranes.
A small molecule drug in development by Vertex Pharmaceuticals Inc has reduced pain in patients with diabetic peripheral neuropathy, a type of nerve damage that can occur with the disease. A Phase 2 trial of VX-548 which inhibits NaV1.8, a voltage-gated sodium channel, showed a statistically significant reduction in a measure of pain, Vertex announced on 13 December 2023. Patients with diabetic peripheral neuropathy are often treated with anti-seizure drugs or antidepressants, according to the Mayo Clinic. Opioid analgesics can also be used as second or third-line agents.
A Phase 3 trial of a medicine for primary immune thrombocytopenia (ITP), an autoimmune disorder that can lead to excessive bleeding and anaemia, has failed to meet its primary and secondary endpoints, the developer argenx SE announced on 28 November. The drug, Vyvgart Hytrulo (efgartigimod alfa), is an antibody fragment targeting the neonatal Fc receptor. Vyvgart has been approved in the US for generalised myasthenia gravis, another autoimmune disease, in both intravenous and subcutaneous formulations.
Sonelokimab, a single-domain antibody product, has achieved positive results from a Phase 2 trial of patients with active psoriatic arthritis. The ARGO trial, which enrolled 207 patients, met its primary endpoint with a statistically significant greater proportion of patients treated with the antibody achieving an American College of Rheumatology 50 response compared with those on a placebo at week 12. Secondary endpoints such as minimal disease activity also showed promising levels of response at week 12, according to the developer MoonLake Immunotherapeutics AG of Switzerland.
Results from a Phase 3 trial evaluating semaglutide (Wegovy) as a treatment for adults with cardiovascular disease have shown a statistically significant 20% reduction in the risk of major adverse cardiovascular events. The results, disclosed on 11 November, were simultaneously published in the New England Journal of Medicine. Called SELECT, the placebo-controlled trial enrolled 17,604 adults in 41 countries. The participants had an established cardiovascular disease and were overweight or obese but without diabetes.
A Phase 3 trial investigating a potential new treatment for atrial fibrillation has been stopped early because of inferior efficacy, Bayer AG announced on 19 November. The trial OCEANIC-AF, was investigating asundexian, a, small molecule drug being studied in patients with atrial fibrillation (AF) at risk of stroke. It was seen as a potential alternative to traditional anticoagulants for the treatment of AF, an irregular and often very rapid heart rhythm.
MorphoSys AG reported positive results on 20 November from a Phase 3 study of the BET inhibitor pelabresib as a treatment for myelofibrosis – a key development for the company’s evolving portfolio. Pelabresib is a small molecule drug designed to promote anti-tumour activity by inhibiting the function of bromodomain and extra-terminal domain (BET) proteins to decrease the expression of abnormally expressed genes in cancer. If approved, it would be the company’s second wholly-owned product on the market.
Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.