Clinical Research

argenx pemphigus trials fails

Country
Netherlands

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. 

Vertex drug reduces pain

Country
United States

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.

argenx trial in thrombocytopenia fails

Country
Netherlands

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 shows promise for psoriatic arthritis

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.

Semaglutide reduces risk of cardiovascular events

Country
Denmark

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.

Bayer atrial fibrillation trial is stopped

Country
Germany

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’ myelofibrosis data are positive

Country
Germany

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 gives data for DMD gene therapy

Country
United States

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.

Positive data for ADC in breast cancer

Country
United Kingdom

AstraZeneca Plc has reported positive data from a Phase 3 trial of its antibody-drug conjugate datopotamab deruxtecan in patients with metastatic hormone receptor (HR) positive, HER2-low or negative breast cancer, the most common breast cancer subtype, accounting for more than 65% of cases diagnosed globally. The trial enrolled 700 patients across five continents.

Cell therapy treats refractory lymphoma

Country
United States

Yescarta, a chimeric antigen receptor (CAR) T cell therapy, has delivered positive results in a Phase 2 study of patients with relapsed or refractory large B cell lymphoma after one prior therapy. The patients were all ineligible for chemotherapy and autologous stem cell transplantation. Yescarta (axicabtagene ciloleucel) is already approved for several lymphoma indications in the US and Europe.