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.
Immunocore Holdings Plc, a developer of T cell receptor immunotherapies, has reported third quarter revenue for its first approved product, Kimmtrak (tebentafusp), for metastatic uveal melanoma. The therapy was approved in both the US and the EU in 2022. In the third quarter Kimmtrak delivered net sales of £49.7 million, an increase of 9% from a year earlier. Of this sum, £34.5 million was generated in the US; £15 million from Europe; and £0.2 million from the rest of the world.
Freeline Therapeutics Holdings Plc, a UK gene therapy company, is to become a private company once again following an agreement with a portfolio company of Syncona, its majority shareholder and co-founder, to acquire all of the shares that Syncona doesn’t already own. Syncona co-founded Freeline in 2015 with the aim of developing gene therapies for chronic debilitating diseases. In 2020, Freeline went public with the issue of 8.8 million American Depositary Shares (ADSs) on the US Nasdaq market, raising $158.8 million.
The world’s first regulatory approval of a gene-edited therapy, Casgevy (exagamglogene autotemcel), was issued by the UK Medicines and Healthcare products Regulatory Authority (MHRA) on 16 November, paving the way for the launch in the UK of a new treatment for sickle cell disease and transfusion-dependent beta thalassaemia. Both diseases are inherited blood disorders that affect red blood cells which are essential for carrying oxygen to all organs and tissues in the body.
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.
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.
Netherlands-based VectorY Therapeutics has raised €129 million in a Series A financing round to support the clinical development of a product for amyotrophic lateral sclerosis (ALS) based on vectorised antibodies. Co-led by EQT Life Sciences and the Forbion Growth Opportunities Fund, the money will be used to develop VTx-002 and other programmes for neurodegenerative diseases.
AstraZeneca Plc has acquired rights to an experimental drug for obesity in a significant expansion of its pipeline of cardiometabolic medicines. The agreement, with Eccogene Inc of China, was announced on 9 November concurrent with the release of the company’s third-quarter results. These showed a 5% increase in revenue to $11.5 billion. The revenue increase, which includes product sales, and collaboration and alliance revenue, was 6% at constant exchange rates.