News

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. 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.

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. Excluding revenue from products developed for the Covid-19 pandemic, the revenue increase was a double-digit figure.

The first vaccine for the prevention of disease caused by the chikungunya virus was approved by the US Food and Drug Administration on 9 November – the result of years of research and development by the French company Valneva SE. The vaccine, Ixchiq, was given an accelerated approval on evidence of its ability to arrest the spread of chikungunya virus disease, an emerging global health threat.

Switzerland-based MetrioPharm AG has received a rare paediatric disease designation from the US Food and Drug Administration for its candidate therapy for Duchenne muscular dystrophy (DMD). The drug, MP1032, is a small molecule immune modulator that targets dysregulated mitochondrial metabolism in macrophages. In a preclinical study it was able to increase muscle strength comparable to the corticosteroid prednisolone, but without the corticosteroid’s serious side effects.

An Austrian start-up company, HeartBeat.bio AG, has raised €4.5 million in pre-Series A financing to develop an organoid screening platform for the discovery of new drugs for heart disease. Organoids are miniature, simplified versions of human organs that are used in medical research and development. The investor syndicate included i&i Biotech Fund, Invest AG, aws Gründungsfonds II, and Tensor Ventures. The financing will support the company’s needs until 2025 when a full Series A round is expected to be launched.