News

H. Lundbeck A/S is to acquire Longboard Pharmaceuticals Inc of the US for $2.6 billion in a transaction that will significantly expand the Danish company’s neurology pipeline. Longboard has a small molecule asset in Phase 3 that is being developed for the treatment of seizures associated with a group of rare epilepsies – a market segment not currently covered by the Danish company. The drug, bexicaserin, is currently in a study for Dravet syndrome, a rare form of epilepsy that begins in infancy.

Itovebi (inavolisib), a kinase inhibitor for use in combination with two other drugs to treat breast cancer, has been approved by the US Food and Drug Administration. This follows a priority review and breakthrough therapy designation for the combination with the marketed breast cancer drugs palbociclib (Ibrance) and fulvestrant (Faslodex). Itovebi targets the PI3K enzyme, a product of the PIK3CA gene. According to the US National Cancer Institute, mutations in the PIK3CA gene have been identified in multiple cancers including breast, lung, ovary, stomach and brain.

Immatics NV, a clinical stage biotech company, has announced the pricing of a new share offering in the US which is expected to raise $150 million for the development of T cell receptor (TCR) therapies for cancer. The underwritten public offering of 16,250,000 ordinary shares was priced at $9.25 per share. The offering is expected to close on 15 October.

This year’s Nobel Prize in Chemistry has been awarded to David Baker, Demis Hassabis, and John Jumper for their work in the field of protein science. Dr Baker, a professor at the University of Washington, US, has been recognised for his pioneering research in computational protein design. Drs Hassabis and Jumper, both from Google DeepMind, are credited with developing AlphaFold2, an artificial intelligence model that predicts the structure of proteins with great accuracy.

Purespring Therapeutics Ltd, a gene therapy company developing treatments for kidney diseases, has raised £80 million from private investors to bring its lead product into clinical development. This is the latest in a series of transactions among biotech companies in the kidney disease sector but the first for a preclinical gene therapy. Purespring’s product candidate, PS-002, is an adeno-associated viral vector with a gene payload for the treatment of IgA nephropathy (IgAN), a chronic kidney disease primarily affecting young adults. 

A Phase 2 trial of a small molecule drug for patients with mild dementia due to Alzheimer’s disease has been stopped due to lack of efficacy, the developer Sage Therapeutics Inc announced on 8 October. The drug, dalzanemdor (SAGE-718), did not show a statistically significant difference in patients with the disorder, compared with a placebo, on the basis of a widely-used intelligence test. 

Victor Ambros and Gary Ruvkun have been awarded this year’s Nobel Prize in Physiology or Medicine for their discovery of microRNA and its role in post-transcriptional gene regulation. Their work has revealed an entirely new mechanism that controls gene expression, transforming our understanding of how cells regulate the production of proteins essential for their function and development.

A consortium of universities and hospitals, with financial support from the UK government and industry, is to undertake a four-year project to collect data on patients who have received immunotherapy for cancer to establish the benefits and risks of these treatments. The treatments, which include checkpoint inhibitors, have extended the lives of many patients by enabling the body’s immune system to recognise and destroy cancer cells.

AstraZeneca Plc is to pay $100 million upfront and up to $1.92 billion in milestone payments for rights to a pre-clinical lipid-lowering therapy from China. The developer, CSPC Pharmaceutical Group Ltd, is listed on the Hong Kong Stock Exchange and has R&D facilities in both China and the US. The asset being acquired is a small molecule lipoprotein inhibitor which is in pre-clinical development for patients suffering from dyslipidaemia, a disorder involving abnormal levels of lipids in the bloodstream. Dyslipidaemia is a risk factor for cardiovascular diseases.

An Italian biotech company, Aptadir Therapeutic Srl, which is developing a new class of RNA therapies, launched on 24 September with pre-seed funding of $1.6 million and plans to tackle cancer by blocking aberrant DNA methylation. DNA methylation is an essential biological process that enables the expression of genes in humans. But this process can be thrown off balance by external factors such as age or diets, leading to cancer and genetic disorders.