News

A new gene therapy has been given a favourable scientific review in Europe paving the way for its use in patients with beta-thalassaemia as early as the second quarter. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March.

Antibody-drug conjugates (ADCs) have assumed new prominence as cancer treatments in the wake of a deal between AstraZeneca Plc and Daiichi Sankyo Co Ltd of Japan to co-develop trastuzumab deruxtecan, an ADC for a group of HER2-mutated cancers.

A type of inflammatory arthritis that affects the spine but cannot be detected by x-ray now has an approved treatment. The US Food and Drug Administration has authorised Cimzia (certolizumab pegol) for the treatment of non-radiographic axial spondyloarthritis, a disease that starts in patients under the age of 45 years and can get progressively worse. The fact that the disorder cannot be identified by x-ray sets it apart from other inflammatory conditions in the same disease group.

Galapagos NV and Gilead Sciences Inc have announced positive data from two ongoing Phase 3 trials of the Janus kinase-1 (JAK1) inhibitor filgotinib in rheumatoid arthritis (RA). The trials, FINCH 1 and FINCH 3, met the primary and key secondary endpoints with favourable safety data at the 24 week cut-off date.

France-based Genfit SA has raised $135 million in a global share offering that includes a listing on the Nasdaq over-the-counter market in the US. The company’s US listing is in the form of American Depositary Shares (ADSs). The company has been trading on the Euronext exchange in Paris since 2006.

The US Food and Drug Administration has approved a new drug for the treatment of patients with multiple sclerosis (MS) whose disease gradually worsens over time leading to a disruption of brain function through inflammation and tissue loss. The drug, Mayzent (siponimod), is a small molecule indicated for active secondary progressive MS.

A prospective therapy for birch allergy failed to achieve the primary endpoint in a Phase 3 multi-center trial, according to the developer Allergy Therapeutics Plc. The therapy, B301, is a short-course treatment for birch-pollen induced seasonal allergic rhinitis.

It failed to show statistical significance relative to placebo. The safety and tolerability profile of the therapy were positive.

Record sums were invested in biotech companies in 2018 – especially in those at the early stage of development. According to the US industry association BIO, some $12.4 billion in venture capital was raised in the US for biotech, double the amount generated in 2017, while $600 million was raised in Europe.

Celgene Corp has contracted the UK artificial intelligence company Exscientia Ltd to supply drug discovery services over a period of three years in order to identify new candidate molecules for three programmes in the areas of oncology and autoimmunity.

The alliance is one of several for Exscientia which has also secured collaborations with Roche, GlaxoSmithKline Plc, Sanofi SA and Evotec AG. Celgene, which is soon to be taken over by Bristol-Myers Squibb Co, is also an investor in Exscientia.

Pfizer Inc has entered into an option agreement to acquire Vivet Therapeutics SAS, a privately-held French company with a gene therapy for Wilson disease, a rare genetic disorder that prevents the body from regulating copper. The disease can lead to liver and brain damage.

Pfizer will initially take a 15% equity interest in Vivet with an exclusive option to acquire the remaining shares in the future. The exercise of the option is linked to the performance of the therapy in a Phase 1/2 trial.