News

Efgartigimod, the antibody treatment being developed by argenx SE for a spectrum of autoimmune diseases leapt, over another hurdle on 17 August with news that it had met the primary endpoint in a trial of two rare muscle diseases. The Phase 3 study showed that the antibody fragment, in combination with hyaluronidase enzymes, was able to restore the strength and muscle function in patients with two types of myositis: immune-mediated necrotising myopathy (IMNM) and dermatomyositis (DM). Both diseases cause muscle weakness leading potentially to long-term disability.

Switzerland-based Vaderis Therapeutics AG has raised $152 million from a Series B financing round to support development of a small molecule drug, engasertib, for a rare vascular disease for which no treatments are currently approved. The disease is hereditary haemorrhagic telangiectasia (HHT), a genetic disorder affecting the vasculature, which manifests in nose bleeds, anaemia, and visceral arteriovenous malformations (AVMs) AVMs are tangles of blood vessels that can cause internal bleeding and organ damage.

On 4 August, the European Commission completed the final legal steps for the launch of the Scaleup Europe Fund, a public-private partnership that will invest in European companies with potential to compete globally in advanced technologies. The fund is being launched with a target capital of €5 billion. The fund’s first investment, a day after launch, was in the Finnish space technology company ICEYE. 

Ireland-based Jazz Pharmaceuticals Plc is to expand its presence in the epilepsy field with the acquisition of privately-held Actio Biosciences of San Diego, US, which has a clinical-stage product for a rare genetic epilepsy. In parallel, Jazz will take a minority stake in a spin-out of Actio, which will develop a cluster of rare disease products.

GSK Plc gave further details of its investment plans on 28 July with the release of its financial results for the second quarter. Turnover for the quarter was £8.4 billion, up by 5% in both actual and constant exchange rates, leading the company to forecast growth for the year of 3% to 5% at constant rates. The company expects to pay a dividend of 70 pence per share for the full year.

AstraZeneca reaffirmed its ambition to achieve revenue of $80 billion by 2030 after a second quarter that featured 12 regulatory approvals across four regions and five positive Phase 3 readouts. The positive trial results included one for a new antibody-drug conjugate, sonesitatug vedotin, that showed improved overall survival for patients with advanced gastric cancers.

argenx SE is to expand its immunology portfolio with the acquisition of Forte Biosciences Inc of Dallas, Texas, US, whose lead product is being investigated in two autoimmune diseases. The Netherlands-based company is to pay $77 per share in cash for the company, representing an equity value of about $2.2 billion – it’s largest deal to date. Both companies are listed on the US Nasdaq exchange.

Scribe Therapeutics Inc, a company co-founded by the Nobel laureate Jennifer Doudna, has raised $128.7 million in an initial public offering on the US Nasdaq market – one of a growing number of biotech companies to go public this year. The funds will be used to advance the company’s lead programme for patients with elevated low-density lipoprotein cholesterol (LDL-C) through Phase 1, and to develop two preclinical projects for cardiovascular and metabolic disorders.

At a time of patent expiries and a strong Swiss franc, the Roche Group is expanding its presence in oncology and other disease areas in order to stimulate growth. Sales for the first half year were CHF 30.4 billion, an increase of 6% at constant exchange rates, and 8% in US dollars. Measured in Swiss francs however, sales declined by 2%. The trend, driven by an appreciation of the franc, was visible across both the pharmaceutical and diagnostics divisions.

Netherlands-based AdJane Holding BV has reported the first clinical data for a new vaccine platform that is being positioned as a technology for use in preparing for future pandemics. The early data showed that outer membrane vesicles, which are vesicles released from the outer membranes of Gram-negative bacteria, were safe and well-tolerated in 40 healthy adults. The vaccine, based on outer membrane vesicles, was administered intranasally and combined with a SARS-CoV-2 spike protein- the structural component of the virus that caused the Covid-19 pandemic.