Company News

The US Food and Drug Administration granted an accelerated approval on 19 August for a new gene therapy for patients with an inherited disorder caused by the shortage of an enzyme needed to maintain stable blood sugar levels in the body. The therapy, Genglycos (pariglasgene brecaparvovec), is the first treatment to be authorised for the disorder – glycogen storage disease type la. Also called von Gierke disease, the disorder results in the  build-up of glycogen, a complex sugar, in the body’s cells.

A messenger RNA (mRNA)-based therapy designed for cancer has delivered positive Phase 3 results in patients with resected, advanced myeloma representing a first of its kind treatment in oncology. Announced on 19 August, the trial, INTerpath-001, delivered recurrence-free survival and distant metastasis-free survival for patients who had tumours removed but were still at risk of disease. The trial enrolled 1,137 patients who were randomised to receive intismeran, the mRNA therapy, and Keytruda (pembrolizumab) or Keytruda alone.

AstraZeneca Plc reported two positive outcomes and one negative outcome for three Phase 3 lung cancer trials on 17 August. The trials were all testing treatments for non-small cell lung cancer, the most prevalent type, which makes up about 85% of all lung cancers globally, according to the World Health Organization. The largest trial, which was testing a dual checkpoint inhibitor bispecific antibody, failed to show efficacy. However the two other studies, targeting cancers with specific genetic mutations, achieved statistically significant and clinically meaningful results.

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.

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.