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.
Sanofi SA is to divest its consumer healthcare business unit in order to concentrate its development and commercialisation resources on innovative, proprietary medicines. This follows a similar decision by GSK Plc which also spun out its consumer products business into a separate, listed business in 2022. Sanofi announced the decision on 27 October, saying that the undertaking would most likely involve a capital markets transaction and could take place as early as the fourth quarter of 2024.
The Roche Group expects sales to decline this year following a steep drop in demand for diagnostic tests for Covid-19. Excluding this factor, sales of both the pharmaceuticals and diagnostics base businesses are projected to increase. Roche said it plans to increase its dividend in Swiss francs.
Novartis gave its first financial forecast, post the spin-out of Sandoz, with the release of third quarter earnings on 24 October. The quarterly figures showed a 12% increase in net sales to $11.78 billion from a year earlier, and a large rise in spending on research and development. The Switzerland-based company is forecasting an increase in net sales for the year of a high single digit figure and an increase in non IFRS operating income of a mid to high teens figure.
GSK Plc highlighted disease prevention as a strategic objective for the company with the release of its third quarter results on 1 November. These showed a strong performance for the company’s vaccines for herpes zoster (shingles) and respiratory syncytial virus (RSV) which together generated £1.53 billion in the quarter, or 19% of total group turnover of £8.15 billion. Arexvy, an RSV vaccine for older adults, was approved in the US in May and in the EU in June.
AstraZeneca Plc is to work with gene editing technologies developed by Cellectis SA of France to identify up to 10 candidate cell and gene therapies for cancer, and autoimmune and rare diseases. Founded in 1999, Cellectis has technology for creating allogeneic chimeric antigen receptor (CAR) T cells by gene editing cells from a healthy donor. The editing technology disables genes that cause donor cells to attack the host.
Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.
Bimzelx (bimekizumab), a monoclonal antibody that inhibits two cytokines driving inflammation, has been approved in the US for the treatment of moderate to severe plaque psoriasis. The Food and Drug Administration cleared the drug for use on 18 October, more than a year after issuing a complete response letter to the developer, UCB SA of Belgium. The CRL was related to certain “pre-approval inspection observations,” according to the company.