Company News

A gene therapy for Wiskott-Aldrich syndrome, whose development was supported by the Italian non-profit organisation, Fondazione Telethon ETS, received a positive opinion from the European Medicines Agency on 14 November. Wiskott-Aldrich syndrome is a rare genetic disorder that keeps a child’s immune system from functionally normally. It mainly affects young boys. The treatment, Waskyra (etuvetidigene autotemcel) is the first gene therapy to be evaluated for this disease.

The US Food and Drug Administration has updated a warning about the safety of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), and restricted its use. This follows reports earlier this year of fatal acute liver failure in certain non-ambulatory, male, paediatric patients taking the drug. Following the incidents, the developer, Sarepta Therapeutics Inc, voluntarily paused distribution of Elevidys to this patient group. This has now been formalised in a notice issued on 14 November.

A candidate treatment for malaria developed by Novartis has reached the primary endpoint in a Phase 3 trial, paving the way for regulatory submission. If approved, the drug would be a new approach for tackling resistance to current treatments across Africa and beyond. The drug, KLU156, combines a novel compound, ganaplacide, with a new once-daily formulation of the existing antimalarial lumefantrine. It was developed in collaboration with the Swiss non-profit organisation Medicines for Malaria Venture and received funding from several European governments.

argenx SE achieved sales of $1.13 billion in the three months ended 30 September, a quarterly record for the company which has successfully exploited the therapeutic potential of a single biologic asset. Announced on 30 October, the quarterly sales were nearly double the $573 million generated a year earlier. They were helped by high demand for a prefilled syringe formulation of the lead product Vyvgart (efgartigimod). Vyvgart binds to the neonatal Fc receptor (FcRn), which is responsible for maintaining the circulation of immunoglobuline G (IgG) in the body.

Baxdrostat, a small molecule inhibitor of the hormone, aldosterone, achieved a statistically significant reduction in systolic blood pressure in a Phase 3 trial, confirming its efficacy as a treatment for patients with hard-to-control hypertension. The data were presented on 9 November at the American Heart Association meeting in New Orleans, US. The developer, AstraZeneca Plc, is expected to start regulatory submissions by the end of the year.

A photovoltaic retinal implant developed by Science Corporation has restored central vision in patients with advanced dry age-related macular degeneration (AMD), according to results from the multicentre PRIMAvera clinical study. 

After a robust third quarter, AstraZeneca Plc confirmed its revenue guidance for the year which is expected to increase by a high single-digit percentage. At the same time, it gave an update on products in late-stage development and a glimpse of new technologies on the horizon. Chief Executive Pascal Soriot outlined the company’s ambitions in a call with journalists on 6 November, which also included comments on new investment in the US.

A venture capital syndicate has mobilised $141 million in Series B financing for an Italian biotech which is developing two gene therapies for retinal disorders. The company, AAVantgarde Bio Srl of Milan, is developing therapies for Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B. Both conditions are inherited retinal diseases for which there are no approved therapies. AAVantgarde is a spin-off of the Telethon Institute for Genetics and Medicine, an international research institute based in Naples.

Ipsen SA is to acquire a privately-owned French biotech with an oncology product that has shown promise for patients with acute myeloid leukaemia (AML) not amenable to standard treatments. The target company, ImCheck Therapeutics SAS, has found of a way of simultaneously modulating the innate and adaptive immune systems with a monoclonal antibody, ICT01, that both identifies and kills cancer cells.

Novartis has signalled its growing interest in neuroscience and RNA technologies with the proposed acquisition of Avidity Biosciences Inc which has three late clinical-stage assets for genetic neuromuscular diseases. Novartis will pay approximately $12 billion in cash for the San Diego, US, company, in a transaction that is expected to close in the first half of 2026. Prior to the close, Avidity will spin-off earlier-stage programmes in cardiology into a new, separate company which could potentially be sold to a third party.