News

Scenic Biotech BV of the Netherlands is to work with a unit of the US National Institutes of Health to advance its therapeutic programme for the treatment of Niemann-Pick Type C (NPC) disease, a severe, progressive neurological disorder. Scenic is developing a small molecule compound for the disorder by identifying genetic modifiers. These are naturally occurring genes that compensate for mutations that are responsible for severe diseases. The goal of the collaboration is to get clinical insight into genes that might reduce the severity of NPC in patients.

In the wake of the Covid-19 pandemic, CEPI, a public-private partnership working to improve global access to new vaccines, is investing in manufacturing capacity in Africa. On 19 January, it announced an agreement with Institut Pasteur in Dakar, Senegal to invest up to $50 million over 10 years to expand capacity. This will include capacity to produce routine immunisation vaccines across multiple technologies, and reserve capacity to supply new vaccines in the event of an emergency. The countries covered by the agreement are in Africa, Latin America and the Caribbean.

AstraZeneca Plc continued to diversify its portfolio in 2022 with medicines for rare diseases contributing 16% of total revenue – the third largest component after oncology and drugs for cardiovascular and renal diseases. This followed the company’s $39 billion acquisition of Alexion Pharmaceuticals in July 2021, a deal that broadened its product profile and gave it a new footprint in the US. Work is underway to set up a new research site in Kendall Square, Cambridge, US, which would complement the company’s new facilities in Cambridge, UK.

Lundbeck A/S delivered revenue of DKK 18.2 billion ($2.62 billion) in 2022 supported by double digit growth for its four main products for neurological disorders. At the same time, it outlined plans for multiple investments, taking advantage of a period during which it expects a limited financial impact from patent expiries.

Long-term data from two Phase 3 trials of Cosentyx (secukinumab), an approved drug for plaque psoriasis and other inflammatory indications, have shown sustained efficacy and symptom improvement in patients with hidradenitis suppurativa (HS). HS is a recurrent skin disease causing abscesses that can lead to wounds and scarring. The two trials enrolled more than 1,000 patients in 40 countries.

The Roche Group has reported positive data from a Phase 3 programme of the monoclonal antibody crovalimab in people with paroxysmal nocturnal haemoglobinuria (PNH), a rare and life-threatening blood condition. PNH occurs when red blood cells are destroyed by the complement system, part of the innate immune system. Participants in the second of two studies had not been previously treated with complement inhibitors.

An antibody drug developed by Boehringer Ingelheim GmbH to prevent flares in generalised pustular psoriasis (GPP), a rare skin disease, has met its primary and key secondary endpoints in a Phase 2b trial of patients with the disease. Spesolimab (Spevigo) has already been approved for the treatment of the disease.

A new company in Scotland, UK has secured £2.4 million from KHAN Technology Transfer Fund I (KHAN-I) and existing investor Cumulus Oncology to pursue research into candidate molecules targeting DNA damage response. Nodus Oncology Ltd was founded in September 2022 to develop inhibitors of proteins involved in the repair of cells with DNA damage.

Evonetix Ltd, a UK synthetic biology company, has raised $24 million from a group of private investors to continue development of technology to enable the synthesis of DNA on a silicon chip. The latest funding is an extension of a Series B financing round that raised $30 million in 2020. The $54 million now available to the company will support development and commercialisation of the company’s semiconductor chips for multiple applications.

Diabetes drugs, especially the glucagon-like peptide-1 (GLP-1) medicines, drove sales at Novo Nordisk A/S to their highest level in five years giving a significant boost to both operating and net profits. Announcing its 2022 financial results on 1 February, the Danish company declared a total dividend for the year of DKK 12.40, up by 19% from a year earlier.

Net sales in 2022 were DKK 176.9 billion ($25.4 billion), up by 26%. At constant exchange rates the increase was 16%. Operating profit was DKK 74.8 billion, up by 28%, and represented 42.3% of sales.