A digital twin lung simulation technology, Twinhale, has been used for the first time in idiopathic pulmonary fibrosis (IPF) in a groundbreaking new study. The study by Munich, Germany-based company Ebenbuild, supports the development of a novel inhaled anti-CCN2 (formerly called CTGF) biologic, PRS-220, developed by Pieris Pharmaceuticals - now Pavella Therapeutics Inc.
A new drug for haemophilia that targets antithrombin, a protein that inhibits blood clotting, has been approved by the US Food and Drug Administration. Discovered by Alnylam Pharmaceuticals Inc and licensed to Sanofi SA, Qfitlia (fitusiran) will be used to treat patients with both haemophilia A and haemophilia B with or without inhibitors. The standard treatment for patients with haemophilia are clotting factor replacement therapies where patients with haemophilia A receive replacement for factor VIII and those with haemophilia B, replacement for factor IX.
Effective 7 April, the US Food and Drug Administration had lost 28 senior leaders who left the agency voluntarily or were fired following the Trump Administration’ decision to reduce the workforce by 3,500 people. The figures, compiled by the STAT news agency, show that departures included deputy directors and commissioners across all departments from the centres for devices and radiological health, to the centres for small molecule and biologic evaluation, and for tobacco regulation.
The US Food and Drug Administration announced plans on 10 April to phase out animal testing for monoclonal antibodies and other drugs – a safety requirement for the development of human medicines that was first introduced in 1938. The reason is to adopt new methods for evaluating safety that could harness technologies such as artificial intelligence while reducing research and development costs.
Only hours before announcing countermeasures against prospective US tariffs on steel and aluminium, the European Commission has pulled back in response to a pause from the Trump Administration. The US announced a 90-day pause on its tariff measures on 9 April. The pause affects a second tier of tariffs that would have driven the tax on exports by the US’ trading partners up by double-digit figures. The pause does not affect China.
Isomorphic Labs Ltd, a pioneer in the use of artificial intelligence for drug discovery, announced a capital raise of $600 million on 31 March, the company’s first external financing since its launch in 2021. Isomorphic is a spin-out of the DeepMind laboratory of Alphabet Inc, the parent of Google, and led by Demis Hassabis, a Nobel laureate. DeepMind is the developer of the AlphaFold artificial intelligence programme. Isomorphic will use the new funds to develop a portfolio of drugs both in-house and in partnership with Novartis and Eli Lilly and Co.
The European Union member states have agreed to enact countermeasures against US tariffs on steel and aluminium from 15 April should negotiations over trade fail to reach a fair and balanced outcome by that time, the European Commission announced on 9 April. The vote by member states gave support to a proposal from the Commission in response to a US decision in March to impose import duties on the two products.
The UK biotech Epsilogen has significantly expanded its repertoire of antibody technologies with the acquisition of TigaTx Inc, of Boston, US, in order to treat more patients with cancer. The deal, announced on 7 April, equips Epsilogen, a clinical-stage company, with technology and assets of the IgA antibody subtype. These activate neutrophils, a type of white blood cell that is an essential part of the immune system. Epsilogen already has IgG antibody assets, the most abundant subtype, and IgE assets, a less common isotype which can activate macrophages.
GSK Plc has entered into a licensing agreement with ABL Bio Inc of South Korea for rights to a delivery platform for carrying prospective drugs across the blood-brain barrier to treat neurodegenerative diseases. The deal, valued at up to £2.15 billion, is one of the company’s largest transactions, reflecting its interest in treating diseases of the brain.
Arvinas Inc, a US biotech with a portfolio of protein degrader molecules, has reported positive data for a potential treatment for neurological diseases. The data were presented at the International Conference on Alzheimer’s and Parkinson’s Diseases in Vienna, Austria on 4 April, showing that the molecule was both safe and able to engage with the cellular target. The target is leucine-rich repeat kinase 2 (LRRK2), a protein that has been implicated in Parkinson’s disease and progressive supranuclear palsy. The Phase 1 study was conducted in healthy male volunteers.