Next-gen, effective payloads – from AI-designed small molecules to RNA-based therapeutics and gene therapies – have revolutionized medicine. Yet, the main hurdle remains targeted delivery: avoiding immune reactions (seen with many viral vectors) or undesired organ accumulation (evidenced with LNPs in the liver). Scientists are now harnessing a technology that could overcome these hurdles by using vesicles that cells naturally produce: exosomes.
Switzerland-based Pharvaris’ once-daily oral deucrictibant XR reduced hereditary angioedema (HAE) attacks by 83% versus placebo in a pivotal Phase 3 trial, putting the experimental prophylactic into roughly the same efficacy range as several leading injected treatments.
Novartis has suffered another pipeline setback after del-desiran, an antibody-oligonucleotide conjugate acquired with Avidity Biosciences, failed to significantly improve hand function over placebo in the Phase 3 HARBOR study in myotonic dystrophy type 1.
North Rhine-Westphalia (NRW), a state located in the North-West of Germany, is investing €200 million to connect university hospitals in Aachen, Bochum, Bonn, Düsseldorf, Essen, Cologne and Münster, as well as the Technical University Dortmund in an AI network for cancer medicine.
Spanish biotech Vaxdyn has raised an €11 million Series A to take K-VAX, its vaccine against Klebsiella pneumoniae and Acinetobacter baumannii, into a first-in-human Phase 1 trial. Portuguese investor Biovance Capital led the round with a €4 million investment, joined by the European Innovation Council (EIC) Fund and the Impacto Andalusia fund managed by Arcano Partners.
Copenhagen’s BioInnovation Institute (BII) is providing six early-stage companies with €1.3 million each in convertible loans, taking its total financing of each startup to €1.8 million.
Novartis’ pelacarsen failed to reduce cardiovascular events in the first Phase 3 outcomes trial of an Lp(a)-lowering drug, despite successfully lowering the genetically driven cardiovascular risk factor.
Novartis could pay up to $3.223 billion (€2.8 billion) for access to Alteogen’s technology for turning intravenous biologics into subcutaneous drugs. Behind the eye-catching figure is more than patient convenience: Drug delivery is becoming an increasingly important tool for lifecycle management and emerging drug classes.
Italian pharma Menarini Group has licensed European rights to bofanglutide for obesity and excess weight, a long-acting GLP-1 receptor agonist from China’s Gan & Lee Pharmaceuticals dosed once every two weeks, for €62 million upfront, up to €664 million in milestones, and “double-digit” royalties. Menarini will handle regulatory submissions and commercialization across 39 European markets.
French biotech Transgene said all 16 head and neck cancer patients treated with its personalized cancer vaccine TG4050 in a randomized Phase 1 trial remained disease-free after three years, while three of 16 patients in the control arm relapsed.