News

NodThera Ltd of the UK, with offices in Boston and Seattle, US, has reported data from a small molecule drug being tested in volunteers which showed reductions in neuroinflammatory and inflammatory biomarkers – the first human data from a project intended to treat Parkinson’s disease. The results were reported on 11 July. The drug, NT-0796, was administered to elderly volunteers and within seven days, showed a decline in biomarkers including the cerebrospinal fluid (CSF) neurofilament light chain (NfL), a key indicator of neurodegeneration.

Twenty years after its launch as a nanomedicines company, Nanobiotix SA of France has secured a major licensing deal enabling it to complete development and commercialise a radioenhancer for use with radiotherapy to treat patients with multiple, difficult cancers. The agreement with Janssen Pharmaceutica NV, part of Johnson & Johnson Inc, was announced on 10 July. It carries potential payments of up to $1.8 billion in development, regulatory and sales milestones as well as royalties on net sales of the product NBTXR3.

The European Medicines Agency is reviewing the safety of three medicines used for the treatment of weight loss and type 2 diabetes in light of reports from the Icelandic Medicines Agency of suicidal thoughts and attempted self-injury amongst patients taking the drugs. The review will focus on the glucagon-like peptide 1 (GLP-1) receptor agonists liraglutide (Saxenda) and semaglutide (Ozempic and Wegovy), all three of which have been developed by Novo Nordisk A/S. The investigation will involve an analysis of 150 reports of possible self-harm and suicidal thoughts.

The first gene therapy to treat severe haemophilia A was approved by the US Food and Drug Administration on 30 June, adding a novel treatment to the existing group of drugs for the bleeding disorder. The therapy, Roctavian, is an adeno-associated virus (AAV) vector-based therapy intended to be used as a one-time treatment. It is administered as a single dose by intravenous infusion.

AstraZeneca Plc said that its antibody-drug conjugate (ADC) datopotamab deruxtecan generated positive data from patients in a Phase 3 lung cancer trial, but that some Grade 5 events were observed in the study. A spokesperson for AstraZeneca said on 11 July that some Grade 5 interstitial lung disease events were observed which are being investigated. Overall the safety profile of the drug was consistent with previous studies, she added.

The US Food and Drug Administration approved the first allogeneic cell therapy for type 1 diabetes on 28 June, making it possible for people with severe hypoglycaemia to receive an additional treatment for achieving targeted blood glucose levels. Lantidra is an allogeneic pancreatic islet cell therapy made from deceased donor pancreatic cells.

Eli Lilly and Co is to expand its pharmaceutical pipeline with the acquisition of Sigilon Therapeutics Inc of Cambridge, US, which has a pre-clinical asset for type 1 diabetes. The two companies have been developing the asset, an encapsulated cell therapy called SIG-002, since 2018. The product is in lead optimisation and poised to enter clinical development.

The US Food and Drug Administration has awarded a rare paediatric disease designation to a subsidiary of Nippon Shinyaku Co Ltd for an experimental treatment for Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive weakness and the loss of skeletal, cardiac and respiratory muscles. It occurs primarily in males.

Leqembi (lecanemab), a monoclonal antibody that binds to beta-amyloid protofibrils, was approved by the US Food and Drug Administration for Alzheimer’s disease on 6 July, the first drug to show clinical benefit in patients with a mild stage of the disease. The full approval comes six months after Leqembi was given an accelerated authorisation on 6 January which was based on a surrogate endpoint – reducing amyloid plaques in the brain.

AAVantgarde Bio Srl of Italy has raised €61 million in a Series A financing to develop genetic medicines for inherited retinal diseases. The financing was co-led by Atlas Venture of the US and Netherlands-based Forbion.