A Phase 2a study that was evaluating a potential treatment for paediatric patients with non-alcoholic fatty liver disease was stopped early in August after the first three patients showed an increase in liver fat and elevations in serum alanine transaminase (ALT) levels, raising concerns about liver toxicity.
A study published in the journal Clinical Cancer Research concludes that elevated levels of kidney injury molecule-1 (KIM-1), a transmembrane protein in blood plasma, can predict renal cell carcinoma (RCC) incidence up to five years before a diagnosis.
An early warning of kidney cancer could make the difference between life and death. When diagnosed early, eight out of 10 people will survive for five years or more. But if the disease is diagnosed late, only one out of 10 survive, according to the charity Cancer Research UK.
Oxford BioMedica Plc has teamed up with two UK partners to produce a gene therapy for cystic fibrosis that could be administered to patients through repeat dosing. Boehringer Ingelheim GmbH has an option to commercialise the product.
The small molecule drug, laquinimod, has failed to meet the primary endpoint in a Phase 2 study of patients with Huntington’s disease, a rare neurodegenerative disease. In May 2017 the same drug failed a Phase 3 trial in multiple sclerosis.
The drug is being developed by Teva Pharmaceutical Industries Ltd under licence from Active Biotech AB of Sweden. It is an immunomodulatory therapy that aims to prevent neurodegeneration and inflammation in the central nervous system.
Researchers at the Wellcome Sanger Institute in the UK have reported unexpected genetic changes in mouse embryonic stem cells following the use of the CRISPR-Cas9 gene editing tool. They warn that the scientific community may be overestimating the potential for gene editing as a human therapy, and urge that more work be done to better understand the safety profile of the technology.
An international research group has reported promising data from an early clinical trial of a prophylactic vaccine against HIV-1 in healthy volunteers and a parallel study of the same vaccine in rhesus monkeys. The study results were published online in The Lancet on 6 July.
A prospective drug for Alzheimer’s disease that failed to show efficacy in late 2017 based on a Bayesian analysis of patient data, has now demonstrated its promise using more conventional statistical methods, according to Eisai Co Ltd and Biogen Inc.
The drug, BAN2401, is a monoclonal antibody targeting beta-amyloid, protein fragments in the brain which are thought to contribute to the neurodegenerative process in Alzheimer’s disease.
Ipsen SA received further validation of its in-licensed compound cabozantinib following the publication in the 5 July edition of the New England Journal of Medicine (NEJM) of Phase 3 data showing the drug’s effectiveness in advanced liver cancer. Cabozantinib, which trades commercially as Cabometyx, has already been approved in the US and Europe for advanced kidney cancer. The drug was developed by Exelixis Inc and out-licensed to Ipsen in 2016 for commercialisation outside the US, Canada and Japan. Ipsen later acquired rights to Canada.
A candidate vaccine to prevent infection from the chikungunya virus has been accepted into the European Medicines Agency’s PRIME scheme, a programme of regulatory support for developers of medicines that meet a major public health need.
The vaccine is a biologic developed by Themis Bioscience GmbH of Austria and is currently undergoing Phase 2 studies in 600 patients across the US, the EU and South and Central America. The company hopes to receive marketing approval within three years.
Valneva SE is poised to bring its prophylactic vaccine for Lyme disease into a Phase 2 clinical study by the end of 2018 following positive discussions with the US Food and Drug Administration, the company announced on 2 July. The agency has given the product ‘fast track’ status in light of its potential contribution to public health.