Shire Plc is advancing a candidate gene therapy acquired from its 2016 purchase of Baxalta, with plans to take the product into a first human trial in patients with haemophilia A. The product is a factor VIII gene therapy delivered in an adeno-associated virus (AAV) vector.
Three multiple myeloma studies in which Merck & Co’s checkpoint inhibitor Keytruda (pembrolizumab) was being investigated in combination with drugs produced by Celgene Corp have been stopped following patient deaths, Merck announced on 5 July.
The term biopsy has traditionally been associated with the examination of tissue from a patient in order to detect the presence of disease. Tissue samples can be taken from the bone marrow, or organs such as the bladder, lung or liver, using procedures of varying complexity from the insertion of a needle into the skin, to surgery.
Epidiolex, a cannabinoid drug for a type of childhood epilepsy known as Dravet syndrome has reduced the frequency of seizures in a Phase 3 trial of children and adolescents with the disease, all of whom were not getting relief from current treatments.
The Roche Group’s checkpoint antibody, Tecentriq (atezolizumab), has missed the primary endpoint of overall survival in a Phase 3 trial, designed to confirm the drug’s efficacy in urothelial cancer and convert an earlier accelerated approval in the US to a full approval.
The first of two pivotal Phase 3 trials of a new anti-interleukin-13 drug for severe asthma has failed to meets its primary endpoint of a significant reduction in the annual asthma exacerbation rate, AstraZeneca Plc announced on 10 May.
Poxel SA has reported positive data from a Phase 2b study in Japan of imeglimin, its treatment for Type 2 diabetes. The primary endpoint of the trial was met, and was more pronounced in the Japanese patients than had been the case in similar Phase 2b trials in the US and the EU.
A Phase 3 trial of an experimental drug for relapsing-remitting multiple sclerosis, laquinimod, has failed to meet its primary endpoint which was to show an improvement in the time to confirmed disease progression.
Roche said that its kinase inhibitor Alecensa (alectinib) has produced better results in a Phase 3 trial of patients with a specific type of lung cancer than a rival treatment marketed by Pfizer Inc.
Sweden-based Medivir AB has reported positive Phase 2 data for a recently acquired histone deacetylase (HDAC) inhibitor in a trial of patients with early-stage cutaneous T cell lymphoma (CTCL), a haematologic cancer that presents in the skin.