Clinical Research

AstraZeneca Plc’s Bruton tyrosine kinase inhibitor Calquence, already approved in the US for mantle cell lymphoma, has delivered positive results in a Phase 3 trial of previously-treated patients with chronic lymphocytic leukaemia. The trial showed a statistically-significant and clinically-meaningful improvement in progression-free survival and will stop early, the company announced on 6 May. Calquence was administered as a monotherapy and compared with a combination of rituximab and a physician’s choice of either idelalisib or bendamustine.

MorphoSys AG has reported positive data from a single-arm Phase 2 study of its investigational monoclonal antibody tafasitamab with the cancer drug lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma. The drug, previously known as MOR208, is being prepared for a regulatory submission to the US Food and Drug Administration by the end of the year.

Another attempt to produce an effective therapy for glioblastoma multiforme (GBM), an aggressive brain cancer, has failed. This time it was a study testing the checkpoint antibody Opdivo (nivolumab) plus radiation in patients with newly diagnosed disease. The Phase 3 trial, known as CheckMate-498, did not meet its primary endpoint of overall survival at final analysis, according to the sponsor Bristol-Myers Squibb Co.

The US Food and Drug Administration has issued its first-ever draft guidance on the development of stimulant drugs to treat attention deficit hyperactivity disorder (ADHD), a condition whose symptoms include inattention and high levels of activity. While many of the recommendations are already in practice, the document gives clarification on the agency’s expectations for clinical trial design.

A Phase 3 trial investigating a new small molecule drug for advanced fibrosis failed to meet its primary endpoint, sending the developer Gilead Sciences Inc back to the drawing board. The drug, selonsertib, was being investigated in patients with stage 3 fibrosis due to non-alcoholic steatohepatitis (NASH), a progressive liver disease characterised by the accumulation of fat and inflammation in the liver.

A new player in the gamma delta T cell therapy arena has brought a cancer therapy to the clinic – this time for acute myeloid leukaemia. Glasgow, UK-based TC BioPharm Ltd has started a Phase 1 study of an allogeneic cell therapy consisting of activated and expanded gamma delta T cells.

The Belgian cell therapy company Promethera Biosciences SA has reported a positive safety profile and early efficacy signals from a Phase 2a study of its lead therapy for liver disease, HepaStem. HepaStem is an allogeneic therapy derived from the stem cells of healthy donors.

The stem cells are administered by infusion into the bloodstream of patients where they circulate until reaching the liver, settle and support regeneration.

Gilead Sciences Inc and Novo Nordisk A/S of Denmark have decided to pool their resources to test a new combination therapy for the treatment of nonalcoholic steatohepatitis, a chronic and progressive liver disease characterised by the accumulation of fat and inflammation in the liver.

Alnylam Pharmaceuticals Inc has reported positive Phase 3 data for a trial of its ribonucleic acid interference (RNAi) drug, givosiran, in patients with acute hepatic porphyria (AHP). AHP refers to a family of rare, genetic diseases characterised by potentially life-threatening attacks and for some patients, chronic debilitating symptoms that negative affect their quality of life.

UK-based Destiny Pharma Plc is stepping up the pace of development for its lead antibacterial XF-73 with the start of a 200-patient Phase 2b study in April. The topical treatment is being investigated for the prevention of post-surgical staphylococcal infections including methicillin resistant Staphylococcus aureus (MRSA), a major public health threat. XF-73 is administered as a nasal gel where it has been shown to reduce the nasal carriage of the S. aureus bacteria.