Clinical Research

An RNA oligonucleotide drug that is being tested as a combination therapy in patients with pancreatic and colorectal cancers has produced positive data in a Phase 1 clinical study. Top-line results from the combination of NOX-A12 (olaptesed pegol) and Keytruda produced responses from patients whose cancers had progressed rapidly on prior therapy.

A Chinese scientist’s reported use of the Crispr gene editing tool to alter a gene in two  embryos which were implanted into a mother’s womb has created an uproar in the scientific community. The edit was performed when the embryos were just a day old and involved alterations to the CCR5 gene in order to prevent the offspring from contracting HIV. They were reported on YouTube videos, rather in a scientific journal, after twin girls bearing the DNA changes were born.

Immunocore Ltd is expanding a 2013 collaboration with Genentech (Roche) to co-develop a bi-specific biologic for cancer. The compound is based on soluble T cell receptors that have been designed to recognize intracellular cancer antigens, and then kill cancer cells through an anti-CD3 effector function.

AstraZeneca Plc’s Phase 3 trial of the Imfinzi and tremelimumab checkpoint antibodies did not meet the primary endpoint of improving overall survival compared with standard-of-care chemotherapy in previously untreated patients with Stage IV metastatic non-small cell lung cancer, the company announced on 16 November. This follows the failure of the same combination in July 2017 to reach statistical significance in progression-free survival.

AstraZeneca Plc has reported positive outcome data for its licensed diabetes medicine Farxiga (dapagliflozin) showing that the drug significantly reduces the risk of hospitalisation for heart failure or cardiovascular death among patients with Type 2 diabetes. In a Phase 3 trial, the reduction of risk was 17% compared with a placebo.

A prospective treatment for patients with schizophrenia, who have not responded to other drugs, failed to show statistical superiority in a Phase 3 trial compared with conventional therapy, H. Lundbeck A/S announced on 25 October.

AbbVie Inc is to take full control of a joint cystic fibrosis (CF) research and development programme with Galapagos NV with the goal of bringing a triple combination CF therapy to the market. Currently in early clinical development, the programme consists of potentiator and corrector drug molecules that are intended to increase the activity of mutated copies of the cystic fibrosis transmembrane conductance regulator protein that causes the disease.

Positive data from two Phase 2 studies of filgotinib, a Janus kinase 1 inhibitor being developed for several inflammatory conditions, were published online on 22 October 2018 in The Lancet, Gilead Sciences Inc and Galapagos NV announced.

An enzyme therapy developed by Hansa Medical AB has successfully inactivated donor-specific antibodies in patients with kidney disease enabling them to receive kidney transplants. The result comes from follow-up data from two Phase 2 trials of highly sensitised patients who otherwise would not have been eligible for transplantation.

Nightstar Therapeutics Plc has reported positive Phase 1/2 data from a trial of its gene therapy for X-linked retinitis pigmentosa, a rare inherited retinal disorder primarily affecting males. The 18-patient study showed preliminary evidence of improved visual function.