Clinical Research

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.

The first trial, called EQUATOR, is in patients with psoriatic arthritis. The second, TORTUGA, is in patients with ankylosing spondylitis, a form of arthritis that primarily affects the spine.

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.

Galapagos NV has raised $300 million in a secondary offering on Nasdaq two days after the Belgian company and its partner Gilead Sciences Inc announced positive Phase 3 results for a new JAK1 inhibitor to treat rheumatoid arthritis.

Nicox has begun a Phase 2 study of a candidate treatment for glaucoma designed to secure a position in global ophthalmology. The treatment, NCX 470, is being tested in patients with open-angle glaucoma, the most common form of glaucoma, accounting for about 90% of all cases.

AstraZeneca Plc has announced that a Phase 3 trial of its proposed antibody treatment for systemic lupus erythematosus (SLE) failed to meet the trial’s primary endpoint of showing a statistically-significant reduction in disease activity as measured at 12 months by a responder index. Called TULIP 1, the trial was a double-blinded, 52-week placebo-controlled study evaluating the safety and efficacy of anifrolumab as a treatment for adult patients with moderate-to-severe SLE.

Pfizer has stopped two clinical studies that were evaluating a proposed antibody treatment for Duchenne muscular dystrophy (DMD) because of lack of efficacy. The first study was a Phase 2 trial testing the treatment for safety and efficacy, while the second was an open-label extension study, the company announced on 30 August.

The glucagon-like peptide-1 (GLP-1) analogue semaglutide has demonstrated an ability to promote weight loss in people without diabetes, in some cases up to 13.8%, when combined with diet and exercise. The data comes from a Phase 2 trial comparing semaglutide with liraglutide, another GLP-1 analogue, and with placebo.

Novartis has reported positive Phase 3 data from a trial investigating a new treatment for breast cancer targeting a protein in the PI3K pathway, a signalling pathway for human cancers. The compound, BYL719 (alpelisib), showed an improvement in progression-free survival.