Clinical Research

Positive long-term data have been reported for a cell therapy being tested in patients with retinitis pigmentosa, a group of hereditary diseases of the eye that can lead to a progressive loss of vision and blindness. Results from the ongoing US Phase 1/2a study of the human retinal progenitor cell therapy showed a meaningful clinical effect at all points in time out to 12 months after treatment.

The mode of action of an experimental treatment for septic shock has been confirmed in a Phase 2 study, paving the way for further development of the therapy in patients with an early stage of the disease. Septic shock is a life-threatening organ dysfunction caused by the body’s response to infection. The trial, AdrenOSS-2, enrolled 301 patients who were identified by a novel biomarker as having early septic shock and elevated blood levels of the peptide hormone adrenomedullin.

GlaxoSmithKline Plc has expanded further into cell therapy with a partnership aimed at generating drug candidates for solid tumours. The collaboration is with Immatics Biotechnologies GmbH of Germany which has been working for several years with the MD Anderson Cancer Center in Texas, US on engineering T cells to fight cancer.

Two experimental drugs that were being tested in people with dominantly inherited Alzheimer’s disease failed to meet their primary endpoint in a study sponsored by  Washington University School of Medicine in St. Louis, US.

Called DIAN-TU, the study was designed to find out whether either treatment could slow the rate of cognitive decline and improve disease-related biomarkers in people known to have a genetic mutation for inherited Alzheimer’s disease.

An antisense oligonucleotide directed against a non-coding microRNA in cardiac tissue has shown an ability to reverse heart failure in preclinical studies involving a variety of animal models including a clinically relevant pig model of heart failure. The results of the study were reported in Nature Communications on 31 January 2020. The lead author is Ariana Foinquinos of Hannover Medical School in Germany.

A combination of the anti-platelet medicine Brilinta (ticagrelor) with aspirin can reduce the risk of stroke and death in patients with minor acute ischaemic stroke or at high risk of a transient ischaemic attack, according to AstraZeneca Plc. Data from a Phase 3 trial involving more than 11,000 patients showed a statistically significant and clinically meaningful reduction in the risk of stroke for those who received Brilinta and aspirin compared with aspirin alone.

A Phase 3 trial of a lipid-lowering drug involving more than 13,000 patients has been discontinued after the study’s monitors concluded the medicine was unlikely to deliver benefits to patients. The sponsor, AstraZeneca Plc, was attempting to show that patients with mixed dyslipidaemia who are at risk of cardiovascular disease could be helped by Epanova (omega-3 carboxylic acids), a compound derived from fish oil. The patients were all on optimal statin therapy.

Germany-based Medigene AG has reported positive top-line data from an early clinical study of a dendritic cell vaccine for cancer which showed both safety and tolerability as well as early signs of efficacy. The vaccine was administered to 20 patients with acute myeloid leukaemia, of whom half were 60 years of age or older. This patient cohort is often ineligible for haematopoietic stem cell transplantation and has poorer treatment outcomes, the company said.

A Phase 3 study of the checkpoint antibody Tecentriq (atezolizumab) did not meet its primary endpoint of disease-free survival compared with observation in patients with muscle-invasive urothelial cancer, the developer Roche announced on 24 January.

An experimental treatment for Type 1 spinal muscular atrophy (SMA) has reached its primary endpoint in a pivotal trial of infants, paving the way for a US regulatory decision by late May, according to the developer Roche. The drug, risdiplam, is a small molecule that works by increasing the amount of protein produced by the survival motor neuron-2 (SMN2) gene.