Clinical Research

Data from a Phase 3 trial of a combination vaccine candidate to prevent meningococcal disease, a bacterial infection that can cause death or permanent disability, were positive, according to the developer GSK Plc. The vaccine candidate showed non-inferiority in primary endpoints for five Neisseria meningitidis serogroups compared with Bexsero and Menveo, two marketed GSK vaccines, which together cover the same serogroups. The five Neisseria meningitidis serogroups account for nearly all invasive meningococcal disease cases in the world.

Gene editing technology has been used by an international team of researchers to create a new therapeutic that targets Escherichia coli in the guts of patients with blood cancers who are at risk of infection. A preclinical study of the new therapy was published in Nature Biotechnology on 4 May. The therapy, SNIPR001, was developed by Denmark-based SNIPR Biome ApS which is currently conducting a Phase 1 trial of the drug in the US. The drug has received a fast track designation from the Food and Drug Administration.

Preclinical data for the small molecule drug vidofludimus calcium has revealed its activity in relation to a neuroprotective transcription factor in addition to its ability to inhibit an enzyme which plays a key role in the metabolism of overactive immune cells and virus-infected cells, Immunic Inc announced on 17 May. The findings have been published in the Journal of Medicinal Chemistry.

Fenebrutinib, a small molecule drug being investigated for multiple sclerosis, was able to reduce brain lesions in people with the disease, the developer Roche announced on 17 May. The results were obtained from a Phase 2 trial of fenebrutinib, a Bruton’s tyrosine kinase inhibitor, which is being studied in adults with relapsing forms of the disease. Data from the study showed that fenebrutinib significantly reduced the total number of new gadolinium-enhancing T1 brain lesions compared with a placebo, the primary endpoint of the trial.

H. Lundbeck A/S scored two important regulatory goals in April and May, just weeks after closing its books for the first quarter, a period during which it produced its highest three-month revenue figure ever. Revenue was DKK 5 billion (€670 million) for the period, up by 15% from a year earlier.

Eli Lilly and Co reported positive results for its Alzheimer’s disease drug donanemab on 3 May saying that the Phase 3 TRAILBLAZER-ALZ 2 trial met the primary and all secondary endpoints measuring cognitive and functional decline. The company expects to make a regulatory submission to the US Food and Drug Administration by 30 June. Lilly submitted a regulatory application for donanemab to the FDA in January but the agency turned it back citing insufficient data.

Just a year after closing a €60 million Series B financing, Tubulis GmbH of Germany has concluded a licensing agreement with Bristol Myers Squibb Company to develop antibody-drug conjugates (ADC). Announced on 20 April, the deal will give BMS rights to Tubulis’ technology platform to develop ADCs for solid tumours. The number of products to be developed wasn’t disclosed.

The Food and Drugs Authority (FDA) of Ghana has approved a vaccine developed by the University of Oxford, UK, to prevent malaria – the first regulatory clearance for the product globally. The vaccine, R21/Matrix-M, is a subunit vaccine made up of a protein secreted by the malaria parasite and fused to a fragment of the hepatitis B virus. It is delivered alongside a plant-based adjuvant that enhances a person’s immune response. The adjuvant was developed by Novavax Inc of the US. The vaccine is being manufactured and scaled up by the Serum Institute of India PvT Ltd.

Dupixent (dupilumab), a monoclonal antibody approved to treat asthma, atopic dermatitis and three other inflammatory diseases, has now been shown to be effective in chronic obstructive pulmonary disease (COPD), the third leading cause of death in the world. Results of a Phase 3 trial of the drug showed that it achieved a 30% reduction in moderate or severe acute exacerbations in COPD, Sanofi SA announced on 23 March. Dupixent is being jointly developed by Sanofi and Regeneron Pharmaceuticals Inc.

Acesion Pharma ApS of Denmark has reported that proof-of-mechanism has been achieved in a Phase 2 trial of patients with atrial fibrillation who were treated with its SK ion channel inhibitor. The drug, AP30663 is intended to convert atrial fibrillation, the most common cardiac arrhythmia, into a normal sinus rhythm. This is achieved through SK channel inhibition.