News

GSK Plc is to develop oligonucleotide therapeutics under a new collaboration with the genetic medicines company Wave Life Sciences Ltd. The agreement, which includes an upfront payment of $170 million in cash and equity as well as future milestone payments, involves the discovery and development of medicines based on genetic targets.

The oligonucleotide medicines are short DNA or RNA molecules that can modulate RNA through several mechanisms. The primary function of RNA is to create proteins via translation. The Wave technology is capable of editing, splicing and silencing RNA.

CureVac NV and its partner GSK Plc have reported promising preliminary data from ongoing Phase 1 studies of vaccine candidates directed against both Covid-19 and influenza. The experimental medicines are based on messenger RNA. The results showed that vaccine candidates using a modified second-generation mRNA backbone produced promising immunogenicity and reactogenicity profiles in both indications. The modified vaccines are directed against specific antigens.

Lecanemab, a monoclonal antibody drug to treat Alzheimer’s disease, was given an accelerated approval by the US Food and Drug Administration on 6 January, based on evidence that it reduced amyloid plaque, a marker for the disease. Known commercially as Leqembi, the drug is only the second antibody treatment to be given a preliminary authorisation to treat the disease, which is an irreversible, progressive brain disorder affecting millions of people. The first was Aduhelm (aducanumab), which also targets amyloid plaque.

The developers of the first chimeric antigen receptor (CAR) T cell therapies are to merge their assets under a deal that they say will speed up manufacturing and create new opportunities for treating solid tumours. The deal involves the acquisition of Tmunity Therapeutics Inc of Philadelphia, US, by Kite Pharma, a business unit of Gilead Sciences Inc. The financial terms of the transaction were not disclosed.

Switzerland-based MetrioPharm AG has reported top-line data for a candidate treatment for Covid-19, showing that the compound is as effective as two approved antiviral medicines for the disease. The drug, MP1032, is a small molecule macrophage metabolic modulator which targets activated macrophages. It acts by normalising oxidation-reduction reactions at a cellular level which is essential for the body’s immune response to infections and autoimmune and inflammatory diseases.

A second Phase 3 trial of ensifentrine, a candidate product for chronic obstructive pulmonary disease (COPD), has met its primary endpoint, paving the way for a regulatory submission in the US in the first half of 2023, the developer Verona Pharma Plc announced on 20 December. Ensifentrine is a dual inhibitor of the enzymes phosphodiesterase 3 and 4, a mode of action that enables it to combine both bronchodilator and anti-inflammatory effects in a single compound.

The US Food and Drug Administration has approved a gene therapy for bladder cancer, the second authorisation of a novel gene treatment within a month. The new drug, Adstiladrin (nadofaragene firadenovec), is indicated for the treatment of patients with bladder cancer which hasn’t yet invaded the muscle and is unresponsive to the Bacillus Calmette-Guérin (BCG) immunotherapy. The decision comes not long after the agency approved a gene therapy for haemophilia B.

An experimental messenger RNA (mRNA) cancer vaccine has generated positive data in combination with Keytruda (pembrolizumab) in patients with advanced melanoma, the developers Moderna Inc and Merck & Co announced on 13 December.

Plans to develop a candidate therapy for solid tumours using a T cell receptor (TCR) approach were disclosed on 2 December by Immunocore Holdings Plc, which has concluded an oncology collaboration with Gadeta BV of the Netherlands. The two companies are at different stages of development, but they share an interest in using TCRs as agents for fighting disease.

TCRs are a group of proteins found on T cells that bind to certain antigens on cancer cells causing T cells to attack the malignancy.

An allogeneic dendritic cell vaccine for patients who have residual cancer after previous successful treatments with chemotherapy has shown positive survival data, according to the developer Mendus AB. The treatment, DCP-001, is being evaluated in a Phase 2 trial as a maintenance therapy in acute myeloid leukaemia (AML) for patients with measurable residual disease. Fresh data from the trial, ADVANCE II, was reported at the American Society of Hematology Annual Meeting on 12 December.