Clinical Research

US-based ReCode Therapeutics Inc has strengthened its ties with the Cystic Fibrosis Foundation as it progresses an experimental messenger RNA (mRNA) therapy for the disease through the clinic. Simultaneously, it promoted Heather Clark on 1 July to the role of chief executive from vice president and head of the company’s cystic fibrosis franchise. Ms Clark joined ReCode in 2022. She has nearly 30 years of experience in rare disease drug development of which more than half were spent at Vertex Pharmaceuticals Inc, developer of five marketed cystic fibrosis products.

The US Food and Drug Administration granted an accelerated approval on 19 August for a new gene therapy for patients with an inherited disorder caused by the shortage of an enzyme needed to maintain stable blood sugar levels in the body. The therapy, Genglycos (pariglasgene brecaparvovec), is the first treatment to be authorised for the disorder – glycogen storage disease type la. Also called von Gierke disease, the disorder results in the  build-up of glycogen, a complex sugar, in the body’s cells.

A messenger RNA (mRNA)-based therapy designed for cancer has delivered positive Phase 3 results in patients with resected, advanced myeloma representing a first of its kind treatment in oncology. Announced on 19 August, the trial, INTerpath-001, delivered recurrence-free survival and distant metastasis-free survival for patients who had tumours removed but were still at risk of disease. The trial enrolled 1,137 patients who were randomised to receive intismeran, the mRNA therapy, and Keytruda (pembrolizumab) or Keytruda alone.

AstraZeneca Plc reported two positive outcomes and one negative outcome for three Phase 3 lung cancer trials on 17 August. The trials were all testing treatments for non-small cell lung cancer, the most prevalent type, which makes up about 85% of all lung cancers globally, according to the World Health Organization. The largest trial, which was testing a dual checkpoint inhibitor bispecific antibody, failed to show efficacy. However the two other studies, targeting cancers with specific genetic mutations, achieved statistically significant and clinically meaningful results.

Efgartigimod, the antibody treatment being developed by argenx SE for a spectrum of autoimmune diseases, leapt over another hurdle on 17 August with news that it had met the primary endpoint in a trial of two rare muscle diseases. The Phase 3 study showed that the antibody fragment, in combination with hyaluronidase enzymes, was able to restore the strength and muscle function in patients with two types of myositis: immune-mediated necrotising myopathy (IMNM) and dermatomyositis (DM). Both diseases cause muscle weakness leading potentially to long-term disability.

Switzerland-based Vaderis Therapeutics AG has raised $152 million from a Series B financing round to support development of a small molecule drug, engasertib, for a rare vascular disease for which no treatments are currently approved. The disease is hereditary haemorrhagic telangiectasia (HHT), a genetic disorder affecting the vasculature, which manifests in nose bleeds, anaemia, and visceral arteriovenous malformations (AVMs) AVMs are tangles of blood vessels that can cause internal bleeding and organ damage.

Netherlands-based AdJane Holding BV has reported the first clinical data for a new vaccine platform that is being positioned as a technology for use in preparing for future pandemics. The early data showed that outer membrane vesicles, which are vesicles released from the outer membranes of Gram-negative bacteria, were safe and well-tolerated in 40 healthy adults. The vaccine, based on outer membrane vesicles, was administered intranasally and combined with a SARS-CoV-2 spike protein- the structural component of the virus that caused the Covid-19 pandemic.

GSK Plc has stopped development of a small molecule drug for chronic cough following only limited efficacy for the product in a Phase 3 programme. However the drug will continue to be evaluated for irritable bowel syndrome. The drug, camlipixant, entered GSK’s portfolio with the acquisition of the Canadian company, BELLUS Health Inc in 2023. At the time, the acquisition was valued at $2 billion. Camlipixant is an antagonist of the P2X3 receptor, a type of receptor primarily found in sensory neurons.

The US Food and Drug Administration issued new draft guidance on 29 May about how companies can reduce the use of animals in drug testing – this time for cancer medicines. Entitled, Oncology Pharmaceuticals: Streamlined Nonclinical Safety Studies for Biologics and Conjugated Products, the guidance recommends new criteria for general toxicology studies, including when animal testing may be unnecessary because there is no binding or pharmacologic activity.

The European Medicines Agency, in coordination with the African Medicines Agency and national regulators in Africa, started talks in early June about organising clinical trials to evaluate possible vaccines and therapies against Ebola disease caused by the Bundibugyo virus. The virus is currently circulating in the Democratic Republic of Congo and Uganda and was declared a public health emergency of international concern by the World Health Organization on 17 May. As of 18 June, the WHO had reported just over 900 cases of the disease and approximately 235 deaths.