Clinical Research

Data from an early clinical trial of a small molecule drug intended to treat solid tumours by modulating an enzyme that plays a key role in the presentation of antigen to the human immune system were positive, according to the developer Grey Wolf Therapeutics Ltd. Data from the Phase 1/2 study were presented at the 2024 American Society of Clinical Oncology annual meeting on 3 June. The drug, GRWD5769 was well-tolerated after repeat dosing. It also showed an ability to modulate the immunopeptidome.

A gene therapy being investigated in a Phase 3 trial of boys with Duchenne muscular dystrophy (DMD) has failed to reach its primary endpoint of improvement in motor function, the developer Pfizer Inc announced on 12 June. The therapy, fordadistrogene movaparvovec, missed the primary endpoint at one year as well as key secondary endpoints compared with a placebo. The secondary endpoints included an improvement in velocity in a 10 metre run or walk. Participants were boys between the ages of four and seven who were on a daily regimen of glucocorticoids.

Eli Lilly and Co has reported positive data for tirzepatide, an approved drug for obesity and type 2 diabetes, which is now being investigated for metabolic dysfunction-associated steatohepatitis (MASH), a progressive liver disease. Formerly known as non-alcoholic steatohepatitis, MASH results from an excess of fat in the liver which can lead to liver-related complications and death. The global prevalence of the disease is increasing.

ITM Isotope Technologies Munich SE has raised €188 million from a group of investors to prepare for the commercial launch of its lead radiopharmaceutical product and increase its manufacturing capacity for radioisotopes. The equity financing was led by Temasek Holdings Ltd, a Singapore-based investment group, and supported by four other entities including the Qatar Investment Authority. ITM has a pipeline of candidate radiopharmaceuticals and diagnostics for cancer as well as manufacturing agreements to supply medical radioisotopes to others, including Novartis.

France-based SparingVision SA, which is developing gene therapies for retinal diseases, has presented data from an ongoing natural history study of rod-cone dystrophy showing the structural features of the disease and identifying a subset of patients with a higher rate of disease progression. This is to inform the company’s development of an ocular gene therapy for the disease which is in a separate Phase 1/2 trial in patients for severe rod-cone dystrophy.

ADC Therapeutics SA of Switzerland reached agreement with investors on 6 May to sell shares and warrants valued at $105 million to support its portfolio of antibody-drug conjugates for cancer. The new financing will lengthen the company’s cash runway enabling it to advance new indications for Zynlonta (loncastuximab tesirine), a marketed drug, and clinical-stage assets in its portfolio. ADC is developing antibody-drug conjugates for both haematological cancers and solid tumours. On 31 March it had cash and cash equivalents of $234.3 million compared with $278.6 million on 31 December 2023.

A gene therapy being developed by Regeneron Pharmaceuticals Inc has enabled a young child, suffering from a congenital hearing loss, to overcome deafness. The child, dosed at 11 months of age, is one of two to have responded to the experimental therapy which uses gene replacement to address mutations in the OTOF gene – the cause of the disease. A second child, who received the therapy at the age of four, showed initial improvements after dosing.

A confirmatory Phase 3 trial of a drug for patients with amyotrophic lateral sclerosis (ALS) failed to meet its primary and secondary endpoints, raising the possibility that the therapy, originally approved in 2022, could be withdrawn from the market. The developer, Amylyx Pharmaceuticals Inc, announced the trial results on 8 March, saying that a voluntary market withdrawal had not been ruled out. The therapy, AMX0035, is a combination of two small molecules, sodium phenylbutyrate and taurursodiol, which each target different neurodegenerative pathways.

NodThera Ltd said that its small molecule inhibitor of the NLRP3 inflammasome has shown evidence of reducing neuroinflammatory and inflammatory biomarkers in Parkinson’s disease. Data from a Phase 1b/2a a trial, disclosed on 7 March, showed that the drug, NT-0796, delivered mean reductions of these markers in the cerebrospinal fluid of elderly patients, bringing them to levels approximating those of healthy elderly controls.

Novo Nordisk A/S’s semaglutide (Ozempic), currently approved to reduce the risk of adverse cardiovascular events in adults with type 2 diabetes and cardiovascular disease, has achieved another milestone. This was evidence that the drug reduced the risk of kidney disease-related events by 24% in people with type 2 diabetes and chronic kidney disease. The data comes from the Phase 3 FLOW trial, which was stopped early in October 2023 due to efficacy. The headline results from this trial were released on 5 March.