Pharmaceutical Market Europe • February 2026 • 7
NEWS
Sanofi announces positive results for venglustat in type 3 Gaucher disease
Sanofi has announced positive results from its study of venglustat in adults and children over 12 with neurological manifestations of type 3 Gaucher disease (GD3).
Gaucher disease is a rare, inherited lysosomal storage disease caused by deficiency of the glucocerebrosidase enzyme. This leads to molecules known as glycosphingolipids (GSLs) building up, especially in macrophages of the spleen, liver, bone marrow and lungs.
There are three main types of Gaucher disease. In GD3, neurological symptoms such as ataxia and cognitive issues are seen alongside the systemic manifestations that characterise GD1.
Enzyme replacement therapy (ERT) can be used to treat systemic symptoms of GD3, but there are currently no approved treatments for its neurological symptoms.
The LEAP2MONO study showed that venglustat improved both neurological and non-neurological outcomes. Improvements in neurological symptoms, which were the primary endpoint of the study, were measured at week 52. These scores were then compared with those of patients receiving ERT.
Venglustat was found to be well tolerated by participants, and no new safety signals were observed.
Skyhawk Therapeutics’ SKY-0515 shows positive results in Huntington’s disease
Skyhawk Therapeutics has announced positive interim results from its phase 1 trial of SKY-0515, an investigational treatment for Huntington’s disease.
The phase 1 clinical trial of SKY-0515 showed that patients receiving SKY-0515 had a mean Huntington’s Disease Rating Scale improvement from baseline of 0.64 points at nine months of treatment, where a worsening of 0.73 points would usually be expected. There was a dose-dependent reduction of mHTT protein in the blood of 62% at the 9mg dose, and a dose-dependent PMS1 mRNA reduction of 26%.
Huntington’s disease is a rare, hereditary neurodegenerative disorder affecting more than 40,000 patients in the US and hundreds of thousands globally. There are no approved treatments to inhibit the progression of the disease, which is ultimately fatal.
Ed Wild, professor of neurology at University College London, said: “These open-label trial results, due to be validated in the ongoing placebo-controlled FALCON-HD trial, give an expectation of meaningful impact for people living with HD across the world – for whom an orally administered HTT-lowering treatment such as SKY-0515 will be truly transformative.”
Novo Nordisk announces positive results for CagriSema in type 2 diabetes
Novo Nordisk has announced positive headline results from its trial of CagriSema (cagrilintide and semaglutide) in weight loss and blood glucose control.
Data from the phase 3 REIMAGINE 2 trial, part of the global REIMAGINE programme of clinical trials, showed that the weight loss and blood glucose control demonstrated by CagriSema at 68 weeks was superior to those offered by semaglutide. These results were consistent across all tested doses.
The trial evaluated 2,728 people living with type 2 diabetes who were inadequately controlled with metformin with or without an SGLT2 inhibitor. Around 40% of participants were treated with an SGLT2 inhibitor prior to trial initiation.
Those with a baseline body weight of 101kg lost an average of 14.2% after 68 weeks while using, with no weight loss plateau observed at week 68. This is compared to a weight loss of 10.2% for those using semaglutide 2.4mg.
In addition, blood glucose control improved by 1.9%, from a baseline of 8.2%, after 68 weeks for those using CagriSema 2.4mg, compared to 1.76% for those using semaglutide 2.4mg.
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