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New strategy for Vivoryon as second failed Alzheimer’s trial shows kidney promise

Varoglutamstat was being considered in the treatment of early Alzheimer’s disease, but Vivoryon will now consider it in kidney treatments. Credit: Max Acronym / Shutterstock.

Vivoryon Therapeutics has shared the results of its VIVA-MIND Phase IIa trial, considering varoglutamstat – an investigational oral glutaminyl cyclase (QPCT/L) inhibitor – in the treatment of early Alzheimer’s disease.

The trial (NCT03919162) will not progress into Phase IIb after it mirrored the company’s earlier VIVIAD trial (NCT04498650) and missed its primary and secondary endpoints. Instead, the company will change its development strategy after both showed promising effects on kidney function.

Similar to the company’s failed VIVIAD study, shared in March 2024, the VIVA-MIND trial was an independent double-blind placebo-controlled Phase II study, which investigated the dose of 600mg varoglutamstat BID in a similar patient population with early Alzheimer’s.

The reported top-line analysis of VIVA-MIND data in AD demonstrated that the trial had not met its primary endpoint of measuring clinically meaningful and statistically significant differences between varoglutamstat 600mg BID and placebo, assessed through Clinical Dementia Rating – Sum of Boxes (CDR-SB) scores.

It also failed to meet key secondary endpoints including Cognitive Function Composite 2 (CFC2) and Alzheimer’s Disease Assessment Schedule-Cognitive Subscale (ADAS-Cog) 13, in patients treated with varoglutamstat compared to placebo, in line with the previously reported results in Alzheimer’s from VIVIAD.

Despite missing all endpoints, varoglutamstat was also observed to demonstrate a favourable safety and tolerability profile in VIVA-MIND.

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As a result of the VIVIAD results, fewer patients were also recruited in VIVA-MIND, with 112 randomised and 109 treated in Phase IIa. The VIVA-MIND study was discontinued early and will not move into Phase IIb.

However, the data did reveal a significant finding in kidney function improvement. Analysis of kidney function in patients treated with varoglutamstat showed a statistically significant increase in eGFR of >4ml/min/1.73m² compared to placebo (p<0.001).

Vivoryon reported that the VIVA-MIND trial results will continue to shape the company’s development strategy to advance into a Phase II study in diabetic kidney disease.

Ahead of the company’s Q3 financial results today (10 December), Vivoryon CEO Frank Weber said: “All results support the development of varoglutamstat as a convenient oral therapeutic in diabetic kidney disease. Varoglutamstat also has potential across a broad range of other kidney diseases, including rare diseases affecting kidney function such as Fabry disease and Alport syndrome.”

Optimism for the potential of varoglutamstat in the treatment of kidney function has been reflected in the company’s stock prices, which saw a 43.3% increase on 9 December as the VIVA-MIND results piqued investors’ interest.

However, Vivoryon’s stock prices are still yet to recover from the blow delivered by VIVIAD’s results at the beginning of March when 90.5% of the company’s stock price was wiped.

Regarding the disappointing results for Alzheimer’s, Weber said: “These confirm the data seen in VIVIAD and are therefore not unexpected. While we would have wished for a different outcome of this study, we would like to thank all patients, their families and caregivers, the Alzheimer’s Disease Cooperative Study at the University of California San Diego School of Medicine and clinical study investigators, as well as the US National Institutes of Health – enabling the VIVA-MIND study through a grant – for their commitment to the study and the varoglutamstat programme.”

The total Alzheimer’s and mild cognitive impairment market was $2.2bn across the eight major markets (the US, France, Germany, Italy, Spain and the UK) in 2020, according to GlobalData. Although Vivoryon will now count itself out of the growing sector, the compound annual growth rate of the market was forecast at more than 20% between 2020 and 2030.

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