Trial of Oral Varoglutamstat Cleared to Take Place in China

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by Teresa Carvalho MSc |

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A clinical trial application for varoglutamstat, or PQ912, an investigational therapy for Alzheimer’s disease, was approved in China, its developers, Vivoryon Therapeutics and Simcere Pharmaceutical Group, announced.

“We are proud to have obtained [China’s Center for Drug Evaluation] CDE approval of our Clinical Trial Application of varoglutamstat which represents our strong commitment in the battle against Alzheimer’s disease,” Renhong Tang, executive vice president of Simcere, said in a press release.

“We will push forward the Phase 1 clinical study of varoglutamstat in China with all our efforts, and prepare to join the active global Phase 2 efforts,” Tang added.

Varoglutamstat is being evaluated in two Phase 2 clinical trials in Europe and the U.S., the VIVIAD (NCT04498650) and VIVA-MIND (NCT03919162) studies.

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VIVIAD, underway at several sites in Denmark, Germany, and the Netherlands, seeks to recruit 250 patients with mild cognitive impairment and mild dementia due to Alzheimer’s. More information on enrollment can be found here.

VIVA-MIND, which is ongoing at six locations in the U.S., plans to enroll more than 400 patients with early stages of Alzheimer’s disease. Additional information may be found here.

Alzheimer’s disease is characterized by clumps of two proteins, called amyloid plaques and tau tangles, that accumulate to toxic degrees within nerve cells in the brain, causing them to die.

Varoglutamstat is a small molecule that blocks the activity of the enzyme glutaminyl cyclase. This enzyme, known to help in forming N3pE amyloid (a toxic form of amyloid-beta), is present in abnormally high levels in the brains of those with Alzheimer’s.

N3pE amyloid is a kind of seeding factor as it promotes amyloid-beta accumulation. High levels of this protein are known to be related to cognitive decline.

Unlike other treatments aimed at decreasing amyloid-beta plaque levels, varoglutamstat — by blocking N3pE amyloid — may act earlier and inhibit its formation.

Moreover, given the importance of glutaminyl cyclase in the stability and activity of a pro-inflammatory protein called CLL2, which also helps tau protein to aggregate, varoglutamstat also has the potential to reduce neuro-inflammation.

Varoglutamstat was found to be well tolerated in a first-in-human Phase 1 trial, with 205 healthy volunteers. The therapy has also been considered safe in a three-month Phase 2a trial called SAPHIR (NCT02389413), which enrolled 120 patients with early Alzheimer’s treated with varoglutamstat twice daily for three months. The study also supported the therapy’s effectiveness in improving cognitive function, memory, and attention.

“We are delighted with Simcere’s rapid progress towards bringing new treatment options to the millions of patients suffering from Alzheimer’s disease in China,” said Michael Schaeffer, chief business officer of Vivoryon.

“Varoglutamstat has already demonstrated encouraging clinical results in earlier studies, and we look forward to building on our experience from the completed and ongoing studies in the EU and U.S. to support our partner during this significant phase in development,” Schaeffer added.

Simcere and Vivoryon announced a partnership last year to develop and market therapies targeting N3pE to treat Alzheimer’s in China. Besides varoglutamstat, the deal also includes a monoclonal N3pE-antibody (PBD-C06).

Also in 2021, the U.S. Food and Drug Administration (FDA) granted Vivoryon a fast track designation for varoglutamstat in early Alzheimer’s. The designation enables Vivoryon to have more frequent interactions with the FDA during the development process. The main goal of the fast-track program is to accelerate the review of therapies that potentially improve care for serious diseases.