Alzheimer’s Drug Discovery Foundation Awards $2m for Promising Swiss Merck Serono Spin-off

Alzheimer’s Drug Discovery Foundation Awards $2m for Promising Swiss Merck Serono Spin-off

The Alzheimer’s Drug Discovery Foundation (ADDF) awarded $2.2 million to a biotech venture called Asceneuron SA, an emerging leader in the development of treatments for neurodegenerative diseases to help finance a first-in-human Phase 1 study of the venture’s next generation O-GlcNAcase inhibitor, ASN51. The trial is due to recruit healthy volunteers and Alzheimer’s disease patients at sites in Europe and Australia, to start Q2 2021, with first interim data projected for Q3, 2021. Of course, that date assumes the trial moves according to a pre established clinical plan.

TrialSite introduces the TrialSite Network to this novel therapy and company.

What is O-GlcNAcase?

O-GlcNAcase is an emerging drug target in central nervous system (CNS) drug development since deficient glycosylation patterns of intracellular proteins have been associated with diseases of aging and neuronal dysfunction. O–GlcNAcase inhibitors prevent the elimination of intracellular protein glycosylation, thereby halting the decline of the steady-state levels of this post translational modification. O–GlcNAcase inhibitors have initially been pursued exclusively for tau-related diseases. Emerging preclinical data suggest a wider application to intracellular proteinopathies such as Alzheimer’s disease and related disorders, and diseases of disturbed neuronal network function in general, with the potential to provide both disease-modifying and symptomatic benefits at the same time as multimodal drugs.

What does the funding support?

The award will support a Phase 1 single ascending dose arm and a positron emission tomography (PET) target engagement study in healthy volunteers. The Phase 1 clinical trials will assess safety and tolerability of ASN51, and closely examine multiple biomarkers relevant to the target mechanism and neurodegeneration in healthy subjects and Alzheimer’s patients.

Australian Study

The company’s forthcoming Australian study (NCT04759365) evaluates ASN51-101 in a randomized double-blind, placebo-controlled, phase 1 first in human (FIH) safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) study of oral ASN51 in healthy young adult and elderly subjects and elderly subjects with AD. The study is comprised of three parts (Part 1, Part 2 and Part 3).

The Company

Based out of Switzerland, this privately held venture is financed by a renowned syndicate of investors including Sofinnova Partners, M Ventures, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and Kurma Partner.

Founded in 2012 via spin-off of Merck Serono’s Alzheimer’s drug discovery portfolio and research group in Switzerland, the company pursues a semi-virtual operating model. They use in-house resources to pursue their unique biochemical assays, biomarker discovery and pharmacology models while they collaborate with contract research organizations for other activities such as medicinal chemistry. The company operates in a modern, state-of-the-art facility at the EPFL Innovation Park in Lausanne, in close proximity to academic centers in the Lake Geneva area.

Their lead product is an O-GlcNAcase inhibitor which has been demonstrated to modulate tau pathology in preclinical studies and is now in human studies. The first indication targeted is the orphan tauopathy Progressive Supranuclear Palsy (PSP). The pipeline reflects our ambition to develop treatments for a wide range of neurodegenerative diseases including orphan tauopathies, Alzheimer’s and Parkinson’s diseases. The lead program ASN120290, an O-GlcNAcase inhibitor, is being developed for the orphan tauopathy progressive supranuclear palsy (PSP). Asceneuron is also developing a next generation O-GlcNAcase inhibitor ASN51 to target Alzheimer’s disease and related disorders.

R&D Strategy

The company strives to address high unmet needs in neurodegenerative diseases such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases. With a pipeline consisting of small-molecule therapeutics aiming for disease modulation of tauopathies and cognitive impairment, they develop molecules that are orally bioavailable, meaning that they are suitable for dosage as pills. For each program, the company implements a clinical biomarker, which helps demonstrate that the drug reaches the intended target in humans and will support dose selection for the seminal proof-of-concept trials in patients.

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