According to WHO, “only a quarter of countries worldwide have a national policy, strategy or plan for supporting people with dementia and their families […]. . Half of these countries are in WHO’s European Region, with the remainder split between the other Regions. Yet even in Europe, many plans are expiring or have already expired, indicating a need for renewed commitment from governments.

At the same time, the number of people living with dementia is growing according to the report: WHO estimates that more than 55 million people (8.1 % of women and 5.4% of men over 65 years) are living with dementia. This number is estimated to rise to 78 million by 2030 and to 139 million by 2050.”

In the article “Precision And Prevention Key To Future Of Alzheimer’s Treatment – A Look at Novel Approaches In The Alzheimer’s Pipeline” published by In Vivo on August 4th, 2021, William Masters gathered the recommentations of leading CEOs from early- to mid-stage biotechs to advance AD treatment.

Award will fund first in human clinical trials of next generation O-GlcNAcase inhibitor, ASN51, to start Q2 2021

Lausanne, SWITZERLAND and San Francisco, CA, USA, 9 March, 2021 – Asceneuron SA, an emerging leader in the development of treatments for neurodegenerative diseases, announced today the receipt of a USD 2.2 million award from the Alzheimer’s Drug Discovery Foundation (ADDF) for a first in human Phase I study of the Company’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 in Q2 2021, with first interim data due in Q3 2021.

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.

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 posttranslational 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.

Dirk Beher, Chief Executive Officer and a Co-Founder of Asceneuron, commented: 

“This Phase I clinical study targeting the root cause of neurodegeneration comes at a time when our current knowledge of the biology of O GlcNAcase has reached a critical inflection point. As a leader in this field, and with the welcome support of ADDF, we plan to translate our novel and exciting biology into significant health benefits for patients with neurodegenerative diseases.”

Howard Fillit, M.D., Founding Executive Director and Chief Science Officer of the ADDF, commented: 

“Alzheimer’s disease is a complex neurodegenerative disease, and many studies highlight the role of reducing neurodegeneration as a promising path to effective treatment. The ADDF is pleased to help advance Asceneuron’s research program to test this drug candidate in Alzheimer’s patients in a Phase 1 clinical trial.”

Peter Van Vlasselaer, Chairman of Asceneuron added: 

“We’re delighted to have received this substantial recognition from the ADDF for our work in neurodegeneration, and the potential of our pipeline. Together with support of the ADDF, we look to treatments for not just AD, but all and related dementias, providing new hope for the medical community, the patients, and their families / one of the greatest healthcare challenges of our time.”

Asceneuron’s CEO and founder Dirk Beher shared his views on the remaining challenges of tackling diseases like PSP and Alzheimer’s disease in an article by George Underwood in Pharmaphorum.

Atypical parkinsonian syndromes are a group of hypokinetic neurodegenerative diseases other than Parkinson’s disease: Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Multiple System Atrophy (MSA) and Dementia with Lewy Bodies (DLB) are the most prominent representatives of this group. This course is organised by the International Parkinson and Movement Disorder Society (MDS), a professional society of clinicians, scientists, and other healthcare professionals who are interested in Parkinson’s disease, related neurodegenerative and neurodevelopmental disorders, hyperkinetic movement disorders, and abnormalities in muscle tone and motor control.

The workshop is designed to provide young neurologist and future experts in Movement Disorders the essential knowledge and skills required for qualified medical care and high end research in atypical parkinsonian syndromes. 

Without adequate treatments, neurodegenerative diseases such as Alzheimer’s and Parkinson’s remain an important unmet medical burden for aging societies. In a discussion at the 2019 Biotech Showcase, Asceneuron CEO Dirk Beher tells Mike Ward about the progress that the 2012 startup is making with its small-molecule therapeutics that reduce the accumulation of toxic aggregates of the tau protein into the neurofibrillary tangles that are widely recognized as the main cause of neurodegeneration and clinical symptoms in the majority of dementia cases, including Alzheimer’s disease.