Asceneuron SA, an emerging leader in the development of novel, oral small molecule therapeutics targeting tauopathies for Alzheimer’s and related neurodegenerative diseases, today announced that the Alzheimer’s Drug Discovery Foundation (ADDF) awarded it a $325,000 grant to support the development of Asceneuron’s tau modulators.
“We are honored to receive this prestigious grant and proud that our tau modulators have been chosen through the rigorous peer-review process of the ADDF,” said Dirk Beher, CEO of Asceneuron. “The award will fund critical research as we prepare for initial clinical testing of ASN-561, our lead O-GlcNAcase inhibitor, in humans in early 2016.”
“We are interested in supporting new and innovative research to address the pressing need for effective therapeutics to treat Alzheimer’s disease,” said ADDF Founding Executive Director and Chief Science Officer, Howard Fillit, MD. “We are excited about accelerating the development of tau modulators because one of the major hallmarks of Alzheimer’s disease is the presence of tangles composed of the tau protein. Research suggests that reducing the formation of these tangles may be an effective way to treat the disease as a neuroprotective strategy.”
Neurofibrillary tangles form when microtubule-associated protein tau aggregates into paired helical filaments and larger structures, a process associated with tau hyperphosphorylation. The enzyme O-linked N-acetylglucosaminidase (O GlcNAcase) reverses a naturally occurring posttranslational modification of tau termed O-GlcNAcylation. Since O-GlcNAcylation protects against the aggregation of tau, inhibiting O-GlcNAcase may prevent neurofibrillary tangle formation and, most importantly, the resulting neurodegeneration.
ASN-561 is the first orally available small molecule O-GlcNAcase inhibitor in late preclinical testing. At the 2014 Alzheimer's Association International Conference, Asceneuron reported that in a preclinical model characterized by neurofibrillary tangles, oral doses of ASN-561 caused dose-dependent increases in O-GlcNAcylated tau in the forebrain, brain stem and spinal cord that reached levels up to 12 times higher than levels seen control subjects. Asceneuron aims to be among the first to confirm that this mechanism has therapeutic value for human patients.
Asceneuron plans to pursue the clinical development of ASN-561 in patients with progressive supranuclear palsy (PSP) and other tauopathies while also pursuing partnerships with larger companies for clinical development in Alzheimer’s disease (AD). PSP, an Orphan disease, is a neurodegenerative disorder associated with pathological tau accumulation (i.e. a tauopathy) in the brain. Asceneuron has also developed a proprietary blood biomarker of the O-GlcNAcase mechanism which has already been validated preclinically and will be used in the clinical trials.
About Asceneuron SA
Asceneuron SA (www.asceneuron.com) researches and develops oral small molecule therapeutics targeting tauopathies for treating Progressive Supranuclear Palsy (PSP), other related neurodegenerative diseases and Alzheimer’s disease. The lead product, a preclinical O-GlcNAcase inhibitor which targets tau, is scheduled to begin clinical testing in early 2016. Neuronal deposits of the microtubule-associated tau protein are a common feature of tauopathies, known to be a major contributor to neurodegenerative diseases. Asceneuron was formed in October 2012 through an MS Ventures-led spinout of the Alzheimer’s disease drug discovery portfolio and research group from Merck Serono, the pharmaceutical division of Merck.