Novo leads $100m financing for Alzheimer's player Asceneuron

Novo Nordisk's parent company, Novo Holdings, has led a $100 million third-round financing for Swiss biotech Asceneuron, which is developing a non-amyloid therapy for Alzheimer's disease.

The new funds will be used for mid-stage clinical trials of ASN51, an oral small-molecule inhibitor of O-GlcNAcase (OGA), which has emerged in recent years as a possible target for the treatment of tauopathies, including Alzheimer's.

Studies have shown that OGA inhibitors promote the glycosylation of tau protein, which clumps together to form tangles in Alzheimer's in a manner similar to the aggregation of amyloid into plaques.

OGA inhibitors promote glycosylation of tau, which seems to prevent aggregation and stabilise the protein in a soluble, non-pathogenic form. Tau tangles are also a feature of other neurodegenerative diseases, including Parkinson's and amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND).

Asceneuron has already completed five phase 1 trials of ASN51 which showed that the drug was taken up into the central nervous system uptake and bound to OGA. A phase 2 clinical study is due to start later this year, as Asceneuron tries to keep pace with rivals in the category, which include Eli Lilly, Biogen, and MSD.

Lilly's LY3372689 is already in a phase 2 trial involving patients with early symptomatic Alzheimer's in North America, Australia, Japan, and Poland, with results expected later this year. Meanwhile, Biogen reported phase 1 data with its BIIB113 candidate in healthy volunteers at this year's International Conference on Alzheimer's and Parkinson's Diseases (AD/PD), while MSD/Alectos have an inhibitor called MK-8719 in preclinical testing.

There are a host of other tau-targeting candidates coming through the pipeline – with mixed results in clinical trials – as developers try to follow Eisai/Biogen and Lilly in bringing anti-amyloid therapies to market for Alzheimer's. Given that the first generation of amyloid drugs has shown modest disease-modifying efficacy in trials, there is keen interest to see if combining drugs with different mechanisms of action could improve outcomes.

"Alzheimer's disease is undergoing a transformational moment," commented Naveed Siddiqi, senior partner for venture investments, at Novo Holdings.

"We are now witnessing the approvals of the first disease-modifying antibody-based injectable therapies," he added. "Asceneuron's innovative oral small molecule drug targeting intracellular tau offers the potential for a paradigm shift in the way this neurodegenerative disease is treated."

EQT Life Sciences, OrbiMed, SR One M Ventures, Sofinnova Partners, and the venture capital arms of GSK and Johnson & Johnson also participated in the round.

It is the second nine-figure financing round led by Novo Holdings in the last few days, coming after it participated in a $115 million first round for UK cancer drug developer Myricx Bio.

Photo by Mii Luthman on Unsplash