Alzheimer’s disease, a neurodegenerative disorder, has been traditionally targeted through treatments focusing on the amyloid protein plaques in the brain, with recent drug approvals such as Eisai’s Leqembi and Eli Lilly’s Kisunla leading the way. These intravenously infused therapies use antibodies to break up amyloid proteins but come with risks such as bleeding and brain swelling. Asceneuron, however, is pioneering a different approach by directly targeting another protein, tau, which is also implicated in Alzheimer’s pathology.
Founded in 2012 and originally spun out of Merck Serono, Asceneuron has shifted its focus towards orally administered therapies designed to interfere with tau protein aggregation, a process believed to contribute to the progression of Alzheimer’s. The company’s most promising candidate, ASN51, is a small molecule drug that inhibits the enzyme OGA, which is involved in protein aggregation. By blocking this enzyme, ASN51 aims to prevent tau proteins from accumulating, thereby potentially slowing the disease’s progression. Initial Phase 1 testing in healthy volunteers showed that ASN51 effectively reached the central nervous system and targeted a significant percentage of OGA enzymes.
The progression of ASN51 into Phase 2 clinical trials is bolstered by a $100 million Series C financing round led by Novo Holdings, underpinning a robust support from investors that include EQT Life Sciences – LSP Dementia Fund, OrbiMed, SR One, M Ventures, Sofinnova Partners, GSK Equities Investments Limited, and Johnson & Johnson Innovation—JJDC. This funding will support the upcoming Phase 2 study specifically focused on assessing ASN51’s effects on Alzheimer’s patients.
Asceneuron’s approach is part of a broader trend in Alzheimer’s research focusing on tau proteins. Competitive efforts include those by AC Immune, which is developing both immunotherapies and small molecules targeting tau under partnerships with Johnson & Johnson and Eli Lilly. Similarly, Takeda Pharmaceutical is exploring anti-tau small molecules in collaboration with Cure Network Dolby Acceleration Partners.
Aside from ASN51, Asceneuron has developed another clinical-stage drug, ASN90, targeted at treating progressive supranuclear palsy (PSP), a rare neurological disorder also associated with tau. ASN90 has already attracted commercial interest, with Barcelona-based Ferrer acquiring global licensing rights last year for an undisclosed sum.
With the fresh influx of capital, Asceneuron is not only advancing ASN51 into mid-stage testing but is also exploring other potential applications of its OGA inhibition technology in various neurodegenerative diseases including Parkinson’s and amyotrophic lateral sclerosis (ALS). The company’s innovative approach targeting protein aggregation pathways holds promise for developing therapies against a range of disorders characterized by protein misfolding and aggregation.
The optimism surrounding Asceneuron’s approach is encapsulated by CEO Barbara Angehrn Pavik’s statement on the potential of the company’s OGA inhibitor pipeline and its leadership in tackling tauopathies, diseases linked to tau proteins. The move to Phase 2 trials with ASN51 represents a significant milestone for Asceneuron as it seeks to expand treatment options for patients with Alzheimer’s disease and potentially other disorders.
As Alzheimer’s research continues to evolve, the focus on tau proteins, alongside amyloid, highlights the growing complexity in understanding and treating this devastating disease. Asceneuron’s advancement into later stages of clinical testing will be closely watched, as it represents a potential shift in the treatment paradigm and hope for patients seeking new avenues for management of their condition.
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