Novo Holdings has led a $100 million Series C financing round for Asceneuron, a Swiss biotech company. This funding aims to advance Asceneuron’s pioneering therapeutics for neurodegenerative diseases, including Alzheimer’s disease and progressive supranuclear palsy (PSP).
Background Information
About Novo Holdings
Novo Holdings is a leading international life science investor, responsible for managing the assets and wealth of the Novo Nordisk Foundation. With a strong focus on creating long-term value, Novo Holdings invests in life science companies at all stages of development. Their portfolio includes significant investments in companies developing innovative treatments for a range of diseases, emphasizing their commitment to advancing healthcare solutions globally.
About Asceneuron
Founded in 2012, Asceneuron is a clinical-stage biotech company based in Lausanne, Switzerland. The company focuses on developing small molecule therapeutics that target neurodegenerative diseases characterized by tau protein aggregation, such as Alzheimer’s disease and PSP. Asceneuron’s lead drug candidates, ASN51 and ASN90, are designed to inhibit the OGA enzyme, which plays a crucial role in tau protein aggregation.
Details of the Financing Round
Investment Details
The $100 million Series C financing round was led by Novo Holdings, with participation from new investors EQT Life Sciences Dementia Fund, OrbiMed, and SR One. Existing investors, including M Ventures, Sofinnova Partners, GSK Equities Investments Limited, and Johnson & Johnson Innovation – JJDC, Inc., also participated in the round. The funds will primarily support the clinical development of ASN51, Asceneuron’s lead asset targeting Alzheimer’s disease.
Strategic Importance
This significant investment will enable Asceneuron to advance ASN51 into phase II clinical trials. ASN51 is an oral small molecule drug designed to inhibit OGA, thereby preventing the aggregation of tau proteins, which are implicated in Alzheimer’s disease. The unique mode of action and oral formulation of ASN51 offer a potentially transformative approach to treating Alzheimer’s, providing a convenient alternative to current therapies that are often administered via injection or infusion.
Asceneuron has already completed multiple phase I trials demonstrating the drug’s uptake in the central nervous system and its impact on the OGA enzyme. The phase II study, set to begin later this year, aims to further validate ASN51’s efficacy and safety in treating Alzheimer’s disease.
Research and Development
Pipeline Overview
Asceneuron’s research and development efforts are focused on addressing neurodegenerative diseases through their innovative O-GlcNAcase (OGA) inhibitors, ASN51 and ASN90. These small molecule inhibitors are designed to target proteinopathies involving tau and alpha-synuclein, which are implicated in diseases such as Alzheimer’s, Parkinson’s, and progressive supranuclear palsy (PSP).
ASN51
ASN51 is Asceneuron’s next-generation OGA inhibitor, currently in clinical development for Alzheimer’s disease. Preclinical studies and phase I trials have demonstrated that ASN51 is well-tolerated, shows significant brain penetration, and effectively targets OGA, preventing the aggregation of tau proteins. The drug has shown promise in both single and multiple dosing regimens, maintaining high brain OGA occupancy and elevating tau glycosylation in peripheral blood cells. Asceneuron plans to begin phase II clinical trials for ASN51 in the latter half of 2024, aiming to provide a disease-modifying oral therapy for Alzheimer’s patients.
ASN90
ASN90, another OGA inhibitor, has shown potential as a multimodal therapy for both tau and alpha-synuclein-related diseases. Licensed to Ferrer Pharmaceuticals for the treatment of PSP, ASN90 has completed multiple phase I trials, demonstrating safety and efficacy in increasing O-GlcNAcylation of tau and alpha-synuclein proteins. This inhibition reduces the formation of toxic aggregates, thereby slowing disease progression in models of PSP and Parkinson’s disease. ASN90 is now moving into phase II/III trials to further evaluate its therapeutic potential.
Impact on the Industry
Market Potential
The market potential for new treatments targeting neurodegenerative diseases is substantial, given the high unmet medical needs and the growing prevalence of conditions such as Alzheimer’s and Parkinson’s. Current treatments often focus on symptom management rather than disease modification. Asceneuron’s approach with OGA inhibitors represents a significant advancement by aiming to modify disease progression directly through the inhibition of protein aggregation.
Broader Implications
Asceneuron’s innovative research could have wide-reaching implications for the biotech industry. The successful development and commercialization of OGA inhibitors may pave the way for new therapeutic strategies across various neurodegenerative diseases. Additionally, the collaboration between Asceneuron and prominent investors like Novo Holdings, EQT Life Sciences, and others underscores the growing interest and investment in neurodegenerative research.
Conclusion
Novo Holdings’ $100 million investment in Asceneuron marks a pivotal moment for the advancement of treatments for neurodegenerative diseases. With promising candidates like ASN51 and ASN90 progressing through clinical trials, Asceneuron is well-positioned to make significant strides in providing effective therapies for conditions that currently have limited treatment options. This investment not only supports Asceneuron’s mission but also highlights the critical need for innovative solutions in the fight against debilitating neurodegenerative diseases.
