• Financing led by Novo Holdings, including new investment from EQT Life Sciences – LSP Dementia Fund, OrbiMed and SR One with participation from existing investors M Ventures, Sofinnova Partners, GSK Equities Investments Limited and Johnson & Johnson Innovation – JJDC, Inc.
• Funds will be used to progress lead asset ASN51, an oral small molecule OGA inhibitor, into Phase 2 clinical development for the treatment of Alzheimer’s disease

Lausanne, SWITZERLAND and San Francisco, CA, USA, 16 July 2024

Asceneuron SA, a clinical stage biotech company developing small molecules targeting tau protein aggregation, a root cause of neurodegenerative disease, today announces an $100 million oversubscribed Series C financing to advance the clinical development of its groundbreaking clinical pipeline of OGA inhibitors for the treatment of neurodegenerative diseases. The financing was led by Novo Holdings with new investment from EQT Life Sciences – LSP Dementia Fund, OrbiMed and SR One, alongside participation from existing investors M Ventures, Sofinnova Partners, GSK Equities Investments Limited and Johnson & Johnson Innovation – JJDC, Inc.

The financing will be used to advance Asceneuron’s lead asset ASN51 into Phase 2 clinical development for the treatment of Alzheimer’s disease. ASN51 is an oral small molecule drug designed to inhibit OGA, an enzyme implicated in protein aggregation. By preventing the aggregation of tau proteins, ASN51 aims to slow the progression of Alzheimer’s disease. OGA inhibition has also shown promising potential to prevent the aggregation of proteins that are central to other neurodegenerative diseases, including Parkinson’s disease and amyotrophic lateral sclerosis.

ASN51’s unique mode of action and convenient oral formulation make it an ideal therapy for patients with Alzheimer’s disease. Asceneuron has completed five Phase 1 clinical trials, demonstrating complete central nervous system uptake and high OGA enzyme occupancy. Asceneuron plans to initiate its first Phase 2 clinical study later this year.

Barbara Angehrn Pavik, Chief Executive Officer of Asceneuron, said:
“This high caliber life sciences investor syndicate further validates the potential of our OGA inhibitor pipeline and leadership in the field of tauopathies. We are excited to advance our lead asset ASN51 into Phase 2 clinical development, recognizing its potential to significantly expand treatment options for patients with Alzheimer’s disease.”

Naveed Siddiqi, MD, Senior Partner, Venture Investments, Novo Holdings, commented:
“Alzheimer’s disease is undergoing a transformational moment. Millions are afflicted by this devastating disease and there are very few therapeutic options. Validated biomarkers are allowing for more focused and rapid development. We are now witnessing the approvals of the first disease modifying antibody based injectable therapies. 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.”

Hakan Goker, PhD, Managing Director of M Ventures, the founding investor of Asceneuron, remarked on behalf of the existing investors:
“Asceneuron has successfully developed highly differentiated oral OGA inhibitors from pre-clinical development to where they are today, entering Phase 2 development in patients with Alzheimer’s disease. ASN51 holds great promise as a next generation treatment for Alzheimer’s disease in addition to addressing other neurodegenerative diseases, including Parkinson’s disease and amyotrophic lateral sclerosis. Together with the current blue-chip investors like Sofinnova Partners, who led the series A round, we welcome the new strong investor group to continue supporting the company in this significant phase of development.”

In connection with the financing, Naveed Siddiqi of Novo Holdings, Philip Scheltens of EQT Life Sciences – LSP Dementia Fund and Dina Chaya of OrbiMed will join the Asceneuron board of directors, chaired by Abbas Hussain. Amit Shah, board director, will now represent new investor SR One (previously representing GSK). These Board directors join existing investor directors, Henrijette Richter of Sofinnova Partners and Hakan Goker of M Ventures.

• Consortium brings together industry, academic, and regulatory experts to advance research and bridge gaps in drug development targeting Alzheimer’s disease
• Asceneuron to contribute its expertise in neurodegenerative disease to advance biomarkers for drug development in Alzheimer’s disease

Lausanne, SWITZERLAND and San Francisco, CA, USA, 26 March 2024

Asceneuron SA, a clinical stage biotech company dedicated to targeting the root causes of neurodegenerative diseases, today announces that it has joined the Critical Path Institute’s, Critical Path for Alzheimer’s Disease (CPAD) Consortium. With its highly experienced leadership team and world class Scientific Advisory Board of leading experts in neurodegenerative diseases, Asceneuron is well positioned to contribute to the consortium.

Asceneuron has an exemplary pipeline of O-GlcNAcase (OGA) inhibitors in clinical development that have the potential to halt disease progression in neurodegenerative diseases. Its lead asset, ASN51, is a potential best-in-class, orally administered, OGA inhibitor targeting tau aggregation and will be progressing into Phase II clinical development later this year. The CPAD consortium is proceeding to advance important biomarkers for Alzheimer’s disease (AD), such as tau Positron Emission Tomography (PET) and is accelerating the pathway for approval of promising new therapies. As a member of CPAD, Asceneuron will work collaboratively with the consortium to help address the large unmet medical need of patients and families suffering due to neurodegenerative diseases.

Ryan Schubert, Senior Vice President Research and Development at Asceneuron, said:
“We are honored to be collaborating with the CPAD consortium alongside esteemed fellow members, all of whom are dedicated to accelerating drug development to improve patients’ lives. CPAD has gathered high quality data and experts from industry, academia and regulatory agencies with a focus on bringing new treatment options to patients suffering with AD. We look forward to contributing to and impacting the treatment pathway for AD as we advance our lead intracellular tau-targeting asset, ASN51, into Phase II clinical development later this year. Further information will be shared at upcoming scientific conferences.”

Yashmin Karten, PhD, MBA, Associate Director at Critical Path for Alzheimer’s Disease, added: “C-Path is proud to welcome Asceneuron as the newest member of its CPAD Consortium. Asceneuron’s research on molecules that prevent the formation of toxic tau aggregates in the brain to slow down or stop the progrecision-making tools to advance drug development and improve the lives of those living with AD and related dementia (ADRD).

CPAD’s mission is to accelerate the drug development process for patients with chronic neurodegenerative disease leading to dementia. Its primary focus is on AD. CPAD works with industry, regulatory authorities, academia, and patient advocacy organizations to aggregate anonymized patient-level data consensus standards with the aim of advancing drug development tools for evaluating drug efficacy and safety, to optimize novel clinical trial designs, and facilitating the regulatory review process.”
CPAD’s integrated and standardized patient-level database comprises over 100,000 patient level records across 73 separate clinical trial datasets. These records are utilized by over 370 institutions worldwide. Alongside its biotech and pharmaceutical company members, the initiative incorporates feedback from all its stakeholders, including government and regulatory agencies. CPAD has ongoing efforts as part of its pre-competitive working groups to evaluate the potential of tau PET as a reliable surrogate endpoint, with the aim of enhancing efficiency in Alzheimer’s disease drug development.