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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, March 26, 2024 /PRNewswire/ -- 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 progression of AD provides a welcome addition to the intellectual brain power and wealth of scientific knowledge within the consortium. CPAD's diverse stakeholders and members are dedicated to enhancing regulatory decision-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. About Asceneuron Asceneuron is a clinical stage biotech company focused on the development of orally bioavailable therapeutics for debilitating neurodegenerative disorders with high unmet medical need. The company's pipeline reflects its ambition and commitment to developing treatments for a wide a range of neurodegenerative diseases including Alzheimer's and Parkinson's disease, as well as orphan tauopathies. Asceneuron has two clinical-stage small molecule O-GlcNAcase inhibitors in development for the treatment of proteinopathies: OGA inhibitor ASN90 (licensed to Ferrer Pharmaceuticals) for the treatment of progressive supranuclear palsy (PSP) and a potential best-in-class OGA inhibitor, ASN51 for Alzheimer's disease. Asceneuron is backed by a renowned syndicate of investors consisting of Sofinnova Partners, M Ventures, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and Kurma Partners. For more information, please visit . About Critical Path Institute Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA's Critical Path Initiative. C-Path's mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path's global headquarters is located in Tucson, Arizona and C-Path's Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit . About Critical Path for Alzheimer's Disease (CPAD) Consortium Founded in 2008, the Critical Path for Alzheimer's Disease (CPAD) enhances regulatory decision-making tools to advance drug development and improve the lives of those living with Alzheimer's disease and related dementia (ADRD). The primary objective of Critical Path for Alzheimer's Disease (CPAD), a pre-competitive consortium of the Critical Path Institute, is to promote, support, and manage data and knowledge sharing from Alzheimer disease (AD) drug development stakeholders. CPAD generates the appropriate tools for the specific challenges identified by our stakeholders, to accelerate drug development for AD and advancing better treatments for people worldwide. For more information, please visit . SOURCE Asceneuron SA

Biogen has two clinical-stage Alzheimer's disease assets behind Leqembi, both of which target tau proteins or their dysfunction. “What does it mean to be a leader in Alzheimer’s disease?” That’s the question Biogen is asking itself, and the question many of the company’s peers are working fervently to answer as the heated race to differentiate from the first batch of Alzheimer’s treatments turns to a simmer.  Adam Keeney, Ph.D., corporate development chief for the Leqembi co-maker, said in an interview with Fierce Biotech that Biogen is "not only just satisfied with [amyloid] beta" and instead "looking at the different pillars of disease." Keeney says the first stop on the portfolio-building circuit is tau. The company’s phase 2 antisense drug, BIIB-080, targets the protein buildup that accompanies amyloid beta in patients with Alzheimer’s. It’s an intrathecal injection—an injection into the spinal cord to bypass the blood-brain barrier— which Keeney described as “not ideal” though probably satisfactory for a first-generation option. But Biogen is planning for and think-tanking easier methods, like IV delivery and even oral compounds.  “We're thinking about degraders and molecular glues and different technologies that we can use,” Keeney said. Biogen already has one oral option in the clinic, the O-GlcNACase inhibitor BIIB-131, a small molecule that looks to block the enzyme research has tied to tau’s dysfunction in Alzheimer’s patients.  Keeney described oral delivery as a goal that’s on a “longer time horizon” routing the work back to the original question: What does it mean to be a leader in the space?  Biogen has experienced the ups and downs of Alzheimer's drug discovery more acutely than probably any company. Its well-documented Aduhelm failure came to a much-expected conclusion earlier this year, when Biogen wound down all work on the approved med. Not only was it made obsolete by Leqembi, but the therapy was a corporate representation of time wasted. Meanwhile, Eli Lilly pressed on from behind, raising up donanemab, which is expected to get an FDA ruling any day now.  The amyloid beta battle may not ultimately be an automatic reflection of tomorrow’s tau contest, or other options that may be discovered. Deeper into Biogen’s research work, the company is tinkering with novel anti-inflammatory targets, Keeney says. Targeting rogue tau buildup has proven difficult, just like amyloid. Lilly cut a phase 2 large-molecule zagotenemab in October 2021 after it failed to hit phase 2 endpoints. This followed phase 1 data showing four cases of brain bleeding (ARIA-H) across three patients, representing 19% of treated patients. Kenney says it remains to be seen whether that’s a genuine signal tied to tau-busting treatments—but it's certainly been an issue with Leqembi and donanemab.  “I think it's probably too early to say there's a signal or not in that,” he said. “We have no signal that is preventing us to move forward.”  Beyond drug development, critical advancements are happening in the Alzheimer’s diagnostic sector, with companies working to improve the tests that could detect amyloid buildup. There’s been such improvement in this area that a working group convened by the Alzheimer’s Association and the National Institute on Aging recently revised its criteria for diagnosing Alzheimer’s to include blood-based biomarkers. Keeney says those tests may not immediately be a primary diagnosis, but a negative result could preclude someone from an additional PET scan.  “So we're also investing and working with collaborators because there are blood-brain barrier biomarkers that are out there,” Keeney said, noting they aren’t yet 100% predictive. “We're helping also get the predictive validity improved.”  Despite the blood-based tests needing refinement, authors of the new testing criteria made clear that the future is now, and some tests provide “excellent diagnostic performance.” “The field is now in a transition phase during which plasma biomarkers are being integrated with traditional CSF and PET biomarkers,” the guidance states.