AC Immune’s Targeted Anti-pTau Active Immunotherapy for Alzheimer’s Disease Advances into Phase 2b Trial
AC Immune’s Targeted Anti-pTau Active Immunotherapy for Alzheimer’s Disease Advances into Phase 2b Trial
- Potentially registration-enabling Phase 2b study (ReTain) will evaluate the effect of ACI-35.030 on cognition and Tau pathology in approximately 500 participants with preclinical Alzheimer’s disease (AD)
- Anti-pTau active immunotherapy being designed to potentially prevent or reduce cognitive decline could address need of over 315 million people globally1 with preclinical AD
AC Immune to receive approximatelyCHF 40 million in total milestone payments under terms of the licensing agreement, following trial initiation and enrollment milestone
Lausanne,
Under the terms of the licensing agreement with
Dr.
The Phase 2b ReTain trial is a potentially registration-enabling trial and is a randomized, multicenter, double-blind, placebo-controlled clinical study in participants with preclinical AD to assess the clinical effect of active immunization with ACI-35.030. It is designed to test the hypothesis that ACI-35.030 has a disease-modifying effect that can delay or prevent the onset of cognitive impairment or other clinical symptoms in individuals with preclinical AD through inhibition of seeding and spreading of pathological Tau.
- Approximately 500 participants with preclinical AD (cognitively normal, amyloid positive, Tau positive) will be randomized in a 1:1 ratio to a single dose level of ACI-35.030 or placebo and administered as intramuscular injections for a maximum of 4 years.
- The primary endpoint will measure cognitive decline as assessed by the Preclinical AD Cognitive Composite 5 (PACC-5) score, which combines tests that evaluate episodic memory, timed executive function, and global cognition. It is sensitive enough to detect early changes in cognitive function, even before the first clinical signs of mild cognitive impairment (MCI) are apparent2.
- The key secondary efficacy endpoint will assess the effect of ACI-35.030 on the propagation and/or accumulation of Tau pathology compared with placebo, as measured by Tau PET imaging in the Tau Naïve Composite region of interest. PET imaging for pathological Tau will be performed at baseline and annually for 4 years. This endpoint may be sufficient for a Biologics License Application (BLA) filing seeking accelerated approval from the
U.S. Food & Drug Administration (FDA), with the primary endpoint serving as the basis for a traditional approval.
- Gustavsson et al. Alzheimer’s and Dement. 2023 19:658-670. https://doi.org/10.1002/alz.12694
- Donohue MC, Sperling RA, Salmon DP, Rentz DM, Raman R, Thomas RG, Weiner M, Aisen PS, Australian Imaging, Biomarkers, and Lifestyle Flagship Study of Ageing, Alzheimer’s Disease Neuroimaging Initiative, and Alzheimer’s Disease Cooperative Study (2014) The preclinical Alzheimer cognitive composite: measuring amyloid-related decline. JAMA Neurol 71(8):961–970. https://doi.org/10.1001/jamaneurol.2014.803
- Gustavsson et al. Alzheimer’s and Dement. 2023 19:658-670. https://doi.org/10.1002/alz.12694
About ACI-35.030 (JNJ-64042056)
ACI-35.030 (JNJ-64042056) is an investigational active immunotherapy designed using AC Immune’s SupraAntigen® platform. Its liposomal formulation incorporates a conformationally-constrained, membrane bound target peptide antigen, phosphorylated Tau (pTau), in addition to adjuvants and non-Tau T-helper peptides. Immunization with ACI-35.030 has been shown in a recent Phase 1b/2a clinical trial to rapidly elicit antibodies after the first injection against extracellular pathological pTau in 100% of elderly patients with Alzheimer’s disease. Importantly, the antibody response was sustained, boostable, and focused on pathological pTau, including enriched paired helical filaments (ePHF). Aggregation of pTau leads to the formation of neurotoxic ePHF and Tau tangles. Antibodies against non-phosphorylated Tau diminished over time. To date, no safety or tolerability issues have been observed following ACI-35.030 immunization.
About the SupraAntigen® platform
AC Immune’s clinically validated SupraAntigen® platform uses proprietary liposomes to rapidly generate novel product candidates for active immunotherapy as well as best-in-class monoclonal antibodies for passive immunization against key neurodegenerative disease targets. Antibodies generated by the platform are highly specific for the pathological conformations of misfolded proteins and have shown strong safety. The SupraAntigen® platform has successfully generated two active immunotherapies and two antibody candidates that have been validated in clinical studies and has led to multiple global partnerships with world-leading pharmaceutical companies. In addition to targeting Amyloid-beta and Tau,
About
AC Immune SA is a clinical-stage biopharmaceutical company that aims to become a global leader in precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and NeuroOrphan indications driven by misfolded proteins. The Company’s two clinically validated technology platforms, SupraAntigen® and Morphomer®, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features ten therapeutic and three diagnostic candidates, five of which are currently in Phase 2 clinical trials and one of which is in Phase 3. AC Immune has a strong track record of securing strategic partnerships with leading global pharmaceutical companies including Genentech, a member of the Roche Group, Eli Lilly and Company, and others, resulting in substantial non-dilutive funding to advance its proprietary programs and >$3 billion in potential milestone payments.
SupraAntigen® is a registered trademark of AC Immune SA in the following territories: AU, EU, CH, GB, JP, RU, SG and USA. Morphomer® is a registered trademark of AC Immune SA in CN, CH, GB, JP, KR, NO and RU.
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Source: AC Immune SA