Full Press Release Details
AC Immune's Targeted Anti-pTau Active Immunotherapy
for Alzheimer's Disease Advances into Phase 2b Trial
Lausanne, Switzerland, December 15, 2023
- AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases,
today announced that its development partner has programmed the launch of a Phase 2b clinical study to evaluate ACI-35.030 (JNJ-64042056)
in patients with preclinical Alzheimer's disease (AD), those individuals not yet showing symptoms. ACI-35.030 is an investigational
targeted active immunotherapy, selective for pathological phosphorylated Tau (pTau). Studies have shown that pTau correlates with AD progression
and the trial aims to show that ACI-35.030 can prevent or slow down the progression of tau pathology and onset of clinical symptoms.
Under the terms of the licensing agreement with
Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, AC Immune will now receive a milestone payment of CHF 15 million
and will receive another milestone payment of CHF 25 million related to achieving a non-disclosed enrollment target. The partnership with
Janssen aims to develop and commercialize therapeutic anti-Tau active immunotherapies for the treatment of AD and potentially other Tauopathies.
Dr. Andrea Pfeifer, CEO of AC Immune SA, commented:
"In the recently completed Phase 1b/2a trial, data showed that ACI-35.030 was able to activate patients' immune systems with
a robust polyclonal antibody response against phosphorylated Tau and its neurotoxic aggregated form, which is believed to contribute to
the pathology and progression of Alzheimer's disease. Our partner's decision to move forward with this robust clinical trial
shows that treatment with this active immunotherapy so early in the disease process that individuals are not yet showing symptoms, holds
tremendous promise to slow or possibly even prevent progression to symptomatic AD."
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.
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, AC Immune has generated
conformation-specific antibodies against emerging neurodegenerative disease targets including alpha-synuclein, TDP-43, and the NLRP3 inflammasome
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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