Clinically validated platform accelerates the discovery and development of first-in-class small molecule therapeutics in parallel with companion diagnostics
LAUSANNE, Switzerland, March 31, 2021 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, will host an investor webinar today to discuss the unique benefits of the Company’s innovative MorphomerTM technology platform, which generates first-in-class therapeutic and diagnostic candidates to power AC Immune’s precision medicine approach for neurodegenerative diseases. The live webinar begins at 10am ET and can be accessed here.
Prof. Andrea Pfeifer, Ph.D., CEO of AC Immune SA, commented: “Our industry-leading precision medicine approach aims to address the complexity and heterogeneity of neurodegenerative diseases by pairing highly selective and conformation-specific therapeutics with first- or best-in-class companion diagnostics. This approach is fueled, in part, by our clinically validated MorphomerTM platform, which combines decades of medicinal chemistry and drug development expertise to accelerate the design, synthesis, and development of brain- and cell-penetrant small molecules capable of binding to intracellular pathological proteins.
“Through the successful application of this platform, we are advancing a comprehensive pipeline of therapeutic and diagnostic Morphomers™ that address key targets such as Tau, TDP-43, alpha-synuclein, and the NLRP3 inflammasome. We are pleased to provide an overview of the discovery and development of these programs, several of which have been validated in the clinic, as well as outline the significant milestones we expect to achieve across our portfolio or Morphomer™- and SupraAntigen™-derived portfolio over the coming months.”
The webinar features presentations and a Q&A session with members of AC Immune’s Management and Research and Development Teams: Prof. Andrea Pfeifer; Dr. Marie Kosco-Vilbois, Chief Scientific Officer; Dr. Sonia Poli, Life Cycle Leader; and Dr. Francesca Capotosti, Group Leader, in vivo Pharmacology and Non-Clinical Safety.
Key highlights from the event:
AC Immune’s Morphomer™ technology enables the Company to deliver on its vision for precision medicine by fulfilling the need for differentiated therapies and a more comprehensive panel of diagnostic agents to characterize neurodegenerative diseases at the individual patient level.
Brain- and cell-penetrant Morphomer™ therapeutics that disrupt and/or inhibit intracellular protein aggregation may preserve neurons and prevent spreading of pathology and disease progression
Earlier, more reliable detection of specific neuropathologies may unlock the value of disease-modifying therapeutics and create a pathway for personalized combination therapies
AC Immune’s Morphomer™ platform has generated significant value through partnership and clinical validation and will continue to drive future value as early-stage programs mature.
Tau-targeted therapeutic and diagnostic Morphomers™ have generated more than CHF 174 million to date from strategic partnerships with the potential for considerable future milestone payments and royalties
The Company’s current portfolio of therapeutic and diagnostic Morphomer™ candidates targeting TDP-43, alpha-synuclein and the NLRP3 inflammasome can address multiple significant market opportunities in neurodegenerative and non-CNS indications
The Morphomer™ platform can rapidly deliver first-/best-in-class small molecule candidates against emerging targets
The unique benefits and competitive advantages of AC Immune’s MorphomerTM platform include:
A highly focused, proprietary library of ~12,000 conformation-specific small molecules reflecting years of know-how and enrichment for compounds that bind mis-folded, aggregated protein structures
Efficient generation of CNS-optimized compounds with favorable brain penetration and pharmacokinetics
Rational design and deep expertise in medicinal chemistry to ensure optimized candidates bind selectively to pathological forms of misfolded proteins inside neurons at the earliest stage of disease
A proprietary suite of cellular and functional assays that use patient-derived brain samples to enable rapid identification and validation of successful compounds
Broad applicability as potentially disease-modifying therapies as well as precision diagnostics, directed against both established and novel neurodegenerative disease targets
Validation both clinically and through collaboration agreements with industry leaders Eli Lilly and Company and Life Molecular Imaging
Morphomer Tau candidate ACI-3024 achieves potentially therapeutic target levels in the cerebrospinal fluid (CSF) at the highest administered dose in a Phase 1 study – the first therapeutic Morphomer candidate to be tested in humans.
Single and multiple dosing with ACI-3024 resulted in dose-dependent exposure, and after multiple doses, ACI-3024 concentrations in CSF exceeded target concentrations based on animal studies
ACI-3024 is being evaluated for efficacy in models of rare Tauopathies, while AC Immune and strategic partner Eli Lilly and Company have decided to pursue other promising Tau Morphomer candidates with the desired CSF exposure and selectivity for pathological aggregated Tau for potential clinical development in Alzheimer’s disease
Platform validation has positive readthrough for earlier-stage candidates targeting a-syn and NLRP3
Morphomer™-derived positron emission tomography (PET) imaging agents are first-/best-in-class, representing potential game changers for alpha-synuclein-, TDP-43- and certain Tau-driven diseases.
Published results for AC Immune’s differentiated Tau PET tracer, PI-2620, demonstrate its ability to reliably detect 3- and 4-repeat Tau protein, identifying early Tau pathology in Alzheimer’s disease patients and reproducibly differentiating patients with Alzheimer’s and progressive supranuclear palsy from those with other disease pathologies such as alpha-synucleinopathies
Clinical-stage alpha-synuclein PET tracer ACI-12589 provides specific signal in tissue from Parkinson’s disease patients and differentiates between disease and non-disease controls
First-in-class TDP-43 PET tracer candidates show nanomolar affinities on patient-derived brain tissue and rapid uptake in vivo
Dr. Kosco-Vilbois concluded: “As the field expands its understanding of co-pathologies and the heterogeneity inherent to neurodegenerative diseases, it becomes increasingly clear that precision medicine will be the future of this treatment landscape. Our proprietary Morphomer™ and SupraAntigen™ technology platforms and the deep expertise at AC Immune give us the ability to remain at the forefront of this paradigm shift.”
About AC Immune SA
AC Immune SA is 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, SupraAntigenTM and MorphomerTM, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features nine therapeutic and three diagnostic candidates, six of which are currently in clinical trials. 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 Janssen Pharmaceuticals, Inc., resulting in substantial non-dilutive funding to advance its proprietary programs and >$3 billion in potential milestone payments.
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Forward looking statements
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