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.
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 millionto 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 Immuneand 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
For further information, please contact:
|Head of Investor Relations
Phone: +1 917 809 0814
LaVoie Health Science
Phone: +1 617 792 3937
Phone: +41 79 367 6254
Forward looking statements
This press release contains statements that constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements are statements other than historical fact and may include statements that address future operating, financial or business performance or AC Immune’s strategies or expectations. In some cases, you can identify these statements by forward-looking words such as “may,” “might,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “potential,” “outlook” or “continue,” and other comparable terminology. Forward-looking statements are based on management’s current expectations and beliefs and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. These risks and uncertainties include those described under the captions “Item 3. Key Information – Risk Factors” and “Item 5. Operating and Financial Review and Prospects” in AC Immune’s Annual Report on Form 20-F and other filings with the
Source: AC Immune SA