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AC IMMUNE SA filed this Form 20-F on 03/21/2019
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Morphomer alpha-synuclein: Our Morphomer alpha-synuclein product candidate is a small molecule that reduces the cytotoxicity of alpha-synuclein aggregates by a decrease in their beta sheet content. In pre-clinical studies, Morphomer alpha-synuclein significantly reduced in vivo the formation of alpha-synuclein pathological structures accompanied by improvement of a neuronal marker relevant to PD. Ongoing activities are focused on optimizing potency and pharmacokinetic properties and preparing compounds for pre-clinical development activities.


Anti-alpha-synuclein antibody and TDP-43 antibody: The two antibody programs targeting alpha-synuclein and TDP-43 were discovered using the SupraAntigen technology platform. Both antibody programs have unique binding properties allowing them to bind to unique epitopes of the pathological forms of alpha-synuclein and TDP-43, respectively. Alpha-synuclein is an established target for Parkinson’s disease and other Lewy body diseases, while TDP-43 is a recently identified target of growing interest for neuro-orphan indications such as Frontotemporal Lobar Degeneration. Interestingly, TDP-43 also plays an important role in other significant neurodegenerative indications such as AD.


Neuroinflammation: Neuroinflammation has been linked to the pathology associated with neurodegenerative diseases. Furthermore, scientists believe that neuroinflammatory biomarkers may aid in the early detection and monitoring of disease progress. Thus, in addition to our programs targeting proteinopathies, we are using our two proprietary platforms to generate molecules that may serve as therapeutics as well as diagnostic tools. Currently, we have five projects in the discovery stage with the first lead to advance into preclinical development in 2020.




Scientists believe that early detection of neurodegenerative diseases is critical to enhancing the effectiveness of both symptomatic and disease-modifying therapies. As a result, therapeutic development for AD increasingly focuses on treating early stage disease to delay or prevent progression and to preserve the maximum amount of cognitive function before it is irreversibly lost. Most clinical studies now target mild or even pre-clinical stages of the disease increasing the need for accurate diagnosis that is independent of potentially subjective cognitive metrics. At least one study estimates that as many as one-third of patients in previous AD studies did not in fact have AD. Accurate and early diagnosis of AD is thus a substantial unmet market need, and diagnostic products will have a key role in generating a new treatment paradigm, including by selecting more uniform and stage-specific clinical study subjects, tracking patient progress and results, managing patients receiving treatment, and ultimately diagnosing disease at its earliest stage for immediate treatment.


Figure 26: The need of Precision Medicine in AD: High level of other proteinopathies and co-pathologies in AD



Ref: Adapted from Robinson, et. al., Brain, 2018



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