Our two proprietary technology platforms are product focused and highly productive engines for the sustained growth of our Company.
Our biological SupraAntigenTM and chemical MorphomerTM platforms are our scientific basis to generate a robust pipeline of antibodies, vaccines and small molecules which selectively bind to misfolded proteins representing the underlying causes of a broad range of neurodegenerative diseases.
Our platforms address two key issues in the development of therapeutics and diagnostics for neurodegenerative diseases:
- The body does not make antibodies against misfolded proteins because, although pathogenic, they are still recognized as “self-proteins” and do not trigger an immune response.
- The difference between a normal protein and a pathological protein is only related to a conformational change in protein structure making drug specificity difficult to achieve.
Immunotherapy against conformation specific targets
The SupraAntigen™ technology platform was initially developed by our co-scientific founders, Dr. Claude Nicolau in collaboration with Dr. Fred van Leuven to solve the problem of the lack of immunogenicity of “self” proteins. This technology generates conformation specific antibodies and is used to create products for active immunization (vaccines) and passive immunization (antibodies).
The vaccine product approach is based on the ability of peptide antigens attached to liposomes to elicit the body’s own immune system to produce antibodies against “self”-proteins. The vaccines are composed of synthetic peptides and liposomal anchors to mimic the pathological conformation of the antigen, an adjuvant to enhance the immunogenicity and liposomes as carriers for the peptides and adjuvant. The key features of the vaccines derived from this platform include the high selectivity for conformational targets and a favorable safety profile due to a T-cell independent mechanism of action that does not trigger T-cell correlated inflammation.
The anti-Abeta vaccine ACI-24, currently in a Phase 2 clinical trial in Alzheimer's disease and in a Phase 1b clinical trial in Down syndrome, and the anti-pTau vaccine ACI-35, currently in a Phase 1b/2a clinical study, were derived from the active immunization approach of the SupraAntigen™ platform.
Our SupraAntigen™ platform can also be used to generate antibodies that can be used as therapeutic and diagnostic products. Such antibodies are generated by injecting the SupraAntigen constructs in mice and by selecting the antibodies for their ability to break up aggregated forms of misfolded proteins and to change the equilibrium from the insoluble pathological to the soluble forms which are depleted by the antibodies.
The anti-Abeta antibody crenezumab, currently in a Phase 2 Alzheimer prevention trial, and the anti-Tau antibody semorinemab, in two Phase 2 clinical trials, were derived from the passive immunization approach of the SupraAntigen™ platform.
Generation of conformation specific small molecules
The Morphomer™ technology platform was initially developed through a collaboration of our co-scientific founders Dr. Jean-Marie Lehn and Dr. Claude Nicolau. The rational chemical design enables us to generate small molecules, so called Morphomers, which bind very specifically to misfolded proteins, break up neurotoxic aggregates and inhibit their aggregation and seeding. Other key assets of the robust library of Morphomers include promising CNS drug features such as excellent brain penetration, bioavailability and metabolic stability which are important for the development of both therapeutic and diagnostic agents for multiple neurodegenerative diseases.Three therapeutic (Morphomer Tau, Morphomer Abeta and Morphomer a-synuclein) and two diagnostic development candidates (Tau-PET imaging agent and Morphomer a-synuclein (PET) imaging agent) originate from the Morphomer™ technology platform.