Discovering novel antibodies derived from the human body itself to treat neurodegenerative diseases

Neurodegenerative diseases are devastating diseases with huge unmet medical needs. Decades of research have been invested in finding cures, however almost none have been successful so far. Antibodies are great therapeutic molecules as they can target different aspects of disease-causing processes with high specificity. Within this class, human-derived antibodies stand out for their unique properties and therapeutic potential. For example, they are naturally designed by the human immune system to fight toxic moieties and have matured in the human body, resulting in an expected higher efficacy and specificity compared to antibodies deriving from synthetic libraries or animal models. However, human subjects harboring target-specific antibodies for neurodegenerative diseases are extremely rare, down to 1 every 10,000 people. Thus, to identify such rare individuals an extremely large number of blood samples needs to be screened, which is rarely available to drug discovery.

Mabylon’s antibody discovery platform overcomes these limitations with access to one of the largest and most diverse libraries of blood samples deriving from up to 100,000 human subjects per year. The library further distinguishes itself by containing both diseased and healthy patient samples which, together with access to patient medical history, advanced data-analytics, and cutting-edge B-cell screening technology, overall increases the chance of finding protective, therapeutically relevant antibodies.

Mabylon’s lead molecule is an antibody against pathological TDP-43 for the treatment of
amyotrophic lateral sclerosis (ALS) and a second antibody for Alzheimer’s disease (AD) therapy.

For the SMEi phase 1 feasibility study, we investigated the technical validity, the market potential and competitor landscape of our primary lead candidate targeting TDP-43 and our antibody in AD. Moreover, we established partners for preclinical development and validated our licensing strategy. We have consolidated these results in a business plan to support future funding activities.

Task 1.1 Market and competitor Research of neurodegenerative diseases market for ALS and FTD.
We have performed an extensive market and competitor investigation for our TDP-43 antibody, primarily focusing on ALS. As a result, we devised a realistic perspective on our market position for the development of our TDP-43 antibody. In addition to this market research we also performed technical validation studies consulting Key-opinion-leaders in the field to create a multi-perspective feasibility analysis for our TDP-43 antibody.

Task 1.2 Market and competitor Research for pipeline diversification
We have performed an extensive market and competitor investigation for our novel antibody in AD. As a result, we devised a realistic perspective on our market position for the development of our novel antibody in AD. In addition to this market research we also performed technical validation studies consulting Key-opinion-leaders in the field to create a multi-perspective feasibility analysis for our antibody in AD.

Task 1.3 Partnerships search for brain delivery and animal models
We established several crucial partnerships for the development of our platform, as well as initiated discussion with partners for brain delivery systems and animal model systems.

Task 1.4 Development of licensing strategy
We had conversations with multiple potential licensing partners, which validated our strategy to out-license one or several antibodies after completion of a preclinical proof-of-concept.

Task 1.5 Business plan consolidation
Based on the findings in Task 1.1-1.4 we consolidated our results in a business plan that includes a strategy for clinical validation, regulatory approval, IPR, and market launch. Furthermore, the business plan contains risk and mitigation analyses, financial models, and commercialization models.

The results of the feasibility study put Mabylon in an ideal position to complete a preclinical Proof-of-Concept and to leverage these results into a licensing deal within a year, which will be crucial for the development of our technology platform for treatment of patients with neurodegenerative diseases.

This will help to bring one of the first truly effective therapies for a neurodegenerative disease to patients, by leveraging the unique properties of naturally designed, Mabylon engineered, fully human antibodies. With this we aim to have a long-lasting significant impact on the treatment landscape for neurodegenerative diseases.

Periodic Reporting for period 1 - HominAb (Discovering novel antibodies derived from the human body itself to treat neurodegenerative diseases)

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