AXENIS HUMICE - Humanized mice as innovative tools for transversal biomedical research & preclinical validation.
Biomedical research faces a variety of ‘Grand Challenges’ in vaccine development & infectious diseases, control of chronic diseases with inflammation-related pathology, and definition of innovative cancer treatment strategies. Among these challenges, validation of new therapeutic approaches increasingly depends on access to relevant experimental models to screen human-dedicated, clinical products in a preclinical, in vivo setting. In the era of personalized molecular medicine, rodent models that do not properly reflect human cell physiology or do not efficiently translate into advances for human health are still used too often in biomedical research, therefore limiting the capacity to generate new and efficient therapeutic advances.
As a spin-off of the renowned Institut Pasteur (Paris, France), AXENIS is an innovative company exploiting cutting-edge humanized, immuno-deficient mouse models for research and development (R&D) preclinical studies. AXENIS goal is to become the ‘first-to-market’ with its forefront innovative tools that represent a major technological breakthrough in the huge market of vaccine and drug discovery/development. AXENIS mission is to develop and offer to the biopharmaceutical community rigorous, reproducible and standardized animal models that give access to prospective ‘read-out’ of human immune functions.
The unique AXENIS chimeric mouse models harboring components of the human immune system (a.k.a. ‘Human Immune System’ [HIS] mice) are intended to overcome the well-documented limitations of species barriers for bio-therapeutics testing in existing animal models – including standard rodent models and non-human primates.
AXENIS HIS mice dramatically broaden the scope of pre-clinical testing of human immune cell behavior, with numerous potential applications in fields such as immunology, infectious diseases, stem cell biology, inflammation or immuno-oncology – affecting ≥10 million new patients per year worldwide and a preclinical testing market of ~2500M€ in 2012. Overall, HIS mice represent a crucial tool for this large, transversal market.
A mature technology & a right timing for market penetration
The HIS mouse models marketed by AXENIS are backed up by approximately 30 years of research with key technologies protected by patents granted to the Institut Pasteur (owners). AXENIS has secured exclusive, worldwide licenses for these patents and ‘know-how’ until 2022. HIS mouse models require highly specific expertise in their handling and in results interpretation. They are the main focus of AXENIS internal R&D efforts. AXENIS aims at being the principal actor in this area within the next 5 years and at staying at the forefront of this rapidly evolving field in biology by constantly developing its products, its market penetration, and collaborative strategies.
Over the past 5 years, AXENIS has developed its commercial activity in two directions, namely:
(i) Direct sales of HIS mice to international customers, e.g. large biopharmaceutical companies, contract research organizations (CRO), biotech small & medium enterprises (SME) and academic institutions;
(ii) Hosting of R&D projects outsourced by customers (CRO activity);
As a result, AXENIS currently valorizes several proof-of-concept results that clearly demonstrate the added value of HIS mice over the existing models, as a family of predictive models for the screening of therapeutic molecules and identification of human biomarkers. This major achievement is the outcome of collaborative joined forces with academic partners (research collaborations; academic technology transfer; public incentives and consortia) and private customers (~10 different biotech companies and one CRO). Technology-wise, AXENIS has also validated its products & services development strategy.
Action plan
Facing increasing worldwide market demands for accessing its products (HIS mice) & services (CRO), AXENIS aims at scaling up its production capacity while maintaining its quality standards. Over the next 3 years, the major goal of AXENIS is therefore to enhance both direct sales and CRO operations, in terms of production facilities, commercial organization and company structuration into respectively dedicated business units (BU). To achieve these objectives, the overall company project focuses on building a ‘first-in-class’ humanized mouse factory for increased distribution capacity and the support of its CRO/R&D activity. Humanized mice produced in this ‘humanization’ BU will offer all compliance features (reliability, reproducibility, quality, ethics) required for biomedical R&D.
To value this scale-up strategy, AXENIS aims at setting-up a ‘beta-testing’ program with big players in the aforementioned application fields (a biopharmaceutical company – e.g. Sanofi – and a large CRO – e.g. EVOTEC), and therefore anticipate market evolutions that will determine internal and external R&D efforts. Market dissemination of such innovative in vivo models is expected to take some time (3 years). The European Union (EU) support to AXENIS development plan is therefore of utmost importance at this initial stage to guaranty its positioning visibility, success at the international level and in attracting additional private investors.
The investments required for the development of the next generation of HIS mouse models and up scaling of AXENIS’ operations have been estimated at ~5.1M€ over the 2016-2018 time frame (including ~3.5M€ eligible costs in the context of the H2020-SME call). The development strategy of AXENIS takes into account the logistic and regulation compliance related to animal experimentation and shipment of living animals. The company aims at consolidating its position on the European market (2015-2017) before establishing joint ventures in North America (NA) and Asia (2018+) to facilitate the distribution channels and operations.
HIS mouse models as unique research tools represent a small but essential ‘missing link’ in the drug discovery/development and preclinical processes. This positions AXENIS as an excellent European company candidate to receive the support of EU and attract new investors.
