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A Unique Pancreatic Human Beta Cells to revolutionize the Diabetes Drug Discovery

Periodic Reporting for period 1 - BETASCREEN (A Unique Pancreatic Human Beta Cells to revolutionize the Diabetes Drug Discovery)

Reporting period: 2016-08-01 to 2017-01-31

Univercell-Biosolutions’ mission is to develop human cell-based assays for Diabetes drug discovery and development. Since 2011, Univercell-Biosolutions (UB) has successfully implemented its first version of immortalized Endoc-BH1 human pancreatic Beta Cells. This represents the first human adult beta cell available on the market to test potential new anti-diabetic drugs in the preclinical phase with high predictive clinical value. To date UB’s Endoc-BH1 has been adopted by 200 prestigious academic centers and 10 world pharmaceutical industry leaders. If immortalization feature of Endoc-BH1 can be seen as strength in term of unlimited quantity accessibility this also represents a market limit in term of business model and industrial applications for drug discovery (insulin stimulated insulin secretion, proliferation, apoptosis). Today, Univercell-Biosolutions will have the opportunity to further develop, improve and industrialize human beta cells assays closer to human reality, to adapt them to industrial high throughput screening assays and larger market demand, and to develop novel beta cells disease models for diabetes drug discovery.

Main objectives of the SME instrument project will be (a) to further further develop, improve and industrialize a fully physiological non proliferative human beta cells assays closer to human reality, (b)to adapt them to industrial high throughput screening assays and larger market demand, and (c) to develop novel beta cells disease models for diabetes drug discovery.

The feasibility study initially programmed within the SME instrument phase 1 included several different complementary deliverables to precise and validate the opportunity to further develop and improve human cell based assays closer to human reality for diabetes drug discovery segment. Our major aim is to determine the market interest and add value of Univercell-Biosolutions Endoc-BH unique approach compared with competitive current solutions available i.e. human primary pancreatic islets, cell lines and hiPSc derived Beta cells.

Objectives of the work plan designed on the SME instrument Phase 1 were:

1- Development of a targeted marketing strategy,
2- Validation of the industrial needs and their interest,
3- Validation of Univercell-Biosolutions IP freedom to operate and IP robustness
4- Definition of a comprehensive Business plan including pricing and commercial strategy
5- Anticipate large scale commercialisation by providing adapted technical results
Within BETASCREEN SME instrument Phase 1, UB has furthermore contacted more than 40 international potential clients/partners to precisely validate its novel approach based on a new business model and associated commercialization strategy. We have precisely defined the design, the shipping solution, the adequate production process and acceptable pricing strategy adapted to partner needs in coherence with market’s targeted different segments (academia and industry). This has led to a robust business model associated with a realistic business plan.
Then, Univercell-Biosolutions has validated the relevance of its existing IP portfolio and the “freedom to operate”.
Industrialization of the production process of Endoc-BH1 ®proposed on SME instrument stands out very positively and demonstrates the significant add value when compared with other human induced pluripotent stem cells (hiPSc) derived beta cells and marketed solutions currently available. Interviews of current customers and potential new prospects have furthermore identified 6 industrials from majors European and US companies of the pharmaceutical industry to be part of our industrialization program as co-developers and “early adopter”. This represents the best validation from the market specific need and strong attempts.

The first outcome of BETASCREEN project is a fully physiological non-proliferative pancreatic beta cell line totally identical to primary human beta islets, with ultimate objective of replication to human beta cells disease models. Scientific preliminary results performed on SME instrument phase 1 demonstrate that our novel strategy to excise the immortalization gene of Endoc-BH lead to impressive level of insulin content (X14 compared with Endoc-BH1) absolutely similar to human primary pancreatic islets from patients. This extraordinary result represents a major progression which provides a solution to the major bottleneck highlighted by industrials and related to Endoc-BH version 1. So, this important technical validation brings an evident robust answer to open large Endoc-BH1 non accessible commercial applications.
Cell based assay robustness in term of reproducibility and sensibility to existing pharmacology compounds of Endoc-BH5 is qualified by industrials and academia clients/partners as a significant progression regarding other human beta cells already available on the market and current Endoc-BH1.

This represents for our clients a significant evolution beyond the current state of the art which opens extraordinary perspectives on implementation of innovative cutting edge drug discovery strategies within the Diabetes field and development of associated novel Therapeutics. Endoc-BH5 is a fully functional human beta cell displaying physiological level of insulin secretion associated to the capacity to assign new important questions related to Diabetes as beta cell protection (apoptosis) and proliferation.