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Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - TREGS4DM1 (Commercialization of TREG cells sorting and proliferation in vitro for the production of vaccines for the treatment of type 1 diabetes.)

Teaser

Summary

Diabetes mellitus type 1 (DM1) represents up to 10% of all diabetes cases. The disease can occur at any age, but the onset is more common in children and young adults. It is estimated that worldwide approximately 86,000 of new cases of DM1 are diagnosed in children under the age of 15 each year, and the incidence is increasing by approximately 3% annually (IDF Diabetes Atlas, 2015).
The most important factor underlying the etiology of DM1 is impairment in the regulation of the immune system, associated with a T cell dependent autoimmune attack. In diabetic patients insulin-producing Î² cells are recognized and destroyed by self-immune cells. Despite the fact that we are currently able to detect DM1 before complete loss of insulin-producing cells and to identify individuals being in the asymptomatic stage of DM1 development (presence of autoantibodies against Î² cell antigens), there are presently no available effective therapies to prevent disease progression and clinical manifestation. The current medical management for DM1 is based on insulin replacement therapy. The aim is to lower fasting blood glucose levels as much as possible to the normal range of 3.9-5.5 mmol/L. Good glycemic control helps to avoid acute episodes of life-threatening hyper- and hypoglycemia, as well as long-term neurological dysfunctions and vascular complications, leading to severe disability (e.g. vision loss, urinary incontinence, loss of feeling in limbs, muscle weakness and pain, and end-stage kidney disease).
The overall objective of the project was to carry out a set of actions leading to a feasibility report on full-scale market commercialization. The project was aimed at de-risking the investment in an innovative project and a company for investors. Phase 1 proved to be instrumental in the Companyâ€™s understanding the regulatory and business environment. The company has performed a thorough investigation of many assumptions which in the end allowed for an evolution of the business idea, that attracted both VC and PE investors. The Company has identified key market barriers, which are of regulatory nature and analyzed the market to validate the above-the-standard effect of the therapy and influence on the national healthcare (including possible reimbursement by governments with relation to the overall costs of the diabetes care). The main aim for PolTREG was to increase clinical availability of vaccine using autologous TREGs for the benefit of the patient and design of cost-effective process thus increase likelihood of market uptake and distribution of innovative (better targeted) therapy developed by an European SME.

Work performed

During the course of the Phase 1, the Company has carried out multiple actions aimed at moving closer to commercial application of its innovative cell therapy, treating one of autoimmune diseases (and possibly more in the future). Feasibility report includes the results of performed works compiled in a comprehensive document. The knowledge gained thanks to the Phase 1 was instrumental in attracting two investment funds to the Company.
After the Company was awarded Advanced Therapy Medicinal Product (ATMP) status in the first half of 2016 by the European Medicines Agencyâ€™s (EMA) Committee for Advanced Therapies (CAT), a thorough analysis of regulatory pathways was carried out to find the best option for introducing a cell-based medicinal product, including accelerated approval. The Company has held talks with the EMA, which resulted in acknowledging that, based on current knowledge, it is known that activity of Tregs is crucial for autoimmunity prevention and lack of these cells leads to onset of multiple autoimmune diseases including Diabetes Mellitus Type 1 (DM1). Due to the potentially high impact of Tregs on cells of the innate and adaptive immune responses, they could be a very effective tool as a cell therapy to delay progression of DM1 and preserve Î² cell mass. This would prolong insulin independence in early diagnosed diabetic patients and protect them from acute and late diabetic complications. In market and business analysis as well as talk with potential stakeholders (Ministry of Health), the Company has concluded, that there is pressing need to offer a solution lowering the costs of care over consequences of DM1, that are more costly than the disease itself. Market analysis has also proved that the price suggested by PolTREG is competitive in comparison to other cell therapies available on the market and that there is willingness to pay, mainly due to the fact that relatively high price is compensated by a mechanism of approaching the underlying cause of the disease and not only mitigating its effects.
Works during the Phase 1 have also validated that the technology used by PolTREG is in compliance with current best practice. In addition, the company has also validated its business model towards market entry strategies to other countries under the Hospital Exemption rule as well as towards its own GMP facility and IP protection strategy.

Final results

Phase 1 allowed the company to conclude a strategy that proves that the project shall continue with few alterations. The vision has evolved towards a more focused approach to marketing authorization, especially accelerated approval. Focus on full market authorization also arises from the fact, that the more advanced clinical phase, the more profitable royalty fees. The Company will focus on finishing the construction of its own GMP facility (co-financed by the Operational Program), training its own laboratory staff and focusing on clinical trial. This will form a sold proof-of-concept that the process is viable not only on a laboratory scale (as it is now performed in Gdansk) but also on a small-industrial scale.
Phase 1 has also allowed us for a conclusion that investors and payers (governments) seek for platform solutions and the notion that Company should focus on process improvement has changed towards researching new applications for TREGs. Investors acknowledged that the Company has the potential to form a multi-disciplinary team that can find new solutions to pressing medical needs using a personalized and targeted technology. Thanks to the project, the Company is better prepared to attract investors and other capital needed for clinical trials and then marketing authorization, which will bring the therapy to all in need and allow for a governmental reimbursement.