Letter from the CEO

John Tchelingerian_web_0_0.jpg

Dear stakeholders,                                                        

The human body is made from about trillions of individual cells. In a healthy state, all cells interact with each other in balance and harmony, thus creating a homeostatic state. In a diseased state, however, this equilibrium is in disarray with miscommunication and abnormal activities on a cellular level leading to acute, progressive and often chronic diseases.

Our industry has evolved from treating diseases purely with chemically derived substances to using biomolecular factors or biologicals, such as antibodies. In today’s clinical practice, these molecules are used to block a certain signaling pathway, pull a switch to heat up or cool down the immune system, flag cancer cells for destruction and many other tasks. In the human body, all these modes of action are initiated, orchestrated and performed by living cells and the full range of proteins and effectors they produce. Cells were designed by nature to act as a living source of simple and sophisticated molecules with therapeutic modulating effects. Unlocking the promise of cell-based medicines is therefore the next frontier in modern medicine.

The liver is a remarkable organ and one of the largest of the human body. Its regenerative potential surpasses many if not all other organs. It is rich with stem and stem-like cells that form a regenerative complex offering multiple layers of protection for the crucial functions this unique organ serves. At Promethera Biosciences, we acknowledge the centrality of the liver in human health, as a point where many diseases manifest and therefore as a central point of therapeutic intervention. As our industry is fostering the maturation of cell-based medicines, we at Promethera are highly convinced that the healthy human liver can be the superior source of innovative and potent cell therapy products.

Promethera considers itself an innovator and a pioneer in the cell therapy space of liver diseases. Promethera just celebrated its 8th Anniversary and has gathered a robust body of expertise and experience both on an individual and organizational level across all aspects a biopharmaceutical company requires. To point out just a few examples, Promethera’s team members have already successfully developed products independently into and through clinical development and up to registration and market launch, including the first ATMP in Europe. In 2016, we acquired and integrated the key assets of our competitor, Cytonet, which brought us complimentary technologies, manufacturing capabilities and a real US presence with an experienced team.

The two programs Promethera is currently developing independently are based on its proprietary HepaStem lead platform, which has been at the core of the Company since its inception. In 2017, we intend to advance the clinical evaluation of this cell therapy approach both in Acute-on Chronic Liver Failure, ACLF, and Nonalcoholic steatohepatitis, or NASH. Investment in these programs is our top priority, as they provide our best and largest opportunity to ­generate substantial value for the Company.

ACLF represents a very tough to treat indication with a high mortality and no effective therapy outside ICU interventions. Novel therapies that have the potential to modulate the acute systemic and in particular liver inflammation in these patients, improve disease progression and have a positive impact on overall survival could bring a significant benefit to these patients. At the same time, evaluating the anti-inflammatory potential of HepaStem early in a clinical setting in ACLF could facilitate our development plan in NASH.

NASH is already considered the ‘next big global epidemic’. Currently there are an estimated 62 million people living with NASH in the US, EU and Japan alone.. We believe the multi-factorial intervention HepaStem cells could provide for, with at least both anti-inflammatory and anti-fibrotic effects, has clear advantages over existing competition (mainly small molecules and some biologicals) and could allow carving out a significant position in this market with a successfully developed product.

Our most advanced cell therapeutics today are derived from human livers with the aim to treat liver diseases. Our ambitions, however, go beyond that. We believe liver-derived cells can be augmented with additional therapeutic functions using modern gene transfer methods and could therefore treat with targeted effects even broader range of diseases.

Overall, Promethera is well positioned to continue its path of making liver-derived cell therapies for serious medical conditions and patients suffering from such diseases a reality. In biopharmaceutical drug development, many hurdles and uncertainties must be overcome, critical decisions made and success is never guaranteed. We approach this challenge with respect for the science and biology, patients response complexity, diligence and with a certain degree of courage, because being at the forefront of the next medical revolution is not for the faint hearted.

 

Respectfully Yours,

John Tchelingerian, PhD

CEO