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2012 

Phase I/II: Promethera® Biosciences treats first patients with Promethera® HepaStem

Promethera Biosciences has treated its first four patients with its innovative treatment based on allogeneic adult liver stem cell technology (Promethera ® HepaStem). These patients were treated at the Cliniques Universitaires Saint-Luc (Brussels, Belgium). Two patients with Crigler-Najjar syndrome and two patients with a deficiency in ornithine transcarbamylase deficiency received the low dose. Patients were aged from 17 years old to 9 months at the time of cells administration.

 

The trial, a prospective, open label, multicenter- phase I/II study testing Promethera® HepaStem is designed to evaluate the safety and the preliminary efficacy of Promethera® HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients.

 

Development of a mobile unit (Van) for the formulation and distribution of the innovative cell therapy product, Promethera® HepaStem

The cell therapy product developed by Promethera (Promethera® HepaStem) is a hepatic progenitor cell suspension that must be infused to the patient in a medical setting within 6 hours after its final formulation. The underlying idea was to transpose the aseptic and temperature-controlled formulation process performed at Promethera’s unique plant at Mont-Saint-Guibert into a mobile unit (=van) brought near the clinical site, so as to timely and consistently deliver the product within its shelf-life.

 

A closed formulation system, where the product does not come into contact with the environment, was designed and validated to aseptically prepare the final product in a non-aseptic environment. A temperature controlled production room was designed and fitted on a truck frame. It is used to both transport the required equipment to the clinical site and to perform the formulation.

 

This innovation enables the delivery of the cell therapy product to remote patients who cannot be reached within 6 hours from Promethera’s facilities. The process and the equipment have been inspected and approved by the Belgian authorities (AFMPS).

 

The closed system ability to maintain the aseptic conditions in an uncontrolled environment has been fully demonstrated and, as part of the validation process, a specification-compliant batch of HepaStem was successfully formulated in the mobile unit at the Birmingham Children Hospital (UK) as well. Several patients were then treated with HepaStem formulated in the mobile unit in the Cliniques Universitaires Saint-Luc (Brussels, Belgium) and in the Hôpital Jeanne de Flandre (Lille, France).

 

Promethera® Biosciences raises EUR 23.6 million (~ USD 31.4 million) in Series-B financing round in March 2012

Promethera® Biosciences attracted new investors in this second round, securing a further capital increase of EUR 17 million. In addition, the Walloon Region, which has backed the project since its inception, has granted a loan of EUR 6.6 million to support the clinical development of the HepaStem product.

Five new investors have acquired equity in Promethera: Boehringer Ingelheim Venture Fund, the venture capital fund of the pharmaceutical giant; Shire, a leading biopharmaceutical company; Mitsui Global Investment, the venture capital fund of Japanese industrial conglomerate Mitsui & Co Ltd.; ATMI, a global leader in semiconductors and innovative culture systems.

In addition to these four groups of international scope and reputation is Sambrinvest, the investment fund of the Gosselies region in Wallonia. This Series-B fundraising has been successful thanks to the major achievements made by Promethera Biosciences since its Series-A fundraising in 2009. 

 

Approval by the Belgian authorities (AFMPS) and British authorities (MHRA) of a Phase I/II clinical trial involving Crigler Najjar and Urea Cycle Disorders patients.

Promethera® Biosciences obtained the first authorizations from the British (MHRA) and Belgian (AFMPS) regulatory authorities to begin the phase I/II clinical study of Promethera® HepaStem. As part of the clinical study, the cell therapeutic product is assessed in a paediatric setting involving children suffering from Crigler-Najjar syndrome or urea cycle disorders. Most young patients have only very limited therapeutic options and may die at an early age. 

 

GMP accreditation of the manufacturing facility located in Mont-Saint-Guibert granted by the Belgian Authorities.

GMP certification of Promethera’s facilities has been delivered for the manufacture and the quality control of Promethera’s core product Promethera® HepaStem. Promethera’s GMP facilities consist in a platform covering 327 m² including three grade B aseptic rooms, dedicated Quality Control areas and warehouses.

This accreditation of Promethera's high level quality system has allowed the initiation of the manufacturing campaign for the clinical lots that will be used in the phase I/II clinical trial.