The United States Food and Drug Administration (FDA) has accepted TxCell SA’s Investigational New Drug (IND) application for its lead product, Ovasave, currently in a phase 2b clinical trial for the treatment of patients with refractory Crohn’s disease.

The study is currently one of largest ever-controlled studies for a personalised T cell immunotherapy product.

The activation of the IND authorises TxCell to extend the CATS29 study to the United States. The CATS29 study is currently on-going in Europe following its start in December 2014. There are currently 30 study sites operating the study in 6 countries in the EU. It has been designed to include 160 severe refractory Crohn’s disease patients. The extension of this study to US.sites could be initiated in the first part of 2016. Ovasave materials manufactured in a US or EU GMP facility will be required to start enrolment in a clinical trial under the IND.

“This active US IND is a very important milestone for TxCell as it is the first ever obtained by the company with one of its products,” said Stéphane Boissel, chief executive officer of TxCell. “It is a sign of maturity for TxCell and, importantly, for its cellular immunotherapy technology based on regulatory T cells products, a field in which our company is a pioneer and a world leader.”

“The receipt of the IND for the CATS29 study with Ovasave from the FDA will give TxCell an option to extend the trial in the US. The extension to this trial, already one of largest ever-controlled studies for a personalised T cell immunotherapy product, could give additional resources, namely in terms of patient recruitment, to accelerate the study,” said Miguel Forte, chief operating officer of TxCell. “The IND provides further opportunities for Ovasave to progress smoothly and rapidly to phase 3 and commercialisation. It also opens the possibility to assess the benefit of our antigen specific T regulatory cell therapy in refractory Crohn’s disease patients who have no alternative treatment options, from US sites.”

The CATS29 study is a multi-center, randomized, double-blinded, placebo-controlled, multi-dose and multi-injection, 4 parallel groups study. The study is currently on-going in 30 study sites in 6 countries (Austria, Belgium, France, Germany, Italy and United Kingdom). This study could now be extended to include US sites. The trial has been designed to include 160 severe refractory Crohn’s disease patients. The primary objective of the CATS29 study is the evaluation of the response rate for a single intravenous injection of 1.106 cells dose of Ovasave (ovalbumin-specific autologous Treg cells (Ova-Treg)) compared to placebo 6 weeks post administration. Response is defined as a decrease is greater than or equal to 100 points in the Crohn’s Disease Activity Index (CDAI), the gold standard regulatory measure of response in Crohn’ disease. Patients will receive, double-blinded, two intravenous (iv) injections 8 weeks apart of either 1.104, 1.106, or 1.107 cells of Ovasave or placebo. Patients will then receive either an open-label treatment with 2 additional iv injections of 1.106 cells of Ovasave or a safety follow-up with no injection. Finally, there will be an extended safety follow-up for all patients.

TxCell develops innovative, personalised T cell immunotherapies for the treatment of severe chronic inflammatory diseases with high medical need.