Industry Update: Clinical trials

Latest developments compiled from 01 — 31 December 2014

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions 01 — 31 December 2014, scheduled to be published in Volume 10 Issue 3 of Regenerative Medicine.

Clinical trials

CBMG

Cellular Biomedicine Group (CBMG; CA, USA; www.cellbiomedgroup.com) released the 48-week follow-up data analysis of its Phase I/IIa clinical trial for Rejoinâ„¢ human adipose-derived mesenchymal progenitor cell (haMPC) regenerative therapy for Knee Osteoarthritis. The trial, conducted by Shanghai Renji Hospital, tested the safety and efficacy of intra-articular injections of autologous haMPCs in order to reduce inflammation and repair damaged joint cartilage. Additional information about CBMG’s clinical trials of haMPCa therapy for Knee Osteoarthritis is available at http://clinicaltrials.gov (ID: NCT01809769 and NCT02162693)

TxCell

TxCell SA (France; www.txcell.com), a biotechnology company developing novel and innovative, cost-effective personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, has enrolled the first patient in Phase IIb clinical trial of its lead product Ovasave in refractory Crohn’s disease. TxCell has received approval for this multi-center, multinational trial from the regulatory authorities in all six European countries in which the trial will be performed.The Phase IIb study, named CATS29 (Crohn’s and Treg study), includes 32 study sites in 6 countries (Austria, Belgium, France, Germany, Italy and United Kingdom). The trial has been designed to include 160 severe refractory rCrohn’s disease patients. The primary objective of the CATS29 study is the evaluation of the response rate for a single intravenous (i.v.) injection of 1×10⁶ cells dose of Ovasave compared to placebo 6 weeks post administration. Response is defined as a decrease ≥100 points in the Crohn’s Disease Activity Index (CDAI), the gold standard regulatory measure of response in Crohn’s disease. The CATS29 study is a multicentre, randomised, double-blinded, placebo-controlled, multi-dose and multi-injection, 4 parallel groups study. It will evaluate the efficacy and the safety of Ovasave (ovalbumin-specific autologous Treg cells (Ova-Treg)) in patients with active refractory Crohn’s disease. Patients will receive, double-blinded, two i.v. 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 i.v. injections of Ovasave or a safety follow-up with no injection. Finally, there will be an extended safety follow-up for all patients. Top line results of this study are expected at the end of 2016 to early 2017. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT02327221)

Unum

Unum Therapeutics (MA, USA; www.unumrx.com) has begun recruitment in the first clinical trial of the Antibody-Targeted Tumor Cell Killing ATTCK20 therapy. The Phase I study will examine the feasibility, safety and potential efficacy of infusing the ATTCK20 combination therapy in patients with B-cell malignancies and persistent disease following standard therapy. The clinical program commences shortly after the company’s official launch in October 2014. ATTCK happens when T-cells expressing an antibody-coupled T-cell receptor (ACTR) engage a tumor-targeting antibody on the surface of a cancer cell. ATTCK20 is a combination of a patient’s ACTR T-cells administered with rituximab, a monoclonal antibody targeting CD20. The first Phase I dose escalation study for ATTCK20 is taking place in Singapore at National University Hospital (NUH) and Singapore General Hospital (SGH). T-cells from patients in the study are processed at the Tissue Engineering & Cell Therapy (TECT) Laboratory at NUH. The Hematology-Oncology Research Group Trial Unit within the National University Cancer Institute, Singapore (www.ncis.com.sg) manages and supports all aspects of the clinical trial.