CAT-192 is safe but efficacy in doubt

UK-headquartered Cambridge Antibody Technology and the USA's Genzyme Corp have revealed that Phase I/II trials of CAT-192 for scleroderma showed the human anti-TGF-Beta1 monoclonal antibody to be safe and well tolerated across all dose levels, although no conclusions regarding efficacy of the compound could be made.

The primary endpoint of the double-blind, placebo-controlled, 45-patient trial was to assess the safety, tolerability and pharmacokinetics of CAT-192. Patients received one of three dose levels (0.5mg/kg, 5mg/kg or 10mg/kg) or matching placebo, intravenously every six weeks for four doses. Results show that CAT-192 met the primary goal and no treatment-related adverse effects were observed.

The secondary objective of the trial was to evaluate various clinical endpoints and biological markers potentially indicative of disease progression, and preliminary examination of the results demonstrated that gender and disease duration were significant factors. However, patients taking the placebo did not deteriorate as expected during the trial, and this, coupled with a small sample size, led to inconclusive results regarding the efficacy of the drug.