BioWorld International Correspondent
LONDON - Cambridge Antibody Technology Group plc and partner Genzyme Corp. announced preliminary results from a Phase I/II trial of CAT-192 in the treatment of scleroderma, showing the anti-TGF beta-1 antibody was safe and well tolerated, but providing no definitive indications of efficacy.
At the same time, CAT said it is ending its collaboration with Elan Corp. plc, of Dublin, Ireland, in neurology and pain, three years into a four-year agreement.
CAT CEO Peter Chambré told BioWorld International: "The CAT-192 trial met its primary endpoint of safety and got a big tick for that. The secondary objective was to understand more about the disease and how anti-TGF beta-1 works in it. We learned an enormous amount."
The trial enrolled 45 patients suffering from diffuse systemic sclerosis who were randomized to receive one of three dose levels of CAT-192, or placebo.
CAT-192 was generally safe and well tolerated at each dose level, and elimination half-life was around three weeks. A number of clinical endpoints and biological markers, potentially indicative of disease progression, also were studied. The skin score of patients in the placebo group did not, as anticipated, deteriorate during the trial, leading the two partners to conclude that "no definitive conclusions regarding the efficacy of CAT-192 can be drawn at this time."
Chambré said CAT and Cambridge, Mass.-based Genzyme would be working in the next few months on plans to take CAT-192 forward, based on the findings of the trial. CAT and Genzyme also expect that Phase I trials of GC-1008 in idiopathic pulmonary fibrosis will start soon. The human monoclonal antibody is active against TGF beta-1, -2 and -3.
The companies have been collaborating on TGF beta since September 2000. In September 2003 they agreed to broaden the alliance, with CAT increasing its financial contribution to 50 percent in return for a significant increase in its share of profits. At the same time, Genzyme invested £22.9 million (US$42.6 million) in CAT, taking its stake to 11 percent.
The move to strengthen the relationship with Genzyme was part of CAT's strategy to focus its financial resources on core programs in which it will get a high rate of return, while licensing its antibody technology as widely as possible to partners who are prepared to pay development costs.
Chambré said the decision to end the Elan collaboration, agreed in January 2001, was in line with that strategy.
"Neurology and pain is not part of our future; I don't believe we would end up co-funding a development program," he said. "That doesn't mean our technology can't be used in that area, and we feel our ability [to find other partners] has been constrained by the exclusive deal with Elan."
CAT and Elan have worked on a number of targets, but Chambré said that no products have been discovered during the collaboration.