Washington Editor

Hoffmann-La Roche Inc. agreed to spend upward of $215 million in a global partnership with Isotechnika Inc. to develop a transplantation drug with blockbuster potential.

The drug is named ISATX247, and so far, Isotechnika, of Edmonton, Alberta, has spent about $20 million taking the calcineurin inhibitor to early Phase II clinical trials. Isotechnika, and now Roche, is looking to develop ISATX247 as an immunosuppressive treatment for organ transplantation and autoimmune diseases.

In a conference call with analysts and reporters Tuesday, representatives from both companies said early trials of ISATX247 have shown evidence that the compound produces less toxicity and more potency than existing agents when used in transplantation cases.

“There’s been a 20-year search for a better cyclosporine, for one that prevents rejection without the toxic problems that are associated with the existing calcineurin inhibitors,” Robert Gordon, medical director for transplantation at Roche Laboratories in Nutley, N.J., told BioWorld Today. “Especially the toxicity to the kidney, which is not only a problem with kidney transplants, but in liver transplants and in heart transplants because even though they don’t have transplanted kidneys, they still have a kidney.

“Of all the molecules we’ve seen, this is the only one that looks like it might have the potential to make it as a marketable product that would offer a dramatically different therapeutic window and therapeutic index for a calcineurin inhibitor-type class of drugs. Roche does not have a drug in this class at this time,” Gordon said.

Indeed, to seal the deal with Isotechnika, Roche has agreed to pay 70 percent of the development costs and to give Isotechnika a percentage of the gross profits. In exchange, Roche gained exclusive worldwide marketing rights. Specifics were not provided on how the deal’s potential $215 million total would be earned.

The annual U.S. market for calcineurin inhibitors is $1.5 billion, the companies said.

“After years of painstaking research and business development, our business, medical and scientific teams have brought us to the point where our lead drug, ISATX247, has caught the attention of many,” Robert Foster, chairman and CEO of Isotechnika, said in the conference call. “This agreement between our two companies represents the largest single development deal between a large multinational pharmaceutical company and a biotechnology company in all of Canadian history. It is a landmark deal on a global basis.”

In August, Isotechnika filed applications with the FDA and the Health Protection Branch of the government of Canada to begin Phase II trials of ISATX247 in kidney transplantation. The trials were set to enroll 120 patients at 16 centers in North America. (See BioWorld Today, July 23, 2001.)

“We think the compound has a very bright future in transplantation and immunosuppression,” Dennis Burns, vice president, global head of business development for Roche, said in the conference call. “We look forward to taking ISATX247 from Phase II all the way to global commercialization so that its potential can be fully realized. We believe we can bring an important new therapeutic alternative to physicians, and improved efficacy and quality of life to the patients they treat.”

Aside from the transplantation indication, company representatives said the compound will be tested in other diseases.

When asked specifics about future indications or timelines on Phase II and Phase III trials, Foster deferred to a “joint decision-making governance” committee that will be established to shepherd ISATX247 through development and commercialization. The committee, he said, will make all future decisions on the compound.

In terms of a timeline to market, Foster said, “Typically you are looking at four to seven years for development to commercialization, once you get a drug like this into man.”

Roche hopes that ISATX247 can be used in conjunction with its other drugs for transplantation and life after transplantation. They are Zenapax (daclizumab), which works to prevent the acute rejection of the newly transplanted organ; CellCept (mycophenolate mofetil), a long-term immunosuppressive therapy; and Cytovene (ganciclovir), for prevention and treatment of cytomegalovirus (CMV), a viral infection associated with transplantation. Additionally, Valcyte (valganciclovir), a Roche product for CMV, is in Phase III trials.

Last year, Isotechnika raised $16.2 million through the sale of warrants to help pay for Phase II trials of ISATX247, which also is being studied in psoriasis.

Isotechnika has a diagnostic division that focuses on products such as Helikit, a breath-based kit to help diagnosis ulcers.