Parkinson's partnership

SAN DIEGO—Ceregene Inc. is getting a big boost in an already successful area of drug development thanks to a deal with Cambridge, Mass.-based Genzyme Corp. for the development and commercialization of CERE-120, Ceregene's proprietary lead program for the treatment of Parkinson's disease. Genzyme gets in on a compound that is complementary with its own Parkinson's drug development work and Ceregene gets an accelerated and expanded commercial reach for its product.

"We've been very successful so far, initiating this program and taking it forward by ourselves up to this point," says Dr. Raymond T. Bartus, Ceregene's senior VP of clinical and preclinical R&D and COO, as well as chairman of the partnership's joint steering committee. "We even completed a Series C round of financing as well for $27 million, so financially we were in great shape for this program and our other programs."

The additional money Genzyme brings is useful, Bartus admits—a $25 million up-front payment in exchange for certain partnership-related expenses and development-related milestone payments of up to $125 million, as well as 50 percent reimbursement of the worldwide Phase III development costs. And Genzyme's own technical experience is a valuable add-on to Ceregene's.

But the real payoff wasn't money or technical know-how but rather what Genzyme brings in terms of infrastructure, regulatory knowledge, international presence and marketing expertise.

"These are the kinds of things that will allow us to move into Europe and the Pacific Rim much more rapidly than we could have imagined otherwise," Bartus says. "Everything we have hoped from our CERE-120 program has been borne out, and much more than we hoped, and that isn't just hype and hubris. But despite knowing that we had something good and could carry it on our own, the attraction of being able to expand to other countries, continents and marketplaces and make the therapy available to great numbers of people more rapidly was too attractive to ignore."

"We are very enthusiastic about the potential that CERE-120 may have to improve the lives of people with Parkinson's disease, which currently affects at least one million Americans," said David Meeker, president of Genzyme's Lysosomal Storage Disorders unit, announcing the deal. "We look forward to helping further the development of this exciting approach, building on our long-term commitment to gene therapy and neurodegenerative diseases."

The two companies will work together outside of North America, with Genzyme gaining marketing rights in all markets outside of the United States and Canada, and Genzyme paying Ceregene tiered royalties based on sales in markets where it has rights. Ceregene will retain exclusive rights to CERE-120 in the United States and Canada.

"We have driven the discovery and development process in North America up to this point and we will continue to do so," Bartus says.

Genzyme is itself conducting a separate Phase I-II clinical trial of its own gene therapy for Parkinson's disease. Erin Emlock, a spokesperson for Genzyme, says there are no plans at this point to create any direct connections between that program and the CERE-120 program, but it still speaks to synergies that exist between the two companies, she says.

Unlike Ceregene's CERE-120, which aims to restore damaged and dying neurons, the Genzyme gene therapy is designed to improve the therapeutic effectiveness of levo-Dopa in Parkinson's patients by enhancing the brain's ability to convert it into dopamine. "It's a slightly different approach than Ceregene is taking, but an important one," Emlock says. "As Parkinson's disease kills cells in the brain, you also lose an enzyme called AADC, which is needed to convert levo-Dopa, and which makes the drug less effective over time. We're looking to help restore the effectiveness and make levo-Dopa therapy viable for a longer period of time."