Pfizer reported cutting a deal with San Diego's Zacharon Pharmaceuticals today that could pay the latter up to $210 M. It is a very early stage deal allowing Pfizer access to Zacharon's molecules related to treating lysosomal storage disorders. Pfizer agreed to an undisclosed upfront payment and will also contribute R&D funding. Zacharon can earn additional milestone payments for commercialization and sales targets. The project's success will be the responsibility of Pfizer's new orphan and genetic diseases division.
Orphan disease targets have become increasing favorites of Big Pharma as about 25% of all new drug approvals since 2005 have been aimed at them according to the FDA. Orphan drugs can receive expedited review by the agency, often command higher prices, and receive market exclusivity which requires less marketing expense as well.
Current treatments for lysosomal disorders can relieve some of the symptoms but usually have little effect on neurological pathology as they don't typically cross the blood-brain barrier. Zacharon has been able to identify molecules that can cross the barrier and thereby offer hope of a therapeutic effect in the brain. See WSJ.
Posted by Bruce Lehr April 6th 2011.