OPKO Health, Inc. and Pfizer have entered into a worldwide agreement for the development and commercialization of OPKO’s long-acting hGH-CTP for the treatment of growth hormone deficiency (GHD) in adults and children, as well as growth failure in children who fail to show catch-up by 2 years of age. hGH-CTP has the potential to reduce the required dosing frequency of human growth hormone to a single weekly injection from one injection per day. Currently in a Phase III trial in adults and a Phase II trial in children, hGH-CTP has orphan drug designation in the U.S. and Europe for GHD.
 
OPKO will receive an upfront payment of $295 million and is eligible to receive an additional $275 million based on certain regulatory milestones, and is eligible to receive initial royalty payments associated with commercialization. Pfizer will receive the exclusive license to commercialize hGH-CTP worldwide. OPKO will lead clinical activities and will be responsible for funding the development programs for the key indications. Pfizer will be responsible for all development costs for additional indications as well as all post-marketing studies and commercialization activities.
 
“We believe this collaboration will help advance our commitments to patients with Adult and Pediatric Growth Hormone Deficiency as we believe Pfizer’s strengths, expertise and presence in the human growth hormone space makes them the ideal partner for our hGH-CTP program. We believe that the global growth hormone market is currently valued at more than $3 billion, and believe that hGH-CTP has the potential to be the best in class long-acting growth hormone product. Our long acting human growth hormone is our most advanced product candidate utilizing our CTP technology to extend the half-life of a broad range of therapeutic peptides and proteins. By reducing the number of injections, our technology can improve patient compliance,” said OPKO’s chief executive officer, Phillip Frost, M.D.
 
“This agreement strengthens Pfizer’s commitment to rare diseases, and we are pleased to work with OPKO to help provide a potential next-generation therapy for patients with Adult and Pediatric Growth Hormone Deficiency,” said Geno Germano, group president, Pfizer Global Innovative Pharma (GIP). “Long-acting growth hormone is the first innovation in the GHD space in 20 years. hGH-CTP would be complementary to our existing Genotropin franchise, and could potentially provide an option that could improve patients’ adherence to treatment with once weekly dosing.”