Pfizer inks deal with OPKO for growth hormone deficiency drug candidate

MIAMI & NEW YORK—A worldwide agreement has been announced between OPKO Health Inc. and Pfizer Inc. to develop and commercialize OPKO's long-acting hGH-CTP for the treatment of growth hormone deficiency (GHD) in adults and children and growth failure in children born small for gestational age (SGA) who fail to show catch-up growth by 2 years of age. Per the agreement, Pfizer will pay OPKO $295 million up front, with the potential for up to an additional $275 million if certain regulatory milestones are achieved. Pfizer will receive an exclusive license to commercialize hGH-CTP worldwide. OPKO also stands to receive initial royalty payments tied to the commercialization of hGH-CTP for adult GHD. Should the product be launched for pediatric GHD, the royalties will transition to gross profit sharing for both hGH-CTP and Pfizer's Genotropin.

While OPKO will lead clinical activities and assume responsibility for funding the development programs for the key indications, Pfizer will be responsible for all development costs in additional indications, as well as all post-marketing studies. Pfizer will also fund commercialization activities for all indications and lead manufacturing activities covered by the global development plan.

"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," Geno Germano, group president of Pfizer Global Innovative Pharma, said in a press release. "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."

OPKO's hGH-CTP could reduce the dosing frequency for patients to a single weekly injection, rather than the current standard-of-care treatment of a daily dose. The compound is currently under investigation in a global Phase 3 trial in adults and a global Phase 2 trial in children, and has been granted orphan drug designation in both the United States and Europe for children and adults with GHD.

"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. Our collaboration enables full alignment between Pfizer and OPKO to optimize development and potentially bring an innovative treatment to patients. 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," Dr. Phillip Frost, CEO of OPKO, commented in a statement.

SOURCE: OPKO press release