OPKO Health, a biopharmaceutical company, and Pfizer have formed a worldwide agreement for the development and commercialization of OPKO's long-acting recombinant human growth hormone analogh (hGH-CTP) for treating growth hormone deficiency (GHD) in adults and children as well as for treating growth failure in children born small for gestational age (SGA) who fail to show catch-up growth by two years of age. hGH-CTP has the potential to reduce the required dosing frequency of human growth hormone to a single weekly injection from the current standard of one injection per day. hGH-CTP is currently in a global Phase III trial in adults and a global Phase II trial in children and has orphan drug designation in the US and Europe for both adults and children with GHD.

Under the terms of the agreement, OPKO will receive an upfront payment of $295 million and is eligible to receive up to an additional $275 million upon the achievement of certain regulatory milestones. Pfizer will receive the exclusive license to commercialize hGH-CTP worldwide. In addition, OPKO is eligible to receive initial royalty payments associated with the commercialization of hGH-CTP for adult GHD that is subject to regulatory approval. Upon the launch of hGH-CTP for pediatric GHD, which is subject to regulatory approval, the royalties will transition to gross profit sharing for both hGH-CTP and Pfizer's Genotropin.

OPKO will lead the clinical activities and will be responsible for funding the development programs for the key indications, which includes adult and pediatric GHD and pediatric SGA. Pfizer will be responsible for all development costs for additional indications as well as all post-marketing studies. In addition, Pfizer will fund the commercialization activities for all indications and lead the manufacturing activities covered by the global development plan.

The transaction is subject to customary Hart-Scott-Rodino approval and is expected to close during the first-quarter of 2015.

hGH-CTP is intended to reduce the burden of daily injection therapy by requiring only weekly injections potentially improving compliance and treatment outcomes. OPKO's proprietary technology allows the company to extend the hormone's half-life without the use of polymers, encapsulation techniques, or nanoparticles. This technology is based on a natural peptide, the C-terminal peptide (CTP) of the beta chain of human chorionic gonadotropin (hCG).

Source: Pfizer