Opko HealthInc., a multinational biopharmaceutical and diagnostics company, and Pfizer Inc have entered into a worldwide agreement for the development and commercialisation of Opko's long-acting hGH-CTP for the treatment of growth hormone deficiency (GHD) in adults and children, as well as for the treatment of growth failure in children born small for gestational age (SGA) who fail to show catch-up growth 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 the current standard of one injection per day. hGH-CTP is currently in a global phase 3 trial in adults and a global phase 2 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 licence to commercialise hGH-CTP worldwide.
In addition, Opko is eligible to receive initial royalty payments associated with the commercialisation of hGH-CTP for Adult GHD which is subject to regulatory approval. Upon the launch of hGH-CTP for Paediatric 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 programmes for the key indications, which includes Adult and Paediatric 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 commercialisation activities for all indications and lead the manufacturing activities covered by the global development plan.
"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 programme. Our collaboration enables full alignment between Pfizer and Opko to optimise 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 utilising 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
cief executive officer, Phillip Frost, MD.
"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 Paediatric 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."
The transaction is subject to customary Hart-Scott-Rodino approval and is expected to close during the first-quarter of 2015.
hGH-CTP is a novel, long-acting recombinant human growth hormone analog being developed by Opko for the treatment of children with growth failure due to inadequate endogenous growth hormone secretion, and adults with growth hormone deficiency (GHD) of either childhood or adult-onset etiology. 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). Opko has an ongoing pivotal phase 3 clinical trial in adults for hGH-CTP and a phase 2 clinical trial in paediatric patients. hGH-CTP has been granted orphan drug designation in the US and Europe for both adults and children with growth hormone deficiency.