Pfizer To Develop GHD Drug With OPKO Health

Pfizer announced it has partnered with Miami-based OPKO Health to develop the latter's long-acting hGH-CTP as treatment for growth hormone deficiency (GHD) syndrome in both adults and children, as well as for growth failure in children born small for gestational age (SGA) who fall short of catch-up growth standard by 2 years old.

hGH-CTP is a novel, long-acting recombinant human growth hormone analog in development for the treatment of pediatric patients with growth failure due to inadequate endogenous growth hormone secretion and adults with growth hormone deficiency (GHD) of either childhood or adult-onset etiology. The drug presents the opportunity to reduce the burden of daily therapeutic injections to a single weekly injection. hGH-CTP is being investigated in a late stage trial in adults and a global mid-stage trial in pediatric patients for GHD. It has also received Orphan Drug Designation both in the U.S. and EU in both pediatric and adult indications.

Under the terms of the partnership, Pfizer will pay OPKO an upfront fee of $295 million and will be eligible to receive up to $275 million in milestone payments based on the achievement of regulatory goals. Pfizer in turn will gain exclusive rights to commercialize the drug worldwide. OPKO will be responsible for providing necessary funding for development programs in the key indications of adult and pediatric GHD as well as pediatric SGA. The company will also fund all commercialization and manufacturing activities for the drug as part of its global development plan.

OPKO's CEO,  Phillip Frost, said, “We believe that the global growth hormone market is currently valued at more than $3 billion, and we 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.”

Geno Germano, group president of Pfizer Global Innovative Pharma (GIP), said, “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. 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 companies said they expect the transaction to be completed during the first quarter of 2015.