OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and diagnostics company, today announced 6 month results of a Phase 2 dose-finding study evaluating the safety and efficacy of its novel long-acting human growth hormone product (Lagova) to treat pediatric growth hormone deficiency disorder (GHD).
All three Lagova once-weekly doses demonstrate strong catch-up growth during the six months treatment. The annualized growth rates are above 12 cm in all three doses. The results are supported by excellent dose dependent pharmacokinetics (PK) and pharmacodynamics (PD) profiles. Lagova shows a promising safety profile with no serious adverse events reported. Glucose and lipid metabolism markers are within the normal ranges. No lipoatrophy was observed in any patients dosed, and no clinically significant local tolerability issues were identified.
“The interim results further affirm that a once-weekly administration of Lagova can replace daily injections of marketed hGH in pediatric GHD patients. The results enable dose selection for the company’s upcoming Phase 3 pediatric trial,” said Dr. Ron Rosenfeld, clinical advisor on the study and professor of Pediatrics (emeritus), Stanford University and professor of Pediatrics at Oregon Health and Science University (emeritus). “Because Lagova consists of native human growth hormone attached to a C-Terminal Peptide of endogenous hormone, one would anticipate low immunogenicity,” Rosenfeld noted.
“Based on these encouraging safety and efficacy results, OPKO plans to move aggressively into a single confirmatory pivotal Phase 3 study for pediatric GHD patients. We hope to make Lagova available to pediatric GHD patients as soon as possible,” said CEO, Phillip Frost, M.D. “Lagova is one of a family of important products being developed at OPKO Biologics designed to improve compliance and offer ease of administration to patients.”Study Design The randomized, comparator-controlled Phase 2 study was conducted in up to 56 pre-pubertal, naïve GHD children receiving one of three Lagova doses as once-weekly regimen (0.25, 0.48, 0.66mg/Kg/week; equivalent of 0.18, 0.35, 0.48 mg/Kg/week of hGH) or daily hGH (34µg/Kg/day) subcutaneously. In order to introduce naïve patients to the allocated Lagova dose in a gradual manner, a stepwise dose increase approach was implemented. Once patients reached the targeted doses, Lagova, GH, IGF-1 and IGF-BP3 concentrations were measured and PK-PD analysis was conducted utilizing a population based approach.