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OPKO Announces Positive Interim Six-Month Lagova™ (hGH-CTP) Phase 2 Data in Pediatric Growth Hormone Deficiency Disorder

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Interim efficacy results show that a single weekly injection of Lagova (hGH-CTP) can replace seven consecutive daily injections of currently marketed human growth hormone (hGH)

June 23, 2014

OPKO Health, Inc., 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.

Study Results

An interim analysis of 6 months data demonstrated that all doses of Lagova used in the study provided strong catch-up growth response better than historical controls of daily growth hormone therapy.

The baseline characteristics of all patients were comparable among all groups. Interim analysis of the PK profile following administration of Lagova demonstrates a significantly extended half-life as reflected by the T1/2 and AUC respectively. A dose dependent PD (IGF-1) response was observed between Lagova cohorts, reaching steady state with no accumulation or excessive levels. All cohorts demonstrated promising “catch-up” growth, in line with reported age and GHD severity-matched data. The annualized height velocities are more than 12 cm, which correlates with the PK/PD profile in those patients.

About Lagova (hGH-CTP)

In June 2013, OPKO initiated a pivotal Phase 3 clinical trial in adults for its proprietary long-acting version of hGH-CTP (Lagova). Lagova has been awarded orphan drug designation in the U.S. and Europe for both adults and children with growth hormone deficiency.

ABOUT OPKO HEALTH

OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development and commercialization expertise and novel and proprietary technologies. For more information, visit www.opko.com.

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