OPKO Health, Inc. products

Rayaldee® (calcifediol) is an extended-release prohormone of the active form of vitamin D3 that both raises 25-hydroxyvitamin D and lowers intact parathyroid hormone (iPTH) levels in patients with stage 3 or 4 CKD. Rayaldee® is under development for adult patients with stage 5 chronic kidney disease (CKD) with SHPT and vitamin D insufficiency who require regular dialysis.

Somatrogon, a long-acting human growth hormone (hGH) molecule, is a once-weekly injectable, created using recombinant technology, for the treatment of pediatric and adult growth hormone deficiency (GHD). The molecule consists of the natural peptide sequence of native growth hormone and the 28 amino acids of the C-Terminus Peptide (CTP) of the human chorionic gonadotropin hormone. This molecule, as compared to current GH replacement therapies, is intended to reduce the injection frequency from a daily to once a week in adults and children with GHD.

Selective Androgen Receptor Modulators (SARMs) have recently garnered attention as possible therapeutic agents due to their increased specificity for anabolic androgen receptors, and yet potentially serving as an antagonist to androgen receptors in the prostate. OPK88004 is being assessed as a treatment for muscle wasting, decreased physical strength and function, sarcopenia, and frailty in end stage renal disease (ESRD) patients on dialysis and for prostate cancer patients on Androgen Deprivation Therapy (ADT). The selective anabolic effects of SARM on lean body mass and physical function should provide advantages over other therapies with potential effects on the prostate in aging males.

Oxyntomodulin is a naturally occurring hormone produced in the colon, shown to bind both the GLP-1 and glucagon receptor, regulating both blood glucose appetite and lipid metabolism. OPKO Health is developing OPK88003, a long-acting, once weekly oxyntomodulin analog for the treatment of type 2 diabetes and obesity.

In our Natural Antisense Transcripts Antagonist (AntagoNAT) technology, an oligonucleotide molecule is designed to interfere with regulatory gene expression in order to enhance production of endogenous functional proteins. This strategy is unique and contrasts with established approaches which down-regulate protein production. This platform can lead to the discovery of new drugs to target relevant genes that regulate the optimal production of endogenous functional proteins. One of the leading applications of our AntagoNAT technology is OPK88001 which is designed for the treatment of Dravet syndrome by targeting the mutated SCN1A gene.