OPKO Biologics (formerly Prolor Biotech), a subsidiary of OPKO Health Inc., is a clinical stage company developing next-generation, bio-better, long-acting versions of therapeutic proteins and peptide drugs, utilizing CTP and reversible PEGylation technologies. The CTP technology involves fusing one or more copies of a naturally occurring glycosylated peptide by rDNA technology to the protein chain ends. When attached to a therapeutic protein, CTP significantly extends its longevity in the body.
OPKO Biologics’ second technology is reversible PEGylation, a synthetic chemistry-based platform for enhancing the half-life of therapeutic peptides and small molecules. Using a hydrolysable linker bypasses the disadvantages of using a standard PEGylated molecule, i.e. the potential for steric hindrance that limits its function, and the limited ability to cross cellular membranes or the blood-brain barrier.
OPKO Biologics currently presents two bio-better long-acting products:
- hGH-CTP (Somatrogon, MOD-4023): CTP-modified human growth hormone (hGH). OPKO completed a pivotal Phase 3 study in growth hormone-deficient (GHD) adults and is currently conducting a Phase 3 study in a GHD pediatric population. The company completed a Phase 2 study in GHD adults, demonstrating that a single weekly injection of hGH-CTP could potentially substitute seven consecutive daily injections of regular hGH, and showing an excellent safety profile.
- OPK88005: CTP-modified long-acting coagulation factor VIIa, potentiality supporting every-other-day prophylactic treatment or twice a week regimen in hemophilia patients. OPK88005 is currently evaluated in an ongoing Phase 2a clinical study.