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.
Using those two technologies, OPKO Biologics presents three bio-better long-acting products:
- MOD-4023: CTP-modified human growth hormone (hGH). OPKO initiated a pivotal Phase 3 study in growth hormone-deficient (GHD) adults and is currently conducting an advanced Phase 2 study in a GHD pediatric population. The company completed a Phase 2 study in GHD adults, demonstrating that a single weekly injection of MOD-4023 could potentially substitute seven consecutive daily injections of regular hGH, and showing an excellent safety profile.
- MOD-5014: CTP-modified long-acting coagulation factor VIIa, potentiality supporting every-other-day prophylactic treatment or twice a week regimen in hemophilia patients. MOD-5014 is in an advanced pre-clinical development stage, targeted for Phase 2a by Q4 2015.
- MOD-6031: long-acting reversibly PEGylated oxyntomodulin (OXM) for the treatment of type 2 diabetes and obesity. MOD-6031 injected once weekly for five weeks in obese and diabetic mice resulted in marked weight loss and glucose control, as compared to twice-daily injections of the native OXM peptide at the same dose. MOD-6031 is in advanced pre-clinical development, targeted for Phase 1 by Q1 2016.