BEL-0216 (PEG-HGH) – Growth Disorders.
BEL-0216 is a novel, long-acting recombinant human growth hormone (“HGH”) being developed to treat growth hormone deficiency (GHD) and Turner’s Syndrome, an orphan disease. BEL-0216 utilizes Belrose’s proprietary PEGylation technology to create a more effective treatment for GHD and Turner’s Syndrome.
BEL-0216 is the only long-acting HGH development program that leverages the FDA’s approval of the recombinant HGH molecule (somatropin) to accelerate the regulatory approval process:
- BEL-0216 uses Belrose’s proprietary releasable linker technology that prolongs exposure in the body and then releases the native FDA-approved drug in its original, unmodified form, without a “tail” or residue.
- Belrose anticipates the regulatory requirements and clinical risk of BEL-0216 to be lower than other development-stage GHD programs due to the combination of “clean” releasable linker and FDA-approved native molecule.
Mechanism of Action / Advantages
Human growth hormone (GH or HGH), also known as somatotropin or somatropin, is a peptide hormone that stimulates growth, reproduction and regeneration in humans. HGH is produced by the pituitary gland and spurs growth in children and adolescents. It also helps to regulate body composition, body fluids, muscle and bone growth, sugar and fat metabolism, and possibly heart function. Synthetic human growth hormone was developed in 1985 and approved by the FDA for specific uses in children and adults. In children, HGH injections are approved for treating short stature of unknown cause as well as poor growth due to a number of medical causes such as GHD, Turner’s Syndrome and Idiopathic Short Stature (“ISS”).
BEL-0216 is a proprietary, long-acting human growth hormone (HGH) that Belrose has developed using an FDA-approved HGH and its proprietary releasable linker technology:
- Conjugating the recombinant human growth hormone (rHGH) with releasable PEGylation allows BEL-0216 to be made available to the body in its original form with no residual components.
- This proprietary therapeutic improves the clinical outcomes of a range of patients by reducing dosing frequency, enhancing the side effect profile and improving patient compliance.
Clinical Trial Overview
Belrose is preparing to hold a pre-IND meeting with the FDA to confirm the specific clinical and non-clinical requirements to support approval. Belrose expects to progress BEL-0216 through a final preclinical toxicology program followed by a Phase I human clinical trial in 2015, followed by two Phase II/III pivotal clinical trials to support approval for the initial orphan indication.