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news.XOMA Corporation (XOMA), a leader in the discovery and development of therapeutic antibodies, announced XOMA 358, a fully human allosteric monoclonal antibody that reduces both the binding of insulin to its receptor and downstream insulin signaling, has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for the treatment of congenital hyperinsulinism (HI).
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"The orphan drug designation for XOMA 358 recognizes its potential to address a significant unmet medical need for patients with congenital hyperinsulinism. Patients with hyperinsulinism, a rare and devastating disease, secrete inappropriate and excessive insulin, which cause dangerously low blood sugar levels that can lead to brain damage or, in rare cases, death. Currently options to manage many of these patients are limited to continuous ingestion of glucose or pancreatectomy," said Paul Rubin, M.D., Senior Vice President, Research and Development, and Chief Medical Officer at XOMA.
"We are developing XOMA 358 as a first-in-class therapeutic for conditions of hyperinsulinemic hypoglycemia. We recently completed a positive Phase 1 study, results of which suggest XOMA 358 is active against the insulin receptor and shows potential in treating patients who experience an endogenous over-production of insulin. Congenital hyperinsulinism is one of the indications we are considering for a Phase 2 study, which we expect to initiate later this year."
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the United States.
The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance, and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
The OOPD also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
Insulin is the major hormone for lowering blood glucose levels. Abnormal increases in insulin secretion can lead to profound hypoglycemia (low blood sugar), a state that can result in significant morbidities including cerebral damage and epilepsy. In some instances, profound hypoglycemia can be fatal.
XOMA, leveraging its scientific expertise in allosteric monoclonal antibodies, developed the XMet platform. This platform consists of three classes of selective insulin receptor modulators (SIRMs) that could have a major effect on treating patients with abnormal metabolic states.
XOMA 358, the lead antibody in the XMetD program (designed to deactivate the insulin receptor), is a fully human allosteric monoclonal antibody that binds to insulin receptors and attenuates insulin action. XOMA 358 is being investigated as a novel treatment for non-drug-induced, endogenous hyperinsulinemic hypoglycemia (low blood glucose caused by excessive insulin production) and other related disorders.
A therapy that safely and effectively mitigates insulin-induced hypoglycemia has the potential to address a significant unmet therapeutic need for certain rare medical conditions associated with hyperinsulinism.
XOMA presented positive Phase 1 data on XOMA 358 at the Endocrine Society’s Annual meeting (ENDO 2015) in March 2015. Results of the study, in which 14 healthy volunteers received XOMA 358 and 5 received placebo, showed XOMA 358 reduced insulin receptor signaling and increased glucose production after exogenous insulin injection. In the study, XOMA 358 appeared to be well tolerated, with no serious adverse events observed.