Naia Limited, an international drug development company today announced that its subsidiary, Naia Rare Diseases, has been granted by the U.S. Food and Drug Administration (FDA) orphan drug designation for NB1001, its long-acting GLP-1 receptor agonist, for the treatment of Short Bowel Syndrome (SBS).
“The receipt of orphan drug designation for NB1001 is further indication that there is a great unmet medical need for the treatment of Short Bowel Syndrome, and our drug candidate may represent a promising new approach for the treatment of this disease,” said H. Daniel Perez, M.D., co-founder, chairman and chief executive officer of Naia Limited.
Mark Bagnall, co-founder and chief financial officer of Naia Limited, added, “We are committed in advancing NB1001 through clinical trials with the initiation of a Phase 1b dose de-escalating in SBS patients by the second quarter of 2016.”
NB1001 (XTEN™-GLP-1) is a long-acting Glucagon-like peptide-1 (GLP-1) receptor agonist that combines exenatide with a proprietary extended half-life technology. NB1001 will be administered as a replacement therapy (replacing endogenous GLP-1 lost by bowel resection), in contrast to GLP-1 agonists used to treat Type 2 diabetes, which are administered at pharmacologic levels in order to lower blood sugar. Lower doses of NB1001 combined with a longer half-life will provide a differentiated, safe, effective and convenient therapeutic approach for these patients.
GLP-1 is produced by the L cells of the ileum. Upon food ingestion, low concentrations of endogenous GLP-1 act as a negative modulator of gastric and duodenal contractility, slowing the transit of food from the stomach. Slow transit results in better digestion of food and better absorption of nutrients in the small bowel. After extensive intestinal resection, SBS patients lack endogenous GLP-1. Deficiency of GLP-1 leads to fast transit of food through a shortened bowel and diminished absorption of nutrients leading to severe nutritional deficiency and fluid imbalance.
FDA orphan drug designation is granted to investigational therapies addressing rare medical diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
NB1001 was previously being developed for Type 2 diabetes, and in a 70-patient Phase 1b study, NB1001 was shown to be safe, well-tolerated and lowered HbA1c in a dose-dependent manner.
About Naia Limited
Naia Limited builds and funds new biotech companies using de-risked clinical stage assets. Naia’s structure spreads overhead costs and resources during early development of assets thus reducing costs and time. The company has an international base of operations to develop therapeutics better, faster and more inexpensively – in proximity to target markets. Naia’s operating structure includes a Cayman Island-based management company and several holding companies that focus on development activities of like assets. The company is developing a diverse portfolio of therapeutics that address regional and global unmet medical needs in various markets with an initial focus on rare and metabolic diseases.