Short Bowel Syndrome (“SBS”, or short gut syndrome) is a disease caused by the surgical removal of the small intestine, or rarely due to the complete dysfunction of a large segment of bowel. SBS leads to the malabsorption of nutrients and as a result, SBS patients suffer from dehydration and malnutrition. The 4-year survival rate in newborns is 70%, but for those with only 10% of expected bowel length the survival rate is only 10%.
Many patients require chronic treatment with Total Parenteral Nutrition (TPN) a procedure in which nutrients are introduced intravenously. SBS patients on TPN often need intravenous feeding for 6 to 8 hours per day, 5 to 7 days per week, significantly affecting their quality of life. In addition, the use of a catheter increases the likelihood of serious and life-threatening infections.
Naia Rare Diseases is developing drugs for rare gastrointestinal diseases. The company’s lead program, NB 1001, is being developed for Short Bowel Syndrome and is expected to launch in 2019 for use in adults and in 2020 for children.
The goal of SBS therapy is therefore to wean patients off TPN and increase their ability to absorb nutrients. A number of methods are used, including allowing the natural process of intestinal adaption to occur, surgery and treatment of symptoms such as diarrhea and bloating. All of these have varied effects on the ability of patients to wean off TPN. In 2012, the USFDA approved a new drug, teduglutide, a glucagon-like peptide-2 analog for SBS which works by increasing the surface area of the remaining gut. While effective at lowering the volume of TPN, treatment with teduglutide has not allowed many additional patients to be weaned off TPN.
NB 1001 was developed based on an observation by Dr. Mark Pimentel of Cedars Sinai, that treating SBS patients with a GLP-1 agonist, such as the diabetes drug, Byetta, has a positive effect on their ability to wean themselves off TPN. GLP-1 is produced by in the small intestine and slows the transit of food from stomach. Slower transit results in better digestion of food and better absorption of nutrients by the shortened small bowel. This natural “brake” is lost after intestinal resection in SBS patients.
Based on observations with GLP-1s, we believe that a GLP-1 agonist that is specifically designed to treat SBS patients (vs. those developed to treat diabetes) will have a significant effect on the need for TPN and could act as a functional cure for the disease for many patients.
NB 1001 will enter clinical studies in 2016 and could be on the market as soon as 2019.