Our Focus
We are committed to pursuing severe rare diseases with well-defined biology that can be targeted with therapies tailored to patients’ needs. Our approach leverages unique scientific and clinical insight into the design and development of product candidates with a well understood mechanism of action.
Our Science
About Short Bowel Syndrome
Short Bowel Syndrome (SBS) is a malabsorptive disorder caused by the loss of functional small intestine. SBS typically occurs as a consequence of extensive intestinal resection due to chronic inflammatory bowel disease (IBD), acute events such as trauma, mesenteric infarction, bariatric surgery or congenital abnormalities. The severity of SBS ranges across an anatomical spectrum, and some patients who are initially diagnosed with intestinal insufficiency suffer from progressively worse food and fluid absorption, leading to SBS with chronic intestinal failure (SBS-IF).
As an organ failure condition with unmet medical need, patients with SBS-IF require parenteral support (PS), the intravenous delivery of essential fluids and nutrients, to survive. Patients with the most severe SBS-IF require PS infusions for up to 10 to 15 hours per day. SBS is associated with frequent complications, significant morbidity and mortality, high economic burden and an impaired quality of life. An estimated 32,000 people are thought to suffer from SBS in the U.S. and Europe, of whom 15,000 suffer from SBS-IF and require lifelong PS.
About Apraglutide
Apraglutide* is an investigational drug being developed as a once-weekly treatment for patients who have SBS with intestinal failure (SBS-IF). It is a next-generation, long-acting, synthetic GLP-2 analog that acts as a selective, full agonist of the GLP-2 receptor.
Apraglutide has the potential to enable patients to minimize the burden from PS by increasing intestinal absorption of fluids, calories and nutrients. Apraglutide has successfully completed phase 2 studies and is currently being evaluated in a pivotal phase 3 clinical trial. Based on preclinical and clinical data to date, apraglutide has the potential to advance the treatment SBS-IF, by establishing less frequent dosing and improving outcomes in a clinically meaningful fashion to address the needs of patients across the anatomical spectrum that characterizes the disease.
We plan to assess the safety and efficacy of apraglutide in pediatric SBS-IF and in other conditions where we believe the mechanism of action of GLP-2 has the potential to provide therapeutic benefit due to its impact on intestinal growth and absorption, gastrointestinal (GI) blood flow and GI barrier function and immunity.
Our Pipeline
Our Clinical Trials
We are evaluating the safety and efficacy of apraglutide in a global Phase 3 clinical trial called STARS (STudy of ApRaglutide in SBS) in adults with short bowel syndrome with intestinal failure (SBS-IF).
STARS represents the largest phase 3 trial ever conducted in SBS-IF and has several important features. It is the first trial of a next-generation, long-acting GLP-2 analog designed to exclusively evaluate a once-weekly dosing interval. STARS is also designed to take patient factors, like remnant bowel anatomy, into account. With an anatomically heterogenous disease like SBS, individual patients’ caloric needs will vary while weaning off of parenteral support.
We are actively enrolling patients for the STARS trial. For more information on this trial, study locations and eligibility criteria, please visit ClinicalTrials.gov.
To receive information when we initiate future clinical trials, please send your enquiry to: [email protected].
