APM-176: Targeting Non Cystic Fibrosis CFTR-Related Chronic Diseases
- Many surfaces in the body, including the nasal passageways, pancreas, liver, lungs, and intestines depend on CFTR to maintain the balance of fluids.¹
- 1 in 25-30 people in the US carry CFTR Mutations.²
- Carrying a CFTR mutation increases the probability of a CFTR Related Disorder (CFTR-RD).³
- We are targeting the following indications: Chronic rhinosinusitis (with or without polyps), pancreatitis, IBS-C, liver cirrhosis, bronchiectasis.
APM-176 is a differentiated Phase 2 ready oral small molecule that has been shown to directly potentiate wildtype and mutated CFTR in in-vitro studies.
References:
¹ MacDonald K, McKenzie K, Zeitlin P. Cystic fibrosis transmembrane regulator protein mutations: ‘class’ opportunity for novel drug innovation. Pediatr Drugs. 2007; 9(1): 1–10.
² Philip M. Polgreen, Alejandro P. Comellas. Clinical Phenotypes of Cystic Fibrosis Carriers. Annual Review of Medicine. 2022; 73(1): 563–574.
³ Miller AC, et al. Cystic fibrosis carriers are at increased risk for a wide range of cystic fibrosis-related conditions. Proc Natl Acad Sci U S A. 2020 Jan 21;117(3):1621–1627.