Scientists at CymaBay are committed to identifying unique targets in pathways that have critical roles in disease. We are developing novel small molecules that regulate metabolism, inflammation, and fibrosis with the potential to address unmet needs in the treatment of inflammatory liver diseases and other chronic diseases.
| Indication or Program | Phase 1 | Phase 2 | Phase 3 |
|---|---|---|---|
|
Primary Biliary Cholangitis incomplete responders (PPARĪ“ agonist) |
Phase 1 Phase complete
|
Phase 2 Phase complete
|
Phase 3 Phase in progress
|
Seladelpar
Primary Biliary Cholangitis |
Phase 1 Phase complete
|
Phase 2 Phase complete
|
Phase 3 Phase in progress
|
|
Primary Sclerosing Cholangitis |
Phase 1 Phase in progress
|
Phase 2 Phase not started
|
Phase 3 Phase not started
|
|
Diabetic Hypoglycemia (GPR 119 agonist) |
Phase 1 Phase complete
|
Phase 2 Phase in progress
|
Phase 3 Phase not started
|
Seladelpar, is a first-in-class oral, selective delpar, or peroxisome proliferator-activated receptor-delta (PPARĪ“) agonist, that has been shown to regulate critical metabolic and liver-disease pathways. It is currently being evaluated in a Phase 3 study of patients with primary biliary cholangitis (PBC), a progressive cholestatic inflammatory liver disease. Seladelpar has received Breakthrough Therapy Designation (FDA) in the United States for the treatment of early stage PBC and PRIME status (EMEA), as well as Orphan Drug Designation in the United States and Europe for the treatment of patients with PBC. It is backed by clinical evidence and global patient experience through its Phase 2 and Phase 3 studies,1 and we initiated an international pivotal Phase 3 study of seladelpar in patients with PBC in 2021.1 Future research could indicate whether it has the potential to be effective in treating other liver diseases such as primary sclerosing cholangitis (PSC).
MBX-2982, an agonist of G protein-coupled receptor (GPR119), is being evaluated in a Phase 2 proof-of-pharmacology trial for the treatment of diabetic hypoglycemia conducted by the AdventHealth Translational Research Institute in Orlando, Florida and is fully funded by The Leona M. and Harry B. Helmsley Charitable Trust. CymaBay retains full commercial rights to MBX-2982. In published preclinical studies, GPR119 agonists were shown to enhance glucagon secretion from human pancreatic islets in response to low glucose levels. They were able to prevent insulin-induced hypoglycemia via a glucagon response in animal models. To confirm these findings in patients with type 1 diabetes, MBX-2982 is in a Phase 2a proof-of-pharmacology study to determine whether the drug can enhance glucagon secretion during insulin-induced hypoglycemia in subjects with type 1 diabetes (T1D).
Glucagon is a hormone secreted by the pancreas that naturally reverses hypoglycemia by signaling the body to release stored glucose. This process can become defective in individuals with insulin-dependent diabetes, such as T1D. If left uncorrected, hypoglycemia can lead to unconsciousness or death. Preventing episodes of hypoglycemia in persons with diabetes is an unmet medical need: according to the 2016 global HAT study of 27,000 people, 4 out of 5 individuals with T1D reported hypoglycemia, with a rate of severe hypoglycemia (requiring the assistance of another person) of approximately five events per patient-year.
1. Li NX, et al. Diabetes. 2018;67:1401-1413.
