CymaBay Therapeutics Announces IND to Commence a Clinical Study of Seladelpar

CymaBay Therapeutics (NASDAQ:CBAY) has announced that the US Food and Drug Administration (FDA) has cleared the company’s investigational new drug application for seladelpar to treat primary sclerosing cholangitis (PSC).

As quoted in the press release:

The company intends to initiate a Phase 2 study to evaluate the safety, tolerability, and efficacy of seladelpar in patients with PSC in the third quarter.

PSC is a rare, chronic cholestatic liver disease that is characterized by diffuse inflammation and fibrosis of the bile ducts. The disease predominantly affects the medium to large-sized bile ducts inside and outside the liver and is manifested by ongoing ductal destruction leading to cholestasis, advanced fibrosis, and cirrhosis. There are currently no FDA-approved treatments for PSC. Seladelpar is an orally administered, potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist that is also in development for primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). In clinical studies, seladelpar has demonstrated anti-cholestatic and anti-inflammatory activity, suggesting the potential of seladelpar as a therapeutic option for the treatment of PSC.

The planned Phase 2 study will be a randomized, placebo-controlled, dose-ranging study that will enroll approximately 100 patients at 60 sites globally. Seladelpar at doses of 5, 10, and 25 mg once daily will be studied versus placebo in a 1:1:1:1 randomization. The primary efficacy outcome will be the relative change in alkaline phosphatase (AP) from baseline at 24 weeks.

“PSC patients currently have significant unmet need for effective treatment options. I am encouraged by the effects of seladelpar observed in the PBC clinical program, such as the anti-cholestatic and anti-inflammatory activity as well as the favorable safety and tolerability profiles, which suggest a potential benefit for PSC patients,” said Dr. Cynthia Levy, Division of Hepatology, University of Miami.

Click here to read the full press release.