CymaBay Therapeutics Inc (NASDAQ:CBAY) stock climbed Tuesday after it said the Food and Drug Administration cleared the company’s investigational new drug (IND) application for seladelpar to treat a chronic liver disease, called primary sclerosing cholangitis (PSC).
The Newark, California-based company intends to initiate a Phase 2 study to evaluate the safety, tolerability, and potency of seladelpar in patients with PSC in the third quarter.
Investors hailed the development, sending the stock up nearly 2% to $6.7.
PSC is a rare, chronic liver disease that is characterized by inflammation and fibrosis of the bile ducts. The disease predominantly affects the medium to large-sized bile ducts inside and outside the liver and causes ductal destruction leading to fibrosis, and cirrhosis. There are currently no FDA-approved treatments for PSC.
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, said the company.
Launching Phase 2 study in 3Q
The Phase 2 study will be a randomized, placebo-controlled, dose-ranging study that will enroll approximately 100 patients at 60 sites globally, said the company in a statement.
Seladelpar at doses of 5, 10, and 25 milligrams 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 from baseline at 24 weeks.
“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, at the University of Miami.
Dr Pol Boudes, chief medical officer at CymaBay, said the initiation of a Phase 2 study of seladelpar in PSC is a “significant step” forward in addressing the high unmet need that exists for PSC patients.
“As we continue to progress the Phase 3 development of seladelpar in primary biliary cirrhosis (PBC), it is important to expand the potential indications where the drug may have clinical activity,” said Dr Boudes. “We are eager to commence this dose-ranging study to further evaluate our hypothesis that seladelpar can improve the clinical course for patients with PSC.”
The FDA approval gives CymaBay Therapeutics a boost after it suffered a setback earlier this month after reporting that its lead drug, seladelpar, posted disappointing results in a mid-stage clinical trial linked to nonalcoholic steatohepatitis (NASH).
NASH is liver inflammation and damage caused by a buildup of fat in the liver. Patients with this disease are often told they have a "fatty liver."
Contact Uttara Choudhury at [email protected]