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CymaBay Down on Dismal Interim Data From Mid-Stage NASH Study

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Shares of CymaBay Therapeutics, Inc. plunged significantly after it announced disappointing interim data from a mid-stage study on lead pipeline candidate, seladelpar. 

Seladelpar is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in development for non-alcoholic steatohepatitis (NASH) and primary biliary cholangitis (PBC).

The double-blind, placebo-controlled study randomized 181 subjects with biopsy-confirmed NASH and a liver fat content (LFC) greater than 10% to receive either placebo or seladelpar 10 mg, 20 mg, or 50 mg once-daily. The primary endpoint was the relative change in LFC from baseline to 12 weeks.

Top-line results from the ongoing 52-week phase IIb dose-ranging, paired liver biopsy study of seladelpar at 12 weeks showed that treatment with seladelpar resulted in minimal reductions in liver fat that were not significant enough compared to placebo.

Nevertheless, reductions in markers of liver injury were robust and clinically meaningful. The study remains blinded and will continue to 52 weeks, with assessments including a liver biopsy, non-invasive imaging evaluations, and biomarker assessments of inflammation and fibrosis.

Seladelpar demonstrated a favorable safety and tolerability profile at all doses evaluated in the study.

A chronic liver disease, NASH, is caused by excessive fat accumulation in the liver or steatosis. The ailment is anticipated to be the leading reason behind liver transplantation by 2020. Currently, NASH is the primary reason for liver transplants in people under 50 years in the United States. With no treatments currently approved to address this disease, the market opportunity is substantially huge. Thus, many companies are investing a major chunk of their R&D budget on the same.

Hence, the dismal interim data disappointed investors.

CymaBay’s stock has lost 23.1% in the year so far compared with 16.1% decline for the industry.

 

NASH is a complex disease and there have been quite a few failures of late. Conatus Pharmaceuticals’ phase IIb ENCORE-NF study in patients with biopsy-confirmed NASH and liver fibrosis also failed to achieve goals.

Earlier, biotech bigwig Gilead Sciences, Inc. (GILD - Free Report) too suffered a setback with the failure of a late-stage study on pipeline candidate, selonsertib, in patients with compensated cirrhosis (F4) due to NASH.

Intercept Pharmaceuticals, Inc had earlier announced positive top-line results from its pivotal phase III REGENERATE study of obeticholic acid (OCA) on patients with liver fibrosis due to NASH. While the results showed promise, investors weren’t impressed with additional data.

We expect investors to focus on further updates from the study.

Zacks Rank

CymaBay currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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