CymaBay Therapeutics Inc (NASDAQ: CBAY) stock plummet 45.45% on June 11th, 2019 (Source: Google finance) after the company’s data from an ongoing Phase 2 trial showed patients with nonalcoholic steatohepatitis (NASH) who were treated with investigational drug seladelpar did not show significant reductions in liver fat when compared with those on a placebo. The stock slightly recovered over 9.1% in the pre market session of June 12th, 2019.
CBAY has announced 12-week topline results from an ongoing 52-week Phase 2b dose-ranging, paired liver biopsy study of seladelpar for the treatment of nonalcoholic steatohepatitis (NASH). Seladelpar is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in development for NASH and primary biliary cholangitis (PBC). In the Phase 2 study, three oral doses of seladelpar, all performed worse than a harmless placebo, based on a non-invasive imaging test that measures decreases in liver fat. The study had enrolled 181 patients with a moderate stage of NASH and treated them for 12 weeks. However, the treatment with seladelpar did result in a reduction in biomarkers associated with liver injury. The 52-week liver biopsy data is expected to allow the company to understand whether the improvement in liver injury markers will translate into histological, or microscopic improvement.
Moreover, these data demonstrated a notable decrease in biochemical markers of liver injury despite lack of overall improvement in liver fat. The dose-response relationship in serum ALT reduction is very encouraging and supports the potential histopathologic benefit of seladelpar in patients with NASH.
Meanwhile, NASH is a liver disease driven recently due to the obesity epidemic and sugary diets. It occurs when the livers of a portion of patients with fatty liver disease becomes inflamed, stiff, and scarred, and it leads to thousands of liver transplants a year in the US alone. There are no marketed drugs for NASH. Intercept Pharmaceuticals is expected to file for approval of what could be the first NASH drug, obeticholic acid (OCA), later this year. OCA is the first in a long, growing line of experimental treatments to near the finish line, followed by a drug from the French firm Genfit and many more.
On the other hand, the company had raised $107.7 million in net proceeds through the March public offering of common stock. The company has held $264.8 million in cash, cash equivalents and marketable securities at March 31, 2019. Existing cash is expected to fund the current operating plan into 2021.