Tempest’s Amezalpat Gains FDA Fast Track For Liver Cancer After Promising Trial Data

Tempest Therapeutics, Inc. is a biotechnology company developing best-in-class targeted and immune-mediated therapeutics to fight cancer. Today, the firm announced the approval of Fast Track Designation (FTD) for its lead molecule, Amezalpat. The drug is an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC). This is the second regulatory designation granted to Amezalpat.

Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest, said in a statement, “We are thrilled to receive Fast Track designation from the FDA. This designation, following the Orphan Drug designation granted last month, reinforces the promise of amezalpat as a potential treatment option for patients affected by HCC. We look forward to working closely with the FDA and foreign regulatory agencies to develop amezalpat with the goal of bringing this promising therapy to patients.”

In January, the company obtained an Orphan Drug Designation (ODD) for amezalpat from the U.S. FDA based on its clinical study performance with positive safety and efficacy data at multiple endpoints when combined with atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone for first-line treatment in patients with unresectable or metastatic HCC. 

The randomized comparison trial recorded positive results showing a six-month longer median overall survival (OS) among patients taking amezalpat combination drugs at a hazard ratio (HR) value of 0.65. This survival advantage was preserved when amezalpat was evaluated as an add-on treatment for multiple patient subtypes based on PD-L1 staining status. This finding matches how amezalpat works by attacking cancer cells and activating immune defence systems within patients.