Tempest Therapeutics, Inc. (TPST), a clinical-stage biotechnology company, announced on Monday that the U.S. Food and Drug Administration has granted Orphan Drug Designation to amezalpat or TPST-1120 an oral, small molecule, selective PPAR antagonist, for the treatment of hepatocellular carcinoma or HCC.
Amezalpat is being evaluated in a global Phase 1b/2 clinical study, which has shown positive results when combined with the standard-of-care therapies, atezolizumab and bevacizumab, for first-line treatment of patients with unresectable or metastatic HCC.
HCC, an aggressive and hard-to-treat cancer, is projected to become the third-leading cause of cancer death by 2030.
The combination therapy demonstrated a significant 6-month improvement in median overall survival with a hazard ratio of 0.65, along with a 30 percent objective response rate or ORR versus 13 percent for the standard treatment alone.
The positive data also showed that the survival benefits from adding amezalpat were maintained in key sub-populations, including patients with PD-L1-negative disease and ß-catenin mutations.
These findings align with amezalpat's proposed mechanism of action, targeting both tumor cells directly and modulating the immune system.
The FDA's Orphan Drug Designation for amezalpat highlights the urgent need for new treatment options in this area. This designation provides benefits such as tax credits for clinical testing, fee waivers, and seven years of market exclusivity upon approval.
Tempest is currently preparing for a pivotal Phase 3 study for amezalpat in first-line HCC patients and continues to work with both the FDA and European Medicines Agency or EMA to advance the therapy's development.
Cash position:
Tempest ended the third quarter with $22.1 million in cash and cash equivalents.
Currently, TPST is trading at $0.96 up by 1.27%.
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