Tempest Gets FDA Approval for Phase 3 Trial of Amezalpat Combo Therapy for First-Line Liver Cancer

On November 12, 2024, Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company, announced it has received approval from the U.S. Food and Drug Administration (FDA) to proceed with a pivotal Phase 3 clinical trial. This trial will evaluate the efficacy of amezalpat (TPST-1120) in combination with atezolizumab and bevacizumab, compared to the standard treatment alone, for the first-line treatment of unresectable or metastatic hepatocellular carcinoma (HCC).

Dr. Sam Whiting, chief medical officer and head of R&D at Tempest, expressed the team's excitement about the FDA's approval. He highlighted previously reported Phase 2 data that suggested amezalpat could improve survival rates for patients with this severe condition. The dedicated team at Tempest is focused on advancing this program to make amezalpat available to patients as soon as possible.

The Phase 3 trial is designed as a global, blinded, randomized study. It will compare the combination of amezalpat, atezolizumab, and bevacizumab to a regimen of atezolizumab and bevacizumab with a placebo, the current standard of care. In August 2024, Tempest received the FDA's agreement on the study design, dosage, and statistical plan, which includes a pre-specified efficacy analysis potentially expediting the primary analysis timeline. The company aims to initiate the Phase 3 trial in the first quarter of 2025.

Amezalpat is an oral small molecule that selectively antagonizes PPAR⍺. Research suggests it combats cancer by directly targeting tumor cells and modulating immune suppressive cells and angiogenesis within the tumor microenvironment. In ongoing global Phase 1b/2 studies, amezalpat combined with atezolizumab and bevacizumab showed superiority across multiple endpoints, including overall survival for both the entire population and key subpopulations, compared to the standard treatment alone. These findings were further supported by results from a Phase 1 trial involving patients with advanced solid tumors, such as renal cell carcinoma and cholangiocarcinoma, who had received extensive previous treatments.

Tempest Therapeutics is headquartered in Brisbane, California. The company is committed to developing a broad array of small molecule therapies that target tumors and/or modulate the immune system, with the potential to treat various cancers. Their innovative programs span from early research to advanced stage studies in global, randomized trials for first-line cancer patients.