On April 21, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TPST-1495, the company’s novel dual receptor inhibitor of prostaglandin (PGE2) signaling, for the treatment of patients with Familial Adenomatous Polyposis (FAP) (Press release, Tempest Therapeutics, APR 21, 2025, View Source [SID1234651994]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"Receiving orphan drug designation for TPST-1495, our second clinical program, is a significant milestone in our mission to bring innovative therapies to patients with unmet medical need," said Stephen Brady, President and CEO of Tempest. "This designation for the treatment of FAP underscores Tempest’s mission to make a meaningful difference in the lives of patients and builds on the momentum from prior designations received for amezalpat in hepatocellular carcinoma."
A Phase 2 study evaluating TPST-1495 in patients with FAP is set to begin this year, conducted by the Cancer Prevention Clinical Trials Network and funded by the National Cancer Institute (NCI) Division of Cancer Prevention. Data from this study are expected in 2026.
About Familial Adenomatous Polyposis
FAP is a high-risk inherited syndrome associated with the development of cancer in affected patients and has no approved medical therapies. In the US, the disease affects approximately one in 5,000 to 10,000 individuals2. FAP is caused by autosomal dominant inactivating mutations in the tumor suppressor gene APC. Patients with FAP develop large numbers of adenomatous polyps throughout the gastrointestinal tract, often starting in their teenage years. These growths have a high risk of malignant transformation and can give rise to invasive cancers of the colon, stomach, duodenum, rectum, and other tissues. Standard of care treatment for patients with FAP is surgical removal of the colon (colectomy) early in life to reduce the likelihood of cancer development. Even after colectomy, patients must receive careful surveillance for development of cancer elsewhere in the GI tract throughout their lifetime. While surgical management and surveillance have improved the prognosis for patients with FAP, cancer remains a major cause of death for patients with FAP.