Tempest Therapeutics announces FDA Orphan Drug Designation for amezalpat in treating hepatocellular carcinoma, highlighting significant clinical study results.
Quiver AI Summary
Tempest Therapeutics, Inc. announced that the FDA has granted Orphan Drug Designation to amezalpat (TPST-1120), a selective PPAR⍺ antagonist, for treating hepatocellular carcinoma (HCC), which has a high mortality rate and limited treatment options. The designation highlights the urgent need for new therapies for HCC, as the company prepares for a pivotal Phase 3 study. Recent clinical trials showed that amezalpat, when combined with standard treatments, significantly improved median overall survival and objective response rates in patients with advanced HCC. This drug targets both tumor cells and the immune environment, reflecting its potential in addressing this aggressive cancer. The Orphan Drug Designation provides Tempest with various benefits to facilitate further development of amezalpat.
Potential Positives
- The FDA has granted Orphan Drug Designation to amezalpat for the treatment of hepatocellular carcinoma, highlighting its potential to address an unmet medical need.
- Amezalpat demonstrated clinical superiority in a global randomized Phase 1b/2 study, showing significant improvement in overall survival compared to the current standard of care.
- The positive clinical results were preserved in key patient sub-populations, indicating broad applicability and potential efficacy of amezalpat across diverse patient profiles.
- The Orphan Drug Designation offers Tempest Therapeutics potential benefits, including tax credits for clinical testing and seven years of market exclusivity upon approval, which could enhance competitive positioning in the market.
Potential Negatives
- The press release emphasizes the need for new treatment options for HCC, highlighting the significant challenges faced in treating this aggressive disease, which may imply limitations in current standard care options.
- The mention of unexpected safety or efficacy data during clinical trials as a potential risk factor raises concerns about the reliability of the current positive results.
- Forward-looking statements caution investors against relying on anticipated outcomes, indicating uncertainty about future performance and regulatory approvals.
FAQ
What is Orphan Drug Designation and its significance?
The Orphan Drug Designation provides benefits like tax credits and market exclusivity for treatments of rare diseases affecting fewer than 200,000 people.
What is amezalpat and its intended use?
Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist aimed at treating patients with hepatocellular carcinoma (HCC).
How does amezalpat improve HCC treatment outcomes?
Clinical trials showed amezalpat combined with standard therapies improved overall survival and response rates in advanced HCC patients.
What are the benefits of the FDA's approval for Tempest Therapeutics?
The FDA's approval allows Tempest to advance amezalpat into pivotal studies, reflecting its commitment to innovative cancer treatments.
What challenges does hepatocellular carcinoma present to patients?
HCC is aggressive, with high recurrence rates even after surgery, contributing to rising mortality rates, particularly in specific regions.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$TPST Insider Trading Activity
$TPST insiders have traded $TPST stock on the open market 2 times in the past 6 months. Of those trades, 0 have been purchases and 2 have been sales.
Here’s a breakdown of recent trading of $TPST stock by insiders over the last 6 months:
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$TPST Hedge Fund Activity
We have seen 13 institutional investors add shares of $TPST stock to their portfolio, and 8 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- ALTIUM CAPITAL MANAGEMENT LP added 545,000 shares (+inf%) to their portfolio in Q3 2024
- CITADEL ADVISORS LLC removed 92,232 shares (-100.0%) from their portfolio in Q3 2024
- GEODE CAPITAL MANAGEMENT, LLC added 65,826 shares (+42.7%) to their portfolio in Q3 2024
- VIRTU FINANCIAL LLC removed 62,027 shares (-100.0%) from their portfolio in Q3 2024
- FULLCIRCLE WEALTH LLC added 62,000 shares (+inf%) to their portfolio in Q3 2024
- SUSQUEHANNA INTERNATIONAL GROUP, LLP added 45,259 shares (+inf%) to their portfolio in Q3 2024
- MORGAN STANLEY added 37,415 shares (+59.6%) to their portfolio in Q3 2024
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Full Release
BRISBANE, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class
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targeted and immune-mediated therapeutics to fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC).
“Receiving orphan drug designation for amezalpat to treat HCC underscores the critical need for new treatment options for patients suffering from this historically hard to treat disease,” said Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest. “Tempest is dedicated to developing groundbreaking cancer treatments that will improve patients’ lives, and with broad agreement in hand from both the FDA and EMA, the team continues to prepare for a pivotal phase 3 study for amezalpat in first-line HCC patients.”
This important regulatory designation follows positive data across multiple key study efficacy and safety endpoints in a global randomized Phase 1b/2 clinical study evaluating amezalpat plus standard-of-care atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone in the first-line treatment of patients with unresectable or metastatic HCC. Notable positive outcomes of the randomized comparison include a six-month improvement in median overall survival (OS) with a hazard ratio (HR) of 0.65 for patients receiving the amezalpat combination therapy and an objective response rate (ORR) of 30% vs 13% favoring the amezalpat arm. In addition, survival benefit from the addition of amezalpat was preserved in key sub-populations including PD-L1 negative disease and b-catenin mutated disease, which is consistent with amezalpat’s proposed mechanism of action to target both the tumor cells directly and the patient’s immune system.
About Hepatocellular Carcinoma
HCC is an aggressive cancer with rising mortality and is projected to become the third leading cause of cancer death by 2030.
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Every year, more than 900,000 people worldwide are diagnosed with HCC.
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Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US.
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In the US, HCC represents the fastest-rising cause of cancer-related death.
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Nine out of ten cases of HCC are caused by chronic liver disease, which includes chronic hepatitis B and C infection, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), alcohol-related liver disease (ALD) and cirrhosis resulting from these conditions.
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Even if diagnosed in the early stage, an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery.
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Early recurrence is associated with poorer prognosis and shorter survival.
5,
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Tumor size, number of tumors, and portal vein invasion are associated with an increased risk of recurrence.
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About Amezalpat
Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist. Data suggest that amezalpat treats cancer by targeting tumor cells directly and by modulating immune suppressive cells and angiogenesis in the tumor microenvironment. In a global randomized phase 1b/2 study of amezalpat in combination with atezolizumab and bevacizumab in first-line patients with advanced HCC, the amezalpat arm showed clinical superiority across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to atezolizumab and bevacizumab alone, the standard of care. These randomized data were supported by additional positive results observed in the Phase 1 clinical trial in patients with heavily pretreated advanced solid tumors, including renal cell carcinoma and cholangiocarcinoma.
About Orphan Drug Designation
The FDA's Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
About Tempest Therapeutics
Tempest Therapeutics is a clinical-stage biotechnology company advancing a diverse portfolio of small molecule product candidates containing tumor-targeted and/or immune-mediated mechanisms with the potential to treat a wide range of tumors. The company’s novel programs range from early research to later-stage investigation in a randomized global study in first-line cancer patients. Tempest is headquartered in Brisbane, California. More information about Tempest can be found on the company’s website at
www.tempesttx.com
.
Forward-Looking Statements
This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (the “Securities Act”)) concerning Tempest Therapeutics, Inc. These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Tempest Therapeutics, as well as assumptions made by, and information currently available to, management of Tempest Therapeutics. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “could”, “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions. All statements that are not historical facts are forward-looking statements, including any statements regarding: the design, initiation, progress, timing, scope and results of clinical trials, including the anticipated Phase 3 study for amezalpat; anticipated therapeutic benefit and regulatory development of the Company’s product candidates the Company’s ability to advance into a late-stage clinical company; and the Company’s ability to achieve its operational plans. Forward-looking statements are based on information available to Tempest Therapeutics as of the date hereof and are not guarantees of future performance. Any factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied are discussed in greater detail in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q filed for the quarter ended September 30, 2024 and other documents filed by the Company from time to time with the Securities and Exchange Commission. Except as required by applicable law, Tempest Therapeutics undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. These forward-looking statements should not be relied upon as representing Tempest Therapeutics’ views as of any date subsequent to the date of this press release and should not be relied upon as prediction of future events. In light of the foregoing, investors are urged not to rely on any forward-looking statement in reaching any conclusion or making any investment decision about any securities of Tempest Therapeutics.
Investor & Media Contacts:
Sylvia Wheeler
Wheelhouse Life Science Advisors
swheeler@wheelhouselsa.com
Aljanae Reynolds
Wheelhouse Life Science Advisors
areynolds@wheelhouselsa.com
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If approved by the FDA
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Rahib, L. et al. Projecting cancer incidence and deaths to 2030: the unexpected burden of thyroid, liver, and pancreas cancers in the United States.
Cancer Res.
74, 2913-2921 (2014).
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World Health Organization. Liver Cancer Factsheet. Globocan. 2020. Available at: https://gco.iarc.fr/today/data/factsheets/cancers/11-Liver-fact-sheet.pdf. Last accessed: April 2023.
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Llovet, J. M., Kelley, R. K., Villanueva, A., et al. Hepatocellular carcinoma. Nature Reviews Disease Primers. 2021, 7(1), 6.
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Office for Health Improvement & Disparities. Liver disease profiles: November 2021 update. Available at: https://www.gov.uk/government/statistics/liver-disease-profiles-november-2021-update/liver-disease-profiles-november-2021-update. Last accessed: April 2023.
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Hack SP, Spahn J, Chen M et al. IMbrave 050: a Phase III trial of atezolizumab plus bevacizumab in high-risk hepatocellular carcinoma after curative resection or ablation. Future Oncology. 2020 May;16(15):975-989.
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Saito A, Toyoda H, Kobayashi M et al. Prediction of early recurrence of hepatocellular carcinoma after resection using digital pathology images assessed by machine learning. Modern Pathology. 2021. 34, 417-425.
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