Tempest Receives Orphan Drug Designation from the U.S. Food and Drug Administration for Amezalpat to Treat Patients with Hepatocellular Carcinoma (HCC)
MWN-AI** Summary
Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotech firm focused on innovative cancer treatments, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational drug, amezalpat (TPST-1120), for the treatment of hepatocellular carcinoma (HCC). This designation highlights the urgent need for new therapeutic options for a cancer known for its challenges in treatment.
Amezalpat is an oral small-molecule selective PPAR? antagonist designed to target cancer cells directly and enhance the immune response. Tempest's clinical data from a global randomized Phase 1b/2 study indicate promising outcomes, showing a significant improvement in median overall survival for patients receiving amezalpat in combination with standard-of-care treatments compared to those on standard therapy alone. Patients treated with the combination therapy experienced a six-month improvement in overall survival, alongside a notable objective response rate of 30%.
HCC is an aggressive cancer, with increasing incidence and mortality rates, making it a leading cause of cancer-related death. Frequent causes include chronic liver diseases such as hepatitis B and C, non-alcoholic fatty liver disease, and alcohol-related liver disease. Despite advances in diagnosis and management, early-stage HCC often recurs, leading to poorer prognoses.
With the ODD from the FDA, Tempest Therapeutics is poised to leverage benefits such as tax credits for clinical testing and market exclusivity upon approval, helping to advance the development of amezalpat. The company is now actively preparing for a pivotal Phase 3 study aimed at evaluating the drug's efficacy further in first-line HCC patients, reflecting its commitment to addressing the unmet needs within this patient population.
MWN-AI** Analysis
Tempest Therapeutics, Inc. (Nasdaq: TPST) has made a significant stride by receiving Orphan Drug Designation from the FDA for its drug amezalpat (TPST-1120), aimed at treating hepatocellular carcinoma (HCC). This designation highlights the dire need for new treatment options in a disease that is notoriously difficult to manage and is becoming an increasingly pressing public health issue, particularly in the U.S. where HCC is the fastest-rising cause of cancer-related deaths.
Investors should view this news positively, as orphan drug status not only offers potential market exclusivity for seven years if approved but also comes with critical benefits such as tax credits for clinical testing and fee waivers. Given that HCC affects a substantial patient population, with growing incidence rates globally, amezalpat's market potential appears promising.
In recent clinical trials, the combination of amezalpat with standard treatments showed improved overall survival and a higher objective response rate compared to current standards of care, which positions Tempest favorably in a competitive landscape. Key data showing a six-month median overall survival improvement signifies significant potential utility in treating HCC.
However, while the prospects are buoyant, prospective investors should remain cautious. The biotechnology sector is inherently volatile, with substantial reliance on successful clinical trial outcomes, regulatory approvals, and market acceptance. As Tempest prepares for pivotal Phase 3 trials, it will be critical to monitor enrollment and data readouts closely.
In summary, investors may consider a cautious accumulation strategy in TPST shares, particularly as clinical data emerge and regulatory milestones are approached. However, due diligence is advised given the risks inherent in drug development and the unpredictability of FDA evaluations.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
BRISBANE, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class 1 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. 2 Every year, more than 900,000 people worldwide are diagnosed with HCC. 3 Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US. 4 In the US, HCC represents the fastest-rising cause of cancer-related death. 3
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. 5
Even if diagnosed in the early stage, an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery. 6 Early recurrence is associated with poorer prognosis and shorter survival. 5, 7 Tumor size, number of tumors, and portal vein invasion are associated with an increased risk of recurrence. 6
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
1 If approved by the FDA
2 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).
3 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.
4 Llovet, J. M., Kelley, R. K., Villanueva, A., et al. Hepatocellular carcinoma. Nature Reviews Disease Primers. 2021, 7(1), 6.
5 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.
6 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.
7 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.
FAQ**
How does the FDA's Orphan Drug Designation for Tempest Therapeutics Inc. TPST's amezalpat enhance its potential market position in the treatment of hepatocellular carcinoma?
What key safety and efficacy data from the Phase 1b/2 study of Tempest Therapeutics Inc. TPST's amezalpat could influence investor confidence ahead of the pivotal Phase 3 trial?
How might the clinical superiority of amezalpat over standard-of-care therapies impact Tempest Therapeutics Inc. TPST's valuation and future funding strategies?
Considering the rising incidence of hepatocellular carcinoma, what strategic advantages does Tempest Therapeutics Inc. TPST have in developing therapies like amezalpat in a competitive biotech landscape?
**MWN-AI FAQ is based on asking OpenAI questions about Tempest Therapeutics Inc. (NASDAQ: TPST).
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