Tempest Granted Fast Track Designation from the U.S. Food and Drug Administration for Amezalpat to Treat Patients with Hepatocellular Carcinoma
Tempest Therapeutics (NASDAQ: TPST) has received Fast Track Designation (FTD) from the FDA for amezalpat, their oral PPAR⍺ antagonist drug for treating hepatocellular carcinoma (HCC). This follows the Orphan Drug Designation (ODD) granted in January after positive Phase 1b/2 clinical trial results.
The global randomized study evaluated amezalpat combined with standard-of-care atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone in first-line treatment of unresectable or metastatic HCC. Key results showed a six-month improvement in median overall survival with a hazard ratio of 0.65 for patients receiving the combination therapy. The survival benefit was maintained in key sub-populations, including PD-L1 negative disease patients, supporting amezalpat's proposed mechanism targeting both tumor cells and the patient's immune system.
Tempest Therapeutics (NASDAQ: TPST) ha ottenuto la Designazione Fast Track (FTD) dalla FDA per amezalpat, il loro farmaco antagonista PPAR⍺ orale per il trattamento del carcinoma epatocellulare (HCC). Questo segue la Designazione di Farmaco Orfano (ODD) concessa a gennaio dopo risultati positivi degli studi clinici di Fase 1b/2.
Lo studio globalmente randomizzato ha valutato amezalpat combinato con il trattamento standard atezolizumab e bevacizumab rispetto a atezolizumab e bevacizumab da soli nel trattamento di prima linea dell'HCC non resecabile o metastatico. I risultati chiave hanno mostrato un miglioramento di sei mesi della sopravvivenza globale mediana con un rapporto di rischio di 0,65 per i pazienti che ricevevano la terapia combinata. Il beneficio di sopravvivenza è stato mantenuto nelle principali sottopopolazioni, inclusi i pazienti con malattia PD-L1 negativa, supportando il meccanismo proposto di amezalpat che mira sia alle cellule tumorali che al sistema immunitario del paziente.
Tempest Therapeutics (NASDAQ: TPST) ha recibido la Designación de Vía Rápida (FTD) de la FDA para amezalpat, su fármaco antagonista oral de PPAR⍺ para el tratamiento del carcinoma hepatocelular (HCC). Esto sigue a la Designación de Medicamento Huérfano (ODD) concedida en enero después de resultados positivos en ensayos clínicos de la Fase 1b/2.
El estudio global randomizado evaluó amezalpat combinado con el tratamiento estándar atezolizumab y bevacizumab frente a atezolizumab y bevacizumab solos en el tratamiento de primera línea de HCC irresecable o metastásico. Los resultados clave mostraron una mejora de seis meses en la supervivencia global media con una razón de riesgo de 0.65 para los pacientes que recibieron la terapia combinada. El beneficio de supervivencia se mantuvo en subpoblaciones clave, incluidos los pacientes con enfermedad negativa para PD-L1, apoyando el mecanismo propuesto de amezalpat que apunta tanto a las células tumorales como al sistema inmunológico del paciente.
템페스트 테라퓨틱스 (NASDAQ: TPST)는 간세포암 (HCC) 치료를 위한 경구 PPAR⍺ 길항제인 아메잘파트에 대해 FDA로부터 신속 승인(Fast Track Designation, FTD)을 받았습니다. 이는 1b/2상 임상 시험에서 긍정적인 결과를 바탕으로 1월에 부여된 희귀의약품 지정(Orphan Drug Designation, ODD) 이후의 일입니다.
전세계 무작위 연구에서는 아메잘파트와 표준 치료제인 아테졸리주맙 및 베바시주맙을 병용한 것과 아테졸리주맙 및 베바시주맙 단독 치료를 비교하여 수술이 불가능하거나 전이성 HCC의 1차 치료로 평가하였습니다. 주요 결과는 병합 요법을 받은 환자에서 중간 전체 생존율이 6개월 향상된 것으로 나타났으며, 위험비는 0.65였습니다. 생존 이점은 PD-L1 음성 질환 환자를 포함한 주요 하위 집단에서도 유지되어 아메잘파트의 제안된 기전이 종양 세포와 환자의 면역계를 모두 표적한다는 것을 지지하였습니다.
Tempest Therapeutics (NASDAQ: TPST) a reçu la désignation Fast Track (FTD) de la FDA pour amezalpat, leur médicament antagoniste PPAR⍺ par voie orale pour le traitement du carcinome hépatocellulaire (HCC). Cela fait suite à la désignation de médicament orphelin (ODD) accordée en janvier après les résultats positifs des essais cliniques de phase 1b/2.
La étude randomisée à l'échelle mondiale a évalué amezalpat en combinaison avec les traitements standards atezolizumab et bevacizumab par rapport à atezolizumab et bevacizumab seuls dans le traitement de première ligne du HCC irrésecable ou métastatique. Les résultats clés ont montré une amélioration de six mois de la survie globale médiane avec un ratio de risque de 0,65 pour les patients recevant la thérapie combinée. Le bénéfice de survie a été maintenu dans les sous-populations clés, y compris les patients atteints de la maladie négative pour PD-L1, soutenant le mécanisme proposé d'amezalpat visant à cibler à la fois les cellules tumorales et le système immunitaire du patient.
Tempest Therapeutics (NASDAQ: TPST) hat von der FDA die Fast Track Designation (FTD) für amezalpat, ihren oralen PPAR⍺-Antagonisten zur Behandlung von hepatozellulärem Karzinom (HCC), erhalten. Dies folgt der Orphan Drug Designation (ODD), die im Januar nach positiven Ergebnissen der Phase 1b/2-Studien gewährt wurde.
Die globale randomisierte Studie bewertete amezalpat in Kombination mit dem Standardbehandlungsansatz Atezolizumab und Bevacizumab im Vergleich zu Atezolizumab und Bevacizumab allein bei der Erstlinientherapie von nicht resezierbarem oder metastasiertem HCC. Die wichtigsten Ergebnisse zeigten eine Verbesserung der medianen Gesamtüberlebenszeit um sechs Monate mit einem Hazard Ratio von 0,65 für Patienten, die die Kombinationstherapie erhielten. Der Überlebensvorteil wurde in wichtigen Subpopulationen, einschließlich Patienten mit PD-L1-negativer Erkrankung, aufrechterhalten und unterstützt den vorgeschlagenen Mechanismus von amezalpat, der sowohl Tumorzellen als auch das Immunsystem des Patienten angreift.
- Received FDA Fast Track Designation for amezalpat
- Recently obtained Orphan Drug Designation
- Phase 1b/2 trial showed 6-month improvement in median overall survival (HR=0.65)
- Demonstrated efficacy in PD-L1 negative disease subgroup
- None.
Insights
The FDA's Fast Track Designation for amezalpat marks a important milestone in addressing the significant unmet need in hepatocellular carcinoma treatment. The designation, coupled with January's Orphan Drug status, provides multiple regulatory advantages including expedited review and more frequent FDA interactions, potentially accelerating the path to market.
The clinical data is particularly compelling: the hazard ratio of 0.65 indicates a 35% reduction in mortality risk, which is substantial in HCC where survival improvements are typically modest. Most notably, the efficacy in PD-L1 negative patients addresses a critical gap in current immunotherapy approaches, as these patients typically show response to existing treatments.
Amezalpat's unique mechanism as a PPAR⍺ antagonist represents a novel approach in HCC treatment. The dual-action strategy - targeting both tumor cells directly while enhancing immune response - differentiates it from current standard-of-care options. This could be particularly valuable when combined with atezolizumab and bevacizumab, potentially offering a new paradigm in first-line HCC treatment.
For a small-cap biotech like Tempest (
Fast Track Designation highlights potential of amezalpat to address unmet need for patients with HCC
Amezalpat builds momentum with both Fast Track and Orphan Drug Designations
BRISBANE, Calif., Feb. 10, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC).
“We are thrilled to receive Fast Track designation from the FDA,” said Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest. “This designation, following the Orphan Drug designation granted last month, reinforces the promise of amezalpat as a potential treatment option for patients affected by HCC. We look forward to working closely with the FDA and foreign regulatory agencies to develop amezalpat with the goal of bringing this promising therapy to patients.”
This is the second regulatory designation granted to amezalpat. The company announced in January that the U.S. FDA granted amezalpat with Orphan Drug Designation (ODD) following 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. In addition, a survival benefit from the addition of amezalpat was preserved in key sub-populations including PD-L1 negative 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.ii Every year, more than 900,000 people worldwide are diagnosed with HCC.iii Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US.iv In the US, HCC represents the fastest-rising cause of cancer-related death. iii
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.v
Even if diagnosed in the early stage, an estimated 70
About Amezalpat
Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist. Data suggests 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 in first-line HCC patients, amezalpat in combination with atezolizumab and bevacizumab showed clinical superiority across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to patients receiving 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 Fast Track Designation
Fast Track designation is intended to help rapidly advance the development and review processes for promising therapeutic candidates for serious conditions that may fill an unmet medical need. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process and may also be eligible for accelerated approval and priority review when relevant criteria are met.
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:
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Wheelhouse Life Science Advisors
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i If approved by the FDA
ii 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).
iii 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.
iv Llovet, J. M., Kelley, R. K., Villanueva, A., et al. Hepatocellular carcinoma. Nature Reviews Disease Primers. 2021, 7(1), 6.
v 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.
vi 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.
vii 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
What are the clinical trial results for TPST's amezalpat in HCC treatment?
What regulatory designations has TPST received for amezalpat in 2025?
How does TPST's amezalpat work in treating hepatocellular carcinoma?
What is the combination therapy being tested with TPST's amezalpat?
