Palazestrant, a novel oral Complete Estrogen Receptor Antagonist (CERAN) and Selective Estrogen Receptor Degrader (SERD), in patients with ER+/HER2− advanced or metastatic breast cancer: phase 1/2 study results

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Publicat a:Breast Cancer Research vol. 27 (2025), p. 1-16
Autor principal: Hamilton, Erika P
Altres autors: Patel, Manish R, Borges, Virginia F, Meisel, Jane L, Okera, Meena, Alemany, Carlos A, Pluard, Timothy J, Wesolowski, Robert, Sabanathan, Dhanusha, Miller, Kathy D, Conlin, Alison K, McCarthy, Nicole, Shaw, Morena, Tonda, Margaret, Shilkrut, Mark, Lin, Nancy U
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Springer Nature B.V.
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022 |a 1465-5411 
022 |a 1465-542X 
024 7 |a 10.1186/s13058-025-02049-y  |2 doi 
035 |a 3227648849 
045 2 |b d20250101  |b d20251231 
084 |a 242930  |2 nlm 
100 1 |a Hamilton, Erika P 
245 1 |a Palazestrant, a novel oral Complete Estrogen Receptor Antagonist (CERAN) and Selective Estrogen Receptor Degrader (SERD), in patients with ER+/HER2− advanced or metastatic breast cancer: phase 1/2 study results 
260 |b Springer Nature B.V.  |c 2025 
513 |a Journal Article 
520 3 |a BackgroundEndocrine resistance is a major challenge in treating patients with ER+ /HER2− metastatic breast cancer (MBC) necessitating a switch from endocrine therapy to more toxic therapies. Mutations in ESR1 constitute a key mechanism of resistance to endocrine therapy in ER+ /HER2− BC. Therapies that overcome endocrine resistance are needed. Palazestrant is a novel oral complete estrogen receptor (ER) antagonist (CERAN) and selective ER degrader (SERD) belonging to a new class of ER-targeting agents that completely blocks estrogen-induced transcriptional activity, regardless of ESR1 mutation status. This first-in-human, open-label, multicenter, phase 1/2 dose-escalation/expansion study was designed to determine the recommended phase 2 dose (RP2D) and to evaluate safety, pharmacokinetics, and antitumor activity of palazestrant in patients with ER+ /HER2− MBC with disease progression on prior treatment.MethodsAdults with ER+ /HER2‒ MBC who received ≥ 1 prior line of endocrine therapy for advanced disease and ≤ 2 prior chemotherapy regimens for metastatic disease were eligible. Patients received once-daily oral palazestrant (30–300 mg) in 28-day cycles until progression or intolerable toxicity.ResultsThis study enrolled 146 patients. No dose-limiting toxicities were observed at doses up to 300 mg/day palazestrant. Confirmed partial responses were observed with 60 and 120 mg/day palazestrant. Both doses showed similar and tolerable safety profiles, favorable pharmacokinetics, and steady-state plasma concentrations above the predicted threshold for complete ER inhibition. Greater clinical benefit at palazestrant 120 mg/day (46%) versus 60 mg/day (19%) led to selection of 120 mg/day as RP2D and study expansion dose. At 120 mg/day, the median progression-free survival was 4.8 months (95% CI, 3.5–7.1) overall and 5.6 months (95% CI, 4.8–NE) among patients with cancers with ESR1 mutations. Most treatment-emergent adverse events (TEAEs) were grade 1–2. The most common TEAEs were nausea (62.8%), vomiting (29.1%), and fatigue (25.6%). The most common grade ≥ 3 TEAE was transient neutropenia (10.5%) managed by dose interruption and reduction.ConclusionsPalazestrant demonstrated a manageable safety profile, with antitumor activity observed in patients with heavily pretreated cancers with wild-type and ESR1-mutated BC. These data support the ongoing phase 3 study evaluating palazestrant in patients with ER+ /HER2 − MBC.Trial registrationClinicalTrials.gov, NCT04505826. Registered August 6, 2020. 
651 4 |a United States--US 
653 |a Plasma 
653 |a Womens health 
653 |a Metastasis 
653 |a Toxicity 
653 |a Cancer therapies 
653 |a Mutation 
653 |a Antitumor activity 
653 |a Laboratories 
653 |a Breast cancer 
653 |a Metastases 
653 |a Estrogens 
653 |a Kinases 
653 |a Estrogen receptors 
653 |a ErbB-2 protein 
653 |a Endocrine therapy 
653 |a Patients 
653 |a Neutropenia 
653 |a Pharmacokinetics 
653 |a Cyclin-dependent kinases 
653 |a Biomarkers 
653 |a Chemotherapy 
653 |a Tumors 
653 |a Cancer 
700 1 |a Patel, Manish R 
700 1 |a Borges, Virginia F 
700 1 |a Meisel, Jane L 
700 1 |a Okera, Meena 
700 1 |a Alemany, Carlos A 
700 1 |a Pluard, Timothy J 
700 1 |a Wesolowski, Robert 
700 1 |a Sabanathan, Dhanusha 
700 1 |a Miller, Kathy D 
700 1 |a Conlin, Alison K 
700 1 |a McCarthy, Nicole 
700 1 |a Shaw, Morena 
700 1 |a Tonda, Margaret 
700 1 |a Shilkrut, Mark 
700 1 |a Lin, Nancy U 
773 0 |t Breast Cancer Research  |g vol. 27 (2025), p. 1-16 
786 0 |d ProQuest  |t Health & Medical Collection 
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