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Elacestrant

Elacestrant, sold under the brand name Orserdu, is an oral (SERD) approved by the U.S. (FDA) for the treatment of (ER)-positive, human 2 (HER2)-negative, ESR1-mutated advanced or in postmenopausal women or adult men whose disease has progressed following at least one line of endocrine therapy. Originally discovered by Co., Ltd., elacestrant (also known as RAD1901) was licensed to Health, Inc. in June 2006 for development outside , with Radius acquiring full global rights by March 2015. In July 2020, Radius entered an exclusive global license agreement with the Group, under which Stemline Therapeutics (a Menarini subsidiary) handles commercialization in the United States. The drug received FDA and fast track designation prior to its approval on January 27, 2023, marking it as the first oral therapy specifically indicated for patients with ESR1 mutations in this setting. Elacestrant functions as a selective degrader and partial , binding preferentially to ERα (IC50 48 nmol/L) over ERβ (IC50 870 nmol/L), which inhibits ER-dependent transcription, reduces ER protein levels, and suppresses tumor in ER-positive models. It exhibits oral of approximately 10%, with a time to maximum concentration of 1–4 hours and an elimination half-life of 30–50 hours, primarily metabolized via the enzyme. The recommended dose is 345 mg taken orally once daily with food, and it is approved in conjunction with the Guardant360 CDx assay as a diagnostic to detect ESR1 mutations in tumor tissue. Approval was based on the phase 3 EMERALD (NCT03778931), a randomized study of 478 patients with ER-positive, HER2-negative advanced or who had prior endocrine therapy exposure. In the ESR1-mutated subgroup (n=228), elacestrant significantly prolonged median (PFS) to 3.8 months compared to 1.9 months with standard-of-care endocrine therapy ( [HR] 0.55; p=0.0005). Overall, across all patients, median PFS was 2.8 months with elacestrant versus 1.9 months with control ( 0.70). Common adverse reactions (≥10%) include musculoskeletal pain, , increased , fatigue, decreased , and . Ongoing research explores elacestrant's role in earlier-stage and combinations with other therapies, such as CDK4/6 inhibitors, to address endocrine resistance driven by ESR1 mutations, which occur in up to 40% of patients after prolonged exposure. Elacestrant was approved for medical use in the in September 2023.

Medical use

Indications

Elacestrant is indicated for the treatment of postmenopausal women or adult men with (ER)-positive, human 2 (HER2)-negative, ESR1-mutated advanced or with disease progression following at least one line of endocrine therapy. This approval is based on the results of the phase 3 EMERALD (NCT03778931), which demonstrated a significant improvement in (PFS) in the ESR1-mutated subgroup. In this randomized study of 228 patients with ESR1 mutations, elacestrant extended median PFS to 3.8 months compared to 1.9 months with standard-of-care endocrine monotherapy ( or an ), with a of 0.55 (95% CI: 0.39-0.77; p=0.0005). Current guidelines, such as those from the (NCCN), recommend elacestrant for this specific patient population after progression on prior endocrine , aligning with the FDA-approved indication. No off-label uses are routinely recommended in major guidelines at this time.

Administration and dosing

Elacestrant is administered orally as tablets, with the recommended dose being 345 mg taken once daily with food until disease progression or unacceptable toxicity. Patients should take the dose at approximately the same time each day and swallow the tablets whole, without chewing, crushing, or splitting them; if a dose is missed by more than 6 hours or if vomiting occurs after dosing, the dose should be skipped and resumed the following day. with food is advised to enhance . Patient selection for elacestrant requires confirmation of an ESR1 mutation using an FDA-approved companion diagnostic test, such as Guardant360 CDx, which detects ESR1 mutations in from . Dose modifications are recommended based on the severity of adverse reactions, graded according to the Common Terminology Criteria for Adverse Events (CTCAE). For Grade 1 toxicities, continue the current dose; for Grade 2, interrupt therapy until resolution to Grade 1 or better, then resume at the same dose. For Grade 3 toxicities, interrupt until resolution to Grade 1 or better, then reduce to 258 mg daily (three 86 mg tablets); if recurrence occurs, further reduce to 172 mg daily (two 86 mg tablets). For Grade 4 toxicities or recurrent intolerable Grade 3 events, interrupt until resolution to Grade 1 or better, reduce by one dose level, and permanently discontinue if further reduction is needed or if toxicity persists. Examples of adverse events warranting modification include severe , , or elevations in liver enzymes. No dose adjustment is required for patients with mild hepatic impairment (Child-Pugh Class A). For moderate hepatic impairment (Child-Pugh Class B), reduce the dose to 258 mg daily; elacestrant is not recommended for use in patients with severe hepatic impairment (Child-Pugh Class C). No dose adjustments are needed for patients with mild or moderate renal impairment, and data are limited for severe renal impairment.

Safety and tolerability

Adverse effects

In the phase III EMERALD , the most common adverse reactions (occurring in >10% of patients treated with elacestrant) included musculoskeletal pain (41%, grade 3/4: 7%), (35%, grade 3/4: 2.5%), increased (30%, grade 3/4: 1%), increased (29%), increased triglycerides (27%, grade 3/4: 2%), (26%, grade 3/4: 2%), decreased hemoglobin (26%, grade 3/4: 1%), (19%), increased (17%), decreased sodium (16%), increased (16%), decreased (15%), (13%), (12%), (12%), (11%), hot flush (11%), and dyspepsia (10%). These gastrointestinal toxicities, such as and , were more frequent with elacestrant compared to standard-of-care endocrine (nausea: 35% vs. 19%; vomiting: 19% vs. 8%). Serious adverse reactions occurred in 12% of patients in the EMERALD trial, with the most common (>1%) being musculoskeletal pain (1.7%) and nausea (1.3%); fatal adverse reactions occurred in 1.7% of patients, including cardiac arrest and septic shock. Grade 3/4 adverse events were reported in 27% of elacestrant-treated patients versus 21% in the standard-of-care arm, with notable examples including back pain (2.5%) and increased ALT (2.1%). Treatment discontinuations due to adverse events occurred in 6.3% of patients on elacestrant, compared to 4.4% on standard of care, with treatment-related discontinuations at 3.4% versus 0.9%. Common laboratory abnormalities (>10%) included increased cholesterol (30%), increased AST (29%), increased triglycerides (27%), decreased hemoglobin (26%), increased ALT (17%), decreased sodium (16%), and increased creatinine (16%). Elacestrant is not associated with clinically significant QT prolongation at the recommended dose. As of early 2025, post-marketing surveillance from the FDA Adverse Event Reporting System identified nausea (703 reports), fatigue (494), disease progression (332), vomiting (306), and diarrhea (319) as the most frequently reported adverse events, with serious events comprising a subset consistent with trial findings and 75 signals of disproportionate reporting, including new events like arthralgia and bone pain not emphasized in the label. Management of adverse effects involves dose modifications: interrupt for grade 3 events until recovery to grade ≤1 or baseline, then resume at a reduced dose (258 mg for first reduction, 172 mg for second); permanently discontinue for recurrent grade 4 or intolerable events. For , prophylactic or symptomatic antiemetics (e.g., ) are recommended, along with on small frequent meals. Lipid profiles and liver function should be monitored periodically, with statins considered for and dose interruption for significant elevations in liver enzymes. Musculoskeletal pain can be managed with acetaminophen or NSAIDs if no contraindications exist. Certain drug interactions may exacerbate gastrointestinal adverse effects like .

Contraindications and drug interactions

Elacestrant should not be used in patients with known to elacestrant or any of its excipients, as hypersensitivity reactions may occur. Elacestrant should be avoided in patients with severe hepatic impairment (Child-Pugh class C), as the drug has not been studied in this population and may lead to increased exposure and toxicity. Additionally, elacestrant can cause fetal harm when administered to a and should be avoided during ; females of reproductive potential should verify negative pregnancy status prior to initiation. should be avoided during treatment with elacestrant and for at least one week after the final dose, as elacestrant may be excreted in human milk and pose risks to nursing infants. Relative precautions are advised for patients with moderate hepatic impairment (Child-Pugh class B), where the recommended dose should be reduced to 258 mg once daily to mitigate risks of elevated plasma concentrations. Concomitant use with strong inhibitors, such as , requires caution or avoidance, as these can increase elacestrant exposure approximately twofold (AUC increased 2.6-fold), potentially heightening adverse effects; moderate inhibitors should similarly be avoided if possible. Key drug interactions involve modulators, where strong inducers like rifampin can decrease elacestrant exposure by about 50% ( reduced 0.5-fold), thereby potentially compromising therapeutic efficacy, and thus should be avoided. As elacestrant is both a substrate and inhibitor of (P-gp), coadministration with sensitive P-gp substrates (e.g., ) may result in altered substrate levels, necessitating dose adjustments and monitoring per the substrate's prescribing information. Hormonal contraceptives should be avoided in females of reproductive potential using elacestrant; non-hormonal methods are recommended due to potential interactions affecting contraceptive efficacy and the drug's modulation. Monitoring is essential for patients on elacestrant, including baseline and periodic lipid panel assessments to detect (incidence 30%) or (incidence 27%), which may require management. When inhibitors are unavoidable, ECG monitoring for prolongation is recommended, although no clinically significant QTc changes were observed at therapeutic doses. Patient counseling should emphasize taking elacestrant with to minimize interpatient variability in and reduce gastrointestinal symptoms like , as high-fat meals increase (AUC by 22%). Patients should be advised to avoid and other strong inhibitors to prevent excessive drug exposure.

Pharmacology

Pharmacodynamics

Elacestrant is a (SERD) that functions as a potent of (ERα), binding to the receptor and promoting its ubiquitination and subsequent proteasomal degradation, thereby inhibiting estrogen-dependent signaling in ER-positive cells. This mechanism disrupts ERα dimerization and nuclear translocation, leading to a dose-dependent reduction in ERα protein levels, with an of approximately 0.6 nM for ERα degradation in cells after 48 hours of exposure. Unlike partial agonists such as , elacestrant exhibits pure antagonistic activity without any estrogen-like agonism, even at high concentrations, ensuring complete blockade of ERα transcriptional activity. The binding affinity of elacestrant for ERα is high, with an of 48 nM in competitive binding assays, demonstrating 18-fold selectivity over ERβ ( of 870 nM), and it shows minimal interaction with other receptors at concentrations up to 5 μM. This selectivity profile limits off-target effects while effectively targeting ERα-driven pathways. Elacestrant maintains comparable potency against mutant forms of ERα, including common ESR1 mutations such as Y537S and D538G, which are associated with resistance to prior endocrine therapies like inhibitors. Downstream, elacestrant suppresses ERα-mediated gene transcription, significantly reducing the expression of key estrogen-responsive genes such as (PGR), GREB1, and TFF1 in ER-positive cell lines like and T47D, with near-complete inhibition observed at clinically relevant doses. This transcriptional blockade culminates in antiproliferative effects and induction of in ER-positive cells, particularly those harboring ESR1 mutations, by depleting functional ERα and halting progression. By degrading mutant ERα variants, elacestrant restores sensitivity to endocrine therapy in resistant models, addressing a major mechanism of acquired resistance in advanced disease.

Pharmacokinetics

Elacestrant exhibits low oral of approximately 10% in the fasted state, with a time to maximum concentration (Tmax) of 1 to 4 hours. Administration with a high-fat increases the maximum concentration (Cmax) by 42% and the area under the concentration-time curve () by 22%, prompting the recommendation to take the drug with to enhance and mitigate gastrointestinal adverse effects. The is highly bound to proteins (>99%), primarily to and alpha-1-acid glycoprotein, independent of concentration. Elacestrant has a large (approximately 5,800 L at ), indicating extensive distribution into tissues, including penetration into tumor tissue as demonstrated in preclinical models. Elacestrant undergoes hepatic metabolism primarily via (fraction metabolized approximately 87%), with minor contributions from and , forming metabolites such as the conjugate of 4-[2-(ethylamino)ethyl], which accounts for about 41% of radioactivity. The terminal elimination ranges from 30 to 50 hours, with steady-state concentrations achieved after approximately 8 days of daily dosing. Elimination occurs mainly through (82% of dose, with 34% as unchanged ) and to a lesser extent (7.5% of dose, <1% unchanged), with a mean apparent clearance of 186 L/hour. In special populations, show no clinically significant differences based on age (24 to 89 years), , or body weight (41 to 143 ). Mild to moderate does not require dose adjustment due to minimal renal excretion (renal clearance ≤0.14 L/hour). For hepatic , mild (Child-Pugh A) cases show no major changes, while moderate (Child-Pugh B) increases by approximately 83%, necessitating a dose reduction to 258 mg daily; severe (Child-Pugh C) is contraindicated due to predicted substantial exposure increases. Coadministration with moderate CYP3A4 inhibitors approximately doubles elacestrant exposure ( increase 2.3-fold), requiring dose reduction, whereas strong inhibitors increase exposure up to 5.3-fold, also necessitating adjustment.

History and development

Discovery and early development

Elacestrant, also known as RAD1901, was discovered by Co., Ltd. as a novel, orally bioavailable (SERD) designed to overcome resistance to endocrine therapies in receptor-positive (ER+) breast cancer, particularly in cases involving ESR1 mutations. Developed in the mid-2000s, the compound emerged from efforts to create nonsteroidal agents that could bind the (ER), induce its degradation, and inhibit ER-mediated transcription more effectively than existing treatments, addressing the unmet need for oral options in endocrine-resistant disease. In June 2006, granted Health, Inc. an exclusive worldwide license for elacestrant, excluding , enabling to advance its , , and commercialization. This agreement provided with rights to key patents and know-how related to the compound. In March 2015, secured the Japanese rights from through an amendment, obtaining full global control and paying an initial license fee of $0.4 million, which solidified its position to pursue worldwide . Preclinical studies demonstrated elacestrant's potent ER degradation in ER+ breast cancer cell lines, such as MCF-7, T47D, and HCC1428, reducing ER protein levels in a dose-dependent manner comparable to fulvestrant while downregulating ER target genes like PGR, TFF1, and GREB1. In animal models, including MCF-7 xenografts and patient-derived xenografts (PDXs), elacestrant achieved complete tumor growth inhibition at doses of 30-60 mg/kg and showed superior antitumor activity over fulvestrant in ESR1-mutant models, such as those harboring the Y537S mutation, where fulvestrant exhibited limited efficacy. These findings highlighted elacestrant's oral bioavailability—approximately 10% with a favorable pharmacokinetic profile—as a key advantage over injectable SERDs, supporting its potential in treating endocrine-resistant breast cancer. The Investigational New Drug (IND) application for elacestrant became effective on December 17, 2014, under IND 124748. Radius Health initiated phase 1 clinical trials for elacestrant in 2015 to evaluate its safety, tolerability, , and preliminary efficacy in patients with advanced +, HER2-negative . The first-in-human (NCT02338349) began enrolling in January 2015, focusing on dose escalation from 200 mg to 1000 mg orally once daily in postmenopausal women.

Clinical trials and regulatory approval

The development of elacestrant progressed through early-phase clinical trials that established its dosing and preliminary efficacy. In the phase 1 study (NCT02338349) conducted from 2015 onward, dose-escalation identified 400 mg once daily as the recommended phase 2 dose (RP2D), with the regimen demonstrating a clinical benefit rate of approximately 43% among heavily pretreated patients with estrogen receptor-positive, HER2-negative advanced . The approved dose of 345 mg once daily was selected for later trials based on pharmacokinetic data with food intake and tablet formulation to achieve comparable exposure. The pivotal phase 3 EMERALD (NCT03778931) was a randomized, open-label comparing elacestrant at 345 mg once daily to standard-of-care endocrine therapy ( or an ) in 478 postmenopausal women or men with receptor-positive, HER2-negative advanced or metastatic breast cancer who had progressed on prior endocrine therapy and CDK4/6 inhibitors. The met its primary endpoint of in the ESR1-mutated subgroup, with a of 0.55 (95% , 0.39-0.77; P=0.0005), indicating a significant improvement over standard therapy. Regulatory approval followed the EMERALD results, with the U.S. granting accelerated approval for elacestrant (Orserdu) on January 27, 2023, for postmenopausal women or adult men with receptor-positive, HER2-negative, ESR1-mutated advanced or after one or more lines of endocrine therapy. Concurrently, the FDA approved the Guardant360 CDx as a diagnostic to detect ESR1 mutations via liquid biopsy. The authorized elacestrant on September 15, 2023, for similar indications in postmenopausal women or men. approved the drug in 2023. As part of the accelerated approval, post-approval commitments include confirmatory trials to verify clinical benefit, such as the ongoing ELEVATE study evaluating elacestrant in combination with other therapies. In July 2020, global development and commercialization rights (excluding ) were licensed to the Menarini Group (via Stemline Therapeutics) from Radius Health for an upfront payment of $75 million plus potential milestones up to $525 million. In June 2023, transferred all future economic rights for elacestrant in to the Menarini Group. In , regulatory review remains ongoing as of November 2025, with no approval granted.

Research

Completed pivotal trials

The EMERALD trial (NCT03778931) was a multicenter, open-label, phase 3 study that randomized 477 patients with estrogen receptor-positive, HER2-negative advanced or metastatic breast cancer—who had received one or two prior lines of endocrine therapy, including a CDK4/6 inhibitor—1:1 to elacestrant (345 mg orally once daily with food) or standard-of-care endocrine monotherapy (fulvestrant or an aromatase inhibitor). Stratification occurred by ESR1 mutation status (detected via central next-generation sequencing of blood), prior fulvestrant exposure, and presence of visceral metastases. The primary endpoint was progression-free survival (PFS) assessed by blinded independent central review, with a key analysis in the ESR1-mutated subgroup (228 patients, 48% of total). Median PFS was 2.8 months with elacestrant versus 1.9 months with standard of care in the intent-to-treat population (hazard ratio [HR] 0.70, 95% CI 0.55-0.88, P=0.002), and 3.8 months versus 1.9 months in the ESR1-mutated subgroup (HR 0.55, 95% CI 0.39-0.77, P=0.0005). Objective response rate was 12.4% with elacestrant versus 7.3% with standard of care in the ESR1-mutated subgroup. A post-hoc subgroup analysis of the EMERALD trial identified enhanced benefit in patients with ESR1 mutations and at least 12 months of prior endocrine therapy plus CDK4/6 inhibition in the metastatic setting, where median PFS reached 8.6 months with elacestrant versus 1.9 months with (HR 0.41, 95% 0.25-0.65). Secondary endpoint overall survival remained immature as of 2024 analyses, with an HR of approximately 0.94 (95% not reached statistical significance) overall and 0.90 (95% 0.63-1.30) in the ESR1-mutated , showing no definitive benefit. Supporting phase 2 dose-optimization efforts, including study RAD1901-005, evaluated escalating doses in approximately 200 patients across early development phases to establish the recommended phase 2 dose of 345 mg daily, confirming tolerability with primarily grade 1-2 gastrointestinal events. These studies also identified a positive food effect, with a high-fat meal increasing elacestrant exposure (area under the curve by 22%, maximum concentration by 42%), which informed administration recommendations to enhance and reduce incidence. Subgroup analyses from EMERALD demonstrated consistent PFS benefits across patients regardless of prior CDK4/6 inhibitor exposure duration, with numerically greater improvements in those with visceral disease ( 0.61 in ESR1-mutated patients with visceral metastases). Limitations of these trials include immature overall survival data with no definitive benefit, higher rates of gastrointestinal toxicity with elacestrant ( in 35% versus 19% with ; grade 3/4 in approximately 2%), and reduced efficacy signals in ESR1 wild-type patients. Primary results from EMERALD were published in the Journal of Clinical Oncology in 2022, with initial presentation at the annual meeting that year; subsequent subgroup analyses and updates, including post-hoc data, appeared at ASCO meetings in 2023, 2024, and 2025.

Ongoing and future studies

As of November 2025, the ELCIN trial (NCT05596409), a phase 2 study, is evaluating the efficacy and safety of elacestrant versus standard in patients with estrogen receptor-positive (ER+), human 2-negative (HER2-) who have received prior but no prior CDK4/6 inhibitors in the metastatic setting. The primary endpoint is (PFS), with the trial recruiting a planned 60 patients across multiple sites; recruitment is ongoing. Additional ongoing phase 2 trials, including the ELEVATE study (NCT05563220), are investigating elacestrant in combination with PI3K inhibitors like for PIK3CA-mutated cancers, demonstrating feasibility, tolerability, and clinical activity in heavily pretreated patients with ESR1 and PIK3CA co-mutations. Building on results from the phase 3 EMERALD , updated analyses presented at ASCO 2025 from ELEVATE highlight elacestrant's potential as a backbone endocrine therapy, particularly in combinations that improve outcomes, such as with or in ER+/HER2- patients progressing after endocrine therapy and CDK4/6 inhibition (preliminary objective response rates up to 40%). In early-stage settings, the EORTC-2129-BCG trial (NCT05512364), initiated in 2023, is a phase 3 study assessing elacestrant versus standard endocrine therapy in high-risk, node-positive ER+/HER2- early , with primary of distant metastasis-free and screening over 1,900 patients across European sites. Similarly, the global phase 3 ELEGANT trial (NCT06492616) is recruiting approximately 4,220 patients to compare elacestrant with standard endocrine therapy ( inhibitors or ) in high-risk early with elevated recurrence risk. Future research directions include expanding elacestrant to premenopausal women through ovarian suppression protocols, as preclinical and early data suggest comparable activity to postmenopausal settings. Enhanced ESR1 mutation diagnostics via liquid biopsy are being integrated into trials to refine patient selection, while studies on biomarkers aim to identify predictors of response beyond ESR1 status. Challenges in ongoing development encompass managing liver elevations (/ increases) observed in long-term use, which occur in up to 10% of patients and require monitoring. Head-to-head comparisons with other selective degraders (SERDs), such as camizestrant, are anticipated in future trials to assess differential efficacy and toxicity profiles in ESR1-mutated populations.