Background: Previous reports of ELEVATE-TN (NCT02475681) at median follow-up of up to 58.2 months (mo) demonstrated superior efficacy of acalabrutinib (A) ± obinutuzumab (O) compared with O + chlorambucil (Clb) in patients (pts) with treatment-naive (TN) chronic lymphocytic leukemia (CLL). Herein, updated results at 74.5 mo of follow-up are reported.

Methods: Pts received A monotherapy, A+O, or O+Clb. Pts who progressed on O+Clb could cross over to A monotherapy. Investigator (INV)-assessed progression-free survival (PFS), INV-assessed overall response rate (ORR), overall survival (OS), and safety were evaluated. All analyses are ad-hoc and P-values are descriptive.

Results: A total of 535 pts (A, n=179; A+O, n=179; O+Clb, n=177) were randomized: median age was 70 years, 63% had unmutated immunoglobulin heavy chain variable region genes (uIGHV), and 14% had del(17p) and/or TP53 mutation ( TP53m). At a median follow-up of 74.5 mo (range, 0.0-89.0; data cutoff March 3, 2023), median PFS was not reached (NR) for A+O and A vs 27.8 mo for O+Clb (hazard ratio [HR] vs O+Clb: 0.14 and 0.23, respectively; P<0.0001 for both; HR A+O vs A: 0.58; P=0.0229); estimated 72-mo PFS rates were 78%, 62%, and 17%, respectively (Figure 1). For the 79 pts who crossed over from O+Clb to A, median PFS2 (time to second disease progression or death) was NR; estimated 72-mo PFS2 rate was 54%. Median OS was NR in any treatment arm and was significantly longer with A+O vs O+Clb (HR: 0.62; P=0.0349); estimated 72-mo OS rates were 84% for A+O, 76% for A, and 75% for O+Clb ( Figure 2). In 337 pts with uIGHV, median PFS was NR in both the A+O and A arms vs 22.2 mo for O+Clb (HR: 0.08 and 0.12, respectively; P<0.0001 for both), and estimated 72-mo PFS rates were 75%, 60%, and 5%, respectively, with median OS NR in all treatment arms and estimated 72-mo OS rates of 84%, 76%, and 74%, respectively. In 73 pts with del(17p) and/or TP53m, median PFS was 73.1 mo for A+O and NR for A vs 17.5 mo for O+Clb (HR: 0.28 and 0.23, respectively; P≤0.0009 for both), and estimated 72-mo PFS rates were 56%, 56%, and 18%, respectively. Median OS was NR in both the A+O and A arms vs 74.9 mo for O+Clb (HR: 0.53 and 0.46, respectively, neither statistically significant) and estimated 72-mo OS rates were 68%, 72%, and 53%, respectively. ORR was significantly higher with A+O (96%; 95% confidence interval [CI]: 92-98; P<0.0001) and A (90%; 95% CI: 85-94; P=0.0499) vs O+Clb (83%; 95% CI: 77-88). Combined complete response (CR) plus CR with incomplete hematologic recovery rates were higher with A+O (37%) and A (19%) vs O+Clb (14%; P≤0.0499 for both).

Median treatment exposure was 74.4 mo and 72.0 mo for A in the A+O and A arms, respectively; 5.5 mo and 5.6 mo for O in the A+O and O+Clb arms, respectively; and 5.5 mo for Clb in O+Clb arm. Adverse events (AEs) for the O+Clb arm have been previously reported. The most common (≥5% of pts) grade ≥3 AEs for A+O and A, respectively, were neutropenia (31% and 12%), thrombocytopenia (8% and 3%), diarrhea (6% and 1%), COVID-19 (9% and 7%), pneumonia (7% and 6%), syncope (5% and 2%), and hypertension (4% and 5%). For events of clinical interest, grade ≥3 atrial fibrillation, hypertension, and secondary primary malignances were reported in 2%, 4%, and 10% of pts treated with A+O and 2%, 5%, and 5% of pts treated with A, respectively. Treatment is ongoing in 54% (A+O; n=96) and 47% (A; n=84) of pts; the most common reasons for treatment discontinuation were AEs, observed in 21% (n=38) of pts treated with A+O and 18% (n=32) of pts treated with A, and progressive disease, observed in 6% (n=10) of pts treated with A+O and 14% (n=25) of pts treated with A. For pts who crossed over from O+Clb to A, 41% (n=32) discontinued A due to AEs in 13% (n=10) of pts and progressive disease in 16% (n=13) of pts.

Conclusions: With a median follow-up of 74.5 mo, the efficacy and safety of A+O and A monotherapy were maintained in pts with TN CLL, including in pts with high-risk genetic features. At 6 years of follow-up, PFS was significantly longer in pts treated with A+O vs A. Median OS was NR in any treatment arm and was significantly longer in pts treated with A+O vs O+Clb.

Sharman:AbbVie, AstraZeneca, BeiGene, BMS, Genentech, Inc., Lilly: Consultancy; Merck, Novartis: Consultancy; AbbVie, AstraZeneca, BMS, Beigene, Lilly, Genentech, Inc., Genmab: Consultancy; Seattle Genetics: Research Funding. Jurczak:AbbVie: Consultancy; AstraZeneca: Consultancy; BeiGene: Consultancy; Eli Lilly: Consultancy; Pfizer: Consultancy; Roche: Consultancy; SOBI: Consultancy; Takeda: Consultancy; AbbVie: Research Funding; AstraZeneca: Research Funding; Bayer: Research Funding; BeiGene: Research Funding; Celgene: Research Funding; Janssen: Research Funding; Eli Lilly: Research Funding; Merck: Research Funding; Pfizer: Research Funding; Roche: Research Funding; SOBI: Research Funding; Takeda: Research Funding. Skarbnik:MorphoSys: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; SeaGen: Consultancy, Honoraria, Speakers Bureau; Jazz Pharmaceuticals: Honoraria, Speakers Bureau; TG Therapeutics: Consultancy, Honoraria, Speakers Bureau; AstraZeneca: Consultancy, Honoraria, Speakers Bureau; Lilly: Consultancy, Honoraria, Speakers Bureau; Kite Pharma: Consultancy, Honoraria, Speakers Bureau; Epizyme: Consultancy, Honoraria; Genmab: Consultancy, Honoraria, Speakers Bureau; Pharmacyclics: Consultancy, Honoraria, Speakers Bureau; Janssen: Consultancy, Honoraria, Speakers Bureau; Genentech, Inc.: Consultancy, Honoraria, Speakers Bureau; Alexion: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria, Speakers Bureau; Bristol-Myers-Squibb: Consultancy, Honoraria; Beigene: Honoraria, Speakers Bureau; ADC therapeutics: Honoraria, Speakers Bureau. Patel:Xencor: Consultancy, Research Funding; Epizyme: Consultancy, Research Funding; Merck: Consultancy, Research Funding; Nurix: Research Funding; Morphosys: Consultancy; BeiGene: Consultancy; Kite: Consultancy, Research Funding, Speakers Bureau; ADC Therapeutics: Consultancy; Trillium Therapeutics/Pfizer: Consultancy, Research Funding; TG Therapeutics: Consultancy, Speakers Bureau; Bristol Myers Squibb: Consultancy, Research Funding, Speakers Bureau; Caribou Biosciences: Consultancy; Sunesis Pharmaceuticals: Research Funding; AstraZeneca: Consultancy, Research Funding, Speakers Bureau; Fate Therapeutics: Research Funding; Curis, Inc: Research Funding; Loxo Oncology: Consultancy, Research Funding; MEI Pharma: Consultancy, Research Funding; Genentech/Roche: Consultancy, Research Funding; Pharmacyclics/Janssen: Consultancy, Research Funding; CRISPR Therapeutics: Research Funding; Adaptive Biotechnologies: Research Funding; Abbvie: Consultancy. Flinn:Century Therapeutics: Consultancy; Genentech: Consultancy; Genmab: Consultancy; Kite: Consultancy; Hutchinson MediPharma: Consultancy; Secura Bio: Consultancy; Servier Pharma: Consultancy; BeiGene: Consultancy; AbbVie: Consultancy; Vincerx Pharma: Consultancy; Myeloid Therapeutics: Consultancy; Novartis: Consultancy; Innocare Pharma: Consultancy; TG Therapeutics: Consultancy. Kamdar:Seagen: Speakers Bureau; Genentech, Inc., Celgene for IDMC: Membership on an entity's Board of Directors or advisory committees; Novartis: Research Funding; AbbVie, AstraZeneca, Celgene/ Bristol-Myers Squibb, Adaptive Biotechnologies, ADC therapeutics, BeiGene, Genentech, Inc., syncopation, caribou biosciences: Consultancy. Munir:Alexion: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BeiGene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sobi: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AstraZeneca: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Abbvie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Walewska:AbbVie, AstraZeneca, Janssen, Beigene: Other: meeting attendancies. Herishanu:Lilly: Honoraria; Abbvie: Honoraria; AstraZeneca: Honoraria; Roche: Honoraria; Janssen: Honoraria, Research Funding. Banerji:Janssen: Honoraria; Lymphoma Canada: Research Funding; AstraZeneca: Honoraria, Research Funding; Merck: Honoraria; Beigene: Honoraria; CancerCare Manitoba Foundation: Research Funding; Hairy Cell Leukemia Foundation: Research Funding; CIHR: Research Funding; Abbvie: Honoraria; Biogen, U of M: Patents & Royalties: GSK-3 inhibtors and use there of, NAMPT OF COMPLEX I INHIBTORS OR USE THERE OF; LLSC: Research Funding. Follows:Centessa: Consultancy, Honoraria, Speakers Bureau; Janssen: Consultancy, Honoraria, Speakers Bureau; Beigene: Consultancy, Honoraria, Speakers Bureau; Lilly: Consultancy, Honoraria, Speakers Bureau; AstraZeneca: Consultancy, Honoraria, Speakers Bureau; Takeda: Consultancy, Honoraria, Speakers Bureau; Roche: Consultancy, Honoraria, Speakers Bureau; Abbvie: Consultancy, Honoraria, Speakers Bureau; Genesis Care: Consultancy, Honoraria. Ghia:Janssen: Consultancy, Honoraria, Research Funding; BeiGene: Consultancy, Honoraria, Research Funding; AstraZeneca: Consultancy, Honoraria, Research Funding; AbbVie: Consultancy, Honoraria, Research Funding; Roche: Consultancy, Honoraria, Research Funding; Lilly/Loxo Oncology: Consultancy, Honoraria, Research Funding; MSD: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria, Research Funding. Janssens:Argenx: Consultancy; Abbvie: Consultancy; AstraZeneca: Consultancy, Speakers Bureau; MSD: Consultancy; Janssen-Cilag: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Eli-Lilly: Speakers Bureau; Novartis: Speakers Bureau; Beigene: Consultancy, Speakers Bureau; Gilead: Consultancy; Amgen: Speakers Bureau; Roche: Consultancy; Sanofi: Speakers Bureau. Cymbalista:Lilly: Honoraria; AstraZeneca: Honoraria; Abbvie: Honoraria. Byrd:Orange Grove Bio: Membership on an entity's Board of Directors or advisory committees; Newave: Membership on an entity's Board of Directors or advisory committees, Research Funding; Vincerx: Current equity holder in publicly-traded company, Membership on an entity's Board of Directors or advisory committees; Orbimed: Consultancy, Research Funding; Kurome: Current equity holder in publicly-traded company, Membership on an entity's Board of Directors or advisory committees; OSU Drug Devel. Inst.: Consultancy; Eilean Therapeutics: Consultancy, Current equity holder in private company, Membership on an entity's Board of Directors or advisory committees, Research Funding; American Cancer: Membership on an entity's Board of Directors or advisory committees; AstraZeneca: Other: TRAVEL, ACCOMMODATIONS, EXPENSES. Ferrajoli:Beigene: Research Funding; AstraZeneca: Honoraria, Research Funding; Abbvie: Honoraria, Research Funding; GenMab: Research Funding; Genetech: Honoraria; Janssen: Honoraria. Wierda:Pharmacyclics LLC: Research Funding; Accutar Biotechnology: Research Funding; Numab THerapeutics: Research Funding; NIH P30 CA016672/MDACC Cancer Center Support Grant: Research Funding; AbbVie: Consultancy, Research Funding; Genentech: Research Funding; Janssens Biotech: Research Funding; KITE Pharma: Research Funding; Nurix THerapeutics: Research Funding; Juno Therapeutics: Research Funding; Loxo Oncology, Inc./Lilly: Research Funding; GlaxoSmithKline: Research Funding; Janssens Biotech Inc: Research Funding; Bristol Myers Squibb (Juno & Celgene): Consultancy, Research Funding; Gilead Sciences: Research Funding; AstraZeneca/Acerta Pharma: Consultancy, Research Funding; Miragen: Research Funding; National Comprehensive Cancer Network: Other: Nonrelevant Financial Relationship/Chair, CLL). Supported by the NIH/NCI under award number P30 CA016672 and used MDACC Cancer Center Support Grant (CCSG) shared resources; Sunesis: Research Funding; Oncternal Therapeutics, Inc.: Research Funding; Cyclacel: Consultancy, Research Funding; GSK/Novartis: Research Funding. Munugalavadla:Gilead Sciences: Current equity holder in publicly-traded company; AstraZeneca: Current Employment, Current equity holder in publicly-traded company. Wachira:Pfizer: Current equity holder in publicly-traded company; AstraZeneca: Current Employment. Wun:AstraZeneca: Current Employment. Woyach:AbbVie Inc, ArQule Inc, AstraZeneca Pharmaceuticals LP, BeiGene Ltd, Genentech, a member of the Roche Group, Janssen Biotech Inc, Loxo Oncology Inc, a wholly owned subsidiary of Eli Lilly & Company, Newave Pharmaceutical Inc, Pharmacyclics LLC, an AbbVie: Other: Advisory Committee and Consulting Agreements; AbbVie Inc, Karyopharm Therapeutics, Loxo Oncology Inc, a wholly owned subsidiary of Eli Lilly & Company, MingSight Pharmaceuticals, MorphoSys, Schrödinger, Verastem Inc.: Other: Contracted Research.

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