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Last update 24 May 2025

Palbociclib

Last update 24 May 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Iburance, Palbociclib (JAN/USAN), Palbociclib Isethionate

+ [10]

Target

CDK4 x CDK6

Action

inhibitors

Mechanism

CDK4 inhibitors(Cyclin-dependent kinase 4 inhibitors), CDK6 inhibitors(Cyclin-dependent kinase 6 inhibitors)

Therapeutic Areas

NeoplasmsSkin and Musculoskeletal DiseasesImmune System Diseases+ [10]

Active Indication

Breast cancer recurrentHormone receptor positive HER2 negative breast cancerER-positive/HER2-negative Breast Cancer+ [112]

Inactive Indication

acute leukemiaAcute myeloid leukaemia with 11q23 abnormalityAdvanced HER2-Positive Breast Carcinoma+ [50]

Originator Organization

Pfizer Inc.

Active Organization

The University of Texas MD Anderson Cancer Center

National Cancer Institute

Pfizer Australia Pty Ltd.

+ [20]

Inactive Organization

Hoffmann-La Roche, Inc.Xynomic Pharmaceuticals, Inc.

Calithera Biosciences, Inc.

+ [5]

License Organization

Dr. Reddy's Laboratories Ltd.

Amgen, Inc.

Ascentage Pharma Group International Co., Ltd.

+ [11]

Drug Highest PhaseApproved

First Approval Date

United States (03 Feb 2015),

ER-positive/HER2-negative Breast Cancer

RegulationBreakthrough Therapy (United States), Accelerated Approval (United States), Priority Review (China), Special Review Project (China)

Structure/Sequence

Molecular FormulaC24H29N7O2

InChIKeyAHJRHEGDXFFMBM-UHFFFAOYSA-N

CAS Registry571190-30-2

414

Clinical Trials associated with Palbociclib

NCT06757634

/ Not yet recruitingPhase 3

VIKTORIA-2: A Randomized, Open-Label, Phase 3 Study of Fulvestrant and CDK4/6 Inhibitors With or Without Gedatolisib as First-Line Treatment in Patients With HR-Positive and HER2-Negative Advanced Breast Cancer

This is a Phase 3, open-label, randomized, clinical trial evaluating the efficacy and safety of gedatolisib plus fulvestrant and CDK4/6 Inhibitors for the treatment of patients with locally advanced or metastatic HR+/HER2- advanced breast cancer.

Start Date30 Jun 2025

Sponsor / Collaborator

Celcuity, Inc.

NCT06962969

/ Not yet recruitingNot Applicable

Real-world Database Study to Observe Safety and Effectiveness of Ibrance

The purpose of this real-world study is to look at breast cancer patients receiving Palbociclib using a large real-world database collected under real-world practice. This study also looks at the safety of Ibrance, including any side effects. Side effects are undesired effects of a medicine or other type of treatment.
This study will include the data of the following participants:
* Adult women (more than18 years of age) with at least one visit with a breast cancer
* Patients with locally advanced breast cancer or metastatic breast cancer with a staging classification of stage III, stage IV. Advanced cancer is a term that is often used to describe cancer that is unlikely to be cured. Metastatic cancer is the cancer which is spread from the place where it started to other places in the body.
* Patients with a laboratory test positive for hormone receptor and negative for HER2 before or up to 60 days after advanced/metastatic breast cancer diagnosis date.
* Patients who received at least one initial prescription of Ibrance following a diagnosis of advanced/metastatic breast cancer.
This study will look at the safety of palbociclib treatment by looking at the number and severity of the side effects.

Start Date01 May 2025

Sponsor / Collaborator

Pfizer Inc.

NCT06947811

/ Not yet recruitingPhase 1/2IIT

A Multicenter, Open-Label, Single-Arm, Phase Ib/II Clinical Study of Almonertinib Combined With Palbociclib in the Treatment of Patients With KRAS Mutations-Positive Advanced Solid Tumors

The main purpose of this study is to evaluate the safety and tolerability of almonertinib combined with palbociclib in patients with advanced solid tumors harboring KRAS gene mutations, and to conduct a preliminary observation of its efficacy

Start Date30 Apr 2025

Sponsor / Collaborator

Sun Yat-Sen University

100 Clinical Results associated with Palbociclib

100 Translational Medicine associated with Palbociclib

100 Patents (Medical) associated with Palbociclib

3,508

Literatures (Medical) associated with Palbociclib

01 Sep 2025·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Rationally designed Pyrazolo[1,5-a]pyrimidines as dual inhibitors of CA IX/XII and CDK6: A novel approach for NSCLC treatment

Article

Author: Salem, Rofaida ; Giovannuzzi, Simone ; Ghabbour, Hazem A ; Ismail, Rania S M ; El-Refaie, Wessam M ; Elkelesh, Islam A ; Nocentini, Alessio ; Supuran, Claudiu T ; Abdel-Aziz, Hatem A ; Fares, Mohamed ; Almehizia, Abdulrahman A ; Elkotamy, Mahmoud S ; Eldehna, Wagdy M ; Naglah, Ahmed M

Developing novel anticancer agents that target critical pathways in non-small cell lung cancer (NSCLC) presents a considerable challenge. This study synthesized 16 pyrazolo[1,5-a]pyrimidine derivatives with zinc-binding groups through molecular hybridization to achieve dual-target inhibition of tumor-associated carbonic anhydrase (CA) isoforms IX/XII and cyclin-dependent kinase 6 (CDK6). In-vitro assays indicated that sulfonamide-bearing compounds displayed enhanced CA inhibition, with compounds 7d, 11b, and 11d presenting K_i values of 11.2, 18.4, and 19.7 nM for CA IX, while compounds 11a and 11c exhibited K_i values of 14.8 and 8.7 nM for CA XII. Cytotoxicity assays conducted on NSCLC cell lines A549 and NCI-H1734 demonstrated that compounds 7c, 7d, 7i, and 11d exhibited superior activity relative to Roscovitine in both cell lines. While these compounds demonstrated limited inhibition of cyclin-dependent kinase 4 (CDK4), 7d and 11d effectively inhibited CDK6, with IC₅₀ values of 0.054 and 0.069 μM, respectively, which are comparable to Palbociclib. Analyses of the cell cycle and apoptosis demonstrated a strong G1 arrest and a notable induction of apoptosis. Molecular docking confirmed essential binding interactions with CA IX/XII and CDK6, while in-silico ADMET predictions suggested favorable pharmacokinetics, despite potential toxicity concerns. Compounds 7d and 11d represent potential dual-target inhibitors for the treatment of NSCLC.

01 Jul 2025·BREAST CANCER RESEARCH AND TREATMENT

Non-adherence of cyclin-dependent kinases 4 and 6 inhibitors reduces overall and progression-free survival in patients with hormone receptor–positive breast cancer

Article

Author: LaBorde, Krista ; Trivedi, Meghana V ; Alfartosy, Shahad S ; De La Torre, Rodrigo ; Abughosh, Susan ; Lau, Connie ; Brown, Erika N ; Fatima, Bilqees

PURPOSE:

Cyclin-dependent kinase 4/6 inhibitors (CDK4/6i) combined with endocrine therapy improve survival in patients with hormone receptor-positive, HER2-negative (HR + /HER2-) breast cancer (BC). This retrospective study aimed to evaluate CDK4/6i adherence, factors associated with non-adherence, the impact of health-system specialty pharmacy on adherence, and effects of non-adherence on survival outcomes in HR + /HER2- BC patients.

METHODS:

Data was collected from Houston Methodist Hospital System (HM) from HR + /HER2- BC patients with medication fill history utilizing electronic medical records. CDK4/6i adherence was calculated using the mean possession ratio ≥ 80%. Multivariable logistic regression model and Kaplan-Meier analysis were utilized to evaluate factors associated with non-adherence and its impact on survival, respectively.

RESULTS:

A total of 121 patients were assessed and analyzed; 55% patients received abemaciclib, 40% were on palbociclib, and 4% were on ribociclib. More patients were on aromatase inhibitors and tamoxifen (79%) than fulvestrant (21%). Most of the patients were Caucasian (64%), non-Hispanic or Latino (84%), and postmenopausal (66%). Overall, 52 patients (43%) were non-adherent. Patients ≥ 65 years of age (OR: 0.304, [95% CI: 0.110-0.840], P-value: 0.022) and those of Hispanic or Latino ethnicity (OR: 0.291, [95% CI: 0.086-0.985], P-value: 0.047) were more likely to be non-adherent to CDK4/6i. Non-adherence to CDK4/6i was associated with worse overall survival and progression-free survival.

CONCLUSION:

43% patients were non-adherent to CDK4/6i. Older patients and those of Hispanic ethnicity were more likely to be non-adherent. Non-adherent patients had worse survival outcomes, highlighting the unmet need to implement interventions to improve CDK4/6i adherence in these patients.

01 Jun 2025·Clinical Genitourinary Cancer

CDK4/6 Inhibition With Abemaciclib in Patients With Previously Treated Advanced Renal Cell Carcinoma

Article

Author: Xu, Wenxin ; Kaelin, William G ; McGregor, Bradley A ; Choueiri, Toni K ; McDermott, David ; Xie, Wanling ; Berg, Stephanie A ; Signoretti, Sabina ; Viswanathan, Srinivas R

BACKGROUND:

Preclincal data provide a rationale for cyclin-dependent kinases 4 and 6 (CDK4/6) inhibitors alone and in combination with HIF-2α inhibitors in treatment of clear cell renal cell carcinoma (ccRCC), with randomized phase 2 clinical trials currently open exploring the combination of palbociclib with belzutifan vs belzutifan in treatment resistant ccRCC (NCT05468697). However, single agent activity for CDK4/6 inhibitors in ccRCC has not been reported. In this multi-center phase 1b clinical trial (NCT04627064), we investigated the safety and efficacy of monotherapy with abemaciclib, an oral CDK4/6 inhibitor in patients with advanced pretreated RCC.

METHODS:

Adult patients with advanced RCC with a clear cell component and ECOG status of ≤ 2 progressing after at least 1 prior regimen including immunotherapy and a VEGFR TKI received abemaciclib 200 mg twice daily in 4-week cycles until progression or unacceptable toxicity. The primary objective was to evaluate the objective response rate (ORR) of abemaciclib with a secondary endpoint of safety. First imaging was performed after 8 weeks or 2 cycles. Response was assessed per RECIST 1.1 and toxicity graded per CTCAE v5.0.

RESULTS:

Eleven patients were enrolled between December 31, 2020 and October 03, 2023. Median age was 62 years (range 54-68); 73% (n = 8) had IMDC intermediate risk disease and 1 patient had translocation RCC with a clear cell component. Median number of prior therapies was 4 (range 1-9). ORR was 0% (0/11; 8 progressive disease, 1 stable disease stopping for clinical progression, 2 not evaluable with clinical progression). About 27% (n = 3) experienced grade ≥3 treatment-related adverse events (diarrhea n = 1, nausea n = 1, neutropenia n = 1).

CONCLUSION:

In patients with heavily pretreated metastatic RCC, abemaciclib monotherapy had no clinically meaningful activity without new toxicity signals. This data will offer important insight into interpretation of results for ongoing trials exploring CDK4/6 inhibition in combination with HIF-2α inhibitors and immunotherapy.

422

News (Medical) associated with Palbociclib

23 May 2025

·www.globenewswire.com

CHMP recommends EU approval of Roche’s Itovebi for PIK3CA-mutated, ER-positive, HER2-negative, advanced breast cancer

Positive recommendation based on phase III INAVO120 data showing ItovebiTM (inavolisib) in combination with palbociclib and fulvestrant more than doubled progression-free survival in the first-line setting1The Itovebi-based regimen also demonstrated a statistically significant and clinically meaningful benefit in overall survival (OS) in the final OS analysisFinal OS data will be presented in an oral session at the 2025 American Society of Clinical Oncology Annual Meeting Basel, 23 May 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Itovebi™ (inavolisib), in combination with palbociclib (Ibrance®) and fulvestrant, for the treatment of adult patients with PIK3CA-mutated, oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment. If approved, the Itovebi-based regimen has the potential to transform the standard of care in this first-line setting, where treatments are currently limited.1 A final decision regarding the approval is expected from the European Commission in the near future. “The positive CHMP recommendation for the Itovebi-based regimen represents a significant step towards providing people in the EU with PIK3CA-mutated, ER-positive advanced breast cancer with a targeted therapy in the first-line setting,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “This recommendation is further enforced by the recent final overall survival results from the INAVO120 study, showing the regimen can meaningfully extend survival.” The presence of a PIK3CA mutation, found in approximately 40% of hormone receptor (HR)-positive breast cancers, can make the disease more aggressive and worsen survival outcomes.2,3 This underscores the importance of testing for PIK3CA mutations prior to first-line treatment so that people with a poor prognosis can benefit from an effective, PI3K-targeted therapy as soon as possible. The CHMP’s positive opinion is based on the phase III INAVO120 results, published in the New England Journal of Medicine in October 2024, which showed a 57% reduction in the risk of disease worsening or death (progression-free survival [PFS]) with the Itovebi-based regimen compared with palbociclib and fulvestrant alone (15.0 months vs. 7.3 months; hazard ratio [HR]=0.43, 95% CI: 0.32-0.59, p<0.001) in the first-line setting.1 The PFS benefit was consistent across all pre-specified subgroups, including people whose disease had spread to three or more locations, characterised as difficult-to-treat disease.1 The Itovebi-based regimen was well tolerated, with no new safety signals observed.1 Positive topline results from the final overall survival (OS) analysis, announced in January 2025, showed a statistically significant and clinically meaningful OS benefit with the Itovebi-based regimen. OS data were immature at the time of primary analysis, but a clear positive trend was observed (stratified HR=0.64, 95% CI: 0.43-0.97, p=0.0338 (boundary of 0.0098)).1 The full results from the OS analysis will be presented in an oral session at the 2025 American Society of Clinical Oncology Annual Meeting. The Itovebi-based regimen is approved for the treatment of adults with endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative, locally advanced or metastatic breast cancer in the United States, Switzerland, Canada, Australia, United Arab Emirates and China, with data from INAVO120 under review with other global health authorities. Beyond INAVO120, Itovebi is currently being investigated in three company-sponsored phase III studies (INAVO121, INAVO122, INAVO123), all in PIK3CA-mutated, locally advanced or metastatic breast cancer in various combinations.4-7 We are exploring additional studies in breast cancer and other tumour types with the hope of providing the benefit of this targeted therapy to more people with PIK3CA mutations. About Itovebi TM (inavolisib)Itovebi is an oral, targeted treatment that has been shown to provide well-tolerated and durable disease control in people with advanced PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer, who often have a poor prognosis and are in urgent need of new treatment options.1-3 Itovebi has been designed to help minimise the overall burden and toxicity of treatment and is differentiated from other PI3K inhibitors due to its high potency and specificity for the PI3K alpha isoform versus other isoforms, and unique mechanism of action that facilitates the degradation of mutated PI3K alpha.8,9 About the INAVO120 studyThe INAVO120 study [NCT04191499] is a phase III, randomised, double-blind, placebo-controlled study evaluating the efficacy and safety of Itovebi™ (inavolisib) in combination with palbociclib and fulvestrant versus placebo plus palbociclib and fulvestrant in people with PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer whose disease progressed during treatment or within 12 months of completing adjuvant endocrine therapy and who have not received prior systemic therapy for metastatic disease.4 The study included 325 patients, who were randomly assigned to either the investigational or control treatment arm.4 The primary endpoint is progression-free survival, as assessed by investigators, defined as the time from randomisation in the clinical trial to the time when the disease progresses, or a patient dies from any cause.4 Secondary endpoints include overall survival, objective response rate, and clinical benefit rate.4 Beyond INAVO120, Itovebi is currently being investigated in three additional company-sponsored phase III clinical studies in PIK3CA-mutated, locally advanced or metastatic breast cancer in various combinations:5-7 in combination with fulvestrant versus alpelisib plus fulvestrant in HR-positive/HER2-negative breast cancer post cyclin-dependent kinase 4/6 (CDK4/6) inhibitor and endocrine combination therapy (INAVO121; NCT05646862).in combination with pertuzumab plus trastuzumab for subcutaneous injection (SC) versus pertuzumab plus trastuzumab for SC and optional physician's choice of endocrine therapy as a maintenance treatment in HER2-positive disease (INAVO122; NCT05894239).in combination with a CDK4/6 inhibitor and letrozole versus placebo plus a CDK4/6 inhibitor and letrozole in the first-line setting in PIK3CA-mutated HR-positive/HER2-negative, endocrine-sensitive breast cancer (INAVO123; NCT06790693). About hormone receptor (HR)-positive breast cancerHR-positive breast cancer is the most prevalent type of all breast cancers, accounting for approximately 70% of cases.10,11 A defining feature of HR-positive breast cancer is that its tumour cells have receptors that attach to one or both hormones – oestrogen or progesterone – which can contribute to tumour growth. People diagnosed with HR-positive metastatic breast cancer often face the risk of disease progression and treatment side effects, creating a need for additional treatment options.11-13 The PI3K signalling pathway is commonly dysregulated in HR-positive breast cancer, often due to activating PIK3CA mutations, which have been identified as a potential mechanism of intrinsic resistance to standard of care endocrine therapy in combination with cyclin-dependent kinase 4/6 inhibitors.3 About Roche in breast cancerRoche has been advancing breast cancer research for more than 30 years with the goal ofhelping as many people with the disease as possible. Our medicines, along with companiondiagnostic tests, have contributed to bringing breakthrough outcomes in human epidermal growth factor 2-positive and triple-negative breast cancers. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for other subtypes of the disease, including oestrogen receptor-positive breast cancer, which is a form of hormone receptor-positive breast cancer, the most prevalent type of all breast cancers.10,11About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law.References[1] Turner NC, et al. Inavolisib-Based Therapy in PIK3CA-Mutated Advanced Breast Cancer. NEJM. 2024;391(17):1584-96.[2] Fillbrunn M, et al. PIK3CA mutation status, progression and survival in advanced HR+/HER2- breast cancer: a meta-analysis of published clinical trials. BMC Cancer. 2022;22:1002.[3] Anderson E, et al. A Systematic Review of the Prevalence and Diagnostic Workup of PIK3CA Mutations in HR+/HER2– Metastatic Breast Cancer. Int J Breast Cancer. 2020;2020:3759179.[4] ClinicalTrials.gov. A Study Evaluating the Efficacy and Safety of Inavolisib + Palbociclib + Fulvestrant vs Placebo + Palbociclib + Fulvestrant in Patients With PIK3CA-Mutant, Hormone Receptor-Positive, Her2-Negative, Locally Advanced or Metastatic Breast Cancer (INAVO120) [Internet; cited 2025 May]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT04191499. [5] ClinicalTrials.gov. A Study Evaluating the Efficacy and Safety of Inavolisib Plus Fulvestrant Compared With Alpelisib Plus Fulvestrant in Participants With HR-Positive, HER2-Negative, PIK3CA Mutated, Locally Advanced or Metastatic Breast Cancer Post CDK4/6i and Endocrine Combination Therapy (INAVO121) [Internet; cited 2025 May]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT05646862. [6] ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Inavolisib in Combination With Phesgo Versus Placebo in Combination With Phesgo in Participants With PIK3CA-Mutated HER2-Positive Locally Advanced or Metastatic Breast Cancer (INAVO122) [Internet; cited 2025 May]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT05894239.[7] ClinicalTrials.gov. A Study Evaluating the Efficacy and Safety of Inavolisib Plus CDK4/6 Inhibitor and Letrozole vs Placebo + CDK4/6i and Letrozole in Participants With Endocrine-Sensitive PIK3CA-Mutated, Hormone Receptor-Positive, HER2-Negative Advanced Breast Cancer (INAVO123) [Internet; cited 2025 April]. Available from: https://clinicaltrials.gov/study/NCT06790693. [8] Juric D, et al. A phase I/Ib study of inavolisib (GDC-0077) in combination with fulvestrant in patients (pts) with PIK3CA-mutated hormone receptor-positive/HER2-negative (HR+/HER2–) metastatic breast cancer. Presented at San Antonio Breast Cancer Symposium, 2020 December 7-10; San Antonio, USA. Abstract #P5-17-05.[9] Hong R, et al. GDC-0077 is a selective PI3K alpha inhibitor that demonstrates robust efficacy in PIK3CA mutant breast cancer models as a single agent and in combination with standard of care therapies. Cancer Res. 2018;78(4):4-14.[10] National Cancer Institute: Surveillance, Epidemiology and Ends Result Program. Cancer Stat Facts: Female Breast Cancer Subtypes [Internet; cited 2025 April]. Available from: https://seer.cancer.gov/statfacts/html/breast-subtypes.html. [11] Lim E, et al. The natural history of hormone receptor-positive breast cancer. Oncology (Williston Park). 2012;26(8):688-94,696.[12] Tomas R and Barrios CH. Optimal management of hormone receptor positive metastatic breast cancer in 2016. Ther Adv Med Oncol. 2015;7(6):304-20.[13] Galipeau N, et al. Understanding key symptoms, side effects, and impacts of HR+/HER- advanced breast cancer: qualitative study findings. J Patient-Rep Outcomes. 2019;3(1):10. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Roche Investor Relations Dr Bruno EschliPhone: +41 61 68-75284e-mail: bruno.eschli@roche.com Dr Sabine BorngräberPhone: +41 61 68-88027e-mail: sabine.borngraeber@roche.com Dr Birgit MasjostPhone: +41 61 68-84814e-mail: birgit.masjost@roche.com Investor Relations North America Loren KalmPhone: +1 650 225 3217e-mail: kalm.loren@gene.com Attachment Media & Investor Release Itovebi CHMP Opinion English

Phase 3Clinical ResultASCODrug Approval

23 May 2025

·firstwordpharma.com

Blenrep comeback continues with EU recommendation, plus a thumbs up for Itovebi

GSK's bid to bring back multiple myeloma therapy Blenrep (belantamab mafodotin) got a major boost on Friday when the antibody-drug conjugate (ADC) received a positive opinion from the EU's drug advisory panel. Also receiving a positive vote from the European Medicine Agency's Committee for Medicinal Products for Human Use (CHMP) was Roche's breast cancer drug Itovebi (inavolisib). Survival data for the PI3K inhibitor was recently spotlighted in an abstract released ahead of the American Society of Clinical Oncology (ASCO) meeting (see – ASCO25 abstract roundup: Roche's Itovebi cuts risk of death).Autolus' CAR-T cell therapy Aucatzyl (obecabtagene autoleucel) also received a thumbs up from CHMP, as well as mirdametinib, a MEK inhibitor recently acquired by Merck KGaA in its $3.9-billion takeout of SpringWorks Therapeutics. Blenrep is backBlenrep was withdrawn from global markets in 2022 following disappointing results from a confirmatory trial, but after positive readouts from two pivotal Phase III trials — DREAMM-7 and DREAMM-8 — GSK has been working to bring the BCMA-targeted ADC back. Specifically, CHMP recommended Blenrep to treat relapsed or refractory multiple myeloma in combination with bortezomib and dexamethasone (BVd) for adults who have received at least one prior therapy; and in combination with Bristol Myers Squibb's Pomalyst (pomalidomide) plus dexamethasone for patients who have received at least one prior treatment, including BMS's Revlimid (lenalidomide). In DREAMM-7, Blenrep plus BVd achieved a progression-free survival (PFS) of 36.6 months, versus 13.4 months for the comparator regimen of Johnson & Johnson's Darzalex (daratumumab) plus BVd.DREAMM-8 compared Blenrep to bortezomib, both in combination with Pomalyst and dexamethasone. At nearly 22 months follow-up, median PFS was not yet reached with Blenrep compared to 12.7 months in the control arm.In April, the UK became the first country to reauthorise Blenrep. Regulatory applications for the ADC are under review in a dozen other countries, including the US, Switzerland, Japan, China and Canada. The FDA is expected to make a decision by July 23, although there is concern that recent mass layoffs at the FDA could slow down the review process (see – Spotlight On: Could key drug approvals be put at risk of delay by FDA layoffs?).GSK is positioning the drug as a key growth driver, projecting peak annual sales exceeding £3 billion (see – Spotlight On: Can GSK deliver much needed pipeline momentum?).Itovebi impressesItovebi scored a positive opinion for its use in combination with palbociclib and fulvestrant to treat PIK3CA‑mutated, ER‑positive/HER2‑negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.The PI3K inhibitor won a similar approval in the US last October based on data from the Phase III INAVO120 study showing a 57% benefit for the Itovebi triplet on the primary endpoint of PFS compared to palbociclib and fulvestrant alone. Patients achieved PFS of 15 months in the experimental arm versus 7.3 months for placebo.At ASCO, Roche plans to share updated survival data for Itovebi. The abstract revealed that patients who received the triplet experienced a median overall survival of 34 months compared to 27 months for controls, representing a 33% reduction in the risk of death. Additionally, the combination therapy delayed the time to chemotherapy from 12.6 months in the control arm to 35.6 months with Itovebi.Go-ahead for Aucatzyl, mirdametinibA CHMP recommendation also went to Aucatzyl to treat patients aged 26 and older with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (ALL).Autolus' CD19-directed cell therapy was cleared by the FDA in November for a similar indication, based on data from the Phase Ib/II FELIX trial. Of 65 patients with ALL who received the CAR-T, 27 (42%) achieved a complete response (CR) within three months, with a median CR duration of 14.1 months.Meanwhile, Merck KGaA's newly gained mirdametinib received a positive opinion to treat symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. If fully approved by the European Commission, it will have the brand name of Ezmekly.In February, the MEK inhibitor was approved in the US for the same patient population, where it's marketed as Gomekli.

Clinical ResultDrug ApprovalPhase 3AcquisitionASCO

22 May 2025

·www.einpresswire.com

Menarini Group Presents Updated Data Underscoring the Combinability of Elacestrant (ORSERDU®) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the ASCO 2025 Annual Meeting

Preliminary efficacy analysis from the elacestrant plus everolimus and ribociclib cohorts of the ELEVATE study, along with updated safety data from additional cohorts of elacestrant plus targeted therapy combination arms, will be presented. These data highlight elacestrant’s potential role as an endocrine therapy backbone in combination with various targeted agents for patients with ER+/HER2- mBC. The robust elacestrant clinical development program across wide-ranging studies further solidifies its potential in monotherapy and combination settings, in mBC and in early breast cancer. FLORENCE, Italy and NEW YORK, May 22, 2025 (GLOBE NEWSWIRE) -- The Menarini Group (“Menarini”), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, focused on bringing transformational oncology treatments to cancer patients, will present updated preliminary efficacy and safety results from the Phase 1b/2 ELEVATE study in patients with estrogen receptor-positive (ER+), HER2-negative (HER2-) locally advanced or metastatic breast cancer (mBC). The ELEVATE study was designed to evaluate the safety and efficacy of oral-oral combination treatment options to overcome different resistance mechanisms observed in ER+/HER2- mBC and improve patient outcomes. Additionally, various other trial-in-progress updates will be presented, investigating elacestrant’s potential to become an endocrine therapy (ET) backbone across the spectrum of breast cancer. These data will be presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. The ELEVATE study is comprised of six treatment regimens evaluating elacestrant in combination with CDK4/6 inhibitors (palbociclib, abemaciclib and ribociclib) and with inhibitors of the PI3K/AKT/mTOR pathway (everolimus, alpelisib and capivasertib). ELEVATE results reported at ASCO 2025 (abstract 1070/49) include updated efficacy data which demonstrate favorable preliminary progression-free survival (PFS) from the elacestrant plus ribociclib and the elacestrant plus everolimus cohorts. A recommended phase 2 dose (RP2D) was determined to be elacestrant 345 mg plus ribociclib 400 mg. The RP2D of elacestrant 345 mg plus everolimus 7.5 mg was previously reported. “It is encouraging to see the positive preliminary efficacy and safety results when everolimus and ribocilib, respectively, are combined with elacestrant. These findings are consistent with the promising elacestrant plus abemaciclib cohort data from the same study that was presented last December, which also demonstrated favorable preliminary efficacy and safety in this setting,” said Hope S. Rugo, MD, Director, Women's Cancers Program and Division Chief, Breast Medical Oncology, City of Hope Comprehensive Cancer Center. “As the progression-free survival data and safety data continue to mature across the various cohorts of the ELEVATE study, we are encouraged by elacestrant’s potential to become an endocrine therapy backbone in combination regimens for the treatment of metastatic breast cancer.” Additional data reported separately (abstract 1079/58) provided updated Phase 1b/2 safety results from four cohorts of the ELEVATE study, including elacestrant plus ribociclib, everolimus, alpelisib, and capivasertib. These updated preliminary results show that the combinations are consistent with the known safety profiles of each targeted therapy plus standard of care endocrine therapy. “These data continue to underscore the potential value of elacestrant as a combination partner in the ER+/HER2- metastatic breast cancer treatment landscape,” said Elcin Barker Ergun, CEO of the Menarini Group. “We are also exploring the potential of elacestrant in other patient populations, including our currently enrolling ELEGANT study, which is designed to assess its potential benefit in early breast cancer patients with high risk of recurrence.” In addition, the company will be presenting other data at the ASCO Annual Meeting. See below for a complete list of Menarini Stemline abstracts. Menarini Stemline Abstracts: Presentation Title: Elacestrant (Ela) combinations with ribociclib (Ribo) and everolimus (Eve) in patients (pts) with ER+/HER2- locally advanced or metastatic breast cancer (mBC): Update from ELEVATE, a phase (Ph) 1b/2, open-label, umbrella study. Abstract Number: 1070 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 49 Presenting Author: Hope S. Rugo Presentation Title: Elacestrant combinations in patients (pts) with ER+/HER2- locally advanced or metastatic breast cancer (mBC): Safety update from ELEVATE, a phase (Ph) 1b/2, open-label, umbrella study. Abstract Number: 1079 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 58 Presenting Author: Nancy Chan Presentation Title: ADELA: a double-blind, placebo-controlled, randomized phase 3 trial of Elacestrant (ELA) + everolimus (EVE) versus ELA + placebo (PBO) in ER+/HER2- advanced breast cancer (aBC) patients with ESR1-mutated tumors progressing on endocrine therapy (ET) + CDK4/6i. Abstract Number: TPS1129 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 103b Presenting Author: Antonio Llombart-Cussac Presentation Title: ELCIN: Elacestrant in women and men with CDK4/6 Inhibitor (CDK4/6i)-naïve estrogen receptor-positive (ER+), HER2-negative (HER2-) metastatic breast cancer (mBC): an open-label multicenter phase 2 study. Abstract Number: TPS1127 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 102b Presenting Author: Virginia G. Kaklamani Presentation Title: ELEGANT: Elacestrant versus standard endocrine therapy (ET) in women and men with node-positive, estrogen Receptor-positive (ER+), HER2-negative (HER2-), early breast cancer (eBC) with high risk of recurrence in a global, multicenter, randomized, open-label phase 3 study. Abstract Number: TPS619 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 210a Presenting Author: Aditya Bardia Presentation Title: EORTC-2129-BCG: Elacestrant for treating ER+/HER2- breast cancer patients with ctDNA relapse (TREAT ctDNA) Abstract Number: TPS620 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 210b Presenting Author: Michail Ignatiadis About The Elacestrant Clinical Development Program Elacestrant is also being investigated in several company-sponsored clinical trials in metastatic breast cancer disease, alone or in combination with other therapies. ELEVATE ( NCT05563220 ) is a phase 1b/2 clinical trial evaluating the safety and efficacy of elacestrant combined with alpelisib, everolimus, capivasertib, palbociclib, ribociclib or abemaciclib. ELECTRA ( NCT05386108 ) is an open-label phase 1b/2, multicenter study evaluating elacestrant in combination with abemaciclib in patients with ER+, HER2- breast cancer. The phase 2 portion evaluates this treatment regimen in patients with brain metastases. ELCIN ( NCT05596409 ) is a phase 2 trial evaluating the efficacy of elacestrant in patients with ER+, HER2- advanced/metastatic breast cancer who received one or two prior hormonal therapies and no prior CDK4/6 inhibitors in the metastatic setting. ADELA ( NCT06382948 ) is a phase 3 randomized, double-blinded trial evaluating elacestrant in combination with everolimus in patients with ER+, HER2- mBC with ESR1-mut tumors. Elacestrant is also being evaluated in additional investigator-led trials, in trials conducted in collaboration with other companies, in metastatic breast cancer as well as in early disease. About ORSERDU (elacestrant) U.S. Indication: ORSERDU (elacestrant), 345 mg tablets, is indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1 -mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Full prescribing information for the U.S. can be found at www.orserdu.com . Important Safety Information Warning and Precautions Dyslipidemia : Hypercholesterolemia and hypertriglyceridemia occurred in patients taking ORSERDU at an incidence of 30% and 27%, respectively. The incidence of Grade 3 and 4 hypercholesterolemia and hypertriglyceridemia were 0.9% and 2.2%, respectively. Monitor lipid profile prior to starting and periodically while taking ORSERDU. Embryo-Fetal Toxicity : Based on findings in animals and its mechanism of action, ORSERDU can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the final dose. Adverse Reactions Serious adverse reactions occurred in 12% of patients who received ORSERDU. Serious adverse reactions in >1% of patients who received ORSERDU were musculoskeletal pain (1.7%) and nausea (1.3%). Fatal adverse reactions occurred in 1.7% of patients who received ORSERDU, including cardiac arrest, septic shock, diverticulitis, and unknown cause (one patient each). The most common adverse reactions ( > 10%) , including laboratory abnormalities, of ORSERDU were musculoskeletal pain (41%), nausea (35%), increased cholesterol (30%), increased AST (29%), increased triglycerides (27%), fatigue (26%), decreased hemoglobin (26%), vomiting (19%), increased ALT (17%), decreased sodium (16%), increased creatinine (16%), decreased appetite(15%), diarrhea (13%), headache (12%), constipation (12%), abdominal pain (11%), hot flush (11%), and dyspepsia (10%). Drug interactions Concomitant use with CYP3A4 Inducers and/or inhibitors : Avoid concomitant use of strong or moderate CYP3A4 inhibitors with ORSERDU. Avoid concomitant use of strong or moderate CYP3A4 inducers with ORSERDU. Use in specific populations Lactation : Advise lactating women to not breastfeed during treatment with ORSERDU and for 1 week after the last dose. Hepatic Impairment : Avoid use of ORSERDU in patients with severe hepatic impairment (Child-Pugh C). Reduce the dose of ORSERDU in patients with moderate hepatic impairment (Child-Pugh B). The safety and effectiveness of ORSERDU in pediatric patients have not been established. To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . About The Menarini Group The Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of $4.7 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini’s products are available in 140 countries worldwide. For further information, please visit www.menarini.com . About Stemline Therapeutics Inc. Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on bringing transformational oncology treatments to patients. Stemline commercializes elacestrant, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1 -mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, in the U.S., Europe, and other global regions. Stemline also commercializes tagraxofusp-erzs, a novel targeted therapy directed to CD123, for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States, Europe, and other global regions. In addition, Stemline commercializes selinexor, an XPO1 inhibitor for multiple myeloma, in Europe. The company is also conducting multiple label-expansion studies with elacestrant and tagraxofusp in breast and hematologic cancer indications, respectively, and has an extensive clinical pipeline of additional drug candidates in various stages of development for a host of solid and hematologic cancers. Media Contacts The Menarini Group Valeria Speroni Cardi Email: pressoffice@menarini.com Stemline Therapeutics, Inc. Cheya Pope Email: media@menarinistemline.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Clinical ResultASCOPhase 2Phase 3

100 Deals associated with Palbociclib

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KEGG	Wiki	ATC	Drug Bank
D10372	Palbociclib	L01XE33	DB09073

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Breast cancer recurrent	Japan	Pfizer Japan, Inc.	27 Sep 2017
Hormone receptor positive HER2 negative breast cancer	United States	Pfizer Inc.	19 Feb 2016
ER-positive/HER2-negative Breast Cancer	United States	Pfizer Inc.	03 Feb 2015

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Human Papillomavirus-Related Squamous Cell Carcinoma	Phase 3	United States	Pfizer Inc.	06 Apr 2022
Squamous Cell Carcinoma of Head and Neck	Phase 3	United States	Pfizer Inc.	06 Apr 2022
Squamous cell carcinoma of head and neck metastatic	Phase 3	United States	Pfizer Inc.	06 Apr 2022
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	Austria	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	France	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	Hungary	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	Italy	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	Spain	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	Switzerland	Pfizer Inc.	27 Aug 2019
HER2-negative breast cancer	Phase 3	United States	Pfizer Inc.	16 Jul 2019

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05361655 (Pubmed \| Breast)	Not Applicable	Metastatic breast cancer First line	1,302	Palbociclib dose adjustments	ffrapzvryf(scxmemzfag) = xlytqlzswu gvzvqqvwwk (xjsqqnfuku ) View more	Positive	01 Jun 2025
				No palbociclib dose adjustments	ffrapzvryf(scxmemzfag) = ybcjqkvfni gvzvqqvwwk (xjsqqnfuku ) View more
NCT03530696 (ESMO_BC 12025 \| ESMO_BC 22025) Manual	Phase 2	HER2 Positive Breast Cancer HER2 Positive	52	T-DM1	hvwrkiembu(orfyzrxjuc) = uidgflqdms cmcgvvdpfd (oxhirtwngg ) View more	Positive	16 May 2025
				T-DM1 + Palbociclib	hvwrkiembu(orfyzrxjuc) = lilbnnmsko cmcgvvdpfd (oxhirtwngg ) View more
ESMO_BC2025	Not Applicable	Metastatic breast cancer First line	974	Palbociclib + Aromatase Inhibitor	hzklgnrjsl(itsibdckqb) = aqpziabgpg fpixyyeucl (sfadwpfoxt, 56.2 - 79.6) View more	Positive	14 May 2025
				Aromatase Inhibitor alone	hzklgnrjsl(itsibdckqb) = lcuruegddm fpixyyeucl (sfadwpfoxt, 44.8 - 65.1) View more
ESMO_BC2025	Not Applicable	Metastatic human epidermal growth factor 2 positive carcinoma of breast	105	Palbociclib 125mg	odvroahhsg(jrimaimlsf) = kkktfccwqp mrdhrsyqsd (eoofwwqmdi ) View more	Positive	14 May 2025
ESMO_BC2025	Not Applicable	First line	320	Palbociclib + Letrozole	ovqzhhduku(vselznghwb) = ejdkszdbkm jkhqtxmdnj (axsfelqqdn )	Positive	14 May 2025
				Palbociclib + Fulvestrant	ovqzhhduku(vselznghwb) = kgxkqmqbtm jkhqtxmdnj (axsfelqqdn )
NCT06086340 (Pubmed \| Cancer Med)	Not Applicable	Metastatic breast cancer \| Hormone receptor positive HER2 negative breast cancer HR+ \| HER2-	779	Palbociclib + Aromatase Inhibitor	oxgxannnxs(nshauwillu) = mjuoayepwq wpkvpdzshs (fptoeuypcm )	Positive	01 Apr 2025
				Aromatase Inhibitor Alone	oxgxannnxs(nshauwillu) = ntizjytepy wpkvpdzshs (fptoeuypcm )
NCT01864746 (PENELOPE-B, Pubmed \| JCO Precis Oncol)	Phase 3	Neoadjuvant BRCA1/2	445	Palbociclib + Endocrine Therapy	alwcgnuygb(patbbsrzsf) = ddkoaluamz jsndofigqj (csgxyvfexz ) View more	Negative	01 Apr 2025
				Placebo + Endocrine Therapy	alwcgnuygb(patbbsrzsf) = yjfbvkkzld jsndofigqj (csgxyvfexz ) View more
NCT04494958 (PALBOBIN, CTgov)	Phase 1/2	Advanced Triple-Negative Breast Carcinoma	24	Palbociclib+Binimetinib	bqmsbubihv = qcqplswsld esgapxxxqt (lxzwhgztiy, vwqrnruwdv - uqzbniodni) View more	-	24 Mar 2025
NCT03774472 (CTgov)	Phase 1/2	ER-positive/HER2-negative Breast Cancer \| Metastatic breast cancer	15	HCQ (HCQ at 400 mg)	meovusrifp = htuibrictq aazekwfkhy (hugldycymk, dqyqaikczz - pwtqflscys) View more	-	17 Feb 2025
				HCQ (HCQ at 600 mg)	meovusrifp = savsempqlr aazekwfkhy (hugldycymk, vexaouxkzw - uurnxyzddb) View more
Pubmed \| Future Oncol	Not Applicable	Metastatic breast cancer	-	Palbociclib-fulvestrant	zkefyjfaul(awllxsctbh) = ktigjpgkbn lwncrvwqvr (ptefpzixgy, 15.2 - 23.6)	-	02 Jan 2025

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