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HKI-272

Phase 2

Breast Cancer | Small molecule | Oncology |Puma Biotechnology Inc|Last Updated: Jan 23, 2026

Success Probability

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Market & Valuation

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Trial Design

ACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment140

FDA Designations

No designations recorded

Clinical trial landscape

HKI-272 · 4 trials · 6 indications

Phase 2 2Phase 1 2
NCT01494662HKI-272 for HER2-Positive Breast Cancer and Brain MetastasesBreast Cancer
COMPLETED140 Analytics
NCT00266877Study Evaluating the Safety Of HKI-272 (Neratinib) In Subjects With Advanced Non-Small Cell Lung CancerCarcinoma, Non-Small-Cell Lung
COMPLETED172 Analytics
PHASE2COMPLETED
HKI-272 for HER2-Positive Breast Cancer and Brain Metastases
Breast CancerUnlock trial analytics
PHASE2COMPLETED
Study Evaluating the Safety Of HKI-272 (Neratinib) In Subjects With Advanced Non-Small Cell Lung Cancer
Carcinoma, Non-Small-Cell LungUnlock trial analytics

Study Endpoints

Primary Endpoints

Objective Response Rate Per Composite Response Criteria
2 years

Defined as the percentage of patients achieving a complete response (CR) or partial response (PR) of Central Nervous System (CNS) lesions based on composite criteria, reported separately in Cohorts 1, 3A, 3B. For the volumetric criteria, an objective CR will be defined as the following: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks; no new lesions; stable or decreasing steroid dose; stable or improved neurological symptoms. An objective PR by volumetric criteria will be defined as the following: At least a 50% decrease in the sum volume of CNS target lesions, taking as reference the baseline sum volume sustained for at least 4 weeks; no new lesions; stable or decreasing steroid dose; stable or improved neurological symptoms.

Objective Response Rate Per RANO-BM Criteria
2 years

Defined as the percentage of patients achieving a complete response (CR) or partial response (PR) of Central Nervous System (CNS) lesions based on RANO-BM criteria, reported separately in Cohorts 4A, 4B, and 4C. For the RANO-BM criteria, an objective CR will be defined as the following: Disappearance of all CNS target and non-target lesions sustained for at least 4 weeks; no new lesions; no corticosteroids; stable or improved clinically. An objective PR by RANO-BM criteria will be defined as the following: At least a 30% decrease in the sum LD of CNS target lesions, taking as reference the baseline sum LD sustained for at least 4 weeks; no new lesions; stable to decreased corticosteroid dose; stable or improved clinically.

Objective Response Rate for Neratinib in Patients With Non-small Cell Lung Cancer
From first dose date to progression/death or last tumor assessment, up to three years.

Objective response rate as reported by Independent Assessment (radiographic review by independent radiologists) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.0: Complete Response (CR), disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; and Non-PD for non-target lesions, and no new lesions.

Dose Limiting Toxicity Incidence of Neratinib in Combination With Paclitaxel
From first dose date through day 28

Dose Limiting Toxicity in subjects with solid tumors treated with neratinib, administered daily, in combination with paclitaxel 80 mg/m² IV on days 1, 8, and 15 of a 28 day cycle.

Maximum Tolerated Dose
From first dose date through day 28.

Maximum Tolerated Dose (MTD) of neratinib, daily, in combination with paclitaxel 80 mg/m², intravenous at days 1, 8, and 15, associated with the dose limiting toxicity data.

Objective Response Rate
From first dose date to progression or last tumor assessment, up to 140 weeks

Subjects with partial response (PR) or complete response (CR) with ERBB2 positive breast cancer treated at the maximum tolerated dose (MTD) of neratinib in combination with paclitaxel, per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v.1.0: CR, disappearance of all target lesions; PR, \>=30% decrease in the sum of the longest diameter of target lesions; and no progressive disease (PD) for non-target lesions, and no new lesions.

16-week Progression-free Survival (PFS) Rate
From first dose date to progression status (PD or death) at 16-week

16-week progression-free survival (PFS) rate for subjects with advanced breast cancer who receive neratinib at the maximum tolerated dose (MTD) in combination with trastuzumab, evaluable population.

Secondary Endpoints

Progression-Free Survival
Assessed from date of first treatment until the date of first documented progression or date of death from any cause, whichever came first, up to 3 years
Overall Survival
Assessed from date of trial registration until the date of death from any cause, up to 5 years
CNS Response by Macdonald Criteria (Bidirectional Criteria)
2 years
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1ACTIVE_COMPARATORPatients With Progressive Brain Metastases Intervention: HKI-272 (Neratinib)340 mg orally, once daily.
Cohort 2ACTIVE_COMPARATORPatients Who Are Candidates For Craniotomy. Intervention: HKI-272 (Neratinib) 240 mg orally, once daily. Surgical resection (biopsy). Neratinib concentrations from craniotomy specimen, CSF, plasma Neratinib.
Cohort 3a/3bACTIVE_COMPARATORCohort 3a will be made up of participants with No Prior Lapatinib Treatment. They will receive Neratinib 240mg Orally daily and 750mg/m2 Capecitabine twice per day for 14 days followed by 7 days rest. Cohort 3b will be made of of participants with Prior Lapatinib Treatment. Cohort 3b participants will receive Neratinib 240mg Orally daily and 750mg/m2 Capecitabine twice per day for 14 days followed by 7 days rest.
Cohort 4a/4b/4cACTIVE_COMPARATORCohort 4a will be made up of participants with previously untreated brain metastases. They will receive Neratinib 160mg Orally daily and T-DM1 3.6mg/kg IV every 3 weeks. Cohort 4b will be made up of participants with progressive brain metastases. They will receive Neratinib 160mg Orally daily and T-DM1 3.6mg/kg IV every 3 weeks. Cohort 4c will be made up of participants with progressive brain metastases and prior T-DM1. They will receive Neratinib 160mg Orally daily and T-DM1 3.6mg/kg IV every 3 weeks.
Prior Tarceva or Iressa With EGFR MutationEXPERIMENTALHKI-272 administered to patients whose disease has progressed following \> or = 12 weeks of treatment with Tarceva or Iressa and who have a tumor with an EGFR mutation demonstrated at screening
Prior Tarceva or Iressa w/o EGFR MutationEXPERIMENTALHKI-272 administered to patients whose disease has progressed following \> or = 12 weeks of treatment with Tarceva or Iressa and who have a tumor without an EGFR mutation demonstrated at screening
No Prior EGFR Tyrosine Kinase Inhibitor TreatmentEXPERIMENTALHKI-272 administered to patients with no prior EGFR tyrosine kinase inhibitor treatment, adenocarcinoma, \< or = 20 pack-year smoking history, and current non-smoker (no requirement for EGFR mutation)
HKI-272 dose level 1EXPERIMENTALPart 1: Subjects with solid tumors receiving HKI-272 (neratinib) 160 mg daily by mouth in combination with paclitaxel 80 mg/m\^2 weekly IV.
HKI-272 dose level 2EXPERIMENTALPart 1: Subjects with solid tumors receiving HKI-272 (neratinib) 240 mg daily by mouth in combination with paclitaxel 80 mg/m\^2 weekly IV.
HKI-272 expanded MTD cohort, arm AEXPERIMENTALPart 2: Subjects with metastatic breast cancer who have not received more than 1 prior cytotoxic chemotherapy treatment regimen for metastatic disease receiving HKI-272 (neratinib) 240 mg daily by mouth in combination with paclitaxel 80 mg/m\^2 weekly IV.
HKI-272 expanded MTD cohort, arm BEXPERIMENTALPart 2: Subjects with metastatic breast cancer who have not received more than 3 prior cytotoxic chemotherapy treatment regimen for metastatic disease receiving HKI-272 (neratinib) 240 mg daily by mouth in combination with paclitaxel 80 mg/m\^2 weekly IV.
Part 1 - dose level 1 (160 mg)EXPERIMENTALAll subjects receiving HKI-272 dose level 1 in combination with trastuzumab
Part 1 - dose level 2 (240 mg)EXPERIMENTALAll subjects receiving HKI-272 dose level 2 in combination with trastuzumab
Part 2 - expanded MTD cohortEXPERIMENTALAll subjects receiving HKI-272 in combination with trastuzumab

Interventions

NameTypeDescription
HKI-272DRUG240 mg orally, once daily
Surgical ResectionPROCEDURENeratinib concentrations from craniotomy specimen, CSF, plasma Neratinib.
CapecitabineDRUG750 mg/m2 orally, twice daily (1,500 mg/m2 daily) for 14 days followed by 7 days off
Ado-Trastuzumab EmtansineDRUG3.6 mg/kg IV every 3 weeks
PaclitaxelDRUG -
trastuzumabDRUGtrastuzumab 4 mg/kg IV as a loading dose followed by trastuzumab 2 mg/kg weekly thereafter
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites18

Inclusion Criteria: * Patients (men or women) must have histologically or cytologically confirmed invasive breast cancer, with metastatic disease. Patients without pathologic or cytologic confirmation of metastatic disease should have unequivocal evidence of metastasis by physical exam or radiologi...

Countries:United StatesFranceHungaryPolandSpainBelgiumCanadaChinaHong KongIndiaSouth KoreaUkraineSwitzerland
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Competitive Landscape -Breast Cancer 402 trials

Top 20 of 92 competitors

CompanyTickerTrialsLead PhaseDrugs
AstraZeneca PLCAZN47PHASE3Fulvestrant, Capivasertib
Merck & Co., Inc.MRK12PHASE3Pembrolizumab, Paclitaxel, Doxorubicin, Epirubicin, Cyclophosphamide
Eli Lilly and CompanyLLY27PHASE3Abemaciclib, Standard Adjuvant Endocrine Therapy
BioNTech SE Sponsored ADRBNTX7PHASE3DB-1303/BNT323, T-DM1
Gilead Sciences, Inc.GILD13PHASE3Sacituzumab Govitecan-hziy, Eribulin, Capecitabine Product, Gemcitabine, Vinorelbine
Novartis AG Sponsored ADRNVS30PHASE3Ribociclib
Pfizer Inc.PFE34PHASE3ARV-471, Fulvestrant
BeOne Medicines Ltd. Sponsored ADRONC5PHASE3BGB-43395, Letrozole, Abemaciclib, Palbociclib, Ribociclib
Olema Pharmaceuticals, Inc.OLMA5PHASE3Palazestrant, Fulvestrant, Anastrozole, Letrozole, Exemestane
Jazz Pharmaceuticals Public Limited CompanyJAZZ3PHASE3Zanidatamab, Trastuzumab, Eribulin, Vinorelbine, Gemcitabine
Celcuity Inc.CELC3PHASE3Gedatolisib, Palbociclib, Fulvestrant, Alpelisib
Relay Therapeutics, Inc.RLAY2PHASE3Zovegalisib, Capivasertib, Fulvestrant
GSK plc Sponsored ADRGSK2PHASE3Niraparib
Greenwich LifeSciences, Inc.GLSI1PHASE3GLSI-100
Bristol-Myers Squibb CompanyBMY5PHASE2Iza-bren, Nab-paclitaxel, Paclitaxel, Capecitabine, Carboplatin
BriaCell Therapeutics CorpBCTX2PHASE3SV-BR-1-GM, Cyclophosphamide, Interferon infiltration of the inoculation site, Retifanlimab, Treatment of Physician's Choice
Incyte CorporationINCY4PHASE2Ruxolitinib, Capecitabine, Regorafenib
Natera, Inc.NTRA3PHASE2Discontinuation of the anti-HER2 maintenance therapy
Puma Biotechnology, Inc.PBYI3PHASE2Neratinib, Loperamide, Colesevelam
Atossa Therapeutics, Inc.ATOS1PHASE2endoxifen, goserelin
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Frequently asked questions about HKI-272

What is HKI-272 used for?

HKI-272, also known as neratinib, is an investigational small molecule being studied for the treatment of advanced non-small cell lung cancer, advanced breast cancer, and breast cancer. It has been evaluated in clinical trials for these oncology indications, including in combination with other therapies.

What does HKI-272 target?

HKI-272 is a small molecule that targets HER2, a protein involved in cell growth that is overexpressed in some cancers. By inhibiting HER2, HKI-272 aims to block signaling pathways that promote tumor growth, which is relevant in HER2-positive breast cancer and other solid tumors.

Who makes HKI-272?

HKI-272 is being developed by Puma Biotechnology Inc, a biopharmaceutical company. The company's stock is traded under the ticker symbol PBYI on the stock market.

What phase is HKI-272 in?

HKI-272 has completed Phase 1 and Phase 2 clinical trials. It is an investigational drug and has not been approved by the FDA. The completed trials include studies in advanced non-small cell lung cancer and advanced breast cancer.

What clinical trials is HKI-272 in?

HKI-272 has been studied in several completed clinical trials. These include NCT00266877 for advanced non-small cell lung cancer, NCT00398567 for advanced breast cancer in combination with trastuzumab, NCT00445458 for solid tumors and breast cancer with paclitaxel, and NCT01494662 for HER2-positive breast cancer with brain metastases.

Is HKI-272 the same as neratinib?

Yes, HKI-272 is also known as neratinib. The drug has been referred to by both names in clinical research and development contexts, and they refer to the same investigational compound.