Review of adjuvant and neo-adjuvant abstracts from SABCS 2011 January 7 th 2012

Similar documents
The Role of Pathologic Complete Response (pcr) as a Surrogate Marker for Outcomes in Breast Cancer: Where Are We Now?

Positive HER-2 tumor. How to incorporate the new drugs into neoadjuvance

Novel Preoperative Therapies for HER2-Positive Breast Cancer. Debu Tripathy, MD University of Southern California Norris Comprehensive Cancer Center

The next wave of successful drug therapy strategies in HER2-positive breast cancer. Hans Wildiers University Hospitals Leuven Belgium

PRO: Pathologic Complete Response Does Predict Outcome for Early Stage Breast Cancer Patients

Neo-adjuvant and adjuvant treatment for HER-2+ breast cancer

New Targeted Agents Demonstrate Greater Efficacy and Tolerability in the Treatment of HER2-positive Breast Cancer

Sustained benefits for women with HER2-positive early breast cancer JORGE MADRID BIG GOCCHI PROTOCOLO HERA

DR. BOMAN N. DHABHAR Consulting Oncologist Jaslok Hospital, Fortis Hospital Mulund, Wockhardt Hospital Mumbai & BND Onco Centre INDIA

Novel Preoperative Therapies for HER2-Positive Breast Cancer

Highlights. Padova,

NeoadjuvantTreatment In BC When, How, Who?

Nadia Harbeck Breast Center University of Cologne, Germany

Treatment of Early Stage HER2-positive Breast Cancer (One size does not fit all)

Treatment of Early-Stage HER2+ Breast Cancer

Biomarkers for HER2-directed Therapies : Past Failures and Future Perspectives

pan-canadian Oncology Drug Review Initial Clinical Guidance Report Pertuzumab (Perjeta) Neoadjuvant Breast Cancer April 30, 2015

Locally Advanced Breast Cancer: Systemic and Local Therapy

Lo Studio Geparsepto. Alessandra Fabi Oncologia Medica 1

FDA Briefing Document Oncologic Drugs Advisory Committee Meeting. September 12, sbla /51 Pertuzumab (PERJETA ) Applicant: Genentech, Inc.

DR LUIS MANSO UNIDAD TUMORES DE MAMA Y GINECOLÓGICOS HOSPITAL 12 DE OCTUBRE MADRID

San Antonio Breast Cancer Symposium, December 5-9, San Antonio Breast Cancer Symposium, December 5-9, 2017

Breast : ASCO Abstracts for Review

Immunoconjugates in Both the Adjuvant and Metastatic Setting

Cáncer de mama HER2+/RE+ vs HER2+/RE : Una misma enfermedad? Dra E. Ciruelos Departamento de Oncología Médica Hospital Universitario 12 de Octubre

Taking NeoadjuvantTreatment into the Clinic

Clinical Research on PARP Inhibitors and Triple-Negative Breast Cancer (TNBC)

Rethinking neoadjuvant therapy: neoadjuvant therapy as a platform for drug development in HER2 positive breast cancer

Treatment of Early Stage HER2-positive Breast Cancer

BREAST CANCER RISK REDUCTION (PREVENTION)

Dennis J Slamon, MD, PhD

Systemic Therapy Considerations in Inflammatory Breast Cancer

(Neo)Adjuvant Chemotherapy and biological Agents (essentials in HER2 and TN early breast cancer)

Non-Anthracycline Adjuvant Therapy: When to Use?

Impact of BMI on pathologic complete response (pcr) following neo adjuvant chemotherapy (NAC) for locally advanced breast cancer

that the best available evidence has not demonstrated that pcr can predict long-term outcomes in the neoadjuvant setting.

Point of View on Early Triple Negative

TNBC: What s new Déjà vu All Over Again? Lucy R. Langer, MD MSHS Compass Oncology - SABCS 2016 Review February 21, 2017

Systemic therapy: HER-2 update. Hans Wildiers Multidisciplinair Borst Centrum/Algemene medische oncologie UZ Leuven

Triple Negative Breast cancer New treatment options arenowhere?

HER2-positive Breast Cancer

Advances in Breast Cancer Therapeutics in the Adjuvant and Metastatic Settings. Eve Rodler, MD University of California at Davis October 2016

非臨床試験 臨床の立場から 京都大学医学部附属病院戸井雅和

Supplementary appendix

Best of San Antonio 2008

Evolving Paradigms in HER2+ MBC: Strategies for Individualizing Therapy with Available Agents

Systemic Therapy of HER2-positive Breast Cancer

Overview of nab-paclitaxel in Breast Cancer

Adjuvant Chemotherapy + Trastuzumab

Ruth M. O Regan, MD Professor and Vice-Chair for Educational Affairs, Department of Hematology and Medical Oncology, Emory University, Chief of

Triple Negative Breast Cancer: Part 2 A Medical Update

2014 San Antonio Breast Cancer Symposium Review

Introduction. Approximately 20% of invasive breast cancers

Breast Cancer Earlier Disease. Stefan Aebi Luzerner Kantonsspital

XII Michelangelo Foundation Seminar

OPTIMIZING NONANTHRACYLINES FOR EARLY BREAST CANCER. Stephen E. Jones, M.D. US Oncology Research, McKesson Specialty Health The Woodlands, Tx

Recent advances in the management of metastatic breast cancer in older adults

Locally Advanced Breast Cancer: Systemic and Local Therapy

Update on the Management of HER2+ Breast Cancer. Christian Jackisch, MD, PhD Sana Klinikum Offenbach Offenbach, Germany

Systemic Therapy for Locally Advanced Breast Cancer

The absolute benefit from chemotherapy for both older and younger patients appeared most significant in ER-negative populations.

Roche s Perjeta regimen approved in Europe for use before surgery in early stage aggressive breast cancer

Breast cancer treatment

The Expert Thoughts. Alessandra Fabi Oncologia Medica 1

10/15/2012. Inflammatory Breast Cancer vs. LABC: Different Biology yet Subtypes Exist

Neoadjuvant therapy a new pathway to registration?

Lecture 5. Primary systemic therapy: clinical and biological endpoints

EARLY STAGE BREAST CANCER ADJUVANT CHEMOTHERAPY. Dr. Carlos Garbino

(NEO-)ADJUVANT THERAPY FOR HER-2+ EBC

Targe:ng HER2 in Metasta:c Breast Cancer in 2014

Karcinom dojke. PANEL: Semir Bešlija, Zdenka Gojković, Robert Šeparović, Tajana Silovski

Update in the treatment of Her2- overexpressing breast cancers. Fabrice ANDRE Institut Gustave Roussy Villejuif, France

Advanced HER2+ Breast Cancer: New Options and How to Deploy Them. José Baselga MD, PhD

Her 2 Positive Advanced Breast Cancer: From Evidence to Practice

(Neo) Adjuvant systemic therapy for HER-2+ EBC

Any News in EBC? Ann H. Partridge, MD, MPH Dana-Farber Cancer Institute November 11, 2016

Role of chemotherapy in BRCA and Triple negative breast cancer. Fernando Moreno Servicio de Oncología Médica Hospital Clinico San Carlos

Post-ESMO 2012: Tamara Rordorf Klinik für Onkologie UniversitätsSpital Zürich T.Rordorf, SAMO Luzern 1

Media Release. FDA grants Roche s Perjeta accelerated approval for use before surgery in people with HER2-positive early stage breast cancer

Disease Update: Metastatic Breast Cancer

Existe-t-il un sous groupe à risque qui pourrait bénéficier d une modification de la durée de traitement par trastuzumab? X. Pivot CHRU De Besançon

Roohi Ismail-Khan, MD, MS

St Gallen 2017 controversies & consensus

Corporate Medical Policy

The Neoadjuvant Model as a Translational Tool for Drug and Biomarker Development in Breast Cancer

Triple negative breast cancer -neoadjuvant and adjuvant systemic therapy

pertuzumab 420mg concentrate for solution for infusion vial (Perjeta ) SMC No. (1121/16) Roche Products Limited

Emerging Agents in HER2-positive Disease. Mary Cianfrocca, DO Director, Breast Oncology Program Banner MD Anderson Cancer Center Gilbert, AZ

NEW ZEALAND DATA SHEET

AUSTRALIAN PRODUCT INFORMATION Perjeta (pertuzumab)

What to do after pcr in different subtypes?

Expanding Therapeutic Strategies for HER2-Positive Metastatic Breast Cancer

Highlights in Metastatic Breast Cancer From the 2011 San Antonio Breast Cancer Symposium (SABCS)

Adjuvant Chemotherapy TNBC & HER2 Subtype

PROGNOSTICO DE PACIENTES COM CA DE MAMA METASTATICO HER2+: PODEMOS FAZER MAIS? TDM-1 AND BEYOND!

Terapia sistemica neoadiuvante: in quali tumori? Quali risultati? Dott. Giacomo Pelizzari

FEC-T plus trastuzumab & pertuzumab

Systemic Therapy of HER2-positive Breast Cancer

HER2-Targeted Rx. An Historical Perspective

Accelerated approval of Perjeta for neoadjuvant use also converted to full approval

Transcription:

Review of adjuvant and neo-adjuvant abstracts from SABCS 2011 January 7 th 2012 Ruth M. O Regan, MD Professor and Vice-Chair for Educational Affairs, Department of Hematology and Medical Oncology, Emory University, Chief of Hematology and Medical Oncology, Georgia Cancer Center for Excellence, Grady Memorial Hospital

Abstracts Delayed inhibition of HER2 in early stage breast cancer (Abstract S4-7) HER2-directed agents in the neoadjuvant setting (S5-6, S5-4, S5-1) Management of patients non-responsive to pre-operative therapy (Abstracts S3-2, S3-6)

Results of a randomized, double-blind, multicenter, placebo-controlled (TEACH) study of adjuvant lapatinib in women with early stage ErbB2-overexpressing breast cancer Goss et al Abstract S4-7

Study rationale At the time that results of the adjuvant trastuzumab trials were made available, many countries worldwide did not have access to trastuzumab TEACH trial designed to determine The natural history of HER2-positive breast cancers and whether there was continued recurrences overtime Whether delayed anti-her2 therapy with lapatinib would decrease these delayed recurrences if they occurred

TEACH trial design Stage 1-3c primary breast cancer HER2+ (3+ or FISH+) No prior trastuzumab (Neo) adjuvant chemotherapy Appropriate endocrine therapy Stratification: Time from diagnosis 4 vs >4 years Lymph node + vs ER+ and/or PR+ vs ER-, PR- RANDOMIZE Lapatinib 1500mg qd X 1 year n = 3147 Placebo qd X 1 year Diagnosis 4 years

TEACH: Endpoints and statistics Primary endpoint: DFS Hypothesis: Lapatinib will decreases recurrence by 23% (requires HR of 0.769), assuming an annual recurrence rate of 7% in the lapatinib arm and 10% in the placebo arm

TEACH: Baseline characteristics Lapatinib N = 1571 Placebo N = 1576 Median age 51 52 Median time from initial diagnosis 2.7 years 2.75 years Years since initial diagnosis: 4 years 71% 72% > 4 years 29% 28% 0 1 year 20% 21% Hormone receptor status: ER and/or PR + 59% 59% ER, PR-negative 41% 41% Lymph node status: Positive 56% 56%

TEACH RESULTS: Ongoing Risk of Recurrence from Diagnosis in Placebo Arm Year(s) Disease-free Patients (%) Number of patients at risk Placebo 1576 1569 1504 1430 1323 1043 781 539 310 181 96 1 99.9% 2 96.7% 3 93.1% 4 91.0% 5 87.8% 6 84.4% 7 81.1% 8 79.2% 9 77.2% 10 74.9% 8

TEACH: K-M Plot of DFS According to Hormone Receptor (HR) Status in untreated (placebo) ITT Population ER/PgR+ve ER/PgR -ve 0.0 Number of patients at risk HR+ placebo 927 880 845 814 789 757 626 420 193 HR placebo 649 607 567 529 506 490 422 286 134

TEACH Primary Endpoint: K-M Plot of DFS in ITT Population Time From Randomization HR 0.83 (0.70-1.00); p=0.053 a Lapatinib Placebo Median Follow up: 4 years 0.0 Number of patients at risk Lapatinib 1500 mg 1571 1431 1349 1293 1233 1168 1001 661 299 Placebo 1576 1487 1412 1343 1295 1247 1048 706 327 a p value based on 2-sided stratified log-rank test

TEACH: K-M Plot of OS in ITT Population Time From Randomization Lapatinib Placebo HR 0.99 (0.74-1.31); p=0.966 a Lapatinib (n=1571) Placebo (n=1576) Died, n (%) 92 (6%) 97 (6%) 0.0 Number of patients at risk Lapatinib 1500 mg 1571 1518 1477 1444 1402 1340 1153 777 351 Placebo 1576 1555 1528 1487 1448 1389 1188 805 374 a p value based on 2-sided stratified log-rank test. 11

TEACH: Forest Plot of DFS for Subgroups in ITT Population L=lapatinib; P=placebo. 12

TEACH: K-M Plot of DFS According to Hormone Receptor (HR) Status in treated ITT Population ER/PgR+ve: HR 0.98 (0.77-1.25); p=0.886 a ER/PgR-ve: HR 0.68 (0.52-0.89); p=0.006 a 0.0 Number of patients at risk HR+ lapatinib 1500 mg 932 847 794 761 731 693 593 384 169 HR+ placebo 927 880 845 814 789 757 626 420 193 HR lapatinib 1500 mg 639 584 555 532 502 475 408 277 130 HR placebo 649 607 567 529 506 490 422 286 134 a p value based on 2-sided stratified log-rank test.

TEACH: K-M Plot of DFS in Confirmed FISH+ Population Time From Randomization HR 0.82 (0.67-1.00); p=0.04 a Lapatinib Placebo 0.0 Number of patients at risk Lapatinib 1500 mg 1230 1137 1069 1026 980 934 810 533 245 Placebo 1260 1186 1125 1075 1035 993 840 578 275 a p value based on 2-sided stratified log-rank test.

TEACH: Sites of BRCA Recurrences and Second Primaries in Confirmed FISH+ Population Lapatinib 1500 mg (n=1230) Placebo (n=1260) Any recurrence of disease, second primary or contralateral BRCA, n (%) 157 (13%) 208 (17%) Local recurrence 24 (2%) 37 (3%) Regional recurrence 19 (2%) 25 (2%) Distant recurrence 102 (8%) 133 (11%) CNS 12 (<1%) 20 (2%) Contralateral BRCA 10 (<1%) 13 (1%) Second primary malignancy 22 (2%) 24 (2%)

TEACH: Time-to-First BRCA Recurrences in Confirmed FISH+ Population Lapatinib 1500 mg (n=1230) Placebo (n=1260) Any recurrence or contralateral BRCA, n (%) a 137 (11%) 183 (15%) Patients with recurrence at yearly time points, % 1 yr 3.7% 6% 2 yr 7.9% 10.5% 3 yr 10.6% 13.2% Any recurrence HR (95% CI) 0.79 (0.63-0.98) 2-sided stratified log-rank p value b 0.033 Patients with CNS recurrence at yearly time points, % 1 yr 0.5% 0.7% 3 yr 1.1% 1.3% CNS recurrence HR (95% CI) 0.66 (0.33, 1.34) 2-sided stratified log-rank p value b 0.286 a Events not included were death and second primary cancer (competing risk). b p value stratified by time from initial diagnosis, HR status, and lymph node involvement.

TEACH: Adverse events Lapatinib associated with more AEs, especially diarrhea and rash 20% drug discontinuation on lapatinib arm No significant difference in cardiac events between the two arms Lapatinib associated with elevated LFTs in 8% of patients

TEACH: conclusions Patients with HER2+ cancers who do not receive trastuzumab remain at an ongoing risk of recurrence up to 10 years (regardless of HR status) DFS was not significantly improved with delayed lapatinib in the ITT population but did benefit patients: With ER/PR-negative cancers Within one year of diagnosis

Neoadjuvant Pertuzumab and Trastuzumab Concurrent or Sequential with an Anthracycline-Containing or Concurrent with an Anthracycline-Free Standard Regimen: A Randomized Phase II Study (TRYPHAENA) Schneeweiss et al Abstract S5-6

Primary study objective To make a preliminary assessment of the tolerability of neoadjuvant treatment with pertuzumab and trastuzumab plus anthracycline-taxanebased or carboplatin-taxane-based standard chemotherapy regimens in HER2-positive EBC EBC, early breast cancer; HER2, human epidermal growth factor receptor

Study design Cycles 1 3 4 6 A FEC Docetaxel HER2-positive EBC centrally confirmed (n = 225) B C Pertuzumab + trastuzumab FEC Docetaxel Docetaxel Pertuzumab + trastuzumab Pertuzumab + trastuzumab S u r g e r y Trastuzumab to complete 1 year Carboplatin All 3 arms were experimental Study dosing q3w: FEC: 500 mg/m 2, 100 mg/m 2, 600 mg/m 2 Carboplatin: AUC 6 Trastuzumab: 8 mg/kg loading dose, 6 mg/kg maintenance Pertuzumab: 840 mg loading dose, 420 mg maintenance Docetaxel: 75 mg/m 2 (escalating to 100 mg/m 2 if tolerated, in Arms A and B only) AUC, area under the plasma concentration-time curve; EBC, early breast cancer; FEC, 5-fluorouracil, epirubicin, cyclophosphamide

Study endpoints Primary endpoint: Cardiac safety Symptomatic LVSD (grade 3) LVEF declines ( 10 percentage points and below 50%) Secondary endpoints: Toxicity pcr (defined as the absence of invasive tumor residues in the breast at surgery; remaining in situ lesions allowed; ypt0/is) Study was not powered for formal comparison between arms Clinical response rate Rate of breast-conserving surgery Disease-free survival and overall survival Biomarker evaluation LVEF, left ventricular ejection fraction; LVSD, left ventricular systolic dysfunction; pcr, pathologic complete response

Baseline characteristics in the safety population FEC+H+P x3 T+H+P x3 n = 72 FEC x3 T+H+P x3 n = 75 TCH+P x6 n = 76 Median age, years (range) 49.0 (27 77) 49.0 (24 75) 50.0 (30 81) ECOG PS 0, n (%) 1, n (%) ER and/or PR positive, n (%) ER and PR negative, n (%) Disease type, n (%) Operable Locally advanced Inflammatory HER2 IHC 0 and 1+, n (%) 2+, n (%) 3+, n (%) HER2 FISH positive, n (%) FISH negative, n (%) Unknown, n (%) 65 (91.5) 6 (8.5) 39 (53.4) 34 (46.6) 53 (72.6) 15 (20.5) 5 (6.8) 1 (1.4) 5 (6.8) 67 (91.8) 69 (94.5) 0 (0.0) 4 (5.5) 66 (88.0) 9 (12.0) 35 (46.7) 40 (53.3) 54 (72.0) 17 (22.7) 4 (5.3) 0 (0.0) 1 (1.3) 74 (98.7) 69 (92.0) 1 (1.3) 5 (6.7) 67 (88.2) 9 (11.8) 40 (51.9) 37 (48.1) 49 (63.6) 24 (31.2) 4 (5.2) 0 (0.0) 2 (2.6) 75 (97.4) 73 (94.8) 2 (2.6) 2 (2.6) ECOG PS, Eastern Cooperative Oncology Group performance status; ER, estrogen receptor; FEC, 5-fluorouracil, epirubicin, cyclophosphamide; FISH, fluorescence in situ hybridization; H, trastuzumab; IHC, immunohistochemistry; P, pertuzumab; PR, progesterone receptor; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Cardiac events during neoadjuvant treatment FEC+H+P x3 T+H+P x3 n = 72 FEC x3 T+H+P x3 n = 75 TCH+P x6 n = 76 Symptomatic LVSD (grade 3), n (%) 0 (0.0) 2 (2.7) 0 (0.0) LVSD (all grades), n (%) 4 (5.6) 3 (4.0) 2 (2.6) LVEF decline 10% points and below 50%, n (%) 3 (4.2) 4 (5.3) 3 (3.9) FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; LVEF, left ventricular ejection fraction; LVSD, left ventricular systolic dysfunction; P, pertuzumab; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Mean change in LVEF 8 Central readings FEC+H+P x3 T+H+P x3 (n = 72) FEC x3 T+H+P x3 (n = 75) TCH+P x6 (n = 76) 6 Mean absolute change in LVEF (%) 4 2 0-2 -4-6 -8-10 -12-14 Cycle Patients with assessment, n Neoadjuvant 2 66 65 62 4 64 58 62 6 69 65 68 10 36 33 35 12 34 27 28 15 19 18 17 Adjuvant 18 13 End of treatment 15 FEC+H+P x3 T+H+P x3 10 FEC x3 T+H+P x3 16 TCH+P x6 FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; LVEF, left ventricular ejection fraction; P, pertuzumab; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

10 most common grade 3 adverse events excluding cardiac events during neoadjuvant treatment Adverse event, n (%) FEC+H+P x3 T+H+P x3 n = 72 FEC x3 T+H+P x3 n = 75 TCH+P x6 n = 76 Neutropenia 34 (47.2) 32 (42.7) 35 (46.1) Febrile neutropenia 13 (18.1) 7 (9.3) 13 (17.1) Leukopenia 14 (19.4) 9 (12.0) 9 (11.8) Diarrhea 3 (4.2) 4 (5.3) 9 (11.8) Anemia 1 (1.4) 2 (2.7) 13 (17.1) Thrombocytopenia 0 (0.0) 0 (0.0) 9 (11.8) Vomiting 0 (0.0) 2 (2.7) 4 (5.3) Fatigue 0 (0.0) 0 (0.0) 3 (3.9) Alanine aminotransferase inc. 0 (0.0) 0 (0.0) 3 (3.9) Drug hypersensitivity 2 (2.8) 0 (0.0) 2 (2.6) FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; inc., increased; P, pertuzumab; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Pathologic complete response Pathologic complete response (%) 100 90 80 70 60 50 40 30 20 10 0 61.6 ypt0/is FEC+H+P x3 T+H+P x3 (n = 73) 57.3 FEC x3 T+H+P x3 (n = 75) 66.2 TCH+P x6 (n = 77) FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; P, pertuzumab; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Pathologic complete response Pathologic complete response (%) 100 90 80 70 60 50 40 30 20 10 0 61.6 ypt0/is 50.7 FEC+H+P x3 T+H+P x3 (n = 73) 57.3 45.3 FEC x3 T+H+P x3 (n = 75) ypt0 ypn0 66.2 TCH+P x6 (n = 77) 51.9 FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; P, pertuzumab; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Pathologic complete response by hormone receptor status Pathologic complete response (%) ER- and PR-negative 79.4 46.2 FEC+H+P x3 T+H+P x3 (n = 73) 65.0 ER- and/or PR-positive 48.6 FEC x3 T+H+P x3 (n = 75) 83.8 TCH+P x6 (n = 77) 50.0 ypt0/is ER, estrogen receptor; FEC, 5-fluorouracil, epirubicin, cyclophosphamide; H, trastuzumab; P, pertuzumab; PR, progesterone receptor; T, docetaxel; TCH, docetaxel/carboplatin/trastuzumab

Summary and conclusions Results from TRYPHAENA indicate a low incidence of symptomatic and asymptomatic LVSD across all arms Concurrent administration of pertuzumab plus trastuzumab with epirubicin resulted in similar cardiac tolerability compared with sequential administration or the anthracycline-free regimen Neutropenia, febrile neutropenia, leukopenia, and diarrhea were most frequently reported adverse events (grade 3) across all arms Regardless of chemotherapy chosen, the combination of pertuzumab with trastuzumab in the neoadjuvant setting resulted in high pcr rates (57 66%) Lower PCR in HR-positive versus HR-negative TRYPHAENA supports the ongoing APHINITY study, a Phase III trial to evaluate pertuzumab and trastuzumab plus standard chemotherapy in the adjuvant setting (NCT01358877)

Comparison of survival according to pathological complete response (pcr) in patients with HER2-positive breast cancer receiving neoadjuvant chemotherapy with and w/o trastuzumab compared to patients with HER2-negative tumors, Loibl et al Abstract S5-4

TECHNO Study - Overall Survival pcr no pcr EC x4 Paclitaxel + Trastuzumab x4 Untch et al. J Clin Oncol 2011; 29:3351

Objectives Definition of three subgroups: HER2-positive with trastuzumab HER2-positive without trastuzumab HER2-negative Compare DDFS and OS in these subgroups: pcr vs. no pcr hormone receptor positive and -negative tumors

Methods All neoadjuvant trials with follow-up were included Known HER2 status (locally or centrally assessed) Hormone receptor positivity was defined as 10% cells positive for estrogen and/or progesterone receptor (locally or centrally assessed) pcr defined as no invasive and no non-invasive residuals in breast and lymph nodes (ypt0 ypn0) Adjustment for trial

All patients 6377 Eligible with known HER2-status 4387 HER2 negative HER2 positive HER2 positive 3060 w/o trastuzumab with trastuzumab 665 662 pcr no pcr pcr no pcr pcr no pcr 454 2606 119 546 181 481 pcr-rate* pcr-rate* pcr-rate* 14.8% 17.9% 27.3% *ypt0 ypn0

Patients Characteristics Age median 49 (22-81) years % ct1-3 87 cn+ 53 Ductal invasive 82 Grading 3 40 Hormone receptor positive 66 HER2-negative 70

DDFS and OS in the three subgroups vs vs P=0.040 P=0.382 vs vs P=0.142 P=0.103 n= 662 HER2+ with trastuzumab n= 3060 HER2 negative n= 665 HER2+; no trastuzumab

DDFS and OS by pcr HER2-negative pcr pcr no pcr no pcr Log-rank p<0.001 Log-rank p<0.001

DDFS and OS by pcr HER2-positive Without Trastuzumab pcr pcr no pcr no pcr Log-rank p=0.007 Log-rank p=0.037

DDFS and OS by pcr HER2-positive with Trastuzumab pcr pcr no pcr no pcr Log-rank p<0.001 Log-rank p=0.001

No pcr OS analysis by pcr pcr vs vs p=0.295 p=0.384 Log-rank vs vs p=0.058 p=0.134 Arm N Events Arm N Events positive w trast 481 35 positive w trast 181 1 positive w/o trast 546 75 positive w/o trast 119 9 negative 2606 310 negative 454 14 n= 662 HER2+ with trastuzumab n= 3060 HER2 negative n= 665 HER2+; no trastuzumab

DDFS and OS in Hormone Receptor-positive vs vs P=0.606 P=0.795 vs vs P=0.614 P=0.652 n= 662 HER2+ with trastuzumab n= 3060 HER2 negative n= 665 HER2+; no trastuzumab

DDFS and OS in Hormone Receptor Negative vs p=0.012 vs p=0.123 vs p=0.059 vs p=0.029 n= 662 HER2+ with trastuzumab n= 3060 HER2 negative n= 665 HER2+; no trastuzumab

Summary Patients with HER2-positive primary breast cancer treated with trastuzumab and chemotherapy achieve a higher pcr rate DDFS and OS was significantly better with pcr in HER2- negative, HER2-positive non- trastuzumab and HER2-positive trastuzumab patients In pcr patients OS tended to be superior with trastuzumab compared to HER2-positive, non-trastuzumab and HER2- negative patients In particular HER2-positive, hormone receptor negative patients have a better DDFS and OS compared to HER2- positive, non-trastuzumab and HER2-negative patients

Neoadjuvant chemotherapy adapted by interim response improves overall survival of primary breast cancer patients Results of the GeparTrio trial. von Minckwitz et al Abstract 3-2

Aims To take advantage from the in vivo chemosensitivity test situation of neoadjuvant treatment To develop specific treatment strategies for patients with or without response to 2 cycles TAC: Responding patients: treatment intensification by increased cycle number Non-responding patients: switch to non-cross resistant treatment

GeparTrio Trial Design N=2072 Core biopsy: uni/bilateral ct2-4a-d cn0-3 size 2 cm* *low risk patients were excluded (T2 + ER/PR pos. + cno + G1/2 + > 35 yrs) NC R Sonography CR/ PR R NX TACx6 response- guided arms conventional arms TACx6 TACx8 von Minckwitz et al, JNCI 100: 542, 2008 von Minckwitz et al. JNCI 100; 552, 2008

Short Term Efficacy (pcr = ypt0 ypn0) Responder Non-Responder N=1344 N=604 30% P=0.27 P=0.73 20% 10% 21.0 23.5 0 5.3 6.0 TACx6 TACx8 TACx6 TAC-NX von Minckwitz et al, JNCI 100: 542, 2008 von Minckwitz et al. JNCI 100; 552, 2008

Objectives Primary: Pathologic response (responder) Sonographic response (non-responder) Secondary (actual with median follow up of 62 months): To determine 5-year DFS and OS To examine treatment effects by breast cancer phenotype (post-hoc analysis)

Study Population Characteristic Conventional Response-guided TACx6 TACx8 or TAC-NX N=1025 N=987 % % Age < 40 years 16.9 18.2 ct> 40 mm 60.5 61.5 ct4a-c 9.0 8.7 ct4d 4.6 4.3 cn + 55.3 54.7 Lobular type 13.8 13.1 Grade 3 41.0 35.1 HR-negative 36.8 34.4 HER2-positive 30.5 29.1

DFS and OS after conventional (TACx6) vs. response-guided (TACx8/TAC-NX) treatment Median follow up 62 months

Adjusted Analysis for DFS Variable Group HR p-value Treatment Response-guided 0.71 0.001 Age 40 years 0.92 0.6 T-stage ct1-3 0.60 <0.001 T-size <40 mm 0.81 0.08 cn negative 0.56 <0.001 Histological type lobular 0.99 0.9 Grade 1-2 0.84 0.12 HR positive 0.49 <0.001 HER2 negative 0.88 0.3

DFS after TACx6 vs TACx8 in responding patients

DFS after TACx6 vs TAC-NX in non-responding patients

Subgroup analysis comparing DFS after conventional vs response-guided treatment

Breast Cancer phenotypes (St. Gallen definition*) Phenotype Definition Conventional Response-guided % % Luminal A HR+, HER2-, G1/2 34.4 37.1 Luminal B (HER2-) HR+, HER2-, G3 13.5 12.8 Luminal B (HER2+) HR+, HER2+ 17.3 17.8 HER2+ (non-luminal) HR-, HER2+ 11.7 10.4 Triple-negative HR-, HER2-23.1 22.0 Missing N=181 N=227 *Goldhirsch A, Ann Oncol 2011

pcr (%) pcr Rates by Subtype 40 35 30 25 20 15 10 5 0 Luminal A (N=572) Luminal B (HER2-) Luminal B (HER2+) HER2+ (non-luminal) Triple-negative (N=211) (N=281) (N=178) N=362)

DFS in Luminal A tumors by pcr by treatment

DFS in Luminal B (HER2-) by pcr by treatment

DFS in Luminal B (HER2+) tumors by pcr by treatment

DFS in HER2+(non-luminal) tumors by pcr by treatment

DFS in Triple Negative Tumors by pcr by treatment

Conclusion Interim response-guided (longer or sequential) neoadjuvant chemotherapy improved survival. Treatment effects on survival derived from luminal-type tumors. This treatment effect could not be predicted by pcr as these tumors have lower pcr rates and their prognosis does not depend on pcr. Patients with HER2+ or triple-negative tumors did not benefit from response-guided treatment. pcr is highly prognostic in these subgroups. Lack of treatment effect on pcr rate corresponds to lack of long term treatment.

NEOADJUVANT CHEMOTHERAPY OF PACLITAXEL WITH OR WITHOUT RAD001 - RESULTS OF THE NON-RESPONDER PART OF THE GEPARQUINTO STUDY (GBG 44) Huober et al Abstract 3-6

Introduction HER2- Non-Responder The oral signal transduction inhibitor everolimus (RAD001 = Rad), binds selectively to mtor (mammalian target of rapamycin) mtor is an intracellular protein kinase controlling cellular proliferation of activated T-lymphocytes and neoplastic cells. In vitro synergistic reactions with Rad and several chemotherapeutic drugs including paclitaxel were observed1 Additional neoadjuvant treatment strategies are needed for patients without early clinical response 1 O`Reilly T et al. Anti-Cancer Drugs 2011

HER2- Non-Responder Study Design HER-2 negative Pw Core biopsy 2nd core NC R Surgery R EC +/- B RAD001 Pw RAD +/- Bevacizumab T +/- B PR CR/ +/- Bevacizumab E = Epirubicin C = Cyclophosphamide T = Docetaxel B = Bevacizumab Pw = Paclitaxel, weekly (80 mg/m2: day 1 q day 8-12 weeks) R = RAD001 (5 mg / day from day 13 after a dose escalation starting from 2.5 mg every other day to 5mg every day)

HER2- Non-Responder Patients & Tumor Characteristics Pw Pw+Rad N=198 N=197 age (median yrs) 51 50 Age < 40 years (%) 13.1 9.1 palpable T-size (median mm) 40 40 % % ct 4 (a-c) 8.6 8.1 inflammatory 8.1 9.1 cn + 55.7 59.1 lobular type 11.1 10.2 hormone receptor positive 71.2 73.1 grade 3 35.5 33.7

HER2- Non-Responder 10% 9% 8% 7% 6% 5% 4% 3% pcr (no invasive & no non-invasive residuals in breast & nodes based on central pathology report review N=395) P=0.476 2% 5.6% 3.6% 1% 0% N=11 N=7 2.8-9.7% 1.4-7.2% Pw n= 198 Pw+Rad n=197

HER2- Non-Responder pcr Rates According to Secondary Endpoint Definitions no invasive residuals in breast & nodes (ypt0/tis, ypn0) Houston 20% 20% 18% 18% P=0.836 15% 15% 13% 13% 10% 10% 8% 8% no invasive residuals in breast (ypt0/tis) NSABP P=0.689 5% 6.1% 5% 7.1% 3% 5.1% 3% 6.6% 0% 0% Pw Pw+Rad Pw Pw+Rad

HER2- Non-Responder ccr+cpr (clinical complete and partial response at surgery n=379) 100% 90% 80% 70% 60% 50% 40% 30% P=0.061 62.1% 52.2% 20% 54.8-68.9% 44.7-59.6% 10% 0% Pw n= 195 Pw+Rad n=184

HER2- Non-Responder Conclusions pcr rate is low (4.6%) in patients not responding to the initial 4 cycles of neoadjuvant chemotherapy with or without Bev Addition of Rad to 12 weeks paclitaxel did not improve pcr rate in these patients (Pw 5.6% vs. Pw+Rad 3.6%; P=0.476) Toxicity was higher in the group treated with Rad DFS and OS have to be awaited because pcr might not be the appropriate endpoint (high number HR+) A large biomarker program is ongoing to identify predictive markers

Neo-adjuvant pertuzumab and trastuzumab: Biomarker analyses of a 4-arm randomized phase 2 trial (NeoSphere) in patients with HER2-positive breast cancer Gianni et al Abstract S5-1

NeoSphere: Correlative results PI3-kinase mutations were not associated with rate of PCR No role for truncated forms of HER2, including p95 HER2 in predicting PCR IGF1R, HER3, PTEN and EGFR were higher in ER-positive cancers, while HER2 was higher in ER-negative breast cancers

Practice changing? Potentially important: Late recurrences in HER2-positive breast cancers Confirmatory: Decreased incidence (and importance) of PCR in HR-positive, HER2-positive breast cancers Equivalence of anthracycline and non-anthracycline containing regimens in HER2-positive breast cancers Dual targeting of HER2 superior to single agents in preoperative setting Depressing: Poor outcome for patients with TNBC who do not obtain a PCR and Lack of improvement in PCR rate with non-resistant chemotherapeutics and novel agents