Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23

Poster display

2659 - PIK-ORL: A phase II, multicenter trial aiming to evaluate BKM120 in monotherapy in patients (pts) with metastatic/recurrent head and neck squamous cell carcinoma (HNSCC) after failure of platin and cetuximab or anti-EGFR-based therapy


09 Oct 2016


Poster display


Jérome Fayette


Annals of Oncology (2016) 27 (6): 328-350. 10.1093/annonc/mdw376


J. Fayette1, L. Digue2, C. Ferlay3, I. Treilleux4, G. Garin3, Q. Wang5, C. Hebert6, C. Even7, D. Cupissol8, S. Couchon-Thaunat9, L. Jaouen10, A. Guyennon9, C. Le Tourneau11, G. Lefebvre12, A. Mailliez12, M. Matias7, M. Degardin12, S. Tartas13, G. Clapisson14, D. Perol10

Author affiliations

  • 1 Medical Oncology, Centre Léon Bérard, 69008 - Lyon/FR
  • 2 Department Of Medical Oncology, CHU Bordeaux Hopital St. André, Bordeaux/FR
  • 3 Clinical Research, Centre Léon Bérard, Lyon/FR
  • 4 Biopathology Department, Centre Léon Bérard, Lyon/FR
  • 5 Translational Research Department, Centre Léon Bérard, Lyon/FR
  • 6 Medical Oncology, Centre Antoine Lacassagne, Nice/FR
  • 7 Medical Oncology, Institut Gustave Roussy, Villejuif/FR
  • 8 Medicine, Clinique Val d'Aurelle, Montpellier/FR
  • 9 Department Of Radiology, Centre Léon Bérard, Lyon/FR
  • 10 Clinical Research, Centre Léon Bérard, 69008 - Lyon/FR
  • 11 Dept Of Medical Oncology, Institut Curie, 75005 - Paris/FR
  • 12 Medical Oncology, Centre Oscar Lambret, Lille/FR
  • 13 Medical Oncology, Hospice Civil de Lyon centre Hospitalier Lyon Sud, Lyon/FR
  • 14 Biological Ressouces Center, Centre Léon Bérard, Lyon/FR


Abstract 2659


The PI3K/AKT pathway activation is an early event in HNSCC and an independent marker of poor outcome that seems to be involved in resistance to cetuximab. BKM120 is an oral, pan-Class I PI3K inhibitor that inhibits tumor growth in HNSCC xenografts.


This Phase II evaluates the clinical benefit of BKM120 (100mg/d, po) in 2 parallel cohorts of HNSCC pts with or without mutation in PI3KCA. Eligible pts progressed after platin and cetuximab or anti-EGFR therapy, and had documented PIK3CA status (exons 9 and 20). The primary endpoint was 2-month Disease Control Rate (DCR2m) as per RECIST 1.1. Secondary endpoints were ORR, PFS, OS, and safety. Blood and tumor samples were obtained for pharmacodynamics (PD). Imaging data were centrally reviewed. Considering that BKM120 would be uninteresting if DCR2m ≤ 10% and promising if ≥ 30% and using Simon's optimal two-stage design (α: 5% unilateral, power: 90%), 7 successes/35 evaluable pts were required for each cohort. Only results of the cohort without PI3KCA mutation are presented. .


36 HNSCC pts without PI3KCA mutation (median age: 58.6 yrs) received at least one dose of BKM120. They were heavily pretreated (33% with at least 3 previous lines). Median treatment duration was 8 wks [min-max: 4 d-55.9 wks]. At the end of 2nd Simon's Stage, the DCR2m was 38.9% (14/36pts). No OR was observed. The most common related AE (>25%) included hyperglycemia, asthenia, anxiety, depression, lymphopenia, anemia, leucocyte increase, hematocrit decrease, nausea and diarrhea. 20 pts (55.6%) presented at least one related AE ≥ Grade 3 (>5%: hyperglycemia, lymphopenia, asthenia, Na or K decrease) and 10 pts (27.8%) prematurely discontinued BKM120 due to an AE. 11pts (30.6%) experienced a related SAE including 3 SUSARs (1 fatal hyperglycemic coma, 1 death of uncertain relationship, 1 severe dehydration). PFS, OS and PD data will be presented at the meeting. Cohort with PI3KCA mutation is ongoing (n = 17).


BKM120 deserves further investigation in relapsing HNSCC pts without PI3KCA mutation, nevertheless with close monitoring of metabolic tolerance.

Clinical trial identification


Legal entity responsible for the study

Centre Leon Bérard


Institut National du Cancer and Fondation ARC


All authors have declared no conflicts of interest.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings