Bringing the Oncology Community Together

Dr. Cortes on the Phase II Ponatinib PACE Trial

Jorge E. Cortes, MD
Published Online: Wednesday, December 12, 2012
Jorge E. Cortes, MD, Department of Leukemia, University of Texas, MD Anderson Cancer Center, describes results from a 12 month follow up of the phase II PACE study that examined the potent, oral, pan-BCR-ABL inhibitor ponatinib in patients with chronic myeloid leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).

The PACE trial enrolled 449 patients who were resistant or intolerant (R/I) to dasatinib or nilotinib, or with the T315I mutation.

In the trial, patients were stratified into 6 cohorts based on disease stage and mutation status. In the chronic phase (CP)-CML cohort the endpoint was major cytogenetic response (MCyR). For those with accelerated phase (AP)-CML, blast phase (BP)-CML, and Ph+ ALL the endpoint was major hematologic response (MHR).

Overall, 271 patients were stratified to the CP-CML cohort. A MCyR was observed in 56% of the patients who were R/I to dasatinib or nilotinib and 70% with T315I mutation. Additionally, complete cytogenetic response was seen in 46%.

The AP-CML cohort contained 79 patients and showed similar response rates to those with CP-CML. Overall, 57% of R/I patients and 50% of patients harboring T315I mutations experienced a MHR. Among the 94 patients with BP-CML and Ph+ALL, MHR was observed in 35% of those R/I and 33% with T315I mutations. Five patients were treated but did not qualify for any of the cohorts.

Cortes says that ponatinib was well tolerated in the trial and had significant activity in heavily pretreated patients with CML and Ph+ ALL. Overall, this agent showed success in an area where patients have limited treatment options.

On December 14, 2012, the FDA approved ponatinib based on information from the PACE trial (Find out more).

Related Articles
New MRD Assay Helps Move Testing Forward in Multiple Myeloma Research
An improved assay for assessing MRD status in patients with multiple myeloma is contributing to the momentum for using MRD as a surrogate endpoint for survival in clinical trials and as a tool with the potential to help guide therapy choices.
FDA Approves First-Line Ofatumumab for Fludarabine-Ineligble Patients With CLL
The FDA has approved ofatumumab plus chlorambucil for previously untreated patients with chronic lymphocytic leukemia who are considered inappropriate for treatment with fludarabine therapy.
Epizyme Maintains Independence, Advances HMT-Inhibitor Platform
Epizyme, a Cambridge, Massachusetts-based biotech, opted for independence, with a successful IPO in 2013.
Most Popular Right Now
More Reading
External Resources

American Journal of Managed Care
Pharmacy Times
Physicians' Education Resource
Physician's Money Digest
Specialty Pharmacy Times
OncLive Resources

OncLive TV
Oncology Nurses
Web Exclusives

About Us
Advisory Board
Contact Us
Forgot Password
Privacy Policy
Terms & Conditions
Intellisphere, LLC
666 Plainsboro Road
Building 300
Plainsboro, NJ 08536
P: 609-716-7777
F: 609-716-4747

Copyright OncLive 2006-2014
Intellisphere, LLC. All Rights Reserved.