Quantum Leap Healthcare Collaborative

Press Release

Widely hailed as the future of phase II drug development, the adaptive I-SPY2 platform trial has set a new benchmark for efficiency, through innovation in trial design and clinical operations.

Plexxikon and QuantumLeap Healthcare Collaborative Announce Selection of PLX3397 for I-SPY 2 TRIAL in Breast Cancer

Plexxikon’s drug candidate, PLX3397, has been selected for study in the I-SPY 2 TRIAL

BERKELEY, CA, and SAN FRANCISCO, CA—December 11, 2014 – Plexxikon Inc., a member of Daiichi Sankyo Group, and QuantumLeap Healthcare Collaborative today announced that Plexxikon’s drug candidate, PLX3397, has been selected for study in the I-SPY 2 TRIAL (Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging And moLecular Analysis 2). I-SPY 2 is a standing phase 2 randomized, controlled, multicenter trial for women with newly diagnosed, locally advanced breast cancer (minimum of Stage 2) that is designed to test whether adding investigational drugs to standard chemotherapy is better than standard chemotherapy alone in the neoadjuvant setting (prior to surgery).

About the I-SPY 2 Trial
I-SPY 2 is conducted by a consortium that brings together the Food and Drug Administration (FDA), National Cancer Institute (NCI), pharmaceutical companies, leading academic medical centers, and patient advocacy groups under its umbrella. The trial is sponsored by QuantumLeap Healthcare Collaborative (QLHC), a 501(c)(3) non-profit organization dedicated to accelerating healthcare solutions.

The I-SPY 2 TRIAL employs a unique adaptive trial design to match experimental therapies with patients. Genetic or biological markers (“biomarkers”) from individual patients’ tumors are used to screen promising new treatments, identifying which therapies are most effective in specific patient subgroups. Regimens that have a high Bayesian predictive probability of showing superiority in a 300 patient phase 3 confirmatory trial in at least one of 10 predefined signatures may “graduate” from I-SPY. This high efficacy bar and rapid turnaround time allows the trial to identify the right drug for the right patient in the most expeditious fashion.

“We are pleased that PLX3397 has been included in the I-SPY 2 trial and see this as an excellent opportunity to validate this promising agent in this setting,” stated Gideon Bollag, Ph.D, chief executive officer of Plexxikon. “PLX3397 is designed to block a key component of both the tumor and its micro-environment, ultimately resulting in reduction of tumor size. Importantly, PLX3397 is expected to have applicability to multiple types of cancer.”

“The I-SPY TRIAL is designed to evaluate multiple promising new and emerging agents simultaneously with the goal of getting them to patients much more quickly. The goal is to compress the timeline to get the right drugs to the right patients, and to do so more affordably. We are excited to bring Plexxikon’s agent, which specifically targets the tumor immune microenvironment, into I SPY and learn about where it will be most effective,” stated Laura J. Esserman, M.D., M.B.A., principal investigator of I-SPY 2 and director of the Carol Franc Buck Breast Care Center at the UCSF Helen Diller Family Comprehensive Cancer Center.

"Our experience in Phase 1 studies with PLX3397 in combination with chemotherapy has shown it to be a viable and safe candidate with a unique mechanism of action to test in our patient population,” stated Hope S. Rugo, M.D., professor of medicine at the UCSF Helen Diller Family Comprehensive Cancer Center and future chaperone of this arm.

About QuantumLeap Healthcare Collaborative
QuantumLeap Healthcare Collaborative, a non-profit foundation, was established in 2005 as a collaboration between medical researchers at University of California at San Francisco, and Silicon Valley entrepreneurs. QuantumLeap’s mission is to accelerate transfer of high-impact research in clinical processes and systems technology into widespread adoption so that patients and physicians can benefit from the research as soon as practicable. QuantumLeap provides operational, financial and regulatory oversight to I-SPY 2 and is also the sponsor of its companion phase 3 confirmatory trial, I-SPY 3. For more information, visit: http://www.quantumleaphealth.org.

About PLX3397
PLX3397 is a novel oral small molecule that potently and selectively inhibits CSF1R, KIT, and mutant FLT3 kinases. CSF1R and KIT regulate key components of both the tumor and its microenvironment (macrophages, osteoclasts, mast cells). In addition to breast cancer, PLX3397 is being evaluated in several other clinical indications, including pigmented villonodular synovitis (PVNS), glioblastoma, melanoma and acute myelogenous leukemia. For more information on PLX3397 clinical trials, please visit www.clinicaltrials.gov.

About Plexxikon
Plexxikon, a member of the Daiichi Sankyo Group since April 2011, is a leader in the structure-guided discovery and development of novel small molecule pharmaceuticals to treat human disease. The company’s drug Zelboraf®, (vemurafenib/PLX4032) was approved by the FDA in 2011, and is being co-promoted in the U.S. by Daiichi Sankyo Inc. and Genentech. Plexxikon is developing a portfolio of preclinical and clinical stage compounds to address significant unmet medical needs in oncology and other therapeutic areas. Plexxikon’s Scaffold-Based Drug DiscoveryTM platform integrates multiple state-of-the-art technologies, including structural screening as a key component that provides a significant advantage over other drug discovery approaches.

UC Disclaimer
The information stated above was prepared by Plexxikon and QuantumLeap Healthcare Collaborative, and reflects solely the opinions of those companies. Nothing in this statement shall be construed to imply any support or endorsement of Plexxikon or any of its products, by The Regents of the University of California, its officers, agents and employees.

For Inquiries, Please Contact:
For Plexxikon:
Jennifer Cook Williams
Cook Williams Communications, Inc.
(360) 668-3701 - office

For QuantumLeap/I-SPY 2:
Caren Browning
King + Company PR
(212) 561-7464

Media Contact:

Karyn DiGiorgio
Quantum Leap Healthcare Collaborative