BioMarin Provides BMN 673 Program Update

GlobeNewswire
Share
Thursday, July 25th 2013
Advanced

RECIST Response Rate Increases to 50% in gBRCA Breast Cancer; Study Ongoing

Phase 3 Trial to Start in 3Q 2013

National Breast Cancer Coalition Announces Intention to Assist With Trial Recruitment

SAN RAFAEL, Calif., July 25, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today provided an update on its ongoing Phase 1/2 study for its poly ADP-ribose polymerase (PARP) inhibitor BMN 673 for the treatment of solid tumors. As of July 24, the RECIST response rate from the ongoing trial is nine out of 18 breast cancer patients, or 50 percent, including one confirmed complete response. This response rate includes three additional confirmed responses since the last update at the ASCO Annual Meeting, and two new patients are yet to be confirmed. Four patients are ongoing with stable disease with potential for additional responses. The study is still ongoing, and the company will provide additional updates later this year, including data in ovarian cancer, Ewing's sarcoma and small cell lung cancer. 

Since the ASCO Annual Meeting, the company has met with the FDA and MAA to review its planned clinical program in deleterious gBRCA mutation metastatic breast cancer. At those meetings, health authorities confirmed that a primary endpoint of Progression Free Survival (PFS) could serve as the basis for a potential approval. BioMarin now expects to initiate a Phase 3 trial for BMN 673 in deleterious gBRCA mutation metastatic breast cancer in late third quarter of 2013, earlier than previously announced at the ASCO Annual Meeting in early June.

National Breast Cancer Coalition (NBCC) has announced its intention to assist BioMarin's development of the PARP inhibitor for the treatment of hereditary breast cancer with BRCA mutations. NBCC intends to participate with BioMarin on enrollment initiatives in the United States and around the world. In addition, NBCC will be a resource on study design, implementation and execution.     

"NBCC is an organization committed to ending breast cancer in large part by participating in and contributing to the scientific process. The application of drug development science to well-defined problems is an important step in our mission. Therefore, we are pleased to collaborate with BioMarin to develop BMN 673 as a potential first and/or best in class PARP inhibitor for patients with hereditary forms of cancer, namely BRCA mutations. This project offers the hope of improving the length and/or quality of life for patients," said Fran Visco, President of the National Breast Cancer Coalition.

"We look forward to conducting a world-class study, in collaboration with the finest clinical investigators in the world to evaluate the safety and efficacy of BMN 673 in the metastatic setting. Our advocates are poised to change the world through our actions. Ultimately, we hope to prevent women from getting breast cancer and prevent it from spreading outside the breast," Visco added.

"BioMarin is an organization that wants to make big differences in the lives of patients. The data emerging from the ongoing BMN 673 study is progressively more encouraging. We are committed to the successful execution of the Phase 3 program in hopes of offering patients a safe and easily administered pill, which is an effective treatment option," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "We are honored to include a seat at the table for our colleagues from NBCC. The purpose of our efforts is best served hearing their voice from the beginning and taking action together."

About the National Breast Cancer Coalition (NBCC)

The National Breast Cancer Coalition (NBCC) is dedicated to knowing how to end breast cancer by January 1, 2020 through the power of grassroots action and advocacy. NBCC increases funding for innovative breast cancer research; monitors how those funds are spent; expands access to quality health care for all; and ensures that trained advocates influence all decision making in breast cancer. Join NBCC, learn more and take action visit BreastCancerDeadline2020.org. 

About Hereditary Breast Cancer with BRCA Mutation

(C) Copyright 2013 GlobeNewswire, Inc. All rights reserved.