Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review to the Company's supplemental New Drug Application (sNDA) for BRAFTOVI (encorafenib) in combination with ERBITUX (cetuximab) (BRAFTOVI Doublet) based on results from the Phase 3 BEACON CRC trial, which evaluated the efficacy and safety of BRAFTOVI in combination with ERBITUX with or without MEKTOVI (binimetinib) in patients with advanced BRAFV600E-mutant metastatic colorectal cancer (mCRC), following one or two lines of therapy.
As published in The New England Journal of Medicine (NEJM), results from the BEACON CRC trial showed improvements in overall survival (OS) and objective response rates (ORR) for both the BRAFTOVI Doublet and BRAFTOVI Triplet (BRAFTOVI, MEKTOVI and ERBITUX) combination, compared to ERBITUX plus irinotecan-containing regimens (Control).1 In descriptive analyses comparing the Doublet and Triplet arms, the results showed comparable efficacy between the Doublet and Triplet in the overall population. The BRAFTOVI Triplet and Doublet showed no unexpected toxicities.
'The FDA's acceptance of our application for the BRAFTOVI Doublet is highly encouraging news for patients with mCRC harboring a BRAFV600E mutation,' said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology, Pfizer Global Product Development. 'Currently, there are no FDA-approved treatments specifically for patients with BRAF-mutant mCRC who have received prior treatment. If approved, the BRAFTOVI Doublet would become the first targeted regimen for this patient population, who typically have a poor prognosis. We also look forward to continuing to explore this targeted Doublet regimen with or without MEKTOVI in earlier lines of BRAF-mutant mCRC, including in the ongoing, Phase 2 ANCHOR study in previously untreated patients.'
The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is in April 2020.
On November 2, 2019, the European Medicines Agency (EMA) also started the review procedure for Pierre Fabre's Type II variation applications based on the BEACON CRC trial.
About Colorectal Cancer
Worldwide, colorectal cancer is the third most common type of cancer in men and the second most common in women, with approximately 1.8 million new diagnoses in 2018.2,3 In the U.S. alone, an estimated 140,250 patients were diagnosed with cancer of the colon or rectum in 2018, and approximately 50,000 are estimated to die of their disease each year.4 BRAF mutations are estimated to occur in up to 15% of patients with mCRC and represent a poor prognosis for these patients.5,6,7,8,9,10 The V600 mutation is the most common BRAF mutation and the risk of mortality in CRC patients with the BRAFV600E mutation is more than two times higher than for those with wild-type BRAF.7,8 BRAFV600E-mutant mCRC is an area of high unmet need as there are currently no approved therapies specifically indicated for patients with BRAF-mutant mCRC.11,12, 13
About BEACON CRC
BEACON CRC is a randomized, open-label, global trial evaluating the efficacy and safety of BRAFTOVI, MEKTOVI and ERBITUX in patients with BRAFV600E-mutant mCRC whose disease has progressed after one or two prior regimens.
The randomized portion of the BEACON CRC trial is designed to assess the efficacy and safety of BRAFTOVI in combination with ERBITUX with or without MEKTOVI compared to ERBITUX and irinotecan-based therapy. 665 patients were randomized 1:1:1 to receive the Triplet combination, the Doublet combination (BRAFTOVI and ERBITUX) or the control arm (irinotecan-based therapy and ERBITUX). The study was amended to include an interim analysis of endpoints including ORR. The primary overall survival endpoint is a comparison of the Triplet combination to the control arm. Secondary endpoints address efficacy of the Doublet combination compared to the control arm, and the Triplet combination compared to the doublet therapy. Other secondary endpoints include progression-free survival, duration of response, safety and tolerability. The trial is being conducted at over 200 investigational sites in North America, South America, Europe and the Asia Pacific region. The BEACON CRC trial is being conducted with support from Ono Pharmaceutical Co. Ltd., Pierre Fabre and Merck KGaA, Darmstadt, Germany (support is for sites outside of North America).
About BRAFTOVI + MEKTOVI
BRAFTOVI is an oral small molecule BRAF kinase inhibitor and MEKTOVI is an oral small molecule MEK inhibitor which target key enzymes in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Inappropriate activation of proteins in this pathway has been shown to occur in many cancers including melanoma and colorectal cancer. In the U.S., BRAFTOVI + MEKTOVI are approved for the treatment of unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for treatment of patients with wild-type BRAF melanoma. In Europe, the combination is approved for adult patients with unresectable or metastatic melanoma with a BRAFV600 mutation, as detected by a validated test. In Japan, the combination is approved for unresectable melanoma with a BRAF mutation.
Pfizer has exclusive rights to BRAFTOVI and MEKTOVI in the U.S. and Canada. Ono Pharmaceutical Co. Ltd. has exclusive rights to commercialize both products in Japan and South Korea, Medison has exclusive rights to commercialize both products in Israel and Pierre Fabre has exclusive rights to commercialize both products in all other countries, including Europe, Latin America and Asia (excluding Japan and South Korea).
About Pfizer Oncology
At Pfizer Oncology, we are committed to advancing medicines wherever we believe we can make a meaningful difference in the lives of patients. Today, Pfizer Oncology has an industry-leading portfolio of 22 approved innovative cancer medicines and biosimilars across more than 30 indications, including breast, prostate, kidney and lung cancers, as well as leukemia and melanoma. Pfizer Oncology is striving to change the trajectory of cancer.
Pfizer Inc.: Breakthroughs that change patients' lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com.
DISCLOSURE NOTICE
The information contained in this release is as of Dec. 18, 2019. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about the BRAFTOVI (encorafenib) and ERBITUX (cetuximab) combination (BRAFTOVI Doublet) and a potential new indication for the treatment of advanced BRAFV600E-mutant metastatic colorectal cancer, following one or two lines of therapy, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of the BRAFTOVI Doublet; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications for the Doublet combination for the potential new indication may be filed with regulatory authorities in any other jurisdictions; whether and when the FDA and the European Medicines Agency will approve the pending applications for the potential new indication and whether and when regulatory authorities in any other jurisdictions may approve any such other applications that may be pending or filed, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether the Doublet combination for the potential new indication will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of BRAFTOVI, MEKTOVI or the Doublet Combination and competitive developments.
A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned 'Risk Factors' and 'Forward-Looking Information and Factors That May Affect Future Results', as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.
Contact:
Jessica Smith
Tel: (212) 733-6213
Email: Jessica.M.Smith@pfizer.com
PFIZER, INC. 
