Blueprint Medicines Publishes Strategy for Advancing Kinase Drug Discovery

CAMBRIDGE, Mass., May 11, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC) today announced that the Journal of Clinical Investigation (JCI) published an overview of the Company's kinase drug discovery and development strategy. The publication underscores Blueprint Medicines' focus on identifying novel genomically defined disease drivers and the use of novel chemistry from its proprietary compound library to craft highly selective kinase inhibitors for new and difficult-to-drug kinase targets. "Targeting cancer with kinase inhibitors," authored by Blueprint Medicines' scientists, was published in the May 2015 issue of JCI, a leading journal focused on science and clinical research to advance medicine.

"Despite the success of kinase inhibitors in treating cancer, approved drugs focus on less than 5 percent of all kinases and the function of most kinases remains unknown," said Sir Philip Cohen, PhD, Professor of Enzymology, Medical Research Council Protein Phosphorylation and Ubiquitylation Unit, University of Dundee, Scotland. "There is a tremendous need and opportunity to further our understanding of kinase biology, identify novel disease drivers and design highly targeted therapies. Blueprint Medicines' approach of using novel chemistry to target newly discovered genomic drivers of cancer as well as known drivers that have been historically difficult to drug has the potential to greatly improve the lives of patients who currently have no or limited treatment options."

The JCI review article describes how the abnormal activation of kinases drives many hallmarks of tumor biology. Kinases are proven cancer drug targets, but progress in recent years has been largely incremental. In the article, Blueprint Medicines scientists call attention to important opportunities for advancing the field that serve as the foundation for the Company's drug discovery and development strategy:

• Uncovering novel disease drivers and drug targets: New genomics and sequencing approaches are needed to elucidate the biology of the full kinome, which is comprised of 518 kinases, and to uncover the function of kinases of unknown biology (or KUBs) and their potential for drug discovery.
• Crafting highly selective kinase inhibitors: Novel chemical matter and structure-informed design are needed to create more selective and potent therapies that can inhibit new and difficult-to-drug kinase targets. Complete target inhibition with minimal off-target effects is also needed to provide more durable responses with few toxicities, improving outcomes for patients.
• Predicting resistance mutations: Novel approaches in structural biology and computational chemistry are needed to predict future resistance mutations which arise from targeted therapies. Novel chemistry is needed to design a single therapy that can target both the original primary disease driver and the subsequent mutations that cause patients to become refractory to treatment.
• Developing novel combination therapies: Combinations of highly targeted kinase inhibitors acting within a single kinase pathway or between parallel kinase pathways are needed to mount a multi-pronged attack that improves efficacy and delays the onset of treatment resistance.

"We're entering a new era of precision medicine, in which we can craft highly selective kinase inhibitors against previously unaddressed drivers of disease and identify patients who are most likely to respond based on the molecular profile of their cancers," said Christoph Lengauer, PhD, MBA, Blueprint Medicines Chief Scientific Officer. "The strategies we describe in the JCI paper are at the heart of our drug discovery and development efforts. We believe that our ability to identify new drug targets, coupled with our proprietary library of compounds, will enable us to uniquely fulfill our mission of delivering targeted medicines for patients with genomically defined diseases."

Blueprint Medicines expects to advance its lead programs into Phase 1 clinical trials in mid-2015: BLU-554, a selective inhibitor of fibroblast growth factor receptor 4 (FGFR4), in hepatocellular carcinoma, and BLU-285, a selective inhibitor of PDGFRα D842V and KIT Exon 17 mutants, in gastrointestinal stromal tumors and systemic mastocytosis. The company also has a drug discovery program targeting RET kinase fusions and predicted resistance mutations.

About Blueprint Medicines 
Blueprint Medicines makes kinase drugs to treat patients with genomically defined diseases. Led by a team of industry innovators, Blueprint Medicines integrates a novel target discovery engine and a proprietary compound library to understand the blueprint of cancer and craft highly selective therapies. This empowers the Blueprint Medicines team to develop patient-defined medicines aimed at eradicating cancer.

Forward-Looking Statements

Various statements in this release concerning Blueprint Medicines' future expectations, plans and prospects, including without limitation, statements regarding the foundation for Blueprint Medicines' drug discovery and development strategy, expectations regarding Blueprint Medicines' ability to craft highly selective kinase inhibitors and deliver targeted medicines for patients with genomically defined cancers, Blueprint Medicines' expectations regarding BLU-554 as a treatment for hepatocellular carcinoma, Blueprint Medicines' expectations regarding BLU-285 as a treatment for gastrointestinal stromal tumors and systemic mastocytosis, statements concerning the timing of  planned Phase 1 clinical trials for BLU-554 and BLU-285, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. In particular, it should be noted that the strategies described in the JCI paper are preliminary and interim in nature; the Phase 1 clinical trials for BLU-554 and BLU-285 have not yet commenced. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties.  Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, the risk of delay of any planned clinical trials and/or development of Blueprint Medicines' drug product candidates,  Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug product candidates, the pre-clinical and clinical results for its drug product candidates, which may not support further development of such drug product candidates, the risk that Blueprint Medicines' collaboration with Alexion Pharma Holdings will not continue or will not be successful, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Blueprint Medicines' ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, competition from others developing products for similar uses, Blueprint Medicines' ability to manage operating expenses, Blueprint Medicines' ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Blueprint Medicines' dependence on third parties for various functions, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in  the final prospectus related to Blueprint Medicines' initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act, as well as discussions of potential risks, uncertainties, and other important factors in Blueprint Medicines' subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Blueprint Medicines' views only as of today and should not be relied upon as representing its views as of any subsequent date. Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

CONTACT:

Investor Relations: 
Beth DelGiacco
Stern Investor Relations, Inc.
212-362-1200
[email protected]

Media Relations: 
Beth Keshishian
inVentiv Health PR
212-229-8417
[email protected]

SOURCE Blueprint Medicines

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