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MD Anderson and Blueprint Medicines Announce Strategic Collaboration to Accelerate BLU-222 Development

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Blueprint Medicines Corporation (BPMC) and MD Anderson Cancer Center announced a three-year strategic research collaboration to advance BLU-222, a precision therapy targeting cyclin-dependent kinase 2 (CDK2). The partnership aims to explore cancer types responsive to this selective CDK2 inhibitor, develop mono- and combination therapies, and identify biomarkers for treatment response. Blueprint Medicines plans to initiate a Phase 1 clinical trial of BLU-222 in patients with CCNE-aberrant cancers by mid-2022. MD Anderson will receive payments based on future development stages of BLU-222.

Positive
  • Collaboration with MD Anderson expected to enhance the clinical development of BLU-222.
  • Potential to identify novel biomarkers for better patient selection.
  • Plans for a Phase 1 clinical trial of BLU-222 in CCNE-aberrant cancers in H1 2022.
  • BLU-222 shows robust anti-tumor activity in preclinical models.
Negative
  • Dependence on future development payments from MD Anderson may impact financial planning.
  • Existing financial conflict of interest due to the relationship with MD Anderson.

HOUSTON and CAMBRIDGE, Mass., July 28, 2021 /PRNewswire/ -- The University of Texas MD Anderson Cancer Center and Blueprint Medicines Corporation (NASDAQ: BPMC) today announced a three-year strategic research collaboration focused on accelerating development of BLU-222, an investigational precision therapy designed to target cyclin-dependent kinase 2 (CDK2).

The collaboration brings together MD Anderson translational research scientists, the drug development capabilities of MD Anderson's Therapeutics Discovery division and Blueprint Medicines' precision therapy pipeline and expertise. The teams seek to characterize the range of cancer types susceptible to treatment with a selective CDK2 inhibitor, advance BLU-222 mono- and combination-therapy strategies with the potential to maximize patient benefit, and identify novel biomarkers that may better predict treatment response and optimize patient selection.

"This collaboration highlights our commitment to rapidly advance innovative science and builds on our prior efforts – also supported by MD Anderson investigators – that led to two FDA-approved and breakthrough therapy-designated precision therapies for patients with cancer," said Fouad Namouni, M.D., President of Research and Development at Blueprint Medicines. "By leveraging the power of MD Anderson's expertise in translational research, we aim to reveal the broad potential of BLU-222 and optimize our clinical development strategy to bring treatment innovation to as many patients as possible."

Under the preclinical collaboration agreement, Blueprint Medicines expects to collaborate primarily with MD Anderson's Translational Research to Advance Therapeutics and Innovation in Oncology (TRACTION) platform, which leads cutting-edge translational biology research to rapidly position new therapies for clinical trials. TRACTION is a core component of MD Anderson's Therapeutics Discovery division, an integrated team of clinicians, researchers and drug development experts working to advance impactful therapies that address patient needs.

MD Anderson and Blueprint Medicines will jointly design translational studies, and Blueprint Medicines will provide funding, research compounds and additional support. As part of the agreement, MD Anderson is eligible to receive certain payments based on the future development of BLU-222.

"As a critical regulator of the cell cycle, CDK2 is an exciting therapeutic target in oncology with broad potential across multiple patient populations and treatment settings," said Timothy Heffernan, Ph.D., executive director of TRACTION and head of oncology research in MD Anderson's Therapeutics Discovery division. "Through our collaboration with Blueprint Medicines, we aim to improve the scientific understanding of the role of CDK2 inhibition in cancer treatment and advance a new therapeutic option for patients with difficult-to-treat tumors, which further demonstrates our commitment to work at the forefront of cancer research."

Cyclin-dependent kinases and their cyclin partners regulate the cell cycle, which is the process of cell growth and division. In certain cancers, aberrant cyclin E (CCNE) hyperactivates CDK2, resulting in cell cycle dysregulation and tumor proliferation. Aberrant CCNE has been observed as a primary driver of disease in subsets of patients across a wide range of tumor types and has been shown to promote resistance to CDK4/6 inhibitors and other therapies. BLU-222 is a potential best-in-class CDK2 inhibitor that has shown robust anti-tumor activity in preclinical models of CCNE-aberrant ovarian, breast and gastric cancer. Blueprint Medicines plans to initiate a first-in-human Phase 1 clinical trial of BLU-222 in patients with CCNE-aberrant cancers in the first half of 2022.

Disclosures

MD Anderson's relationship with Blueprint Medicines creates a financial conflict of interest, and therefore MD Anderson is implementing an Institutional Conflict of Interest Management and Monitoring Plan for this research.

About MD Anderson

The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institution's sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 51 comprehensive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is No.1 for cancer in U.S. News & World Report's "Best Hospitals" rankings. It has been named one of the nation's top two hospitals for cancer since the rankings began in 1990 and has ranked first 16 times in the last 19 years. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

About Blueprint Medicines

Blueprint Medicines is a global precision therapy company that invents life-changing medicines for people with cancer and hematologic disorders. Applying an approach that is both precise and agile, we create therapies that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering our approved medicines to patients in the United States and Europe, and we are globally advancing multiple programs for genomically defined cancers, systemic mastocytosis, and cancer immunotherapy. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the research collaboration between Blueprint Medicines and The University of Texas MD Anderson Cancer Center; plans and timelines for the research and development of BLU-222; the potential benefits of BLU-222 in treating patients; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the impact of the COVID-19 pandemic to Blueprint Medicines' business, operations, strategy, goals and anticipated milestones, including Blueprint Medicines' ongoing and planned research and discovery activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Blueprint Medicines' ability and plans in continuing to establish and maintain a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; Blueprint Medicines' ability to successfully expand the approved indications for AYVAKITTM /AYVAKYT® (avapritinib) and GAVRETO® (pralsetinib) or obtain marketing approval for AYVAKIT/AYVAKYT and GAVRETO in additional geographies in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for AYVAKIT/AYVAKYT, GAVRETO or any drug candidates it is developing; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for AYVAKIT/AYVAKYT, GAVRETO or any of its current and future drug candidates; and the success of Blueprint Medicines' current and future collaborations, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

Trademarks

Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.

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SOURCE Blueprint Medicines Corporation

FAQ

What is the purpose of the collaboration between Blueprint Medicines and MD Anderson?

The collaboration aims to accelerate the development of BLU-222, focusing on cancer types that may respond to CDK2 inhibition.

When is the Phase 1 clinical trial for BLU-222 expected to start?

Blueprint Medicines plans to initiate the Phase 1 clinical trial of BLU-222 in the first half of 2022.

What is BLU-222 designed to target?

BLU-222 is an investigational precision therapy designed to target cyclin-dependent kinase 2 (CDK2).

What are the anticipated benefits of BLU-222?

BLU-222 has shown robust anti-tumor activity in preclinical models and aims to optimize treatment for patients with CCNE-aberrant cancers.

What financial implications does the collaboration have for Blueprint Medicines?

MD Anderson is eligible for payments based on the future development of BLU-222, which may influence Blueprint Medicines' financial planning.

Blueprint Medicines Corporation

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