Blueprint Medicines and PatientCrossroads Launch Registry for Mastocytosis Patients to Increase Understanding of Disease and Speed Therapeutic Development

CAMBRIDGE, Mass. and SAN MATEO, Calif., Dec. 1, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, and PatientCrossroads, a pioneer in spearheading the adoption of patient-entered disease registries, today announced the launch of Mast Cell Connect, a patient registry to advance the understanding of mastocytosis and help speed the development of new therapies. Blueprint Medicines, which is developing a treatment for patients with systemic mastocytosis, is sponsoring the registry. PatientCrossroads' robust platform will be used to host and manage the registry, including patient privacy, data collection and access.

"We are proud to support the creation of Mast Cell Connect, the first-ever open-model registry to capture the perspective of patients living with mastocytosis," said Jeffrey Albers, Chief Executive Officer of Blueprint Medicines. "This new resource will bring Blueprint Medicines, researchers and the broader medical community greater insights about the needs of people living with the disease and enable patients to sign up for updates about clinical trials and other important developments."

Participation in Mast Cell Connect is voluntary and open to those diagnosed with mastocytosis, a rare disease in which immune cells known as mast cells abnormally build up in the skin, bone marrow and other parts of the body. The registry will include patients with all forms of the disease, including systemic mastocytosis, cutaneous mastocytosis and their subtypes. By participating in Mast Cell Connect, patients will be able to view de-identified perspectives from other patients living with the disease, and may consent to be notified of clinical trials and other research studies. Recruitment efforts for the registry will initially be focused in the United States, but Mast Cell Connect is open to patients from anywhere in the world.

"We have observed over the years how patient registries have made an impact on advancing clinical development for other rare diseases, like Duchenne Muscular Dystrophy and Friedreich's ataxia. These databases of detailed medical information have proven to be a valuable tool in better understanding such disease areas," said Valerie Slee, RN, BSN, Chair of the Board of Directors for The Mastocytosis Society (TMS), the U.S.-based patient advocacy organization for the disease. "Mast Cell Connect will galvanize patients to share information about their experiences with mastocytosis with the greater medical community."

"PatientCrossroads was founded on the philosophy that making patient registries available to disease communities and sharing this data via an open model will facilitate a new level of cross-collaboration, and ultimately, the development of new therapies," said Kyle Brown, Chief Executive Officer and Founder of PatientCrossroads. "We believe that Mast Cell Connect will contribute to better care for people with mastocytosis by systematically documenting the impact and natural history of this rare disease and increasing participation in clinical studies."

In addition to launching Mast Cell Connect, Blueprint Medicines today launched a disease awareness website called "Together with Systemic Mastocytosis." The goal of this website is to inform, empower and engage patients with systemic mastocytosis and their caregivers.

About Mastocytosis
Mastocytosis is a rare disease in which immune cells known as mast cells abnormally build up in the skin, bone marrow and other parts of the body. In healthy people, mast cells produce mediators, like histamine, that help activate the immune system and appropriately direct disease-fighting blood cells. In mastocytosis, however, the abnormal build-up of mast cells leads to high levels of these mediators, and can cause symptoms that resemble allergies, including hives, flushing, shortness of breath and anaphylactic shock. The signs, symptoms and severity of mastocytosis vary widely, but in more severe cases, mast-cell accumulation in the organs results in organ function impairment and shortened life span.

In patients with mastocytosis, mast cells can accumulate in the skin (known as cutaneous mastocytosis) and in other tissues including bone, bone marrow, liver, spleen and the gastrointestinal tract (known as systemic mastocytosis, or SM).

In almost all patients with SM, a genetic mutation known as KIT D816V is believed to be the root cause of the disease. The genetic mutation is not hereditary, and it is highly unusual for SM to run in families. Today, most treatments for SM only provide symptom relief and do not treat the underlying cause. There is no cure for the disease.

About Blueprint Medicines
Blueprint Medicines is developing a new generation of highly selective and potent kinase medicines to improve the lives of patients with genomically defined diseases. The Company's approach is rooted in a deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases. Blueprint Medicines is advancing three programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma and systemic mastocytosis, as well as multiple programs in research and preclinical development. For more information, please visit www.blueprintmedicines.com.

About PatientCrossroads
Since 2005, PatientCrossroads has pioneered the use of patient registries to accelerate therapeutic development and empower patients. PatientCrossroads registry programs connect patients with researchers, advocates and industry organizations working to understand or treat specific diseases and conditions. Dedicated to openly accessible registry programs, PatientCrossroads has been recognized for its efficacy as the recipient of an NIH Merit Group Award. PatientCrossroads also is the only full U.S. based partner in the European Union FP7 funded RD-CONNECT program, which supports the international goal of developing 200 therapeutics for rare diseases by 2020. For more information, visit http://www.patientcrossroads.com.

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 potential benefits of Mast Cell Connect to Blueprint Medicines, patients, researchers and the broader medical community. 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 delay of any current or planned clinical trials or the development of Blueprint Medicines' drug product candidates, including BLU-285 and BLU-554; Blueprint Medicines' advancement of multiple early-stage efforts, including its RET program; Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug product candidates; the preclinical and clinical results for Blueprint Medicines' drug product candidates, which may not support further development of such drug product candidates; and actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' Quarterly Report on Form 10-Q for the quarter ended September 30, 2015, as filed with the Securities and Exchange Commission (SEC) on November 9, 2015, and other filings that Blueprint Medicines 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. Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

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SOURCE Blueprint Medicines

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