Targeting the future of medicine

Our commitment to patients

At Blueprint Medicines, we work together in pursuit of one common goal: to dramatically improve the lives of people with genomically defined cancers and rare diseases.

We combine a deep understanding of the genetic blueprint of cancer and rare diseases with expertise in kinase biology to craft precision therapies specifically designed to target the underlying molecular basis of disease. With this approach, we aim to develop breakthrough medicines that allow patients to live longer, healthier lives.

patients-graphic
“Though deep scientific knowledge forms the basis of our company, we go beyond intellectual curiosity to achieve a common goal: to allow patients with genomically defined cancers and rare diseases to live longer, healthier lives.”
Andy Boral, M.D., Ph.D., Chief Medical Officer

Our precision therapy approach

Our goal is to rapidly and reproducibly design precision therapies that treat the underlying cause of genomically defined cancers and rare diseases. With this approach, we aim to deliver important new medicines to patients in need.

Identify

a genomic driver of disease

Design a highly selective therapy

to target the genomic driver

Conduct clinical trials in patients

harboring the genomic driver

Diseases we target

Gastrointestinal stromal tumor

  • Gastrointestinal stromal tumor, or GIST, accounts for the most common sarcoma of the gastrointestinal tract, or GI tract. Tumors arise within cells in the GI tract wall, most often the stomach or small intestine.
  • Most patients are diagnosed between ages 50-80, typically triggered by GI bleeding, incidental findings during surgery or imaging, or in rare cases, tumor rupture or GI obstruction. Symptoms include pain or discomfort in the abdomen, nausea, vomiting, anemia and fatigue. Patients diagnosed at an early stage may undergo surgery.
  • Although treatment options have improved in recent years for patients with metastatic disease, a meaningful percentage either do not respond to treatment or relapse afterwards, and for these patients there is a vital need for effective treatments.

Systemic mastocytosis

  • Systemic mastocytosis (SM) is characterized by the abnormal buildup of mast cells – immune cells that produce histamine and other mediators of the body’s inflammatory and allergic responses. In patients with SM, mast cells produce high levels of these mediators, causing symptoms that range from mild to life-threatening, including pain, nausea, hives, bleeding, fever and anaphylaxis.
  • There are several forms of SM including indolent SM and advanced SM. Many patients with indolent SM have a high symptom burden that severely impacts activities of daily living and quality of life. In advanced cases, including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia, mast cells accumulate in organs such as the bone marrow, spleen and liver, often resulting in life-threatening organ dysfunction.
  • There are no approved treatments designed to selectively target KIT D816V, the primary disease driver in most patients with SM, and there is a clear need for new therapies.

RET-altered non-small cell lung cancer, medullary thyroid cancer and other solid tumors

  • RET is a protein kinase involved in important cell functions including organ development and tissue stability. Genetic alterations and mutations in RET have been shown to cause non-small cell lung cancer and medullary thyroid cancer (MTC), as well as other solid tumors including colon and breast cancers.
  • Currently, no approved therapies selectively target RET-driven cancers. There is a need for precision therapies that provide durable clinical benefit by selectively targeting RET alterations and resistance mutations.

Hepatocellular carcinoma

  • Liver cancer (mainly hepatocellular carcinoma, or HCC) is the second leading cause of cancer-related deaths worldwide and the fastest rising cause of cancer-related death in the United States.
  • The incidence of HCC has tripled over the last twenty years, while the five-year survival rate remains below 12%.
  • While patients diagnosed at an early stage can receive potentially curative liver transplants, surgery or ablation therapies, patients with advanced HCC face a poor prognosis; more treatment options are needed for these patients.

Fibrodysplasia ossificans progressiva

  • Fibrodysplasia ossificans progressiva (FOP) is a rare, severely disabling and ultimately life-shortening genetic disease characterized by episodic soft tissue edema (flares) and the progressive replacement of skeletal muscle, ligaments and connective tissue by heterotopic bone.
  • Most cases result from new mutations in the ACVR1 gene, whereas a small number of people inherit a mutation. There are no approved treatments to slow or stop the progression of FOP.
Our clinical trials

Our clinical trials

Clinical trials are an important part of helping to bring safe and effective medicines to patients. All drugs go through rigorous series of trials, and based on the results, regulatory agencies including the US Food and Drug Administration and European Medicines Agency decide whether to approve medicines.

By taking part in clinical trials, patients can make an important contribution to ongoing research and better understanding of a disease. For patients who lack effective treatments, clinical trials are also an opportunity to be a part of testing a new drug.

Learn more about participating in clinical trials
Explore our clinical trials

Patient community events

We host informational webinars for patients, caregivers and families in each of the above disease areas. Read on to find archived events of interest.

Informational Webcast on Avapritinib Clinical Trial Program in Systemic Mastocytosis (SM)

On Tuesday, September 18, 2018, The Mastocytosis Society and Blueprint Medicines hosted a webinar for the systemic mastocytosis (SM) community about avapritinib (also known as BLU-285), an investigational medicine for people with SM.

Dr. Andy Boral, Chief Medical Officer at Blueprint Medicines, shared an update on recently presented clinical results, as well as plans for future studies in advanced, indolent and smoldering forms of SM. A replay is available by clicking below:

Patients who are interested in planned studies can contact the Blueprint Medicines study director at studydirector@blueprintmedicines.com or 1-617-714-6707 to learn more about opportunities to participate.

Additional information about the PATHFINDER trial for people with advanced SM is available
at pathfindertrial.com or clinicaltrials.gov/ct2/show/NCT03580655.

Additional information about the PIONEER trial for people with indolent and smoldering SM is available at
pioneertrial.com or clinicaltrials.gov/ct2/show/NCT03731260.