Blueprint Medicines Announces Proof-of-Concept Data from Phase 1 Clinical Trial of BLU-285 in Patients with Advanced Systemic Mastocytosis
"Advanced systemic mastocytosis is a rare and severe disease that shortens life expectancy with a wide range of debilitating symptoms and organ damage," said
"We are excited by these early data demonstrating a favorable safety profile as well as signs of clinical activity starting at the first dose level," said
Data from the Ongoing Phase 1 Clinical Trial
BLU-285 is currently being evaluated in the dose escalation portion of a Phase 1 clinical trial in patients with advanced SM. As of the data cutoff date of
Consistent with preliminary data reported by
Preliminary Safety Data
As of the data cutoff date of
Preliminary Clinical Activity Data
As of the data cutoff date of
- Investigators observed decreases in bone marrow mast cell infiltrate (measured by bone marrow biopsy) in six of the eight patients who had a bone marrow biopsy after starting treatment with BLU-285. Three of the six patients had a decrease of bone marrow mast cell infiltrate of more than 50% from baseline, including one patient with no residual mast cells in the bone marrow.
- Based on measurements at a central laboratory, serum tryptase decreased in ten of 12 patients. The serum tryptase decrease was greater than 50% in eight patients.
- The allele burden of D816V mutant KIT decreased within the first two treatment cycles in five of six patients in circulating tumor DNA and bone marrow.
- Rash improved in five patients with urticaria pigmentosa from baseline based on investigator assessments. Urticaria pigmentosa is an allergy-mediated rash common in SM patients.
- Weight increased in ten patients and albumin increased in 11 patients, suggesting improvements in malabsorption.
- Ten of 12 patients remained on treatment with treatment duration ranging from 1 month to 8.1 months.
Clinical Development Plans for BLU-285 in SM
Based on the favorable safety profile and encouraging clinical activity observed to date in the Phase 1 clinical trial for BLU-285 for the treatment of advanced SM,
Conference Call Information
About the Phase 1 Clinical Trial for BLU-285 in Advanced SM
There are several forms of SM, including indolent SM and more advanced forms of SM, which include aggressive SM, SM-AHN and MCL. SM is characterized by the buildup of mast cells, which are immune cells that produce histamine and other mediators of the body's inflammatory and allergic responses. In patients with SM, mast cells release high levels of these mediators, causing symptoms that range from mild to life-threatening symptoms, including pain, nausea, rash, fever, fatigue and anaphylaxis. In patients with advanced SM, including aggressive SM, SM-AHN and MCL, mast cell infiltration in bone marrow, liver and other vital organs can eventually lead to organ dysfunction and lower life expectancy, with a median overall survival of approximately four years or less. Patients with indolent SM do not have a lower life expectancy, but they do suffer from a broad range of acute and chronic symptoms that can have a significant impact on their quality of life. There are no approved treatments that target the KIT D816V mutation, which is the primary driver of disease in approximately 90 to 95 percent of SM patients, and there is a clear need for more effective therapies for patients with advanced SM and for patients with indolent SM who have a heavy symptom burden.
BLU-285 is an orally available, potent and highly selective inhibitor of D816V mutant KIT.
Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases. Its 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.
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 plans and timelines for the clinical development of BLU-285;
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Investor Relations: Kristin Williams, Blueprint Medicines Corporation, 617-714-6674, firstname.lastname@example.org; Media Relations: Rachel Hutman, W2O Group, 301-801-5540, email@example.com