Blueprint Medicines Announces Proof-of-Concept Data from Global Phase 1 Clinical Trial of BLU-554 in Patients with Advanced Hepatocellular Carcinoma
"We desperately need new treatment options for patients with liver cancer, the second leading cause of cancer deaths worldwide," said
"We are very encouraged to see early anti-tumor activity in patients with confirmed FGF19 overexpression and consistent evidence of FGFR4 pathway modulation during the dose escalation part of this first-in-human clinical trial," said
Data from the Ongoing Global Phase 1 Clinical Trial
BLU-554 was evaluated in the dose escalation stage of a Phase 1 clinical trial in patients with advanced HCC. As of the data cutoff date of
Pharmacokinetic (PK) data across all dose levels showed rapid oral absorption, a mean half-life of approximately ten hours, and exposure in the expected therapeutic range based on HCC xenograft models.
Pharmacodynamic (PD) data demonstrated FGFR4 pathway inhibition with BLU-554, as evidenced by effects on metabolic pathways downstream of FGFR4, with increases in the bile acid precursor C4, decreases in cholesterol, and feedback upregulation of the ligand FGF19 in blood.
Preliminary Safety Data
As of the data cutoff date of
Preliminary Clinical Activity Data
As of the data cutoff date of
- One patient had a confirmed partial response (PR), and remained on the clinical trial for eight 28-day dosing cycles. Twelve patients had stable disease (SD), including seven who had tumor reduction but did not reach the threshold for a PR.
- Of ten evaluable patients with FGF19 overexpression in their tumors, five had radiographic tumor reduction, including one patient with a confirmed PR. Seven of the ten patients with FGF19 overexpression remain on treatment as of the data cutoff.
- Among all 25 evaluable patients, seven patients remain on treatment as of the data cutoff, with a duration of treatment ranging from 0.8 to 7.6 months.
- Eighteen patients discontinued treatment with BLU-554, including 15 patients due to disease progression, two patients due to treatment-related AEs and one patient due to the investigator's decision.
Clinical Development Plans for BLU-554
Conference Call Information
To participate in the conference call, please dial 1-855-728-4793 (domestic) or 1-503-343-6666 (international) and refer to conference ID 10770449. A live webcast of the presentation will also be available under "Events and Presentations" in the Investors section of
About the Global Phase 1 Clinical Trial for BLU-554 in Advanced HCC
Liver cancer is the second leading cause of cancer-related deaths worldwide, with HCC accounting for most liver cancers. In
BLU‑554 is an orally available, potent, highly selective and irreversible inhibitor of the kinase FGFR4, while sparing the other three FGFR paralogs.
Blueprint Medicines is developing a new generation of targeted 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. For more information, please visit www.blueprintmedicines.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 plans and timelines for the clinical development of BLU-554; our ability to implement our clinical development plans for BLU-554 for the treatment of advanced HCC; our ability to enroll patients in our ongoing Phase 1 clinical trial for BLU-554 in advanced HCC; and
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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