Blueprint Medicines Announces New Data from Ongoing Phase 1 Clinical Trial of BLU-554 in Patients with Advanced Hepatocellular Carcinoma
"Patients with hepatocellular carcinoma face a very poor prognosis with few therapeutic options," said
"We are encouraged by the updated BLU-554 Phase 1 data presented at ESMO, which build on our prior clinical experience and suggest that BLU-554 may offer meaningful benefit to patients with FGFR4-driven HCC," said
Updated Data from the Ongoing Phase 1 Clinical Trial
BLU-554 is currently being evaluated in a Phase 1 clinical trial in patients with advanced HCC. Following the completion of the dose escalation portion of the trial and determination of the maximum tolerated dose (MTD) of 600 mg once daily (QD),
As of the data cutoff of
Pharmacokinetic (PK) analysis demonstrated rapid oral absorption across all dose levels, with a mean half-life of approximately 17 hours and exposure in the expected therapeutic range based on HCC xenograft models. Collectively, these data support a once-daily dosing regimen.
Safety Data
As of the data cutoff of
Clinical Activity Data
As of the data cutoff of
In patients with FGFR4-driven HCC (n=38), the data showed an ORR of 16 percent (95 percent confidence interval 6-31 percent). In addition, 49 percent of patients had radiographic tumor reduction, and clinical activity was observed regardless of disease etiology or geography. As of the data cutoff date:
- One patient had an unconfirmed complete response.
- Five patients had a partial response, with four confirmed and one unconfirmed.
- An additional 20 patients had stable disease, representing a disease control rate of 68 percent.
- No responses were observed in patients without FGFR4 pathway activation (n=29).
Among all 77 patients treated with BLU-554, 19 remained on treatment as of the data cutoff date, including 15 patients with FGFR4-driven HCC. Median progression free survival was 3.7 months among patients with FGFR4-driven HCC.
In addition, five TKI-naïve patients with FGFR4-driven HCC were evaluable for response assessment as of the data cutoff date. Within this group, preliminary evidence of prolonged disease control was observed. Two TKI-naïve patients remain on treatment as of the data cutoff with a duration of treatment of 11.4 months and 12.3 months, respectively.
Clinical Development Plans for BLU-554
Conference Call Information
About the Phase 1 Clinical Trial for BLU-554 in Advanced HCC
About HCC
Liver cancer is the second leading cause of cancer-related deaths worldwide, with HCC accounting for most liver cancers. In the
About BLU-554
BLU-554 is an orally available, potent, irreversible inhibitor of FGFR4 discovered and being developed by
About
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 four programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, 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, including plans and timelines for clinical trials evaluating BLU-554 in TKI-naïve patients with FGFR4-driven HCC or BLU-554 in combination with an immune checkpoint inhibitor;
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Media and Investor Relations Contacts: Kristin Hodous, 617-714-6674, KHodous@blueprintmedicines.com; Jim Baker, 617-844-8236, JBaker@blueprintmedicines.com