Blueprint Medicines Highlights Advances in Preclinical Discovery Efforts
"We are excited about the ways in which we continue to leverage our novel library of kinase inhibitors to advance our drug discovery pipeline and target kinases as drivers of disease," said
Oncology—Preclinical Discovery Highlights
At the 9th Annual Conference of the
- The new preclinical data presented by
Blueprint Medicinesat ILCA further validate the advancement of BLU-554 into clinical development. BLU-554 is an exquisitely selective inhibitor of fibroblast growth factor receptor 4 (FGFR4). The new data show that BLU-554 demonstrated anti-tumor activity in preclinical models of HCC with aberrant FGFR4 signaling. Blueprint Medicinesis currently enrolling patients in a dose-escalation, Phase 1 clinical trial of BLU-554 for the treatment of advanced HCC and cholangiocarcinoma.
- In addition,
Blueprint Medicinescontinues to pursue research on a novel drug target for fibrolamellar hepatocellular carcinoma (FL-HCC). FL-HCC is a rare subtype of liver cancer that arises in adolescents and young adults. Preclinical data presented by Blueprint Medicinesat ILCA indicate that more than 90 percent of patients with FL-HCC have the DNAJB1-PRKACA fusion, which is likely a driver of FL-HCC. The new data highlight the role of PRKACA in FL-HCC and its potential as a novel target for therapeutic intervention.
In addition, utilizing its proprietary platform,
Rare Genetic Diseases—Preclinical Discovery Highlights
Continuing its focus on rare genetic diseases,
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
 Russo M, Misale S, Wei G et al. Acquired resistance to the TRK inhibitor entrectinib in colorectal cancer [published online
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