New drug search for fibrolamellar carcinoma announced

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Blueprint Medicines Corporation, of Cambridge, Mass.,  a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, today disclosed a new drug discovery program targeting protein kinase cAMP-activated catalytic subunit alpha (PRKACA) fusions for the treatment of fibrolamellar carcinoma (FLC). The announcement was made during an oral presentation on September 8, 2016 at the 10th ILCA Annual Conference in Vancouver, Canada on Blueprint Medicines’ efforts to develop targeted therapies for patients with hepatocellular carcinoma.

“Patients with liver cancers have historically suffered from a lack of genomically defined therapeutic options. Our new drug discovery program represents our commitment to patients with orphan cancers,” said Christoph Lengauer, Chief Scientific Officer of Blueprint Medicines. “The addition of this program is a testament to the strength of our scientists and genomics platform, as well as to the quality of our unique library of kinase inhibitors and continued momentum of our discovery efforts.”

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Distinct type of liver cancer

FLC is a rare and distinct subtype of liver cancer that typically arises in young adults. Currently, there are no approved therapies, and surgery is the only available treatment option for some patients, but most patients inevitably progress. Research published in 2014 by the laboratory of Dr. Sanford Simon, Professor and Head of the Laboratory of Cellular Biophysics at The Rockefeller University in New York in Science (Honeyman J.N. et al., Detection of a recurrent DNAJB1-PRKACA chimeric transcript in fibrolamellar hepatocellular carcinoma) and Blueprint Medicines in Nature Communications (Stransky N. et al., The landscape of kinase fusions in cancer) identified PRKACA kinase fusions in FLC. Blueprint Medicines estimates that more than ninety percent of patients with FLC harbor the PRKACA fusion, which is the only known recurrent genomic event in FLC and is considered to be the driver gene of the disease. Given the high medical need and the opportunity for a potentially transformative therapeutic impact, Blueprint Medicines is developing drug candidates for the selective inhibition of PRKACA.

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“FLC is a devastating disease, and patients are in great need of effective therapeutic options,” said Dr. Sanford Simon. “Targeting PRKACA represents a promising opportunity to develop innovative therapeutics that could make a difference in these patients’ lives.”

The unveiling of the PRKACA program expands Blueprint Medicines’ existing pipeline, which includes Phase 1 clinical programs for its drug candidates BLU-554, a selective inhibitor of FGFR4 for patients with hepatocellular carcinoma, and BLU-285, a selective inhibitor of both exon 17 mutant KIT and D842V mutant PDGFRα for patients with advanced systemic mastocytosis and unresectable, treatment-resistant gastrointestinal stromal tumors.  Blueprint Medicines expects to report preliminary data from the dose escalation portion of each of these Phase 1 clinical trials by the end of 2016.  Blueprint Medicines’ existing pipeline also includes preclinical programs for its drug candidate BLU-667, a selective inhibitor of RET activating mutations, fusions and predicted resistant mutants found in non-small cell lung cancer and thyroid cancer, a rare genetic disease program in collaboration with Alexion Pharma Holding and cancer immunotherapy programs in collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. – PRNewswire.

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