Blueprint Medicines Corp (NASDAQ:BPMC), 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 inVancouver, 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.”

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.

“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. (Original Source)

Shares of Blueprint Medicines closed yesterday at $28.04, down $0.19 or -0.67%. BPMC has a 1-year high of $31.94 and a 1-year low of $13.04. The stock’s 50-day moving average is $24.77 and its 200-day moving average is $19.80.

On the ratings front, Blueprint has been the subject of a number of recent research reports. In a report issued on August 9, Cowen analyst Eric Schmidt reiterated a Buy rating on BPMC. Separately, on the same day, Canaccord Genuity’s Arlinda Lee reiterated a Buy rating on the stock and has a price target of $26.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Eric Schmidt and Arlinda Lee have a total average return of 22.5% and -17.1% respectively. Schmidt has a success rate of 54.0% and is ranked #71 out of 4147 analysts, while Lee has a success rate of 30.5% and is ranked #4020.

Blueprint Medicines Corp. is a biopharmaceutical company, which engages in developing of kinase inhibitors for genomically defined subsets of cancer patients. It has developed a platform that combines genomics with a novel library of kinase inhibitors, enabling the development of potent highly selective compounds against clear genomic driver targets.