Blueprint Medicines Corp (NASDAQ:BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, today provided an update on certain of its preclinical discovery efforts in oncology and rare genetic diseases, in addition to reporting its financial results for the third quarter ended September 30, 2015.

“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 Christoph Lengauer, Ph.D., Chief Scientific Officer of Blueprint Medicines. “During the third quarter of 2015, we presented preclinical data validating the advancement of BLU-554 into clinical development and identifying PRKACA as a novel target for therapeutic intervention. In addition, we have predicted resistance mutations in NTRK, some of which have recently been clinically observed by others, and we are advancing compounds that inhibit NTRK and resistant mutants into preclinical safety studies. We have also entered into a collaboration with theChildren’s Hospital of Eastern Ontario seeking to identify new therapeutic opportunities for rare genetic diseases.”

Oncology—Preclinical Discovery Highlights

At the 9th Annual Conference of the International Liver Cancer Association (ILCA) in September, Blueprint Medicines presented new preclinical data on BLU-554, one ofBlueprint Medicines’ lead drug candidates, and DNAJB1-PRKACA, a recurrent fusion in certain hepatocellular carcinoma (HCC) patients.

  • The new preclinical data presented by Blueprint Medicines at 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 Medicines is 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 Medicines continues 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 Medicines at 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, Blueprint Medicines has predicted resistance mutations in the neurotrophic tyrosine receptor kinase (NTRK).  Recently, some of these mutations were clinically observed by others in tumors that developed resistance after initially responding to an NTRK inhibitor.[1] Blueprint Medicines is advancing compounds that inhibit NTRK and resistant mutants into preclinical safety studies. Research published by Blueprint Medicines scientists in the September 10, 2014 edition of Nature Communications (Stransky N. et al., The landscape of kinase fusions in cancer) suggests NTRK  fusion events may drive tumorigenesis in multiple cancers, with a total of 23 NTRK1, NTRK2 and NTRK3 fusions identified across nine tumor types.

Rare Genetic Diseases—Preclinical Discovery Highlights

Continuing its focus on rare genetic diseases, Blueprint Medicines is currently collaborating with the Children’s Hospital of Eastern Ontario (CHEO). Blueprint Medicines and CHEO are seeking to identify potential therapeutic opportunities for rare genetic diseases utilizing Blueprint Medicines’ proprietary compound library, which will allow Blueprint Medicines to initiate development of future programs in rare genetic diseases. (Original Source)

Shares of Blueprint Medicines closed today at $22.82, up $0.50 or 2.24%. BPMC has a 1-year high of $37.17 and a 1-year low of $18. The stock’s 50-day moving average is $22.09 and its 200-day moving average is $26.67.

On the ratings front, Blueprint Medicines has been the subject of a number of recent research reports. In a report issued on July 14, Wedbush analyst David Nierengarten reiterated a Buy rating on BPMC, with a price target of $41, which implies an upside of 84.8% from current levels. Separately, on July 13, Cowen’s Eric Schmidt reiterated a Buy rating on the stock .

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, David Nierengarten and Eric Schmidt have a total average return of 47.0% and 31.0% respectively. Nierengarten has a success rate of 50.5% and is ranked #14 out of 3829 analysts, while Schmidt has a success rate of 61.5% and is ranked #37.

Blueprint Medicines Corp is a biopharmaceutical company. It is focused on improving the lives of patients with genomically defined diseases driven by abnormal kinase activation.