Blueprint Medicines Corp. (NASDAQ: BPMC) watched its shares make a solid gain on Monday morning after the firm announced results for its ongoing early-stage trial for advanced systemic mastocytosis (SM). Specifically, the company released new data from its Phase 1 clinical trial of avapritinib (formerly known as BLU-285).

This data will be presented as part of 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, Georgia.

Ultimately, this new data from the dose escalation portion of the Phase 1 trial showed strong clinical activity regardless of advanced SM subtype, prior treatment with midostaurin or the presence of additional mutations.

What stands out here is that the data showed an overall response rate of 72% and a disease control rate of 100% in patients evaluable for response. As of the data cutoff date (October 4), avapritinib was well-tolerated and most adverse events reported by investigators were Grade 1 or 2. In addition, there were no discontinuations due to treatment-related adverse events.

Daniel J. DeAngelo, M.D., Ph.D., Director of Clinical and Translational Research, Adult Leukemia at Dana-Farber Cancer Institute, Associate Professor of Medicine at Harvard Medical School, and an investigator on the Phase 1 trial, commented:

The new clinical trial results for avapritinib reported at the ASH plenary scientific session represent an exciting milestone for the systemic mastocytosis community. The data showed a remarkably high response rate and a favorable tolerability profile, suggesting avapritinib has the potential to transform the treatment of this devastating rare disease. The data also provide strong evidence that selective inhibition of D816V mutant KIT, a disease-driver present in nearly all patients across the spectrum of SM, is a potentially important treatment strategy that may offer patients improved outcomes. These data strongly support continued development of avapritinib in a broad population of patients with SM.

Shares of Blueprint traded up about 26% to $90.61 early Monday, with a consensus analyst price target of $79.75 and a 52-week range of $25.34 to $92.00.

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