Blueprint Medicines $BPMC jumped onto the radar at ASH on Sunday, outlining some remarkable data on their lead drug as the biotech lays the foundation for a registration program with the FDA. The Cambridge, MA-based biotech, which went public in 2015 and has a market cap that’s grown to $2.8 billion, definitely wants to get the word out.
Investigators for the company reported that their lead drug — avapritinib, formerly known as BLU-285 — hit an impressive 72% overall response rate for rare cases of advanced systemic mastocytosis and a 100% disease control rate.
Blueprint shares shot up 26% in pre-market trading.
Researchers gather a group of 32 patients with this disease — characterized by the proliferation of mast cells in tissue and organs. And after treating them with their drug, a KIT and PDGFRα inhibitor, they recorded a greater than 50% drop in serum tryptase in all patients, which is a key biomarker for the disease.
The patients with radiographic scans demonstrated a decrease in spleen volume. And the 25 patients with bone marrow mast cell burden? In that group, 21 patients had at least a 50% decrease and 15 patients achieved a CR for bone marrow mast cell burden.
They seem to be eager to discuss this ahead of the market open tomorrow. Their conference call is set for 6 am.
Said Blueprint Medicines CMO Andy Boral:
We look forward to engaging with global regulatory authorities in the first half of 2018 to obtain feedback on a potential registration pathway. In addition, based on data showing a favorable tolerability profile and strong clinical activity from the lowest dose levels tested, we plan to expand our clinical development program to address the full spectrum of SM, including advanced, smoldering and indolent forms of the disease, over the course of 2018.
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