Blueprint Medicines Corporation (NASDAQ: BPMC) outlined upcoming portfolio milestones that are anticipated to extend its position as a leading precision therapy company.

“With two precision therapies expanding their global reach across multiple approved indications, four INDs filed from our portfolio in 2021, and a range of clinical data inflection points anticipated over the next 12 to 18 months, Blueprint Medicines is entering a new, transformative phase of growth,” said Jeff Albers, Chief Executive Officer of Blueprint Medicines. “As we look ahead to 2022, we have the foundation in place to dramatically expand our opportunity to improve the lives of increasingly broad populations of patients with cancer and blood disorders. We will do this by advancing our systemic mastocytosis franchise to improve treatment across the spectrum of disease, progressing multiple innovative precision therapies for lung cancer and other genomically defined cancers toward registration, and diversifying our scientific platform to maintain unparalleled research productivity. Supported by strong commercial performance for AYVAKIT in advanced systemic mastocytosis and a clear pathway to financial independence, we are well positioned to usher in a new era of leadership and innovation in the field of precision medicine.”

The company’s key strategies and upcoming goals are to:

1. Expand the company’s leadership position in systemic mastocytosis (SM), improving treatment options for patients across the spectrum of the disease.

AYVAKIT®/AYVAKYT® (avapritinib) and BLU-263: SM

Obtain regulatory approval from the European Medicines Agency and launch AYVAKYT in advanced SM in Europe in the first half of 2022.
Report topline data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2022.
Submit a supplemental new drug application to the U.S. Food and Drug Administration for AYVAKIT in non-advanced SM in the second half of 2022.
Present data from the HARBOR trial of BLU-263 in non-advanced SM in the second half of 2022.

2. Advance a robust portfolio of innovative clinical programs towards registration.

BLU-945 and BLU-701: EGFR-driven NSCLC

Present preclinical data supporting the combination of BLU-945 and BLU-701 in the first quarter of 2022.
Present initial clinical data from the Phase 1/2 SYMPHONY trial of BLU-945 in the first half of 2022.
Present initial clinical data from Phase 1/2 HARMONY trial of BLU-701 in the second half of 2022.
BLU-451 (formerly LNG-451): EGFR exon 20 insertion-positive NSCLC

Initiate a Phase 1/2 trial of BLU-451 in the first quarter of 2022.
Present preclinical data for BLU-451 in the first half of 2022.
BLU-222: Cyclin-E aberrant cancers

Initiate the Phase 1/2 VELA trial of BLU-222 in the first quarter of 2022.
Present preclinical data for BLU-222 in the first half of 2022.
GAVRETO® (pralsetinib): RET-altered cancers

Submit additional marketing applications for GAVRETO for RET-altered NSCLC and thyroid cancers across multiple additional global geographies in 2022, via the company’s ongoing global collaboration with Roche.
3. Grow the R&D pipeline with diverse, high-value programs from company’s prolific scientific platform.


Expand pipeline with two new development candidates in 2022.
Share the company’s research vision, including scientific platform expansion plans, at an R&D Day in the second half of 2022.
Financial Guidance

Blueprint Medicines also announced preliminary1 global product revenues for AYVAKIT for full year 2021 and the fourth quarter of 2021 were approximately $52.9 million, and $20.0 million, respectively, representing an increase of approximately 150 percent and 230 percent over the same periods in 2020 based on strong initial U.S. demand in advanced SM. Full year 2021 total revenues, including collaboration revenues, are expected to be at the higher-end of previous guidance of $170-$180 million. Cash, cash equivalents and investments as of December 31, 2021 were approximately $1.0 billion.1 Based on its current operating plans, Blueprint Medicines continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

1The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2022.