Blueprint Medicines Provides 2023 Portfolio Goals to Achieve Precision at Scale
-- Expect to achieve high-end of 2022 total revenue guidance of
-- Positioned for significant near-term growth with potential FDA approval of AYVAKIT® (avapritinib) in indolent systemic mastocytosis in the middle of 2023 --
-- Anticipate multiple clinical data catalysts across mast cell disorder, EGFR-mutant non-small cell lung cancer, and breast cancer programs in 2023 --
In the coming year, we plan to scale the impact of AYVAKIT by addressing the medical needs of a substantially larger group of patients with an anticipated label expansion in indolent systemic mastocytosis, where AYVAKIT has the potential to be a disease modifying therapy and a major treatment advance. Our experienced team, who is in the field bringing AYVAKIT to patients with advanced systemic mastocytosis, is ready to expand our efforts upon FDA approval.
In addition, the data we plan to generate this year will start to truly characterize the potential of our clinical-stage pipeline to solve complex medical problems in EGFR-mutant lung cancer and CDK2-vulnerable breast cancer, which both represent large established commercial opportunities.
This diverse set of growth drivers, together with our existing commercial portfolio and proven discovery platform, put us squarely on the path to achieve our vision for doubling our impact between now and 2027."
The company's key strategies and upcoming goals are to:
1. Launch AYVAKIT in indolent systemic mastocytosis (SM), improving treatment options for patients across the spectrum of the disease and expanding the company's leadership position in SM.
- Present registrational PIONEER trial data for AYVAKIT in indolent SM at the
American Academy of Allergy, Asthma and Immunology Annual Meeting in February 2023
European Medicines Agencyvalidation of a type II variation marketing authorization application for AYVAKYT for indolent SM in the first half of 2023
- Achieve approval from the
Food and Drug Administration(FDA) and initiate the launch of AYVAKIT in indolent SM in the U.S.in the middle of 2023
- Present HARBOR Part 1 trial data for elenestinib (BLU-263) in indolent SM in the second half of 2023
2. Advance a robust portfolio of innovative clinical programs, addressing broader patient populations, towards registration.
- Submit an investigational new drug application to the FDA for BLU-525 in the first half of 2023
- Present initial CONCERTO trial dose escalation data for BLU-451 in EGFR exon 20-positive NSCLC in the first half of 2023
- Provide an initial clinical data update on SYMPHONY trial expansion for the combination of BLU-945 and osimertinib in first-line EGFR L858R-positive NSCLC in the second half of 2023
Breast and other CDK2-vulnerable cancers:
- Present initial VELA trial dose escalation data for BLU-222 in CDK2-vulnerable cancers in the first half of 2023
3. Grow the R&D pipeline with diverse, high-value programs from the company's prolific scientific platform.
- Nominate a development candidate targeting wild-type KIT for treatment of chronic urticaria in the middle of 2023
J.P Morgan Healthcare Conference Presentation Information
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding FDA approval for AYVAKI for the treatment of indolent SM; plans and timing for presenting data from the PIONEER trial of AYVAKIT in patients with indolent SM; statements regarding plans, strategies, timelines and expectations for interactions with the FDA and other regulatory authorities, statements regarding plans and expectations for
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