Blueprint Medicines Announces 2020 Corporate Goals
"2020 is off to a great start for
RECENT PORTFOLIO MILESTONES AND 2020 GOALS
Avapritinib: gastrointestinal stromal tumors (GIST)
- In
January 2020 , announced theU.S. Food and Drug Administration (FDA ) granted a full approval to AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Read the press release here and visit www.AYVAKIT.com for full Prescribing Information.
2020 goals:
- Report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST in the second quarter of 2020
- Gain regulatory approval and launch avapritinib in fourth-line GIST in the U.S. in the second quarter of 2020
- Gain regulatory approval and launch avapritinib in PDGFRA D842V GIST in
Europe in the third quarter of 2020 - Present the registration dataset from the VOYAGER trial of avapritinib in third-line GIST in 2020
- Submit a supplemental new drug application (NDA) to the
FDA for third-line GIST in the second half of 2020
Avapritinib and BLU-263: systemic mastocytosis (SM) and other mast cell disorders
- Today announced an update on the planned submission of a supplemental NDA to the
FDA for avapritinib for advanced SM. Based on ongoing discussions with theFDA , the company plans to focus its supplemental NDA on data from patients in the Phase 1 EXPLORER and Phase 2 PATHFINDER trials who started at the proposed indicated dose of 200 mg once daily (QD), supported by pooled data from all doses. To allow for a sufficient number of patients and follow-up,Blueprint Medicines now plans to submit the supplemental NDA to theFDA in the second half of 2020. - In
December 2019 , reported initial data from Part 1 of the PIONEER trial of avapritinib in patients with indolent SM. The data showed rapid and robust reductions in serum tryptase, a measure of mast cell burden, in patients treated with 25, 50 or 100 mg QD of avapritinib. All dose levels of avapritinib tested were well-tolerated, and no patients discontinued treatment due to an adverse event (AE). Read the press release here. - Today announced updated data from Part 1 of the PIONEER trial have been accepted for presentation as a late-breaking oral abstract at the
American Academy of Allergy , Asthma & Immunology 2020 Annual Meeting inPhiladelphia onMarch 14, 2020 .
2020 goals:
- Present updated data from Part 1 of the PIONEER trial of avapritinib in indolent SM in the first quarter of 2020
- Initiate a Phase 1 trial of BLU-263 in healthy volunteers in the first half of 2020
- Submit a supplemental NDA to
FDA for avapritinib for advanced SM in the second half of 2020 - Present updated data from the EXPLORER and PATHFINDER trials of avapritinib in advanced SM in the second half of 2020
- Complete enrollment of Part 2 of the PIONEER trial of avapritinib in indolent SM by the end of 2020
Pralsetinib: RET-altered cancers
- In
January 2020 , reported top-line data from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive non-small cell lung cancer (NSCLC) as of a data cutoff date ofNovember 18, 2019 in patients treated with the proposed indicated dose of 400 mg QD. In patients with previously treated RET fusion-positive NSCLC, the overall response rate (ORR) was 61 percent (95% CI: 50-72%) per central review (two responses pending confirmation), and the median duration of response was not reached. In patients with treatment-naïve RET fusion-positive NSCLC, the ORR was 73 percent (95% CI: 52-88%) per central review (all responses confirmed), with 12 percent of patients achieving a complete response. Pralsetinib was well-tolerated, and most reported AEs were Grade 1 or 2 with only four percent of patients discontinuing treatment with pralsetinib due to treatment-related AEs. In addition, the company announced it had initiated a rolling NDA submission to theFDA for pralsetinib for the treatment of RET fusion-positive NSCLC. Read the press release here. - Today announced the activation of the first trial site for the company's Phase 3 AcceleRET Lung trial of pralsetinib in patients with first-line RET fusion-positive NSCLC. More information about the trial is available at www.clinicaltrials.gov (NCT04222972).
2020 goals:
- Complete the submission of a rolling NDA to the
FDA for pralsetinib for RET fusion-positive NSCLC in the first quarter of 2020 - Submit an NDA to the
FDA for pralsetinib for previously treated RET-mutant medullary thyroid cancer (MTC) in the second quarter of 2020 - Submit an MAA to EMA for pralsetinib for RET fusion-positive NSCLC in the second quarter of 2020
- Present registration datasets from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive NSCLC and RET-mutant MTC in 2020
- Initiate a Phase 3 trial of pralsetinib in first-line RET-mutant MTC in the second half of 2020
- Gain regulatory approval and launch pralsetinib in RET fusion-positive NSCLC in the U.S. by the end of 2020
Research portfolio
- In
November 2019 , atBlueprint Medicines' first R&D Day, announced four new research programs enabled by the company's integrated precision medicine platform. Read the press release here. - Today announced the nomination of a potential first-in-class development candidate for the treatment of resistant EGFR-positive triple mutant NSCLC.
2020 goals:
- Nominate up to two additional development candidates by the end of 2020
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SOURCE
Investor and Media Contact: Jim Baker, 617-844-8236, media@blueprintmedicines.com; Investor Contact: Kristin Hodous, 617-714-6674, ir@blueprintmedicines.com