On January 13, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported corporate goals for 2020, which provide a path to achieve the company’s "2020 Blueprint" strategy for launching its global commercial business (Press release, Blueprint Medicines, JAN 13, 2020, View Source [SID1234553100]).

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"2020 is off to a great start for Blueprint Medicines with the recent FDA approval of our first medicine and a U.S. launch now underway. As we complete our evolution into a fully-integrated biopharmaceutical company this year, we will also aim to bring a second product to market, expand across multiple indications and extend our global commercial footprint with our first anticipated regulatory approval in Europe," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, our future growth will be fueled by an expanded strategic focus on systemic mastocytosis and related mast cell disorders, which represent a large population of underserved patients with significant medical needs. This focus is anchored by avapritinib, which is now FDA-approved for the treatment of PDGFRA exon 18 mutant GIST. Avapritinib was specifically designed to treat the underlying cause of systemic mastocytosis and has demonstrated remarkable and consistent clinical activity across the spectrum of the disease."

RECENT PORTFOLIO MILESTONES AND 2020 GOALS

Avapritinib: gastrointestinal stromal tumors (GIST)

In January 2020, announced the U.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 the FDA, 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 the FDA 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 in Philadelphia on March 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 of November 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 the FDA 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, at Blueprint 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