Blueprint Medicines Announces Accelerated Regulatory Submission Plans and Recent Clinical Progress
"The updates we are announcing today for avapritinib, BLU-667 and BLU-782 represent the acceleration of multiple clinical-stage programs, including planned marketing applications for avapritinib and BLU-667, and highlight our commitment to executing a portfolio-based strategy," said
Summary of Accelerated Regulatory Submission Plans and Recent Clinical Progress
Avapritinib: Gastrointestinal Stromal Tumors (GIST)
Blueprint Medicinesnow plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency(EMA) for avapritinib for the treatment of both PDGFRα D842V mutant GIST and fourth-line GIST in the third quarter of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)
Blueprint Medicinesnow plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration( FDA) for avapritinib for the treatment of advanced SM in the first quarter of 2020, subject to continuing discussions with the FDAunder its breakthrough therapy program to determine the required clinical data for an NDA submission.
BLU-667: RET-Altered Cancers
- Top-line interim data from the Phase 1 ARROW clinical trial of BLU-667 for patients with RET-fusion non-small cell lung cancer (NSCLC) and RET-mutant medullary thyroid cancer (MTC) who were response evaluable and treated at the recommended Phase 2 dose of 400 mg once daily, as of
November 14, 2018with follow-up through a data cutoff date of March 1, 2019, showed the following:
- In 34 patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 62 percent.(1)
- In 16 patients with RET-mutant MTC previously treated with the approved multi-kinase inhibitors cabozantinib and/or vandetanib, the ORR was 63 percent.(2)
- BLU-667 was generally well-tolerated, and most adverse events (AEs) reported by investigators were mild or moderate (Grade 1 or 2). Across all doses and treatment cohorts (n=217), only eight patients discontinued treatment with BLU-667 due to treatment-related AEs, with one Grade 5 AE (pneumonia/lung infection) determined by the investigator to be possibly related to BLU-667.
- Detailed clinical safety and efficacy data from the ARROW trial in RET-fusion NSCLC patients and RET-mutant MTC patients have been submitted for presentation at the
American Society of Clinical OncologyAnnual Meeting in June 2019.
- The enrollment target has been reached and patient screening is now closed for the registration-enabling ARROW trial cohort for patients with previously treated RET-fusion NSCLC.
Blueprint Medicinesanticipates reaching the enrollment target for the registration-enabling ARROW trial cohort for patients with previously treated MTC in the second quarter of 2019.
- Based on the early achievement of the enrollment target for the RET-fusion NSCLC cohort,
Blueprint Medicinesnow plans to submit an NDA to the FDAfor BLU-667 for the treatment of patients with NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020. Blueprint Medicinescontinues to expect to submit an NDA to the FDAfor BLU-667 for the treatment of patients with RET-mutant MTC previously treated with an approved multi-kinase inhibitor in the first half of 2020.
BLU-782: Fibrodysplasia Ossificans Progressiva (FOP)
- Based on the progress of the ongoing Phase 1 clinical trial in healthy volunteers and input from clinical experts,
Blueprint Medicinesnow plans to initiate a Phase 2a clinical trial of BLU-782 in patients with FOP in the fourth quarter of 2019.
(1) 95 percent confidence interval (CI): 44-78 percent; five responses pending confirmation.
(2) 95 percent CI: 35-85 percent; three responses pending confirmation.
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 plans and timelines for the development of avapritinib, BLU-667 and BLU-782; plans and timelines for submitting an MAA to the EMA for avapritinib for the treatment of PDGFRα D842V mutant GIST and fourth-line GIST; plans and timelines for submitting an NDA to the
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