Blueprint Medicines Reports Portfolio Milestones and Outlines 2021 Roadmap for Precision Medicine Leadership
"For the first time, we enter a new year as a fully integrated, global biopharmaceutical company, with four regulatory approvals in
- AYVAKIT received breakthrough therapy designation from the
U.S. Food and Drug Administration(FDA) for the treatment of moderate to severe indolent systemic mastocytosis (SM), which encompasses the majority of patients with SM, highlighting the medical need in this population as well as the clinical potential of AYVAKIT to demonstrate substantial improvement over the current standard of care.
- Positive top-line results from a Phase 1 trial in healthy volunteers showed BLU-263 was well-tolerated across a range of single- and multiple-ascending doses predicted to potently inhibit D816V mutant KIT, the underlying SM disease driver. These data support development of BLU-263 as a potential treatment for patients with SM and other mast cell disorders.
- The company nominated a selective, brain-penetrant development candidate for treatment-resistant double-mutant EGFR-driven non-small cell lung cancer (NSCLC), with the potential to be first-in-class, showing potent activity against the activating L858R or exon 19 deletion mutations and the acquired C797S mutation, the most common on-target resistance mutation to osimertinib.
- The company nominated a development candidate targeting MAP4K1, a kinase believed to play a role in T-cell regulation, with the potential to be best-in-class. The program was developed under the company's cancer immunotherapy collaboration with Roche. In addition,
Blueprint Medicinesand Roche have amended their agreement to focus on MAP4K1 and one additional undisclosed target, collectively identified as the most promising targets of the collaboration to date.
Entering 2021, the company's key strategies and goals include:
1. Accelerate global adoption of AYVAKIT and GAVRETO™ (pralsetinib)
AYVAKIT, a selective KIT and PDGFRA inhibitor, is approved in the
- Obtain FDA approval and launch AYVAKIT in advanced SM in the
U.S.in the second half of 2021.
- Submit a Type II variation marketing authorization application (MAA) to the
European Medicines Agency(EMA) for AYVAKYT® (avapritinib) for advanced SM in the first quarter of 2021.
- Present registrational data from the PATHFINDER trial of AYVAKIT in advanced SM in the first half of 2021.
- Complete enrollment of the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2021.
GAVRETO, a selective RET inhibitor, is approved in the
- Obtain regulatory approval from the
European Commissionand launch GAVRETO in RET fusion-positive NSCLC in Europein the first half of 2021.
- Submit a Type II variation MAA to the EMA for GAVRETO for RET-altered thyroid cancers in the second half of 2021.
- Initiate a GAVRETO cohort in Roche's TAPISTRY tumor-agnostic platform trial in the second half of 2021.
- Submit marketing applications for GAVRETO for RET-altered NSCLC and thyroid cancers across multiple additional global geographies in 2021.
2. Advance a new wave of innovative therapeutic candidates into clinical development, with plans to achieve rapid proof-of-concept and regulatory approval.
BLU-263, a next-generation selective KIT inhibitor
- Initiate the Phase 2 HARBOR trial of BLU-263 in patients with non-advanced SM in mid-2021.
Development candidates for treatment-resistant EGFR-driven NSCLC
- Initiate a Phase 1 trial of BLU-945, a triple-mutant EGFR inhibitor, in patients with treatment-resistant EGFR-driven NSCLC in the first half of 2021.
- Initiate a Phase 1 trial of the company's double-mutant EGFR inhibitor in patients with treatment-resistant EGFR-driven NSCLC by the end of 2021.
- Present foundational preclinical data for the company's double-mutant EGFR inhibitor in the first half of 2021.
- Present preclinical data supporting combination of the company's wholly owned double- and triple-mutant EGFR inhibitors in treatment-naïve EGFR-driven NSCLC in the second half of 2021.
Development candidate targeting MAP4K1, under the cancer immunotherapy collaboration with Roche
- Present foundational preclinical data in the first half of 2021.
3. Further expand the company's precision medicine pipeline with a focus on delivering transformational benefit to patients with cancer and hematologic disorders.
- Expand pipeline with one or more development candidates in 2021.
- Pursue external opportunities to complement the company's precision medicine pipeline.
Based on its current operating plans,
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
View original content to download multimedia:http://www.prnewswire.com/news-releases/blueprint-medicines-reports-portfolio-milestones-and-outlines-2021-roadmap-for-precision-medicine-leadership-301204817.html
Media Contact: Andrew Law, 617-844-8205, email@example.com; Investor Contact: Kristin Hodous, 617-714-6674, firstname.lastname@example.org