On January 11, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) reported an update on key portfolio milestones and outlined a strategic roadmap to become the world’s leading precision therapy company (Press release, Blueprint Medicines, JAN 11, 2021, View Source [SID1234573835]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"For the first time, we enter a new year as a fully integrated, global biopharmaceutical company, with four regulatory approvals in the United States and Europe in 2020, a pipeline of eight wholly owned or partnered precision therapies, and the strongest financial position since our inception," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "With this solid foundation, we are now scaling our ambition and aim to make real the promise of precision medicine to improve and extend life for as many people with cancer and hematologic disorders as possible. We will do this by bringing our medicines to more patients globally, rapidly advancing a wave of new therapeutic candidates to clinical proof-of-concept, and further expanding our platform-enabled research pipeline."
In addition, Blueprint Medicines reported the achievement of several portfolio milestones:
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 Medicines and 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 U.S. and Europe for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumor driven by certain PDGFRA mutations.
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 U.S. for the treatment of patients with certain advanced or metastatic RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Under a global collaboration, Blueprint Medicines and Roche are developing and commercializing GAVRETO for the treatment of RET-altered cancers.
Obtain regulatory approval from the European Commission and launch GAVRETO in RET fusion-positive NSCLC in Europe in 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.
Financial Guidance
Based on its current operating plans, Blueprint Medicines continues to anticipate its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.