On April 29, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) reported its financial results and provided a business update for the first quarter ended March 31, 2021 (Press release, Blueprint Medicines, APR 29, 2021, View Source [SID1234578806]).
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"In recent months, we made significant progress toward achieving our key 2021 portfolio goals, illustrating our clinical leadership in systemic mastocytosis and the rapid advancement of our next wave of therapeutic candidates with first- or best-in-class potential," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "With a potential U.S. approval for AYVAKIT in advanced systemic mastocytosis in the second quarter, we are looking forward to welcoming a new era of precision medicines care for people living with this debilitating disease. In parallel, we are initiating multiple clinical trials across our pipeline, beginning with the Phase 1 trial of BLU-945 and Phase 2/3 HARBOR trial of BLU-263, as we continue to translate our research productivity and precision therapy expertise to address patient need across cancers and hematologic malignancies. Taken together, these accomplishments position Blueprint Medicines to help an increasing number of patients globally as we advance our robust portfolio."
First Quarter 2021 Highlights and Recent Progress
AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM)
● Reported data at the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, including registrational PATHFINDER trial data in advanced systemic mastocytosis (SM), which demonstrated an overall confirmed response rate of 75 percent, as well as PIONEER Part 1 data highlighting the impact of AYVAKIT on skin manifestations in non-advanced SM. Read the full data here.
● Received European Medicines Agency (EMA) validation of the Type II variation marketing authorization application (MAA) for AYVAKYT for the treatment of advanced SM. Validation of the MAA confirms that the submission is sufficiently complete to begin the formal review process. Read the press release here.
AYVAKIT/AYVAKYT (avapritinib): gastrointestinal stromal tumor (GIST)
● Recorded $7.1 million in net product revenue during the first quarter of 2021 for AYVAKIT/AYVAKYT, which was approved by the U.S. Food and Drug Administration (FDA) in January 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and by the European Commission in September 2020 as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
● Received National Medical Products Administration (NMPA) approval in China, via our collaboration with CStone Pharmaceuticals, for the treatment of adults with unresectable or metastatic PDGFRA exon 18 mutant GIST, the first approved precision therapy for this patient population in China.
GAVRETO (pralsetinib): RET-altered cancers
● Recorded $1.8 million in net product revenue during the first quarter of 2021 for GAVRETO, which was approved by the FDA in September 2020 for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test and in December 2020 for the treatment of patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Blueprint Medicines is commercializing GAVRETO in the U.S. together with Genentech, Inc., a member of the Roche Group.
● Received approval in China, via our collaboration with CStone Pharmaceuticals, for the treatment of adults with locally advanced or metastatic RET fusion-positive NSCLC after platinum-based chemotherapy, the first approved selective RET inhibitor in China.
BLU-263: SM
● Reported new data at AACR (Free AACR Whitepaper) from a Phase 1 trial in healthy volunteers, showing that BLU-263 was generally well-tolerated across a range of single- and multiple-ascending doses, with a half-life supporting once-daily dosing. Based on these data, Blueprint Medicines plans to evaluate BLU-263 at doses ranging from 25 to 100 mg once daily in Part 1 of the Phase 2/3 HARBOR trial in non-advanced SM. Read the full data here.
BLU-945 and BLU-701: treatment-resistant EGFR-driven NSCLC
● Reported new preclinical data at AACR (Free AACR Whitepaper) showing the potential for the company’s potent and selective double-mutant EGFR inhibitor, BLU-701, and potent and selective triple-mutant EGFR inhibitor, BLU-945, to be used alone or in combination, together or with other agents, to overcome or prevent on-target resistance across multiple lines of treatment. Read the full data here.
● Received clearance from the FDA for an investigational new drug (IND) application for BLU-945 for the treatment of patients with EGFR-driven NSCLC.
BLU-222: Cyclin E-aberrant cancers
● Nominated BLU-222, a potentially best-in-class selective and potent CDK2 inhibitor development candidate, for the treatment of cyclin E-aberrant cancers.
● Reported new preclinical data at AACR (Free AACR Whitepaper) for a set of CDK2 inhibitors, showing that selective CDK2 inhibition arrested the cell cycle and blocked tumor proliferation in cyclin E (CCNE)-amplified cell lines and demonstrated robust and sustained anti-tumor activity in vivo in models of CCNE-amplified ovarian, breast and gastric cancer, with improved tolerability compared to a pan-CDK inhibitor and chemotherapy. Read the full data here.
BLU-852: Cancer immunotherapy
● Nominated BLU-852, a potentially best-in-class selective and potent MAP4K1 inhibitor development candidate, developed under the company’s cancer immunotherapy collaboration with Roche.
● Reported new preclinical data at AACR (Free AACR Whitepaper) for a set of MAP4K1 inhibitors, including BLU-852, which were shown to enhance intratumoral immune cell activation, overcome T cell suppression, and reduce tumor burden both as a monotherapy and in combination with checkpoint inhibition. Read the full data here.
Key Upcoming Milestones
The company expects to achieve the following near-term milestones:
● Obtain regulatory approval from the FDA and, if approved, launch AYVAKIT for the treatment of patients with advanced SM in the second quarter of 2021.
● Present clinical data from the ARROW trial of pralsetinib in patients with RET fusion‒positive non-small cell lung cancer and in patients with solid tumors at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in the second quarter of 2021.
● Initiate a global Phase 1 trial of BLU-945 in patients with treatment-resistant EGFR-driven NSCLC in the second quarter of 2021.
● Complete enrollment of the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2021.
● Initiate the Phase 2/3 HARBOR trial of BLU-263 in patients with non-advanced SM in mid-2021.
● Initiate a Phase 1 trial of BLU-701 in patients with treatment-resistant EGFR-driven NSCLC in the second half of 2021.
● Present preclinical data supporting combination of BLU-945 and BLU-701 in treatment-naïve EGFR-driven NSCLC in the second half of 2021.
First Quarter 2021 Financial Results
● Revenues: Revenues were $21.6 million for the first quarter of 2021, including $7.1 million of net product revenues from sales of AYVAKIT/AYVAKYT, $1.8 million of net product revenues from sales of GAVRETO and $12.6 million in collaboration revenues. Blueprint Medicines recorded revenues of $6.2 million in the first quarter of 2020, including $3.5 million of net product revenues from sales of AYVAKIT and $2.7 million in collaboration revenues.
● Cost of Sales: Cost of sales was $0.1 million for the first quarter of 2021, as compared to less than $0.1 million for the first quarter of 2020.
● R&D Expenses: Research and development expenses were $79.7 million for the first quarter of 2021, as compared to $84.1 million for the first quarter of 2020. This decrease was primarily due to reimbursement from the global development cost sharing arrangement under our collaboration with Roche for pralsetinib. Research and development expenses included $8.9 million in stock-based compensation expenses for the first quarter of 2021.
● SG&A Expenses: Selling, general and administrative expenses were $42.0 million for the first quarter of 2021, as compared to $35.7 million for the first quarter of 2020. This increase was primarily due to increased costs associated with building our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT and GAVRETO, partially offset by reimbursement under our collaboration with Roche for pralsetinib. General and administrative expenses included $11.7 million in stock-based compensation expenses for the first quarter of 2021.
● Net Loss: Net loss was $99.7 million for the first quarter of 2021, or a net loss per share of $1.72, as compared to a net loss of $111.0 million for the first quarter of 2020, or a net loss per share of $2.11.
● Cash Position: As of March 31, 2021, cash, cash equivalents and investments were $1,430.1 million, as compared to $1,549.7 million as of December 31, 2020.
Conference Call Information
Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss first quarter 2021 financial results and recent business activities. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international), and referring to conference ID 9292306. A webcast of the call will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.