On February 16, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2021 (Press release, Blueprint Medicines, FEB 16, 2022, View Source [SID1234608162]).
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"Following a year of outstanding execution across our portfolio, we are well positioned in 2022 to drive significant year-over-year product revenue growth, achieve multiple anticipated clinical data milestones for our precision therapies in systemic mastocytosis and lung cancer, and expand our portfolio with new therapeutic candidates, all supported by a strong cash position," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, as we execute on this near-term vision, we will put in place drivers of future growth, including the expansion of our research platform to incorporate targeted protein degradation, as well as plans for targeted sourcing of external innovation. Together, these efforts have the potential to significantly strengthen and broaden our impact as a leading global precision therapy company."
Fourth Quarter 2021 Highlights and Recent Progress
AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM) and gastrointestinal stromal tumor (GIST)
Recorded global net product revenues of $53.0 million and $20.0 million for the full year and the fourth quarter of 2021, respectively, representing approximately 150 percent year-over-year growth based on strong initial U.S. demand in advanced SM.
Received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use for AYVAKYT for the treatment of adult patients with advanced SM, including aggressive SM, SM with an associated hematological neoplasm, or mast cell leukemia, after at least one systemic therapy. Read the press release here.
Announced two publications in Nature Medicine on the registration-enabling EXPLORER and PATHFINDER trials highlighting AYVAKIT’s robust efficacy and safety data in advanced SM. Read the press release here.
GAVRETO (pralsetinib): RET-altered cancers
Following the transition of certain responsibilities from Blueprint Medicines to Roche in the third quarter of 2021, Roche recorded and reported $9.8 million in U.S. net end user product revenues for the second half of 2021. Full year 2021 U.S. net end user product revenues for GAVRETO were $14.5 million, including sales booked by Blueprint Medicines in the first half of 2021.
Received, via the collaboration with Roche, conditional marketing authorization by the European Commission for GAVRETO for the treatment of adults with RET fusion-positive non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.
BLU-945, BLU-701 and BLU-451 (formerly LNG-451): EGFR-driven NSCLC
Treated the first patient in the Phase 1/2 HARMONY trial of BLU-701 in EGFR-driven NSCLC.
Presented new preclinical data supporting the development of BLU-945 and BLU-701 combination therapy in EGFR-driven NSCLC at the British Thoracic Oncology Group annual conference. Read the poster presentation here.
Multiple abstracts accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, including initial Phase 1/2 SYMPHONY trial dose escalation data for BLU-945 in EGFR-driven NSCLC.
Announced a strategic collaboration and license agreement with Zai Lab Limited for the development and commercialization of BLU-945 and BLU-701 for the treatment of EGFR-driven NSCLC in Greater China. Read the press release here.
Completed the acquisition of Lengo Therapeutics and its lead compound BLU-451, a potential best-in-class precision therapy for EGFR exon 20 insertion-positive NSCLC. Read the press release here.
Received clearance from the U.S. Food and Drug Administration (FDA) for an investigational new drug (IND) application for BLU-451 in EGFR exon 20 insertion-positive NSCLC.
BLU-222: ovarian, breast and other cyclin E-CDK2 aberrant cancers
Received FDA clearance for an IND application for BLU-222 for cyclin E-CDK2 aberrant cancers.
Corporate
Announced Kate Haviland, who has served as Chief Operating Officer and previously Chief Business Officer since 2016, has been appointed by the company’s Board of Directors to succeed Jeff Albers as President and Chief Executive Officer, effective April 4, 2022. At that time, Mr. Albers will transition from his current role as Chairman, President and Chief Executive Officer to Executive Chairman of the Board of Directors. In addition, Ms. Haviland will join the Board of Directors. Read the press release here.
Announced promotions for Christina Rossi to Chief Operating Officer and Philina Lee, Ph.D., to Chief Commercial Officer. In addition, announced a role expansion for Helen Ho, Ph.D., to Chief Business Officer. Read the press releases here and here.
Announced the appointment of Daniella Beckman, Chief Financial Officer of Tango Therapeutics, to the company’s Board of Directors. Read the press release here.
Key Upcoming Milestones
The company plans to achieve the following milestones by mid-2022:
Initiate a Phase 1/2 trial of BLU-222 in cyclin E-CDK2 aberrant cancers in the first quarter of 2022.
Initiate a Phase 1/2 trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC in the first quarter of 2022.
Present initial clinical data from the Phase 1/2 SYMPHONY trial of BLU-945 in the second quarter of 2022.
Present preclinical data supporting the development of BLU-451 in EGFR exon 20 insertion-positive NSCLC in the second quarter of 2022.
Present preclinical data supporting the development of BLU-222 in cyclin E-CDK2 aberrant cancers in the second quarter of 2022.
Launch AYVAKYT in advanced SM in Europe in the second quarter of 2022.
Report topline data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2022.
Fourth Quarter and Year End 2021 Results
Revenues: Revenues were $107.0 million for the fourth quarter of 2021, including $20.0 million of net product revenues from sales of AYVAKIT and $87.0 million in collaboration revenues. Revenues for the year ended December 31, 2021 were $180.1 million, including $53.0 million of net product revenues from sales of AYVAKIT, $4.7 million of net product revenues from sales of GAVRETO and $122.4 million in collaboration revenues. Blueprint Medicines recorded $34.1 million and $793.7 million in revenues in the fourth quarter and year ended December 31, 2020, respectively.
Cost of Sales: Cost of sales was $7.5 million for the fourth quarter of 2021 and $17.9 million for the year ended December 31, 2021, as compared to $0.1 million for the fourth quarter of 2020 and $0.4 million for the full year ended December 31, 2020. This increase was primarily driven by lower margin drug product sold to our collaboration partners during the fourth quarter of 2021.
R&D Expenses: Research and development expenses were $356.9 million for the fourth quarter of 2021 and $601.0 million for the year ended December 31, 2021, as compared to $77.4 million for the fourth quarter of 2020 and $326.9 million for the year ended December 31, 2020. Research and development expense for the fourth quarter of 2021 included $260.0 million incurred to acquire in-process research and development compounds through the acquisition of Lengo Therapeutics which was the primary driver of the increase in expenses over the prior year. Research and development expenses also included $10.0 million in stock-based compensation expenses for the fourth quarter of 2021 and $39.7 million in stock-based compensation for the year ended December 31, 2021.
SG&A Expenses: Selling, general and administrative expenses were $54.2 million for the fourth quarter of 2021 and $195.3 million for the year ended December 31, 2021, as compared to $42.5 million for the fourth quarter of 2020 and $157.7 million for the year ended December 31, 2020. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $12.7 million in stock-based compensation expenses for the fourth quarter of 2021 and $52.0 million in stock-based compensation for the year ended December 31, 2021.
Net Income (Loss): Net loss was $(318.7) million for the fourth quarter of 2021 and $(644.1) million for the year ended December 31, 2021, or a diluted net loss per share of $(5.40) and diluted net loss per share of $(11.01), respectively, as compared to a net loss of $(85.7) million for the fourth quarter of 2020 and a net income of $313.9 million for the year ended December 31, 2020, or a diluted net loss per share of $(1.53) and a diluted net income per share of $5.59, respectively.
Cash Position: As of December 31, 2021, cash, cash equivalents and marketable securities were $1,034.6 million, as compared to $1,549.7 million as of December 31, 2020.
2022 Financial Guidance
Blueprint Medicines reported it anticipates approximately $180M to $200M in total revenues in 2022, including approximately $115M to $130M in AYVAKIT net product revenues. The company continues to expect that 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.
Conference Call Information
Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss fourth quarter and full year 2021 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 936793. A webcast of the call will also 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.
Upcoming Investor Conferences
Blueprint Medicines will participate in two upcoming investor conferences:
11th Annual SVB Leerink Global Healthcare Conference on Friday, February 18, 2022 at 10:40 a.m. ET.
Cowen 42nd Annual Health Care Conference on Tuesday, March 8, 2022 at 9:10 a.m. ET.
A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.