On February 16, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2022 (Press release, Blueprint Medicines, FEB 16, 2023, View Source [SID1234627295]).
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"In 2022, AYVAKIT became the standard of care for patients with advanced systemic mastocytosis in the U.S., and we continued to solidify our global leadership in mast cell disorders. Today, we are at the precipice of a pivotal moment in Blueprint’s history as we near a potential FDA approval of AYVAKIT for a much broader population of patients with indolent SM, which will enable us to address the significant medical needs of SM patients and create a critical inflection point in our growing revenue stream. In addition to our commercial execution, we advanced additional clinical development programs and continued to bolster our financial strength through our strategic financing and business development," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "In 2023, Blueprint Medicines has a diversity of fundamental value drivers that position us well on the path to achieve our 2027 Blueprint for Precision at Scale and translate the promise of precision medicine into reality for thousands of patients globally."
Fourth Quarter 2022 Highlights and Recent Progress
Systemic mastocytosis
- Recorded global AYVAKIT/AYVAKYT (avapritinib) net product revenues of $111.0 million and $30.1 million for the full year and the fourth quarter of 2022, respectively, representing more than 100 percent year-over-year growth.
- Anticipate $130 million to $140 million in AYVAKIT net product revenues in 2023 for advanced systemic mastocytosis (SM) and GIST. This guidance excludes revenue from the anticipated AYVAKIT indication expansion in indolent systemic mastocytosis (ISM) in mid-2023.
- Announced acceptance by the U.S. Food and Drug Administration (FDA) of the company’s supplemental new drug application for AYVAKIT for the treatment of adults with moderate-to-severe ISM. The FDA granted priority review with an action date of May 22, 2023. Read the press release here.
- Achieved validation of a type II variation marketing authorization application from the European Medicines Agency for AYVAKYT for the treatment of adult patients with moderate-to-severe ISM.
- Announced plans to present positive data from the PIONEER trial of AYVAKIT in ISM at the 2023 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting. Read the press release here.
- Presented new clinical data demonstrating AYVAKIT significantly improved survival in patients with all sub-types of advanced SM at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual conference. Read presentation and posters available here.
- Advanced the development of elenestinib and announced topline results from Part 1 of the HARBOR trial demonstrating clinical activity and safety. Read the press release here.
EGFR-driven non-small cell lung cancer (NSCLC)
- Presented new clinical data at the company’s Investor Day informing the development strategy for BLU-945, including plan to prioritize development of BLU-945 in combination with osimertinib in first-line EGFR L858R-positive NSCLC. This included data demonstrating that BLU-945 monotherapy treatment led to ctDNA responses and tumor shrinkage in late-line patients with EGFR-driven NSCLC, as well as early dose escalation data showing BLU-945 in combination with osimertinib has been generally well-tolerated to-date. Read the Investor Day presentation here.
- Presented new preclinical data supporting the development of BLU-525 as a potent and selective EGFR inhibitor at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting. Read the poster here.
CDK2-vulnerable breast and other cancers
- Announced a partial clinical trial hold for the Phase 1/2 VELA study of BLU-222 due to transient and reversible visual adverse events observed in a limited number of patients. Read the press release here.
- Presented new data supporting BLU-222 as monotherapy or in combination with ribociclib in preclinical models at San Antonio Breast Cancer Symposium. Read the poster presentation here.
Corporate
- Presented the company’s 2027 Blueprint to achieve Precision at Scale, a five-year business strategy to double the company’s impact at Investor Day 2022. Read the Investor Day presentation here.
- Announced the appointment of John Tsai, M.D, former President, Global Drug Development and Chief Medical Officer at Novartis AG, to the company’s Board of Directors.
Key Upcoming Milestones
The company plans to achieve the following milestones by mid-2023:
- Present registrational PIONEER trial data in ISM at the AAAAI annual meeting in the first quarter of 2023.
- Receive approval from the FDA for AYVAKIT for use in adults with ISM in the second quarter of 2023.
- Present dose escalation data from the CONCERTO trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC in the first half of 2023.
- Present dose escalation data from the VELA trial of BLU-222 in patients with CDK2-vulnerable cancers in the first half of 2023.
- Submit an investigational new drug (IND) application to the FDA for BLU-525 in the first half of 2023.
- Nominate a development candidate for inhibition of wild-type KIT for the treatment of chronic urticaria in mid-2023.
Fourth Quarter and Year End 2022 Results
- Revenues: Revenues were $38.8 million for the fourth quarter of 2022, including $30.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $8.7 million in collaboration revenues. Revenues for the year ended December 31, 2022 were $204.0 million, including $111.0 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $93.0 million in collaboration and license revenues. Blueprint Medicines recorded $107.0 million and $180.1 million in revenues in the fourth quarter and year ended December 31, 2021, respectively.
- Cost of Sales: Cost of sales was $4.8 million for the fourth quarter of 2022 and $17.8 million for the year ended December 31, 2022, as compared to $7.5 million for the fourth quarter of 2021 and $17.9 million for the full year ended December 31, 2021. This decrease was primarily driven by lower costs related to collaboration product sales.
- R&D Expenses: Research and development expenses were $117.8 million for the fourth quarter of 2022 and $477.4 million for the year ended December 31, 2022, as compared to $356.9 million for the fourth quarter of 2021 and $601.0 million for the year ended December 31, 2021. Research and development expense for the year ended December 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 decrease in expenses for the year ended December 31, 2022. Research and development expenses also included $9.8 million in stock-based compensation expenses for the fourth quarter of 2022 and $40.3 million in stock-based compensation for the year ended December 31, 2022.
- SG&A Expenses: Selling, general and administrative expenses were $64.0 million for the fourth quarter of 2022 and $237.4 million for the year ended December 31, 2022, as compared to $54.2 million for the fourth quarter of 2021 and $195.3 million for the year ended December 31, 2021. This increase was primarily due to increased internal and external costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $16.4 million in stock-based compensation expenses for the fourth quarter of 2022 and $58.7 million in stock-based compensation for the year ended December 31, 2022.
- Net Income (Loss): Net loss was $(158.6) million for the fourth quarter of 2022 and $(557.5) million for the year ended December 31, 2022, or a diluted net loss per share of $(2.65) and diluted net loss per share of $(9.35), respectively, as compared to a net loss of $(318.7) million for the fourth quarter of 2021 and a net loss of $(644.1) million for the year ended December 31, 2021, or a diluted net loss per share of $(5.40) and a diluted net loss per share of $(11.01), respectively.
- Cash Position: As of December 31, 2022, cash, cash equivalents and marketable securities were $1,078.5 million, as compared to $1,034.6 million as of December 31, 2021.
2023 Financial Guidance
Blueprint Medicines reported it anticipates approximately $130 million to $140 million in AYVAKIT net product revenues for advanced SM and GIST in 2023, and $40 million to $50 million in collaboration revenues from existing collaborations. This guidance excludes revenue from the anticipated AYVAKIT indication expansion in ISM in mid-2023. 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 8:00 a.m. ET today to discuss fourth quarter and full year 2022 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 286085. 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:
Cowen 43rd Annual Health Care Conference on Monday, March 6, 2023 at 2:10 pm ET.
Barclays Global Healthcare Conference, on Wednesday, March 15, 2023 at 2:35 pm 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.