Blueprint Medicines Reports Fourth Quarter and Full Year 2022 Results
-- Achieved
-- Continued progress toward regulatory approvals of AYVAKIT/AYVAKYT for indolent systemic mastocytosis, with FDA granting priority review of a supplemental new drug application and EMA validating a type II variation marketing authorization application --
-- Positive data from PIONEER trial of AYVAKIT in indolent systemic mastocytosis to be presented at the AAAAI Annual Meeting --
"In 2022, AYVAKIT became the standard of care for patients with advanced systemic mastocytosis in the
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 ofMay 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 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-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 endedDecember 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 endedDecember 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 endedDecember 31, 2022 , as compared to$7.5 million for the fourth quarter of 2021 and$17.9 million for the full year endedDecember 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 endedDecember 31, 2022 , as compared to$356.9 million for the fourth quarter of 2021 and$601.0 million for the year endedDecember 31, 2021 . Research and development expense for the year endedDecember 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 endedDecember 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 endedDecember 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 endedDecember 31, 2022 , as compared to$54.2 million for the fourth quarter of 2021 and$195.3 million for the year endedDecember 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 endedDecember 31, 2022 . - Net Income (Loss): Net loss was
$(158.6) million for the fourth quarter of 2022 and$(557.5) million for the year endedDecember 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 endedDecember 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 ofDecember 31, 2021 .
2023 Financial Guidance
Conference Call Information
Upcoming Investor Conferences
- Cowen 43rd Annual Health Care Conference on
Monday, March 6, 2023 at2:10 pm ET . Barclays Global Healthcare Conference , onWednesday, March 15, 2023 at2:35 pm ET .
A live webcast of each presentation will be available by visiting the Investors & Media section of
About
Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering approved medicines directly to patients in the United States and Europe, and we are globally advancing multiple programs for systemic mastocytosis, lung cancer, breast cancer and other genomically defined cancers, and cancer immunotherapy. For more information, visit www.BlueprintMedicines.com and follow us on
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 plans, strategies, timelines and expectations for
Trademarks
|
|||||
(1) Blueprint Medicines defines working capital as current assets less current liabilities. |
|||||
|
|
||||
2022 |
2021 |
||||
Cash, cash equivalents and investments |
$ |
1,078,472 |
$ |
1,034,643 |
|
Working capital (1) |
863,417 |
404,260 |
|||
Total assets |
1,349,902 |
1,252,225 |
|||
Deferred revenue (2) |
18,291 |
36,576 |
|||
Liability related to the sale of future royalties and revenues (2) |
430,330 |
- |
|||
Term loan (2) |
139,083 |
- |
|||
Total liabilities |
835,225 |
281,490 |
|||
Total stockholders' equity |
514,677 |
970,735 |
(1) |
|
(2) |
Includes both current and long-term portions of the balance |
|
||||||||||||||||||||||
Three Months Ended |
Years Ended |
|||||||||||||||||||||
|
|
|||||||||||||||||||||
2022 |
2021 |
2022 |
2021 |
|||||||||||||||||||
Revenues: |
||||||||||||||||||||||
Product revenue, net |
$ |
30,064 |
$ |
20,029 |
$ |
110,993 |
$ |
57,687 |
||||||||||||||
Collaboration and License Revenue |
8,717 |
86,993 |
65,543 |
122,393 |
||||||||||||||||||
License Revenue – |
-- |
-- |
27,500 |
-- |
||||||||||||||||||
Total revenues |
$ |
38,781 |
$ |
107,022 |
$ |
204,036 |
$ |
180,080 |
||||||||||||||
Cost and operating expenses: |
||||||||||||||||||||||
Cost of sales |
4,848 |
7,549 |
17,813 |
17,934 |
||||||||||||||||||
Collaboration loss sharing |
1,872 |
4,531 |
8,948 |
7,801 |
||||||||||||||||||
Research and development |
117,840 |
356,877 |
477,419 |
601,033 |
||||||||||||||||||
Selling, general and administrative |
64,019 |
54,199 |
237,374 |
195,293 |
||||||||||||||||||
Total cost and operating expenses |
$ |
188,579 |
$ |
423,156 |
$ |
741,554 |
$ |
822,061 |
||||||||||||||
Other income (expense): |
||||||||||||||||||||||
Interest income (expense), net |
(9,240) |
463 |
(16,767) |
2,386 |
||||||||||||||||||
Other income (expense), net |
1,435 |
(381) |
2,004 |
(1,489) |
||||||||||||||||||
Total other income (expense) |
$ |
(7,805) |
$ |
82 |
$ |
(14,763) |
$ |
897 |
||||||||||||||
Loss before income taxes |
$ |
(157,603) |
$ |
(316,052) |
$ |
(552,281) |
$ |
(641,084) |
||||||||||||||
Income tax expense |
1,036 |
2,635 |
5,236 |
3,001 |
||||||||||||||||||
Net loss |
$ |
(158,639) |
$ |
(318,687) |
$ |
(557,517) |
$ |
(644,085) |
||||||||||||||
Net loss per share applicable to common stockholders — basic |
$ |
(2.65) |
$ |
(5.40) |
$ |
(9.35) |
$ |
(11.01) |
||||||||||||||
Net loss per share applicable to common stockholders —diluted |
$ |
(2.65) |
$ |
(5.40) |
$ |
(9.35) |
$ |
(11.01) |
||||||||||||||
Weighted-average number of common shares used in net loss per share applicable to common stockholders — basic |
59,873 |
58,985 |
59,642 |
58,518 |
||||||||||||||||||
Weighted-average number of common shares used in net loss per share applicable to common stockholders —diluted |
59,873 |
58,985 |
59,642 |
58,518 |
||||||||||||||||||
View original content to download multimedia:https://www.prnewswire.com/news-releases/blueprint-medicines-reports-fourth-quarter-and-full-year-2022-results-301748416.html
SOURCE
Media Contact, Sarah Mena Guerrero, 617-714-6684, media@blueprintmedicines.com; or Investor Contact, Jenna Cohen, 857-209-3147, ir@blueprintmedicines.com