-- Achieved $204.0
million in total revenues in 2022, including $111.0 million in AYVAKIT®/AYVAKYT®
(avapritinib) net product revenues --
-- 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 --
CAMBRIDGE, Mass., Feb. 16,
2023 /PRNewswire/ -- Blueprint Medicines Corporation
(NASDAQ: BPMC) today reported financial results and provided a
business update for the fourth quarter and full year ended
December 31, 2022.
![Print Print](https://mma.prnewswire.com/media/221118/blueprint_medicines_logo.jpg)
"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 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
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 today announced 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 http://ir.blueprintmedicines.com/. 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 http://ir.blueprintmedicines.com. A replay of the
webcasts will be archived on Blueprint Medicines' website for 30
days following each presentation.
About Blueprint Medicines
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 Twitter (@BlueprintMeds) and LinkedIn.
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
Blueprint Medicines' current or future approved drugs and drug
candidates, including approvals and launches, the initiation of
clinical trials or the results of ongoing and planned clinical
trial; Blueprint Medicines' plans, strategies and timelines to
nominate development candidates; timelines and expectations for
interactions with the FDA and other regulatory authorities;
statements regarding the plans and potential benefits of AYVAKIT in
treating patients with indolent SM; statements regarding plans and
expectations for Blueprint Medicines' current or future approved
drugs and drug candidates; the potential benefits of any of
Blueprint Medicines' current or future approved drugs or drug
candidates in treating patients; and Blueprint Medicines' financial
performance, strategy, goals and anticipated milestones, business
plans and focus. The words "aim," "may," "will," "could," "would,"
"should," "expect," "plan," "anticipate," "intend," "believe,"
"estimate," "predict," "project," "potential," "continue," "target"
and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Any forward-looking statements in this
press release are based on management's current expectations and
beliefs and are subject to a number of risks, uncertainties and
important factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation: the risk the partial clinical hold may or may not be
resolved in a timely manner; there may be additional adverse events
observed that could impact the extent of the partial clinical hold
or Blueprint Medicines' resolution of the partial
clinical hold; there may be amendments to the trial protocol that
impact the timing of the trial or evaluation of the data;
preliminary activity and safety data may not be representative of
more mature data; the COVID-19 pandemic may impact Blueprint
Medicines' business, operations, strategy, goals and anticipated
milestones, including ongoing and planned research and discovery
activities, Blueprint Medicines' ability to conduct ongoing and
planned clinical trials; the risk of delay of any current or
planned clinical trials or the development of Blueprint Medicines'
current or future drug candidates; risks related to Blueprint
Medicines' ability to successfully demonstrate the safety and
efficacy of its drug candidates and gain approval of its drug
candidates on a timely basis, if at all; preclinical and clinical
results for Blueprint Medicines' drug candidates may not support
further development of such drug candidates either as monotherapies
or in combination with other agents or may impact the anticipated
timing of data or regulatory submissions; the timing of the
initiation of clinical trials and trial cohorts at clinical trial
sites and patient enrollment rates may be delayed or slower than
anticipated; actions of regulatory agencies may affect the
initiation, timing and progress of clinical trials; risks related
to Blueprint Medicines' ability to obtain, maintain and enforce
patent and other intellectual property protection for its products
and current or future drug candidates it is developing; and
the success of Blueprint Medicines' current and future
collaborations, financing arrangements, partnerships or licensing
arrangements. Any forward-looking statements contained in this
press release represent Blueprint Medicines' views only as of the
date hereof and should not be relied upon as representing its views
as of any subsequent date. Except as required by law, Blueprint
Medicines explicitly disclaims any obligation to update any
forward-looking statements.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are
trademarks of Blueprint Medicines Corporation.
Blueprint Medicines
Corporation Selected Condensed Consolidated Balance Sheet
Data (in
thousands) (unaudited)
|
|
(1) Blueprint
Medicines defines working capital as current assets less current
liabilities.
|
|
|
|
|
|
|
|
|
December
31,
|
|
December
31,
|
|
|
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)
|
Blueprint Medicines
defines working capital as current assets less current
liabilities.
|
(2)
|
Includes both current
and long-term portions of the balance
|
Blueprint Medicines
Corporation Condensed Consolidated Statements of
Operations Data (in thousands, except per share
data) (unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
|
|
Years
Ended
|
|
|
December
31,
|
|
December
31,
|
|
|
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 –
Related Party
|
|
|
--
|
|
|
--
|
|
|
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
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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SOURCE Blueprint Medicines Corporation