CAMBRIDGE, Mass., Feb. 17, 2021 /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, 2020.
"Last year was transformational for Blueprint Medicines, with
four regulatory approvals in the United
States and Europe, as we
executed on our foundational mission of delivering innovative
precision medicines to patients globally," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "In 2021, as we accelerate ongoing efforts to
drive the global adoption of AYVAKIT and GAVRETO, we look forward
to a potential U.S. approval for AYVAKIT for the most advanced and
deadly form of systemic mastocytosis for which no other precision
medicines are currently available. We also continue to focus on our
next wave of therapeutic candidates, including our two EGFR
inhibitors, which we anticipate will enter the clinic this year
with the goal of achieving rapid clinical proof-of-concept. In
addition, we continue to expand our research pipeline with new
assets that leverage our kinase inhibition expertise, and today, we
are excited to announce our newest research program that
selectively targets CDK2, a kinase involved in driving multiple
cancers."
Fourth Quarter 2020 Highlights and Recent Progress
AYVAKIT™/AYVAKYT® (avapritinib):
systemic mastocytosis (SM)
- Today announced the U.S. Food and Drug Administration (FDA) has
accepted our supplemental new drug application for AYVAKIT for
advanced systemic mastocytosis (SM). The FDA granted priority
review and set an action date of June 16,
2021 under the Prescription Drug User Fee Act.
- Today announced the submission of a Type II variation marketing
authorization application to the European Medicines Agency for
AYVAKYT for the treatment of adult patients with advanced SM.
- Received breakthrough therapy designation from the FDA for the
treatment of moderate to severe indolent SM, which encompasses the
majority of patients with SM. Avapritinib has also previously
received breakthrough therapy designation for the treatment of
patients with advanced SM.
- Presented new data at the virtual 62nd American
Society of Hematology (ASH) Annual Meeting highlighting new
proposed response criteria for advanced SM, the potential of highly
sensitive droplet digital PCR-based KIT D816V testing to accelerate
diagnosis across the spectrum of SM, and data from patients and
healthcare providers characterizing the significant disease burden
across the full spectrum of SM. Read the press release here.
AYVAKIT™/AYVAKYT® (avapritinib):
gastrointestinal stromal tumor (GIST)
- Recorded $6.0 million in net
product revenue during the fourth quarter of 2020 for
AYVAKIT/AYVAKYT, which was approved by the 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 for the
treatment of adult patients with unresectable or metastatic GIST
harboring the PDGFRA D842V mutation.
GAVRETO™ (pralsetinib): RET-altered
cancers
- Recorded $0.7 million in net
product revenue during the fourth quarter of 2020 for GAVRETO,
which was approved by the FDA in September
2020 for the treatment of adult patients with metastatic 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. Read the press release
announcing the expansion of the FDA label for GAVRETO into
RET-mutant MTC and RET fusion-positive thyroid cancer here.
BLU-263: systemic mastocytosis
- Reported positive top-line results from a Phase 1 trial in
healthy volunteers, showing that BLU-263 was well-tolerated across
a range of single- and multiple-ascending doses predicted to
potently inhibit D816V mutant KIT, the underlying SM disease
driver.
Research Portfolio:
- Today announced a highly selective and potent research program
with best-in-class potential targeting CDK2. CDK2, a
cyclin-dependent kinase involved in cell cycle biology, is
activated by its regulatory partner Cyclin E, and can drive cancer
cell proliferation when Cyclin E is aberrantly expressed.
Dysregulated Cyclin E is associated with multiple malignancies and
has been shown to be a mechanism of resistance to targeted
therapies, including CDK4/6 inhibitors.
- Nominated BLU-701, a potential first-in-class, selective,
brain-penetrant development candidate for treatment-resistant
double-mutant EGFR-driven NSCLC.
- Nominated a potential best-in-class development candidate
targeting MAP4K1, a kinase believed to play a role in T-cell
regulation, under the company's cancer immunotherapy collaboration
with Roche.
Corporate:
- Announced leadership transitions for the company's research and
development organization, including the promotion of Becker Hewes,
M.D. to Chief Medical Officer, effective January 11, 2021.
Key Upcoming Milestones
The company plans to achieve the following milestones by
mid-2021:
- Provide portfolio updates across clinical and research programs
at the AACR 2021 Annual Meeting.
- Obtain regulatory approval from the European Commission and
launch GAVRETO in RET fusion-positive NSCLC in Europe in the first half of 2021, under the
ongoing global collaboration with Roche.
- Initiate a Phase 1 trial of BLU-945, a triple-mutant EGFR
inhibitor, in patients with treatment-resistant EGFR-driven NSCLC
in the first half of 2021.
- Obtain FDA approval and launch AYVAKIT in advanced SM in the
U.S. in the first half 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 HARBOR trial of BLU-263, a next-generation
KIT inhibitor, in patients with non-advanced SM in mid-2021.
Fourth Quarter and Year End 2020 Financial Results
- Revenues: Revenues were $34.1
million for the fourth quarter of 2020, including
$6.0 million of net product revenues
from sales of AYVAKIT, $0.7 million
of net product revenues from sales of GAVRETO and $27.4 million in collaboration revenues. Revenues
for the year ended December 31, 2020
were $793.7 million, including
$21.2 million of net product revenues
from sales of AYVAKIT, $0.9 million
of net product revenues from sales of GAVRETO and $771.6 million in collaboration revenues.
Blueprint Medicines recorded $51.5
million and $66.5 million in
collaboration revenues in the fourth quarter and year ended
December 31, 2019, respectively.
- Cost of Sales: Cost of sales was $0.1 million for the fourth quarter of 2020 and
$0.4 million for the year ended
December 31, 2020. Blueprint
Medicines did not incur cost of sales in the fourth quarter or year
ended December 31, 2019, as no
product sales were generated during that period.
- R&D Expenses: Research and development expenses were
$77.4 million for the fourth quarter
of 2020 and $326.9 million for the
year ended December 31, 2020, as
compared to $88.6 million for the
fourth quarter of 2019 and $331.5
million for the year ended December
31, 2019. This decrease was primarily due to reimbursement
from the global development cost sharing arrangement under the
collaboration with Roche for pralsetinib. Research and development
expenses included $8.5 million in
stock-based compensation expenses for the fourth quarter of 2020
and $33.6 million in stock-based
compensation for the year ended December 31,
2020.
- SG&A Expenses: Selling, general and administrative
expenses were $42.5 million for the
fourth quarter of 2020 and $157.7
million for the year ended December
31, 2020, as compared to $32.3
million for the fourth quarter of 2019 and $96.4 million for the year ended December 31, 2019. This increase was primarily
due to increased costs and personnel expenses associated with
building Blueprint Medicines' commercial infrastructure for
AYVAKIT/AYVAKYT and GAVRETO, partially offset by reimbursement
under the collaboration with Roche for pralsetinib in connection
with the commercialization of GAVRETO in the U.S.. Selling, general
and administrative expenses included $11.0
million in stock-based compensation expenses for the fourth
quarter of 2020 and $41.9 million in
stock-based compensation for the year ended December 31, 2020.
- Net Income (Loss): Net loss was $85.7 million for the fourth quarter of 2020 and
net income was $313.9 million for the
year ended December 31, 2020, or a
diluted net loss per share of $1.53
and diluted net income per share of $5.59, respectively, as compared to a net loss of
$66.3 million for the fourth quarter
of 2019 and a net loss of $347.7
million for the year ended December
31, 2019, or a diluted net loss per share of $1.35 and $7.27,
respectively.
- Cash Position: As of December 31,
2020, cash, cash equivalents and marketable securities were
$1,549.7 million, as compared to
$548.0 million as of December 31, 2019. This increase was primarily
related to upfront payments of $775.0
million received in the third quarter of 2020 under
Blueprint Medicines' collaboration with Roche for pralsetinib,
$308.4 million in net proceeds
received from Blueprint Medicines' January
2020 follow-on underwritten public offering and $194.7 million in net proceeds received in the
fourth quarter of 2020 from Blueprint Medicines' "at the market"
stock offering program, partially offset by cash used in operating
activities.
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 2020 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 4844138. A webcast of the call will 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 three upcoming investor
conferences:
- 10th Annual SVB Leerink Global Healthcare
Conference on Thursday, February 25,
2021 at 11:20 a.m. ET.
- Cowen 41st Annual Health Care Conference on Wednesday, March 3, 2021 at 3:20 p.m. ET.
- Barclays Global Healthcare Conference on Wednesday, March 10, 2021 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 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 medicines for people with cancer and
hematologic disorders. Applying an approach that is both precise
and agile, we create therapies 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 our approved medicines to patients in the United
States and Europe, and we are globally advancing multiple
programs for genomically defined cancers, systemic mastocytosis,
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 timelines for marketing applications and
approvals, the initiation of clinical trials or the results of
ongoing and planned clinical trials; Blueprint Medicines' plans,
strategies and timelines to nominate development candidates; plans
and timelines for additional marketing applications for avapritinib
and pralsetinib and, if approved, commercializing avapritinib and
pralsetinib in additional geographies or for additional
indications; the potential benefits of any of Blueprint Medicines'
current or future approved drugs or drug candidates in treating
patients; the potential benefits of Blueprint
Medicines' collaboration with Roche
and Genentech for pralsetinib; and Blueprint
Medicines' 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, risks and uncertainties related to the impact of the
COVID-19 pandemic to Blueprint Medicines' business, operations,
strategy, goals and anticipated milestones, including Blueprint
Medicines' ongoing and planned research and discovery activities,
ability to conduct ongoing and planned clinical trials, clinical
supply of current or future drug candidates, commercial supply of
current or future approved products, and launching, marketing and
selling current or future approved products; Blueprint Medicines'
ability and plans in continuing to establish and expand a
commercial infrastructure, and successfully launching, marketing
and selling current or future approved products; Blueprint
Medicines' ability to successfully expand the approved indications
for AYVAKIT/AYVAKYT and GAVRETO or obtain marketing approval for
AYVAKIT/AYVAKYT in additional geographies in the future; the delay
of any current or planned clinical trials or the development of
Blueprint Medicines' current or future drug candidates; Blueprint
Medicines' advancement of multiple early-stage efforts; 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; the preclinical and
clinical results for Blueprint Medicines' drug candidates, which
may not support further development of such drug candidates;
actions of regulatory agencies, which may affect the initiation,
timing and progress of clinical trials; Blueprint Medicines'
ability to obtain, maintain and enforce patent and other
intellectual property protection for AYVAKIT/AYVAKYT, GAVRETO or
any drug candidates it is developing; Blueprint Medicines' ability
to develop and commercialize companion diagnostic tests for
AYVAKIT/AYVAKYT, GAVRETO or any of its current and future drug
candidates; and the success of Blueprint Medicines' current and
future collaborations, partnerships or licensing arrangements.
These and other risks and uncertainties are described in greater
detail in the section entitled "Risk Factors" in Blueprint
Medicines' filings with the Securities and Exchange Commission
(SEC), including Blueprint Medicines' most recent Annual Report on
Form 10-K, as supplemented by its most recent Quarterly Report on
Form 10-Q and any other filings that Blueprint Medicines has made
or may make with the SEC in the future. 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, GAVRETO and associated
logos are trademarks of Blueprint Medicines Corporation.
Blueprint
Medicines Corporation
Selected Condensed
Consolidated Balance Sheet Data
(in
thousands)
(unaudited)
|
|
|
|
|
|
|
|
|
December
31,
|
|
December
31,
|
|
|
2020
|
|
2019
|
Cash, cash
equivalents and marketable securities
|
$
|
1,549,722
|
|
$
|
547,960
|
Working capital
(1)
|
|
796,957
|
|
|
410,304
|
Total
assets
|
|
1,718,393
|
|
|
707,694
|
Deferred
revenue
|
|
41,158
|
|
|
46,073
|
Total
liabilities
|
|
248,305
|
|
|
243,335
|
Total stockholders'
equity
|
|
1,470,088
|
|
|
464,359
|
|
(1) Blueprint Medicines
defines working capital as current assets less current
liabilities.
|
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,
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue,
net
|
|
$
|
6,688
|
|
$
|
—
|
|
$
|
22,134
|
|
$
|
—
|
Collaboration
revenue
|
|
|
27,419
|
|
|
51,533
|
|
|
771,601
|
|
|
66,512
|
Total
revenues
|
|
$
|
34,107
|
|
$
|
51,533
|
|
$
|
793,735
|
|
$
|
66,512
|
Cost and operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
|
128
|
|
|
—
|
|
|
425
|
|
|
—
|
Research and
development
|
|
|
77,405
|
|
|
88,646
|
|
|
326,860
|
|
|
331,450
|
Selling, general and
administrative
|
|
|
42,541
|
|
|
32,265
|
|
|
157,743
|
|
|
96,388
|
Total cost and
operating expenses
|
|
$
|
120,074
|
|
$
|
120,911
|
|
$
|
485,028
|
|
$
|
427,838
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income,
net
|
|
|
936
|
|
|
2,990
|
|
|
6,599
|
|
|
13,732
|
Other income
(expense), net
|
|
|
50
|
|
|
57
|
|
|
(366)
|
|
|
(100)
|
Total other income
(expense)
|
|
$
|
986
|
|
$
|
3,047
|
|
$
|
6,233
|
|
$
|
13,632
|
Income (loss) before
income taxes
|
|
$
|
(84,981)
|
|
$
|
(66,331)
|
|
$
|
314,940
|
|
$
|
(347,694)
|
Income tax
expense
|
|
|
688
|
|
|
—
|
|
|
1,058
|
|
|
—
|
Net income
(loss)
|
|
$
|
(85,669)
|
|
$
|
(66,331)
|
|
$
|
313,882
|
|
$
|
(347,694)
|
Net income (loss) per
share applicable to common stockholders —
basic
|
|
$
|
(1.53)
|
|
$
|
(1.35)
|
|
$
|
5.76
|
|
$
|
(7.27)
|
Net income (loss) per
share applicable to common stockholders —
diluted
|
|
$
|
(1.53)
|
|
$
|
(1.35)
|
|
$
|
5.59
|
|
$
|
(7.27)
|
Weighted-average
number of common shares used in net income
(loss) per share applicable to
common stockholders — basic
|
|
|
56,072
|
|
|
49,218
|
|
|
54,534
|
|
|
47,829
|
Weighted-average
number of common shares used in net income
(loss) per share applicable to
common stockholders —diluted
|
|
|
56,072
|
|
|
49,218
|
|
|
56,168
|
|
|
47,829
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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SOURCE Blueprint Medicines Corporation