CAMBRIDGE, Mass., Feb. 26, 2019 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC), a precision therapy company
focused on genomically defined cancers, rare diseases and cancer
immunotherapy, today reported financial results and provided a
business update for the fourth quarter and full year ended
December 31, 2018.
![Print Print](https://mma.prnewswire.com/media/221118/blueprint_medicines_logo.jpg)
"Following a year of remarkable clinical progress across our
portfolio in 2018, we are focused on executing our '2020 Blueprint'
vision to transform Blueprint Medicines into a fully-integrated
global precision therapy company," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "The cornerstone of this effort is our planned
NDA submission for avapritinib for patients with PDGFRA Exon 18
mutant GIST and fourth-line GIST in the second quarter. As we work
to bring this new therapy to GIST patients who currently have no
approved treatment options, we are also partnering with treating
physicians, the patient community and testing companies to evolve
the GIST treatment paradigm toward precision medicine, with the
shared goals of maximizing patient outcomes, enabling efficient
clinical trials and delivering value to the healthcare system."
Fourth Quarter 2018 Highlights and Recent Progress:
Avapritinib: Gastrointestinal stromal tumors (GIST):
- Locked the registration database and reported top-line results
from the Phase 1 NAVIGATOR trial of avapritinib in patients with
PDGFRA Exon 18 mutant GIST and fourth-line GIST in preparation for
the planned submission of a New Drug Application (NDA) to the U.S.
Food and Drug Administration (FDA) in the second quarter of 2019.
Read the top-line data here.
- Presented updated data from the Phase 1 NAVIGATOR trial across
treatment lines at the Connective Tissue Oncology Society 2018
Annual Meeting in November 2018 and
disclosed plans to conduct the registration-enabling Phase 3
COMPASS-2L precision medicine trial of avapritinib in second-line
GIST. Read the full data here.
- Under Blueprint Medicines' collaboration with CStone
Pharmaceuticals, announced the China National Medical Products
Administration (NMPA) cleared an Investigational New Drug (IND)
application for the ongoing Phase 3 VOYAGER trial of avapritinib in
third-line GIST.
Avapritinib: Systemic mastocytosis (SM):
- Presented updated data from the Phase 1 EXPLORER trial at the
American Society of Hematology Annual Meeting and Exposition in
December 2018. Read the full data
here.
- Initiated patient dosing in two registration-enabling trials:
the Phase 2 PATHFINDER trial in advanced SM and the Phase 2 PIONEER
trial in indolent and smoldering SM.
BLU-667: RET-altered solid tumors:
- Today announced the FDA has granted Breakthrough Therapy
Designation to BLU-667 for the treatment of RET-mutation-positive
medullary thyroid cancer (MTC) that requires systemic treatment and
for which there are no acceptable alternative treatments.
BLU-554: Hepatocellular carcinoma (HCC):
- Under Blueprint Medicines' collaboration with CStone
Pharmaceuticals, announced the China NMPA cleared an IND
application for the ongoing Phase 1 trial of BLU-554 as a
monotherapy in advanced HCC.
BLU-782: Fibrodysplasia ossificans progressiva (FOP):
- Initiated participant dosing in a Phase 1 trial of BLU-782 in
healthy volunteers in the first quarter of 2019.
- Today announced the FDA has granted Fast Track Designation to
BLU-782 for the treatment of FOP.
Corporate:
- Announced "2020 Blueprint," a two-year global business strategy
under which Blueprint Medicines expects to have two marketed
products, four pending marketing applications in the United States or Europe, six clinical-stage therapeutic
candidates and eight research programs by the end of 2020.
- Announced the promotion of Michael
Landsittel to Chief Financial Officer and the promotion of
Kate Haviland to Chief Operating
Officer in February 2019.
Key Upcoming Milestones:
The company expects to achieve the following milestones by the
end of the second quarter of 2019.
- Submit an NDA for avapritinib for PDGFRA Exon 18 mutant GIST
and fourth-line GIST.
- Present the registration dataset for avapritinib in PDGFRA Exon
18 mutant GIST and fourth-line GIST.
- Present updated data from the Phase 1 EXPLORER trial of
avapritinib in advanced SM.
- Present updated data from the Phase 1 ARROW trial of BLU-667 in
RET-altered cancers.
- Complete enrollment of previously treated NSCLC and MTC patient
cohorts in the Phase 1 ARROW trial of BLU-667.
Fourth Quarter and Year End 2018 Financial Results:
- Cash Position: As of December 31,
2018, cash, cash equivalents and investments were
$494.0 million, as compared to
$673.4 million as of December 31, 2017. This decrease was primarily
related to cash used in operating activities, partially offset by
the $40.0 million upfront payment
received in connection with entering into the collaboration with
CStone Pharmaceuticals and the $10.0
million milestone payment achieved under the Roche
collaboration in June 2018.
- Collaboration Revenues: Collaboration revenues were
$1.0 million for the fourth quarter
of 2018 and $44.5 million for the
year ended December 31, 2018, as
compared to $1.6 million for the
fourth quarter of 2017 and $21.4
million for the year ended December
31, 2017. This increase for the year was primarily due to
revenue recognized under the collaboration agreement with CStone
Pharmaceuticals, partially offset by the termination of the Alexion
agreement in 2017.
- R&D Expenses: Research and development expenses were
$70.5 million for the fourth quarter
of 2018 and $243.6 million for the
year ended December 31, 2018, as
compared to $43.6 million for the
fourth quarter of 2017 and $144.7
million for the year ended December
31, 2017. This increase was primarily due to increased
clinical and manufacturing expenses driven by Blueprint Medicines'
lead development candidates and increased personnel-related
expenses. Research and development expenses included $4.9 million in stock-based compensation expenses
for the fourth quarter of 2018 and $17.0
million in stock-based compensation expenses for the year
ended December 31, 2018.
- G&A Expenses: General and administrative expenses
were $13.6 million for the fourth
quarter of 2018 and $47.9 million for
the year ended December 31, 2018, as
compared to $8.1 million for the
fourth quarter of 2018 and $28.0
million for the year ended December
31, 2017. This increase was primarily due to increased
personnel-related expenses and increased professional fees,
including pre-commercial planning activities. General and
administrative expenses included $3.9
million in stock-based compensation expenses for the fourth
quarter of 2018 and $13.5 million in
stock-based compensation expenses for the year ended December 31, 2018.
- Net Loss: Net loss was $80.3
million for the fourth quarter of 2018 and $236.6 million for the year ended December 31, 2018, or a net loss per share of
$1.83 and $5.39, respectively, as compared to a net loss of
$49.0 million for the fourth quarter
of 2017 and $148.1 million for the
year ended December 31, 2017, or a
net loss per share of $1.23 and
$3.92, respectively.
Financial Guidance:
Based on its current plans, Blueprint Medicines expects that its
existing cash, cash equivalents and investments, excluding any
potential option fees and milestone payments under its existing
collaborations with Roche and CStone Pharmaceuticals, will be
sufficient to enable it to fund its operating expenses and capital
expenditure requirements into the second half of 2020.
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 2018 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 26735762. A webcast of the conference call will be
available in the Investors 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.
About Blueprint Medicines:
Blueprint Medicines is a precision therapy company striving
to improve human health. With a focus on genomically defined
cancers, rare diseases and cancer immunotherapy, we are developing
transformational medicines rooted in our leading expertise in
protein kinases, which are proven drivers of disease. Our uniquely
targeted, scalable approach empowers the rapid design and
development of new treatments and increases the likelihood of
clinical success. We are currently advancing four investigational
medicines in clinical development, along with multiple research
programs. 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 and timelines for the development of avapritinib,
BLU-667, BLU-554 and BLU-782; the potential benefits of Blueprint
Medicines' current and future drug candidates in treating patients;
Blueprint Medicines' "2020 Blueprint" strategy, key goals and
anticipated milestones; plans and timelines for submitting an NDA
to the FDA for avapritinib; plans and timelines for presenting the
registration dataset for avapritinib in PDGFRA Exon 18 mutant GIST
and fourth-line GIST; plans and timelines for presenting updated
data from the Phase 1 EXPLORER trial of avapritinib in advanced SM;
plans and timelines for presenting updated data from the Phase 1
ARROW trial of BLU-667 in RET-altered cancers; plans and timelines
for completing previously treated NSCLC and MTC patient cohorts in
the Phase 1 ARROW trial of BLU-667; expectations regarding
Blueprint Medicines' existing cash, cash equivalents and
investments; and Blueprint Medicines' strategy, business plans and
focus. The words "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 delay of any
current or planned clinical trials or the development of Blueprint
Medicines' drug candidates, including avapritinib, BLU-667, BLU-554
and BLU-782; Blueprint Medicines' advancement of multiple
early-stage efforts; Blueprint Medicines' ability to successfully
demonstrate the safety and efficacy of its drug candidates; 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 develop and commercialize companion
diagnostic tests for its current and future drug candidates,
including companion diagnostic tests for avapritinib for PDGFRα
D842V-driven GIST, BLU-667 for RET-driven NSCLC and BLU-554 for
FGFR4-driven HCC; the success of Blueprint Medicines' current and
future collaborations, including its cancer immunotherapy
collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche
Inc. and its collaboration with CStone Pharmaceuticals. These and
other risks and uncertainties are described in greater detail in
the section entitled "Risk Factors" in Blueprint Medicines'
Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, as filed with the Securities
and Exchange Commission (SEC) on October 30,
2018, 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.
Blueprint
Medicines Corporation
|
Selected Condensed
Consolidated Balance Sheet Data
|
(in
thousands)
|
(unaudited)
|
|
|
|
December
31,
|
|
December 31,
|
|
|
|
2018
|
|
2017
|
|
Cash, cash
equivalents and investments
|
|
$
|
494,012
|
|
$
|
673,356
|
|
Working capital
(1)
|
|
|
439,464
|
|
|
642,615
|
|
Total
assets
|
|
|
540,124
|
|
|
715,737
|
|
Deferred
revenue
|
|
|
46,167
|
|
|
35,373
|
|
Term loan
payable
|
|
|
-
|
|
|
1,518
|
|
Lease incentive
obligation
|
|
|
14,617
|
|
|
16,331
|
|
Total stockholders'
equity
|
|
|
419,009
|
|
|
623,970
|
|
|
(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
|
|
Year
Ended
|
|
|
December
31,
|
|
December
31,
|
|
|
2018
|
|
2017
|
|
2018
|
|
2017
|
Collaboration
revenue
|
|
$
|
1,033
|
|
$
|
1,628
|
|
$
|
44,521
|
|
$
|
21,426
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
70,532
|
|
|
43,629
|
|
|
243,621
|
|
|
144,687
|
General and
administrative
|
|
|
13,643
|
|
|
8,092
|
|
|
47,928
|
|
|
27,986
|
Total operating
expenses
|
|
|
84,175
|
|
|
51,721
|
|
|
291,549
|
|
|
172,673
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income,
net
|
|
|
2,825
|
|
|
1,108
|
|
|
10,459
|
|
|
3,349
|
Interest
expense
|
|
|
(5)
|
|
|
(42)
|
|
|
(73)
|
|
|
(221)
|
Total other income
(expense)
|
|
|
2,820
|
|
|
1,066
|
|
|
10,386
|
|
|
3,128
|
Net loss
|
|
$
|
(80,322)
|
|
$
|
(49,027)
|
|
$
|
(236,642)
|
|
$
|
(148,119)
|
Net loss per
share — basic and diluted
|
|
$
|
(1.83)
|
|
$
|
(1.23)
|
|
$
|
(5.39)
|
|
$
|
(3.92)
|
Weighted-average
number of common shares used in net loss per share — basic and
diluted
|
|
|
43,994
|
|
|
39,988
|
|
|
43,867
|
|
|
37,793
|
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SOURCE Blueprint Medicines Corporation