CAMBRIDGE, Mass., May 9, 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 quarter ended March
31, 2019.
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"Based on significant clinical and regulatory progress in the
first quarter, we accelerated multiple programs and advanced our
'2020 Blueprint' strategy to transform Blueprint Medicines into a
fully-integrated precision therapy company," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "We are especially encouraged that the FDA
granted Breakthrough Therapy Designation to BLU-667 for
RET-fusion-positive NSCLC, and we look forward to presenting
updated data from our Phase 1 ARROW trial at the ASCO Annual
Meeting next month. In addition, our follow-on public offering in
April further strengthened our financial position, enabling us to
continue to build the company ahead of multiple planned marketing
applications for avapritinib and BLU-667 in the United States and Europe over the next 18 months."
First Quarter 2019 Highlights and Recent Progress:
Avapritinib: Gastrointestinal stromal tumors (GIST):
- Announced plans to submit a marketing authorization application
(MAA) to the European Medicines Agency (EMA) for
avapritinib for the treatment of PDGFRα D842V mutant GIST and
fourth-line GIST in the third quarter of 2019.
Avapritinib: Systemic mastocytosis (SM):
- Announced plans to accelerate the submission of a new drug
application (NDA) to the U.S. Food and Drug Administration (FDA)
for avapritinib for the treatment of advanced SM in the first
quarter of 2020, subject to continuing discussions with
the FDA to determine the required clinical data for an NDA
submission.
BLU-667: RET-altered solid tumors:
- Received FDA Breakthrough Therapy Designation for BLU-667 for
the treatment of patients with RET fusion-positive non-small cell
lung cancer (NSCLC) that has progressed following platinum-based
chemotherapy.
- Announced top-line interim data from the Phase 1 ARROW trial of
BLU-667 in patients with previously treated RET-fusion NSCLC and
previously treated RET-mutant medullary thyroid cancer (MTC). Read
the full data here.
- Achieved enrollment targets for registration-enabling ARROW
trial cohorts for patients with previously treated RET-fusion NSCLC
and previously treated RET-mutant MTC. Based on the early
achievement of the enrollment target for the RET-fusion NSCLC
cohort, Blueprint Medicines plans to submit an NDA to the FDA for
BLU-667 for the treatment of patients with NSCLC previously treated
with platinum-based chemotherapy in the first quarter of 2020.
Blueprint Medicines continues to expect to submit an NDA to the FDA
for BLU-667 for the treatment of patients with RET-mutant MTC
previously treated with an approved multi-kinase inhibitor in the
first half of 2020.
BLU-554: Advanced hepatocellular carcinoma (HCC)
- Dosed the first patient in China in the ongoing Phase 1 clinical trial of
BLU-554 in patients with advanced HCC, under Blueprint Medicines'
collaboration with CStone Pharmaceuticals.
BLU-782: Fibrodysplasia ossificans progressiva (FOP):
- Initiated a Phase 1 clinical trial for BLU-782 in healthy
volunteers and, based on the progress of the ongoing trial and
input from clinical experts, announced plans to initiate a Phase 2
clinical trial of BLU-782 in patients with FOP in the fourth
quarter of 2019.
Corporate:
- Closed an underwritten public offering of 4,662,162 shares of
common stock at a public offering price of $74.00 per share, including the exercise in full
by the underwriters of their option to purchase additional shares
of common stock. Blueprint Medicines received estimated net
proceeds of approximately $327.2 million, after deducting underwriting
discounts and commissions and estimated offering expenses.
Key Upcoming Milestones:
The company expects to achieve the following near-term
milestones:
- Submit an NDA to the FDA and an MAA to the EMA for avapritinib
for the treatment of patients with PDGFRA Exon 18 mutant GIST and
fourth-line GIST in the second quarter and third quarter of 2019,
respectively.
- Present the registration dataset for avapritinib in PDGFRA Exon
18 mutant GIST and fourth-line GIST at the 2019 American Society of
Clinical Oncology (ASCO) Annual Meeting.
- Present updated data from the Phase 1 EXPLORER trial of
avapritinib in advanced SM at the 24th Annual Congress
of the European Hematology Society.
- Present updated data from the Phase 1 ARROW trial of BLU-667 in
RET-altered cancers at the 2019 ASCO Annual Meeting.
First Quarter 2019 Financial Results:
- Cash Position: As of March 31,
2019, cash, cash equivalents and investments were
$415.9 million, as compared to
$494.0 million as of December 31, 2018. This decrease was primarily
related to cash used in operating activities. Cash, cash
equivalents and investments as of March 31,
2019 do not include the estimated net proceeds of
approximately $327.2 million from the
company's follow-on underwritten public offering of common stock,
which closed in April 2019.
- Collaboration Revenues: Collaboration revenues were
$0.7 million for the first quarter of
2019, as compared to $1.0 million for
the first quarter of 2018. This decrease was primarily due to
revenue recorded under the Roche collaboration.
- R&D Expenses: Research and development expenses were
$74.3 million for the first quarter
of 2019, as compared to $50.0 million
for the first quarter of 2018. This increase was primarily due to
increased clinical and manufacturing expenses driven by our lead
development candidates and increased personnel-related expenses.
Research and development expenses included $5.8 million in stock-based compensation expenses
for the first quarter of 2019.
- G&A Expenses: General and administrative expenses
were $16.6 million for the first
quarter of 2019, as compared to $9.9
million for the first quarter of 2018. This increase was
primarily due to increased personnel-related expenses,
commercial-readiness activities and increased other professional
fees. General and administrative expenses included $4.5 million in stock-based compensation expenses
for the first quarter of 2019.
- Net Loss: Net loss was $87.4
million for the first quarter of 2019, or a net loss per
share of $1.98, as compared to a net
loss of $56.5 million for the first
quarter of 2018, or a net loss per share of $1.29.
Financial Guidance:
Based on its current plans, Blueprint Medicines expects that its
existing cash, cash equivalents and investments, including the
estimated net proceeds of approximately $327.2 million from its April 2019 follow-on public offering but
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 middle of 2021.
Conference Call Information:
Blueprint Medicines will host a live conference call and
webcast at 8:30 a.m. ET today to discuss first quarter 2019
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 9671728. 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; plans and timelines for submitting an
NDA to the FDA and an MAA to the EMA for avapritinib for the
treatment of GIST; plans and timelines for presenting the
registration dataset for avapritinib in PDGFRA Exon 18 mutant GIST
and fourth-line GIST; plans and timelines for submitting an NDA to
the FDA for avapritinib for the treatment of advanced SM; plans and
timelines for presenting updated data from the Phase 1 EXPLORER
trial of avapritinib in advanced SM; plans and timelines for
submitting NDAs to the FDA for BLU-667 for the treatment of
RET-fusion NSCLC and RET-fusion MTC; plans and timelines for
presenting updated data from the Phase 1 ARROW trial of BLU-667 in
RET-altered cancers; expectations regarding the results from the
Phase 1 clinical trial of BLU-782 in patients with FOP; plans and
timelines for initiating a Phase 2 clinical trial of BLU-782 in
patients with FOP; plans and timelines for patient enrollment in
China in the ongoing global Phase
1 trial of BLU-554 in patients with advanced HCC; the
potential benefits of Blueprint Medicines' current and future drug
candidates in treating patients; expectations regarding Blueprint
Medicines' existing cash, cash equivalents and investments; and
Blueprint Medicines' strategy, goals and anticipated milestones,
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 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 develop and commercialize companion diagnostic tests for
its current and future drug candidates; and 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' Annual Report on Form 10-K for the year
ended December 31, 2018, as filed
with the Securities and Exchange Commission (SEC) on February 26, 2019, 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)
|
|
|
|
March
31,
|
|
December 31,
|
|
|
2019
|
|
2018
|
Cash, cash
equivalents and investments
|
|
$
|
415,856
|
|
$
|
494,012
|
Working capital
(1)
|
|
|
361,753
|
|
|
439,464
|
Total
assets
|
|
|
544,663
|
|
|
540,124
|
Deferred
revenue
|
|
|
45,436
|
|
|
46,167
|
Total
liabilities
|
|
|
200,450
|
|
|
121,115
|
Total stockholders'
equity
|
|
|
344,213
|
|
|
419,009
|
|
(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
|
|
|
March
31,
|
|
|
2019
|
|
2018
|
Collaboration
revenue
|
|
$
|
730
|
|
$
|
954
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development
|
|
|
74,250
|
|
|
49,954
|
General and
administrative
|
|
|
16,553
|
|
|
9,911
|
Total operating
expenses
|
|
|
90,803
|
|
|
59,865
|
Other income
(expense):
|
|
|
|
|
|
|
Other income,
net
|
|
|
2,669
|
|
|
2,394
|
Interest
expense
|
|
|
(3)
|
|
|
(32)
|
Total other
income
|
|
|
2,666
|
|
|
2,362
|
Net loss
|
|
$
|
(87,407)
|
|
$
|
(59,549)
|
Net loss per
share — basic and diluted
|
|
$
|
(1.98)
|
|
$
|
(1.29)
|
Weighted-average
number of common shares used in net loss per share — basic and
diluted
|
|
|
44,097
|
|
|
43,700
|
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SOURCE Blueprint Medicines