CAMBRIDGE, Mass., May 3, 2017 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC), a leader in discovering and
developing targeted kinase medicines for patients with genomically
defined diseases, today reported financial results and
provided a business update for the first quarter ended March 31, 2017.
"In the first quarter of 2017, we expanded our clinical-stage
portfolio of drug candidates with the dosing of the first patient
in our Phase 1 trial of BLU-667, a selective RET inhibitor that
addresses both primary and predicted resistance mutations, while
also progressing our ongoing Phase 1 trials of BLU-554 and
BLU-285," said Jeff Albers, Chief
Executive Officer of Blueprint Medicines. "Our recent financing in
April further strengthened our balance sheet, and we believe that
we are well positioned to continue advancing our robust pipeline,
including three clinical-stage drug candidates and multiple
discovery programs, through important milestones. We look forward
to presenting updated Phase 1 data for BLU-285 in GIST and systemic
mastocytosis and BLU-554 in hepatocellular carcinoma later this
year, which will help inform and guide our future development
efforts for these programs."
Clinical Programs:
BLU-285: Gastrointestinal Stromal Tumors
- Blueprint Medicines continues to enroll patients in the dose
expansion stage of its ongoing Phase 1 clinical trial for BLU-285
in patients with advanced GIST and will present updated data from
this clinical trial in an oral presentation, "Clinical activity of
BLU-285 in advanced gastrointestinal stromal tumor (GIST)," at the
2017 ASCO Annual Meeting on Monday, June 5,
2017 in Chicago,
Illinois.
BLU-285: Systemic Mastocytosis
- Blueprint Medicines recently completed enrollment in the dose
escalation stage of its ongoing Phase 1 clinical trial for BLU-285
in patients with advanced systemic mastocytosis. Blueprint
Medicines anticipates initiating the dose expansion stage of this
clinical trial by the middle of 2017.
BLU-285: Recent Scientific Presentations
- In April 2017 at the Annual
Meeting of the American Association for Cancer Research (AACR),
Blueprint Medicines presented preclinical data demonstrating the
ability of BLU-285 to potently and selectively inhibit KIT and
PDGFRα activation loop mutants, which are key disease drivers for
GIST and systemic mastocytosis. BLU-285 also showed dose-dependent
tumor regression in multiple in vivo disease
models.
- In April 2017, Blueprint
Medicines presented on the discovery and development of BLU-285 at
the 253rd American Chemical Society National Meeting and
Exposition (ACS). The presentation included an in-depth mechanistic
overview of kinase activation loop mutants, and a discussion of
kinome-wide selectivity structure-activity relationship (SAR) and
the optimization of overall drug properties.
BLU-554: Hepatocellular Carcinoma
- In April 2017, Blueprint
Medicines presented on the discovery and development of BLU-554 at
ACS. The presentation detailed the discovery of BLU-554 using
structure-based drug design and SAR development towards the
optimization of overall drug properties for BLU-554.
BLU-667: Non-Small Cell Lung Cancer (NSCLC), Medullary
Thyroid Carcinoma (MTC) and other advanced solid tumors with RET
alterations
- In March 2017, Blueprint
Medicines dosed the first patient in its Phase 1 clinical trial for
BLU-667. This clinical trial is designed to evaluate the safety and
tolerability of BLU-667 in multiple ascending doses in patients
with NSCLC, MTC and other advanced solid tumors with the goal of
establishing a maximum tolerated dose or a recommended dose.
Following the identification of a dose and schedule for BLU-667,
Blueprint Medicines plans to open expansion cohorts for patients
with NSCLC, MTC and other advanced solid tumors with RET
alterations.
Corporate Highlights:
- Closed Public Offering: In April
2017, Blueprint Medicines announced the closing of an
underwritten public offering of 5,750,000 shares of its common
stock at a public offering price of $40.00 per share, including the exercise in full
by the underwriters of their option to purchase additional shares
of common stock. Blueprint Medicines received net proceeds from the
offering of $215.6 million, after
deducting underwriting discounts and commissions and offering
expenses.
First Quarter 2017 Financial Results:
- Cash Position: As of March
31, 2017, cash, cash equivalents and investments were
$236.3 million, as compared to
$268.2 million as of December 31, 2016. This decrease was primarily
related to cash used in operating activities.
- Collaboration Revenue: Collaboration revenues
were $5.8 million for the first
quarter of 2017, as compared to $6.8
million for the first quarter of 2016. This decrease was
primarily due to the recognition of a milestone under Blueprint
Medicines' collaboration with Alexion in the first quarter of 2016
as well as lower reimbursable research and development expenses
under the Alexion collaboration due to the timing of certain
research and development activities.
- R&D Expenses: Research and development
expenses were $28.5 million for the
first quarter of 2017, as compared to $17.6
million for the first quarter of 2016. This increase was
primarily attributable to increased clinical and manufacturing
expenses associated with advancing BLU-285, BLU-554 and BLU-667
into clinical trials and increased personnel-related expenses,
including stock-based compensation expenses.
- G&A Expenses: General and administrative expenses
were $5.6 million for the first
quarter of 2017, as compared to $4.6
million for the first quarter of 2016. This increase was
primarily attributable to increased personnel-related expenses,
including stock-based compensation expense.
- Net Loss: Net loss was $28.0
million for the first quarter of 2017, or a net loss per
share of $0.84, as compared to a net
loss of $15.5 million for the first
quarter of 2016, or a net loss per share of $0.57.
Financial Guidance:
Based on its current plans, Blueprint Medicines expects its
existing cash, cash equivalents and investments, including the net
proceeds from its April 2017
offering, but excluding any potential option fees and milestone
payments under its existing collaborations, will be sufficient to
enable it to fund its operating expenses and capital expenditure
requirements into the second half of 2019.
Conference Call Information
Blueprint Medicines will host a live conference call and
webcast today at 8:30 a.m. ET. The
conference call may be accessed by dialing 855-728-4793 (domestic)
or 503-343-6666 (international) and referring to conference ID
5771361. A webcast of the conference call will also be available in
the Investors section of 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 developing a new generation of
targeted and potent kinase medicines to improve the lives of
patients with genomically defined diseases. Its approach is rooted
in a deep understanding of the genetic blueprint of cancer and
other diseases driven by the abnormal activation of
kinases. Blueprint Medicines is advancing four programs
in clinical development for subsets of patients with
gastrointestinal stromal tumors, hepatocellular carcinoma, systemic
mastocytosis, non-small cell lung cancer, medullary thyroid cancer
and other advanced solid tumors, as well as multiple programs in
research and preclinical development. For more information, please
visit www.blueprintmedicines.com.
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 clinical development of
BLU-285, BLU-554 and BLU-667; the timing of updated clinical data
for Blueprint Medicines' Phase 1 clinical trials for BLU-285 and
BLU-554; 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 BLU-285, BLU-554 and BLU-667; Blueprint
Medicines' advancement of multiple early-stage efforts; Blueprint
Medicines' ability to successfully demonstrate the efficacy and
safety of its drug product candidates; the preclinical and clinical
results for Blueprint Medicines' drug product candidates, which may
not support further development of such drug product candidates;
and 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 BLU-554 with Ventana
Medical Systems, Inc. and for BLU-285 with QIAGEN Manchester
Limited; and the success of Blueprint Medicines' rare genetic
disease collaboration with Alexion Pharma Holding and its cancer
immunotherapy collaboration with F. Hoffmann-La Roche Ltd and
Hoffmann-La Roche Inc. 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, 2016, as filed
with the Securities and Exchange Commission (SEC) on March 9, 2017, and other filings that Blueprint
Medicines 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.
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,
|
|
|
2017
|
|
2016
|
Cash, cash
equivalents and investments
|
|
$
|
236,325
|
|
$
|
268,218
|
Unbilled accounts
receivable
|
|
|
2,826
|
|
|
3,577
|
Working capital
(1)
|
|
|
188,199
|
|
|
191,913
|
Total
assets
|
|
|
252,648
|
|
|
282,795
|
Deferred
revenue
|
|
|
44,220
|
|
|
47,235
|
Term loan
payable
|
|
|
3,246
|
|
|
4,069
|
Lease incentive
obligation
|
|
|
3,226
|
|
|
3,370
|
Total stockholders'
equity
|
|
|
188,222
|
|
|
213,078
|
|
|
|
|
|
|
|
(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,
|
|
|
2017
|
|
2016
|
Collaboration
revenue
|
|
$
|
5,840
|
|
$
|
6,856
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development
|
|
|
28,487
|
|
|
17,635
|
General and
administrative
|
|
|
5,683
|
|
|
4,646
|
Total operating
expenses
|
|
|
34,170
|
|
|
22,281
|
Other income
(expense):
|
|
|
|
|
|
|
Other income
(expense), net
|
|
|
425
|
|
|
61
|
Interest
expense
|
|
|
(72)
|
|
|
(140)
|
Total other income
(expense)
|
|
|
353
|
|
|
(79)
|
Net loss
|
|
$
|
(27,977)
|
|
$
|
(15,504)
|
Net loss per share
applicable to common stockholders — basic and
diluted
|
|
$
|
(0.84)
|
|
$
|
(0.57)
|
Weighted-average
number of common shares used in net loss per share
applicable to
common stockholders — basic and diluted
|
|
|
33,190
|
|
|
27,088
|
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SOURCE Blueprint Medicines