CAMBRIDGE, Mass., March 9, 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 fourth quarter and year
ended December 31, 2016.
"2016 was a transformative year for Blueprint Medicines in which
we achieved all of our corporate goals, including a number of
important milestones," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines.
"When Blueprint Medicines began operations in 2011, we were
committed to building a diversified portfolio, grounded in our
differentiated scientific platform, with the potential to bring
innovative targeted therapies to patients in areas of high medical
need. In 2016 we began to realize this vision, and 2017 will be a
year where that vision comes into clearer focus. In 2016, we
announced proof-of-concept data from three Phase 1 trials in
genomically-defined patient populations, received FDA approval to
begin a Phase 1 trial for BLU-667, continued to expand our pipeline
and announced a collaboration with Roche that enables us to
accelerate and build upon our efforts in cancer immunotherapy.
Looking ahead to 2017, we expect multiple potential milestones,
including presenting updated data for our ongoing Phase 1 trials
for BLU-285 and BLU-554, which will provide further insight into
the safety, clinical activity and clinical development pathways for
these investigational medicines."
Clinical Programs:
BLU-554: Hepatocellular Carcinoma (HCC)
- In November 2016, Blueprint
Medicines presented data from its ongoing, global Phase 1 clinical
trial for BLU-554 in patients with advanced HCC at the
28th EORTC-NCI-AACR Symposium on Molecular Targets and
Cancer Therapeutics (EORTC-NCI-AACR). BLU-554 was designed as a
potent and selective inhibitor of FGFR4 and may offer the first
biomarker-driven targeted therapy for patients with advanced HCC.
The data demonstrated encouraging single agent clinical activity of
BLU-554 in heavily pre-treated advanced HCC patients as well as
pharmacodynamic evidence of FGFR4 pathway modulation. Read the full
data here. The maximum tolerated dose (MTD) was determined to be
600 mg once daily (QD), and enrollment in the dose expansion
portion of this clinical trial is ongoing at the MTD. Blueprint
Medicines anticipates providing updated data from this clinical
trial in 2017.
BLU-285: Gastrointestinal Stromal Tumors (GIST)
- In November 2016, Blueprint
Medicines presented data from its ongoing Phase 1 clinical trial
for BLU-285 in two patient populations with GIST, a rare disease
that is a sarcoma of the GI tract, in an oral presentation at
EORTC-NCI-AACR. BLU-285 is a unique, investigational medicine that
has been designed to target mutations in the PDGFRα and KIT genes,
which are two genetic drivers of GIST. The data showed that BLU-285
demonstrated significant preliminary clinical activity, including
tumor reductions in patients with PDGFRα-driven GIST beginning with
the first dose level and tumor reductions at higher dose levels in
refractory patients with KIT-driven GIST. The data also showed that
BLU-285 was well tolerated with no patients discontinuing treatment
due to adverse events. Read the full data here. Enrollment in the
escalation portion of this clinical trial has been completed, and
an MTD of 400 mg QD has been defined. Enrollment in the dose
expansion portion of this clinical trial is ongoing. Blueprint
Medicines anticipates providing updated data from this clinical
trial in 2017.
BLU-285: Systemic Mastocytosis (SM)
- In December 2016, Blueprint
Medicines presented data from its ongoing Phase 1 clinical trial
for BLU-285 in patients with advanced SM, a rare and severe blood
disease that affects the mast cells, at the 2016 American Society
of Hematology (ASH) Annual Meeting. As highlighted above, BLU-285
was designed to target mutations in the KIT gene, including the
D816V mutation, which is a key disease driver in approximately
90-95% of patients with SM. The data showed encouraging early
clinical activity for BLU-285 in patients with advanced SM,
beginning at the lowest dose levels, and marked decreases in
objective measures of mast cell burden. The data also showed that
BLU-285 was well tolerated, with no patients discontinuing
treatment due to adverse events. Read the full data here.
Enrollment in the dose escalation portion of this clinical trial is
ongoing. Blueprint Medicines anticipates providing updated data
from this clinical trial in 2017.
BLU-667: Non-Small Cell Lung Cancer (NSCLC), Medullary
Thyroid Carcinoma (MTC) and other advanced solid tumors with RET
alterations
- BLU-667 is a selective inhibitor of RET activating fusions,
mutations and predicted resistance mutations.
- In January 2017, Blueprint
Medicines announced that the FDA approved its investigational new
drug application for BLU-667 for the treatment of NSCLC, thyroid
cancer and other advanced solid tumors.
- Blueprint Medicines expects to initiate a Phase 1 clinical
trial for BLU-667 in the first half of 2017. 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 an MTD 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:
- Strengthened executive leadership team: In November 2016, Blueprint Medicines announced the
appointment of Marion Dorsch, Ph.D.
as Chief Scientific Officer and the transition of Christoph Lengauer, Ph.D., MBA, to the role of
Executive Vice President, where he is responsible for driving an
integrated approach to progressing Blueprint Medicines' portfolio
of investigational drugs.
- Closed Public Offering: In December 2016, 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 $25.00 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 approximately $134.5
million, after deducting underwriting discounts and
commissions and estimated offering expenses.
Fourth Quarter and Year End 2016 Financial Results:
- Cash Position: As of December 31, 2016, cash, cash equivalents and
investments were $268.2 million, as
compared to $162.7 million as of
December 31, 2015. This increase was
primarily due to $134.5 million in
net proceeds from the December 2016
follow-on underwritten public offering and an upfront cash payment
of $45.0 million in March 2016 upon execution of the collaboration
agreement with Roche, partially offset by cash used in operating
activities.
- Collaboration Revenue: Collaboration revenues
were $7.7 million for the fourth
quarter of 2016 and $27.8 million for
the year ended December 31, 2016, as
compared to $4.6 million for the
fourth quarter of 2015 and $11.4
million for the year ended December
31, 2015. This increase was due to increased activity in
Blueprint Medicines' collaboration with Alexion and Blueprint
Medicines entering into a collaboration with Roche in March 2016.
- R&D Expenses: Research and development
expenses were $24.1 million for the
fourth quarter of 2016 and $81.1
million for the year ended December
31, 2016, as compared to $16.4
million for the fourth quarter of 2015 and $48.6 million for the year ended December 31, 2015. This increase was primarily
attributable to increased manufacturing and clinical expenses
associated with advancing BLU-285 and BLU-554 into clinical trials,
increased personnel-related expenses, including stock-based
compensation expenses, and continuing to build Blueprint Medicines'
platform and advance its discovery pipeline.
- G&A Expenses: General and administrative expenses
were $5.0 million for the fourth
quarter of 2016 and $19.2 million for
the year ended December 31, 2016, as
compared to $3.6 million for the
fourth quarter of 2015 and $14.5
million for the year ended December
31, 2015. This increase was primarily attributable to
increased personnel-related expenses, including stock-based
compensation expense, and increased professional fees.
- Net Loss: Net loss was $21.3 for the fourth quarter of 2016 and
$72.5 million for the year ended
December 31, 2016, or a net loss per
share of $0.75 and $2.64, respectively, as compared to a net loss of
$15.6 million for the fourth quarter
of 2015 and $52.8 million for the
year ended December 31, 2015, or a
net loss per share of $0.58 and
$3.07, 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, will be sufficient to enable it to fund its
operating expenses and capital expenditure requirements into at
least late 2018.
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
66277428. 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 clinical
trials for subsets of patients with gastrointestinal stromal
tumors, hepatocellular carcinoma, systemic mastocytosis, non-small
cell lung cancer and medullary thyroid cancer, 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 the timing for initiation of Blueprint Medicines' Phase 1
clinical trial for BLU-667; 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 and other potential milestones in 2017;
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
product 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' Quarterly Report on Form 10-Q for the
quarter ended September 30, 2016, as
filed with the Securities and Exchange Commission (SEC) on
November 10, 2016, 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)
|
|
|
|
December
31,
|
|
December 31,
|
|
|
2016
|
|
2015
|
Cash, cash
equivalents and investments
|
|
$
|
268,218
|
|
$
|
162,707
|
Unbilled accounts
receivable
|
|
|
3,577
|
|
|
3,414
|
Working capital
(1)
|
|
|
191,913
|
|
|
151,776
|
Total
assets
|
|
|
282,795
|
|
|
178,898
|
Deferred
revenue
|
|
|
47,235
|
|
|
13,640
|
Term loan
payable
|
|
|
4,069
|
|
|
7,338
|
Lease incentive
obligation
|
|
|
3,370
|
|
|
3,948
|
Total stockholders'
equity
|
|
|
213,078
|
|
|
143,979
|
|
(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,
|
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
Collaboration
revenue
|
|
$
|
7,691
|
|
$
|
4,635
|
|
$
|
27,772
|
|
$
|
11,400
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
24,073
|
|
|
16,432
|
|
|
81,131
|
|
|
48,588
|
General and
administrative
|
|
|
4,991
|
|
|
3,624
|
|
|
19,218
|
|
|
14,456
|
Total operating
expenses
|
|
|
29,064
|
|
|
20,056
|
|
|
100,349
|
|
|
63,044
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income
(expense), net
|
|
|
201
|
|
|
6
|
|
|
551
|
|
|
(429)
|
Interest
expense
|
|
|
(91)
|
|
|
(160)
|
|
|
(469)
|
|
|
(696)
|
Total other income
(expense)
|
|
|
110
|
|
|
(154)
|
|
|
82
|
|
|
(1,125)
|
Net loss
|
|
$
|
(21,263)
|
|
$
|
(15,575)
|
|
$
|
(72,495)
|
|
$
|
(52,769)
|
Convertible preferred
stock dividends
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(3,153)
|
Net loss applicable
to common stockholders
|
|
$
|
(21,263)
|
|
$
|
(15,575)
|
|
$
|
(72,495)
|
|
$
|
(55,922)
|
Net loss per share
applicable to common stockholders — basic
and diluted
|
|
$
|
(0.75)
|
|
$
|
(0.58)
|
|
$
|
(2.64)
|
|
$
|
(3.07)
|
Weighted-average
number of common shares used in net loss per
share applicable to
common stockholders — basic and diluted
|
|
|
28,450
|
|
|
26,962
|
|
|
27,492
|
|
|
18,236
|
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visit:http://www.prnewswire.com/news-releases/blueprint-medicines-reports-fourth-quarter-and-full-year-2016-financial-results-300420776.html
SOURCE Blueprint Medicines