CAMBRIDGE, Mass., May 2, 2018 /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 quarter ended March 31, 2018.
"In the first quarter, we continued to make significant progress
across our portfolio toward our vision of rapidly delivering
potentially transformative kinase medicines to patients with
genomically defined diseases," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines. "In
particular, we were excited to present initial clinical
proof-of-concept data for our highly selective RET inhibitor
BLU-667, which showed consistent clinical activity in patients with
multiple tumor types, RET alterations and prior therapies, along
with a favorable safety and tolerability profile. In addition, we
received positive feedback from the FDA supporting our registration
plan in systemic mastocytosis, including support for a single-arm
registration-enabling Phase 2 trial in patients with advanced
systemic mastocytosis representing a potential expedited path to
registration."
Clinical Programs:
Avapritinib: Gastrointestinal Stromal Tumors (GIST)
- In March 2018, Blueprint
Medicines completed enrollment of the PDGFRα D842V expansion cohort
of its ongoing Phase 1 NAVIGATOR clinical trial. Based on feedback
from the U.S. Food and Drug Administration (FDA) at an End-of-Phase
1 meeting in 2017, Blueprint Medicines believes that data from the
PDGFRα D842V expansion cohort may be sufficient to support a New
Drug Application (NDA) for avapritinib for the treatment of
patients with PDGFRα D842V-driven GIST. Based on the expected
timeline for the collection of data, Blueprint Medicines now
anticipates it will submit an initial NDA to the FDA for
avapritinib in the first half of 2019. In the first quarter,
Blueprint Medicines announced it had completed enrollment of the
third-line or later (KIT-driven) GIST cohort and initiated
enrollment of the second-line GIST cohort in the Phase 1 NAVIGATOR
trial. Blueprint Medicines anticipates presenting updated data from
the NAVIGATOR trial in the second half of 2018.
Avapritinib: Advanced Systemic Mastocytosis (SM)
- Blueprint Medicines recently received positive feedback from
the FDA supporting its proposed registration plan for avapritinib
in patients with advanced, smoldering and indolent SM. Consistent
with feedback from the FDA, Blueprint Medicines plans to initiate a
registration-enabling open-label, single-arm Phase 2 clinical trial
in patients with advanced SM, called the PATHFINDER trial, by the
middle of 2018. In addition, Blueprint Medicines plans to initiate
a registration-enabling Phase 2 clinical trial in patients with
indolent SM and smoldering SM, called the PIONEER trial, by the end
of 2018.
- Enrollment in the expansion portion of the Phase 1 EXPLORER
clinical trial for advanced SM is ongoing, and Blueprint Medicines
anticipates presenting updated data from this trial in the second
half of 2018.
BLU-667: RET-Altered Solid Tumors
- In April 2018, Blueprint
Medicines presented proof-of-concept data from its ongoing Phase 1
ARROW clinical trial of BLU-667 in patients with RET-altered
non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC)
and other advanced solid tumors at the American Association for
Cancer Research (AACR) Annual Meeting. The data showed broad and
robust clinical activity across multiple tumor types and RET
genotypes, including in patients whose disease had progressed on
prior multi-kinase therapy. Radiographic tumor reductions were
observed in 84 percent of patients with RET-altered solid tumors
and measurable target lesions, and preliminary overall response
rates were 50 percent and 40 percent in patients with NSCLC and
MTC, respectively. The data also showed that BLU-667 was generally
well-tolerated, and most adverse events reported by investigators
were Grade 1. Read the full data here. The maximum tolerated dose
(MTD) was determined to be 400 mg once daily, and global enrollment
in the dose expansion portion of the Phase 1 ARROW clinical trial
is ongoing.
- In April 2018, Blueprint
Medicines announced the publication of foundational preclinical
data and clinical proof-of-concept results for BLU-667. The
publication outlined preclinical data characterizing the potency
and selectivity of BLU-667 against multiple oncogenic RET variants
and resistant mutants and anti-tumor activity in multiple solid
tumor models. It also included four patient vignettes from the
ongoing Phase 1 ARROW clinical trial, showing clinical responses in
patients with RET-KIF5B-altered NSCLC and MTC harboring multiple
RET mutations. The paper, titled "Precision targeted therapy with
BLU-667 for RET-driven cancers," was published online in
Cancer Discovery.
Research Programs:
- In the first quarter of 2018, Blueprint Medicines initiated
investigational new drug application-enabling studies for BLU-782,
its development candidate for the treatment of patients with
fibrodysplasia ossificans progressiva. Blueprint Medicines plans to
report preclinical data for the program in 2018.
- Blueprint Medicines also recently nominated a new wholly-owned
discovery program for an undisclosed kinase target.
First Quarter Financial Results:
- Cash Position: As of March 31,
2018, cash, cash equivalents and investments were
$621.1 million, as compared to
$673.4 million as of December 31, 2017. This decrease was primarily
related to cash used in operating activities.
- Collaboration Revenues: Collaboration revenues were
$0.9 million for the first quarter of
2018, as compared to $5.8 million for
the first quarter of 2017. This decrease was primarily due to the
termination of the Alexion agreement in 2017, which resulted in no
revenue recognized under this agreement in the first quarter 2018,
as well as the impact on revenue recognized under the Roche
agreement as a result of the adoption of Accounting Standards
Codification 606 effective January 1,
2018.
- R&D Expenses: Research and development expenses were
$50.0 million for the first quarter
of 2018, as compared to $28.5 million
for the first quarter of 2017. This increase was primarily
attributable to increased clinical and manufacturing expenses
associated with advancing avapritinib, BLU-554, and BLU-667 further
through clinical trials and increased personnel-related expenses.
Research and development expenses included $3.0 million in stock-based compensation expenses
for the first quarter of 2018.
- G&A Expenses: General and administrative expenses
were $9.9 million for the first
quarter of 2018, as compared to $5.7
million for the first quarter of 2017. This increase was
primarily attributable to increased personnel-related expenses and
increased professional fees, including pre-commercial planning
activities. General and administrative expenses included
$2.5 million in stock-based
compensation expenses for the first quarter of 2018.
- Net Loss: Net loss was $56.5
million for the first quarter of 2018, or a net loss per
share of $1.29, as compared to a net
loss of $28.0 million for the first
quarter of 2017, or a net loss per share of $0.84.
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
collaboration with Roche, will be sufficient to enable it to fund
its operating expenses and capital expenditure requirements into
the middle of 2020.
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 7572918. 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 conference 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 disease driven by the abnormal activation of kinases.
Blueprint Medicines is advancing multiple 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
avapritinib, BLU-554, BLU-667 and BLU-782; the potential benefits
of Blueprint Medicines' current and future drug candidates in
treating patients; plans and timelines for presenting preclinical
and clinical data for Blueprint Medicines' current or future
clinical trials; plans and timelines for presenting preclinical
data for the BLU-782 program; plans and timelines for initiating
Blueprint Medicines' PATHFINDER and PIONEER trials; plans and
timelines for engaging regulatory authorities to obtain input on
registration pathways for avapritinib and BLU-667; expectations
regarding the potential for current and future clinical trials to
be registration-enabling for Blueprint Medicines' current and
future drug candidates in one or more indications; expectations
regarding the collection of data for the PDGFRα D842V-driven GIST
cohort of the NAVIGATOR trial; plans and timelines for submitting
an NDA to the FDA for avapritinib; expectations regarding potential
regulatory and commercial milestones; 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-554, BLU-667
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 BLU-554 for FGFR4-driven
HCC, avapritinib for PDGFRα D842V-driven GIST and BLU-667 for
RET-driven NSCLC; and the success of Blueprint Medicines' 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, 2017, as filed
with the Securities and Exchange Commission (SEC) on February 21, 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)
|
|
|
|
March
31
|
|
December 31,
|
|
|
2018
|
|
2017
|
Cash, cash
equivalents and investments
|
|
$
|
621,123
|
|
$
|
673,356
|
Working capital
(1)
|
|
|
575,700
|
|
|
642,615
|
Total
assets
|
|
|
664,468
|
|
|
715,737
|
Deferred
revenue
|
|
|
39,734
|
|
|
35,373
|
Term loan
payable
|
|
|
1,106
|
|
|
1,518
|
Lease incentive
obligation
|
|
|
15,903
|
|
|
16,331
|
Total stockholders'
equity
|
|
|
570,873
|
|
|
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
|
|
|
March
31,
|
|
|
2018
|
|
2017
|
Collaboration
revenue
|
|
$
|
954
|
|
$
|
5,840
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development
|
|
|
49,954
|
|
|
28,487
|
General and
administrative
|
|
|
9,911
|
|
|
5,683
|
Total operating
expenses
|
|
|
59,865
|
|
|
34,170
|
Other income
(expense):
|
|
|
|
|
|
|
Other income,
net
|
|
|
2,394
|
|
|
425
|
Interest
expense
|
|
|
(32)
|
|
|
(72)
|
Total other
income
|
|
|
2,362
|
|
|
353
|
Net loss
|
|
$
|
(56,549)
|
|
$
|
(27,977)
|
Net loss per share
applicable to common stockholders — basic and
diluted
|
|
$
|
(1.29)
|
|
$
|
(0.84)
|
Weighted-average
number of common shares used in net loss per share
applicable to common stockholders — basic
and diluted
|
|
|
43,700
|
|
|
33,190
|
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SOURCE Blueprint Medicines Corporation