CAMBRIDGE, Mass., Oct. 30, 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 third quarter ended September 30, 2018.
"Our third quarter and recent accomplishments represent
tremendous progress across our portfolio, marked by the receipt of
positive regulatory feedback from the FDA for avapritinib and
BLU-667 and the presentation of new data across our clinical- and
research-stage pipeline," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines. "As
we prepare to enter a critical year, we are now accelerating our
efforts with a focus on executing five registration-enabling
studies across seven patient populations, building a global
commercial enterprise to deliver medicines to patients and
investing in the next generation of precision therapies."
Clinical Programs:
Avapritinib: Systemic Mastocytosis (SM)
- Blueprint Medicines announced that the U.S. Food and Drug
Administration (FDA) granted Breakthrough Therapy Designation to
avapritinib for the treatment of patients with advanced SM,
including the subtypes of aggressive SM, SM with an associated
hematologic neoplasm and mast cell leukemia.
- Blueprint Medicines announced that it has screened the first
patient in PATHFINDER, its registration-enabling, open-label,
single-arm Phase 2 clinical trial in patients with advanced SM.
Blueprint Medicines expects to initiate PIONEER, its
registration-enabling, randomized, placebo-controlled Phase 2
clinical trial in patients with indolent and smoldering SM, by the
end of 2018.
- Blueprint Medicines announced that the European Medicines
Agency has granted orphan drug designation to avapritinib for the
treatment of mastocytosis.
- Enrollment in the expansion portion of the Phase 1 EXPLORER
clinical trial for advanced SM is ongoing. Blueprint Medicines
plans to present data from this trial at the 60th
American Society for Hematology (ASH) Annual Meeting and Exposition
in December 2018.
Avapritinib: Gastrointestinal Stromal Tumors (GIST)
- Blueprint Medicines continues to evaluate avapritinib in its
Phase 1 NAVIGATOR clinical trial and will present updated data
across multiple patient populations, including PDGFRA-driven GIST,
third-line or later GIST, and second-line GIST, at the
2018 Connective Tissue Oncology Society (CTOS) Annual
Meeting on November 15, 2018. Based
on data from this trial, Blueprint Medicines plans to submit a new
drug application (NDA) to the FDA for avapritinib for the treatment
of patients with PDGFRα Exon 18 mutant GIST and fourth-line GIST in
the first half of 2019.
BLU-667: RET-Altered Solid Tumors
- Blueprint Medicines recently received written feedback from the
FDA supporting expedited development of BLU-667 and plans to submit
an NDA for BLU-667 in the first half of 2020 based on additional
data from the ongoing Phase 1 ARROW trial. Based on the feedback
from the FDA, Blueprint Medicines currently expects the NDA
submission will be for separate potential indications: (1) patients
with RET-fusion positive NSCLC and papillary thyroid cancer (PTC)
who have progressed following prior systemic therapy and (2)
patients with RET-mutant medullary thyroid cancer (MTC) who have
progressed following treatment with a tyrosine kinase
inhibitor.
- In October 2018, Blueprint
Medicines presented updated data from its ongoing Phase 1 ARROW
clinical trial of BLU-667 in patients with MTC and PTC at the
88th Annual Meeting of the American Thyroid Association.
The data showed that BLU-667 is highly active and well-tolerated in
these patient populations, with increased activity observed at
higher dose levels and longer treatment durations. Ninety percent
of evaluable patients with MTC and PTC experienced radiographic
tumor reductions, regardless of RET alteration or prior
multi-kinase inhibitor therapy. The response rate was 62 percent in
patients with MTC in the 300 and 400 milligram once daily dose
groups who were treated for at least 24 weeks. The data also showed
that BLU-667 was well-tolerated, and most adverse events reported
by investigators were Grade 1. Read the full data here.
- In September 2018, Blueprint
Medicines presented two clinical case studies demonstrating
proof-of-concept for BLU-667 in combination with Tagrisso®
(osimertinib) in patients with treatment-resistant, EGFR-mutant
NSCLC harboring an acquired RET fusion. The data showed that the
combination of BLU-667 and osimertinib overcame resistance to
standard therapy, and both patients achieved a partial response
with a 78 percent reduction in target tumors per RECIST version
1.1. In these two patients, the combination was well-tolerated, and
all reported adverse events were Grade 1 or 2. Read the full data
here.
BLU-554: Hepatocellular Carcinoma (HCC)
- In September 2018, Blueprint
Medicines and its partner, CStone Pharmaceuticals, submitted an
investigational new drug (IND) application for BLU-554 to Chinese
health authorities. Subject to approval of the IND application, the
companies plan to expand Blueprint Medicines' ongoing Phase 1
clinical trial of BLU-554 as a monotherapy for the treatment of
advanced HCC to include clinical sites in Mainland China.
Additionally, the companies plan to initiate a proof-of-concept
clinical trial evaluating BLU-554 in combination with CS1001, a
clinical-stage anti-PD-L1 immunotherapy, in 2019.
Research Programs:
BLU-782: Fibrodysplasia Ossificans Progressiva (FOP)
- In September 2018, Blueprint
Medicines presented preclinical proof-of-concept data for BLU-782,
an investigational precision therapy specifically designed to
target the underlying cause of FOP, at the 2018 American Society
for Bone and Mineral Research Annual Meeting. The data showed
that BLU-782 prevented injury- and surgery-induced heterotrophic
ossification, reduced edema and restored healthy tissue response to
muscle injury in a well-characterized FOP mouse model. Read the
full data here.
- Blueprint Medicines expects to submit an IND application to the
FDA for BLU-782 by the end of 2018, and subject to review of the
IND application, plans to initiate a Phase 1 clinical trial in
healthy volunteers in the first quarter of 2019. Upon completion of
the Phase 1 clinical trial, Blueprint Medicines plans to advance
BLU-782 into a registration-enabling Phase 2 clinical trial in
patients with FOP.
Corporate:
- In October 2018, Blueprint
Medicines announced the expansion of its leadership team with the
appointment of Christina Rossi as
Chief Commercial Officer. Blueprint Medicines also announced the
appointment of Paul Beresford as
General Manager, International.
Third Quarter Financial Results:
- Cash Position: As of September
30, 2018, cash, cash equivalents and investments were
$559.6 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
$1.1 million for the third quarter of
2018, as compared to $8.1 million for
the third quarter of 2017. This decrease was primarily due to the
termination of the Alexion agreement in 2017.
- R&D Expenses: Research and development expenses were
$64.6 million for the third quarter
of 2018, as compared to $39.3 million
for the third quarter of 2017. This increase was primarily
attributable to increased clinical and manufacturing expenses
associated with advancing avapritinib and BLU-667 further through
clinical trials and increased personnel-related expenses. Research
and development expenses included $4.8
million in stock-based compensation expenses for the third
quarter of 2018.
- G&A Expenses: General and administrative expenses
were $12.0 million for the third
quarter of 2018, as compared to $7.4
million for the third quarter of 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 $3.6 million in stock-based compensation expenses
for the third quarter of 2018.
- Net Loss: Net loss was $72.7
million for the third quarter of 2018, or a net loss per
share of $1.66, as compared to a net
loss of $37.7 million for the third
quarter of 2017, or a net loss per share of $0.96.
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.
Earnings Conference Call Information:
Blueprint Medicines will host a live conference call and
webcast at 8:30 a.m. ET today to discuss third quarter 2018
financial results and recent business activities. The conference
call may be accessed by dialing (855) 626-8618 (domestic) or (531)
289-2784 (international) and referring to conference ID 7598866. 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.
CTOS Conference Call Information:
Blueprint Medicines will host a live conference call and webcast
to discuss data being presented at the 2018 CTOS Annual Meeting on
November 15th at
7:30 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 3479587. 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 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 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
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 initiating Blueprint
Medicines' PIONEER trial; the potential benefits of receiving
Breakthrough Therapy Designation for avapritinib for the treatment
of patients with advanced SM, including the subtypes of aggressive
SM, SM with an associated hematologic neoplasm and mast cell
leukemia; plans to present data from the Phase 1 EXPLORER clinical
trial; plans and timelines for submitting an NDA to the FDA for
avapritinib; plans and timelines for submitting an NDA to the FDA
for BLU-667; plans and timelines for expanding Blueprint Medicines'
ongoing Phase 1 clinical trial of BLU-554 monotherapy to include
clinical sites in Mainland China; plans and timelines for
initiating a proof-of-concept clinical trial evaluating BLU-554 in
combination with CS1001; plans and timelines for submitting an IND
application to the FDA for BLU-782; plans and timelines for
initiating a Phase 1 clinical trial for BLU-782 in healthy
volunteers; plans to work with clinical experts and the patient
community to design a potential Phase 2 clinical trial of BLU-782
in patients with FOP; 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. and Blueprint
Medicines' 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 June 30, 2018, as filed with the Securities and
Exchange Commission (SEC) on August 1,
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.
Trademarks:
Tagrisso® is a registered trademark of AstraZeneca plc.
All other trademarks and trade names in this press release are the
property of Blueprint Medicines Corporation.
Blueprint
Medicines Corporation
Selected Condensed
Consolidated Balance Sheet Data
(in
thousands)
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
September
30
|
|
December 31,
|
|
|
|
2018
|
|
2017
|
|
Cash, cash
equivalents and investments
|
|
$
|
559,636
|
|
$
|
673,356
|
|
Working capital
(1)
|
|
|
509,378
|
|
|
642,615
|
|
Total
assets
|
|
|
606,110
|
|
|
715,737
|
|
Deferred
revenue
|
|
|
47,200
|
|
|
35,373
|
|
Term loan
payable
|
|
|
277
|
|
|
1,518
|
|
Lease incentive
obligation
|
|
|
15,046
|
|
|
16,331
|
|
Total stockholders'
equity
|
|
|
489,296
|
|
|
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
September
30,
|
|
Nine Months
Ended
September
30,
|
|
|
2018
|
|
2017
|
|
2018
|
|
2017
|
Collaboration
revenue
|
|
$
|
1,095
|
|
$
|
8,068
|
|
$
|
43,488
|
|
$
|
19,798
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
64,562
|
|
|
39,300
|
|
|
173,089
|
|
|
101,058
|
General and
administrative
|
|
|
12,041
|
|
|
7,378
|
|
|
34,285
|
|
|
19,894
|
Total operating
expenses
|
|
|
76,603
|
|
|
46,678
|
|
|
207,374
|
|
|
120,952
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income,
net
|
|
|
2,799
|
|
|
954
|
|
|
7,635
|
|
|
2,240
|
Interest
expense
|
|
|
(14)
|
|
|
(47)
|
|
|
(69)
|
|
|
(178)
|
Total other
income
|
|
|
2,785
|
|
|
907
|
|
|
7,566
|
|
|
2,062
|
Net loss
|
|
$
|
(72,723)
|
|
$
|
(37,703)
|
|
$
|
(156,320)
|
|
$
|
(99,092)
|
Net loss per share —
basic and diluted
|
|
$
|
(1.66)
|
|
$
|
(0.96)
|
|
$
|
(3.57)
|
|
$
|
(2.67)
|
Weighted-average
number of common shares used in
net loss per share — basic and
diluted
|
|
|
43,915
|
|
|
39,130
|
|
|
43,825
|
|
|
37,053
|
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SOURCE Blueprint Medicines Corporation