CAMBRIDGE, Mass., Nov. 5, 2019 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ:BPMC), a precision therapy company
focused on genomically defined cancers, rare diseases and cancer
immunotherapy, is hosting its first R&D Day in
New York City today.
During the event, Blueprint Medicines will outline its vision to
become a leading platform-enabled, fully-integrated, global
precision therapy company. The R&D Day presentation will
highlight opportunities to expand the reach of the company's
therapeutic candidates to broader patient populations, integrate
and scale scientific, clinical and commercial capabilities to build
therapeutic area leadership, and fully utilize the company's
scientific platform to design innovative medicines targeting novel
kinase biology. In addition, today the company reported financial
results and provided a business update for the quarter ended
September 30, 2019.
"As we prepare to launch our first medicine and submit multiple
additional marketing applications next year, today we are unveiling
our next wave of internally discovered research and clinical-stage
precision therapies with the potential to deliver durable clinical
benefits to additional patient populations," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "By fully leveraging our integrated research
capabilities and reinvesting insights from our ongoing clinical
programs, we continue to build a powerful research engine with the
potential to deliver transformative treatment advances to patients
as well as rapid and sustainable growth to Blueprint
Medicines."
R&D Day Presentation Areas of Focus
- Highlight the significant medical need in indolent systemic
mastocytosis (SM), a rare disease characterized by debilitating and
unpredictable symptoms despite best available therapy. Based on an
improved understanding of the disease, Blueprint Medicines now
estimates there are approximately 75,000 patients with SM in the
major markets, which consist of the
United States, France,
Germany, Italy, Spain,
United Kingdom and Japan.
- Announce a comprehensive strategy to address a broad population
of patients with SM and other mast cell disorders with the
company's drug candidates avapritinib and BLU-263, a
next-generation KIT inhibitor. Blueprint Medicines plans to submit
an investigational new drug (IND) application to the U.S. Food and
Drug Administration (FDA) for BLU-263 for indolent SM in the first
half of 2020.
- Introduce two research programs targeting well-characterized
resistance mutations in patients with EGFR-driven non-small cell
lung cancer (NSCLC), highlighting Blueprint Medicines'
differentiated capability for designing highly selective
investigational medicines that address tumor evolution and
resistance to targeted therapy.
- Highlight a research program under Blueprint Medicines' cancer
immunotherapy collaboration with Roche targeting MAP4K1, which is
believed to play a role in T cell regulation.
Third Quarter 2019 Highlights and Recent Progress
Avapritinib: Gastrointestinal stromal tumors (GIST)
- Completed target enrollment in the Phase 3 VOYAGER trial of
avapritinib versus regorafenib in patients with third- and
fourth-line GIST.
- Announced the FDA intends to administratively split the new
drug application (NDA) for avapritinib into two separate NDAs (one
for PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and
one for fourth-line GIST) and requested top-line data from the
ongoing Phase 3 VOYAGER trial to inform its review of the proposed
fourth-line GIST indication. The PDUFA action date for both
indications is currently February 14,
2020. For the fourth-line indication, an extension of up to
three months for the PDUFA action date will likely be required to
enable Blueprint Medicines to provide the top-line VOYAGER data to
the FDA.
Avapritinib: Systemic mastocytosis (SM)
- Completed enrollment of Part 1 of the Phase 2 PIONEER trial of
avapritinib in patients with indolent SM.
BLU-782: Fibrodysplasia ossificans progressiva (FOP)
- Entered into an exclusive, worldwide license agreement with
Clementia Pharmaceuticals, a subsidiary of Ipsen, for the
development and commercialization of BLU-782 as a potential
treatment for patients with FOP and other indications.
Key Upcoming Milestones
The company expects to achieve the following milestones in the
fourth quarter of 2019:
- Present initial data from Part 1 of the Phase 2 PIONEER trial
of avapritinib in indolent SM at the 61st American
Society of Hematology (ASH) Annual Meeting and Exposition.
- Initiate a Phase 3 trial evaluating pralsetinib in first-line
RET-fusion NSCLC.
- Initiate a Phase 1b/2 trial in
China evaluating fisogatinib in
combination with CS1001, CStone Pharmaceuticals' anti-PD-L1
inhibitor, in patients with HCC.
The company expects to achieve the following milestones related
to planned marketing applications in 2020:
- Submit an NDA to the FDA for avapritinib for the treatment of
advanced SM based on data from the Phase 1 EXPLORER trial and Phase
2 PATHFINDER trial in the first quarter of 2020.
- Submit an NDA to the FDA for pralsetinib for the treatment of
patients with RET-fusion NSCLC previously treated with
platinum-based chemotherapy in the first quarter of 2020.
- Submit an NDA to the FDA for pralsetinib for the treatment of
patients with MTC previously treated with an approved multi-kinase
inhibitor in the first half of 2020.
- Submit a supplemental NDA to the FDA for avapritinib for the
treatment of third‐line GIST in the second half of 2020.
Third Quarter 2019 Financial Results
- Cash Position: As of September
30, 2019, cash, cash equivalents and investments were
$594.5 million, as compared to
$494.0 million as of December 31, 2018. This increase reflects net
proceeds of approximately $327.4
million from the company's follow-on underwritten public
offering of common stock, which closed in April 2019, partially offset by cash used in
operations. Cash, cash equivalents and investments as of
September 30, 2019 do not include the
$25.0 million upfront payment
received in connection with entering into the worldwide license
agreement with Clementia Pharmaceuticals or an $8.0 million research milestone achieved under
the Roche collaboration, both of which were earned in October 2019.
- Collaboration Revenues: Collaboration revenues were
$9.1 million for the third quarter of
2019, as compared to $1.1 million for
the third quarter of 2018. This increase was primarily due to
revenue recognized under the CStone and Roche collaborations.
During the third quarter of 2019, the company recognized
$6.0 million in milestone revenue
under the CStone collaboration compared to no revenue recognized
for the same period in 2018. During the third quarter of 2019, the
company recognized $3.1 million in
revenue under the Roche collaboration compared to $1.1 million for the same period in 2018.
- R&D Expenses: Research and development expenses were
$81.5 million for the third quarter
of 2019, as compared to $64.6 million
for the third quarter of 2018. This increase was primarily due to
increased clinical and manufacturing expenses driven by the
company's lead programs and increased personnel expenses. Research
and development expenses included $7.7
million in stock-based compensation expenses for the third
quarter of 2019.
- G&A Expenses: General and administrative expenses
were $25.6 million for the third
quarter of 2019, as compared to $12.0
million for the third quarter of 2018. This increase was
primarily due to increased personnel expenses and increased
professional fees for commercial-readiness and other activities.
General and administrative expenses included $7.3 million in stock-based compensation expenses
for the third quarter of 2019.
- Net Loss: Net loss was $94.3
million for the third quarter of 2019, or a net loss per
share of $1.93, as compared to a net
loss of $72.7 million for the third
quarter of 2018, or a net loss per share of $1.66.
Financial Guidance
Based on its current plans, Blueprint Medicines expects that its
existing cash, cash equivalents and investments, together with the
$25.0 million upfront cash payment
received under its license agreement with Clementia and an
$8.0 million research milestone
achieved in the fourth quarter of 2019 under the Roche
collaboration, but excluding any additional potential option fees,
milestone payments or other payments from Roche, CStone
Pharmaceuticals or Clementia Pharmaceuticals, will be sufficient to
enable it to fund its operating expenses and capital expenditure
requirements into the second half of 2021.
Conference Call Information
Blueprint Medicines will host a live webcast of its R&D Day
event at 8:30 a.m. ET today. The
webcast may be accessed under "Events and Presentations" in the
Investors & Media 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 90 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 three 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,
pralsetinib, fisogatinib, and BLU-263, including the timing,
designs, implementation, enrollment, plans and announcement of
results regarding Blueprint Medicines' ongoing and planned clinical
trials for its drug candidates, including avapritinib, pralsetinib,
fisogatinib and BLU-263; plans and timelines for nominating
additional development candidates; plans and timelines for
submitting an IND application to the FDA for BLU-263; plans and
timelines for submitting marketing applications for avapritinib and
pralsetinib; the potential benefits of Blueprint Medicines' current
and future drug candidates in treating patients; plans, timelines
and expectations for the FDA's review and administrative split of
the NDA for avapritinib for the treatment of adult patients with
PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and
fourth-line GIST; plans, timelines and expectations for top-line
data from the VOYAGER trial; plans, timelines and expectations for
the commercialization of avapritinib for the treatment of GIST, if
approved by the FDA; potential benefits of the license agreement
between Blueprint Medicines and Ipsen; 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, pralsetinib, fisogatinib and
BLU-263, or licensed products, including BLU-782; the FDA's intent
to administratively split the proposed indications for avapritinib
into two separate NDAs, which may not mean that either indication
is approved; a delay in the review of the proposed indications as a
result of the administrative split of the current NDA; FDA concerns
regarding whether the response rate in the fourth-line GIST
population was reasonably likely to predict clinical benefit in
that population; there can be no assurance that the FDA will not
ask for additional clinical trials for avapritinib; there can be no
assurance that the VOYAGER top-line data will be sufficient for the
FDA's review of the proposed fourth-line indication or that there
will not be a delay in the availability of VOYAGER top-line data;
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 and
licensing arrangement, including its cancer immunotherapy
collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche
Inc., its collaboration with CStone Pharmaceuticals, and its
license to Clementia Pharmaceuticals]. These and other risks and
uncertainties are described in greater detail in the section
entitled "Risk Factors" in Blueprint Medicines' filings with the
Securities and Exchange Commission (SEC), including Blueprint
Medicines' most recent Quarterly Report on Form 10-Q 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)
|
|
|
|
|
|
|
|
September
30,
|
|
December 31,
|
|
|
2019
|
|
2018
|
Cash, cash
equivalents and investments
|
|
$
|
594,459
|
|
$
|
494,012
|
Working capital
(1)
|
|
|
419,584
|
|
|
439,464
|
Total
assets
|
|
|
737,925
|
|
|
540,124
|
Deferred
revenue
|
|
|
41,331
|
|
|
46,167
|
Total
liabilities
|
|
|
221,581
|
|
|
121,115
|
Total stockholders'
equity
|
|
|
516,344
|
|
|
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
|
|
|
September
30,
|
|
|
2019
|
|
2018
|
Collaboration
revenue
|
|
$
|
9,139
|
|
$
|
1,095
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development
|
|
|
81,453
|
|
|
64,562
|
General and
administrative
|
|
|
25,647
|
|
|
12,041
|
Total operating
expenses
|
|
|
107,100
|
|
|
76,603
|
Other income
(expense):
|
|
|
|
|
|
|
Other income
(expense), net
|
|
|
3,692
|
|
|
2,799
|
Interest
expense
|
|
|
(6)
|
|
|
(14)
|
Total other
income
|
|
|
3,686
|
|
|
2,785
|
Net loss
|
|
$
|
(94,275)
|
|
$
|
(72,723)
|
Net loss per
share — basic and diluted
|
|
$
|
(1.93)
|
|
$
|
(1.66)
|
Weighted-average
number of common shares used in net loss per share — basic and
diluted
|
|
|
48,921
|
|
|
43,915
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/blueprint-medicines-outlines-precision-therapy-research-vision-provides-update-on-discovery-and-clinical-stage-portfolio-at-rd-day-and-reports-third-quarter-2019-financial-results-300951462.html
SOURCE Blueprint Medicines