CAMBRIDGE, Mass., May 6, 2020 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC), a precision therapy company
focused on genomically defined cancers, rare diseases and cancer
immunotherapy, today reported financial results and provided a
business update for the first quarter ended March 31, 2020.
"Our progress in recent months is a testament to the agility of
the Blueprint Medicines team and the strength of our
fully-integrated business," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines.
"Despite challenges related to the COVID-19 pandemic and top-line
results from the VOYAGER trial, we broadly executed against our
planned milestones, including the submission of marketing
applications for pralsetinib for RET fusion-positive non-small cell
lung cancer in the U.S. and Europe
and the reporting of transformative datasets for avapritinib in
systemic mastocytosis and pralsetinib in RET-altered cancers,
paving the way for multiple planned launches through
2021."
First Quarter 2020 Highlights and Recent Updates
Avapritinib: systemic mastocytosis (SM)
- Announced results from Part 1 of the Phase 2 PIONEER trial in
patients with indolent SM, which showed treatment with avapritinib
at the recommended Part 2 dose of 25 mg once daily showed
improvements in symptom scores, measures of mast cell burden and
patient-reported quality of life. Avapritinib was well-tolerated,
and no patients discontinued treatment due to an adverse event
(AE). Read the press release here.
Avapritinib: gastrointestinal stromal tumors (GIST)
- Launched AYVAKIT in the U.S. for the treatment of adults with
unresectable or metastatic GIST harboring a PDGFRA exon 18
mutations, including PDGFRA D842V mutations, and achieved net
product revenue of $3.5 million in
the first partial quarter of the launch.
- Announced top-line data from the Phase 3 VOYAGER trial of
avapritinib versus regorafenib in third- or fourth-line GIST. The
VOYAGER trial did not meet its primary endpoint of an improvement
in progression-free survival for avapritinib versus regorafenib.
Based on the top-line results, Blueprint Medicines plans to
discontinue further development of avapritinib in GIST indications
other than PDGFRA exon 18 mutant GIST. Read the press release
here.
Pralsetinib: RET-altered cancers
- Announced top-line data from the Phase 1/2 ARROW trial of
pralsetinib in patients with RET fusion-positive non-small cell
lung cancer (NSCLC). The top-line data showed overall response
rates (ORRs) of 61 percent in patients previously treated with
platinum-based chemotherapy and 73 percent in patients naïve to
prior systemic therapy. In both populations, the median duration of
response (DOR) was not reached. Pralsetinib was well-tolerated, and
most AEs were grade 1 or 2. Read the press release here.
- Completed the submission of a new drug application (NDA) to the
U.S. Food and Drug Administration (FDA) and a marketing
authorization application to the European Medicines Agency for
pralsetinib for the treatment of patients with RET fusion-positive
NSCLC. In connection with the NDA submission, Blueprint Medicines
plans to participate in the FDA's Project Orbis initiative, which
provides a framework for concurrent submission and review of
marketing applications for oncology drugs across participating
global health authorities. Additional information about the FDA's
Project Orbis initiative can be found here.
- Announced top-line data from the Phase 1/2 ARROW trial of
pralsetinib in patients with RET mutant medullary thyroid cancer
(MTC). The top-line data showed ORRs of 60 percent in patients
previously treated with a multi-kinase inhibitor and 74 percent in
patients naïve to prior systemic treatment. In both populations,
the median DOR was not reached. Pralsetinib was well-tolerated, and
most AEs were grade 1 or 2. Read the press release here.
BLU-263: SM
- Received clearance from the FDA for an investigational new drug
(IND) application for BLU-263 for the treatment of patients with
indolent SM.
Key Upcoming Milestones
The company expects to achieve the following near-term
milestones:
- 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 second quarter of 2020 under the FDA's Oncology
Center of Excellence Real-Time Oncology Review pilot program (RTOR
program). The FDA's RTOR program aims to explore a more efficient
review process to ensure that safe and effective treatments are
available to patients as early as possible, while maintaining and
improving review quality by the FDA. Additional information about
the FDA's RTOR program can be found here.
- Present updated data from the ARROW trial of pralsetinib in
RET-altered cancers at the American Society of Clinical Oncology
(ASCO) Annual Meeting.
- Present additional data from Part 1 of the PIONEER trial of
avapritinib in indolent SM at the European Academy of Allergy and
Clinical Immunology (EAACI) 2020 Congress.
- Present updated data from the EXPLORER trial of avapritinib in
advanced SM at the European Hematology Association (EHA)
25th Annual Congress.
- Initiate patient screening for the registration-enabling Part 2
of the PIONEER trial of avapritinib in indolent SM in June 2020.
- Initiate a Phase 1 trial of BLU-263 in healthy volunteers in
June 2020.
- Report top-line data from the EXPLORER and PATHFINDER trials of
avapritinib in advanced SM in the third quarter of 2020.
Response to the COVID-19 Pandemic and Potential Business
Impacts
Due to the evolving and uncertain global impacts of the COVID-19
pandemic, Blueprint Medicines cannot precisely determine or
quantify the impact this pandemic will have on its business,
operations (including clinical trials) and financial performance
for the remainder of our fiscal year ending December 31, 2020 and beyond.
- For ongoing and planned clinical trials, while the company
anticipates and has experienced some temporary delays or
disruptions due to the COVID-19 pandemic, Blueprint Medicines is
working with any impacted clinical trial sites to ensure study
continuity.
- Blueprint Medicines currently has sufficient supply to meet
anticipated global commercial and clinical development needs for
avapritinib, pralsetinib, fisogatinib and BLU-263 through 2021.
However, the COVID-19 pandemic could adversely impact Blueprint
Medicines' suppliers and result in delays or disruptions in the
company's current or future supply chain.
- Blueprint Medicines has shifted commercial and medical affairs
field activities across its portfolio toward virtual formats where
possible in order to allow the company to continue to serve the
needs of healthcare providers, patients and other stakeholders
during this critical time. As the pandemic evolves, the company
anticipates utilizing a mix of in-person and virtual engagement
formats, as is locally and regionally appropriate.
Blueprint Medicines will continue to assess potential impacts of
the COVID-19 pandemic and seek to advance the company's pipeline of
targeted therapies as quickly as possible, while making the health
and safety of the company's employees and their families,
healthcare providers, patients and communities a top priority.
First Quarter 2020 Financial Results
- Cash Position: As of March 31,
2020, cash, cash equivalents and investments were
$750.4 million, as compared to
$548.0 million as of December 31, 2019. This increase was primarily
related to $308.4 million in
estimated net proceeds received from the company's January 2020 follow-on underwritten public
offering, partially offset by cash used in operating
activities.
- Revenues: Revenues were $6.2
million for the first quarter of 2020, including
$3.5 million of net product revenues
from sales of AYVAKIT and $2.7
million in collaboration revenues under the collaboration
agreements with CStone and Roche. Blueprint Medicines recorded
$0.7 million in collaboration
revenues for the first quarter of 2019.
- Cost of Sales: Cost of sales was less than $0.1 million for the first quarter of 2020.
Blueprint Medicines did not incur cost of sales in the first
quarter of 2019 as no product sales were generated during that
period.
- R&D Expenses: Research and development expenses were
$84.1 million for the first quarter
of 2020, as compared to $74.3 million
for the first quarter of 2019. This increase was primarily due to
increased clinical and manufacturing costs and personnel expenses
driven by the progression of Blueprint Medicines' lead programs.
Research and development expenses included $7.8 million in stock-based compensation expenses
for the first quarter of 2020.
- SG&A Expenses: Selling, general and administrative
expenses were $35.7 million for the
first quarter of 2020, as compared to $16.6
million for the first quarter of 2019. This increase was
primarily due to increased costs and personnel expenses associated
with building Blueprint Medicines' commercial infrastructure for
the commercialization of AYVAKIT and to support the overall growth
of the company's business. General and administrative expenses
included $9.1 million in stock-based
compensation expenses for the first quarter of 2020.
- Net Loss: Net loss was $111.0
million for the first quarter of 2020, or a net loss per
share of $2.11, as compared to a net
loss of $87.4 million for the first
quarter of 2019, or a net loss per share of $1.98.
Financial Guidance
Based on its current operating plans, Blueprint
Medicines expects that its existing cash, cash equivalents and
investments, together with anticipated product revenues but
excluding any potential option fees, milestone payments or other
payments under its collaboration or license agreements, will be
sufficient to enable it to fund its operating expenses and capital
expenditure requirements into the second half of 2022.
Conference Call Information
Blueprint Medicines will host a live conference call and webcast
at 8:30 a.m. ET today to discuss
first quarter 2020 financial results and recent business
activities. The conference call may be accessed by dialing (855)
728-4793 (domestic) or (503) 343-6666 (international), and
referring to conference ID 7980947. A webcast of the call will be
available under "Events and Presentations" in the Investors &
Media 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 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 have one precision therapy approved by
the U.S. Food and Drug Administration and are currently
advancing multiple investigational medicines in clinical
development, along with a number of 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 AYVAKIT
(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; plans and timelines for submitting marketing
applications for avapritinib and pralsetinib and, if approved,
commercializing avapritinib for additional indications or
pralsetinib; the impact of the COVID-19 pandemic on Blueprint
Medicines' business, operations and financial performance; the
potential benefits of the FDA's RTOR program or the FDA's Project
Orbis initiative; the potential benefits of Blueprint Medicines'
current and future approved drugs or drug candidates in treating
patients; expectations regarding Blueprint Medicines' existing
cash, cash equivalents and investments; and Blueprint Medicines'
strategy, goals and anticipated milestones, business plans and
focus. The words "aim," "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 impact of the
COVID-19 pandemic to Blueprint Medicines' business, operations,
strategy, goals and anticipated milestones, including Blueprint
Medicines' ongoing and planned research and discovery activities,
ability to conduct ongoing and planned clinical trials, clinical
supply of current or future drug candidates, commercial supply of
current or future approved products, and launching, marketing and
selling current or future approved products; Blueprint Medicines'
ability and plan in establishing a commercial infrastructure, and
successfully launching, marketing and selling its approved product;
Blueprint Medicines' ability to successfully expand the approved
indications for AYVAKIT or obtain marketing approval for AYVAKIT in
additional geographies in the future; the delay of any current or
planned clinical trials or the development of Blueprint Medicines'
drug candidates or licensed product candidate; 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 or licensing arrangements. 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 Annual Report on Form 10-K, as
supplemented by its 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)
|
|
|
March
31,
|
|
December
31,
|
|
2020
|
|
2019
|
Cash, cash
equivalents and investments
|
$
|
750,430
|
|
$
|
547,960
|
Working capital
(1)
|
|
606,395
|
|
|
410,304
|
Total
assets
|
|
913,623
|
|
|
707,694
|
Deferred
revenue
|
|
45,499
|
|
|
46,073
|
Total
liabilities
|
|
230,692
|
|
|
243,335
|
Total stockholders'
equity
|
|
682,931
|
|
|
464,359
|
|
|
|
|
|
|
(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,
|
|
|
2020
|
|
2019
|
Revenues:
|
|
|
|
|
Product revenue,
net
|
|
$
|
3,458
|
|
$
|
-
|
Collaboration
revenue
|
|
|
2,709
|
|
|
730
|
Total
revenues
|
|
|
6,167
|
|
|
730
|
Cost and operating
expenses:
|
|
|
|
|
|
|
Cost of sales
|
|
|
24
|
|
|
-
|
Research and
development
|
|
|
84,146
|
|
|
74,250
|
Selling, general and
administrative
|
|
|
35,655
|
|
|
16,553
|
Total cost and
operating expenses
|
|
|
119,825
|
|
|
90,803
|
Other income
(expense):
|
|
|
|
|
|
|
Interest income
(expense), net
|
|
|
2,904
|
|
|
2,710
|
Other income
(expense), net
|
|
|
(201)
|
|
|
(44)
|
Total other income
(expense)
|
|
|
2,703
|
|
|
2,666
|
Net loss
|
|
$
|
(110,955)
|
|
$
|
(87,407)
|
Net loss per share —
basic and diluted
|
|
$
|
(2.11)
|
|
$
|
(1.98)
|
Weighted-average
number of common shares used in net loss per share —
basic and diluted
|
|
|
52,655
|
|
|
44,097
|
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SOURCE Blueprint Medicines Corporation