CAMBRIDGE, Mass., Feb. 13, 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 fourth quarter and full year
ended December 31, 2019.
"In 2020, we will complete our evolution into a fully-integrated
global biopharmaceutical company and fortify our leadership in the
field of precision medicine with further expansion of our research
pipeline," said Jeff Albers, Chief
Executive Officer of Blueprint Medicines. "Over the course of the
year, we expect a sustained cadence of catalytic milestones across
our portfolio, which began with the commercial launch of AYVAKIT in
January and will continue with the presentation of updated data for
avapritinib in indolent systemic mastocytosis and the completion of
our rolling NDA submission for pralsetinib for RET fusion-positive
non-small cell lung cancer later this quarter. Ultimately, we
believe these and other anticipated achievements throughout the
year will transform the profile of our company and create
substantial value for patients and healthcare providers."
Fourth Quarter 2019 Highlights and Recent Progress
Avapritinib: gastrointestinal stromal tumors (GIST)
- Received U.S. Food and Drug Administration (FDA) approval of
AYVAKIT for the treatment of adults with unresectable or metastatic
GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V
mutations. Read the press release here and visit www.AYVAKIT.com
for full Prescribing Information.
- Announced that the FDA has extended the Prescription Drug User
Fee Act (PDUFA) date for the company's new drug application (NDA)
seeking accelerated approval of avapritinib for the treatment of
adults with fourth-line GIST, by three months from February 14, 2020 to May
14, 2020. Read the press release here.
Avapritinib: systemic mastocytosis (SM)
- Reported initial data from Part 1 of the PIONEER trial of
avapritinib in patients with indolent SM at the American Society of
Hematology Annual Meeting in December
2019 showing rapid and robust reductions in serum tryptase,
a measure of mast cell burden, at all dose levels tested.
Avapritinib was well-tolerated, and most reported adverse events
were Grade 1 or 2. No patients discontinued due to an adverse
event. Read the press release here.
- Announced updated data from Part 1 of the PIONEER trial will be
reported in a late-breaking oral presentation at the American
Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting
on March 14, 2020.
Pralsetinib: RET-altered cancers
- Announced centrally reviewed top-line data from the Phase 1/2
ARROW trial of pralsetinib in patients with RET fusion-positive
non-small cell lung cancer (NSCLC) treated with pralsetinib at 400
mg QD, which is the proposed indicated dose. In patients previously
treated with platinum-based chemotherapy, the overall response rate
(ORR) was 61 percent (95% CI: 50-72%; two responses pending
confirmation). In treatment-naïve patients, the ORR was 73 percent
(95% CI: 52-88%; all responses confirmed), with 12 percent of
patients achieving a complete response. The median duration of
response, regardless of prior treatment, was not reached.
Pralsetinib was well-tolerated, and most AEs were Grade 1 or 2.
Across all patients enrolled in the ARROW trial and treated with
pralsetinib at 400 mg QD, only four percent of patients
discontinued treatment due to treatment-related adverse events.
Read the press release here.
- Announced the initiation of a rolling new drug application
(NDA) submission to the FDA for pralsetinib for the treatment of
RET fusion-positive NSCLC.
- Activated the first clinical trial site for the Phase 3
AcceleRET Lung trial in patients with first-line RET
fusion-positive NSCLC.
Fisogatinib: hepatocellular carcinoma (HCC)
- Dosed the first patient in a Phase 1b/2 trial evaluating fisogatinib in combination
with CS1001 for the treatment of locally advanced or metastatic
HCC, under Blueprint Medicine's collaboration with CStone
Pharmaceuticals. Read the press release here.
Research portfolio
- Nominated a potential first-in-class development candidate for
the treatment of resistant EGFR-positive triple mutant NSCLC.
Corporate
- Closed an underwritten public offering of 4,710,144 shares of
common stock at a public offering price of $69.00 per share. Blueprint Medicines received
estimated net proceeds of approximately $308.2 million, after deducting underwriting
discounts and commissions and estimated offering expenses.
Key Upcoming Milestones
The company expects to achieve the following milestones in the
first half of 2020.
- Present updated data from Part 1 of the PIONEER trial of
avapritinib in indolent SM at AAAAI Annual Meeting in the first
quarter of 2020.
- Complete the submission of a rolling NDA to the FDA for
pralsetinib for RET fusion-positive NSCLC in the first quarter of
2020.
- Report top-line data from the Phase 3 VOYAGER trial of
avapritinib in third-line GIST early in the second quarter of
2020.
- Gain FDA approval and, if approved, launch avapritinib in
fourth-line GIST in the U.S. in the second quarter of 2020.
- Report top-line data from the Phase 1/2 ARROW trial of
pralsetinib in patients with previously treated RET mutant
medullary thyroid cancer (MTC).
- 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.
- Submit a marketing authorization application to the European
Medicines Agency for pralsetinib for RET fusion-positive NSCLC in
the second quarter of 2020.
- Initiate a Phase 1 trial of BLU-263, a next-generation KIT
inhibitor, in healthy volunteers in the first half of 2020.
Fourth Quarter and Year End 2019 Financial Results
- Cash Position: As of December 31, 2019, cash,
cash equivalents and investments were $548.0 million, as
compared to $494.0 million as of December 31, 2018.
This increase was primarily related to $327.5 million in net proceeds received from the
company's April 2019 follow-on
underwritten public offering and the $25.0
million upfront cash payment under the license agreement
Clementia, partially offset by an increase in cash used in
operating activities. Cash, cash equivalents and investments as of
December 31, 2019 do not include the
estimated net proceeds of approximately $308.2 million from the company's follow-on
underwritten public offering of common stock, which closed in
January 2020.
- Collaboration Revenues: Collaboration revenues
were $51.5 million for the fourth quarter of 2019
and $66.5 million for the year ended December 31,
2019, as compared to $1.0 million for the fourth quarter
of 2018 and $44.5 million for the year
ended December 31, 2018. Collaboration revenue for the year
ended December 31, 2019 consisted
primarily of the $25.0 million
upfront payment and a $20.0 million
cash milestone payment due in the third quarter of 2020 under the
license agreement with Clementia, an aggregate of $12.0 million in development and regulatory
milestones that were achieved in 2019 under the CStone
collaboration agreement and $8.2
million under the Roche collaboration agreement.
Collaboration revenue for the year ended December 31, 2018 consisted primarily of the
$40.0 million upfront payment under
the CStone collaboration agreement and $4.5
million under the Roche collaboration agreement.
- R&D Expenses: Research and development
expenses were $88.6 million for the fourth quarter of
2019 and $331.5 million for the year ended December
31, 2019, as compared to $70.5 million for the fourth
quarter of 2018 and $243.6 million for the year
ended December 31, 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 $7.6 million in
stock-based compensation expenses for the fourth quarter of 2019
and $28.6 million in stock-based compensation expenses
for the year ended December 31, 2019.
- G&A Expenses: General and administrative
expenses were $32.3 million for the fourth quarter of
2019 and $96.4 million for the year ended December
31, 2019, as compared to $13.6 million for the fourth
quarter of 2018 and $47.9 million for the year
ended December 31, 2018. This increase was primarily related
to increased costs and personnel expenses associated with building
the company's commercial infrastructure and to support the overall
growth of the business. General and administrative expenses
included $8.1 million in stock-based compensation
expenses for the fourth quarter of 2019 and $26.1
million in stock-based compensation expenses for the year
ended December 31, 2019.
- Net Loss: Net loss was $66.3 million for
the fourth quarter of 2019 and $347.7 million for the
year ended December 31, 2019, or a net loss per share
of $1.35 and $7.27, respectively, as compared to a
net loss of $80.3 million for the fourth quarter of 2018
and $236.6 million for the year ended December 31,
2018, or a net loss per share of $1.83 and $5.39,
respectively.
Financial Guidance
Based on its current operating plans, Blueprint Medicines
expects that its existing cash, cash equivalents and investments
including the $308.2 million in
estimated net proceeds from the January
2020 follow-on public offering, together with anticipated
product revenues but excluding any additional 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 fourth quarter and
full year 2019 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 26735762. 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 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 FDA-approved precision therapy
and are currently advancing multiple 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 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 for avapritinib, pralsetinib, fisogatinib and
BLU-263; plans and timelines for nominating additional development
candidates; plans and timelines for submitting marketing
applications for avapritinib and pralsetinib and, if approved,
commercializing avapritinib for additional indications or
pralsetinib; the potential benefits of Blueprint Medicines' current
and future approved drugs or drug candidates in treating patients;
plans, timelines and expectations for top-line data from the
VOYAGER trial; expectations regarding Blueprint Medicines' existing
cash, cash equivalents and investments; and Blueprint Medicines'
strategy, goals and anticipated milestones, business plans and
focus; the potential benefits of Blueprint Medicines' current and
future drug candidates in treating patients; 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 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)
|
|
|
|
|
|
|
|
|
|
December
31,
|
|
December 31,
|
|
|
2019
|
|
2018
|
Cash, cash
equivalents and investments
|
|
$
|
547,960
|
|
$
|
494,012
|
Working capital
(1)
|
|
|
410,304
|
|
|
439,464
|
Total
assets
|
|
|
707,694
|
|
|
540,124
|
Deferred
revenue
|
|
|
46,073
|
|
|
46,167
|
Lease incentive
obligation
|
|
|
-
|
|
|
14,617
|
Total stockholders'
equity
|
|
|
464,359
|
|
|
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
|
|
Years
Ended
|
|
|
December
31,
|
|
December
31,
|
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
Collaboration
revenue
|
|
$
|
51,533
|
|
$
|
1,033
|
|
$
|
66,512
|
|
$
|
44,521
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
88,646
|
|
|
70,532
|
|
|
331,450
|
|
|
243,621
|
General and
administrative
|
|
|
32,265
|
|
|
13,643
|
|
|
96,388
|
|
|
47,928
|
Total operating
expenses
|
|
|
120,911
|
|
|
84,175
|
|
|
427,838
|
|
|
291,549
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
(expense), net
|
|
|
2,990
|
|
|
2,871
|
|
|
13,732
|
|
|
10,566
|
Other income
(expense), net
|
|
|
57
|
|
|
(51)
|
|
|
(100)
|
|
|
(180)
|
Total other income
(expense)
|
|
|
3,047
|
|
|
2,820
|
|
|
13,632
|
|
|
10,386
|
Net loss
|
|
$
|
(66,331)
|
|
$
|
(80,322)
|
|
$
|
(347,694)
|
|
$
|
(236,642)
|
Net loss per share —
basic and diluted
|
|
$
|
(1.35)
|
|
$
|
(1.83)
|
|
$
|
(7.27)
|
|
$
|
(5.39)
|
Weighted-average
number of common shares used in net
loss per share — basic and diluted
|
|
|
49,218
|
|
|
43,994
|
|
|
47,829
|
|
|
43,867
|
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SOURCE Blueprint Medicines Corporation