CAMBRIDGE, Mass., July 29, 2021 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ:BPMC) today reported financial
results and provided a business update for the second quarter ended
June 30, 2021.
"In the second quarter, we achieved one of our foundational
corporate goals, securing U.S. approval of AYVAKIT for advanced
systemic mastocytosis, and the launch of this therapy is off to a
great start with broad prescriber demand," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "In addition, we continued to progress our
expanding clinical portfolio with the initiation of the Phase 1/2
trial of BLU-945, the initiation of the Phase 2/3 HARBOR trial of
BLU-263 and the announcement of a strategic research collaboration
with MD Anderson to accelerate the development of BLU-222, all of
which exemplify the potential impact of our next wave of
therapeutic candidates. This commercial and clinical progress,
combined with our financial strength, provides a robust foundation
for future growth as we look to address the needs of many more
patients with cancer and hematologic disorders."
Second Quarter 2021 Highlights and Recent Progress
AYVAKIT™/AYVAKYT® (avapritinib): systemic mastocytosis (SM)
and gastrointestinal stromal tumor (GIST)
- Recorded $8.5 million in net
product revenue during the second quarter of 2021 for
AYVAKIT/AYVAKYT, which was approved by the U.S. Food and Drug
Administration (FDA) in June 2021 for
the treatment of adult patients with advanced systemic mastocytosis
(Advanced SM), including aggressive SM (ASM), SM with an associated
hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL). Read
the press release announcing the expansion of the FDA label for
AYVAKIT into advanced SM here. AYVAKIT received its initial
approval from the FDA in 2020 for the treatment of adults with
unresectable or metastatic GIST harboring a PDGFRA exon 18
mutation, including PDGFRA D842V mutations. Please click here to
see the full Prescribing Information for AYVAKIT and visit
www.AYVAKIT.com for more information on AYVAKIT.
GAVRETO® (pralsetinib): RET-altered cancers
- Recorded $2.9 million in net
product revenue during the second quarter of 2021 for GAVRETO,
which was approved by the FDA in September 2020 for the treatment of adult
patients with metastatic rearranged during transfection (RET)
fusion-positive non-small cell lung cancer (NSCLC) as detected by
an FDA approved test and in December
2020 for the treatment of patients 12 years of age and older
with advanced or metastatic RET-mutant medullary thyroid cancer
(MTC) and RET fusion-positive thyroid cancer. Blueprint
Medicines is commercializing GAVRETO in
the U.S. together with Genentech, Inc., a member of
the Roche Group. Please click here to see the full Prescribing
Information for GAVRETO and visit www.GAVRETO.com for more
information on GAVRETO.
- Reported updated data from the Phase 1/2 ARROW clinical trial
of GAVRETO in metastatic RET fusion-positive NSCLC and other
advanced solid tumors at the 2021 American Society of Clinical
Oncology (ASCO) Annual Meeting. The data showed high response rates
to GAVRETO in treatment-naïve patients with RET fusion-positive
NSCLC, clinical activity across a number of RET fusion-positive
tumor types and a safety profile consistent with previously
reported results. Read the press release here.
BLU-263: SM
- Initiated the HARBOR trial, a randomized, double-blind,
placebo-controlled Phase 2/3 trial in non-advanced SM.
BLU-945: treatment-resistant EGFR-driven NSCLC
- Initiated the global Phase 1/2 trial of BLU-945 in patients
with treatment-resistant EGFR-driven NSCLC.
BLU-222: Cyclin E-aberrant cancers
- Announced a strategic research collaboration with The
University of Texas MD Anderson Cancer
Center focused on accelerating development of BLU-222, an
investigational precision therapy designed to target
cyclin-dependent kinase 2 (CDK2). Read the press release here.
Corporate:
- Announced the appointment of Percy
Carter, MBA, Ph.D., as Chief Scientific Officer. In this
role, Dr. Carter oversees all research and preclinical development.
Read the press release here.
Key Upcoming Milestones
The company expects to achieve the following near-term
milestones:
- Present preclinical data supporting combination of BLU-945 and
BLU-701 in treatment-naïve EGFR-driven NSCLC in the second half of
2021.
- Initiate a Phase 1 trial of BLU-701 in patients with
treatment-resistant EGFR-driven NSCLC in the second half of
2021.
- Initiate a Phase 1 trial of BLU-222, a CDK2 inhibitor targeting
cyclin-E aberrant cancers, in the first half of 2022.
- Disclose topline data for the registration-enabling Part 2 of
the PIONEER trial of AYVAKIT in non-advanced systemic mastocytosis
in mid-2022.
Second Quarter 2021 Financial Results
- Revenues: Revenues were $27.3 million for the
second quarter of 2021, including $8.5 million of net product revenues from
sales of AYVAKIT/AYVAKYT, $2.9
million of net product sales from GAVRETO and $15.9
million in collaboration revenues. Blueprint recorded
revenues of $8.3 million in the
second quarter of 2020, including $5.7
million of net product revenues from sales of AYVAKIT and
$2.7 million in collaboration
revenues. The increase in net product revenues was driven by
increased sales quantity and the increase in collaboration revenues
was primarily driven by the sales of drug substance and drug
product to our collaboration partners.
- Cost of Sales: Cost of sales was $6.5 million for the second quarter of 2021,
as compared to $0.1 million for the
second quarter of 2020. Cost of sales includes manufacturing
costs associated with our products sales as well as costs
associated with the sale of drug product to our collaboration
partners. The increase in costs of product sales was primarily
driven by the lower margin product sales to our collaboration
partners during the second quarter of 2021.
- R&D Expenses: Research and development expenses
were $80.0 million for the second quarter of 2021, as
compared to $91.1 million for the
second quarter of 2020. This decrease was primarily due to
decreased expenses associated with clinical supply manufacturing
activities and reimbursement from the global development cost
sharing arrangement under our collaboration with Roche for
pralsetinib, partially offset by increased costs related to early
discovery activities. Research and development expenses
included $10.5 million in stock-based compensation
expenses for the second quarter of 2021.
- SG&A Expenses: Selling, general and
administrative expenses were $49.3 million for the second
quarter of 2021, as compared to $42.2
million for the second quarter of 2020. This increase was
primarily due to increased costs associated with building our
commercial infrastructure for commercialization of AYVAKIT/AYVAKYT
and GAVRETO, partially offset by reimbursement under our
collaboration with Roche for pralsetinib. General and
administrative expenses included $13.8 million in
stock-based compensation expenses for the second quarter of
2021.
- Net Loss: Net loss was $108.4 million for
the second quarter of 2021, or a net loss per share of $1.86,
as compared to a net loss of $123.5 million for the second
quarter of 2020, or a net loss per share of $2.28.
- Cash Position: As of June 30,
2021, cash, cash equivalents and investments were
$1,380.1 million, as compared to
$1,549.7 million as of December 31, 2020.
Conference Call Information
Blueprint Medicines will host a live conference call and webcast
at 8:30 a.m. ET today to discuss
second quarter 2021 financial results and recent business
activities. The conference call may be accessed by dialing
844-200-6205 (domestic) or +44-208-0682-558 (international) and
referring to conference ID 619159. 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.
Upcoming Investor Conference
Blueprint Medicines will participate in the 2021 Wedbush Pacgrow
Healthcare Virtual Conference on Tuesday,
August 10, 2021. Christina
Rossi, Chief Commercial Officer, will participate in a panel
discussion, "Building Back a Better Commercial Infrastructure –
Selling in COVID Times," beginning at 8:35
a.m. ET. A live webcast of the panel discussion will be
available by visiting the Investors & Media section of
Blueprint Medicines' website at http://ir.blueprintmedicines.com. A
replay of the webcast will be archived on Blueprint Medicines'
website for 30 days following the presentation.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company
that invents life-changing medicines for people with cancer and
hematologic disorders. Applying an approach that is both precise
and agile, we create therapies that selectively target genetic
drivers, with the goal of staying one step ahead across stages of
disease. Since 2011, we have leveraged our research platform,
including expertise in molecular targeting and world-class drug
design capabilities, to rapidly and reproducibly translate science
into a broad pipeline of precision therapies. Today, we are
delivering our approved medicines to patients in the United
States and Europe, and we are globally advancing multiple
programs for genomically defined cancers, systemic mastocytosis,
and cancer immunotherapy. 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, strategies, timelines and expectations for
Blueprint Medicines' current or future approved drugs and drug
candidates, including timelines for marketing applications and
approvals, the initiation of clinical trials or the results of
ongoing and planned clinical trials; Blueprint Medicines' plans,
strategies and timelines to nominate development candidates; plans
and timelines for additional marketing applications for avapritinib
and pralsetinib and, if approved, commercializing avapritinib and
pralsetinib in additional geographies or for additional
indications; the potential benefits of any of Blueprint Medicines'
current or future approved drugs or drug candidates in treating
patients; the potential benefits of Blueprint Medicines'
collaborations; 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 plans in continuing to establish and expand a
commercial infrastructure, and successfully launching, marketing
and selling current or future approved products; Blueprint
Medicines' ability to successfully expand the approved indications
for AYVAKIT/AYVAKYT and GAVRETO or obtain marketing approval for
AYVAKIT/AYVAKYT in additional geographies in the future; the delay
of any current or planned clinical trials or the development of
Blueprint Medicines' current or future drug candidates; 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 obtain, maintain and enforce patent and other
intellectual property protection for AYVAKIT/AYVAKYT, GAVRETO or
any drug candidates it is developing; Blueprint Medicines' ability
to develop and commercialize companion diagnostic tests for
AYVAKIT/AYVAKYT, GAVRETO or any of its current and future drug
candidates; and the success of Blueprint Medicines' current and
future collaborations, partnerships 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.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated
logos are trademarks of Blueprint Medicines Corporation.
Blueprint
Medicines Corporation
Selected Condensed Consolidated Balance Sheet Data
(in thousands)
(unaudited)
|
|
|
|
June 30,
|
|
December 31,
|
|
|
2021
|
|
2020
|
Cash, cash
equivalents and investments
|
|
$
|
1,380,051
|
|
$
|
1,549,722
|
Working capital
(1)
|
|
|
578,673
|
|
|
796,957
|
Total
assets
|
|
|
1,568,795
|
|
|
1,718,393
|
Deferred
revenue
|
|
|
38,277
|
|
|
41,158
|
Total
liabilities
|
|
|
239,991
|
|
|
248,305
|
Total stockholders'
equity
|
|
|
1,328,804
|
|
|
1,470,088
|
|
|
|
|
|
|
|
(1) Blueprint 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
|
|
Six Months
Ended
|
|
June 30,
|
|
June 30,
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue, net
|
$
|
11,433
|
|
$
|
5,680
|
|
$
|
20,388
|
|
$
|
9,138
|
Collaboration revenue
|
|
15,862
|
|
|
2,663
|
|
|
28,483
|
|
|
5,372
|
Total
revenues
|
|
27,295
|
|
|
8,343
|
|
|
48,871
|
|
|
14,510
|
Cost and operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
6,493
|
|
|
127
|
|
|
6,595
|
|
|
150
|
Research and
development
|
|
80,027
|
|
|
91,079
|
|
|
159,738
|
|
|
175,225
|
Selling, general and
administrative
|
|
49,286
|
|
|
42,174
|
|
|
91,288
|
|
|
77,829
|
Total cost and
operating expenses
|
|
135,806
|
|
|
133,380
|
|
|
257,621
|
|
|
253,204
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
Interest income, net
|
|
633
|
|
|
1,586
|
|
|
1,371
|
|
|
4,490
|
Other expense, net
|
|
(373)
|
|
|
(23)
|
|
|
(587)
|
|
|
(224)
|
Total other
income
|
|
260
|
|
|
1,563
|
|
|
784
|
|
|
4,266
|
Loss before income
taxes
|
|
(108,251)
|
|
|
(123,474)
|
|
|
(207,966)
|
|
|
(234,428)
|
Income tax
expense
|
|
(193)
|
|
|
—
|
|
|
(193)
|
|
|
—
|
Net loss
|
$
|
(108,444)
|
|
$
|
(123,474)
|
|
$
|
(208,159)
|
|
$
|
(234,428)
|
Net loss per share —
basic and diluted
|
$
|
(1.86)
|
|
$
|
(2.28)
|
|
$
|
(3.58)
|
|
$
|
(4.39)
|
Weighted-average
number of common shares used in net loss per share — basic and
diluted
|
|
58,406
|
|
|
54,217
|
|
|
58,216
|
|
|
53,436
|
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SOURCE Blueprint Medicines Corporation