-- Achieved $36.5
million in total revenues, including $28.5 million in AYVAKIT® (avapritinib) net
product revenues representing 20 percent AYVAKIT revenue growth
from Q1 2022 --
-- Plan to report topline data for
registration-enabling PIONEER trial of AYVAKIT in non-advanced SM
in August 2022 --
-- Licensed KIT exon 13 inhibitor to IDRx in
exchange for a 15 percent Series A preferred equity investment, and
the potential to receive up to $217.5
million in development, regulatory, and sales-based
milestones and tiered percentage royalties --
-- Investor Day planned for November 1, 2022 in New
York, NY --
CAMBRIDGE, Mass., Aug. 2, 2022
/PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC)
today reported financial results and provided a business update for
the second quarter ended June 30,
2022.
"We are now one year into the AYVAKIT launch in advanced
systemic mastocytosis (SM) and continue to see a significant
expansion of our prescriber base as well as strong patient demand,
reinforcing our conviction in the growth opportunity in SM," said
Kate Haviland, Chief Executive
Officer of Blueprint Medicines. "We are on track to announce
topline data from our registration-enabling PIONEER trial in
August, which will catalyze our ability to bring AYVAKIT's
transformative potential to patients living with non-advanced SM.
In addition, we continue to make significant progress across our
pipeline of innovative investigational medicines in EGFR-mutant and
CDK2-vulnerable cancers. We look forward to sharing more about our
strategic vision, including the opportunities we see in SM,
EGFR-mutant lung cancer, and CDK2-vulnerable cancers, and how our
expanding research platform will continue to drive innovation at an
Investor Day on November 1, 2022 in
New York, NY. With our strong
revenue performance, a clinical-stage pipeline of five significant
assets, and well over $1 billion in
cash on our balance sheet as of today, we are uniquely positioned
to drive near- and long-term value for all of our stakeholders by
delivering transformative precision medicines to patients around
the world."
Second Quarter 2022 Highlights and Recent Progress
AYVAKIT®/AYVAKYT® (avapritinib): systemic mastocytosis (SM)
and PDGFRA gastrointestinal stromal tumor (GIST)
- Reported global net product revenues of $28.5 million for the second quarter of
2022.
- Presented new retrospective analyses at the European Hematology
Association (EHA) 2022 Congress, showing that AYVAKIT significantly
improved overall survival in patients with advanced SM when
compared to real-world data for prior best available therapies,
including midostaurin and cladribine. Read the press release
here.
- Upon request by the FDA, changed the primary endpoint for the
registrational PIONEER Part 2 trial of AYVAKIT in patients with
non-advanced SM to the mean change in total symptom score
(TSS).
GAVRETO® (pralsetinib): RET-altered cancers
- As previously recorded and reported by Roche, GAVRETO global
product sales were 7 million CHF,
which excludes sales in the Greater
China territory driven by CStone Pharmaceuticals.
- Received approval in Hong Kong,
China, via the collaboration with CStone Pharmaceuticals,
for the treatment of RET fusion-positive non-small cell lung cancer
(NSCLC).
Corporate
- Announced strategic financing collaborations with Sixth Street
Partners (Sixth Street) and Royalty Pharma plc (Royalty Pharma)
(NASDAQ: RPRX) for up to $1.25
billion, of which $175 million
was funded as of June 30, 2022, and
an additional $400 million funded in
July 2022. Read the press release
here.
- Licensed a development candidate-stage KIT exon 13 inhibitor,
and licensed the compound to IDRx, Inc., a recently launched
clinical-stage biopharmaceutical company, in exchange for a 15
percent Series A preferred equity investment and up to $217.5 million in future milestone payments and
tiered percentage royalty payments.
- Published an inaugural Corporate Responsibility Report,
highlighting the company's long-standing commitment to delivering
sustainable value to patients with cancer and blood disorders, as
well as the communities in which Blueprint Medicines operates. Read
the press release here and access the report here.
- Announced the appointment of Habib
Dable, former Chief Executive Officer of Acceleron Pharma,
Inc., to the company's Board of Directors. Read the press release
here.
Key Upcoming Milestones
The company plans to achieve the following near-term
milestones:
- Present topline data from the registrational PIONEER Part 2
trial for avapritinib in non-advanced SM in August 2022 and submit a supplemental new drug
application to the FDA for AYVAKIT in non-advanced SM in the second
half of 2022.
- Share the strategic vision for Blueprint, including
opportunities in SM, EGFR-mutant lung cancer, and CDK2-vulnerable
cancers, and how our expanding research platform has the potential
to deliver the promise of precision medicine to more patients at an
Investor Day on November 1, 2022 in
New York, NY.
- Present updated BLU-945 monotherapy data and initial dose
escalation data for BLU-945 in combination with osimertinib from
the Phase 1/2 SYMPHONY trial in EGFR-mutant NSCLC in the second
half of 2022.
- Present initial clinical data from the Phase 1/2 HARMONY trial
of BLU-701 in EGFR-mutant NSCLC in the second half of 2022.
- Present initial data from the HARBOR trial of BLU-263 in
non-advanced SM in the second half of 2022.
- Present initial clinical data from the Phase 1/2 CONCERTO trial
of BLU-451 in EGFR-mutant NSCLC in the first half of 2023.
- Present initial clinical data from the Phase 1/2 VELA trial of
BLU-222 in CDK2-vulnerable cancers in the first half of 2023.
Second Quarter 2022 Results
- Revenues: Revenues were $36.5 million for
the second quarter of 2022, including $28.5 million of
net product revenues from sales of AYVAKIT/AYVAKYT and $8.0
million in collaboration revenues. Blueprint
Medicines recorded revenues of $27.3 million in the
second quarter of 2021, including $8.5 million of net
product revenues from sales of AYVAKIT/AYVAKIT, $2.9 million of net product revenues from sales
of GAVRETO and $15.9 million in collaboration
revenues.
- Cost of Sales: Cost of sales was $4.9
million for the second quarter of 2022, as compared
to $6.5 million for the second quarter of 2021.
- R&D Expenses: Research and development expenses
were $128.5 million for the second quarter of 2022, as
compared to $80.0 million for the second quarter of 2021.
This increase was primarily due to increased costs associated with
the progression of our clinical trials and increased costs related
to early discovery effort. Research and development expenses
included $10.5 million in stock-based compensation
expenses for the second quarter of 2022.
- SG&A Expenses: Selling, general and
administrative expenses were $58.7 million for the second
quarter of 2022, as compared to $49.3 million for the
second quarter of 2021. This increase was primarily due to
increased costs associated with expanding our commercial
infrastructure for commercialization of AYVAKIT/AYVAKYT. General
and administrative expenses included $14.9 million in
stock-based compensation expenses for the second quarter of
2022.
- Net Loss: Net loss was $159.7 million for
the second quarter of 2022, or a net loss per share of $2.68,
as compared to a net loss of $108.4 million for the
second quarter of 2021, or a net loss per share of $1.86.
- Cash Position: As of June 30, 2022, cash, cash
equivalents and investments were $947.2 million, as compared
to $1,034.6 million as of December 31, 2021.
Cash as of June 30, 2022, does not
include $400 million gross proceeds
received from our strategic non-dilutive financing agreements with
Sixth Street, which closed and were funded in July 2022.
Financial Guidance
Blueprint Medicines anticipates approximately $180M to $200M in
total revenues in 2022, including approximately $115M to $130M in
AYVAKIT net product revenues. The company continues to expect that
its existing cash, cash equivalents and investments, together with
anticipated future product revenues, will provide sufficient
capital to enable the company to achieve a self-sustainable
financial profile.
Conference Call Information
Blueprint Medicines will host a live conference call and
webcast at 8:00 a.m. ET today to discuss second quarter
2022 financial results and recent business activities. The
conference call may be accessed by dialing 844-200-6205 (domestic)
or 929-526-1599 (international), and referring to conference ID
694684. A webcast of the call will also 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 global precision therapy company that
invents life-changing therapies for people with cancer and blood
disorders. Applying an approach that is both precise and agile, we
create medicines 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 approved medicines
directly to patients in the United
States and Europe, and we
are globally advancing multiple programs for systemic mastocytosis,
lung cancer and other genomically defined cancers, 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
interactions with the FDA and other regulatory authorities; plans
and timelines to update the primary endpoint of the registrational
PIONEER trial of AYVAKIT in patients with non-advanced SM;
expectations regarding the potential benefits of AYVAKIT in
treating patients with non-advanced SM; and the potential benefits
of Blueprint Medicines' collaborations and strategic
financing transactions; and Blueprint
Medicines' financial performance, 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 either
as monotherapies or in combination with other agents or may impact
the anticipated timing of data or regulatory submissions; the
timing of the initiation of clinical trials and trial cohorts at
clinical trial sites and patient enrollment rates; 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; Blueprint
Medicines' ability to successfully expand its operations,
research platform and portfolio of therapeutic candidates, and the
timing and costs thereof; Blueprint Medicines' ability to realize
the anticipated benefits of its executive leadership transition
plan; and the success of Blueprint Medicines' current and future
collaborations, financing arrangements, 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,
|
|
|
2022
|
|
2021
|
Cash, cash equivalents
and investments
|
|
$
|
947,159
|
|
$
|
1,034,643
|
Working capital
(1)
|
|
|
793,419
|
|
|
404,260
|
Total assets
|
|
|
1,192,446
|
|
|
1,252,225
|
Deferred revenue
(2)
|
|
|
22,139
|
|
|
36,576
|
Liability related to
the sale of future royalties (2)
|
|
|
171,254
|
|
|
-
|
Total
liabilities
|
|
|
444,696
|
|
|
281,490
|
Total stockholders'
equity
|
|
|
747,750
|
|
|
970,735
|
|
|
|
|
(1) Blueprint defines
working capital as current assets less current
liabilities.
(2) Includes both
current and long-term portions of the balance
|
|
|
|
Blueprint Medicines
Corporation
|
Condensed
Consolidated Statements of Operations Data
|
(in thousands,
except per share data)
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended,
June
30
|
|
Six Months
Ended,
June
30
|
|
|
2022
|
2021
|
|
2022
|
|
2021
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue,
net
|
|
$
|
28,454
|
$
|
11,433
|
|
$
|
52,295
|
|
$
|
20,388
|
Collaboration
revenue
|
|
|
8,093
|
|
15,862
|
|
|
46,983
|
|
|
28,483
|
Total
revenues
|
|
|
36,547
|
|
27,295
|
|
|
99,278
|
|
|
48,871
|
Cost and operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
|
4,886
|
|
6,493
|
|
|
9,964
|
|
|
6,595
|
Collaboration loss
sharing
|
|
|
2,145
|
|
----
|
|
|
5,410
|
|
|
----
|
Research and
development
|
|
|
128,466
|
|
80,027
|
|
|
231,599
|
|
|
159,738
|
Selling, general and
administrative
|
|
|
58,688
|
|
49,286
|
|
|
115,747
|
|
|
91,288
|
Total cost and
operating expenses
|
|
$
|
194,185
|
|
135,806
|
|
|
362,720
|
|
|
257,621
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
(expense), net
|
|
|
427
|
|
633
|
|
|
869
|
|
|
1,371
|
Other income
(expense), net
|
|
|
632
|
|
(373)
|
|
|
177
|
|
|
(587)
|
Total other income
(expense)
|
|
$
|
1,059
|
|
260
|
|
|
1,046
|
|
|
784
|
Loss before income
taxes
|
|
$
|
(156,579)
|
|
(108,251)
|
|
|
(262,396)
|
|
|
(207,966)
|
Income tax
expense
|
|
|
(3,130)
|
|
(193)
|
|
|
(3,313)
|
|
|
(193)
|
Net loss
|
|
$
|
(159,709)
|
$
|
(108,444)
|
|
$
|
(265,709)
|
|
$
|
(208,159)
|
Net loss per share —
basic and diluted
|
|
$
|
(2.68)
|
$
|
(1.86)
|
|
$
|
(4.47)
|
|
$
|
(3.58)
|
Weighted-average number
of common shares used in net loss per share — basic and
diluted
|
|
|
59,617
|
|
58,406
|
|
|
59,465
|
|
|
58,216
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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SOURCE Blueprint Medicines Corporation