-- Achieved $23.8
million in AYVAKIT® (avapritinib) net product
revenues, and $62.7 million in total
revenues --
-- AYVAKYT® (avapritinib) launch in
Germany underway following
European Commission approval for advanced SM on March 25 --
-- First patients dosed in BLU-701, BLU-451,
and BLU-222 clinical trials; trial cohort initiated for BLU-945 in
combination with osimertinib --
-- On track to report topline data for
registration-enabling PIONEER trial of AYVAKIT in non-advanced SM
in late summer 2022 --
CAMBRIDGE, Mass., May 3, 2022 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC) today reported financial results and
provided a business update for the first quarter ended March 31, 2022.
"The first quarter was marked by a number of important
milestones across our business. We have strong momentum in our
commercial execution, our pipeline is advancing rapidly, and we
continue to build on our scientific leadership," said Kate Haviland, Chief Executive Officer of
Blueprint Medicines. "Our ongoing global launch of
AYVAKIT®/AYVAKYT® (avapritinib) in the U.S. and now also in
Europe is establishing a new
standard of care for the treatment of advanced SM, targeting the
underlying cause of the disease. We are on track to have topline
data from our registration-enabling PIONEER trial in late summer,
further expanding our leadership in SM as we potentially bring the
first and only medicine to patients with the non-advanced form of
the disease. In addition, we continue to progress our pipeline of
innovative investigational medicines in difficult-to-treat and
prevalent cancers such as non-small cell lung cancer and breast
cancer. At the American Association for Cancer Research (AACR)
Annual Meeting, we presented five abstracts highlighting data
across four programs within our EGFR and CDK2 franchises, while
earlier in the quarter we announced a targeted protein degradation
discovery collaboration with Proteovant Therapeutics. The
strength of this past quarter demonstrates that we are well on our
way of delivering on our 2022 goals while also building the
foundation to drive further value for the years ahead."
First Quarter 2022 Highlights and Recent Progress
AYVAKIT®/AYVAKYT® (avapritinib): advanced systemic
mastocytosis (SM) and PDGFRA gastrointestinal stromal tumor
(GIST)
- Recorded global net product revenues of $23.8 million for the first quarter of 2022.
- Received European Commission approval for AYVAKYT for the
treatment of adult patients with advanced SM, including aggressive
SM, SM with an associated hematological neoplasm, or mast cell
leukemia, after at least one systemic therapy, and treated the
first commercial patients in Germany. Read the press release here.
GAVRETO® (pralsetinib): RET-altered cancers
- As previously recorded and reported by Roche, GAVRETO product
sales for their region were 5 million
CHF, which excludes sales in the Greater China territory driven by CStone
Pharmaceuticals.
BLU-945, BLU-701 and BLU-451 (formerly LNG-451): EGFR-driven
NSCLC
- Reported proof-of-concept data at AACR from the Phase 1/2
SYMPHONY clinical trial of BLU-945, showing early evidence of
safety and clinical activity, with dose-dependent decreases in
circulating tumor DNA (EGFR variant allele fractions) and
radiographic tumor reductions, including an unconfirmed partial
response (PR) in a patient treated with 400 mg once daily (QD).
BLU-945 was generally well-tolerated, with no significant adverse
events (AEs) associated with wild-type EGFR inhibition. The maximum
tolerated dose and recommended Phase 2 dose have not yet been
identified, and dose escalation is continuing. These results
support plans to expand the development of BLU-945 in combination
with multiple agents, including osimertinib, with the goal of
preventing or treating tumor resistance to prolong patient benefit.
Read the press release here.
- Entered into a clinical trial supply agreement with AstraZeneca
(LSE/STO/Nasdaq:AZN), under which Blueprint Medicines will evaluate
BLU-945 and BLU-701 in combination with osimertinib in the ongoing
SYMPHONY and HARMONY trials, respectively.
- Also at AACR, reported preclinical data supporting the
development of BLU-451 in EGFR exon 20 insertion-positive
NSCLC.
- Initiated patient dosing in the CONCERTO trial, a Phase 1/2
trial of BLU-451 in patients with EGFR-driven NSCLC harboring exon
20 insertion mutations.
BLU-222: breast, ovarian, and other CDK2-vulnerable cancers,
including CCNE1-amplified tumors
- Reported preclinical data in a CCNE1-amplified ovarian tumor
model at AACR supporting the development of BLU-222 in
CDK2-vulnerable cancers.
- Initiated the VELA trial, a Phase 1/2 trial of BLU-222 in
CDK2-vulnerable cancers, including estrogen-receptor-positive
breast cancer and a range of other CCNE1-amplified tumors, and
dosed the first patient in Part 1 dose escalation.
Corporate
- Announced strategic collaboration with Proteovant Therapeutics
to advance novel targeted protein degrader therapies to address
important areas of medical need. Under the terms of the
collaboration, the companies will jointly research important
targets and advance up to four novel protein degrader therapies
into development candidates. Read the press release here.
- Recognized a $30 million
milestone payment from Clementia related to the initiation of a
Phase 2 trial of BLU-782, which is now called IPN60130, our
out-licensed ALK2 inhibitor in development for the rare bone
disease fibrodysplasia ossificans progressiva.
Key Upcoming Milestones
The company plans to achieve the following near-term
milestones:
- Report topline data from the registration-enabling Part 2 of
the PIONEER trial of AYVAKIT in non-advanced SM in late summer 2022
and submit a supplemental new drug application to the U.S. Food and
Drug Administration for AYVAKIT in non-advanced SM in the second
half of 2022.
- Present initial data from the dose escalation cohort of the
Phase 1/2 SYMPHONY trial evaluating BLU-945 in combination with
osimertinib in the second half of 2022.
- Present initial clinical data from the Phase 1/2 HARMONY trial
of BLU-701 in the second half of 2022.
- Present initial data from Part 1 of the HARBOR trial of BLU-263
in non-advanced SM in the second half of 2022.
- Share the company's research and portfolio vision, including
scientific platform expansion plans, at an R&D Day in the
second half of 2022.
First Quarter 2022 Results
- Revenues: Revenues were $62.7 million for
the first quarter of 2022, including $23.8 million of net
product revenues from sales of AYVAKIT/AYVAKYT and $38.9
million in collaboration revenues. Blueprint
Medicines recorded revenues of $21.6 million in the
first quarter of 2021, including $7.1 million of net
product revenues from sales of AYVAKIT/AYVAKIT, $1.8 million of net product revenues from sales
of GAVRETO and $12.6 million in collaboration
revenues.
- Cost of Sales: Cost of sales was $5.1
million for the first quarter of 2022, as compared
to $0.1 million for the first quarter of 2021.
- R&D Expenses: Research and development expenses
were $103.1 million for the first quarter of 2022, as
compared to $79.7 million for the first quarter of 2021.
This increase was primarily due to increased costs associated with
the progression of our clinical trials, increased costs related to
early discovery efforts, and a decrease in reimbursement from the
global development cost sharing arrangement under our collaboration
with Roche for GAVRETO. Research and development expenses
included $10.0 million in stock-based compensation
expenses for the first quarter of 2022.
- SG&A Expenses: Selling, general and
administrative expenses were $57.1 million for the first
quarter of 2022, as compared to $42.0 million for the
first 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 $13.4 million in stock-based
compensation expenses for the first quarter of 2022.
- Net Loss: Net loss was $106.0 million for
the first quarter of 2022, or a net loss per share of $1.79,
as compared to a net loss of $99.7 million for the first
quarter of 2021, or a net loss per share of $1.72.
- Cash Position: As of March 31, 2022, cash,
cash equivalents and investments were $893.4 million, as
compared to $1,034.6 million as of December 31,
2021.
Financial Guidance
Blueprint Medicines continues to anticipate approximately
$180 to $200
million in total net revenues in 2022, including
approximately $115 to $130 million 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 8:30 a.m. ET today to discuss first 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.
Upcoming Investor Conferences
Blueprint Medicines will participate in three upcoming investor
conferences:
- Jefferies Healthcare Conference on Tuesday, June 9, 2022 in New York, NY.
- JMP Securities 2022 Life Sciences Conference on Thursday, June 16, 2022 in New York, NY.
- Goldman Sachs 43rd Annual Global Healthcare
Conference on Thursday, June 16, 2022
in Rancho Palos Verdes, CA.
A live webcast of each presentation will be available by
visiting the Investors & Media section of Blueprint Medicines'
website at http://ir.blueprintmedicines.com. A replay of the
webcasts will be archived on Blueprint Medicines' website for 30
days following each presentation.
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
Blueprint Medicines' current or future approved drugs and drug
candidates, including timelines for marketing applications and
approvals, the initiation of clinical trials and trial cohorts, or
the results of ongoing and planned clinical trials; expectations
regarding the standard of care for the treatment of advanced
SM; Blueprint Medicines' plans, strategies and timelines to
nominate development candidates; the anticipated benefits of the
preclinical profiles of Blueprint Medicines' drug 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' 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, 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)
|
|
|
|
March
31,
|
|
December
31,
|
|
|
2022
|
|
2021
|
Cash, cash
equivalents and marketable securities
|
$
|
893,351
|
|
$
|
1,034,643
|
Working capital
(1)
|
|
550,219
|
|
|
404,260
|
Total
assets
|
|
1,143,307
|
|
|
1,252,225
|
Deferred
revenue
|
|
23,325
|
|
|
36,576
|
Total
liabilities
|
|
261,641
|
|
|
281,490
|
Total stockholders'
equity
|
|
881,666
|
|
|
970,735
|
|
|
(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,
|
|
2022
|
|
2021
|
Revenues:
|
|
|
|
|
|
Product revenue,
net
|
$
|
23,841
|
|
$
|
8,955
|
Collaboration
revenue
|
|
38,890
|
|
|
12,621
|
Total
revenues
|
$
|
62,731
|
|
$
|
21,576
|
Cost and operating
expenses:
|
|
|
|
|
|
Cost of
sales
|
|
5,079
|
|
|
102
|
Collaboration loss
sharing
|
|
3,265
|
|
|
—
|
Research and
development
|
|
103,133
|
|
|
79,710
|
Selling, general and
administrative
|
|
57,058
|
|
|
42,002
|
Total cost and
operating expenses
|
$
|
168,535
|
|
$
|
121,814
|
Other income
(expense):
|
|
|
|
|
|
Interest income,
net
|
|
442
|
|
|
738
|
Other expense,
net
|
|
(453)
|
|
|
(214)
|
Total other income
(expense)
|
$
|
(11)
|
|
$
|
524
|
Loss before income
taxes
|
$
|
(105,815)
|
|
$
|
(99,714)
|
Income tax
expense
|
|
184
|
|
|
—
|
Net loss
|
$
|
(105,999)
|
|
$
|
(99,714)
|
Net loss per share
applicable to common stockholders — basic and
diluted
|
$
|
(1.79)
|
|
$
|
(1.72)
|
Weighted-average
number of common shares used in net loss per share applicable to
common stockholders — basic and diluted
|
|
59,312
|
|
|
58,023
|
|
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SOURCE Blueprint Medicines Corporation