CAMBRIDGE, Mass., April 28, 2020 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC), a precision therapy company
focused on genomically defined cancers, rare diseases and cancer
immunotherapy, today announced top-line results from the Phase 3
VOYAGER clinical trial of avapritinib versus regorafenib in
patients with locally advanced unresectable or metastatic
gastrointestinal stromal tumor (GIST). The VOYAGER trial did not
meet the primary endpoint of an improvement in progression-free
survival (PFS) for avapritinib versus regorafenib. Top-line safety
data for avapritinib were consistent with those previously
reported.
Blueprint Medicines plans to continue to commercialize AYVAKIT™
(avapritinib) in the United States
for the treatment of adults with unresectable or metastatic GIST
harboring a PDGFRA exon 18 mutation, including PDGFRA D842V
mutations, and seek marketing approval for avapritinib for the
treatment of this patient population in additional geographies.
Blueprint Medicines continues to anticipate a decision from the
European Commission on its marketing authorization application for
the treatment of adults with PDGFRA D842V mutant GIST in the third
quarter of 2020. Based on the top-line VOYAGER data, the company
plans to discontinue further development of avapritinib in GIST
beyond PDGFRA exon 18 mutant GIST.
"While we are disappointed by the outcome of the VOYAGER trial,
we are deeply grateful to the patients, investigators and clinical
site staff who contributed to the completion of this global study.
We hope these data will reveal important insights to improve the
scientific understanding of the disease and inform future
innovations in GIST, and we are committed to sharing the results at
a future medical meeting," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines. "At
Blueprint Medicines, we strive to advance science, building on
successes and learning from setbacks, to create new medicines for
patients with difficult-to-treat cancers and rare diseases. With a
deep portfolio of precision therapies and a strong financial
position, we will continue to advance our pipeline with clear
near-term priorities in systemic mastocytosis and RET-altered
cancers."
Top-line Data from Phase 3 VOYAGER Trial
The VOYAGER trial evaluated the efficacy and safety of
avapritinib (N=240) versus regorafenib (N=236) in patients with
third- or fourth-line GIST.
Avapritinib showed a median PFS of 4.2 months compared to 5.6
months for regorafenib. The difference in median PFS between the
avapritinib and regorafenib groups was not statistically
significant. The overall response rate was 17 percent for the
avapritinib group and 7 percent for the regorafenib group.
Avapritinib was generally well-tolerated with most adverse
events reported as Grade 1 or 2. Top-line safety results were
consistent with previously reported data, and no new safety signals
were observed.
Additional analyses of the VOYAGER trial results are ongoing,
and Blueprint Medicines plans to present the data at a future
medical meeting.
Financial Guidance
Based on its current operating plans, Blueprint Medicines
continues to expect that its existing cash, cash equivalents and
investments 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.
Anticipated savings from the discontinuation of further development
of avapritinib in non-PDGFRA exon 18 mutant GIST indications is
expected to offset any previously forecast revenues from those
indications through 2022.
As of December 31, 2019, Blueprint
Medicines had cash, cash equivalents and investments of
$548.0 million. In addition,
Blueprint Medicines received $308.4
million in estimated net proceeds from its January 2020 follow-on public offering.
Conference Call Information
Blueprint Medicines will host a live webcast today beginning at
8:00 a.m. ET to discuss the top-line
data from the VOYAGER trial. To access the live call, please dial
(855) 728-4793 (domestic) or (503) 343-6666 (international), and
refer to conference ID 2207088. A webcast of the conference call
will be available in the Investors & Media section of 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 the VOYAGER Trial
VOYAGER was a global, open-label, randomized, Phase 3 trial
designed to evaluate the efficacy and safety of avapritinib versus
regorafenib in patients with third- or fourth-line GIST. Patients
were randomized 1:1 to receive either avapritinib (300 mg once
daily dosing) or regorafenib (160 mg once daily dosing for three
out of every four weeks) at multiple sites in the United States, Canada, European Union, Australia and Asia. The primary efficacy endpoint was PFS by
blinded, independent central radiology review, based on modified
Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST
1.1 criteria) for GIST. For more information about the VOYAGER
trial, please visit www.clinicaltrials.gov (ClinicalTrials.gov
Identifier: NCT03465722).
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S.
Food and Drug Administration (FDA) for the treatment of adults with
unresectable or metastatic GIST harboring a PDGFRA exon 18
mutation, including PDGFRA D842V mutations. AYVAKIT is the first
precision therapy approved to treat a genomically defined
population of patients with GIST and the only highly active
treatment for PDGFRA exon 18 mutant GIST. The FDA granted
Breakthrough Therapy Designation to avapritinib for the treatment
of unresectable or metastatic GIST harboring the PDGFRA D842V
mutation. For more information, visit AYVAKIT.com.
Avapritinib is not approved for the treatment of any other
indication in the U.S. or any other jurisdiction by the FDA or any
other health authority.
Blueprint Medicines is developing avapritinib globally for
the treatment of advanced, smoldering and indolent systemic
mastocytosis (SM). The FDA granted Breakthrough Therapy Designation
to avapritinib for the treatment of advanced SM, including the
subtypes of aggressive SM, SM with an associated hematologic
neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license
agreement with CStone Pharmaceuticals for the development and
commercialization of avapritinib and certain other drug candidates
in Mainland China, Hong Kong,
Macau and Taiwan. Blueprint Medicines retains
development and commercial rights for avapritinib in the rest of
the world.
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 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 to continue to commercialize AYVAKIT in
the United States for the
treatment of adults with unresectable or metastatic GIST harboring
a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and
plans to seek marketing approval for avapritinib for the treatment
of this patient population in additional geographies; expectations
and timing for a decision from the European Commission on the
marketing authorization application for the treatment of adults
with PDGFRA D842V mutant GIST; plans and timing for presenting
VOYAGER trial results; plans to discontinue further development of
avapritinib for GIST indications other than PDGFRA exon 18 mutant
GIST; expectations regarding anticipated savings from the
discontinuation of further development of avapritinib in non-PDGFRA
exon 18 mutant GIST indications; expectations regarding Blueprint
Medicines' existing cash, cash equivalents and investments; 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 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, including Blueprint Medicines' ability to
obtain FDA approval for its pending new drug application for
avapritinib for the treatment of fourth-line GIST, 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 efficacy and safety 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.
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