Blueprint Medicines Reports Second Quarter 2018 Financial Results
CAMBRIDGE, Mass., Aug. 1, 2018 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today reported financial results and provided a business update for the second quarter ended June 30, 2018.
"In the second quarter, Blueprint Medicines continued to advance a broad portfolio, with progress across multiple programs," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Importantly, we reported updated data from our Phase 1 EXPLORER trial in patients with advanced systemic mastocytosis that showed profound and durable clinical activity in nearly all patients. These data, combined with previously reported data from our ongoing Phase 1 NAVIGATOR trial in advanced gastrointestinal stromal tumors, reinforce our confidence in avapritinib as a potentially transformative therapy across multiple patient populations. By the end of this year, we expect to have four pivotal clinical trials of avapritinib underway, with the potential to rapidly advance toward approval in defined patient populations."
Clinical Programs:
Avapritinib: Gastrointestinal Stromal Tumors (GIST)
-- In June 2018, Blueprint Medicines announced the dosing of the first
patient in its Phase 3 VOYAGER clinical trial, which will evaluate the
safety and efficacy of avapritinib compared to regorafenib in patients
with third- or fourth-line advanced GIST.
-- In June 2018, Blueprint Medicines presented data from a retrospective
natural history study of patients with advanced PDGFR D842V-driven GIST
at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting.
The data confirmed that patients with advanced PDGFR D842V-driven GIST
are unlikely to respond to currently available tyrosine kinase
inhibitors (TKIs), illustrating the high unmet need for new therapies in
this patient population with a short survival rate.
-- Blueprint Medicines continues to evaluate avapritinib in its Phase 1
NAVIGATOR clinical trial and anticipates presenting updated data across
multiple patient populations, including PDGFRA-driven GIST, third-line
or later GIST and second-line GIST, in the second half of 2018.
Additionally, based on data from this trial, the Company plans to submit
a new drug application (NDA) to the U.S. Food and Drug Administration
(FDA) for avapritinib for the treatment of patients with PDGFRA-driven
GIST and fourth-line KIT-driven GIST in the first half of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)
-- In June 2018, Blueprint Medicines presented updated clinical data from
its ongoing Phase 1 EXPLORER clinical trial of avapritinib in patients
with advanced SM at the 23(rd) Congress of the European Hematology
Association. The data showed an overall response rate of 83 percent and
durable ongoing responses up to 22 months. All evaluable patients showed
marked decreases on one or more objective measures of mast cell burden,
regardless of advanced SM subtype, previous treatment or starting dose
level. The data also showed that avapritinib was generally
well-tolerated. Most adverse events reported by investigators were Grade
1 or 2, and only three patients discontinued due to a treatment-related
adverse event. Read the full data here.
-- Blueprint Medicines plans to initiate screening of patients for
enrollment in PATHFINDER, a registration-enabling, open-label,
single-arm Phase 2 clinical trial in patients with advanced SM, in the
third quarter of 2018, and plans to initiate PIONEER, a
registration-enabling, randomized, placebo-controlled Phase 2 clinical
trial in patients with indolent and smoldering SM, by the end of 2018.
BLU-667: RET-Altered Solid Tumors
-- Blueprint Medicines continues to enroll patients in the expansion
portion of its ongoing Phase 1 ARROW clinical trial of BLU-667 at a dose
of 400 mg once daily. In the expansion, patients are being enrolled in
four defined cohorts: RET-altered non-small cell lung cancer (NSCLC)
patients previously treated with a TKI; RET-altered NSCLC patients who
have not previously received any TKI treatment; patients with medullary
thyroid cancer; and patients with other RET-altered solid tumors.
BLU-554: Hepatocellular Carcinoma (HCC)
-- In June 2018, Blueprint Medicines announced plans to initiate a
proof-of-concept clinical trial with CStone Pharmaceuticals in China to
evaluate BLU-554 in combination with CS1001, a clinical-stage
anti-programmed death ligand-1 (PD-L1) immunotherapy being developed by
CStone Pharmaceuticals, as a first-line treatment for patients with HCC.
Additionally, the companies plan to expand Blueprint Medicines' ongoing
Phase 1 clinical trial of BLU-554 as a monotherapy to include new sites
in Mainland China. The companies expect to submit an investigational new
drug (IND) application for BLU-554 to the Chinese health authorities by
the end of 2018, and plan to initiate the clinical trial evaluating
BLU-554 in combination with CS1001 and the expansion of Blueprint
Medicines' ongoing clinical trial for BLU-554 as a monotherapy in
Mainland China in 2019.
Corporate:
-- In June 2018, Blueprint Medicines announced an exclusive collaboration
and license agreement with CStone Pharmaceuticals to develop and
commercialize avapritinib, BLU-554 and BLU-667 in Mainland China, Hong
Kong, Macau and Taiwan, either as a monotherapy or as part of a
combination therapy. Under the terms of the agreement, Blueprint
Medicines received an upfront cash payment of $40.0 million, will be
eligible to receive up to approximately $346.0 million in potential
milestone payments and tiered percentage royalties in the mid-teens to
low twenties on annual net sales of each licensed product in the
territory.
-- Blueprint Medicines recently received a $10.0 million milestone payment
from Roche following the achievement of a research milestone.
Second Quarter Financial Results:
-- Cash Position: As of June 30, 2018, cash, cash equivalents and
investments were $616.7 million, as compared to $673.4 million as of
December 31, 2017. This decrease was primarily related to cash used in
operating activities, partially offset by the $40.0 million upfront
payment received in connection with Blueprint Medicines entering into
the collaboration with CStone Pharmaceuticals and the $10.0 million
milestone payment received from Roche.
-- Collaboration Revenues: Collaboration revenues were $41.4 million for
the second quarter of 2018, as compared to $5.9 million for the second
quarter of 2017. This increase was primarily due to revenue recognized
under the collaboration agreement with CStone Pharmaceuticals.
-- R&D Expenses: Research and development expenses were $58.6 million for
the second quarter of 2018, as compared to $33.3 million for the second
quarter of 2017. This increase was primarily attributable to increased
clinical and manufacturing expenses associated with advancing
avapritinib, BLU-554 and BLU-667 further through clinical trials and
increased personnel-related expenses. Research and development expenses
included $4.3 million in stock-based compensation expenses for the
second quarter of 2018.
-- G&A Expenses: General and administrative expenses were $12.3 million for
the second quarter of 2018, as compared to $6.8 million for the second
quarter of 2017. This increase was primarily attributable to increased
personnel-related expenses and increased professional fees, including
pre-commercial planning activities. General and administrative expenses
included $3.5 million in stock-based compensation expenses for the
second quarter of 2018.
-- Net Loss: Net loss was $27.0 million the second quarter of 2018, or a
net loss per share of $0.62, as compared to a net loss of $33.4 million
for the second quarter of 2017, or a net loss per share of $0.86.
Financial Guidance:
Based on its current plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, excluding any potential option fees and milestone payments under its existing collaborations with Roche and CStone Pharmaceuticals, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2020.
Conference Call Information:
Blueprint Medicines will host a live conference call and webcast today at 8:30 a.m. ET. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international) and referring to conference ID 5597837. A webcast of the conference call will be available in the Investors 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 Blueprint Medicines:
Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases. Its approach is rooted in a deep understanding of the genetic blueprint of cancer and other disease driven by the abnormal activation of kinases. Blueprint Medicines is advancing multiple programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, as well as multiple programs in research and preclinical development. For more information, please visit www.blueprintmedicines.com.
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 clinical development of avapritinib, BLU-554, BLU-667 and BLU-782; the potential benefits of Blueprint Medicines' current and future drug candidates in treating patients; plans and timelines for presenting preclinical and clinical data for Blueprint Medicines' current or future clinical trials; plans and timelines for initiating Blueprint Medicines' PATHFINDER and PIONEER trials; plans and timelines for submitting an NDA to the FDA for avapritinib or an IND to the Chinese health authorities for BLU-554; expectations regarding Blueprint Medicines' existing cash, cash equivalents and investments; and Blueprint Medicines' strategy, business plans and focus. The words "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 delay of any current or planned clinical trials or the development of Blueprint Medicines' drug candidates, including avapritinib, BLU-554, BLU-667 and BLU-782; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates; 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, including companion diagnostic tests for BLU-554 for FGFR4-driven HCC, avapritinib for PDGFR D842V-driven GIST and BLU-667 for RET-driven NSCLC; and the success of Blueprint Medicines' cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc and Blueprint Medicines' collaboration with CStone Pharmaceuticals. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' Quarterly Report on Form 10-Q for the quarter ended March 31, 2018, as filed with the Securities and Exchange Commission (SEC) on May 2, 2018, 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)
June 30 December 31,
------- ------------
2018 2017
---- ----
Cash, cash equivalents and investments $616,651 $673,356
Working capital (1) 571,880 642,615
Total assets 665,113 715,737
Deferred revenue 48,295 35,373
Term loan payable 692 1,518
Lease incentive obligation 15,474 16,331
Total stockholders' equity 552,932 623,970
(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 Six Months Ended
June 30, June 30,
-------- --------
2018 2017 2018 2017
---- ---- ---- ----
Collaboration revenue $41,439 $5,890 $42,393 $11,730
Operating expenses:
Research and development 58,573 33,271 108,527 61,758
General and administrative 12,333 6,833 22,244 12,516
------ ----- ------ ------
Total operating expenses 70,906 40,104 130,771 74,274
Other income (expense):
Other income, net 2,442 861 4,836 1,286
Interest expense (23) (59) (55) (131)
--- --- --- ----
Total other income 2,419 802 4,781 1,155
----- --- ----- -----
Net loss $(27,048) $(33,412) $(83,597) $(61,389)
======== ======== ======== ========
Net loss per share applicable to common stockholders - basic and $(0.62) $(0.86) $(1.91) $(1.71)
diluted
Weighted-average number of common shares used in net loss per 43,856 38,775 43,779 35,998
share applicable to common stockholders - basic and diluted
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SOURCE Blueprint Medicines
