Blueprint Medicines Reports Second Quarter 2018 Financial Results
"In the second quarter,
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 PDGFRα D842V-driven GIST at the 2018American Society of Clinical Oncology (ASCO ) Annual Meeting. The data confirmed that patients with advanced PDGFRα 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 theU.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 23rdCongress of theEuropean 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 withCStone Pharmaceuticals inChina to evaluate BLU-554 in combination with CS1001, a clinical-stage anti-programmed death ligand-1 (PD-L1) immunotherapy being developed byCStone Pharmaceuticals , as a first-line treatment for patients with HCC. Additionally, the companies plan to expandBlueprint 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 ofBlueprint 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 withCStone Pharmaceuticals to develop and commercialize avapritinib, BLU-554 and BLU-667 in Mainland China,Hong Kong ,Macau andTaiwan , 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 fromRoche 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 ofDecember 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 withBlueprint Medicines entering into the collaboration withCStone Pharmaceuticals and the$10.0 million milestone payment received fromRoche . - 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 withCStone 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,
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
About
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.
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 Corporation |
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Selected Condensed Consolidated Balance Sheet Data |
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(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) |
||||
Weighted-average number of common shares used in net loss per |
43,856 |
38,775 |
43,779 |
35,998 |
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SOURCE
Kristin Hodous, 617 714 6674, khodous@blueprintmedicines.com; Jim Baker, 617 844 8236, jbaker@blueprintmedicines.com