Blueprint Medicines Reports Second Quarter 2017 Financial Results
"Our vision of growing
Clinical Programs
BLU-285: Gastrointestinal Stromal Tumors
- In
June 2017 ,Blueprint Medicines presented updated data from its ongoing Phase 1 clinical trial for BLU-285 in advanced GIST in an oral presentation at the 2017 ASCO Annual Meeting. BLU-285 is a potent and highly selective PDGFRα and KIT inhibitor in development as a potential treatment for patients with PDGFRα-driven GIST and KIT-driven GIST. In evaluable patients with PDGFRα-driven GIST harboring a D842 mutation, the data showed an objective response rate (ORR) of 60 percent by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. In addition, median PFS was not reached for the evaluable PDGFRα-driven GIST patients, and 9-month progression free survival (PFS) was estimated to be 87 percent. In contrast, historical data showed a zero percent ORR and a median PFS of about three months in patients with PDGFRα D842V-driven GIST treated with imatinib. Among patients with treatment-resistant KIT-driven GIST treated at higher dose levels, key observations included tumor reduction in eight of 14 evaluable patients and prolonged progression free survival. The data also showed that BLU-285 was well-tolerated, and most adverse events reported by investigators were Grade 1 or 2. Read the full data here. - Also in
June 2017 ,Blueprint Medicines announced that theU.S. Food and Drug Administration (FDA ) granted Breakthrough Therapy Designation to BLU-285 for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation. Based on feedback from theFDA at the End-of-Phase 1 meeting,Blueprint Medicines believes that additional data from the expansion portion of its ongoing Phase 1 clinical trial of BLU-285 in advanced GIST may be sufficient to support a New Drug Application for BLU-285 for the treatment of patients with PDGFRα D842V-driven GIST.Blueprint Medicines currently estimates it will complete enrollment of the PDGFRα D842V expansion cohort, which is expected to include approximately 50 patients, by the middle of 2018. In the first half of 2018,Blueprint Medicines also plans to initiate a global, pivotal Phase 3 clinical trial of BLU-285 in third line patients with KIT-driven GIST, with the goal of supporting the registration of BLU-285 in a broader GIST patient population.
BLU-285: Systemic Mastocytosis
Blueprint Medicines completed enrollment in the dose escalation portion of its ongoing Phase 1 clinical trial of BLU-285 in patients with advanced systemic mastocytosis and recently began enrolling patients in the dose expansion portion of this clinical trial at the recommended dose of 300 mg once daily.Blueprint Medicines anticipates providing updated data from this clinical trial by the end of 2017.
BLU-554: Hepatocellular Carcinoma
Blueprint Medicines continues to enroll patients in the dose expansion stage of its ongoing Phase 1 clinical trial for BLU-554 in advanced HCC and will present updated data from this clinical trial in an oral presentation, "Phase 1 safety and clinical activity of BLU-554 in advanced hepatocellular carcinoma (HCC)," at the ESMO 2017Congress onSunday, September 10, 2017 inMadrid, Spain . The accepted abstract includes results as of the data cut-off date ofApril 20, 2017 , andBlueprint Medicines expects to present clinical trial results updated since the abstract data cut-off date at the upcoming ESMO 2017Congress .
Corporate Highlights
- Closed Public Offering: In
April 2017 ,Blueprint Medicines announced the closing of an underwritten public offering of 5,750,000 shares of its common stock at a public offering price of$40.00 per share, including the exercise in full by the underwriters of their option to purchase additional shares of common stock.Blueprint Medicines received net proceeds from the offering of$215.6 million , after deducting underwriting discounts and commissions and offering expenses. - Discovery Program in Fibrodysplasia Ossificans Progressiva (FOP):
Blueprint Medicines is evaluating opportunities to advance its rare disease discovery program in FOP as a result of Alexion's decision to discontinue the collaboration withBlueprint Medicines following a strategic review of Alexion's business and research and development portfolio.
Second Quarter 2017 Financial Results
- Cash Position: As of
June 30, 2017 , cash, cash equivalents and investments were$421.0 million , as compared to$268.2 million as ofDecember 31, 2016 . This increase was primarily related to$215.6 million in net proceeds from theApril 2017 follow-on underwritten public offering, partially offset by$58.6 million in cash used to fund operating activities. - Collaboration Revenue: Collaboration revenues were
$5.9 million for the second quarter of 2017, as compared to$7.1 million for the second quarter of 2016. This decrease was primarily due to lower revenue recognized under the Alexion collaboration. - R&D Expenses: Research and development expenses were
$33.3 million for the second quarter of 2017, including$1.5 million of stock-based compensation expense, as compared to$21.3 million for the second quarter of 2016, including$0.8 million of stock-based compensation expense. This increase was primarily attributable to increased clinical and manufacturing expenses associated with the ongoing clinical trials for BLU-285, BLU-554 and BLU-667 and increased personnel-related expenses. - G&A Expenses: General and administrative expenses were
$6.8 million for the second quarter of 2017, including$1.5 million of stock based compensation expense, as compared to$4.7 million for the second quarter of 2016, including$0.7 million of stock based compensation expense. This increase was primarily attributable to increased personnel-related expenses to support our growing operations. - Net Loss: Net loss was
$33.4 million for the second quarter of 2017, or a net loss per share of$0.86 , as compared to a net loss of$18.9 million for the second quarter of 2016, or a net loss per share of$0.70 .
Financial Guidance
Based on its current plans,
Conference Call Information
Blueprint Medicines will host a live conference call and webcast today at
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 diseases driven by the abnormal activation of kinases. Blueprint Medicines is advancing four 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.
Availability of Other Information About Blueprint Medicines
Investors and others should note that
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 BLU-285, BLU-554 and BLU-667; the timing of updated clinical data for
Blueprint Medicines Corporation Selected Condensed Consolidated Balance Sheet Data (in thousands) (unaudited) |
||||||
June 30, |
December 31, |
|||||
2017 |
2016 |
|||||
Cash, cash equivalents and investments |
$ |
421,014 |
$ |
268,218 |
||
Unbilled accounts receivable |
3,075 |
3,577 |
||||
Working capital (1) |
398,336 |
191,913 |
||||
Total assets |
444,721 |
282,795 |
||||
Deferred revenue |
41,405 |
47,235 |
||||
Term loan payable |
2,671 |
4,069 |
||||
Lease incentive obligation |
4,719 |
3,370 |
||||
Total stockholders' equity |
374,089 |
213,078 |
||||
(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, |
|||||||||||
2017 |
2016 |
2017 |
2016 |
|||||||||
Collaboration revenue |
$ |
5,890 |
$ |
7,065 |
$ |
11,730 |
$ |
13,921 |
||||
Operating expenses: |
||||||||||||
Research and development |
33,271 |
21,273 |
61,758 |
38,908 |
||||||||
General and administrative |
6,833 |
4,688 |
12,516 |
9,334 |
||||||||
Total operating expenses |
40,104 |
25,961 |
74,274 |
48,242 |
||||||||
Other income (expense): |
||||||||||||
Other income (expense), net |
861 |
131 |
1,286 |
192 |
||||||||
Interest expense |
(59) |
(129) |
(131) |
(269) |
||||||||
Total other income (expense) |
802 |
2 |
1,155 |
(77) |
||||||||
Net loss |
$ |
(33,412) |
$ |
(18,894) |
$ |
(61,389) |
$ |
(34,398) |
||||
Net loss per share applicable to common stockholders — basic and |
$ |
(0.86) |
$ |
(0.70) |
$ |
(1.71) |
$ |
(1.27) |
||||
Weighted-average number of common shares used in net loss per |
38,775 |
27,170 |
35,998 |
27,129 |
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SOURCE
Media and Investor Relations Contacts: Kristin Hodous, 617-714-6674, khodous@blueprintmedicines.com or Jim Baker, 617-844-8236, jbaker@blueprintmedicines.com