Blueprint Medicines Reports Third Quarter 2017 Financial Results
"In the third quarter, we continued to advance
Clinical Programs
BLU-285: Gastrointestinal Stromal Tumors
Blueprint Medicines continues to enroll patients in the expansion part of the ongoing Phase 1 clinical trial of its highly selective KIT and PDGFRα inhibitor BLU-285 in patients with advanced GIST.Blueprint Medicines expects to complete enrollment of the PDGFRα D842V expansion cohort, which is expected to include approximately 50 patients, by the middle 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 in the first half of 2018, with the goal of supporting the registration of BLU-285 in a broader GIST patient population.- Updated data from
Blueprint Medicines' ongoing Phase 1 clinical trial will be presented at the 22ndConnective Tissue Oncology Society (CTOS) Annual Meeting onFriday, November 10, 2017 inMaui, Hawaii . Following the presentation, a copy of the presentation will also be available under the "Abstracts, Posters and Presentations" section ofBlueprint Medicines' website at http://www.blueprintmedicines.com/abstracts-posters-and-presentations/.
BLU-285: Systemic Mastocytosis
Blueprint Medicines continues to enroll patients in the expansion part of the ongoing Phase 1 clinical trial of BLU-285 in patients with advanced SM.Blueprint Medicines anticipates presenting updated data from this clinical trial by the end of 2017.
BLU-554: Hepatocellular Carcinoma
- In
September 2017 ,Blueprint Medicines presented updated data from its ongoing Phase 1 clinical trial of BLU-554 in patients with advanced HCC at theEuropean Society for Medical Oncology (ESMO) 2017Congress inMadrid, Spain and the 11thInternational Liver Cancer Association (ILCA) Annual Conference inSeoul, South Korea . In evaluable patients with fibroblast growth factor receptor 4 (FGFR4)-driven HCC, the data showed an objective response rate (ORR) of 16 percent by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. In addition, 49 percent of patients with FGFR4-driven HCC experienced radiographic tumor reduction, and clinical activity was observed regardless of disease etiology, prior treatment with immunotherapy, such as nivolumab, or geography. In contrast, currently approved tyrosine kinase inhibitors (TKIs) provide response rates of less than 10 percent. Preliminary evidence of prolonged disease control was observed in patients with FGFR4-driven HCC who had not received prior treatment with a TKI, such as sorafenib. The data also showed that BLU-554 was well-tolerated, and most adverse events reported by investigators were Grade 1 or 2 (mild or moderate). Read the full data here. - Also in
September 2017 ,Blueprint Medicines announced plans to initiate in the first quarter of 2018 an additional cohort in its ongoing Phase 1 clinical trial to evaluate BLU-554 in TKI-naïve patients with FGFR4-driven HCC and to explore opportunities to conduct a clinical trial to evaluate BLU-554 in combination with an immune checkpoint inhibitor.
BLU-667: RET-altered solid tumors
Blueprint Medicines continues to enroll patients in the dose escalation part of its ongoing Phase 1 clinical trial of its highly selective RET inhibitor BLU-667 in patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other RET-altered solid tumors. OnOctober 29, 2017 , new preclinical data supporting the clinical development of BLU-667 in patients with RET-altered solid tumors was presented at theAACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics inPhiladelphia, Pennsylvania . Reported in vitro and in vivo study results showed that BLU-667 is active against KIF5B-RET fusion NSCLC as well as predicted resistance mutations, including the V804M gatekeeper mutation, without inhibition of off-target kinases with known toxicity profiles, such as vascular endothelial growth factor receptor 2.- In
October 2017 ,Blueprint Medicines entered into a collaboration withThermo Fisher Scientific to develop and commercialize a companion diagnostic test to identify NSCLC patients with RET fusions for use with BLU-667.
Research Programs
- Based on a scientific review of discovery-stage portfolio assets,
Blueprint Medicines plans to prioritize continued development of its research program targeting the ALK2 kinase for the treatment of fibrodysplasia ossificans progressiva (FOP) with the goal of identifying a potential development candidate. The termination ofBlueprint Medicines' collaboration withAlexion Pharma Holding (Alexion) became effective inOctober 2017 , enablingBlueprint Medicines to transition the FOP program into its research portfolio as a wholly-owned program. As a result of the termination,Blueprint Medicines' exclusivity obligations under the Alexion agreement terminated, andBlueprint Medicines will not have any current or future payment obligations to Alexion. - Based on a scientific review of its discovery-stage portfolio assets,
Blueprint Medicines plans to deprioritize its research program targeting protein kinase cAMP-activated catalytic subunit alpha fusions (PRKACA) for the treatment of fibrolamellar carcinoma (FLC). Blueprint Medicines also recently initiated an additional discovery program for an undisclosed kinase target.
Corporate Highlights
- In
October 2017 ,Blueprint Medicines announced the appointment ofChristopher Murray , Ph.D., as Senior Vice President, Technical Operations. In this new role, Dr. Murray will be responsible for technical development, manufacturing and supply chain operations, and he will support the expansion of clinical activities forBlueprint Medicines' lead product candidates and plans to address potential future commercial supply demand.
Third Quarter 2017 Financial Results
- Cash Position: As of
September 30, 2017 , cash, cash equivalents and investments were$390.7 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$85.1 million in cash used to fund operating activities for the nine months endedSeptember 30, 2017 . - Collaboration Revenue: Collaboration revenues were
$8.1 million for the third quarter of 2017, compared to$6.2 million for the third quarter of 2016. This increase was primarily due to revenue recognized under the Alexion collaboration in connection with the termination of the collaboration and transition of the FOP program. - R&D Expenses: Research and development expenses were
$39.3 million for the third quarter of 2017, including$1.8 million of stock-based compensation expenses, as compared to$18.2 million for the third quarter of 2016, including$0.6 million of stock-based compensation expense. This increase was primarily due to increased clinical and manufacturing expenses associated with advancingBlueprint Medicines' ongoing clinical trials for BLU-285, BLU-554, and BLU-667 and increased personal-related expenses. - G&A Expenses: General and administrative expenses were
$7.4 million for the third quarter of 2017, including$1.8 million of stock-based compensation expense, as compared to$4.9 million for the third quarter of 2016, including$1.3 million of stock-based compensation expense. This increase was primarily due to increased personnel expenses related to an increase in headcount, as well as increased professional fees. - Net Loss: Net loss was
$37.7 million for the third quarter of 2017, or a net loss per share of$0.96 , as compared to a net loss of$16.8 million for the third quarter of 2016, or a net loss per share of$0.62 .
Financial Guidance
Based on its current plans,
Conference Call Information
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 Blueprint Medicines communicates with its investors and the public using its company website (www.blueprintmedicines.com), including but not limited to investor presentations and scientific presentations, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. You can also connect with Blueprint Medicines on Twitter (@BlueprintMeds) or LinkedIn. The information that Blueprint Medicines posts on these channels and websites could be deemed to be material information. As a result, Blueprint Medicines encourages investors, the media and others interested in Blueprint Medicines to review the information that it posts on these channels, including Blueprint Medicines' investor relations website, on a regular basis. This list of channels may be updated from time to time on Blueprint Medicines' investor relations website and may include other social media channels than the ones described above. The contents of Blueprint Medicines' website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
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) |
||||||
September 30, |
December 31, |
|||||
2017 |
2016 |
|||||
Cash, cash equivalents and investments |
$ |
390,652 |
$ |
268,218 |
||
Unbilled accounts receivable |
3,435 |
3,577 |
||||
Working capital (1) |
364,789 |
191,913 |
||||
Total assets |
420,118 |
282,795 |
||||
Deferred revenue |
36,772 |
47,235 |
||||
Term loan payable |
2,095 |
4,069 |
||||
Lease incentive obligation |
9,900 |
3,370 |
||||
Total stockholders' equity |
341,240 |
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 |
Nine Months Ended |
|||||||||||
September 30, |
September 30, |
|||||||||||
2017 |
2016 |
2017 |
2016 |
|||||||||
Collaboration revenue |
$ |
8,068 |
$ |
6,160 |
$ |
19,798 |
$ |
20,081 |
||||
Operating expenses: |
||||||||||||
Research and development |
39,300 |
18,150 |
101,058 |
57,058 |
||||||||
General and administrative |
7,378 |
4,893 |
19,894 |
14,227 |
||||||||
Total operating expenses |
46,678 |
23,043 |
120,952 |
71,285 |
||||||||
Other income (expense): |
||||||||||||
Other income (expense), net |
954 |
158 |
2,240 |
350 |
||||||||
Interest expense |
(47) |
(109) |
(178) |
(378) |
||||||||
Total other income (expense) |
907 |
49 |
2,062 |
(28) |
||||||||
Net loss |
$ |
(37,703) |
$ |
(16,834) |
$ |
(99,092) |
$ |
(51,232) |
||||
Net loss per share applicable to common stockholders — basic |
$ |
(0.96) |
$ |
(0.62) |
$ |
(2.67) |
$ |
(1.89) |
||||
Weighted-average number of common shares used in net loss per |
39,130 |
27,251 |
37,053 |
27,170 |
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SOURCE
Kristin Hodous, 617-714-6674, khodous@blueprintmedicines.com, Jim Baker, 617-844-8236, jbaker@blueprintmedicines.com