CAMBRIDGE, Mass., Nov. 5, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, is hosting its first R&D Day in New York City today.
During the event, Blueprint Medicines will outline its vision to become a leading platform-enabled, fully-integrated, global precision therapy company. The R&D Day presentation will highlight opportunities to expand the reach of the company's therapeutic candidates to broader patient populations, integrate and scale scientific, clinical and commercial capabilities to build therapeutic area leadership, and fully utilize the company's scientific platform to design innovative medicines targeting novel kinase biology. In addition, today the company reported financial results and provided a business update for the quarter ended September 30, 2019.
"As we prepare to launch our first medicine and submit multiple additional marketing applications next year, today we are unveiling our next wave of internally discovered research and clinical-stage precision therapies with the potential to deliver durable clinical benefits to additional patient populations," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "By fully leveraging our integrated research capabilities and reinvesting insights from our ongoing clinical programs, we continue to build a powerful research engine with the potential to deliver transformative treatment advances to patients as well as rapid and sustainable growth to Blueprint Medicines."
R&D Day Presentation Areas of Focus
Third Quarter 2019 Highlights and Recent Progress
Avapritinib: Gastrointestinal stromal tumors (GIST)
Avapritinib: Systemic mastocytosis (SM)
BLU-782: Fibrodysplasia ossificans progressiva (FOP)
Key Upcoming Milestones
The company expects to achieve the following milestones in the fourth quarter of 2019:
The company expects to achieve the following milestones related to planned marketing applications in 2020:
Third Quarter 2019 Financial Results
Financial Guidance
Based on its current plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, together with the $25.0 million upfront cash payment received under its license agreement with Clementia and an $8.0 million research milestone achieved in the fourth quarter of 2019 under the Roche collaboration, but excluding any additional potential option fees, milestone payments or other payments from Roche, CStone Pharmaceuticals or Clementia Pharmaceuticals, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2021.
Conference Call Information
Blueprint Medicines will host a live webcast of its R&D Day event at 8:30 a.m. ET today. The webcast may be accessed under "Events and Presentations" 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 90 days following the call.
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 are currently advancing three investigational medicines in clinical development, along with multiple 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 and timelines for the development of avapritinib, pralsetinib, fisogatinib, and BLU-263, including the timing, designs, implementation, enrollment, plans and announcement of results regarding Blueprint Medicines' ongoing and planned clinical trials for its drug candidates, including avapritinib, pralsetinib, fisogatinib and BLU-263; plans and timelines for nominating additional development candidates; plans and timelines for submitting an IND application to the FDA for BLU-263; plans and timelines for submitting marketing applications for avapritinib and pralsetinib; the potential benefits of Blueprint Medicines' current and future drug candidates in treating patients; plans, timelines and expectations for the FDA's review and administrative split of the NDA for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST; plans, timelines and expectations for top-line data from the VOYAGER trial; plans, timelines and expectations for the commercialization of avapritinib for the treatment of GIST, if approved by the FDA; potential benefits of the license agreement between Blueprint Medicines and Ipsen; expectations regarding Blueprint Medicines' existing cash, cash equivalents and investments; and Blueprint Medicines' strategy, goals and anticipated milestones, 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, pralsetinib, fisogatinib and BLU-263, or licensed products, including BLU-782; the FDA's intent to administratively split the proposed indications for avapritinib into two separate NDAs, which may not mean that either indication is approved; a delay in the review of the proposed indications as a result of the administrative split of the current NDA; FDA concerns regarding whether the response rate in the fourth-line GIST population was reasonably likely to predict clinical benefit in that population; there can be no assurance that the FDA will not ask for additional clinical trials for avapritinib; there can be no assurance that the VOYAGER top-line data will be sufficient for the FDA's review of the proposed fourth-line indication or that there will not be a delay in the availability of VOYAGER top-line data; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy 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 and licensing arrangement, including its cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., its collaboration with CStone Pharmaceuticals, and its license to Clementia Pharmaceuticals]. 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 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.
Blueprint Medicines Corporation | ||||||
September 30, | December 31, | |||||
2019 | 2018 | |||||
Cash, cash equivalents and investments | $ | 594,459 | $ | 494,012 | ||
Working capital (1) | 419,584 | 439,464 | ||||
Total assets | 737,925 | 540,124 | ||||
Deferred revenue | 41,331 | 46,167 | ||||
Total liabilities | 221,581 | 121,115 | ||||
Total stockholders' equity | 516,344 | 419,009 |
(1) | Blueprint Medicines defines working capital as current assets less current liabilities. |
Blueprint Medicines Corporation | ||||||
Three Months Ended | ||||||
September 30, | ||||||
2019 | 2018 | |||||
Collaboration revenue | $ | 9,139 | $ | 1,095 | ||
Operating expenses: | ||||||
Research and development | 81,453 | 64,562 | ||||
General and administrative | 25,647 | 12,041 | ||||
Total operating expenses | 107,100 | 76,603 | ||||
Other income (expense): | ||||||
Other income (expense), net | 3,692 | 2,799 | ||||
Interest expense | (6) | (14) | ||||
Total other income | 3,686 | 2,785 | ||||
Net loss | $ | (94,275) | $ | (72,723) | ||
Net loss per share — basic and diluted | $ | (1.93) | $ | (1.66) | ||
Weighted-average number of common shares used in net loss per share — basic and diluted | 48,921 | 43,915 |
SOURCE Blueprint Medicines