On August 1, 2018 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported financial results and provided a business update for the second quarter ended June 30, 2018 (Press release, Blueprint Medicines, AUG 1, 2018, View Source [SID1234528283]).
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"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 PDGFRα D842V-driven GIST at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 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 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 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). 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 View Source 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.