On October 30, 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 third quarter ended September 30, 2018 (Press release, Blueprint Medicines, OCT 30, 2018, View Source;q=View Source;source=gmail&ust=1541070221980000&usg=AFQjCNEV72tiQ5N9U9Dom5ERVILd_aDycQ [SID1234530361]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"Our third quarter and recent accomplishments represent tremendous progress across our portfolio, marked by the receipt of positive regulatory feedback from the FDA for avapritinib and BLU-667 and the presentation of new data across our clinical- and research-stage pipeline," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "As we prepare to enter a critical year, we are now accelerating our efforts with a focus on executing five registration-enabling studies across seven patient populations, building a global commercial enterprise to deliver medicines to patients and investing in the next generation of precision therapies."
Clinical Programs:
Avapritinib: Systemic Mastocytosis (SM)
Blueprint Medicines announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to avapritinib for the treatment of patients with advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
Blueprint Medicines announced that it has screened the first patient in PATHFINDER, its registration-enabling, open-label, single-arm Phase 2 clinical trial in patients with advanced SM. Blueprint Medicines expects to initiate PIONEER, its registration-enabling, randomized, placebo-controlled Phase 2 clinical trial in patients with indolent and smoldering SM, by the end of 2018.
Blueprint Medicines announced that the European Medicines Agency has granted orphan drug designation to avapritinib for the treatment of mastocytosis.
Enrollment in the expansion portion of the Phase 1 EXPLORER clinical trial for advanced SM is ongoing. Blueprint Medicines plans to present data from this trial at the 60thAmerican Society for Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2018.
Avapritinib: Gastrointestinal Stromal Tumors (GIST)
Blueprint Medicines continues to evaluate avapritinib in its Phase 1 NAVIGATOR clinical trial and will present updated data across multiple patient populations, including PDGFRA-driven GIST, third-line or later GIST, and second-line GIST, at the 2018 Connective Tissue Oncology Society (CTOS) Annual Meeting on November 15, 2018. Based on data from this trial, Blueprint Medicines plans to submit a new drug application (NDA) to the FDA for avapritinib for the treatment of patients with PDGFRα Exon 18 mutant GIST and fourth-line GIST in the first half of 2019.
BLU-667: RET-Altered Solid Tumors
Blueprint Medicines recently received written feedback from the FDA supporting expedited development of BLU-667 and plans to submit an NDA for BLU-667 in the first half of 2020 based on additional data from the ongoing Phase 1 ARROW trial. Based on the feedback from the FDA, Blueprint Medicines currently expects the NDA submission will be for separate potential indications: (1) patients with RET-fusion positive NSCLC and papillary thyroid cancer (PTC) who have progressed following prior systemic therapy and (2) patients with RET-mutant medullary thyroid cancer (MTC) who have progressed following treatment with a tyrosine kinase inhibitor.
In October 2018, Blueprint Medicines presented updated data from its ongoing Phase 1 ARROW clinical trial of BLU-667 in patients with MTC and PTC at the 88th Annual Meeting of the American Thyroid Association. The data showed that BLU-667 is highly active and well-tolerated in these patient populations, with increased activity observed at higher dose levels and longer treatment durations. Ninety percent of evaluable patients with MTC and PTC experienced radiographic tumor reductions, regardless of RET alteration or prior multi-kinase inhibitor therapy. The response rate was 62 percent in patients with MTC in the 300 and 400 milligram once daily dose groups who were treated for at least 24 weeks. The data also showed that BLU-667 was well-tolerated, and most adverse events reported by investigators were Grade 1. Read the full data here.
In September 2018, Blueprint Medicines presented two clinical case studies demonstrating proof-of-concept for BLU-667 in combination with Tagrisso (osimertinib) in patients with treatment-resistant, EGFR-mutant NSCLC harboring an acquired RET fusion. The data showed that the combination of BLU-667 and osimertinib overcame resistance to standard therapy, and both patients achieved a partial response with a 78 percent reduction in target tumors per RECIST version 1.1. In these two patients, the combination was well-tolerated, and all reported adverse events were Grade 1 or 2. Read the full data here.
BLU-554: Hepatocellular Carcinoma (HCC)
In September 2018, Blueprint Medicines and its partner, CStone Pharmaceuticals, submitted an investigational new drug (IND) application for BLU-554 to Chinese health authorities. Subject to approval of the IND application, the companies plan to expand Blueprint Medicines’ ongoing Phase 1 clinical trial of BLU-554 as a monotherapy for the treatment of advanced HCC to include clinical sites in Mainland China. Additionally, the companies plan to initiate a proof-of-concept clinical trial evaluating BLU-554 in combination with CS1001, a clinical-stage anti-PD-L1 immunotherapy, in 2019.
Research Programs:
BLU-782: Fibrodysplasia Ossificans Progressiva (FOP)
In September 2018, Blueprint Medicines presented preclinical proof-of-concept data for BLU-782, an investigational precision therapy specifically designed to target the underlying cause of FOP, at the 2018 American Society for Bone and Mineral Research Annual Meeting. The data showed that BLU-782 prevented injury- and surgery-induced heterotrophic ossification, reduced edema and restored healthy tissue response to muscle injury in a well-characterized FOP mouse model. Read the full data here.
Blueprint Medicines expects to submit an IND application to the FDA for BLU-782 by the end of 2018, and subject to review of the IND application, plans to initiate a Phase 1 clinical trial in healthy volunteers in the first quarter of 2019. Upon completion of the Phase 1 clinical trial, Blueprint Medicines plans to advance BLU-782 into a registration-enabling Phase 2 clinical trial in patients with FOP.
Corporate:
In October 2018, Blueprint Medicines announced the expansion of its leadership team with the appointment of Christina Rossi as Chief Commercial Officer. Blueprint Medicines also announced the appointment of Paul Beresford as General Manager, International.
Third Quarter Financial Results:
Cash Position: As of September 30, 2018, cash, cash equivalents and investments were $559.6 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 entering into the collaboration with CStone Pharmaceuticals and the $10.0 million milestone payment achieved under the Roche collaboration in June 2018.
Collaboration Revenues: Collaboration revenues were $1.1 million for the third quarter of 2018, as compared to $8.1 million for the third quarter of 2017. This decrease was primarily due to the termination of the Alexion agreement in 2017.
R&D Expenses: Research and development expenses were $64.6 million for the third quarter of 2018, as compared to $39.3 million for the third quarter of 2017. This increase was primarily attributable to increased clinical and manufacturing expenses associated with advancing avapritinib and BLU-667 further through clinical trials and increased personnel-related expenses. Research and development expenses included $4.8 million in stock-based compensation expenses for the third quarter of 2018.
G&A Expenses: General and administrative expenses were $12.0 million for the third quarter of 2018, as compared to $7.4 million for the third quarter of 2017. This increase was primarily due to increased personnel-related expenses and increased professional fees, including pre-commercial planning activities. General and administrative expenses included $3.6 million in stock-based compensation expenses for the third quarter of 2018.
Net Loss: Net loss was $72.7 million for the third quarter of 2018, or a net loss per share of $1.66, as compared to a net loss of $37.7 million for the third quarter of 2017, or a net loss per share of $0.96.
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.
Earnings Conference Call Information:
Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss third quarter 2018 financial results and recent business activities. The conference call may be accessed by dialing (855) 626-8618 (domestic) or (531) 289-2784 (international) and referring to conference ID 7598866. A webcast of the conference call will be available in the Investors section of the 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.
CTOS Conference Call Information:
Blueprint Medicines will host a live conference call and webcast to discuss data being presented at the 2018 CTOS Annual Meeting on November 15th at 7:30 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 3479587. A webcast of the conference call will be available in the Investors section of the 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.