On November 5, 2019 Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported its first R&D Day in New York City (Press release, Blueprint Medicines, NOV 5, 2019, View Source [SID1234550352]).

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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

Highlight the significant medical need in indolent systemic mastocytosis (SM), a rare disease characterized by debilitating and unpredictable symptoms despite best available therapy. Based on an improved understanding of the disease, Blueprint Medicines now estimates there are approximately 75,000 patients with SM in the major markets, which consist of the United States, France, Germany, Italy, Spain, United Kingdom and Japan.
Announce a comprehensive strategy to address a broad population of patients with SM and other mast cell disorders with the company’s drug candidates avapritinib and BLU-263, a next-generation KIT inhibitor. Blueprint Medicines plans to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for BLU-263 for indolent SM in the first half of 2020.
Introduce two research programs targeting well-characterized resistance mutations in patients with EGFR-driven non-small cell lung cancer (NSCLC), highlighting Blueprint Medicines’ differentiated capability for designing highly selective investigational medicines that address tumor evolution and resistance to targeted therapy.
Highlight a research program under Blueprint Medicines’ cancer immunotherapy collaboration with Roche targeting MAP4K1, which is believed to play a role in T cell regulation.
Third Quarter 2019 Highlights and Recent Progress

Avapritinib: Gastrointestinal stromal tumors (GIST)

Completed target enrollment in the Phase 3 VOYAGER trial of avapritinib versus regorafenib in patients with third- and fourth-line GIST.
Announced the FDA intends to administratively split the new drug application (NDA) for avapritinib into two separate NDAs (one for PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and one for fourth-line GIST) and requested top-line data from the ongoing Phase 3 VOYAGER trial to inform its review of the proposed fourth-line GIST indication. The PDUFA action date for both indications is currently February 14, 2020. For the fourth-line indication, an extension of up to three months for the PDUFA action date will likely be required to enable Blueprint Medicines to provide the top-line VOYAGER data to the FDA.
Avapritinib: Systemic mastocytosis (SM)

Completed enrollment of Part 1 of the Phase 2 PIONEER trial of avapritinib in patients with indolent SM.
BLU-782: Fibrodysplasia ossificans progressiva (FOP)

Entered into an exclusive, worldwide license agreement with Clementia Pharmaceuticals, a subsidiary of Ipsen, for the development and commercialization of BLU-782 as a potential treatment for patients with FOP and other indications.
Key Upcoming Milestones

The company expects to achieve the following milestones in the fourth quarter of 2019:

Present initial data from Part 1 of the Phase 2 PIONEER trial of avapritinib in indolent SM at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
Initiate a Phase 3 trial evaluating pralsetinib in first-line RET-fusion NSCLC.
Initiate a Phase 1b/2 trial in China evaluating fisogatinib in combination with CS1001, CStone Pharmaceuticals’ anti-PD-L1 inhibitor, in patients with HCC.
The company expects to achieve the following milestones related to planned marketing applications in 2020:

Submit an NDA to the FDA for avapritinib for the treatment of advanced SM based on data from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial in the first quarter of 2020.
Submit an NDA to the FDA for pralsetinib for the treatment of patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020.
Submit an NDA to the FDA for pralsetinib for the treatment of patients with MTC previously treated with an approved multi-kinase inhibitor in the first half of 2020.
Submit a supplemental NDA to the FDA for avapritinib for the treatment of third‐line GIST in the second half of 2020.
Third Quarter 2019 Financial Results

Cash Position: As of September 30, 2019, cash, cash equivalents and investments were $594.5 million, as compared to $494.0 million as of December 31, 2018. This increase reflects net proceeds of approximately $327.4 million from the company’s follow-on underwritten public offering of common stock, which closed in April 2019, partially offset by cash used in operations. Cash, cash equivalents and investments as of September 30, 2019 do not include the $25.0 million upfront payment received in connection with entering into the worldwide license agreement with Clementia Pharmaceuticals or an $8.0 million research milestone achieved under the Roche collaboration, both of which were earned in October 2019.
Collaboration Revenues: Collaboration revenues were $9.1 million for the third quarter of 2019, as compared to $1.1 million for the third quarter of 2018. This increase was primarily due to revenue recognized under the CStone and Roche collaborations. During the third quarter of 2019, the company recognized $6.0 million in milestone revenue under the CStone collaboration compared to no revenue recognized for the same period in 2018. During the third quarter of 2019, the company recognized $3.1 million in revenue under the Roche collaboration compared to $1.1 million for the same period in 2018.
R&D Expenses: Research and development expenses were $81.5 million for the third quarter of 2019, as compared to $64.6 million for the third quarter of 2018. This increase was primarily due to increased clinical and manufacturing expenses driven by the company’s lead programs and increased personnel expenses. Research and development expenses included $7.7 million in stock-based compensation expenses for the third quarter of 2019.
G&A Expenses: General and administrative expenses were $25.6 million for the third quarter of 2019, as compared to $12.0 million for the third quarter of 2018. This increase was primarily due to increased personnel expenses and increased professional fees for commercial-readiness and other activities. General and administrative expenses included $7.3 million in stock-based compensation expenses for the third quarter of 2019.
Net Loss: Net loss was $94.3 million for the third quarter of 2019, or a net loss per share of $1.93, as compared to a net loss of $72.7 million for the third quarter of 2018, or a net loss per share of $1.66.
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 View Source 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.