On November 1, 2022 Blueprint Medicines (NASDAQ: BPMC) reported its 2027 Blueprint to achieve precision medicine at scale, a five-year business strategy to expand the company’s reach to broader patient populations by leveraging its scientific leadership, proven development capability and integrated business (Press release, Blueprint Medicines, NOV 1, 2022, View Source [SID1234622722]). The company plans to highlight this business strategy, including commercial plans to bring AYVAKIT to patients with non-advanced systemic mastocytosis (SM), at its Investor Day event today. In addition, Blueprint Medicines today reported financial results and provided a business update for the third quarter ended September 30, 2022.

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"The opportunity to bring AYVAKIT to patients with non-advanced SM, based on the positive results of the PIONEER study, will enable us to scale our impact and address the needs of a significantly larger patient population in the near term," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "This morning at our Investor Day, we will highlight our 2027 Blueprint strategy to double our impact in five years across multiple metrics of portfolio strength. We plan to achieve this scale with the potential launch of AYVAKIT in non-advanced SM, multiple advancing clinical development programs for EGFR-mutant lung cancer and CDK2-vulnerable breast cancer, and an expansive precision therapy research pipeline, all of which build on our R&D achievements to date and leverage our fully integrated global infrastructure."

"In addition, today we are reaffirming total revenue guidance for full-year 2022, while we lower product revenue guidance based on performance in the third quarter and our near-term expectations for growth. Over the last year, we have established AYVAKIT as the standard of care in patients who are being actively treated for their advanced SM, and we are now focusing on increasing treatment rates in patients with SM and an associated hematologic neoplasm, where there has been lower adoption. In parallel, we are prioritizing efforts to bring AYVAKIT to patients with non-advanced SM, which, if approved, would represent a 15-fold or larger opportunity based on the number of patients with moderate-to-severe non-advanced SM who are diagnosed, being treated for their SM, and observable in U.S. claims data today."

2027 Blueprint Global Business Strategy

Building on Blueprint Medicines’ significant achievements over the last decade, including the regulatory approval of two internally discovered precision therapies within the company’s first decade, the five-year 2027 Blueprint strategy aims to double the company’s impact across multiple measures of portfolio strength in about half the time.

Blueprint Medicines aims to achieve the following by the end of 2027:

Products: 4+ marketed products for oncology, hematology, or mast cell disorders (versus 2 today)
Portfolio: 3+ disease leadership areas (versus 1, mast cell disease, today)
Clinical: 4+ late-stage clinical programs (versus 2 today)
Research: 25+ cumulative development candidates nominated (versus 14 today) derived from 2 research platforms (versus 1, kinase inhibitor platform, today)
Investor Day Presentation Highlights

At the Investor Day event, Blueprint Medicines plans to:

Review the go-to-market plan for AYVAKIT in non-advanced SM and facilitate a panel discussion with disease experts on perceptions of the registration-enabling PIONEER trial data, the current state of SM care, and the potential role of a new disease-modifying therapy. Participants will include:
Frank Siebenhaar, MD, Assistant Professor, Charité University, and PIONEER trial investigator
Pankit Vachhani, MD, Assistant Professor, University of Alabama, and PIONEER trial investigator
James Wedner, MD, Professor, Washington University in St. Louis
Highlight clinical progress informing development strategies for the company’s EGFR portfolio therapies, including updated Phase 1/2 SYMPHONY trial dose escalation data supporting plans to prioritize development of BLU-945 in combination with osimertinib in first-line EGFR L858R-positive non-small cell lung cancer (NSCLC).
Introduce a new research program targeting wild-type KIT, which aims to build on the company’s KIT target leadership to advance a best-in-class oral precision therapy for common mast cell diseases adjacent to systemic mastocytosis, including chronic urticaria.
Third Quarter 2022 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): SM and PDGFRA gastrointestinal stromal tumor

Reported global net product revenues of $28.6 million for the third quarter of 2022.
Announced positive top-line results from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in patients with non-advanced SM, demonstrating clinically meaningful and highly significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden. AYVAKIT had a favorable safety profile compared to the control arm, supporting the potential for long-term treatment. Read the press release here.
Published results from the TouchStone study of patient and healthcare provider perceptions of SM disease burden, which highlighted that SM is associated with severe and burdensome symptoms including anaphylactic events, frequent emergency department visits, use of multiple symptom-directed medications, reduced ability to work, impaired physical functioning and poor quality of life. Read the press release here.
GAVRETO (pralsetinib): RET-altered cancers

As previously recorded and reported by Roche, GAVRETO global product sales were 20 million CHF year to date which excludes sales in the Greater China territory driven by CStone Pharmaceuticals.
BLU-945, BLU-701, BLU-525, and BLU-451: EGFR-mutant NSCLC

Based on emerging clinical and preclinical data, Blueprint Medicines plans to prioritize development of BLU-525, a back-up EGFR inhibitor candidate, and deprioritize further development of BLU-701. Compared to BLU-701, BLU-525 has a distinct chemical structure with improved kinome selectivity and differentiated metabolism, and equivalent EGFR mutation coverage, wild-type EGFR selectivity, and central nervous system penetration. The company plans to submit an investigational new drug application to the U.S. Food and Drug Administration (FDA) for BLU-525 in the first quarter of 2023.
Blueprint Medicines, with Guardant Health, presented real-world data from patients with EGFR-mutant NSCLC identifying the EGFR C797X mutation as the most common resistance mechanism to osimertinib at the International Association for the Study of Lung Cancer 2022 World Conference on Lung Cancer. Read the press release here.
Key Upcoming Milestones

Blueprint Medicines expects to achieve the following near-term milestones:

Submit a supplemental New Drug Application to the FDA for AYVAKIT for non-advanced SM in the fourth quarter of 2022.
Present detailed data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM at a medical congress in late 2022 or early 2023.
Report top-line 12-week data from Part 1 of the HARBOR trial of BLU-263 in non-advanced SM in the fourth quarter of 2022.
Submit an IND to the FDA for BLU-525 for the treatment of EGFR-mutant NSCLC in the first quarter of 2023.
Present initial data from the Phase 1/2 CONCERTO study of BLU-451 in patients with EGFR-mutant NSCLC in the first half of 2023.
Present initial data from the Phase 1/2 VELA trial of BLU-222 in CDK2-vulnerable cancers in the first half of 2023.
Third Quarter 2022 Results

Revenues: Revenues were $66.0 million for the third quarter of 2022, including $28.6 million of net product revenues from sales of AYVAKIT/AYVAKYT, $9.8 million in collaboration and license revenues, and $27.5 million in license revenues- related party. Blueprint Medicines recorded revenues of $24.2 million in the third quarter of 2021, including $17.3 million of net product revenues from sales of AYVAKIT/AYVAKIT and $6.9 million in collaboration and license revenues.
Cost of Sales: Cost of sales was $3.0 million for the third quarter of 2022, as compared to $3.8 million for the third quarter of 2021.
R&D Expenses: Research and development expenses were $128.0 million for the third quarter of 2022, as compared to $84.4 million for the third quarter of 2021. This increase was primarily due to increased clinical supply manufacturing and clinical development activities due to the progression and expansion of our clinical trials and increased costs related to early discovery effort. Research and development expenses included $10.0 million in stock-based compensation expenses for the third quarter of 2022.
SG&A Expenses: Selling, general and administrative expenses were $57.6 million for the third quarter of 2022, as compared to $49.8 million for the third quarter of 2021. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. General and administrative expenses included $14.1 million in stock-based compensation expenses for the third quarter of 2022.
Net Loss: Net loss was $133.2 million for the third quarter of 2022, or a net loss per share of $2.23, as compared to a net loss of $117.2 million for the third quarter of 2021, or a net loss per share of $2.00.
Cash Position: As of September 30, 2022, cash, cash equivalents and investments were $1,192.6 million, as compared to $1,034.6 million as of December 31, 2021.
Financial Guidance

Blueprint Medicines anticipates it will achieve the high end of previously provided revenue guidance for full-year 2022 of approximately $180 million to $200 million, including approximately $108 million to $111 million in AYVAKIT net product revenues. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today for Investor Day and to discuss third quarter 2022 financial results. The live webcast of the event will be available under "Events and Presentations" in the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 90 days following the event.