On May 4, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the first quarter ended March 31, 2023 (Press release, Blueprint Medicines, MAY 4, 2023, View Source [SID1234631016]).
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"Our first quarter was marked by executional progress across the multiple growth opportunities that Blueprint Medicines has to expand our impact and transform outcomes for thousands of patients worldwide. We drove strong AYVAKIT revenue growth, presented seminal data for AYVAKIT in indolent systemic mastocytosis, and worked to progress our broad pipeline by rapidly resolving the partial clinical hold for BLU-222 and generating early clinical data across our portfolio," said Kate Haviland, Chief Executive Officer at Blueprint Medicines. "Blueprint Medicines is ready to further solidify our leadership in systemic mastocytosis and scale the impact of AYVAKIT by addressing the medical needs of a substantially larger group of patients with our anticipated FDA label expansion for indolent systemic mastocytosis in a few weeks."
First Quarter 2023 Highlights and Recent Progress
Systemic mastocytosis (SM)
· Presented full registrational PIONEER trial data for AYVAKIT in patients with indolent systemic mastocytosis (ISM) at the 2023 American Academy of Asthma, Allergy, and Immunology (AAAAI) Annual Conference. These data included a statistically significant and clinically meaningful improvement in total symptom score that deepened over time, with improvements shown across all individual symptoms and in quality-of-life measures. AYVAKIT showed a favorable safety profile compared to placebo, supporting potential for chronic treatment. Read the press release here.
· U.S. Food and Drug Administration (FDA) target action date for AYVAKIT supplemental new drug application for ISM is May 22, 2023.
EGFR-driven non-small cell lung cancer (NSCLC)
· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 CONCERTO trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC, showing early safety and clinical activity, at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
· Announced plans to present updated results from the dose escalation of the Phase 1/2 SYMPHONY trial showing safety and tolerability of BLU-945 both as a monotherapy and in combination with osimertinib in late-line EGFR-driven NSCLC at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.
· Presented real-world data showing that NSCLC patients with an EGFR L858R mutation have worse outcomes compared to patients with an EGFR exon 19 deletion mutation when treated with first-line osimertinib, and new preclinical data showing the combination of BLU-945 and osimertinib enhanced tumor regression and survival compared to osimertinib alone in a treatment-naïve EGFR L858R-mutant cell-derived model at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Read the poster presentation here.
· Today announced acceptance of an investigational new drug (IND) application to the FDA for BLU-525.
CDK2-vulnerable breast and other cancers
· Announced the FDA lifted the partial clinical hold on patient enrollment in the VELA trial of BLU-222. Patients already enrolled in the trial have continued on the study and trial sites are working to resume patient enrollment.
· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 VELA trial of BLU-222 in breast cancer and other cancers vulnerable to CDK2 inhibition, showing evidence of monotherapy safety and pathway modulation, at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.
Key Upcoming Milestones
The company plans to achieve the following milestones by mid-2023:
· Obtain FDA approval and initiate launch of AYVAKIT in ISM in mid-2023.
· Present initial CONCERTO trial dose escalation data in EGFR exon 20 NSCLC at ASCO (Free ASCO Whitepaper) 2023.
· Present initial VELA trial dose escalation data at ASCO (Free ASCO Whitepaper) 2023.
· Nominate a development candidate targeting wild-type KIT for chronic urticaria by mid-2023.
First Quarter 2023 Results
· Revenues: Revenues were $63.3 million for the first quarter of 2023, including $39.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $24.2 million in collaboration revenues. Blueprint Medicines recorded revenues of $62.7 million in the first quarter of 2022, including $23.8 million of net product revenues from sales of AYVAKIT/AYVAKIT and $38.9 million in collaboration revenues.
· Cost of Sales: Cost of sales was $3.2 million for the first quarter of 2023, as compared to $5.1 million for the first quarter of 2022.
· R&D Expenses: Research and development expenses were $112.1 million for the first quarter of 2023, as compared to $103.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs related to early discovery efforts. Research and development expenses included $10.1 million in stock-based compensation expenses for the first quarter of 2023.
· SG&A Expenses: Selling, general and administrative expenses were $71.0 million for the first quarter of 2023, as compared to $57.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $13.1 million in stock-based compensation expenses for the first quarter of 2023.
· Net Loss: Net loss was $129.6 million for the first quarter of 2023, or a net loss per share of $2.15, as compared to a net loss of $106.0 million for the first quarter of 2022, or a net loss per share of $1.79.
· Cash Position: As of March 31, 2023, cash, cash equivalents and investments were $961.3 million, as compared to $1,078.5 million as of December 31, 2022.
2023 Financial Guidance
Blueprint Medicines has updated its financial guidance and now anticipates approximately $135 million to $145 million in AYVAKIT net product revenues for advanced SM and GIST in 2023, and $40 million to $50 million in collaboration revenues from existing collaborations in 2023. This guidance excludes revenue from the anticipated AYVAKIT indication expansion in ISM in mid-2023. 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 8:00 a.m. ET today to discuss first quarter 2023 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 929-526-1599 (international), and referring to conference ID 668091. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media 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.
Upcoming Investor Conferences
Blueprint Medicines will participate in one upcoming investor conference:
· Goldman Sachs 44th Annual Global Healthcare Conference on Wednesday, June 14, 2023 at 12:20 pm ET.
A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.