On May 3, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the first quarter ended March 31, 2022 (Press release, Blueprint Medicines, MAY 3, 2022, View Source [SID1234613385]).

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"The first quarter was marked by a number of important milestones across our business. We have strong momentum in our commercial execution, our pipeline is advancing rapidly, and we continue to build on our scientific leadership," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our ongoing global launch of AYVAKIT/AYVAKYT (avapritinib) in the U.S. and now also in Europe is establishing a new standard of care for the treatment of advanced SM, targeting the underlying cause of the disease. We are on track to have topline data from our registration-enabling PIONEER trial in late summer, further expanding our leadership in SM as we potentially bring the first and only medicine to patients with the non-advanced form of the disease. In addition, we continue to progress our pipeline of innovative investigational medicines in difficult-to-treat and prevalent cancers such as non-small cell lung cancer and breast cancer. At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, we presented five abstracts highlighting data across four programs within our EGFR and CDK2 franchises, while earlier in the quarter we announced a targeted protein degradation discovery collaboration with Proteovant Therapeutics. The strength of this past quarter demonstrates that we are well on our way of delivering on our 2022 goals while also building the foundation to drive further value for the years ahead."

First Quarter 2022 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): advanced systemic mastocytosis (SM) and PDGFRA gastrointestinal stromal tumor (GIST)

Recorded global net product revenues of $23.8 million for the first quarter of 2022.
Received European Commission approval for AYVAKYT for the treatment of adult patients with advanced SM, including aggressive SM, SM with an associated hematological neoplasm, or mast cell leukemia, after at least one systemic therapy, and treated the first commercial patients in Germany. Read the press release here.
GAVRETO (pralsetinib): RET-altered cancers

As previously recorded and reported by Roche, GAVRETO product sales for their region were 5 million CHF, which excludes sales in the Greater China territory driven by CStone Pharmaceuticals.
BLU-945, BLU-701 and BLU-451 (formerly LNG-451): EGFR-driven NSCLC

Reported proof-of-concept data at AACR (Free AACR Whitepaper) from the Phase 1/2 SYMPHONY clinical trial of BLU-945, showing early evidence of safety and clinical activity, with dose-dependent decreases in circulating tumor DNA (EGFR variant allele fractions) and radiographic tumor reductions, including an unconfirmed partial response (PR) in a patient treated with 400 mg once daily (QD). BLU-945 was generally well-tolerated, with no significant adverse events (AEs) associated with wild-type EGFR inhibition. The maximum tolerated dose and recommended Phase 2 dose have not yet been identified, and dose escalation is continuing. These results support plans to expand the development of BLU-945 in combination with multiple agents, including osimertinib, with the goal of preventing or treating tumor resistance to prolong patient benefit. Read the press release here.
Entered into a clinical trial supply agreement with AstraZeneca (LSE/STO/Nasdaq:AZN), under which Blueprint Medicines will evaluate BLU-945 and BLU-701 in combination with osimertinib in the ongoing SYMPHONY and HARMONY trials, respectively.
Also at AACR (Free AACR Whitepaper), reported preclinical data supporting the development of BLU-451 in EGFR exon 20 insertion-positive NSCLC.
Initiated patient dosing in the CONCERTO trial, a Phase 1/2 trial of BLU-451 in patients with EGFR-driven NSCLC harboring exon 20 insertion mutations.
BLU-222: breast, ovarian, and other CDK2-vulnerable cancers, including CCNE1-amplified tumors

Reported preclinical data in a CCNE1-amplified ovarian tumor model at AACR (Free AACR Whitepaper) supporting the development of BLU-222 in CDK2-vulnerable cancers.
Initiated the VELA trial, a Phase 1/2 trial of BLU-222 in CDK2-vulnerable cancers, including estrogen-receptor-positive breast cancer and a range of other CCNE1-amplified tumors, and dosed the first patient in Part 1 dose escalation.
Corporate

Announced strategic collaboration with Proteovant Therapeutics to advance novel targeted protein degrader therapies to address important areas of medical need. Under the terms of the collaboration, the companies will jointly research important targets and advance up to four novel protein degrader therapies into development candidates. Read the press release here.
Recognized a $30 million milestone payment from Clementia related to the initiation of a Phase 2 trial of BLU-782, which is now called IPN60130, our out-licensed ALK2 inhibitor in development for the rare bone disease fibrodysplasia ossificans progressiva.
Key Upcoming Milestones

The company plans to achieve the following near-term milestones:

Report topline data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in late summer 2022 and submit a supplemental new drug application to the U.S. Food and Drug Administration for AYVAKIT in non-advanced SM in the second half of 2022.
Present initial data from the dose escalation cohort of the Phase 1/2 SYMPHONY trial evaluating BLU-945 in combination with osimertinib in the second half of 2022.
Present initial clinical data from the Phase 1/2 HARMONY trial of BLU-701 in the second half of 2022.
Present initial data from Part 1 of the HARBOR trial of BLU-263 in non-advanced SM in the second half of 2022.
Share the company’s research and portfolio vision, including scientific platform expansion plans, at an R&D Day in the second half of 2022.
First Quarter 2022 Results

Revenues: Revenues were $62.7 million for the first quarter of 2022, including $23.8 million of net product revenues from sales of AYVAKIT/AYVAKYT and $38.9 million in collaboration revenues. Blueprint Medicines recorded revenues of $21.6 million in the first quarter of 2021, including $7.1 million of net product revenues from sales of AYVAKIT/AYVAKIT, $1.8 million of net product revenues from sales of GAVRETO and $12.6 million in collaboration revenues.
Cost of Sales: Cost of sales was $5.1 million for the first quarter of 2022, as compared to $0.1 million for the first quarter of 2021.
R&D Expenses: Research and development expenses were $103.1 million for the first quarter of 2022, as compared to $79.7 million for the first quarter of 2021. This increase was primarily due to increased costs associated with the progression of our clinical trials, increased costs related to early discovery efforts, and a decrease in reimbursement from the global development cost sharing arrangement under our collaboration with Roche for GAVRETO. Research and development expenses included $10.0 million in stock-based compensation expenses for the first quarter of 2022.
SG&A Expenses: Selling, general and administrative expenses were $57.1 million for the first quarter of 2022, as compared to $42.0 million for the first 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 $13.4 million in stock-based compensation expenses for the first quarter of 2022.
Net Loss: Net loss was $106.0 million for the first quarter of 2022, or a net loss per share of $1.79, as compared to a net loss of $99.7 million for the first quarter of 2021, or a net loss per share of $1.72.
Cash Position: As of March 31, 2022, cash, cash equivalents and investments were $893.4 million, as compared to $1,034.6 million as of December 31, 2021.
Financial Guidance

Blueprint Medicines continues to anticipate approximately $180 to $200 million in total net revenues in 2022, including approximately $115 to $130 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 to discuss first quarter 2022 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 694684. 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 three upcoming investor conferences:

Jefferies Healthcare Conference on Tuesday, June 9, 2022 in New York, NY.
JMP Securities 2022 Life Sciences Conference on Thursday, June 16, 2022 in New York, NY.
Goldman Sachs 43rd Annual Global Healthcare Conference on Thursday, June 16, 2022 in Rancho Palos Verdes, CA.
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.