On July 29, 2021 Blueprint Medicines Corporation (NASDAQ:BPMC) reported financial results and provided a business update for the second quarter ended June 30, 2021 (Press release, Blueprint Medicines, JUL 29, 2021, View Source [SID1234585415]).

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"In the second quarter, we achieved one of our foundational corporate goals, securing U.S. approval of AYVAKIT for advanced systemic mastocytosis, and the launch of this therapy is off to a great start with broad prescriber demand," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, we continued to progress our expanding clinical portfolio with the initiation of the Phase 1/2 trial of BLU-945, the initiation of the Phase 2/3 HARBOR trial of BLU-263 and the announcement of a strategic research collaboration with MD Anderson to accelerate the development of BLU-222, all of which exemplify the potential impact of our next wave of therapeutic candidates. This commercial and clinical progress, combined with our financial strength, provides a robust foundation for future growth as we look to address the needs of many more patients with cancer and hematologic disorders."

Second Quarter 2021 Highlights and Recent Progress

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

Recorded $8.5 million in net product revenue during the second quarter of 2021 for AYVAKIT/AYVAKYT, which was approved by the U.S. Food and Drug Administration (FDA) in June 2021 for the treatment of adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL). Read the press release announcing the expansion of the FDA label for AYVAKIT into advanced SM here. AYVAKIT received its initial approval from the FDA in 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Please click here to see the full Prescribing Information for AYVAKIT and visit www.AYVAKIT.com for more information on AYVAKIT.
GAVRETO (pralsetinib): RET-altered cancers

Recorded $2.9 million in net product revenue during the second quarter of 2021 for GAVRETO, which was approved by the FDA in September 2020 for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test and in December 2020 for the treatment of patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Blueprint Medicines is commercializing GAVRETO in the U.S. together with Genentech, Inc., a member of the Roche Group. Please click here to see the full Prescribing Information for GAVRETO and visit www.GAVRETO.com for more information on GAVRETO.
Reported updated data from the Phase 1/2 ARROW clinical trial of GAVRETO in metastatic RET fusion-positive NSCLC and other advanced solid tumors at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data showed high response rates to GAVRETO in treatment-naïve patients with RET fusion-positive NSCLC, clinical activity across a number of RET fusion-positive tumor types and a safety profile consistent with previously reported results. Read the press release here.
BLU-263: SM

Initiated the HARBOR trial, a randomized, double-blind, placebo-controlled Phase 2/3 trial in non-advanced SM.
BLU-945: treatment-resistant EGFR-driven NSCLC

Initiated the global Phase 1/2 trial of BLU-945 in patients with treatment-resistant EGFR-driven NSCLC.
BLU-222: Cyclin E-aberrant cancers

Announced a strategic research collaboration with The University of Texas MD Anderson Cancer Center focused on accelerating development of BLU-222, an investigational precision therapy designed to target cyclin-dependent kinase 2 (CDK2). Read the press release here.
Corporate:

Announced the appointment of Percy Carter, MBA, Ph.D., as Chief Scientific Officer. In this role, Dr. Carter oversees all research and preclinical development. Read the press release here.
Key Upcoming Milestones

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

Present preclinical data supporting combination of BLU-945 and BLU-701 in treatment-naïve EGFR-driven NSCLC in the second half of 2021.
Initiate a Phase 1 trial of BLU-701 in patients with treatment-resistant EGFR-driven NSCLC in the second half of 2021.
Initiate a Phase 1 trial of BLU-222, a CDK2 inhibitor targeting cyclin-E aberrant cancers, in the first half of 2022.
Disclose topline data for the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced systemic mastocytosis in mid-2022.
Second Quarter 2021 Financial Results

Revenues: Revenues were $27.3 million for the second quarter of 2021, including $8.5 million of net product revenues from sales of AYVAKIT/AYVAKYT, $2.9 million of net product sales from GAVRETO and $15.9 million in collaboration revenues. Blueprint recorded revenues of $8.3 million in the second quarter of 2020, including $5.7 million of net product revenues from sales of AYVAKIT and $2.7 million in collaboration revenues. The increase in net product revenues was driven by increased sales quantity and the increase in collaboration revenues was primarily driven by the sales of drug substance and drug product to our collaboration partners.
Cost of Sales: Cost of sales was $6.5 million for the second quarter of 2021, as compared to $0.1 million for the second quarter of 2020. Cost of sales includes manufacturing costs associated with our products sales as well as costs associated with the sale of drug product to our collaboration partners. The increase in costs of product sales was primarily driven by the lower margin product sales to our collaboration partners during the second quarter of 2021.
R&D Expenses: Research and development expenses were $80.0 million for the second quarter of 2021, as compared to $91.1 million for the second quarter of 2020. This decrease was primarily due to decreased expenses associated with clinical supply manufacturing activities and reimbursement from the global development cost sharing arrangement under our collaboration with Roche for pralsetinib, partially offset by increased costs related to early discovery activities. Research and development expenses included $10.5 million in stock-based compensation expenses for the second quarter of 2021.
SG&A Expenses: Selling, general and administrative expenses were $49.3 million for the second quarter of 2021, as compared to $42.2 million for the second quarter of 2020. This increase was primarily due to increased costs associated with building our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT and GAVRETO, partially offset by reimbursement under our collaboration with Roche for pralsetinib. General and administrative expenses included $13.8 million in stock-based compensation expenses for the second quarter of 2021.
Net Loss: Net loss was $108.4 million for the second quarter of 2021, or a net loss per share of $1.86, as compared to a net loss of $123.5 million for the second quarter of 2020, or a net loss per share of $2.28.
Cash Position: As of June 30, 2021, cash, cash equivalents and investments were $1,380.1 million, as compared to $1,549.7 million as of December 31, 2020.
Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss second quarter 2021 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or +44-208-0682-558 (international) and referring to conference ID 619159. A webcast of the call will 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 Conference

Blueprint Medicines will participate in the 2021 Wedbush Pacgrow Healthcare Virtual Conference on Tuesday, August 10, 2021. Christina Rossi, Chief Commercial Officer, will participate in a panel discussion, "Building Back a Better Commercial Infrastructure – Selling in COVID Times," beginning at 8:35 a.m. ET. A live webcast of the panel discussion will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.