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4/29/2021
Ladies and gentlemen, thank you for standing by and welcome to the Blueprint Medicines conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Ms. Kristin Hodes with Blueprint Medicines. Thank you. Please go ahead, ma'am.
Thank you, operator. Good morning, everyone. This is Kristin Hodes of Blueprint Medicines, and welcome to Blueprint Medicines' first quarter 2021 financial and operating results conference call. This morning, we issued a press release which outlines the topics we plan to discuss today. You can access the press release as well as the slides that we'll be reviewing today by going to the investor section of our website at www.blueprintmedicines.com. Today on our call, Jeff Albers, our Chief Executive Officer, will discuss Blueprint Medicine's first quarter 2021 business highlights. Christy Rossi, our Chief Commercial Officer, will provide a commercial update. Becker Hughes, our Chief Medical Officer, will review our clinical progress and highlight upcoming milestones across our growing pipeline. And Mike Lansdell, our Chief Financial Officer, will review our first quarter 2021 financial results. Before we get started, I would like to remind everyone that statements we make on this conference call will include forward-looking statements. Actual events or results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors, including those set forth in the risk factor section of our SEC filings. In addition, any forward-looking statement made on this call represents our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements. Now, here's our CEO, Jeff Albers.
Thanks, Kristen, and good morning, everyone. 2021 is off to a productive start, and I'm pleased to provide an update on recent progress across our growing portfolio. As we outlined on our year-end call, our 2021 efforts are centered around three strategic pillars. First, accelerating the global adoption of Avakit and Gavaretto. Second, advancing a next wave of therapeutic candidates to clinical proof of concept. And third, further expanding our precision therapy research pipelines. Just a few months into the year, we've already made significant progress against each of these goals. As Christy will discuss in a moment, we continue to advance the commercial launches of Avakit and Gavaretto. In parallel, we're preparing for the anticipated approval of Avakit in advanced systemic mastocytosis. As we near our Purdue for date in the U.S., and with a type 2 variation MAA under review with the EMA, we're moving forward with urgency to provide a therapeutic option for as many patients as possible fighting this disease. At the ACR annual meeting earlier this month, we presented registrational data from our Phase II Pathfinder trial in advanced SM, which demonstrated a confirmed overall response rate of 75%, with responses occurring rapidly and continuing to deepen over time. In addition, new patient-reported data showed that treatment with Avakit significantly reduced disease symptoms and improved quality of life. We believe these results reinforce the potential of Avakit to fundamentally change the outlook for patients with advanced systemic mastocytosis. We also shared new data at AACR from several investigational medicines in our portfolio that comprise our next wave of therapeutic candidates. including BLU-263, our next-generation KIT-D816V inhibitor, BLU-701 and BLU-945, our two EGFR development candidates, and our newest programs, BLU-222, targeting CDK2, and BLU-852, targeting MAP4K1. Collectively, these programs have the potential to dramatically expand our ability to reach patients on a global basis. In fact, each of these markets targeted by these new programs is forecast to more than double in size over the next five years, underscoring the opportunity for us to bring innovation to areas of significant medical need. Since our founding 10 years ago, we've worked hard to integrate our discovery, development, and commercialization efforts in order to maximize innovation and efficiency. We've already demonstrated the productivity of this approach, having successfully discovered and developed two approved medicines. In the past year and a half alone, we've expanded our pipeline with five additional development candidates, all of which we intend to advance with the same sense of urgency. To that end, and as Becker will describe in a few minutes, we remain on track to initiate multiple clinical trials in the months ahead, positioning us to generate several significant proof-of-concept data sets in the near future. Importantly, we continue to operate from a position of tremendous financial strength with the resources to invest in our commercial efforts, our pipeline programs, and our research engine. as we aim to make real the promise of precision therapy for as many people with cancer and hematologic disorders as possible. With that, I'll now turn the call over to Christy to discuss our commercial efforts. Christy?
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