Blueprint Medicines(BPMC) - 2019 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended Q2 2019 with $667.3 million in cash, cash equivalents, and investments, which is expected to fund operations into mid-2021 [23] - Operating expenses increased in Q2 due to manufacturing and supply activities, driven by accelerated clinical development and filing timelines for avapritinib and pralsetinib [24] Business Line Data and Key Metrics Changes - The company submitted two marketing applications for avapritinib to the FDA and EMA for adult patients with PDGFRalpha GIST, marking significant progress in their regulatory strategy [7][8] - The company plans to hold its first Research and Development Day on November 5, focusing on indication expansion opportunities and new targets [9] Market Data and Key Metrics Changes - The company aims to increase tumor mutation testing rates among GIST patients, currently at 30% to 40% in the U.S., to improve access to precision medicine [14][15] - The commercial strategy includes educating healthcare providers and patients about avapritinib, targeting a shift in treatment paradigms for GIST [16][17] Company Strategy and Development Direction - The company is focused on evolving the treatment paradigm for GIST and expanding its commercial infrastructure to support multiple product launches [13][19] - The collaboration with CStone Pharmaceuticals in China is progressing well, with multiple clinical trial protocols approved and patient enrollment underway [21][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming regulatory submissions and the potential for avapritinib to transform treatment for GIST patients [6][10] - The company is preparing for significant growth opportunities across its portfolio, with a focus on patient access and education [11][19] Other Important Information - The company plans to provide an update on the avapritinib program in systemic mastocytosis in Q4 2019, with promising clinical data reported [10][11] - The PIONEER trial for indolent systemic mastocytosis is designed to evaluate lower dose levels, with initial data expected by the end of the year [26][36] Q&A Session Summary Question: Can you discuss the side effects profile of the drug in the context of the SM indication particularly the indolent population? - Management indicated that early safety data from the PIONEER study showed low side effects at lower dose levels, which are expected to be well tolerated for patients with indolent systemic mastocytosis [26] Question: Any color around expected patient demographic distribution for VOYAGER now that you're enrolling Chinese patients? - Management noted that the VOYAGER study is open internationally and expects no regulatory impact from the inclusion of Chinese patients [29] Question: How much longer-term safety and efficacy follow-up will you need to demonstrate for chronic conditions? - Management stated that ongoing discussions with regulatory authorities will determine the specifics of safety follow-up for the PIONEER study [30] Question: How much synergy could you expect to capture between indolent and advanced SM? - Management anticipates leveraging learnings from advanced SM to inform strategies for indolent SM, emphasizing the need for precision therapy approaches [31][34] Question: Can you give us an update on the PIONEER trial's dose-ranging portion? - Management confirmed that the PIONEER trial is ongoing, with preliminary information expected by the end of the year [36] Question: What is the expected safety and efficacy if you exclude certain mutations in the GIST program? - Management explained that specific mutations would be excluded from the COMPASS study to ensure appropriate patient selection for avapritinib [49] Question: Will you be including any data on the PRO endpoint in the initial update for PIONEER? - Management indicated that PRO data will not be available in the initial update but is a critical aspect of the study [54]