Alnylam Pharmaceuticals(ALNY) - 2021 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Alnylam Pharmaceuticals reported $135.8 million in total combined product sales for Q1 2021, representing an 89% increase compared to Q1 2020 [31] - ONPATTRO generated $102 million in net revenue, marking a 13% growth from Q4 2020 and a 53% increase from Q1 2020 [29][30] - GIVLAARI achieved $24.7 million in net revenue, reflecting an 11% growth compared to Q4 2020 [31] - OXLUMO generated $9.1 million in net revenue in its first full quarter [31] - Gross margin on total revenues was 83% for the quarter, down from 87% in Q1 2020 [32] Business Line Data and Key Metrics Changes - ONPATTRO's patient count exceeded 1,500, with adherence to treatment stable at pre-COVID levels [10][11] - GIVLAARI had 225 patients on commercial therapy as of March 31, 2021, with strong patient compliance over 90% [12][13] - OXLUMO saw initial demand with approximately 50 patients on treatment in the US and EU [15] Market Data and Key Metrics Changes - ONPATTRO achieved market access in over 30 countries, including regulatory approval in Taiwan [11] - GIVLAARI's geographic expansion is expected to drive growth, particularly in Europe [14] - OXLUMO's market access efforts are progressing well, with over two-thirds of covered US lives confirmed for access [15] Company Strategy and Development Direction - Alnylam announced a five-year strategic plan, Alnylam P5x25, aiming for transformative medicines in both rare and common diseases, with a revenue CAGR of over 40% through 2025 [8] - The company is focused on expanding its pipeline with first and/or best-in-class product candidates and achieving sustainable non-GAAP profitability within the period [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to 2021, highlighting the positive uptake of ONPATTRO and the progress in clinical trials for vutrisiran [7][22] - The company is excited about the potential of its pipeline, particularly in addressing unmet needs in common diseases [25][26] Other Important Information - Alnylam released its first corporate responsibility summary, emphasizing its commitment to using its business as a force for good [9] - The company is advancing multiple late-stage programs, including patisiran and vutrisiran for ATTR amyloidosis, and plans to initiate a biannual dosing regimen for vutrisiran [22][24] Q&A Session Summary Question: Enrollment rate for HELIOS-B - Management attributed the faster enrollment to community enthusiasm for vutrisiran and the global reach in the TTR space [40][41] Question: Breakdown of ONPATTRO patients on concurrent use with stabilizers - Approximately 20% of patients are using ONPATTRO concurrently with stabilizers for cardiomyopathy treatment [42][44] Question: Growth trends for GIVLAARI - Management highlighted geographic expansion and new patient finding as key growth drivers for GIVLAARI [49] Question: Expectations for APOLLO-B trial - The primary endpoint is the six-minute walk distance, which is clinically meaningful for patients with cardiomyopathy [57][58] Question: Interim analysis for APOLLO-B - The interim analysis aims to potentially bring vutrisiran to market ahead of the final results, with the design still needing alignment with regulators [64][66] Question: Value-based payment implementation - The company reported modest deductions associated with value-based agreements, indicating that the products are performing well in the patient community [72][73]