Alnylam Pharmaceuticals(ALNY) - 2021 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Alnylam achieved $662 million in net product revenue for 2021, representing year-over-year growth of 83% [6][30] - Non-GAAP R&D expenses increased by 34% in Q4 2021 compared to Q4 2020, primarily due to investments in mid and late-stage pipeline programs [31] - Non-GAAP operating loss for 2021 was $528 million, showing a $121 million improvement compared to 2020 [32] - Cash, cash equivalents, and marketable securities at the end of 2021 were $2.4 billion, up from $1.9 billion at the end of 2020 [33] Business Line Data and Key Metrics Changes - ONPATTRO generated $139 million in global net product revenues in Q4 2021, a 15% increase quarter-on-quarter and 53% year-over-year [10] - GIVLAARI achieved $41 million in global net product revenues in Q4 2021, representing 28% quarter-on-quarter growth and 84% growth versus Q4 2020 [13] - OXLUMO reported $19 million in global net product revenues in Q4 2021, a 29% increase compared to Q3 2021 [14] Market Data and Key Metrics Changes - In the U.S., ONPATTRO sales increased by 17% versus Q3 2021, driven by patient demand and inventory stocking [11] - GIVLAARI's U.S. sales increased by 35% compared to Q3 2021, influenced by patient demand and inventory stocking [13] - OXLUMO's international markets saw a 40% growth in Q4 2021 compared to Q3 2021, benefiting from increased patient treatment and geographic expansion [15] Company Strategy and Development Direction - Alnylam aims to expand its TTR franchise and become a global leader in delivering impactful medicines [8] - The company plans to expand beyond rare diseases into prevalent diseases, leveraging its sustainable innovation engine [8][29] - Upcoming milestones include the potential approval of vutrisiran and continued advancement of the ATTR franchise [36] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of the TTR franchise, particularly with the upcoming launch of vutrisiran [46] - The company anticipates combined net product revenues for 2022 to be between $900 million and $1 billion, reflecting a 44% growth compared to 2021 [34] - Management acknowledged the ongoing pandemic's impact on operations but remains optimistic about patient demand and product growth [34] Other Important Information - Alnylam's pipeline includes late-stage candidates for ATTR amyloidosis and hypertension, with significant progress in clinical trials [17][26] - The company is also exploring new opportunities in prevalent diseases, such as hypertension, with its investigational product zilebesiran [26] Q&A Session Summary Question: Thoughts on vutrisiran approval and potential label inclusion - Management is excited about vutrisiran's launch and believes it will enhance the TTR franchise without cannibalizing existing products [40][41] Question: Guidance assumptions for ONPATTRO and vutrisiran - The TTR franchise is expected to drive growth, with management confident in the appeal of vutrisiran's profile to patients [46][47] Question: KARDIA-1 Phase 2 study expectations - Management is optimistic about achieving clinically meaningful results in KARDIA-1, with a focus on blood pressure control and patient adherence [50][51] Question: Confidence in APOLLO-B trial design - Management expressed confidence in the study design and execution, citing a strong track record in the TTR space [59][60] Question: Comparison of ramp speed between patisiran and vutrisiran - Management believes vutrisiran has the potential for a faster ramp speed due to its compelling profile and reduced treatment burden [66][67]