Alnylam Pharmaceuticals(ALNY) - 2024 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - In Q1 2024, Alnylam Pharmaceuticals reported total product revenues of $365 million, reflecting a 32% year-over-year growth compared to Q1 2023 [7][26] - The gross margin on product sales was 85%, consistent with Q1 2023, but expected to decrease for the remainder of 2024 due to higher royalties on AMVUTTRA [27][28] - Non-GAAP R&D expenses increased by 13% year-over-year, driven by investments in zilebesiran and the HELIOS-B trial [27] - Non-GAAP SG&A expenses rose by 15% year-over-year, indicating operating leverage as product sales grew by 32% [27] Business Line Data and Key Metrics Changes - The TTR franchise achieved$ 264 million in global net product revenues, representing a 29% growth year-over-year [11][12] - The rare franchise delivered $101 million in combined global net product revenue, marking a 40% increase compared to Q1 2023 [16][17] - AMVUTTRA's U.S. sales increased by 35% year-over-year, driven by strong patient uptake [11][12] Market Data and Key Metrics Changes - The U.S. TTR sales saw a 39% increase in total demand compared to Q1 2023, despite a decrease in patients switching from ONPATTRO to AMVUTTRA [12][14] - International TTR franchise growth increased by 23% year-over-year, primarily due to AMVUTTRA demand in newly launched markets [13][14] Company Strategy and Development Direction - Alnylam aims to position itself as a leader in treating cardiovascular disease through its zilebesiran hypertension program, which has shown positive results in clinical trials [8][21] - The company is focused on advancing its pipeline with plans to file proprietary INDs for nine programs by the end of 2025, aiming to double its clinical pipeline [24][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in a strong start to 2024, with expectations for continued revenue growth and successful outcomes from ongoing clinical trials [7][9] - The company remains optimistic about the HELIOS-B Phase 3 study results, which are expected to support a supplemental NDA filing by the end of 2024 if positive [9][20] Other Important Information - Alnylam ended Q1 2024 with cash, cash equivalents, and marketable securities of$ 2.4 billion, indicating a stable financial position [28] - The company reiterated its 2024 guidance for combined net product revenues between $1.4 billion and$ 1.5 billion, reflecting a growth rate of 13% to 21% [29] Q&A Session Summary Question: What is the relevant improvement over the control arm for HELIOS-B? - Management emphasized that delivering outcomes in the HELIOS-B study is crucial and that they are focused on demonstrating clinical significance in terms of mortality and hospitalization [34][36] Question: What would be the organizational changes if HELIOS-B succeeds or fails? - If HELIOS-B succeeds, no changes to spending guidance are anticipated. However, if it fails, prioritization across the business would be necessary [40][41] Question: How important is the ratio between hospitalization events and deaths in HELIOS-B? - Management indicated that both hospitalization and mortality events are clinically meaningful and should trend in the same direction [70][72] Question: What proportion of patients are mixed phenotype in ATTR? - It was noted that a significant proportion of patients with wild type ATTR may have concomitant polyneuropathy, highlighting the complexity of the disease [75][76] Question: Will subgroup information be shared in the top line release for HELIOS-B? - Management confirmed that they plan to provide information on primary and secondary endpoints, including subgroup analyses, during the top line results presentation [61][80]