Alnylam Pharmaceuticals(ALNY) - 2022 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Alnylam achieved full year 2022 net product revenues of $894 million, representing year-over-year growth of 35$894 million, reflecting a 35% increase compared to 2021, with growth contributions from all four commercial products [34] - Non-GAAP operating loss for 2022 was $554 million, approximately flat compared to 2021, with expectations to reduce this loss in 2023 [37][40] Business Line Data and Key Metrics Changes - The TTR franchise achieved$191 million in global net product revenues in Q4, a 12% increase from Q3 and a 38% increase from Q4 2021 [12][13] - GIVLAARI generated $47 million in global net product revenues in Q4, representing a 3$24 million in global net product revenues in Q4, a 45% increase compared to Q3 [20] Market Data and Key Metrics Changes - In the U.S., combined sales of ONPATTRO and AMVUTTRA increased 12% versus Q3, driven by a 19% increase in demand growth [14][15] - The U.S. market for AMVUTTRA saw a 72% growth compared to Q4 2021, with over 2,975 patients on treatment worldwide by the end of 2022 [13][15] - International sales of ONPATTRO and AMVUTTRA also increased 12% versus Q3, primarily due to initial launches in Germany and Japan [14][18] Company Strategy and Development Direction - Alnylam aims to expand its ATTR franchise and become a global leader in delivering impactful medicines, while also addressing more common disease areas [10] - The company is focusing on sustainable innovation through new platform enhancements and finding new genetically validated targets [10] - Alnylam plans to advance TTRsc04, a new investigational RNAi therapeutic, which may offer a transformative profile with potential for annual dosing [30] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming FDA review and potential approval of patisiran for cardiomyopathy, with a PDUFA date set for October 8, 2023 [24][39] - The company anticipates combined net product revenues for 2023 to be between $1.2 billion and$1.285 billion, reflecting a growth range of 34% to 44% [39][40] - Management highlighted the strong initial commercial performance of AMVUTTRA and its potential to expand the market opportunity for the TTR franchise [21][22] Other Important Information - Alnylam ended 2022 with cash, cash equivalents, and marketable securities of $2.2 billion, down from$2.4 billion at the end of 2021 [38] - The company plans to submit an sNDA in Brazil for patisiran in early 2023, focusing efforts on regions with positive results from ongoing studies [25] Q&A Session Summary Question: Advisory committee meeting topics - Management indicated no specific areas to address for the advisory committee meeting, as no review issues have been identified [44][45] Question: Learnings from AMVUTTRA dosing - Management stated that the quarterly regimen of AMVUTTRA has been effective, and the decision not to pursue a six-month regimen was strategic [48][49] Question: Top line data from ALN-APP study - Management confirmed that the top line data will focus on safety, tolerability, and target engagement, with results expected in early 2023 [52][54] Question: HELIOS-B study design rationale - Management explained that the all-cause mortality endpoint maximizes event capture and provides a clinically meaningful effect [57][59] Question: Impact of J-Code on AMVUTTRA launch - Management noted that the J-Code received would not significantly change the trajectory of the launch, as access strategies were already in place [82][84]