Alnylam Pharmaceuticals (ALNY, Financial) has taken a significant step forward with its RNA interference therapeutic, vutrisiran. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended the approval of vutrisiran for treating cardiomyopathy in adult patients suffering from either wild type or hereditary transthyretin amyloidosis.
This recommendation is crucial as transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a severe, fast-progressing condition with limited treatment options currently available. The proposed extension of vutrisiran's use would address this unmet medical need.
Vutrisiran is already authorized within the European Union under the brand name Amvuttra for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. If approved for cardiomyopathy, this expansion would enhance treatment possibilities for patients battling this life-threatening disease.
Wall Street Analysts Forecast
Based on the one-year price targets offered by 28 analysts, the average target price for Alnylam Pharmaceuticals Inc (ALNY, Financial) is $317.82 with a high estimate of $500.00 and a low estimate of $195.00. The average target implies an upside of 26.34% from the current price of $251.56. More detailed estimate data can be found on the Alnylam Pharmaceuticals Inc (ALNY) Forecast page.
Based on the consensus recommendation from 33 brokerage firms, Alnylam Pharmaceuticals Inc's (ALNY, Financial) average brokerage recommendation is currently 2.1, indicating "Outperform" status. The rating scale ranges from 1 to 5, where 1 signifies Strong Buy, and 5 denotes Sell.
Based on GuruFocus estimates, the estimated GF Value for Alnylam Pharmaceuticals Inc (ALNY, Financial) in one year is $436.84, suggesting a upside of 73.65% from the current price of $251.56. GF Value is GuruFocus' estimate of the fair value that the stock should be traded at. It is calculated based on the historical multiples the stock has traded at previously, as well as past business growth and the future estimates of the business' performance. More detailed data can be found on the Alnylam Pharmaceuticals Inc (ALNY) Summary page.
ALNY Key Business Developments
Release Date: February 13, 2025
- Combined Net Product Revenue: $1.646 billion for 2024, representing 33% growth compared to 2023.
- TTR Franchise Revenue: $343 million in Q4 2024, a 35% increase compared to Q4 2023.
- Full Year TTR Revenue: $705 million in the US, a 39% increase compared to 2023.
- Rare Franchise Revenue: $108 million in Q4 2024, an 18% increase compared to Q4 2023.
- Gross Margin on Product Sales: 81% for the full year 2024, a 3% increase compared to 2023.
- Non-GAAP Operating Income: $95 million for the full year 2024.
- Cash, Cash Equivalents, and Marketable Securities: $2.7 billion at the end of 2024.
- 2025 Revenue Guidance: $2.05 billion to $2.25 billion in combined net product sales.
- 2025 TTR Revenue Guidance: $1.6 billion to $1.725 billion, assuming approval and launch of AMVUTTRA for ATTR cardiomyopathy.
- 2025 Rare Revenue Guidance: $450 million to $525 million.
- 2025 Collaboration and Royalty Revenue Guidance: $650 million to $750 million.
- 2025 Non-GAAP R&D and SG&A Expenses: $2.1 billion to $2.2 billion.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Alnylam Pharmaceuticals Inc (ALNY, Financial) reported strong financial performance with combined net product revenues of over $1.6 billion, achieving a 33% growth compared to 2023.
- The company achieved a significant milestone with $95 million in non-GAAP operating income for the full year, marking its first year of profitability.
- The HELIOS-B Phase III study yielded highly positive results, leading to the submission of global regulatory filings for vutrisiran in ATTR cardiomyopathy.
- Alnylam Pharmaceuticals Inc (ALNY) has a robust pipeline with numerous multibillion-dollar opportunities, including the advancement of Nucresiran, which received orphan drug designation for ATTR amyloidosis.
- The company maintained its award-winning culture, which has been a critical enabler of its success, and is poised for sustainable growth and value creation in 2025 and beyond.
Negative Points
- The launch of AMVUTTRA for ATTR cardiomyopathy is expected to face delays in formulary uptake, with meaningful demand anticipated only in the second half of 2025.
- Gross margin on product sales is expected to decrease in 2025 due to the increased royalty burden on AMVUTTRA sales.
- The company faces competition in the market, particularly in the polyneuropathy segment, which could impact its market share and growth.
- There is uncertainty regarding payer policies and the establishment of value-based agreements for the expanded label of AMVUTTRA in cardiomyopathy.
- Alnylam Pharmaceuticals Inc (ALNY) anticipates increased R&D and SG&A expenses in 2025, driven by clinical investments and the launch of AMVUTTRA, which could impact profitability.
