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Alnylam Pharmaceuticals (ALNY - Free Report) , a commercial-stage company, focuses on developing novel therapeutics based on RNAi. The company’s pipeline of experimental RNAi therapeutics is focused across three strategic therapeutic areas — genetic medicines, cardio-metabolic disease and hepatic infectious disease. Alnylam currently markets four drugs — Amvuttra (vutrisiran), Givlaari (givosiran), Oxlumo (lumasiran) and Onpattro (patisiran).
Portfolio of Marketed Drugs Boosts Revenues
Amvuttra, the company’s lead drug, is approved in the United States and EU for the treatment of adult patients with polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. The drug’s sales contribute significantly to ALNY’s top line. Amvuttra generated sales worth $425.4 million in the first half of 2024, up 82% year over year on a reported basis. The encouraging uptake of the drug is driven by new patients starting treatment as well as patients switching from Onpattro. Notably, Onpattro is approved in the United States and EU to treat hATTR amyloidosis in adults.
In 2019, the FDA approved Givlaari for acute hepatic porphyria. In 2020, the FDA approved Oxlumo injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients.
Alnylam also markets a fifth drug, Leqvio (inclisiran), in collaboration with Novartis (NVS - Free Report) to treat hypercholesterolemia in the EU. In the United States, it is approved to reduce low-density lipoprotein cholesterol (LDL-C) with two doses per year. Per the agreement, Alnylam has granted Novartis exclusive and worldwide rights to manufacture and commercialize RNAi therapeutics targeting PCSK9 for treating hypercholesterolemia and other human diseases, including Leqvio. Novartis has also received FDA approval to expand Leqvio’s label to include earlier use in patients with elevated LDL-C who have an increased risk of heart disease, as an adjunct to diet and statin therapy.
Impressive Pipeline
Apart from its marketed portfolio of drugs, Alnylam also has an impressive clinical-stage pipeline. The company is currently working on expanding Amvuttra’s label. In June 2024, Alnylam reported the success of its phase III HELIOS-B study evaluating vutrisiran for the treatment of patients with ATTR amyloidosis with cardiomyopathy. Based on the encouraging results, the company is gearing up to submit regulatory application, seeking label expansion for Amvuttra, in the United States and several other countries. Year to date, shares of ALNY have rallied 44.9% compared with the industry’s 1.6% growth.
Image Source: Zacks Investment Research
Among other candidates under development in Alnylam’s pipeline, the interesting ones include cemdisiran, mivelsiran and zilebesiran.
Alnylam and its partner, Regeneron (REGN - Free Report) , are developing a cemdisiran and pozelimab combination in late-stage studies for treating myasthenia gravis and paroxysmal nocturnal hemoglobinuria. ALNY is also developing cemdisiran as a monotherapy, which is currently phase III ready for the treatment of IgA nephropathy. Per the modified agreement with Regeneron, signed in June 2024, Alnylam granted exclusive rights to Regeneron to develop cemdisiran as monotherapy for complement-mediated indications.
Alnylam and Regeneron are also jointly developing another candidate, mivelsiran (formerly ALN-APP), for treating early-onset Alzheimer’s disease and cerebral amyloid angiopathy. However, ALNY gained full global development and commercialization rights to mivelsiran in all indications, as Regeneron opted out of further co-development and co-commercialization of mivelsiran. REGN will be eligible to receive low double-digit royalties on sales of mivelsiran if approved.
Last year, the company entered into a strategic collaboration with Roche (RHHBY - Free Report) to co-develop and co-commercialize zilebesiran to treat hypertension. The total value of the zilebesiran deal with Roche is approximately $2.8 billion. Alnylam and Roche have also initiated a third phase II study (KARDIA 3) to evaluate the efficacy of zilebesiran when added to two or more hypertension medications in people with uncontrolled hypertension who are at high cardiovascular risk.
Potential Limiting Factors
However, the company has encountered its fair share of pipeline setbacks in the past. In late 2023, the FDA issued a complete response letter (CRL) to Alnylam’s regulatory filing, seeking label expansion for Onpattro to treat the cardiomyopathy of ATTR amyloidosis. The CRL argued that the existing study data do not establish the clinical meaningfulness of Onpattro’s treatment effects for the above indication. Hence, the application could not be approved in its present form.
Based on the FDA’s CRL, Alnylam has decided to discontinue pursuing an expanded indication for Onpattro in the United States. Such setbacks do not bode well for the company and can hurt growth prospects in the days ahead.
The company also faces significant competition from other companies who are also developing RNA-based drugs, like Ionis, Sarepta Therapeutics and Roche. Some companies like Takeda and Novo Nordisk are even looking to develop chemically synthesized siRNAs as drugs. Also, AstraZeneca and Ionis’ Wainua (eplontersen) are approved in the United States to treat the polyneuropathy of hATTR amyloidosis in adults. This has induced acute competition for Alnylam’s Amvuttra.
In the past five years, shares of Alnylam have skyrocketed more than 230% and we anticipate further growth potential in the years to come. The company’s marketed drugs like Amvuttra, Oxlumo and Givlaari are performing well, generating increasing year-over-year revenues for the company. Additionally, it is also working on expanding Amvuttra’s label to include ATTR amyloidosis with cardiomyopathy. If approved, the label update will expand the eligible patient population for the drug, boosting its sales significantly in the future.
ALNY’s existing collaborations with Regeneron, Novartis and Roche are expected to keep generating significant revenues for the company that will boost its cash balance to fund operating and clinical activities.
Image: Bigstock
Alnylam (ALNY) Amvuttra & Givlaari Fuel Revenues Amid Competition
Alnylam Pharmaceuticals (ALNY - Free Report) , a commercial-stage company, focuses on developing novel therapeutics based on RNAi. The company’s pipeline of experimental RNAi therapeutics is focused across three strategic therapeutic areas — genetic medicines, cardio-metabolic disease and hepatic infectious disease. Alnylam currently markets four drugs — Amvuttra (vutrisiran), Givlaari (givosiran), Oxlumo (lumasiran) and Onpattro (patisiran).
Portfolio of Marketed Drugs Boosts Revenues
Amvuttra, the company’s lead drug, is approved in the United States and EU for the treatment of adult patients with polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. The drug’s sales contribute significantly to ALNY’s top line. Amvuttra generated sales worth $425.4 million in the first half of 2024, up 82% year over year on a reported basis. The encouraging uptake of the drug is driven by new patients starting treatment as well as patients switching from Onpattro. Notably, Onpattro is approved in the United States and EU to treat hATTR amyloidosis in adults.
In 2019, the FDA approved Givlaari for acute hepatic porphyria. In 2020, the FDA approved Oxlumo injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients.
Alnylam also markets a fifth drug, Leqvio (inclisiran), in collaboration with Novartis (NVS - Free Report) to treat hypercholesterolemia in the EU. In the United States, it is approved to reduce low-density lipoprotein cholesterol (LDL-C) with two doses per year. Per the agreement, Alnylam has granted Novartis exclusive and worldwide rights to manufacture and commercialize RNAi therapeutics targeting PCSK9 for treating hypercholesterolemia and other human diseases, including Leqvio. Novartis has also received FDA approval to expand Leqvio’s label to include earlier use in patients with elevated LDL-C who have an increased risk of heart disease, as an adjunct to diet and statin therapy.
Impressive Pipeline
Apart from its marketed portfolio of drugs, Alnylam also has an impressive clinical-stage pipeline. The company is currently working on expanding Amvuttra’s label. In June 2024, Alnylam reported the success of its phase III HELIOS-B study evaluating vutrisiran for the treatment of patients with ATTR amyloidosis with cardiomyopathy. Based on the encouraging results, the company is gearing up to submit regulatory application, seeking label expansion for Amvuttra, in the United States and several other countries. Year to date, shares of ALNY have rallied 44.9% compared with the industry’s 1.6% growth.
Image Source: Zacks Investment Research
Among other candidates under development in Alnylam’s pipeline, the interesting ones include cemdisiran, mivelsiran and zilebesiran.
Alnylam and its partner, Regeneron (REGN - Free Report) , are developing a cemdisiran and pozelimab combination in late-stage studies for treating myasthenia gravis and paroxysmal nocturnal hemoglobinuria. ALNY is also developing cemdisiran as a monotherapy, which is currently phase III ready for the treatment of IgA nephropathy. Per the modified agreement with Regeneron, signed in June 2024, Alnylam granted exclusive rights to Regeneron to develop cemdisiran as monotherapy for complement-mediated indications.
Alnylam and Regeneron are also jointly developing another candidate, mivelsiran (formerly ALN-APP), for treating early-onset Alzheimer’s disease and cerebral amyloid angiopathy. However, ALNY gained full global development and commercialization rights to mivelsiran in all indications, as Regeneron opted out of further co-development and co-commercialization of mivelsiran. REGN will be eligible to receive low double-digit royalties on sales of mivelsiran if approved.
Last year, the company entered into a strategic collaboration with Roche (RHHBY - Free Report) to co-develop and co-commercialize zilebesiran to treat hypertension. The total value of the zilebesiran deal with Roche is approximately $2.8 billion. Alnylam and Roche have also initiated a third phase II study (KARDIA 3) to evaluate the efficacy of zilebesiran when added to two or more hypertension medications in people with uncontrolled hypertension who are at high cardiovascular risk.
Potential Limiting Factors
However, the company has encountered its fair share of pipeline setbacks in the past. In late 2023, the FDA issued a complete response letter (CRL) to Alnylam’s regulatory filing, seeking label expansion for Onpattro to treat the cardiomyopathy of ATTR amyloidosis. The CRL argued that the existing study data do not establish the clinical meaningfulness of Onpattro’s treatment effects for the above indication. Hence, the application could not be approved in its present form.
Based on the FDA’s CRL, Alnylam has decided to discontinue pursuing an expanded indication for Onpattro in the United States. Such setbacks do not bode well for the company and can hurt growth prospects in the days ahead.
The company also faces significant competition from other companies who are also developing RNA-based drugs, like Ionis, Sarepta Therapeutics and Roche. Some companies like Takeda and Novo Nordisk are even looking to develop chemically synthesized siRNAs as drugs. Also, AstraZeneca and Ionis’ Wainua (eplontersen) are approved in the United States to treat the polyneuropathy of hATTR amyloidosis in adults. This has induced acute competition for Alnylam’s Amvuttra.
Alnylam Pharmaceuticals, Inc. Price and Consensus
Alnylam Pharmaceuticals, Inc. price-consensus-chart | Alnylam Pharmaceuticals, Inc. Quote
Conclusion
In the past five years, shares of Alnylam have skyrocketed more than 230% and we anticipate further growth potential in the years to come. The company’s marketed drugs like Amvuttra, Oxlumo and Givlaari are performing well, generating increasing year-over-year revenues for the company. Additionally, it is also working on expanding Amvuttra’s label to include ATTR amyloidosis with cardiomyopathy. If approved, the label update will expand the eligible patient population for the drug, boosting its sales significantly in the future.
ALNY’s existing collaborations with Regeneron, Novartis and Roche are expected to keep generating significant revenues for the company that will boost its cash balance to fund operating and clinical activities.
Therefore, based on the above discussion, we can conclude that ALNY is a good stock to retain in one’s portfolio. Alnylam currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.