RNAi Giant: $12 Billion in Sales!

Recently, RNAi giant Alnylam Pharmaceuticals announced its 2024 financial results, reporting total annual revenue of $2.248 billion, a 23% year-over-year increase. However, due to significant R&D investments, the company remains unprofitable.

Product Revenue

In 2024, Alnylam’s product sales reached $1.646 billion, up 33% year-over-year. This growth was mainly driven by increased sales of AMVUTTRA, as well as higher sales of GIVLAARI and OXLUMO, partially offset by a decline in ONPATTRO sales.

Alnylam's commercial RNAi therapeutics include ONPATTRO (patisiran), AMVUTTRA (vutrisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), and Leqvio (inclisiran).

Among them, the siRNA drugs ONPATTRO (patisiran) and AMVUTTRA (vutrisiran), used for treating transthyretin amyloidosis, recorded 2024 sales of $253 million (-29%) and $970 million (+74%), respectively, totaling $1.223 billion.

Additionally, the RNAi drug OXLUMO (lumasiran) for treating primary hyperoxaluria had sales of $167 million (+52%) in 2024. The RNAi drug GIVLAARI (givosiran) for treating acute hepatic porphyria recorded sales of $256 million (+17%) in 2024, totaling $423 million.

Thus, Alnylam’s four RNAi drugs collectively generated $1.646 billion in sales in 2024 (approximately ￥12 billion, based on the latest exchange rate of 1 USD ≈ ￥7.2846), reflecting a 33% year-over-year increase.

Clinical Progress

• Vutrisiran: The HELIOS-B clinical study achieved both primary and secondary endpoints in June. A supplemental new drug application (sNDA) was submitted to the FDA for treating ATTR amyloidosis cardiomyopathy, with a target action date of March 23, 2025.
• Mivelsiran: Preliminary positive results were observed in the multi-dose Phase 1 study in Alzheimer's patients. After the second 50 mg dose at month six, further reductions in sAPPβ were observed in month seven, exceeding a 90% decrease.
• ALN-HTT02: A Phase 1 clinical study was initiated in adult Huntington’s disease patients, with non-clinical data shared.

R&D Adjustments

Alnylam has suspended further clinical development of the experimental RNAi therapy ALN-KHK for type 2 diabetes. The company is reallocating R&D resources to focus on more promising projects.

Partnerships and Business Expansion

• Collaboration with Roche: Alnylam partnered with Roche to co-develop and commercialize zilebesiran for hypertension treatment. In 2024, Alnylam received a $310 million upfront payment and a $65 million milestone payment.
• Regeneron Partnership Adjustment: Regeneron exited further collaboration and commercialization of Mivelsiran for CAA and Alzheimer's disease. Alnylam regained full global development and commercialization rights for all Mivelsiran indications. If Mivelsiran is approved, Regeneron will receive a low double-digit royalty.

About Alnylam Pharmaceuticals

Founded in 2002, Alnylam is dedicated to developing innovative drugs using RNA interference (RNAi) technology to treat various diseases. The company has an extensive RNAi-based drug pipeline spanning rare diseases, cardiovascular diseases, metabolic disorders, and neurological conditions.