10-KPeriod: FY2024

ALNYLAM PHARMACEUTICALS, INC. Annual Report, Year Ended Dec 31, 2024

Filed February 13, 2025For Securities:ALNY

Summary

Alnylam Pharmaceuticals, Inc. (ALNY) has filed its 2024 10-K report, highlighting significant progress in its RNAi therapeutics pipeline and commercialization efforts. The company has five approved products: ONPATTRO, AMVUTTRA, GIVLAARI, OXLUMO, and Leqvio (commercialized by Novartis). A key development is the filing of a supplemental New Drug Application (sNDA) for AMVUTTRA for ATTR amyloidosis with cardiomyopathy, with a target action date from the FDA set for March 23, 2025. The company also reported strong revenue from its TTR franchise, with ONPATTRO and AMVUTTRA generating $252.9 million and $970.5 million in global net product revenues, respectively, for 2024. Furthermore, GIVLAARI and OXLUMO contributed $255.9 million and $167.1 million in global net revenues. Alnylam continues to advance its "Alnylam P5x25" strategy, aiming to become a top-tier biotech company by the end of 2025. The company ended 2024 with a robust cash position of $2.69 billion, indicating financial stability and the ability to fund ongoing operations and development without immediate need for equity financing. The pipeline features several promising late-stage candidates, including zilebesiran for hypertension, nucresiran for ATTR amyloidosis, and mivelsiran for Alzheimer's disease, with significant updates expected in 2025 from clinical trials and regulatory submissions.

Financial Highlights

48 data points
Beta

Revenue

$2.25B

86.4% Gross Margin

Net Income

-$278.16M

-12.4% Net Margin

EPS (Diluted)

$-2.18

Total Assets

$4.24B

Cash & Equivalents

$966.43M

Free Cash Flow

-$42.59M

Financial Statements
Beta
Revenue$2.25B
Cost of Revenue$306.51M
Gross Profit$1.94B
R&D Expenses$1.13B
SG&A Expenses$975.53M
Operating Expenses$2.43B
Operating Income-$176.88M
Net Income-$278.16M
EPS (Basic)$-2.18
EPS (Diluted)$-2.18
Shares Outstanding (Basic)127.65M
Shares Outstanding (Diluted)127.65M

Key Highlights

  • 1AMVUTTRA (vutrisiran) sNDA for ATTR amyloidosis with cardiomyopathy accepted by FDA with March 23, 2025 PDUFA target action date.
  • 2Strong revenue performance in 2024: ONPATTRO ($252.9M), AMVUTTRA ($970.5M), GIVLAARI ($255.9M), and OXLUMO ($167.1M).
  • 3Ended 2024 with $2.69 billion in cash, cash equivalents, and marketable securities, supporting operational self-sustainability.
  • 4Advancing a robust pipeline with multiple late-stage programs, including zilebesiran for hypertension, with Phase 3 trial initiation expected in H2 2025.
  • 5Fitusiran (hemophilia) NDA with Sanofi has a PDUFA target action date of March 28, 2025.
  • 6Positive Phase 1 data for nucresiran (ATTR amyloidosis) suggests potential for over 90% TTR reduction and annual dosing, with a Phase 3 trial planned for H1 2025.
  • 7Regeneron opted out of further co-development of mivelsiran (Alzheimer's/CAA), returning full global rights to Alnylam.

Frequently Asked Questions

Alnylam has five approved RNAi therapeutics: ONPATTRO and AMVUTTRA for ATTR amyloidosis with polyneuropathy, GIVLAARI for acute hepatic porphyria (AHP), OXLUMO for primary hyperoxaluria type 1 (PH1), and Leqvio (developed with Novartis) for hypercholesterolemia. In 2024, global net product revenues were $252.9 million for ONPATTRO, $970.5 million for AMVUTTRA, $255.9 million for GIVLAARI, and $167.1 million for OXLUMO.

Alnylam has filed a supplemental New Drug Application (sNDA) with the FDA for AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy. The FDA has accepted this submission for review with a Prescription Drug User Fee Act (PDUFA) target action date of March 23, 2025. Regulatory reviews are also underway in Europe and Japan.

As of December 31, 2024, Alnylam reported $2.69 billion in cash, cash equivalents, and marketable securities. The company believes this balance is sufficient to achieve a self-sustainable profile without the need for future equity financing.

Key upcoming milestones include the potential FDA approval for AMVUTTRA in ATTR amyloidosis with cardiomyopathy (March 2025), the FDA action date for Sanofi's fitusiran NDA for hemophilia (March 2025), the initiation of a Phase 3 trial for nucresiran in ATTR amyloidosis with cardiomyopathy (H1 2025), and the expected reporting of results from the KARDIA-3 Phase 2 trial for zilebesiran and initiation of a Phase 3 cardiovascular outcomes trial (H2 2025).