Analyst Conference Summary

biotechnology

conference date: February 13, 2025 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2024 (fourth quarter, Q4)

Forward-looking statements

Overview: Continuing spectacular revenue growth, even had a bit of positive non-GAAP net income.

Basic data (GAAP):

Revenue was $593 million, up 18% sequentially from $501 million, and up 30% from $440 million year-earlier.

Net income was negative $84 million, up sequentially from negative $112 million, and down from negative $74 million year-earlier.

Diluted EPS was negative $0.65, up sequentially from negative $0.87, and up from negative $1.10 year-earlier.

Guidance:

2025 net product revenue $2.05 to $2.25 billion. Non-GAAP Operating expense $2.1 to $2.2 billion. Guiding to non-GAAP profitability.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2024 was another year of impressive execution for Alnylam, generating product revenues of over $1.6 billion, reflecting growth of 33% compared to 2023, and highlighting the strength of our base business in hATTR-PN and Rare in both the U.S. and international markets. We look forward to potential global launches of vutrisiran in ATTR-CM this year, which will mark an inflection point for our TTR franchise and put us on a path to achieve the financial guidance we’ve provided. Furthermore, driven by our proven RNAi platform, we anticipate 2025 will bring major advancements in our pipeline and expect to have over 25 high-value programs in the clinic across diverse indications by the end of the year. We have seen a remarkable pace of progress toward our Alnylam P5x25 goals and believe we are well positioned for the next half of the decade to continue delivering sustainable innovation to patients." Expects to achieve non-GAAP profitability by end of 2025.

In Q4 2024 announced positive results from Phase 1 study of mivelsiran for Alzheaimer's disease. Showed lowering of sAPPB, which should lower amyloid production.

Amvutra (Vutrisiran) Phase 3 ATTR amyloidosis with cardiomyopathy positive topline results (Helios-B) reported in July 2024. Submitted regulatory submissions (FDA and EMA)in Q3 2024 with accelerated review in the US, PDUFA March 23, 2025. Expects to launch in U.S. in early 2025. Believes in the global market 80% of potential ATTR-CM patietns are undiagnosed.

Alnylam will host an R&D Day on February 25, 2025.

In Q4 2024 received FDA Orphan Drug Designation for nucresiran (ALN-TTRsc04). Interim Phase 1 data in patients with ATTR amyloidosis were positive.

In Q4 2024 interim Phase 1 data for mivelsiran for Alzheimer's were positive.

In Q4 2024 initiated a Phase 1 study of ALN-HTT02 in adult patients with Huntington's disease.

In Q4 2024 initiated Phase 1 study of ALN-6400 for a bleeding disorder.

In Q4 2024 initiated Phase 1 study ALN-4324 for type 2 diabetes mellitus.

In Q2 2024 partner Sanofi received a PDUA date of March 28, 2025 for fitusiran. In Q4 2023 Sanofi presented positive Phase 3 extension results for fitusiran for hemophilia A or B.

Net revenue from collaborations was $ million, down from $57 million year-earlier. Royalty revenue was $ million, up from $23 million year-earlier.

Non-GAAP net income $8.0 million, up sequentially from negative $64 million, and up from negative $97 million year-earlier. EPS $0.06, up sequentially from negative $0.50, and up from negative $0.77 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.69 billion, down sequentially from $2.8 billion. Convertible Debt $1.0 billion.

See also Alnylam pipeline. In Q1 2025 had 20 clinical programs including 10 in late stages.

Operating expenses of $698 million consisted of: $ million for cost of goods sold; $300 million for research and development; and $295 million for general and administrative expense; $0 million cost of collaboration. Operating loss $105 million. Interest & other expense was $89 million. $110 million income tax.

Q&A Selective Summary:

Payer timeline for Amvutra? We have a foundation with polyneuropathy. Payer approvals will mainly be in second half of 2025. Formulary a bit longer.

Amvutra pricing v. silencers? It has a great product profile, discussing pricing with payers, who see the value. Expect uptake to be consistent with PN.

Factors in Amvutra pricing? We consider the value we bring to patients. We can use the same J code. We expect Medicare and commercial coverage to remain the same.

Label? The FDA review is going well. We believe the study showed the mortality and other benefits, which should be reflected in the label.

Competitive dynamics with Wainua? We see the category growing, we grew 42% in the U.S. in 2024 despite the competition. We believe Amvutra is highly differentiated from Wainua in PN, and will continue to be in cardiomyopathy.

Nucresiran trial enrollment given availability of current drugs? We think it can be best in class. Want to bring it to market as quickly as possible. More at R&D Day.

Driver of tax benefit? Release of a valuation allowance for Swiss entity. Evaluate every quarter. Difficult to predict going forward.

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