Analyst Conference Summary

biotechnology

conference date: May 1, 2025 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2025 (first quarter, Q1)

Forward-looking statements

Overview: Continued strong revenue growth, still in the red.

Basic data (GAAP):

Revenue was $594 million, up slightly sequentially from $593 million, and up 20% from $494 million year-earlier.

Net income was negative $57 million, up sequentially from negative $84 million, and down from negative $66 million year-earlier.

Diluted EPS was negative $0.44, up sequentially from negative $0.65, and up from negative $0.52 year-earlier.

Guidance:

For 2025 expects product revenue between $2.05 and $2.25 billion. Expects to achieve non-GAAP operating income profitability.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2025 is off to a remarkable start, having ended the first quarter with U.S. regulatory approval of Amvutra for ATTR-CM. This important launch is underway, and we are delighted with our early progress in driving awareness, unlocking patient access, and getting patients on treatment in the initial few weeks since approval. We are also thrilled with the U.S. FDA approval of Qfitlia, under license with Sanofi, which is the sixth Alnylam-discovered RNAi therapeutic approved in the U.S. As we progress the Amvutra launch and further solidify our leadership in TTR, we are excited by the opportunity we have to reinvest in our highly productive RNAi platform, for which we highlighted key programs at our recent R&D Day. These include multiple near- and mid-stage potentially transformative therapies that represent blockbuster opportunities as our pipeline rapidly expands across multiple therapeutic areas. This unique biotech profile will propel us toward achievement of our Alnylam P5x25 goals and continue delivering sustainable innovation to patients for many years to come." Expects to achieve non-GAAP profitability by end of 2025. Reimbursement for -CM is increasing rapidly, with 50% formulary coverage.

Alnylam does not expect any meaningful impacts from any new tariffs on pharmaceuticals.

Amvutra (Vutrisiran) Phase 3 ATTR amyloidosis with cardiomyopathy approved by FDA in March 2025. Positive topline results (Helios-B) were reported in July 2024. Expects to launch in U.S. in early 2025. Believes in the global market 80% of potential ATTR-CM patietns are undiagnosed. Received approval from Brazil and the EU CHMP recommended approval in Q2 2025. Amvutra captured 70% of new patient starts in Q1 2025.

In March 2025 fitusiran for hemophilia A and B was approved by the FDA.

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.

In Q1 2025 announced coming Phase 1 trial for ALN-6400 for bleeding disorders without risk of thrombosis.

In Q1 2025 announced initiation of a Phase 1 trial of ALN-4324, with GRB14 as the target, to sensatize patients for insulin in type 2 diabetes.

In Q1 2025 shared encouraging data from the ALN-HTT02 program, which has a highly differentiated exon-1-targeting approach to lower huntingtin (HTT) for Huntington's disease.

Alnylam plans to initiate the TRITON-CM Phase 3 trial of nucresiran in patients with ATTR-CM in 1H 2025.

A newer potential therapy for ATTR-PN and -CM, nucresiran, should enter Phase 3 trials in 2025.

Net revenue from collaborations was $99 million, down from $119 million year-earlier. Royalty revenue was $26 million, up from $11 million year-earlier.

Non-GAAP net income negative $1 million, down sequentially from $8.0 million, and up from negative $21 million year-earlier. EPS negative $0.01, dowm sequentially from $0.06, and up from negative $0.16 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.62 billion, down sequentially from $2.69 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 $576 million consisted of: $70 million for cost of goods sold; $265 million for research and development; and $240 million for general and administrative expense; $1 million cost of collaboration. Operating income $18 million. Interest & other expense was $60 million. $16 million income tax.

Q&A Selective Summary:

Initial ATTR-CM patients? All the launch indicators are flashing green. Prescriber base is broad, cardiologists, including some that had not previously prescribed Amvuttra.

How much of the quarter increase was due to new CM label? 36% growth in guidance this year. essentially all CM. The category remains untapped in terms of growth, even tafamidis numbers showed.

Plans to capture tafamidis progressors? We have a whole new mechanism of action. Our data showed good results for those already on stabilizers. Physicians want to treat patients early and with the best option. So Amvuttra is the best first line option. Studies show patients do progress (to failure) on stabilizer.

Pricing dynamics? We have a single price, no difference between indications. We help patients navigate a very comples US health system. We see very limited use of our Quick Start program, which we have had in place for years. Most patients have zero copays.

We believe some competitors in this space are "selectively highlighting data points, with creative interpretation."

Doctors know how to recognize when stabilizers are failing and patients are progressing, from long experience and multiple testing parameters.

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