Analyst Conference Summary

biotechnology

conference date: February 15, 2024 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2023 (fourth quarter, Q4)

Forward-looking statements

Overview: Continued strong revenue growth, but back in the red.

Basic data (GAAP):

Revenue was $440 million, down 41% sequentially from $751 million, and up 31% from $335 million year-earlier.

Net income was negative $138 million, down sequentially from $148 million, and up from negative $207 million year-earlier.

Diluted EPS was negative $1.10, down sequentially from $1.15, and up from negative $1.68 year-earlier.

Guidance:

2024 net product revenue $1.4 to $1.5 billion, plus collaboration and royalties.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2023 was a year of strong execution at Alnylam. We delivered robust product revenue growth across our four wholly-owned commercial medicines, with $1.24 billion in global net product revenues, and achieved over 5,000 patients now being treated with an Alnylam commercial medicine. We also extended our leadership in RNAi with the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic, strengthened our business for the future through a landmark partnership with Roche and delivered solid financial performance. Looking ahead to 2024, we are excited for a number of important milestones across the pipeline, including results from the HELIOS-B Phase 3 study of vutrisiran as well as the KARDIA-2 Phase 2 study of zilebesiran, and six clinical study starts. This progress sets us up for delivering on our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial results." Over 5,000 patients are now on Alnylam commercial medicines. But Pfizer's tafamidis will lose patent protection in Q4 2028.

Amvutra (Vutrisiran) Phase 3 ATTR amyloidosis with cardiomyopathy topline results (Helios-B) are expected by July 2024. Increasing follow up to 36 months. Will analyse both combined with tafamadis and as monotherapy.

In Q4 2023 ALN-TTRsc04 demonstrated positive results for TTR reduction.

Zilebesirin for hypertension reported positive Phase 2 topline results in October 2023. Will partner with Roche for global development and distribution.

In Q3 2023 Leqvio (inclisiran) was approved in China and Japan. US launch ongoing. Leqvio results in royalty income.

In Q4 2023 reported positive Phase 1 results for ALN-KHK for type 2 diabetes.

Vir is conducting multiple trials evaluating the potential for ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional cure for chronic hepatitis B. Phase 2 data readouts are on track for Q4 2023.

In Q4 2023 Alnylam filed an NDA for ALB-BCAT for hepatocellular carcinoma. It targets B-catenin.

In Q4 2023 Sanofi presented positive Phase 3 extension results for fitusiran for hemophilia A or B.

ALN-APP phase 1 results are positive, reported Q3 2023, will start part B multidose study. For Alzheimer's.

Net revenue from collaborations was $76 million, up from $71 million year-earlier. Royalty revenue was $17 million, up from $3 million year-earlier.

Non-GAAP net income negative $97 million, down sequentially from $228 million, and up from negative $172 million year-earlier. EPS negative $0.77, down sequentially from $1.83, and up from negative $1.39 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.43 billion, up sequentially from $2.40 billion. Convertible Debt $1.0 billion.

See also Alnylam pipeline.

Operating expenses of $556 million consisted of: $72 million for cost of goods sold; $272 million for research and development; and $198 million for general and administrative expense; $14 million cost of collaboration. Operating income negative $116 million. Interest & other expense was $20 million. $0 million income tax.

Q&A Selective Summary:

If succeed in Helios-B monotherapy success only (Amvutra)? We are confident in both classes, overall and monotherapy. Outcome focussed study. Adjusted after FDA consultations.

Why now with those changes? Just learnings from Apollo-B study, including data from more than 12 months. It took time to consult with the FDA, and to get as much data as we could. In the long-term data from Apollo-B we were encouraged in both populations, including sizeable effects in the monotherapy.

Secondary endpoints? Streamlined them to the most clinically important and differentiated. But will look at the endpoints that were removed. 6 minute walk test is amply powered and differentiates from tafamadis/stabilizers. Then all cause mortality. NYHA class, earlier data showed that class of drugs can show some benefit. We want to focus on the strongest outcomes.

Data to differentiate Amvutra from other therapies? Will share P values on endpoints, quantitative safety, data on subgroups. Physicians believe 75% of patients on stabilizers continue to progress.

Statistical penalties for changes? Just a change in statistical analysis, so no penalty.

Insisted on confidence in the study design and analysis.

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