Analyst Conference Summary

biotechnology

conference date: May 2, 2024 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2024 (first quarter, Q1)

Forward-looking statements

Overview: Remarkable revenue growth while pipeline is strong. Net losses decreasing.

Basic data (GAAP):

Revenue was $494 million, up 12% sequentially from $440 million, and up 55% from $319 million year-earlier.

Net income was negative $66 million, down sequentially from $138 million, and up from negative $174 million year-earlier.

Diluted EPS was negative $0.52, down sequentially from $1.10, and up from negative $1.40 year-earlier.

Guidance:

Reiterated full 2024: $1.4 to $1.5 billion product revenue, $325 million collaboration and royalty.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "We delivered $365 million in global net product revenues, representing 32% year-over-year growth for our four wholly owned products. We also made great progress with our pipeline, particularly with the zilebesiran program in hypertension, for which we reported positive results from the KARDIA-2 Phase 2 study, and initiated the KARDIA-3 Phase 2 study. Looking ahead, we are eagerly awaiting topline results from the HELIOS-B Phase 3 study of vutrisiran in late June or early July, potentially expanding our leadership in ATTR amyloidosis. Additionally, we look forward to early-stage developments with mivelsiran (formerly ALN-APP), where we expect to start a Phase 2 study in cerebral amyloid angiopathy in the coming months. These achievements position us well to deliver on our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial results." Still expects to achieve non-GAAP profitability by end of 2025.

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 Q1 2024, when added to standard of care to control hypertension. Will partner with Roche for global development and distribution. In Q1 2024 initiated a Phase 2 study in adult patients with high cardiovascular risk and uncontrolled hypertension despite treatment with two to four standard of care hypertension medications.

In Q1 received FDA approval for Part B of the Phase 1 study of mivelsiran for early-onset Alzheimer's. A Phase 2 study for cerebral amyloid angiopathy is planned for mid 2024.

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 $119 million, up from $36 million year-earlier. Royalty revenue was $11 million, up from $7 million year-earlier.

Non-GAAP net income negative $21 million, up sequentially from negative $97 million, and up from negative $132 million year-earlier. EPS negative $0.16, up sequentially from negative $0.77, and up from negative $1.06 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.37 billion, down sequentially from $2.43 billion. Convertible Debt $ billion.

See also Alnylam pipeline.

Operating expenses of $538 million consisted of: $55 million for cost of goods sold; $261 million for research and development; and $211 million for general and administrative expense; $11 million cost of collaboration. Operating income negative $43 million. Interest & other expense was $20 million. $2 million income tax.

Q&A Selective Summary:

Relevant improvement over control, to compare competitors? Helios B is an outcome study for cardiomyopathy. Regulators, payors, physicians want to see efficacy. We made changes to our statistical plan. Outcomes will be the important result of the study, and other results will differentiate our drug, vutrisiran (Amvuttra). Topline results in June or July.

Combination with tafamadis with vutrisiran, or only monotherapy for cardiomyopathy? 80% of patients remain undiagnosed, it progresses rapidly and is irreversible. Amvuttra is clearly differntiated from tafamadis. We believe Amvuttra will become the first line agent. Patients treated with tafamadis continue to progress, so physicians are looking for an alternative.

Helios B physicians wanting more that just some improvement? We were able to rapidly enroll patients who were already on tafamadis. We will let the data speak for themselves.

ATTR mixed phenotype % in the real world? Polyneuropathy and Cardiomyopathy are treated as distinct, but the same cause and many patients have both. Patients with multiple manifestations will want the most effective treatment.

Amvuttro pricing? Too early, but will bear in mind patient access needs.

Amvuttro has competed very well with tafamadis in Europe. It is selling well in the U.S., where we only promote it for polyneuropathy, but it is sometimes prescribed more broadly. Hence the rapid revenue growth rate.

ALN-APP study? Data shared last year was exciting. Part B is ongoing, should have more data going forward. At year end plan to start an Alzheimer's study.

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