Analyst Conference Summary

biotechnology

conference date: October 31, 2024 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2024 (third quarter, Q3)

Forward-looking statements

Overview: Continued rapid y/y product revenue growth, but still losing money.

Basic data (GAAP):

Revenue was $501 million, down 24% sequentially from $659 million, and down 33% from $751 million year-earlier.

Net income was negative $112 million, down sequentially from negative $17 million, and down from $148 million year-earlier.

Diluted EPS was negative $0.87, down sequentially from negative $0.13, and down from $1.15 year-earlier.

Guidance:

reiterated

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "Alnylam continued to deliver robust results commercially and clinically in the third quarter. We achieved 34% year-over-year growth, with global net product revenues of $420 million, primarily driven by our TTR business. Further with TTR, we continued to share data for vutrisiran in ATTR-CM, with detailed HELIOS-B results presented at the European Society of Cardiology Congress, and supportive echocardiographic and biomarker data presented at the Heart Failure Society of America Annual Scientific Meeting 2024. We are pleased to have submitted regulatory filings in the U.S. and EU, keeping us on track for a potential U.S. launch in early 2025 and other markets later in the year." Still expects to achieve non-GAAP profitability by end of 2025.

Today announced positive results from Phase 1 study of mivelsiran for Alheaimer'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. Will analyse both combined with tafamadis and as monotherapy. Met primary and secondary endpoints. Gave full results on August 30 at ESC. There are perhaps 300,000 potential global patients, with about 80% untreated. Submitted regulatory submissions (FDA and EMA)in Q3 2024 with accelerated review in the US. Expects to launch in U.S. in early 2025.

Zilebesirin for hypertension reported positive Phase 2 topline results in Q2 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 Q2 2024 initiated dosing Phase 1 of mivelsiran (ALN-APP) in cerebral amyloid angiopathy (CAA) patients. A Phase 2 study for cerebral amyloid angiopathy is planned. Regeneron opted out, so Alnylam now has full global rights.

In Q3 started a Phase 1 study of ALN-HTT02 for Huntington's disease.

Suspended further work on ALN-KHK for type 2 diabetes.

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 $57 million, down from $427 million year-earlier. Royalty revenue was $23 million, up from $10 million year-earlier.

Non-GAAP net income negative $64 million, down sequentially from $74 million, and down from $229 million year-earlier. EPS negative $0.50, down sequentially from $0.58, and down from $1.83 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.77 billion, up sequentially from $2.6 billion. Convertible Debt $1.0 billion.

See also Alnylam pipeline.

Operating expenses of $578 million consisted of: $82 million for cost of goods sold; $271 million for research and development; and $221 million for general and administrative expense; $4 million cost of collaboration. Operating loss $77 million. Interest & other expense was $32 million. $3 million income tax.

Q&A Selective Summary:

RNAi to adipose and muscle tissue? We have highlighted them in our 225 strategy. On track to advance those. Will do update at an R and D day next year.

HTT02 data, goals? Huntington's is our third CNS program. Looking for sustained knockdown and well-tolerated. Preclinical studies looked good. Unique targetting approach, including exon1 fragment. Study is starting up.

Polyneuropathy competition? Despite a new therapy on the market for a year now, our sales continue to rise. There is a high unmet medical need. We have a strong salesforce and a compelling profile for Amvuttra.

Not planning on reducing the price if Amvuttra is approved for cardiomyopathy. Patients in polyneuropathy usually have no copay burden. Will discuss more when we know the PDUFA date.

If patients are progressing on a stabilizer, they will eventually switch them or use a combination therapy. Even if tafamidis becomes generic.

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