Analyst Conference Summary

biotechnology

conference date: February 23, 2023 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2022 (fourth quarter, Q4)

Forward-looking statements

Overview: Revenue continues to expand rapidly, but still deep in the red.

Basic data (GAAP):

Revenue was $335 million, up 27% sequentially from $264 million, and up 29% from $259 million year-earlier.

Net income was negative $207 million, up sequentially from negative $406 million, and up from negative $258 million year-earlier.

Diluted EPS was negative $1.68, up sequentially from negative $3.32, and up from negative $2.16 year-earlier.

Guidance:

Full year 2023 net product revenue expected between $1.20 and $1.285 billion. Collaboration and royalty revenue $100 to $175 million. GAAP op ex $1.79 to $1.89 billion. Non-GAAP op ex $1.575 to $1.650 billion.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2022 was an excellent year at Alnylam . . . Looking ahead to 2023, we are excited for a number of important milestones across the pipeline, with 10 clinical readouts from proprietary and partner-led programs, including first-in-human results for an RNAi therapeutic in the CNS from our Phase 1 study of ALN-APP, as well as Phase 2 results from the Kardia studies of zilebesiran. With the totality of this progress, we believe we are on track to achieve our Alnylam P5x25 goals and position Alnylam as a top-tier biotech company poised to deliver sustainable innovation." Expects 10 data readouts in 2023. Believes cash is sufficient to reach cash-flow break even.

Onpattro (patisiran) Apollo-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy topline results were positive (Q3 2022) and sNDA filed. PDUFA date October 8, 2023.

Alnylam will not proceed with regulatory filings for biannual dosing of Amvuttra, instead advancing ALN-TTRsc04, which could work with annual dosing. Began dosing in the Phase 1 trial in Q1 2023.

In Q4 2022 Alnylam Completed enrollment in the KARDIA-1 Phase 2 monotherapy study of zilebesiran in patients with mild-to-moderate hypertension.

In Q1 2023, in collaboration with Regeneron, ALN-PNP began a Phase 1 study for NASH.

Non-GAAP net income negative $172 million, up sequentially from negative $193 million, and up from negative $208 million year-earlier. EPS negative $1.39, up sequentially from negative $1.58, and up from negative $1.39 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.19 billion, down sequentially from $2.27 billion.

In Q2 2022 Alnylam started a Phase 1 study of ALN-XDN for gout and a Phase 1 trial of ALN-APP for Alzheimer's. Could have top line results in 2023.

In Q3 2022, based on the successful outcome of the ILLUMINATE-C study in children and adults with advanced PH1, received approval from the FDA of an sNDA for Oxlumo, expanding the indication for the treatment of PH1 to lower urinary oxalate and plasma oxalate levels in pediatric and adult patients, also received approval from the EMA of a Type II variation to include the ILLUMINATE-C data in the label.

In Q3 2022 Alnylam filed a Clinical Trial Application for ALN-TTRsc04, the first clinical program from the IKARIA platform, aimed at achieving an annual dosing regimen with highly potent and reversible effects of TTR lowering.

In Q2 2022 Cemdisiran for complement mediated diseases reported positive topline Phase 2 results for immunoglobin A nephropathy. Working with Regeneron to finalize Phase 3 trial plans.

ALN-APP Phase 1 topline results for early-onset Alzheimer's are expected in early 2023.

Leqvio (inclisiran) royalties from Novartis could rise after FDA approval for the treatment of adults with clinical atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia who require additional lowering of LDL-C.

ALN-HSD reported preliminary Phase 1 results for NASH in Q3 2022.

See also Alnylam pipeline.

Operating expenses of $524 million consisted of: $46 million for cost of goods sold; $262 million for research and development; and 210$ million for general and administrative expense; $5 million cost of collaboration. Operating loss $189 million. Interest & other expense was $18 million. $0 million income tax.

Q&A Selective Summary:

Advisory Committee topics for Onpattro? The FDA has not identified any issues. We do not know if there will be an AC meeting. We will be giving a 120 day safety update.

Will lack of biannual dose of Amvuttra effects? Amvuttra has been a game changer for patients, launch is going well. It was a strategic decision, we are concentrating on the quarterly dose, then the one-year dosing would be a real step forward. We will get Phase 1 data later this year on the newer therapy.

ALN-APP details? We are in single ascending dose phase. Primary enpoints are safety and tolerability, but we will be looking for knockdown of amyloid precurser A and B targets.

The Onpattro label expansion would not likely have a significant impact in 2023, more in 2024.

Amvuttra in polyneuropathy J code in January? Pleased we received it, but we already had a generic J code, so we are not expecting a change. But growth is accelerating from the product profile and our sales efforts with prescribers.

Helios A study Death events? Overall safety and adverse events were balanced between the arms. Deaths were not related to the therapy.

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