Analyst Conference Summary

biotechnology

conference date: April 28, 2022 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2022 (first quarter, Q1)

Forward-looking statements

Overview: Continued rapid revenue growth, still deep in the red.

Basic data (GAAP):

Revenue was $213 million, down % sequentially from $258 million, and up 20% from $178 million year-earlier.

Net income was negative $240 million, up sequentially from negative $258 million, and down from negative $200 million year-earlier.

Diluted EPS was negative $2.00, up sequentially from negative $2.16, and down from negative $1.71 year-earlier.

Guidance:

Reduced slightly full year 2022 guidance to $870 to $930 million net product revenue, $175 to $225 collaboration and royalty revenue. Non-GAAP combinded R&D and SG&A expense reduced slightly to $1,390 to $1,450 million.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "We continue to make progress with our pipeline programs, including our TTR franchise, for which we reported positive 18-month results from the HELIOS-A study of vutrisiran, and we remain on track for topline results from the APOLLO-B study of patisiran in the middle of this year. With our earlier stage programs, we’re excited to have initiated our first clinical study of an Alnylam investigational RNAi therapeutic targeting a CNS disorder with the start of our Phase 1 study for ALN-APP in patients with early-onset Alzheimer’s disease, and are announcing today that the Phase 1 study of ALN-XDH in patients with gout also has been initiated." Goal is to bring 2 to 4 new products to clinical trials each year.

At the end of 2021 John Maraganore retired from the CEO position.

Vutrisiran (ALN-TTRsc02) NDA submitted to FDA in Q2 for hATTR-PN, PDUFA April 14, 2022. But on April 4 the FDA extended the timeline, with a new PDUFA of July 14, 2022, to add a packaging facility. Still expects to launch mid-year. Reported positive topline results from the 18-month data for HELIOS-A Phase 3 study in hATTR amyloidosis patients with polyneuropathy in Q3 2021. Completed enrollment the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy in Q3 2021, with full results due in 2024.

In Q1 2022 Alnylam filed patent infringement suits against Pfizer and Moderna re use of biodegradable cationic lipids for delivery.

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 late 2022.

Vir Biotechnology reported ALN-HBV02 (VIR-2218) had good Phase 2 results for HBV in Q2 2022.

Non-GAAP net income negative $180 million, up sequentially from negative $203 million, and up from negative $192 million year-earlier. EPS negative $1.49, up sequentially from negative $1.69, and up from negative $1.64 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.24 billion, down sequentially from $2.44 billion.

Givlaari (Givosiran) for AHP (acute hepatic porphyria) continued launch in Spain and Italy; expects UK launch in 2022. 400 patients worldwide.

Oxlumo (lumasiran) is working on EU approval and reimbursement. Submitted regulatory applications to the U.S. FDA and European Medicines Agency to support label expansion for Oxlumo for the treatment of advanced PH1. A Phase 2 study of patients with recurrent kidney stone disease has been initiated. 160 patients end of Q1 2022.

Onpattro (patisiran) is making progress ex-US with new launches or getting reimbursement. At end of Q1, 2022, over 2,200 patients were on therapy worldwide. Completed enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy in Q2 2021, and data expected in mid 2022. There were over 2,000 patients on commercial Onpattro as of year end 2021.

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. Alnylam recognized a $25 million milestone from Novartis related to the U.S. FDA approval of Leqvio in December 2021.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study reported positive results in Q4 2021. Sanofi Genzyme is a partner in the program. In Q4 2020 received Fast Track Designation from the FDA. Filing date moved back to 2024 to allow for a lower dose cohort.

In mid-2022 Novartis plans to report results from its ORION-3 Phase 2 study of inclisiran for heterozygous familial hypercholesterolemia or pre-existing atherosclerotic cardiovascular disease on background statin ezetimibe therapy.

Cemdisiran for complement mediated diseases expects topline Phase 2 IgA nephropathy results in early 2022. Partnered with Regeneron, which initated Phase 3 studies for myasthenia gravis and paroxysmal nocturnal hemoglobinuria in Q4 2021.

The Ikaria platform for siRNA is being developed aimed at annual dosing.

ALN-HSD expects to report topline Phase 1 results for NASH in mid 2022.

In Q2 ALN-TTRsc04 was introduced for a potential annual dosing regimen, with IND filing planned for end of 2022.

See also Alnylam pipeline.

Operating expenses of $360 million consisted of: $23 million for cost of goods sold; $170 million for research and development; and $154 million for general and administrative expense; $12 million cost of collaboration. Operating loss $147 million. Interest & other expense was $93 million. $1 million income tax.

Q&A Summary:

Onpattro prescriber changes? Please with growth. Some softness in January and February due to omnicron.

Vutrisiran sales expectations for 2022? We see vutrisiran is a potential driver for franchise growth. Hit guidance because of one less quarter on the market in 2022.

In the Vutrisiran delay, the FDA did not ask for additional data or information, it was just due to timing requirements because of the use of the packaging facility. This was a voluntary decision by us, based on our belief it would keep the timeline short.

We were planning to enroll patients in the Ukraine for zilebesiran, so we have had to expand our geographical targets. We still expect enrollment for Kardia-1 to complete in early 2023.

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