Analyst Conference Summary

biotechnology

conference date: July 28, 2022 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2022 (second quarter, Q2)

Forward-looking statements

Overview: Slow revenue growth masks rapid product revenue growth; still deep in the red.

Basic data (GAAP):

Revenue was $225 million, up 6% sequentially from $213 million, and up 2% from $221 million year-earlier.

Net income was negative $277 million, down sequentially from negative $240 million, and down from negative $190 million year-earlier.

Diluted EPS was negative $2.29, down sequentially from negative $2.00, and down from negative $1.61 year-earlier.

Guidance:

Reiterated prior.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "We are pleased with our second quarter results which delivered strong growth in both patient demand and product revenues. A notable achievement in the quarter was the recent approval of Amvuttra, which becomes our fifth RNAi therapeutic approved in under four years, and marks our continued progress in building a multi-product TTR franchise. We are excited to have initiated the U.S. launch and look forward to potential additional global approvals and subsequent rollout. We are also eager for a number of important milestones over the rest of the year, including topline results from the Apollo-B Phase 3 study of patisiran in ATTR amyloidosis with cardiomyopathy patients, which we announced today are expected within the next three weeks. Additionally, we plan to continue to advance our robust pipeline, with clinical data expected across several prevalent disease programs in NASH, HBV, gout, and early-onset Alzheimer's disease with ALN-APP, our first investigational RNAi therapeutic targeting a CNS disorder." Product sales growth was 14% y/y. A future growth driver is expanding from rare diseases into more common diseases. Foreign exchange rates presented a headwind.

Leqvio (inclisiran) launch in the U.S. and other markets is ongoing, with focus on patient onboarding, removing access hurdles and enhancing medical education.

In Q2 2022 the FDA approved Amvuttra (Vutrisiran or ALN-TTRsc02) for polyneuropathy of hATTR amyloidosis. Expects to launch in EU in 2022, already has a positive opinion from the CHMP. Will now seek to expand the label. Helios-A Phase 3 study reported positive topline results for polyneuropathy in April 2022. Also current Phase 3 Helios-B study for cardiomyopathy is fully enrolled, readout in early 2024.

Completed enrollment in the Onpattro (patisiran) Apollo-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy topline results are expected in August 2022.

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.

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.

Non-GAAP net income negative $246 million, down sequentially from negative $180 million, and down from negative $153 million year-earlier. EPS negative $2.03, down sequentially from negative $1.49, and down from negative $1.30 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.11 billion, down sequentially from $2.24 billion.

ALN-APP Phase 1 topline results are expected in late 2022.

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. In Q2 2022 presented new data from Phase 3 studies for PH1. 200 patients end of Q2 2022.

Onpattro (patisiran) is making progress ex-US with new launches or getting reimbursement.

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.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS-PPX study reported positive results in Q2 2022. 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.

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 $ million consisted of: $34 million for cost of goods sold; $205 million for research and development; and $180 million for general and administrative expense; $7 million cost of collaboration. Operating loss $192 million. Interest & other expense was $83 million. $3 million income tax.

Q&A Selective Summary:

Vitrisiran initial prescription sources? Initial launch signs are encouraging. It has just been 5 weeks, we have received about 130 start forms, about 1/3 are new patients. A Part B product, so similar dynamic as Onpattro. We also have new prescribers for it.

Pricing for Patisiran, including when cardiomyopathy added to label? Too soon to talk about cardiomyopathy pricing.

Pfizer's Tafamadis, switching? In U.S. we are indicated for polyneuropathy, Tafamadis is indicated for cardiomyopathy, so no switching. Switching is in ex-US, where both are approved for polyneuropathy, and Onpattro is seen as more effective.

New patients, less severe? Mixed phenotype? Bolus of warehoused patients for Amvuttra? Too little data yet. Seems some patients are younger, seeing a broad range switch or start, part of it may be warehousing. But Onpattro had good growth in Q1, so they were not waiting for Amvuttra.

Start form to on drug timeline for Amvuttra? Building on our robust commercial operation. Subcut every 3 months is attractive. Working with payers, deliveries, formularies. Not seeing any headwind. No specific timeline yet.

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