Analyst Conference Summary

biotechnology

conference date: February 10, 2022 @ 5:30 AM Pacific Time
for quarter ending: December 30, 2021 (fourth quarter, Q4)

Forward-looking statements

Overview: Despite rapid income growth is still generating heaving losses.

Basic data (GAAP):

Revenue was $258 million, up 37% sequentially from $188 million, and up 57% from $164 million year-earlier.

Net income was negative $258 million, down sequentially from negative $204 million, and down from negative $244 million year-earlier.

Diluted EPS was negative $2.16, down sequentially from negative $1.72, and down from negative $2.09 year-earlier.

Guidance:

For the full year 2022 Alnylam expects net product revenue between $900 million and $1.0 billion. Collaboration and royalty revenue between $175 and $225 million. Combined non-GAAP R&D and SGG expenses $1.4 to $1.5 billion.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2021 was another remarkable year at Alnylam, in which we delivered significant product revenue growth of 83% compared to 2020, achieving results at the upper end of our combined product sales guidance range driven by strong patient demand across all products in all regions. Additionally, we made great strides across our pipeline programs in development, completing enrollment in our two key Phase 3 studies in ATTR cardiomyopathy, as well as filing two NDAs or sNDAs, and advancing two programs to the clinic, including our first CNS program which is now in Phase 1." Expansion beyond rare diseases into prevalent diseases will be a growth driver going forward. Believes cash balance can last until Alnylam reaches positive cash flow.

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. 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 the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy in Q3 2021.

Non-GAAP net income negative $203 million, down sequentially from negative $180 million, and down from negative $186 million year-earlier. EPS negative $1.69, down sequentially from negative $1.51, and down from negative $1.60 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.44 billion, up sequentially from $2.33 billion.

Givlaari (Givosiran) for AHP (acute hepatic porphyria) continued launch in Spain and Italy; expects UK launch in 2022. 350 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. 140 patients end of 2021.

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

Vutrisiran (ALN-TTRsc02) NDA submitte to FDA in Q2 for hATTR-PN, PDUFA April 14, 2022. 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 the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy in Q3 2021. Now also being developed as a potential treatment for Stargardt Disease, with a Phase 3 trial expected to initiate in late 2022.

Cemdisiran for complement mediated diseases expects topline 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.

In Q2 Zilebesiran (ALN-AGT) started a Phase 2 trial for mild to moderate hypertension. A trial for it as add-on therapy is planned. In Q4 2021 updated Phase 1 interim results showing sustained SBP and DBP (blood pressure) were reported at AHA.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study continued.

ALF-F12 targeting factor XII is now in development for the treatment of hereditary angioedema and for thromboprophylaxis.

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

Alnylam ALN-XDH for gout to initiate Phase 1 in Q1 2022.

See also Alnylam pipeline.

Operating expenses of $453 million consisted of: $34 million for cost of goods sold; $229 million for research and development; and $186 million for general and administrative expense. Operating loss $453 million. Interest & other expense was $66 million. $2 million income tax benefit.

Q&A Summary:

Potential Vutrisiran label? Excited about launch, a compelling proposition for patients. Believes there are many more global patients awaiting treatment beyond what Onpatro has reached. The exciting exploratory data will not likely be on the label, which will be similar to the Onpattro label.

Vitrisiran in guidance? Onpattro has 90% adherence rate. We are assuming accelerated growth in the TTR franchise. Vitrisiran v. Onpattro will not be a zero sum game, given V is subcutaneous and is not given as frequently.

Zilebesiran goals? We have already demonstrated efficacy (20 mm drop) in early study. There is a straight path to regulatory approval, after a Phase 3 trial. The way BP is reduced is important, reducing fluctuations and restoring night BP dipping that has been lost. Also by administering once every 3 months we could get better adherence.

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