Analyst Conference Summary

biotechnology

conference date: October 28, 2021 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2021 (third quarter, Q2)

Forward-looking statements

Overview: Revenue up y/y despite sequential drop. Still deep in the red while developing pipeline. Missed street estimates for revenue and earnings.

Basic data (GAAP):

Revenue was $188 million, down 15% sequentially from $221 million, and up 49% from $126 million year-earlier.

Net income was negative $204 million, down sequentially from negative $190 million, and up from negative $253 million year-earlier.

Diluted EPS was negative $1.72, down sequentially from negative $1.61, and up from negative $2.18 year-earlier.

Guidance:

Reiterated prior.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "Our third quarter commercial performance was highlighted by strength from Onpattro, and continued execution on the on-going launches of Givlaari and Oxlumo. Another notable achievement was our announcement of positive topline 18-month results from the Helios-A Phase 3 study of vutrisiran in patients with hATTR amyloidosis with polyneuropathy, including improvements in exploratory and cardiac amyloid endpoints, which we believe are encouraging hypothesis-generating data ahead of upcoming readouts from the Phase 3 studies of patisiran and vutrisiran, respectively. We also made significant progress with many of our other programs, and we remain focused on executing on our 'Alnylam P5x25' vision, through which we intend to deliver transformative medicines to patients around the world for rare and prevalent diseases, advancing a robust and high-yielding pipeline of first and/or best-in-class product candidates from our organic product engine, while delivering exceptional financial performance. Lastly, with the announcement this morning of my planned transition from Alnylam at year end, I'd like to emphasize how thrilled I am about the future of Alnylam under Yvonne's upcoming stewardship." Seeing encouraging signs of healthcare systems reopening.

At the end of 2021 Yvonne Greenstreet will become CEO; she is currently President.

Non-GAAP net income negative $180 million, down sequentially from negative $153 million, and up from negative $184 million year-earlier. EPS negative $1.51, down sequentially from negative $1.30, and up from negative $1.58 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.33 billion, up sequentially from $1.90 billion, despite cash use in operations. Received $500 million from Blackstone in September 2021.

Givlaari (Givosiran) for AHP (actue hepatic porphyria) continued launch in Germany, Italy, France. 300 patients worldwide.

Oxlumo working on EU approval and reimbursement.

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 1,875 patients on commercial Onpattro as of 9/30/2021.

Leqvio (inclisiran) royalties from Novartis could rise after FDA PDUFA date of January 1, 2022. Approved in 45 nations.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. 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.

Oxlumo (Lumasiran) for PH1 (primary hyperoxaluria type 1) was approved by the FDA on November 4, 2020. In Q4 2020 presented positive complete results from ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients less than six years of age, including infants, with preserved renal function.

Cemdisiran for complement mediated diseases expects topline IgA nephropathy results in early 2022. Partnered with Regeneron.

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.

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 announced in Q2 2021 plans to advance ALN-XDH to a filing for gout in late 2021.

See also Alnylam pipeline.

Operating expenses of $367 million consisted of: $30 million for cost of goods sold; $183 million for research and development; and $145 million for general and administrative expense. Operating loss $146 million. Interest & other expense was $33 million. $1 million income tax.

Q&A Summary:

Helios A data? Vitrisiran data strengthens belief in the drug, exploratory data in encouraging. So hope Helios B will also generate good data.

ATTR franchise, 30% cardio subpopulation? Helios A full data will be presented at a coming conference.

John Maraganore will stay on the scientific advisory board, will not be on the Board. He will be an advisor to the new CEO during Q1 2022. Will also remain a major shareholder.

ALN-APP with Regeneron development? Extra-hepatic therapies are the next big thing. The CPA (IND) for ALN-APP for Alzheimer's will be filed by the end of this year, would be a milestone for the extra-hepatic and neurodegenerative fields. Excited about CNS delivery system used, which resulted in potent knockdown in animal models.

Believes the new Vutrisiran opportunity is much larger than the current patient base.

Being a quarterly subcutaneous injection, it should help with the compliance issue. Keep in mind patients on Tafamidis for ATTR-CM tend to continue to decline. Despite lack of head-to-head studies, some scan data indicates Onpatro and Vutrisiran provide improvements over Tafamadis. Should see data with Apollo B.

Future strategy? Continue to execute on what we have put in place. Cardiac for the next few years. Zilebesiran for hypertension is a big opportunity.

The safety profile of Vitrusiran is very good so far, both in itself and by comparison to competitors.

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