Analyst Conference Summary

biotechnology

conference date: August 3, 2021 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2021 (second quarter, Q2)

Forward-looking statements

Overview: Strong y/y revenue gains, but far from profitable.

Basic data (GAAP):

Revenue was $221 million, up 63% sequentially from $136 million, and up 113% from $104 million year-earlier.

Net income was negative $190 million, down sequentially from negative $186 million, and down from negative $178 million year-earlier.

Diluted EPS was negative $1.61, down sequentially from negative $1.71, and down from negative $1.56 year-earlier.

Guidance:

Increased net product revenue guidance for 2021 to $640 to $665 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: " The second quarter also marked significant progress for our TTR franchise overall, including NDA submission and acceptance for vutrisiran based on positive HELIOS-A results, initiation of a biannual dosing study for vutrisiran, completion of enrollment in the APOLLO-B Phase 3 trial and imminent enrollment completion for HELIOS-B, and introduction of ALN-TTRsc04 for a potential annual dosing regimen. Together, these efforts highlight our commitment to ATTR amyloidosis for years to come. With sights set on our Alnylam P5x25 five-year vision announced earlier this year, we believe we are well positioned and on our way to achieving these ambitious goals."

Alnylam is collaboring with PeptiDream to discover and develop peptide-siRNA conjugates.

Non-GAAP net income negative $153 million, up sequentially from negative $192 million, and up from negative $191 million year-earlier. EPS negative $1.30, up sequentially from negative $1.64, and up from negative $1.67 year-earlier.

Cash and equivalents balance at the end of the quarter was $1.90 billion, up sequentially from $1.71 billion. Cash increased from a drawdown of the credit facility and exercise of employee stock options.

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

Oxlumo started sales in the U.S. following FDA approval in November 2020. Working on EU approval and reimbursement.

Onpattro (patisiran) is making progress ex-US with new launches or getting reimbursement; got approval in Taiwan in Q4 2020.

Onpattro (patisiran) completed enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy in Q2 2021, and data expected in mid 2022.

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. Initial data expected in late 2021, NDA in H2 2022.

Vutrisiran (ALN-TTRsc02) NDA submitte to FDA in Q2 for hATTR-PN, PDUFA April 14, 2022. Reported positive topline results from the HELIOS-A Phase 3 study in hATTR amyloidosis patients with polyneuropathy in Q1 2021. Continued the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy, with completion in enrollment now expected in 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.

Inclisiran, now Leqvio, for hypercholesterolemia, licensed to Novartis, received marketing authorization from the European Commission in December 2020. But the FDA issued a Complete Response Letter. In Q2 2021 resubmitted, PDUFA date now January 1, 2022.

In Q2 Zilebesiran (ALN-AGT) started a Phase 2 trial for mild to moderate hypertension.

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:

Bigger programs like hypertension, investment v. profitability, partners? We are optimistic about RNA therapies for more prevalent diseases. There is no reason we cannot advance the assets on our own. We do not need funding. But we do want to navigate towards a profitable business.

Cardio dosing regimen? Zilebesiran is like inclisiran in the general dosing and biomarker levels. Zilebesiran will have 2 Phase 2 studies. One as a monotherapy. Second combines with various other anti-hypertension medications. No guidance on completion dates. Enrollment should go quickly. Looking at 3 and 6 monthly dosing regimens.

PeptiDream targets beyond CNS? We are interested in a range of organs. RNAi can work in any cell in the body. Different tissues have different validated targets, including new tissue types. This will help us maintain our leadership in RNAi.

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