Analyst Conference Summary

biotechnology

conference date: April 29, 2021 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2021 (first quarter, Q1)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $136 million, down 17% sequentially from $164 million, but up 89% from $72 million year-earlier.

Net income was negative $186 million, up sequentially from negative $244 million, and up from negative $210 million year-earlier.

Diluted EPS was negative $1.71, up sequentially from negative $2.09, and up from negative $1.62 year-earlier.

Guidance:

Reiterated for 2021. Includes revenue betwee $760 and $860 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "we achieved steady and continued growth for Onpattro with approximately 13% quarterly growth and we observed strong initial demand for Oxlumo in its first full quarter of launch. We also presented positive results from the HELIOS-A Phase 3 study of vutrisiran, and with our recent NDA filing we are one step closer to potentially bringing this transformative medicine to patients. Given the strong pace of enrollment in the HELIOS-B Phase 3 study of vutrisiran, we are announcing today that we expect to complete study enrollment in late 2021, earlier than previously anticipated. Early in the quarter we launched our new five-year vision, Alnylam P5x25, marking our strategy for a planned transition to a top five biotech company in market capitalization by the end of 2025. With Alnylam P5x25, we aim to deliver transformative medicines for rare and prevalent diseases to patients around the world, while advancing a robust and high-yielding pipeline of first and/or best-in-class clinical programs from our organic product engine, while delivering strong topline growth and profitability within the period."

Non-GAAP net income negative $192 million, down sequentially from negative $186 million, and down from negative $172 million year-earlier. EPS negative $1.64, down sequentially from negative $1.60, and down from negative $1.52 year-earlier.

Cash and equivalents balance at the end of the quarter was $1.71 billion, down sequentially from $1.91 billion.

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) continued enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy, with plans to complete enrollment in early 2021 and data 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) reported positive topline results from the HELIOS-A Phase 3 study in hATTR amyloidosis patients with polyneuropathy in Q1 2021, and plans to file an NDA with the FDA in early 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 for Leqvio from the European Commission in December 2020. But the FDA issued a Complete Response Letter.

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 Q4 2020 began Phase 1 dosing of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of non-alcoholic steatohepatitis (NASH), in collaboration with Regeneron.

In May 2020 announced positive initial topline results from the ongoing Phase 1 study (N=48) of ALN-AGT in hypertension. On November 13, 2020 released interim Phase 1 results at AHA.

See also Alnylam pipeline.

Operating expenses of $364 million consisted of: $23 million for cost of goods sold; $186 million for research and development; and $147 million for general and administrative expense. Operating loss $186 million. Interest & other expense was $13 million. $1 million income tax benefit.

Q&A Summary:

Why do you think Helios B is enrolling faster than anticipated? Community enthusiasm for a once-quarterly subq therapy. Outstanding clinical operations team. Planning an interim analysis. Has a 30 month endpoint.

Onpattro with stabilizers? We are indicated for polyneuropathy in the U.S. We see about 20% combinate use. In EU and Japan we are indicated with a stabilizer, we are seeing switches from stabilizers.

Givlaari growth trends? Impact on patients is profound. Growth this year to be driven by geographic expansion in EU.

Value pricing program? Results have been good. Payers see Alnylam as proactive and open-minded, allows for discussion of value. So no payer headwinds, broad access to market. We anticipate no long-term negative impact on our revenue.

OpenIcon Alnylam page

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: