Analyst Conference Summary

biotechnology

conference date: November 5, 2020 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2020 (third quarter, Q3)

Forward-looking statements

Overview: Continued strong revenue ramp, heavy losses.

Basic data (GAAP):

Revenue was $126 million, up 21% sequentially from $104 million, and up % from $70 million year-earlier.

Net income was negative $253 million, down sequentially from negative $179.2 million, and down from negative $208 million year-earlier.

Diluted EPS was negative $2.18, down sequentially from negative $1.56, and down from negative $1.92 year-earlier.

Guidance:

Increased 2020 guidance. Onpattro revenue now expected between $295 and $310 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "We are extremely pleased with the performance of Onpattro and Givlaari in the third quarter, reflecting strong commercial execution and improving market conditions following pandemic phase experienced in the second quarter. We are also excited about the recent positive CHMP opinions for Oxlumo and Leqvio. We believe that this positions Alnylam to potentially exit 2020 with four revenue-generating products bolstering our sustained growth. In addition, our robust late-stage pipeline of investigational medicines continued to advance in the quarter. Notably, we achieved positive results from the ILLUMINATE-B study -- demonstrating efficacy and safety for our investigational RNAi therapeutic lumasiran in children under the age of six, including infants."

Givlaari (Givosiran) for AHP (actue hepatic porphyria) revenue in Q2 2020 was $16.7 million, up sequentially from $11.0 million. Over 150 patients on commercial drug. No payer headwinds to date. Added approval in Canada and NDA in Japan. Achieved reimbursement in Germany and France.

Revenue from collaborators: $26.6 million from Genzyme/Sanofi and Vir.

Onpattro (patisiran) revenue was $82.5 million, up 24% sequentially from $66.5 million and up from $46.1 million year-earlier. Over 1150 patients on therapy. Making progress ex-US with new launches or getting reimbursement. On August 6 Alnylam announced that it achieved agreement on pricing and reimbursement in France, completing patient access for Onpattro in all major European markets in under 2 years following approval. Also approved in Israel in Q3 2020.

Cash and equivalents balance at the end of the quarter was $1.83 billion, down sequentially from $1.95 billion. No debt, but $1.0 billion liability related to sales of future royalties.

Non-GAAP net income negative $184 million, up sequentially from negative $191 million, and down from negative $163 million year-earlier. EPS negative $1.58, up sequentially from negative $1.67, and down from negative $1.50 year-earlier.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. Additional Phase 2 data was presented in Q2 2019. Sanofi Genzyme is a partner in the program.

Vutrisiran (ALN-TTRsc02) subcutaneously administered ATTR treatement completed enrollment of the HELIOS-A Phase 3 study. Continued the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy.

Lumasiran for PH1 (primary hyperoxaluria type 1) in Q3 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. Phase 3 trial had reported positive results from the ILLUMINATE-A at ERA-EDTA, June 7, 2020. Filed an NDA with the FDA, received an action date of December 3, 2020.

Inclisiran for hypercholesterolemia moved over to Novartis, which acquired The Medicines Company in January 2020. 2 Phase 3 results published in New England Journal of Medicine. NDA was submitted to the FDA and the EU. Received a positive EU CHMP opinion in Q3 2020.

In Q2 2020 filed a Clinical Trial Application for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in collaboration with Regeneron.

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.

Plans to file a Clinical Trial Application for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of non-alcoholic steatohepatitis (NASH), in collaboration with Regeneron, by mid-2020.

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 will release interim Phase 1 results at AHA.

See also Alnylam pipeline.

Operating expenses of $351 million consisted of: $22 million for cost of goods sold; $161 million for research and development; and $167 million for general and administrative expense. Operating loss $225 million. Interest & other expense was $27 million. $1 million income tax.

Q&A Summary:

Onpattro Q4 inventory, sales drivers? Q3 saw both net new patient demand and return of patients after pandemic. In Q4 Onpattro growth will be from new patients. Inventory is normal.

Lumasiran patients in Extended Access Program? Have some, if approved will transition to commercial product.

Helios-A study? Excited about patiseran. Once quarterly dosing for HHTTR. Should have topline data early next year: primary, secondary, and safety. Cardiomyopathy in Helios B readthrough depends on proportion of patients in the A trial.

AAT program? We have advanced a molecule into development, now partnering with Dicerna. Positioned to advance quite nicely. Dicerna will pay for the program, we have an option in after Phase 3 for rest of world. This addresses just the liver manifestations of the disease.

Fitusiran in evolving market? Hemophilia A or B, in Phase 3, led by Sanofi, they would release data, decide to advance or not. There is still an unmet need for hemophilia. We would get 15% go 20% royalties if commercialized.

We are excited about our ability to deliver RNAi to organs outside the liver, like the CNS and lungs. ATT with Regeneron should enter Phase 1 in 2021, for Alzheimer's.

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