Analyst Conference Summary

biotechnology

conference date: November 7, 2018 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2018 (third quarter, Q3)

Forward-looking statements

Overview: Onpattro for hattr amyloidosis U.S. was approved in the quarter, but missed the concensus on first revenue. [WPM: No big deal.]

Basic data (GAAP):

Revenue was $2.0 million, plunging sequentially from $29.9 million, and from $17.1 million year-earlier.

Net income was negative $245.3 million, down sequentially from negative $163.6 million, and down from negative $122.9 million year-earlier.

Diluted EPS was negative $2.43, down sequentially from negative $1.63, and down from negative $1.34 year-earlier.

Guidance:

Reiterates its prior guidance for 2018.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "Our recent regulatory and commercial launch experiences will be leveraged for our entire portfolio including late-stage programs such as givosiran, where we will initiate a rolling NDA submission this year; lumasiran, which we have advanced into late-stage development with the recent initiation of the Illuminate-A Phase 3 study; and ALN-TTRsc02, which will enter Phase 3 later this year in the Helios-A study. We believe these accomplishments put us well on our way toward achieving our Alnylam 2020 goal of building a multi-product, global, commercial-stage company with a deep and sustainable clinical pipeline by the end of 2020."

Launched Onpattro (patisiran) in the U.S. and EU. Made value-based agreements with leading health insurers. Had been approved by the FDA in August. Will need to see several quarters of results to be able to see the longer term prescription trajectory.

Onpattro revenue was $0.5 million. Revenue from collaborators: $1.6 million from Genzyme/Sanofi.

Cash and equivalents balance at the end of the quarter was $1.22 billion, down sequentially from $1.48 billion. $30 million in long-term debt.

Non-GAAP net income negative $ million, down sequentially from negative $161.9, and down from negative $ million year-earlier. EPS negative $, down sequentially from negative $1.63, and down from negative $year-earlier.

Alnylam continued a Phase 1 trial for ALN-TTRsc02, an ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a once-quarterly subcutaneous dosing regimen. A Phase 3 trial is expected to begin in late 2018; some details were given. Believes this could be expanded to help carriers, which would greatly expand the patient pool.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. Sanofi Genzyme is a partner in the program.

Givosiran (ALN-AS1) for acute hepatic porphyrias (AHPs) Phase 3 trial completed enrollment and positive topline data was announced, with an NDA possible by the end of 2018.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial started, and topline results are expected in 2019, with an NDA in 2020. Phase 2 results reported in the quarter were positive.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study in Europe continued and updated positive data was presented.

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

Inclisiran (ALN-PCSsc), being developed by The Medicines Company, for hypercholesterolemia, had its fourth efficacy and safety review of the Phase 3 trial, with complete enrollment expected soon. Topline results expected in mid-2019. Alnylam could receive milestones and up to 20% royalties.

Cemdisiran (ALN-CC5) for aHUS (atypical hemolytic-uremic syndrome) had started a Phase 2 study, but enrollment has been slow, so cancelled to focus on an upcoming Phase 2 study in IgA nephropathy.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $256.6 million consisted of: $0.2 million for cost of goods sold; $139.9 million for research and development; and $116.5 million for general and administrative expense. Interest & other income was $9.7 million. Income taxes $0.4 million.

Alnylam hopes to begin commercial sales of Fitusiran in 2019, and Givosiran in 2020.

R&D investor conference will be held on December 6.

Q&A:

Onpattro patients in TTR centers currently? It is a rare and misdiagnosed disease. Many are in clinical studies. Typically visits are every six months. We are seeing an uptick of starts in centers of excellence.

Europe launch? Started in Germany, encouraging so far. Other countries require assessments before starting. Not sharing what we believe will be the order of additional countries after Germany, for competitive reasons.

Onpattro pricing? Early on, could change. Depends on payer mix, government discount. $345,000 is the right number to use. We have had tremendous discussions with payers.

EAP global enrollment? Over 200 EAP patients globally, most in U.S. 60% of start forms are from EAP group as of September 30th.

Givosiran filing process? After positive interim analysis had talks with FDA, their support for rolling submission. Should have full results around March 2019, which means no advantage to filing with just an interim analysis.

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