Analyst Conference Summary

biotechnology

conference date: February 7, 2019 @ 1:30 PM Pacific Time
for quarter ending: December 31, 2018 (fourth quarter, Q4)

Forward-looking statements

Overview: First Onpattro revenue, but no 2019 revenue guidance.

Basic data (GAAP):

Revenue was $21.0 million, up sequentially from $2.0 million, but down from $37.9 million year-earlier.

Net income was negative $211.3 million, up sequentially from negative $245.3 million, and down from negative $142.2 million year-earlier.

Diluted EPS was negative $2.09, up sequentially from negative $2.43, and down from negative $1.48 year-earlier.

Guidance:

Full year 2019 non-GAAP R&D expenses to be between $520 and $560 million and non-GAAP SG&A expenses to be between $390 and $420 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "We ended 2018 with cash and investments on our balance sheet of $1.13 billion, exceeding our 2018 guidance, and we're pleased to have recently strengthened our balance sheet further with our public offering in January resulting in net proceeds of approximately $382 million. . . In 2018, we saw the approval and launch of Onpattro, the world's first RNAi therapeutic, heralding the arrival of a whole new class of medicines. With our planned APOLLO-B Phase 3 study to support potential expansion of Onpattro to ATTR amyloidosis patients with cardiomyopathy and advancement of subcutaneously administered vutrisiran in the HELIOS-A Phase 3 study, we are committing further efforts to help ATTR amyloidosis patients and to support our plans for the sustained and continued growth in our ATTR amyloidosis franchise for years to come. . . In 2019, we intend to execute on six Phase 3 programs - of which two are being advanced together with partners - with Phase 3 data expected from three of these programs, and expect two NDA submissions, assuming positive results. In the meanwhile, we'll continue to benefit from a sustainable RNAi research engine that we expect will fuel future innovation and deliver important medicines for patients."

Launch of Onpattro (patisiran) in the U.S. and EU was in second quarter. Had been approved by the FDA in August. Revenue was $12.1 million. Will need to see several quarters of results to be able to see the longer term prescription trajectory. Reimbursement has been good, and 62% of patients were on Medicare.

Onpattro revenue was $12.1 million, up sequentially from $0.5 million. Revenue from collaborators: $9 million from Genzyme/Sanofi.

Cash and equivalents balance at the end of the quarter was $1.13 billion, down sequentially from $1.22 billion. $30 million in long-term debt. In January 2019, isssued 5,000,000 shares of common stock at $77.50 per share for net proceeds of approximately $382 million.

Non-GAAP net income negative $183.5 million, down sequentially from negative $, and down from negative $115.1 million year-earlier. EPS negative $1.82, down sequentially from negative $, and down from negative $1.20 year-earlier.

Alnylam continued a Phase 1 trial for vutrisiran (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 began in late 2018. Announced plans to initiate an additional Phase 3 study, HELIOS-B, in hereditary and wild-type ATTR amyloidosis with cardiomyopathy in late 2019.

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 rolling submission commenced.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial started, and topline results are expected in 2019, with an NDA in 2020. Announced alignment with the FDA on the trial design for ILLUMINATE-B, a Phase 3 study of lumasiran in PH1 patients less than six years of age with preserved renal function.

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): Alnylam’s partner, The Medicines Company, announced in January 2019 that the Independent Data Monitoring Committee for the ongoing inclisiran Phase 3 clinical trials (ORION 9, 10, and 11) conducted its fifth planned review of safety and efficacy data from the ORION trials and recommended that the trials continue without modification. Topline results expected in mid-2019 with an NDA possible by year end. 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.

Alnylam announced today that it has initiated a Phase 1 study of ALN-AAT02, for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease), with initial results expected in 2019.

With Vir Biotechnology, initiated a Phase 1/2 study of ALN-HBV02 (also known as VIR-2218), with initial results expected in 2019.

See also Alnylam pipeline.

Operating expenses of $241 million consisted of: $2 million for cost of goods sold; $131 million for research and development; and $108 million for general and administrative expense. Interest & other income was $9 million. Slight income tax benefit.

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

Q&A:

Onpattro conversion times? Intention is to get it down to a couple of weeks. The average time has been declining.

Europe ramp? We launched immediately after FDA approval in the U.S. Europe is more complicated, we are going through the process in 15 countries. We are happy with progress so far. We see strong patient demand in Europe and doctor interest.

At home infusion of Onpattro? Home infusion is available in EU and in the U.S. by commercial payers. Most physicians want to infuse once or twice before changin go home. Claims patients like being infused in offices as they get to meet other patients [this is likely a swipe at rival Tegsedi, which is given by injection].

Switches? Tafamidis patients often progress rapidly, so they switch to Onpattro.

Pricing in EU? We are keep a tight band globally.

Onpattro value based agreement color? We offered value based agreements, which helped open dialogs with payers. Payers have been great so far. We have 3 agreements complete and hope to have 90% coverage by end of year.

We give free genetic testing for ATTR, but so do other companies, and insurers pay for independent testing.

Cash runway details? 2019 R&D guidance includes all the Phase 3 trials. The earlier-stage trials are not a big part of the expense.

Stock based compensation growth? We can't guide on that, it depends on performance milestones. But 2019 should be about the same as 2018.

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