Analyst Conference Summary

biotechnology

conference date: November 7, 2017 @ 1:30 PM Pacific Time
for quarter ending: September 30, 2017 (third quarter, Q3)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $17.1 million, up 8% sequentially from $15.9 million, and up 25% from $13.7 million year-earlier. All revenue was from collaborations.

Net income was negative $122.9 million, down sequentially from negative $118.4 million, and down from negative $104.1 million year-earlier.

Diluted EPS was negative $1.34, flat sequentially from negative $1.34, and down from negative $1.21 year-earlier.

Guidance:

Year end cash including restricted investments are expected over $1.0 billion.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "With patisiran, our recent APOLLO Phase 3 study results demonstrate what we believe to be the transformative potential for RNAi therapeutics as a new class of innovative medicines. With these data, we expect to submit our first regulatory filings in the coming months, and are planning for the possibility of having regulatory approval for patisiran in mid-2018."

All revenue was from collaborators: $2.3 million from The Medicines Company, $14.6 million from Genzyme/Sanofi, and $0.2 million from other sources. Some of this revenue resulted from payments for milestones achieved.

Cash and equivalents balance at the end of the quarter was $1.15 billion, down sequentially from $1.25 billion. $150 million in long-term debt.

Non-GAAP net income negative 97.0 million, down from negative $88.5 million year-earlier. EPS negative $1.06, down from negative $1.03 year-earlier.

Alnylam announced a licensing agreement with Vir Biotechnology for infectious diseases.

Patisiran Phase 3 APOLLO study for the treatment of hereditary TTR-mediated amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP), topline results were positive. Alnylam plans to file for regulatory approval in the US by year-end 2017, then in the EU early in 2018.

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. Initial data was reported. A Phase 3 trial is expected to begin in 2018.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study (actually a set of 3) started in July. Sanofi Genzyme is a partner in the program. But study was suspended following a patient death. Dosing could resume before the end of 2017.

Givosiran (ALN-AS1) for acute hepatic porphyrias Phase 3 trial design approved by the FDA and the ENVISION trial was initiated. Interim data is expected mid-2018, with an NDA possible by the end of 2018.

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, started its Phase 3 trial.

Cemdisiran (ALN-CC5) for aHUS (atypical hemolytic-uremic syndrome) started a Phase 2 study.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $142.9 million consisted of: $95.3 million for research and development and $47.6 million for general and administrative expense. Interest & other income was $2.9 million. Income taxes $0 million. Unrealized gain on marketable securities was $0.2 million.

Alnylam hopes to begin commercial sales of Patisiran in 2018, Fitusiran in 2019, and Givosiran in 2020.

Q&A:

Givosiran reduction of ALA to be successful in Phase 3? Not disclosed, but with over 80% reduction in ALA, we have an agreement with with regulators for biomarkers we can comfortably hit. Patients part of interim analysis will continue on therapy for full study, which would be based on attack rate data.

Patisiran competition effect on wild type trial? We will see some results before we lock down our trial. We need to complete the dialog with regulators, especially the cardiac subset, before we put a path forward for wild type attr. We are committed to be leaders in the amyloidosis space, and then with ALN-TTRsc02.

Would you consider branching out of the RNAi space for combination therapies for attr? You make a good point. We will watch the case very carefully. The combination did not provide any great benefit over patisiran alone.

We have plenty of liver based targets. In the future we would go to muscle or ocular targets, but for now our hands are full.

Patisiran v. ALN-TTRsc02, how do you keep up value? We own 100% of Patisiran in North America and Western Europe, with Sanofi as our rest-of-world partner. With ALN-TTRsc02 it is 50-50 in North America and Western Europe, then Sanofi rest of world. We control development and commercialization. We will not cannibalize or impair the value of Patisiran.

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