Analyst Conference Summary

biotechnology

conference date: February 8, 2017 @ 1:30 PM Pacific Time
for quarter ending: December 30, 2016 (fourth quarter, Q4)

Forward-looking statements

Overview: Development stage company continues to make progress with its RNAi therapies. It is running deep in the red due to R&D expenses.

Basic data (GAAP):

Revenue was $17.5 million, up 28% sequentially from $13.7 million, and up 130% from $7.6 million year-earlier. All revenue was from collaborations.

Net income was negative $112.9 million, down sequentially from negative $104.7 million, and worse than negative $90.7 million year-earlier.

Diluted EPS was negative $1.32, down sequentially from negative $1.21, and down from negative $1.07 year-earlier.

Guidance:

At the end of 2017 Alnylam expects to have over $700 million in cash remaining.

Conference Highlights:

"In the fourth quarter of 2016 and recent period, we reported positive clinical results with fitusiran for hemophilia, givosiran for porphyria, and ALN-CC5 for complement-mediated diseases. In addition, our partners at The Medicines Company, reported positive interim clinical data with inclisiran for hypercholesterolemia. On the other hand, we were disappointed to announce the discontinuation of our revusiran program due to safety findings, although our investigation to date indicates the findings are unique to this program and the clinical setting," said John Maraganore, CEO.

All revenue was from collaborators: $2.6 million from The Medicines Company, $14.8 million from Genzyme/Sanofi, and $0.1 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 $1.09 billion, down sequentially from $1.19 billion. $150 million in long-term debt.

Patisiran Phase 3 APOLLO study for the treatment of hereditary TTR-mediated amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP), is now fully enrolled. Topline results should be available in mid 2017. 25-month OLE Phase 2 data will be presented on April 26.

Revusiran program was discontinued due to higher than expected mortality in the treated arm of the trial.

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.

Fitusiran for hemophilia and rare bleeding disorders Phase 1 study interim results were positive, achieving a median estimated annualized bleeding rate of zero for hemophilia A and B. OLE study continued. Positive data was presented at ASH in December. Phase 3 ATLAS study (actually a set of 3) should start in early 2017. Sanofi Genzyme is a partner in the program.

Givosiran (ALN-AS1) for acute hepatic porphyrias Phase 3 trial will be started in 2017. Positive interim Phase 1 results were presented at ASH in December. Granted Orphan Drug designation. But Sanofi Genzyme decided to not opt in, so Alnylam has full global rights. More Phase 1 data will be presented in June.

ALN-CC5 for complement-mediated diseases including PNH continued dosing in a Phase 1/2 trial after reporting positive initial efficacy and safety data. Data was presented at ASH. But Sanofi Genzyme decided not to opt in, so future trial schedule is unclear.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study in Europe continued.

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, had positive interim analysis results from the Phase 2 hypercholesterolemia study. They initiated ORION-2 for patients "with Homozygous Familial Hypercholesterolemia (HoFH) as well as the ORION-3 study, a Phase 2 open-label cross-over extension study for patients completing the ORION-1 study." Full Phase 2 data is to be presented on March 17.

ALN-TTRsc02 for ATTR Amyloidosis Phase 1 study preliminary results were positive.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $133 million consisted of: $105 million for research and development and $28 million for general and administrative expense. Interest & other income was $2 million. Income taxes $0 million. Unrealized loss on marketable securities was $5 million.

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

Q&A:

Rivuseran safety? We are in an ongoing evaluation. Mortality was concentrated in the most advanced patients, suggesting there might have been an imbalance between arms of the study.

Strategic shift to Phase 3 programs? 2017 is a big year, first NDA submission, etc. We are maximizing our efforts on the leading candidates, but we will continue to develop the rest of the pipeline.

HBV program? No guidance on the program, but likely to report some data some time this year.

Key secondary APOLLO endpoints for reimbursement? We will report a p value for a primary endpoint. Safety as a secondary endpoint. Clinical benefit would include quality of life index, gain in muscle strength, walking scores, etc. Based on early Phase 2 data we believe it is likely quality secondary endpoints will be good, for a strong value dosier.

EHAB hemophilia presentation, co-dosing? Patients need lower doses of replacement factors when bleeds do occur. We have discussed that with opinion leaders. We are pleased with our experience to date when patients have a breakthrough bleed.

Cardio endpoints for APOLLO? Comprehensive analysis with biomarkers and echocardiography measures.

Medicines Company stock up, Alnylam down, thoughts? "It doesn't make any sense, does it?" The Incluseran story is exciting, and we are happy for The Medicines Company.

Timeline changes for programs where Genzyme opted out? We think it is an opportunity for us to expand to global commercialization. No comment on timeline yet.

Fitusiran hemophilia A vs. B and powering? Efficacy is relatively straightforward in hemophilia. We can analyze the data by type.

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