Analyst Conference Summary

biotechnology

conference date: May 5, 2017 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2017 (first quarter, Q1)

Forward-looking statements

Overview: Development stage company continues to make progress with its RNAi therapies.

Basic data (GAAP):

Revenue was $19.0 million, up 9% sequentially from $17.5 million, and up 160% from $7.3 million year-earlier. All revenue was from collaborations.

Net income was negative $107.3 million, up sequentially from negative $112.9 million, and up from negative $103.0 million year-earlier.

Diluted EPS was negative $1.25, up sequentially from negative $1.32, and up from negative $1.21 year-earlier.

Guidance:

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

Conference Highlights:

"With our patisiran program, we look forward to the read-out of our APOLLO Phase 3 study and, if the data are positive, our first NDA filing. In addition, we expect to advance three additional programs into Phase 3 trials: fitusiran, givosiran, and - with our partners at The Medicines Company - inclisiran," said John Maraganore, CEO. "We believe our upcoming milestones put us on track to meet our ‘Alnylam 2020' goals of becoming a multi-product, commercial-stage company with a deep and sustainable clinical development pipeline by the end of 2020."

All revenue was from collaborators: $6.4 million from The Medicines Company, $12.3 million from Genzyme/Sanofi, and $0.3 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 $0.96 billion, down sequentially from $1.09 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 (September likely). 25-month OLE (open label extension) Phase 2 data were presented on April 26, showing consistent positive results. Ragged on possible Ionis rival product result.

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 Phase 1 data was presented at EAHAD, with some Phase 2 data to be reported in July. Phase 3 ATLAS study (actually a set of 3) should start by the end of Q2. 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. More Phase 1 data will be presented in June at ISTH. Accepted in Europe's PRIME program, which could allow for an accelerated pth.

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, positive Phase 2 data was presented on March 17.

ALN-HBV Phase 1 study continued.

Discontinued drug Rivusiran investigation should be concluded this summer.

See also Alnylam pipeline.

Operating expenses of $125.5 million consisted of: $87.0 million for research and development and $38.5 million for general and administrative expense. Interest & other expense was $0.8 million. Income taxes $0 million. Unrealized loss on marketable securities was $1.9 million.

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

Q&A:

Fitusiran stability? We have done studies, it will require cold chain storage delivery.

If Patisiran trial reads out positive, what of ALN-TTRsc02? The Phase 1 had exciting results for knockdown, safety, and length of control, we will take that forward after negotiating with regulators.

A number of questions were about building out infrastructure for commericalization, and the answer is Alnylam is doing what is necessary.

Patisiran secondary endpoints from Phase 2? It is not comparative to placebo. The endpoints don't improve, but we would expect them to get worse with time based on natural history. We would gues the endpoints would be better than placebo.

Hemophilia competition? Excitement about emerging medicines. All the approaches show promise and together will disrupt the use of factor. It is a $10 billion market. Fitusiran fits in nicely, depending on the trial results. Works for both A & B and is once per month. Believe gene therapy will have a place, but will take a long time to get to commercialization.

Ionis data, what to look for? A borderline result could reflect their trial was underpowered. We expect positive data, we know they have had adverse events, we need to see the actual data.

Givosiran, what needs to be done before starting Phase 3? Preliminary data are very encouraging. We need to share the data with agencies and get approval for trial design and endpoints. It should be "a fairly straightforward placebo-controlled study." We are going to enrich for patients with multiple attacks, we have to finalize that with regulators.

We are impressed by the number of patisiran patients who volunteered for the biopsy in APOLLO.

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