Analyst Conference Summary

conference date: August 6, 2015 @ 1:30 PM Pacific Time
for quarter ending: June 30, 2015 (second quarter 2015, Q2)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $8.7 million, down sequentially from $18.5 million, and up from $7.3 million year-earlier.

Net loss was $71.8 million, down % sequentially from loss of $50.8 million, and down from loss of $44.1 million year-earlier.

Diluted EPS was negative $0.85, down sequentially from negative $0.62, and down from negative $0.58 year-earlier.

Guidance:

Expects to finish the year with over $1.2 billion in cash.

Conference Highlights:

All revenue was from collaborators: $3.4 million from Takeda, $2.6 million from The Medicines Company, $2.6 million from Genzyme, and $0.1 million from reagent licenses. Some of this revenue resulted from payments for milestones achieved.

In the quarter three therapies announced positive results.

The Patisiran for Familial Amyloidotic Polyneuropathy (FAP) Phase 2 study generated increased evidence for a possible halting of neuropathy progression after the first 12 months of treatment, with study drug administration generally well tolerated out to 17 months.

ALN-AT3 for Hemophilia results from our ongoing Phase 1 trial provide confirmatory evidence that ALN-AT3 has the potential to re-balance hemostasis in people with severe hemophilia through normalization of thrombin generation.

ALN-CC5 for Complement-Mediated Diseases "reported initial positive data from our ongoing Phase 1/2 trial with ALN-CC5 - an investigational RNAi therapeutic for complement-mediated diseases - where we demonstrated what we believe to be promising preliminary clinical activity for single doses of study drug with potent and highly durable knockdown of serum C5 and inhibition of serum complement activity," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.

Cash and equivalents balance at the end of the quarter was $1.40 billion, down sequentially from $1.45 billion.

Strategic program "Alnylam 2020" provides guidance for the commercialization of RNAi therapies through 2020, assuming the clinical data supports that. Expects to have 3 commercial products by 2020, with a robust follow-up pipeline of 10 clinical programs.

Completed enrollment of the ALN-PCSsc Phase 1 study, and results will be presented later in August. Partnered with The Medicines Company. ALN_PCSsc targets PCSK9 to treat hypercholesterolemia (high cholesterol).

Patisiran APOLLO-OLE (open label extension) study started dosing for the treatment of ATTR amyloidosis.

ALN-AS1 for acute hepatic porphyrias and ALN-AAT for ATT deficiency liver disease also began dosing in Phase 1 studies. Results could be available in September.

Alnylam announced today it has a candidate, TTRsc02, for an " ESC-GalNAc-siRNA targeting TTR that is expected to support a once-monthly and possibly once-quarterly subcutaneous dose regimen."

Plans to advance ALN-GO1 for primary hyperoxaluria to development candidate status in mid 2015.

The company reported that the Phase 2 study of revusiran for FAC and senile systemic amyloidosis (SSA) had three patients discontinue due to injection site reactions, but has otherwise been well tolerated. Initial data is expected before the end of 2015. The FDA granted orphan drug status to rivusiran.

See also Alnylam pipeline.

Operating expenses of $81.6 million consisted of: $67.0 million for research and development and $14.6 million for general and administrative expense. Interest income was $1.6 million. Income taxes $0.4 million.

Q&A:

Development candidate for TTR, dosing frequency context with unmet need? We are excited about this candidate. TTRsc02 looks like the most potent of these drugs we have built, based on animal studies. It could be a once-monthly to once-quarterly, lose dose injection.

Injection site reactions details? Of the three discontinued, two had rashes beyond the injection site. This is out of 25 patients in the study. All were out into months of exposure. We have patients who had been dosing longer without this adverse reaction. There were no reactions that got worse and worse. One had a mild rash, one was severe but did not require specific treatment.

Porphyria data, earliness of it? We need to wait a few weeks to talk about specific data. We had good patient enrollment in Sweden.

PCSK9 therapy? We will report the data on 70 subjects, and explored on statins and off statins. We should be able to tell if quarterly dosing is supported. Competitor approvals show LDL lowering is approvable, pricing is good, and we believe the dosing frequency makes our solution attractive.

ALN-CC5 endpoints? We want to understand safety. PNH markers include LDH, and reductions in hemolysis, and substantial reductions in C5 levels. Hopefully the results will accelerate CC5 into Phase 3.

ALN-HBV? The science is that knocking down the Hepatitis B surface antigen can change the way HBV is managed. It is one way the virus can elude immune system detection. It would be added to other therapies, so using a single sRNA should work without allowing resistance to develop. We would most likely retain HBV in the U.S. and possibly partner outside the U.S.

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