Analyst Conference Summary

biotechnology

conference date: August 9, 2017 @ 1:30 PM Pacific Time
for quarter ending: June 30, 2017 (second quarter, Q2)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $15.9 million, down 16% sequentially from $19.0 million, and up 83% from $8.7 million year-earlier. All revenue was from collaborations.

Net income was negative $118.4 million, down sequentially from negative $107.3 million, and down from negative $90.1 million year-earlier.

Diluted EPS was negative $1.34, down sequentially from negative $1.25, and down from negative $1.05 year-earlier.

Guidance:

Plans to end 2017 with over $1 billion in cash.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "With patisiran, we expect to report top-line APOLLO Phase 3 study results in the coming weeks. If positive, these study findings will support our first NDA filing, planned by year-end, and our commercial transition in mid-2018 assuming regulatory approval. In parallel, we continue to advance our late-stage pipeline of investigational RNAi therapeutics, with the recently announced initiation of our ATLAS Phase 3 program for fitusiran in hemophilia and expected Phase 3 starts by year-end for givosiran in acute hepatic porphyrias and, with The Medicines Company, inclisiran in hypercholesterolemia."

All revenue was from collaborators: $1.5 million from The Medicines Company, $14.4 million from Genzyme/Sanofi, and $0 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 $1.25 billion, up sequentially from $0.96 billion. Raised $376.5 million with a stock offering and concurrent private placement with Sanofi Genzyme. $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 fully enrolled. Topline results should be available in mid 2017 (September likely), with full results scheduled for November 2 or 3. 25-month OLE (open label extension) Phase 2 data were presented on April 26, showing consistent positive results. Hope to file an NDA with the FDA before the end of 2017.

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 Positive Phase 2 data was presented in July. Phase 3 ATLAS pivotal study (actually a set of 3) started in July. 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 ICPP. Granted Orphan Drug designation and Breakthrough Therapy designation by the FDA. 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, for hypercholesterolemia made an agreement for the Phase 3 protocol with the FDA. The trail could start in late 2017.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $136.4 million consisted of: $90.6 million for research and development and $45.8 million for general and administrative expense. Interest & other income was $2.0 million. Income taxes $0 million. Unrealized loss on marketable securities was $0.1 million.

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

Q&A:

Registration path for Patisiran, would you include rivusiran data? Rivusiran is a completely different molecule in a different setting, and the data was already shared with regulators. We will share all the safety data from the APOLLO trial.

Commercial plans for Patisiran? There is awareness and efforts to get earlier diagnosis. Cardiology and neurology settings need to know it as a potential diagnosis.

Is genetic testing easier than biopsies for this? Yes.

Slide 44 from RNAi presentation, patisiran vs. givosiran exposure levels? 21 mg every three weeks for patisiran. 28 grams were needed for rivusiran. Givosiran under 2.5 grams per year. So very different exposures for the newer generations of therapies.

Commercial readiness vs. competitor Tafamidis launch? The main lesson is to have a strong efficacy and safety package. If APOLLO has the same type of data as the open-label extension study, then the value proposition is much stronger than the competitor. So pricing should be more like real orphan drug pricing. We have done pricing panels. Pfizer's efforts with Tafamidis has raised awareness, but the relatively poor results caused a lack of enthusiasm in some countries.

APOLLO cardiac subset data expectations? All data will be filed. Regulators will be looking at where patients can end up, robustness of effects vs. placebo.

We expect the results announcement in mid to late September.

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