Analyst Conference Summary

conference date: February 11, 2016 @ 1:30 PM Pacific Time
for quarter ending: December 31, 2015 (fourth quarter 2015, Q4)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $7.6 million, down sequentially from $6.3 million, and down from $24.0 million year-earlier. All revenue was from collaborations.

Net loss was $90.7 million, down sequentially from loss of $76.8 million, and down from loss of $21.4 million year-earlier.

Diluted EPS was negative $1.07, down sequentially from negative $0.91, and down from negative $0.28 year-earlier.

Guidance:

Not given, but outlined likely pipeline events going forward. Expects to end 2016 with over $850 million in cash.

Conference Highlights:

Advancing a very robust RNAi pipeline addressing major unmet medical needs. Expects 10 major clinical readouts in 2016, plus 3 new IND filings to advance candidates into clinical trials. Fitusiran Phase 3 trials should start in 2016.

Enrollment complete in the Patisiran Phase 3 APOLLO ATTR Amyloidosis FAP trial. Alnylam believes it could make its first regulatory filing for commercial approval in late 2017. the Phase 2 open-label extension study (OLE) showed positive 18 month data. Complete data will be released on April 20.

Currently has eight clinical stage programs.

All revenue was from collaborators: $2.9 million from The Medicines Company, $3.6 million from Genzyme, and $1.1 million other. Some of this revenue resulted from payments for milestones achieved.

Sanofi Genzyme exercized its right to purchase 205,030 shares of Alnylam stock at $69.75 per share, resulting in $14.3 million cash. Sanofi owns about 12% of Alnylam stock.

Cash and equivalents balance at the end of the quarter was $1.28 billion, down sequentially from $1.34 billion. Investment in Regulus figured at $51.4 million; it had been $94.6 million at the end of 2014.

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.

ALN-PCSsc Phase 1 trial reported positive initial data. Partnered with The Medicines Company, which plans a Phase 2 trial. ALN-PCSsc targets PCSK9 to treat hypercholesterolemia (high cholesterol). Results may support quarterly dosing. Initial Phase 2 data could be available in late 2016, with a Phase 3 start possible in 2017.

Revusiran Phase 3 ENDEAVOR study continued to enroll ATTR amyloidosis FAC (familial amyloidotic cardiomyopathy) patients. OLE Phase 2 study reported postive 6-month initial data. 12 month data should be available mid-year.

ALN-AT3 (fitusiran) for hemophilia and rare bleeding disorders continued a Phase 1 study and initiated a Phase 1 extension study. Interim results were positive, giving evidence for restoration of hemostasis in severe hemophilia A and B. Phase 3 studies could start in 2016. Genzyme elected to opt into the ALN-AT3 program, with goal of commercializing outside of U.S. and Europe.

ALN-AS1 for acute hepatic porphyrias is expected to report initial data in late 2016. If the data is positive a Phase 3 trial will be started in 2017.

ALN-CC5 for complement-mediated diseases including PNH continued dosing in a Phase 1/2 trial and reported positive initial data. A hase 3 trial could start in 2017. Could be used with or without eculizumab (Alexion's Soliris).

ALN-AAT for ATT deficiency liver disease continued a Phase 1/2 trial.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) is set to start a Phase 1 study in Europe. It received Orphan Drug Designation from the FDA.

ALN-HBV Phase 1 study could begin in early 2016.

See also Alnylam pipeline.

Operating expenses of $100.1 million consisted of: $82.8 million for research and development and $17.2 million for general and administrative expense. Interest & otehr income was $1.8 million. Income taxes $0 million. Unrealized gain on marketable securities was $11.6 million.

By 2015 Alnylam expects to be running five or more Phase 3 trials.

2016 milestones expected:

In early 2016, Alnylam plans to:
• File a CTA for ALN-TTRsc02;
• Initiate a Phase 1 study for ALN-GO1; and
• File a CTA for ALN-HBV.

In mid-2016, Alnylam plans to:

• Present 24-month patisiran Phase 2 OLE data;
• Present 12-month revusiran Phase 2 OLE data;
• Start ALN-TTRsc02 Phase 1 study;
• Present additional fitusiran Phase 1 data;
• Start first fitusiran Phase 3 study;
• Present ALN-CC5 Phase 1/2 data in PNH patients;
• Present initial Phase 1 data with ALN-AAT; and
• Start ALN-HBV Phase 1 study.

R&D expense will continue to increase in 2016 as the number of clinical programs increases. About $100 million will be invested in a manufacturing facility in 2016.

Q&A:

Patisiran, FAP vs. FAC, could patients slide over? We see substantial evidence of clinical benefit on the cardio side. How the data will translate to a label, we don't know yet. Many TTR mutation patients have a mixed pathology.

CC5 possible studies, mono vs. combination? We have begun to enroll PNH patients and include naive patients and those who have used eculizumab. What we do in Phase 3 will depend on prior data. We know we will enroll a monotherapy study. We believe the current data shows it has C5 knockdown that could help patients on eculizumab use less. We believe CC5 is a very compelling approach as it reduces C5 produced by the liver.

TTRsc02 Phase 1 trial design? Phase 1 will be in healthy volunteers, but we also would look for successful knockdown.

ALN-AT3 (fitusiran) for hemophilia inhibitor patients vs. non-inhibitor patients? The inhibitor study likely will have less than 50 patients, the non-inhibitor will likely have around 150 patients.

Patisiran vs. potential FAP therapy competitors? We were interested to see the Ionis GSK knockdown. Patisiran is showing much better knockdown. That gives us a strong foundation for a best-in-class drug, including for safety. We are competitive in timing, having caught up, given the full enrollment in our Phase 3 trial.

Updated fitusiran data could be released in July if our papers get accepted at the meeting then.

We will be doing studies outside of PNH, probably starting in 2017.

OpenIcon Alnylam page

OpenIcon Analyst Conference Summaries Main Page

 

Search

More Analyst Conference Pages: