Analyst Conference Summary

biotechnology

conference date: May 3, 2018 @ 1:30 PM Pacific Time
for quarter ending: March 31, 2018 (first quarter, Q1)

Forward-looking statements

Overview: Preparing for Patisiran launch in 2018, pending approval.

Basic data (GAAP):

Revenue was $21.9 million, down sequentially from $37.9 million, and up from $19.0 million year-earlier. All revenue was from collaborations.

Net income was negative $141.2 million, up slightly sequentially from negative $142.2 million, and down from negative $107.3 million year-earlier.

Diluted EPS was negative $1.41, up sequentially from negative $1.48, and down from negative $1.25 year-earlier.

Guidance:

End of 2018 cash expected around $1.0 billion. Non-GAAP R&D expense $420 to $460 million; SG&S expense $280 to $320 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "we are actively preparing for our U.S. launch of patisiran, following an anticipated mid-2018 FDA approval. With our U.S. field team on-boarded, we believe we are ‘launch ready’ and prepared to get patisiran to patients as soon as it is approved.”

“Beyond patisiran, we were very pleased to announce that we’ve completed enrollment of the first 30 patients in our ENVISION Phase 3 study with givosiran, putting us on track to complete an interim analysis in the September timeframe. We are also excited to announce that we’ve reached alignment with the U.S. FDA on a pivotal study design on our lumasiran program, significantly accelerating our development efforts for this investigational RNAi therapeutic. In sum, we believe our efforts position the Company to achieve its Alnylam 2020 goals of building a multi-product, commercial-stage company with a deep clinical-stage pipeline and robust product engine by the end of 2020, a profile rarely achieved in the biotech industry.”

Positive Phase 3 Apollo results and NDA to FDA for Patisiran for hereditary TTR-mediated amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP) should enable a commercial launch in the second half of 2018. PDUFA date is August 11.

All revenue was from collaborators: $1.3 million from The Medicines Company, $18.9 million from Genzyme/Sanofi, and $1.7 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 $1.60 billion, up sequentially from $1.73 billion. Repaid $ million of its term loan agreement. $30 million in long-term debt.

Non-GAAP net income negative $121.6 million, down sequentially from negative $115.1, and down from negative $91.6 million year-earlier. EPS negative $1.22, down sequentially from negative $1.20, and down from negative $1.06 year-earlier. Excluded non-cash stock based compensation was $19.6 million.

Patisiran for hATTR amyloidosis Phase 3 trial reported positive results. Approval by the FDA is expected mid-2018.

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. Alnylam now owns all the rights. A Phase 3 trial is expected to begin in late 2018. Believes this could be expanded to help carriers, which would greatly expand the patient pool.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. Sanofi Genzyme is a partner in the program.

Givosiran (ALN-AS1) for acute hepatic porphyrias Phase 3 trial enrolled the first 30 patients. Interim data is expected mid-2018, with an NDA possible by the end of 2018.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial should begin mid-2018, but Sanofi declined to opt-in.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study in Europe continued and updated positive data was presented.

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, started its Phase 3 trial, with complete enrollment expected soon. Alnylam could receive milestones and up to 20% royalties.

Cemdisiran (ALN-CC5) for aHUS (atypical hemolytic-uremic syndrome) started a Phase 2 study.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $169 million consisted of: $97 million for research and development and $72 million for general and administrative expense. Interest & other income was $6 million. Income taxes $0 million.

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

Q&A:

Patisiran market opportunity in segments? Biggest hATTR issue is diagnosis. With more voices (competitors) out there will grow the market significantly. We look forward to seeing all the data for all the products. We like our own product's profile best. We don't have wild-type setting data yet.

Confidence in broadness of Patisiran label? The data are strong across the spectrum, our submission to the FDA is broad, but there is a review process.

Patisiran pricing? We believe Patisiran has a very high value. We are working with payers to make sure that access is not a barrier.

Took several opportunities to run down Pfizer's Tafamidis, which might get approved for ATTR in the U.S. later this year (it has been approved in the EU since 2011).

Timeline to complete Apollo open label extension trial? It is going well, the cohort could be dosed for up to five years.

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