Analyst Conference Summary

biotechnology

conference date: August 2, 2018 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2018 (second quarter, Q2)

Forward-looking statements

Overview: Onpattro for hattr amyloidosis U.S. PDUFA date is August 11. Received a positive opinion form EU CHMP, so likely approval there soon.

Basic data (GAAP):

Revenue was $29.9 million, up sequentially from $21.9 million, and up from $15.9 million year-earlier. All revenue was from collaborations.

Net income was negative $163.6 million, down sequentially from negative $141.2 million, and down from negative $118.4 million year-earlier.

Diluted EPS was negative $1.63, down sequentially from negative $1.41, and down from negative $1.34 year-earlier.

Guidance:

Alnylam expects to end the year with about $1.0 billion in cash.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "The second quarter and recent period mark a milepost in the history of Alnylam that has been 16 years in the making – the recommendation from the EU Committee for Medicinal Products for Human Use (CHMP) that the European Medicines Agency approve the first-ever RNAi therapeutic, Onpattro [patisiran]. We believe the positive CHMP opinion in the EU signals the potential for a new therapeutic paradigm in medicine, raising hope for patients and caregivers impacted by hATTR amyloidosis. With this achievement, the expected FDA action by our August 11 PDUFA date, and plans to file our JNDA in Japan later this year, we believe we are poised to deliver on the promise of ONPATTRO on a truly global scale,"

"To date the Company has fulfilled over 200 requests by physicians for eligible patients to begin treatment in the early access or compassionate use programs for patisiran in the U.S. and EU."

[WPM: note the rival Ionis/Akcea drug Tegsedi (inotersen) recently received EU approval and has a PDUFA date of October 6, 2018 in the U.S.]

Revenue from collaborators: $23.1 million from Genzyme/Sanofi, and $6.8 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 $1.48 billion, down sequentially from $1.60 billion. $30 million in long-term debt.

Non-GAAP net income negative $161.9 million, down sequentially from negative $121.6, and down from negative $94.4 million year-earlier. EPS negative $1.63, down sequentially from negative $1.22, and down from negative $1.34 year-earlier. Excluded non-cash stock based compensation was $22.2 million and the $20.6 million gain on litigation settlement.

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. 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 completed enrollment. topline data is expected by end of September, with an NDA possible by the end of 2018.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial should begin mid-2018, and topline results are expected in 2019, with an NDA in 2020.

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, but enrollment has been slow, so initial data in 2019.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $222.3 million consisted of: $137.6 million for research and development and $84.7 million for general and administrative expense. Gain on litigation settlement $20.6 million. Interest & other income was $8.3 million. Income taxes $0 million.

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

Q&A:

ALN-TTRsc02 trial design? We are aligned with the FDA on a single arm open label study, we believe 9 months is sufficient to see the effect on the endpoints.

Lumasiran trial design? We have to show the reduction in urinary oxalate.

New Tafamidis data? Looking for wild type versus hereditary and 20 mg v. 80 mg differentials. Also level of affect, 25% v. 50%. Patients want stabilization or reversal of disease, not just slowing of progression.

Competitive landscape, warehousing of patients? In hereditary there are neurologists and cardiologists, neither warehouse patients. In wild type there is nothing available. Doctors will want to treat patients as soon as a drug becomes available. But cardiologists tend to be more aware of amyloidosis.

Onpattro and ALN-TTRsc02 life cycles? Same patient population, gives them a subcutaneous option.

How large is the givosiran opportunity? It is for an underdiagnosed disease. Out of 100 or so samples we tested, about 15 came back positive. There are a few thousand patients in the system right now. We believe the uptake among known patients will be rapid.

Onpattro advantages over inotersen include: they lack cardiology data; their label is for treating the symptom, neuropathy, ours the underlaying disease.

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