Analyst Conference Summary

biotechnology

conference date: February 6, 2020 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2019 (fourth quarter, Q4)

Forward-looking statements

Overview: Revenue has a nice ramp, but continues to bleed cash.

Basic data (GAAP):

Revenue was $71.7 million, up 2% sequentially from $70.1 million, and way up from $21.0 million year-earlier.

Net income was negative $278 million, down sequentially from negative $208.5 million, and down from negative $211 million year-earlier.

Diluted EPS was negative $2.47, down sequentially from negative $1.92, and up from negative $2.09 year-earlier.

Guidance:

For 2020 expects Onpattro revenue between $285 and $315 million. Collaboration revenue $100 to $150 million. Non-GAAP SG&A and R&D expenes $1.03 to $1.13 billion.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "In 2019 we saw continued and steady growth of patients on ONPATTRO, and we expect growth to continue in 2020, driven by new patient finding, geographic expansion, and evidence-generating activities. We also believe that our ongoing APOLLO-B Phase 3 study, if positive, can potentially enable future label expansion for patisiran to treat the cardiomyopathy of hereditary and wild-type ATTR amyloidosis. With the early U.S. approval of GIVLAARI in the fourth quarter, Alnylam became a multi-product commercial company,"

Working towards a self-sustaining (profitable) company. Timeline not stated.

The FDA approved givosiran for AHP (actue hepatic porphyria) at the end of 2019. 13 start forms were received so far..

Revenue from collaborators: $24.0 million from Genzyme/Sanofi.

Onpattro (patisiran) revenue was $55.8 million, up 21% sequentially from $46.1 million and up from $0.0 million year-earlier. Over 1,000 patients were on therapy at the end of the quarter. Landscape is "increasingly competitive." Making progress ex-US with new launches or getting reimbursement.

Cash and equivalents balance at the end of the quarter was $1.55 billion, down sequentially from $1.74 billion. No debt.

Non-GAAP net income negative $221 million, down sequentially from negative $163 million, and down from negative $183 million year-earlier. EPS negative $1.98, down sequentially from negative $1.50, and down from negative $1.82 year-earlier.

Alnylam continued a Phase 1 trial for vutrisiran (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 began in late 2018. Announced plans to initiate an additional Phase 3 study, HELIOS-B, in hereditary and wild-type ATTR amyloidosis with cardiomyopathy in late 2019.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. Additional Phase 2 data was presented in Q2 2019. Sanofi Genzyme is a partner in the program.

Vutrisiran (ALN-TTRsc02) subcutaneously administered ATTR treatement continued the HELIOS-A Phase 3 study. Initiated the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial reported positive topline results in late 2019. Full results expected March 2020. An NDA planned for in 2020. Announced alignment with the FDA on the trial design for ILLUMINATE-B, a Phase 3 study of lumasiran in PH1 patients less than six years of age with preserved renal function.

Inclisiran for hypercholesterolemia moved over to Novartis, which acquired The Medicines Company in January 2020. 2 Phase 3 studies showed positive results, so an NDA was submitted to the FDA.

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.

Cemdisiran (ALN-CC5) continued a Phase 2 study in IgA nephropathy.

In Q1 2019 received approval to initiate a Phase 1 study of ALN-AGT, an investigational RNAi therapeutic targeting angiotensinogen (AGT) for the treatment of hypertension in high unmet need populations, including patients with resistant or refractory hypertension, chronic kidney disease or heart failure.

ALN-HBV Phase 1 study reported positive results in late 2019..

ALN-AGT Phase 1 study for hypertension reported initial positive results in late 2019.

Alnylam reported positive results from a Phase 1 study of ALN-AAT02, for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease), in late 2019.

With Vir Biotechnology, continued a Phase 1/2 study of ALN-HBV02 (also known as VIR-2218), with initial results expected in 2019.

See also Alnylam pipeline.

Operating expenses of $370 million consisted of: $12 million for cost of goods sold; $201 million for research and development; and $156 million for general and administrative expense. Operating loss $298 million. Interest & other income was $21 million. $0.4 million income tax benefit.

Believes 2019 represents the peak non-GAAP operating loss year.

Q&A Summary:

The 13 start forms came from a variety of prescribers. More data next quarter.

ATTR diagnoses? The diagnostics are evolving. We are focussed on the value for treating polyneuropathy. It is being recognized in a significant portion of the ATTR spectrum. hATTR amyloidosis diagnostics could lead to polyneuropathy diagnoses. Cardiomyopathy overlaps with polyneuropathy.

2020 guidance, Japan ramp? It is the first time we have provided revenue guidance. Will add new products after we have about a year of sales. We expect growth driven by new patients in new markets, including Japan.

Pricing reimbursement outside the US has been very strong.

Onpattro Q4 factors? There was underlying demand growth of 12%. Gross to net reduced that to 6% revemie growth. We expect no seasonality in Q1.

Japan share of Q4 sales? We don't provide that breakdown, but in Q1 we expect Japan to contribute significantly. It contributed to growth in Q4, as did the UK.

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