Analyst Conference Summary

biotechnology

conference date: May 1, 2019 @ 1:30 PM Pacific Time
for quarter ending: March 31, 2019 (first quarter, Q1)

Forward-looking statements

Overview: Onpattro revenue more than doubled sequentially to $26.3 million.

Basic data (GAAP):

Revenue was $33.3 million, up 52% sequentially from $21.0 million, but up 52% from $21.9 million year-earlier.

Net income was negative $181.9 million, up sequentially from negative $211.3 million, and down from negative $141.6 million year-earlier.

Diluted EPS was negative $1.73, up sequentially from negative $2.09, and down from negative $1.41 year-earlier.

Guidance:

Updated 2019 expense guidance: non-GAAP R&D expense $550 to $590 million; SG&A expense $390 to $410 million. The increase is mainly due to the Regeneron collaboration.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "We demonstrated good progress in global commercialization of Onpattro, continued success in our R&D efforts with positive Phase 3 results for givosiran, and a strong commitment to future pipeline growth through our landmark ocular and CNS disease alliance with Regeneron. In addition, we significantly strengthened our balance sheet through both an equity offering in January and partnership-based equity funding and cash that we will receive upon closing of the Regeneron transaction." Believes can now do 3 to 4 INDs annually.

Alnylam announced a partnership with Regeron for CNS and Ocular RNAi therapies. 50-50 structure, but Alnylam to receive milestone payments, including the initial $800 million in Q2..

Onpattro (patisiran) had been approved by the FDA for ATTR in August 2018. At the end of Q1 over 400 patients were on medicine worldwide. Making good progress with payers and reimbursements around the world. Preparing to launch in Canada and Japan upon regulatory approval. Demand is good despite competition.

Onpattro revenue was $26.3 million, up sequentially from $12.1 million. Revenue from collaborators: $7.0 million from Genzyme/Sanofi.

Cash and equivalents balance at the end of the quarter was $1.29 billion, up sequentially from $1.13 billion. $30 million in long-term debt. In January 2019, isssued 5,000,000 shares of common stock at $77.50 per share for net proceeds of approximately $382 million. Will receive $800 million in cash in Q2 from the Regeneron collaboration.

Non-GAAP net income negative $149.9 million, up sequentially from negative $183.5, and down from negative $121.6 million year-earlier. EPS negative $1.42, up sequentially from negative $1.82, and down from negative $1.22 year-earlier.

Onpattro (patisiran) will start a new Phase 3 study in mid-2019 for ATTR Amyloidosis in patients with cardiomyopathy.

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. Sanofi Genzyme is a partner in the program.

Givosiran (ALN-AS1) for acute hepatic porphyrias (AHPs) Phase 3 trial completed enrollment and positive topline data was announced. The NDA should be complete by mid-2019.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial started, and topline results are expected in 2019, with an NDA 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.

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): Alnylam’s partner, The Medicines Company, announced in April 2019 that the Independent Data Monitoring Committee for the ongoing inclisiran Phase 3 clinical trials (ORION 9, 10, and 11) conducted its sixth planned review of safety and efficacy data from the ORION trials and recommended that the trials continue without modification. Topline results expected in mid-2019 with an NDA possible by year end. Alnylam could receive milestones and up to 20% royalties.

Cemdisiran (ALN-CC5) for aHUS (atypical hemolytic-uremic syndrome) had started a Phase 2 study, but enrollment has been slow, so cancelled. Instead initiated 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 continued.

Alnylam continued a Phase 1 study of ALN-AAT02, for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease), with initial results expected in 2019.

With Vir Biotechnology, initiated 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 $222 million consisted of: $3 million for cost of goods sold; $129 million for research and development; and $90 million for general and administrative expense. Operating loss $188 million. Interest & other income was $15 million. Less than $1 million income tax expense.

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

Q&A:

Apollo B trial design? We have a lot of prior data. We think 300 patients is adequate to show a treatment effect. We will give more details when it starts midyear.

Tafamidis? It was approved in many European countries for FAT stage 1. Physicians recognize progression and can then switch to Onpattro. About half the Apollo patients had been on stabilizer previous, just as good as naive patients.

EU pricing thoughts? We are trying to keep a tight vial price, near the U.S. price, around the world.

TTR competitive dynamics vs. Tegsedi? We now have a full quarter with the competitive landscape we will have this year. We are not hearing that much about Tegsedi, just a few patients who prefer an injection rather than an infusion.

We are seeing improved awareness, and desire to set up centers of excellence, in ATTR, and not just in the U.S.

The majority of patients in Apollo B trial will be wild type. We are seeing an increased prevelance of reporting wild type, related to tafamidis trial.

Gross to net in U.S. in 20's? That is the global number.

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