Alnylam Pharmaceuticals Is Launched and Ready for More

Alnylam Pharmaceuticals (ALNY -1.27%) updated investors last month on how the launch of Onpattro, its first drug that was approved to treat transthyretin-mediated amyloidosis (ATTR), was going at the J.P. Morgan Healthcare Conference.

But the fourth-quarter earnings report allowed for another check-in with management and an audited look at how things are going with the biotech's financials.

Alnylam results: The raw numbers

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam this quarter?

• The decrease in overall revenue came from lower collaboration revenue, which tends to be lumpy. Fourth-quarter sales of Onpattro were $12.075 million, a smidgen higher than the $11 million to $12 million that Alnylam estimated when it presented at J.P. Morgan.
• While Onpattro is currently approved for patients with neurological issues, Alnylam reached an agreement with the Food and Drug Administration for its Apollo-B study, which will be used to get Onpattro approved for ATTR patients with heart complications. The study is expected to start in the middle of this year, with potential approval in late 2021 or 2022.
• Alnylam also announced plans to test its next-generation ATTR drug, vutrisiran, in patients with heart complications in a study dubbed Helios-B, which should start in late 2019.
• The larger loss was caused by costs related to the launch of Onpattro as well as increased research and development expenses. Nevertheless, Alnylam still ended the year with $1.13 billion in the bank, giving the biotech a substantial runway to get to profitability.

Image source: Getty Images.

What management had to say

Alnylam president Barry Greene highlighted how the company is trying to speed up the time between patient identification and starting Onpattro: "We have examples now of a start form coming in and patients literally getting on drug in a couple weeks. It does depend on where they're being infused, what the insurance looks like, but those times are coming way down, and we're really excited about the progress we're making."

Akshay Vaishnaw, Alnylam's president of research and development, talked about the competition for patients enrolling in the Apollo-B and Helios-B studies in the face of a launch of Pfizer's (PFE -0.10%) tafamidis:

There are a very significant number of wild-type patients that are currently on no treatment at all. Many of them, despite the potential approval of tafamidis in months to come may be in a territory where tafamidis may not be available or some other therapy may not be available for them. The availability of patients with hereditary TTR similarly varies by territory, and so between the need to do a robust study that encompasses a range of mutations and geographies that satisfies the regulators, the availability of the various drugs ended up being recently approved or about to be approved. We're confident that we can ensure enrollment without competing between the studies or competing between other goals in the company.

Check out he latest Alnylam earnings call transcript.

Looking forward

Management isn't giving guidance for Onpattro sales, which seems completely reasonable given the unknowns. Slow-but-steady seems like a reasonable expectation given the time needed to find patients and get them set up on the drug.

As important as the Onpattro launch is to the short-term success, long-term investors should be focused on the pipeline, including data from a phase 3 study of givosiran in patients with acute hepatic porphyrias, a disease caused by the buildup of neurotoxic intermediates, which is expected in March. Later in the year, investors will get to see phase 3 data for another drug, lumasiran, which treats a kidney disease called primary hyperoxaluria type 1.

If they're both positive, Alnylam could be launching two more drugs in the next two years, taking much of the urgency off the launch of Onpattro.