Alnylam Adds on Sales, Looks to Pipeline for Further Growth

Alnylam Pharmaceuticals (ALNY -0.06%) continued its launch of Onpattro for transthyretin-mediated amyloidosis with solid quarter-over-quarter growth as the biotech looks to its pipeline to further boost its revenue.

Alnylam Pharmaceuticals results: The raw numbers

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam Pharmaceuticals this quarter?

• Sales of Onpattro came in at $38.2 million, up 45% quarter over quarter. Most of the revenue came from the U.S., but the EU contributed $10 million, setting up a solid base for future growth outside the U.S.
• There were more than 500 patients paying for Onpattro at the end of the second quarter, and more than half of the prescriptions in the quarter came from new prescribers, which is a good sign for future growth.
• Onpattro was recently approved for sale in Japan; Alnylam thinks Japan can be the second-best-selling country behind the U.S. by the end of next year.
• While Alnylam is still unprofitable, it's sitting on a large nest egg of $1.97 billion thanks to $800 million in up-front proceeds from its drug-development deal with Regeneron Pharmaceuticals (REGN -1.75%).

Image source: Getty Images.

What management had to say

Alnylam is developing a next-generation transthyretin-mediated amyloidosis drug, vutrisiran, in a clinical trial called Helios-A, which Alnylam's president Barry Greene admits is having a negative effect on sales of first-generation Onpattro: "For every patient that goes into Helios-A, they could be a commercial patient of course, we understand that, but we're investing in that program for the benefit of having a new product offering for patients in the future -- in the not-too-distant future that we think will continue to drive growth of the franchise."

Alnylam is offering free genetic testing for transthyretin-mediated amyloidosis, but Greene reminded investors this is a long-term play. "Now some patients do not yet have penetrance of disease, but we believe that by raising awareness, remember, this is not about a portion of the pie, it is growing your overall pie, that will continue to benefit patients by raising awareness and speed to diagnosis."

Looking forward

In addition to growing sales of Onpattro, investors should be watching the pipeline, which includes givosiran for a rare disease called acute hepatic porphyria that is currently under review by U.S. and European regulators. The FDA is expected to make a decision on or before Feb. 4, 2020, while the European Medicines Agency will probably take a little longer.

On the clinical trial front, Alnylam's partner, The Medicines Company (MDCO), will release data from a pair of late-stage studies testing cholesterol-lowering inclisiran in the next few weeks. And then Alnylam will release data from a late-stage trial testing lumasiran in a kidney disease called primary hyperoxaluria type 1 later this year.

Alnylam could go from one marketed drug to four in a little more than a year.