Alnylam Pharmaceuticals Launches Onpattro

Alnylam Pharmaceuticals (ALNY 0.19%) last week reported the first sales for its hereditary transthyretin-mediated (hATTR) amyloidosis drug Onpattro, which was approved by the Food and Drug Administration in August. With just seven weeks on the market, there wasn't much in the way of sales to get investors excited about in the third-quarter numbers. Fortunately, management shared some additional data about patient trends.

Alnylam Pharmaceuticals results: The raw numbers

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam Pharmaceuticals this quarter?

• Revenue in the year-ago quarter was from collaborations, so the substantial drop is nothing to worry about.
• During the seven weeks that Onpattro was on the market during the quarter, Alnylam generated just $460,000 in sales. The company estimates it can take up to a couple of months to get patients approved by their insurance companies, set up for infusions, and on to the drug, so the slow start shouldn't be too surprising.
• Alnlyam received 125 patient start forms. While it's not guaranteed that all those patients will end up on Onpattro after going through the insurance process and what-not, the start forms are a decent measure of demand. About 60% of those patients were previously receiving free drug on the early access plan (EAP).
• To find more patients, Alnylam is continuing to offer genetic testing of patients suspected of having hATTR amyloidosis. As of the end of October, the company had identified 584 patients with a mutation from more than 8,700 tested samples.
• On the pipeline front, Alnylam reported positive interim results for givosiran in patients with acute hepatic porphyrias, which is caused by the buildup of neurotoxic intermediates. And the company started a phase 3 program for lumasiran, which treats a kidney disease called primary hyperoxaluria type 1.

Image source: Getty Images.

What management had to say

CEO John Maraganore noted that it's going to be hard to predict the sales ramp for Onpattro with many patients coming from the EAP or from doctors associated with Alnylam's Apollo clinical trial program:

We believe the initial start forms likely represent a bolus of patients with a significant number of patients converting from EAP and also diagnosed patients known to Apollo investigators. Therefore, we don't necessarily expect this rate to be reflective of the future run rate. We'll need several quarters to process the initial bolus of patients before we can develop a truer understanding of the run rate of Onpattro demand.

COO Barry Greene talked about insurance coverage and value-based agreements (VBAs) that allow insurers to get discounts if patients don't respond to the drug: "I can tell you that government reimbursement is going well and is fast. And then, on the commercial payer side, and remember we've highlighted that with the VBAs signed or in discussion, we anticipate I think about 70% of commercial patients covered under VBAs."

Looking forward

Clearly, the launch of Onpattro is the most important near-term event for Alnylam, but investors shouldn't forget about the biotech's pipeline while they wait for meaningful Onpattro sales. The full results for the givosiran phase 3 trial should be available early next year, setting up a marketing application submission to the FDA in the middle of 2019. And data from the recently started lumasiran phase 3 program will be available in late 2019, potentially resulting in a third approved drug in 2020.