Alnylam Pharmaceuticals (NASDAQ:ALNY) doesn't have a drug on the market yet, but it was a busy second quarter for the biotech, with movement on the pipeline front as it awaits EU and U.S. approvals for Onpattro that should happen shortly.

Alnylam Pharmaceuticals' Q2 results: The only number that really matters


Q2 2018

Q4 2017

Year-to-Date Change (Decline)

Cash, cash equivalents, marketable debt securities, and restricted investments

$1.48 billion

$1.73 billion

($0.25 billion)

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam Pharmaceuticals this quarter?

  • Without any revenue-generating drugs, the company's nest egg is the only number that really matters at this point. While Alnylam is spending quite a bit, it should have revenue coming in shortly to slow the cash burn rate.
  • The EU Committee for Medicinal Products for Human Use gave a positive opinion on Onpattro, setting up approval for the hereditary TTR amyloidosis (hATTR) drug by the European Commission.
  • In the U.S., Alnylam is expecting an approval for Onpattro from the Food and Drug Administration this week.
  • Alnlyam reached agreement with the FDA on the structure for a clinical trial to get its follow-on ATTR drug, TTRsc02, approved. Likewise, Alnylam and the FDA agreed to a pivotal program for lumasiran, which treats hyperoxaluria type 1. Both trials are expected to start before the end of the year. 
  • The phase 3 Envision study testing givosiran for acute hepatic porphyrias is enrolling quicker than expected with full enrollment expected by the end of September and full data in early 2019.
  • It wasn't all good news for the biotech this quarter. A phase 2 study of Cemdisiran in atypical hemolytic-uremic syndrome won't read out until next year due to slower enrollment in the trial.
Doctor with hand on patient's shoulder

Image source: Getty Images.

What management had to say

"We are launch-ready here in the U.S., and also in Germany, where we expect our first EU launch," a confident CEO John Maraganore said of the company's ability to hit the ground running after Onpattro is approved.

Alnylam has pending competition from Ionis Pharmaceuticals (NASDAQ:IONS) and Pfizer (NYSE:PFE), which both have ATTR drugs with positive phase 3 data. But COO Yvonne Greenstreet, who sees a first-mover advantage for Onpattro, said, "Clearly for diseases like hATTR amyloidosis, I think physicians will be keen to treat their patients as soon as they possibly can when they see those drugs become available, rather than keep patients waiting for a medicine that may or may not come to market downstream."

Looking forward

Beyond the launch of Onpattro, which is clearly a monumental event for the development-stage drugmaker, Alnylam has potential to launch a second drug, givosiran, next year. An interim analysis of the Envision study is expected in September, which could be used to file a marketing application before the end of this year.

And even with five additional drugs behind Onpattro and givosiran ready to drive revenue in 2020 and beyond, Alnylam isn't resting on its laurels. It expects to file applications to start clinical trials for two new drugs later this year.