Alnylam Pharmaceuticals (NASDAQ:ALNY) reported second-quarter earnings last week -- and for the record, the company lost $118.4 million -- but without any products on the market, the focus of the earnings call was the biotech's pipeline and the potential for revenue in the not-too-distant future.

Alnylam Pharmaceuticals results: The only number that really matters


End of Q2 2017

End of 2016

Year-to-Date Change

Cash, cash equivalents, and fixed-income marketable securities

$1.1 billion

$943 million


Data source: Alnylam.

What happened with Alnylam Pharmaceuticals this quarter?

  • Alnylam said to expect data from the phase 3 Apollo trial testing patisiran in patients with hereditary ATTR amyloidosis (a condition characterized by a buildup of protein in the tissues) with polyneuropathy in mid- to late September. Assuming the data are positive, the company plans to submit a marketing application to the U.S. Food and Drug Administration by the end of the year and one to EU regulators shortly thereafter.
  • Patisiran has to be infused, but Alnylam has a follow-on drug, ALN-TTRsc02, which can be injected subcutaneously. After solid phase 1 results that show ALN-TTRsc02 is substantially more potent than patisiran, with potential to be dosed once every few months, Alnylam plans to advance, ALN-TTRsc02 straight into a phase 3 program in 2018.
  • The biotech started its phase 3 Atlas program, a set of three clinical trials testing fitusiran in patients with hemophilia. Results from those trials are expected in mid- to late 2019.
  • Alnylam finished its review of the phase 3 trial for revusiran, which was stopped because more patients who took revusiran died than those who took placebo, but the biotech hasn't been able to figure out why and blamed the discrepancy on bad luck. Nevertheless, the company isn't going to revive the revusiran program.
  • In May, Alnylam raised $355.2 million in a secondary offering, and partner Sanofi bought another $21.4 million worth of shares, helping pad the biotech's coffers.
Doctor talking to patient in exam room

Image source: Getty Images.

What management had to say 

Ionis Pharmaceuticals (NASDAQ:IONS) has a competing ATTR amyloidosis drug, inotersen, that passed its phase 3 trial in May. While competition isn't ideal, John Maraganore, Alnylam's CEO, pointed out that it gives the company confidence that the Apollo trial will be successful:

Moreover, we believe the potential clinical efficacy for a TTR-lowering mechanism of action was also validated by the recent positive top line phase 3 results for inotersen, an investigational antisense oligonucleotide targeting TTR [transthyretin, a hormone-transporting protein].

Pfizer (NYSE:PFE) also has an ATTR amyloidosis drug, Vyndaqel, approved for sale in Europe, but Barry Greene, Alnylam's president, also turned that into a positive:

The fact that Pfizer has been out there for a couple of years certainly helps our efforts because they have raised awareness. The biggest challenge they've had, frankly, is that because of the results, pricing hasn't been in line with orphan pricing and countries like the U.K. are not reimbursing them.

Looking forward

Apollo is obviously the most important near-term event for Alnylam, but the rest of the company's pipeline is progressing well. In addition to the aforementioned phase 3 hemophilia program for fitusiran, the company will start two more phase 3 programs this year: The first is an investigation of givosiran for a rare disease called acute hepatic porphyria; the second is partner The Medicines Company's (NASDAQ:MDCO) planned phase 3 program for the jointly developed cholesterol drug inclisiran.

With all the usual caveats about clinical trials and FDA approvals being risky, Alnylam looks well on its way to its goal of being a multiproduct commercial-stage company by 2020.

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