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Alnylam Pharmaceuticals, Inc. Cranks Out the Data

By Brian Orelli, PhD - Aug 12, 2016 at 4:10PM

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The development-stage biotech may not have any earnings, but it's producing plenty of data to keep investors interested.

Image source: Getty Images.

There isn't much to say about Alnylam Pharmaceuticals' ( ALNY 3.29% ) second-quarter earnings -- or lack thereof -- but it certainly was a data-packed quarter for the development-stage biotech, with more clinical trial results expected in the second half of the year. For the record, the company lost $90.1 million, or $1.05 per share, on a GAAP (generally accepted accounting principles) basis.

Alnylam Pharmaceuticals results: The only metric that really matters


Q2 2016 Actuals

Q4 2016 Actuals

Cash Decline for First Half of 2016

Cash, cash equivalent, marketable securities, and restricted investments

$1.28 billion

$1.28 billion


Data source: Alnylam Pharmaceuticals press release.

What happened with Alnylam Pharmaceuticals this quarter?

  • The company didn't actually burn through $0 in the first half of the year, but the coincidentally identical cash after two quarters likely has to do with the loan Alnylam took to build its new manufacturing facility. Nevertheless, management is guiding for having more than $1 billion on hand at the end of this year, including the $150 million of restricted investments that are securing the loan.
  • On the data front, Alnylam presented relatively positive data for both patisiran and revusiran that treat different aspects of transthyrethin-mediated amyloidosis.
  • Separately, Alnylam presented data for its hemophilia treatment, fitusiran. While the data only come from a phase 1 study, Alnylam thinks they are strong enough to move straight into a phase 3 trial.
  • And then there's ALN-CC5, which appears to be helping patients with paroxysmal nocturnal hemoglobinuria (PNH) who aren't sufficiently helped by Alexion Pharmaceuticals( ALXN ) already-approved Soliris. It's disappointing that ALN-CC5 won't be used as a first-line treatment, but at least Alnylam might be able to capture the estimated 20% to 30% of PNH patients whose symptoms aren't adequately controlled on Alexion's Soliris.

What management had to say 

Management gave a few more details on the new manufacturing plant in Norton, Massachusetts, including that the drug-substance part of patisiran, which could be approved in 2018, will be outsourced to contract manufacturing organizations (CMOs). "The Norton facility really starts approaching the pipeline needs beyond patisiran. And that is planned to be operational in 2018, and we'll still use third-party CMOs and leverage our third-party CMOs as well, but increasingly over time, the Norton facility will be doing the bulk of our drug-substance manufacturing," said Alnylam's CEO John Maraganore.

Beyond hemophilia A and B, Maraganore pointed out that fitusiran could be used for other congenital deficiencies of coagulation factors, and potentially in some platelet disorders. "And we're quite interested in these opportunities. Obviously, these are areas of very high unmet need. There really are no competing drugs in those settings, and we're going to begin to explore some of those studies as early as next year, but it's too soon to give you any more specific details on that at this time," Maraganore said.

Looking forward

By my count, six of Alnylam's 10 clinical-stage drugs will have some sort of data readout in the second half of this year. Of those, arguably the most important will be the phase 2 data for ALN-PCSsc, from a program being run by Alnylam's partner The Medicines Company ( MDCO ). At the American Heart Association meeting in November, investors will get a first look at the proof-of-concept study for ALN-PCSsc, which targets PCSK9. The drug could be dosed as infrequently as twice a year, allowing Alnylam and The Medicines Company to compete with PCSK9 antibodies already on the market.

Of course, Alnylyam's biggest value-inflection point won't come until the middle of next year, when patisiran's Apollo phase 3 trial reads out. If it's successful, the company plans on submitting the data to regulators by the end of next year, for a potential launch in 2018.

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis – even one of our own – helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Stocks Mentioned

Alnylam Pharmaceuticals, Inc. Stock Quote
Alnylam Pharmaceuticals, Inc.
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Alexion Pharmaceuticals, Inc. Stock Quote
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