Even though biotech Alnylam Pharmaceuticals (NASDAQ:ALNY) doesn't have any drugs on the market, it had a solid third quarter in terms of pipeline development, with a drug passing its phase 3 trial, joining two others that are already in phase 3 development.

Alnylam results: The only number that's really important


Q3 2017

Q4 2016


Cash, cash equivalents, fixed income marketable securities, and restricted investments

 $1.15 billion

$1.09 billion

$141 million

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam this quarter?

  • Alnylam is burning a significant amount of cash -- the adjusted net loss was $97 million for the quarter -- but it still has a substantial nest egg after raising about $360 million in May. After the quarter ended, the company raised another $700 million to pad the coffers even more.
  • Patisiran, Alnylam's lead program that treats hereditary ATTR amyloidosis with polyneuropathy, passed the Apollo phase 3 trial, putting it on track to be submitted to the U.S. Food and Drug Administration and EU regulators by the end of the year.
  • Givosiran, which treats another orphan disease, acute hepatic porphyrias, entered a phase 3 trial called Envision, which will have an interim readout in the middle of next year. Alnylam thinks that data will be enough to gain FDA approval.
  • Inclisiran, a cholesterol-lowering drug Alnylam is developing with The Medicines Company (NASDAQ:MDCO), is already in phase 3 development, but given the large number of patients -- about 3,400 across all the trials -- it'll be a while until enrollment is complete and data are available for readouts.
  • Fitusiran, which treats hemophilia, had a setback when a patient died in the clinical trial, but Alnylam met with the FDA and announced last week that it has established protocol changes that will allow the trial to restart later this year.
Doctor talking to patient in exam rooom

Image source: Getty Images.

What management had to say

The interim look at the Envision trial will measure a biomarker called aminolevulinic acid (ALA) rather than a clinical outcome auch as the rate of porphyria attacks that the complete study will look at. But Akshay Vaishnaw, Alnylam Pharmaceuticals' executive vice president of research and development, assured investors that the FDA has signed off on using ALA as a surrogate endpoint: "We have an agreement with regulators and we should comfortably hit the necessary biomarker level."

The FDA appears to be convinced, based on Alnylam's earlier-phase data, that ALA is likely to predict outcomes since, according to CEO John Maraganore, there's no requirement for outcomes data at the interim look: "We'll submit all of the data we have at that point in time, both safety and efficacy data, as part of the interim analysis, but there's no requirement for, based on our discussions, for impact on those other measures as it relates to the interim analysis."

EU regulators haven't signed off on the interim analysis, but Vaishnaw seemed certain they eventually would: "We're still putting the finishing touches on that, but we're confident that we can have a consensus understanding of the endpoint we need to show for ALA and we're confident we can reach that."

Looking forward

Barring some manufacturing issue, patisiran should get approved next year given the rock solid efficacy data for a disease with an unmet need. But Alnylam is going to have to hit the ground running because rival Ionis Pharmaceuticals (NASDAQ:IONS) is slightly ahead, having already submitted marketing applications for inotersen to U.S. and EU regulators.

The follow-on version of the patisiran, ALN-TTRsc02, looks to be substantially more potent, with data supporting treatment once every three to six months.

Beyond patisiran, Alnylam has to continue executing on its pipeline development. Fortunately, with $1 billion in the bank, the biotech has plenty of cash to support development as it ramps up sales of patisiran.