Alnylam Pharmaceuticals, Inc.'s Five-Year Plan

As with most development-stage biotechs, Alnylam Pharmaceuticals' first quarter earnings press release and conference call was focused on the pipeline progress rather than the profit-loss statement.

During the quarter, the biotech launched its Alnylam 2020 plan for the next five years. By the end of this decade, Alnylam expects to have three drugs on the market and 10 drugs in the clinic. Four of those drugs are expected to be in the later stage of development.

Alnylam is well on its way with five drugs already in the clinic. It presented data for two of them this quarter.

In a phase 2 open-label extension trial testing its drug patisiran in Familial Amyloidotic Polyneuropathy, or FAP, patients' neuropathy progression appeared to be halted for a full year of treatment. Investors usually have to be careful with open-label trials since doctors and patients both know that they're taking the drug, but it's less of a worry given the strong data. Patients taking the drug had a mean 2.5 point decrease in modified Neuropathy Impairment Score compared to a 13 to 18 point increase that would be expected for untreated patients over that timeframe; the magnitude of the difference seems unlikely to be due to a placebo effect.

Data from Alnylam's phase 1 trial testing ALN-AT3 in hemophilia comes from just three patients, but were impressive none the less. The drug knocks down the expression of antithrombin, which in turn increases thrombin that causes blood to clot. In the clinical trial, thrombin generation increased up to 334%, and patients saw a marked improvement in whole blood clotting. New patients in the trial will get the dose as a once-monthly shot. If ALN-AT3 is equally effective at that dose, it would be a marked improvement from the infusions hemophilia patients now have to get every few days.

Looking forward, Alnylam has a couple of presentations at medical conferences coming up including more data from the ALN-AT3 trial in patients with hemophilia at the International Society on Thrombosis and Haemostasisin June. Towards the middle of the year, we'll get data from the phase 1 trial testing ALN-PCSsc, which is designed to decrease cholesterol, a potentially large market. And its sixth drug, ALN-AAT for alpha-1 antitrypsin deficiency-associated liver disease, should enter the clinic this year.

For the record...Alnylam had $18.5 million in revenue thanks to its collaborations with Monsanto,Takeda Pharmaceuticals, The Medicines Company, Sanofi, and others, although much of that was amortized revenue from payments received many quarters ago and won't be as high in subsequent quarters of 2015 because some of the amortizations plans wrap up this year.

The biotech recorded a loss of $51 million in the second quarter; don't expect the company to be profitable until it gets its first drug approved, and even then it might take a second drug to get to profitability given its ambitious R&D plans.

Fortunately Alnylam has plenty of cash for the near-term; it ended the quarter with $1.45 billion and expects to burn through less than $250 million of that in the remaining three quarters of the year.

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