Alnylam Pharmaceuticals (NASDAQ:ALNY) released first-quarter results last week, but all eyes were looking forward as the company waits for a potential approval of its hereditary TTR amyloidosis (ATTR) drug, patisiran.

Alnylam results: The only number that really matters


Q1 2018

Q4 2017

Quarter-Over-Quarter Change

Cash, cash equivalents, and fixed income marketable securities

$1.57 billion

$1.70 billion

($136 million)

Data source: Alnylam.

What happened with Alnylam this quarter?

  • Without any drugs on the market -- yet! -- revenue and earnings aren't particularly important to Alnylam. The only thing that really matters is its cash runway, which looks plenty long enough to get patisiran launched. The company is shooting for having approximately $1 billion in cash, cash equivalents, fixed income marketable securities, restricted cash, and restricted investments at the end of the year.
  • Speaking of patisiran, the company still expects an FDA decision on or before its prescription drug user fee act (PDUFA) date of August 11.
  • Alnylam's next most advanced drug, givosiran, which treats a rare disease called acute hepatic porphyrias, is in a phase 3 trial with data due out in September.
  • Behind that, lumasiran, which treats another rare disease called primary hyperoxaluria type 1, is ready to enter phase 3 development in the middle of this year after the company recently reached an agreement with the FDA on the clinical trial design.
  • Alnylam's partner, The Medicines Company (NASDAQ:MDCO), has fully enrolled its phase 3 trials for their cholesterol drug inclisiran, with data expected in 2019.
Doctor talking to patient in exam room

Image source: Getty Images.

What management had to say

Pfizer (NYSE:PFE) threw Alnylam and fellow ATTR drugmaker Ionis Pharmaceuticals (NASDAQ:IONS) a curveball in March, releasing positive results from a trial testing its ATTR drug tafamidis in patients with ATTR cardiomyopathy. But Alnylam isn't backing down with potential plans to run a trial comparing its next-generation TTR drug, ALN-TTRsc02, against tafamidis, as outlined by Akshay Vaishnaw, Alnylam's executive vice president of research and development:

Regarding the wild-type ATTR opportunity, we'll need to see the complete results from Pfizer's ATTR-ACT study with tafamidis later this year, but we're now considering the potential for a head-to-head study comparing TTRsc02 with tafamidis in the setting of ATTR cardiomyopathy, including wild-type disease.

Barry Greene, Alnylam's president, continued to stress that multiple companies developing drugs for ATTR should result in a larger market of potential patients, negating much -- if not all -- of the loss of market share to competitors:

We believe that having other companies actively working to raise disease awareness, shorten the patient journey, and improve patient diagnosis will result in an expansion of the overall market in a way that should ultimately increase the number significantly of patients diagnosed.

Looking forward

It's just a waiting game for Alnylam at this point. With that in mind, investors should remember that the PDUFA date is just a goal, and the FDA often makes decisions early, especially for diseases like ATTR, where there aren't any approved treatments. The lack of a planned advisory committee meeting, where the FDA seeks advice from outside experts, is also a good sign that a decision could come early.

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.