On the brink of gaining approval for its first drug, Alnylam Pharmaceuticals (NASDAQ:ALNY) reported fourth-quarter financial results and updated investors on its stable of RNAi drugs. There was no news-braking clinical trial data during the announcement, but it's good to hear that the company's making progress on its lead drug as well as its pipeline.

Alnylam results: The raw numbers 

Metric

Q4 2017

Q4 2016

Year-Over-Year Change

Revenue

$37.9 million

$17.4 million

118%

Income from operations

($142 million

($113 million)

N/A

Earnings per share

($1.48)

($1.32)

N/A

Data source: Alnylam.

What happened with Alnylam this quarter?

  • All of the revenue comes from collaborators, so don't get too excited about the jump in revenue, especially since much of it was already received as upfront payments that are amortized over the life of the collaboration.
  • Alnylam presented solid results from the Apollo phase 3 trial for patisiran, which treats hereditary ATTR amyloidosis. The data has been submitted in marketing applications to FDA and EU regulators.
  • The company started the Envision phase 3 trial testing givosiran in patients with acute hepatic porphyrias.
  • Alnylam and Sanofi (NYSE:SNY) restructured their 2014 deal with Alnylam getting full rights to patisiran and the follow-on drug ALN-TTRsc02 and Sanofi gaining full rights to their hemophilia drug fitusiran, although both companies will benefit from the drugs they gave up through royalties on sales. Sanofi has jumped full into hemophilia with the acquisition of hemophilia expert Bioverativ last month, which could benefit Alnylam with larger royalties on fitusiran in the future.
  • Alnylam's partner, The Medicines Company (NASDAQ:MDCO), is making good progress with the phase 3 program for inclisiran. The large trials have completed enrollment ahead of schedule, suggesting patients are excited about the potential to only be treated once or twice a year for their high cholesterol.
  • Further back in the pipeline, lumasiran, which used to go by ALN-GO1, looked good in a phase 1/2 trial in patients with primary hyperoxaluria type 1. The drug is part of the pact with Sanofi, which will have to decide in the next few months whether it'll opt in and assume development of the drug.
A scientist working in a lab.

Image source: Getty Images.

What management had to say 

Alnylam's president, Barry Greene, talked a little bit about pricing of patisiran, suggesting that the company would be willing to enter value-based contracts with insurers. Those are often structured with insurers getting lower priced (or free) drugs if the treatment doesn't help the patient, but can result in less bickering over the price:

"With respect to pricing, we're reviewing analogues to other therapies for rare conditions and high unmet needs. As part of our patient access principles, we plan on being proactive on value-based agreements which certain payers have already indicated are of considerable interest."

Akshay Vaishnaw, executive vice president of research and development, laid out phase 3 plans for ALN-TTRsc02, the aforementioned follow-on drug to patisiran, which will start in late 2018:

"We're planning on a comprehensive phase 3 development program that will include studies aimed at confirming safety and efficacy in patients with hATTR amyloidosis, in other words the APOLLO population, and also in patients who are asymptomatic hATTR mutation carriers and the patients with wild-type ATTR amyloidosis. These two later patient groups represented the potential to substantially expand the number of patients who may benefit from TTR knockdown with our investigational RNAi therapeutic TTRsc02."

Looking forward

The FDA has a goal of making a decision on the marketing application for patisiran by Aug. 11, but given the unmet need, the decision could come earlier.

Around that same time, in the middle of the year, interim data from the Envision phase 3 trial is due out. The results could be good enough to gain accelerated approval for givosiran, although Alnlylam will still have to complete the trial to gain full approval.

Alnylam ended 2017 with $1.7 billion in the bank and expects to end 2018 with approximately $1 billion in cash, suggesting it'll be awhile before profits start rolling in.

Brian Orelli has no position in any of the stocks mentioned. The Motley Fool owns shares of and recommends Alnylam Pharmaceuticals. The Motley Fool has a disclosure policy.