Alnylam Pharmaceuticals (ALNY 1.67%) released first-quarter earnings on Friday, but without any drugs on the market, investors are rightfully focused on the biotech's pipeline.

Alnylam Pharmaceuticals results: The only number that really matters


Q1 2017

Q4 2016

Quarter-Over-Quarter Change

Cash, cash equivalents, and fixed income marketable securities $962.2 million $1.09 billion (11.7%)

Data source: Alnylam Pharmaceuticals.

What happened with Alnylam Pharmaceuticals this quarter?

  • In February, Alnylam presented encouraging data for fitusiran in patients with hemophilia A or B at the meeting of the European Association for Haemophilia and Allied Disorders.
  • In March, at the American College of Cardiology meeting, Alnylam's partner The Medicines Company (MDCO) presented positive data from the ORION-1 phase 2 trial of inclisiran. The drug reduced LDL cholesterol -- that's the bad kind -- by over 50%, and sustained this for six months.
  • Last month at the American Academy of Neurology meeting, Alnylam presented extended data from the phase 2 trial for its lead drug patisiran. At 24 months, patisiran showed a mean decrease in the modified neuropathy impairment score (mNIS+7) of seven points, compared to an expected mean increase in mNIS+7 of 26 to 30 points at 24 months, based on historical data of untreated patients.
Doctor and patient

Image source: Getty Images.

What management had to say

Alnylam is developing a few drugs that have potential competitors being developed by rival drugmakers, putting management out in full force defending its position.

CEO John Maraganore was more focused on the potential for Ionis Pharmaceuticals' (IONS 4.56%) IONIS-TTRRx to fail, and the read-through that it would have for patisiran, than on potential competition if they're both approved:

While we do expect [the Ionis] study to be generally positive, confirming the TTR-lowering hypothesis, there is certainly a chance for mixed or even false-negative results based on their smaller sample size, shorter study duration, use of a co-primary endpoint structure that includes quality of life, potentially higher discontinuation rates due to adverse events or other factors, and slower, less robust TTR lowering effect, among others. Accordingly, a potentially mixed or negative Ionis study outcome needs to be viewed in the context of their drug and their study, not ours.

Maraganore is essentially making a "heads I win, tails you lose" argument.

When asked about the hemophilia space, Maraganore also took on Roche's emicizumab (ACE910), and gene therapies such as the one BioMarin Pharmaceutical is developing, which will potentially compete against fitusiran:

Of course, there is nothing in heme B like fitusiran, because emicizumab, or ACE910, is limited to patients with heme A, and of course both emicizumab and fitusiran are going to be very meaningful for patients with inhibitors, and of course that's not a realm where gene-therapy-type products will emerge. I am of the view that gene therapy is going to be important in this space. I don't think it's going to have a very rapid uptake. There is going to be a number of barriers to entry that relate to long-term safety and variability of response. But the data are in fact promising, and I think that gene therapy will emerge as one of the treatment options out there.

Looking forward

Data from the APOLLO phase 3 trial for patisiran is expected in the "September time frame," according to management. Assuming positive results, the company plans to submit marketing applications to U.S. and EU regulators at the end of this year.

Beyond patisiran, Alnylam plans to start three phase 3 trials this year.

The ATLAS phase 3 program will include three separate trials testing fitusiran in hemophilia patients with or without inhibitors, with the first trial expected to start by the end of this quarter.

The phase 3 trial for givosiran to treat porphyria is expected to start in late 2017. Investors will get more information about the potential for this drug next month, when additional phase 1 data is presented at the International Congress on Porphyrins and Porphyrias.

Finally, The Medicines Company plans to start a phase 3 study testing inclisiran in patients with atherosclerotic cardiovascular disease in the middle of this year, with data expected in 2019.