Alnylam Pharmaceuticals (ALNY -0.52%) reported third-quarter earnings on Monday, and like most development-stage biotechs, there was more focus on the pipeline than the dollars and cents.
Alnylam Pharmaceuticals results: The raw numbers
| Metric |
Q3 2015 Actuals |
Q3 2014 Actuals |
Growth (YOY) |
|---|---|---|---|
|
(Loss) from continuing operations |
($78 million) |
($45 million) |
N/A |
|
(Loss) per share |
($0.91) |
($0.58) |
N/A |
|
Cash |
$1.34 million |
$915 million |
46% |
Source: company press release.
What happened with Alnylam Pharmaceuticals this quarter?
- Alynalam presented data from the 18-month update for the patisiran phase 2 open-label extension in transthyretin (TTR)-mediated amyloidosis. The data still look good with evidence for potential halting of neuropathy progression.
- Apollo, the phase 3 trial for patisiran, should be fully enrolled in the next three to four months, putting Alnylam on track to submit a marketing application to the FDA in 2017.
- Alnylam also presented six-month data from its revusiran phase 2 open-label study in familial amyloidotic cardiomyopathy. Patients' walking ability hasn't decreased over the first six months of treatment.
- The phase 3 trial for revusiran, Endeavour, continues to enroll patients, but management hasn't given guidance on when it expects the trial to complete enrollment.
- In addition to patisiran and revusiran, Alnylam Pharmaceuticals has five other drugs in earlier stage development and plans for two more to enter the clinic by the end of the year. You may recall that the company had a 5x15 plan to have five drugs in the clinic by the end of 2015, which it's going to blow past.
- With $1.34 billion in the bank and plans to end the year with more than $1.2 billion, Alynlam has plenty of money to advance those programs.
What management had to say
John Maraganore, Alnylam's CEO commented on the biotech's success record, "We now have reported positive human data in all six of the clinical programs for which we've presented results to date." Much of the success can be attributed to the RNAi platform. As long as the company picks the right targets, the knockdown of the protein levels should be similar for Alnylam's drugs.
Akshay Vaishnaw, chief medical officer and executive vice president of R&D, highlighted a comparison of data for patisiran: "Based on recent competitor data where it was reported that a 76% mean maximal TTR knockdown was achieved after 10 months of dosing. We're pleased with the 93% mean maximal effect achieved for patisiran within just a few days of dosing."
Vaishnaw didn't call out Isis Pharmaceuticals (IONS -0.32%) and GlaxoSmithKline (GSK 1.22%) by name, but it's pretty clear he was talking about Isis Pharmaceuticals' ISIS-TTR Rx that was licensed to GlaxoSmithKline. While Alnylam appears to have the advantage in terms of knockdown of protein, it's possible there may be diminishing returns as the protein levels are lowered beyond a certain point.
Isis Pharmaceuticals and GlaxoSmithKline plan to complete enrolment this year, and their phase 3 trial is slightly shorter than Alnylam's trial -- 65 weeks versus 78 -- potentially giving the duo a slight lead to get on the market. The phase 3 efficacy and safety data -- rather than the protein knockdown or the speed to market -- will probably determine which company eventually wins the competition for patients.
Looking forward
There will be plenty of data to digest this quarter with data on ALN-PCSsc being presented at the American Heart meeting, ALN-HBV data at the Liver Meeting, and new data from ALN-AT3 in hemophilia and ALN-CC5 in complement diseases at the American Society of Hematology meeting in December. If you miss any of those, investors can get a recap on December 10 when Alnylam is hosting an R&D day to provide more information about the company's pipeline.
