Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) Q4 2022 Earnings Call Transcript

BNP: As an example, three out of the five patients in the 50 mg biannual regimen who passed away had glue 89 glen and Serine 77 mutations, which are particularly aggressive, so make the causes too, some CB, but some were non-CB. As an example, one of the patients who passed away in the biannual regimen was diagnosed with acute myeloid leukemia and died after chemotherapy. And then we looked you know, there was no relationship to pharmacokinetics or as you were asking pharmacodynamics a knocked down that we observed. So we’ll provide more details in upcoming scientific meetings. But we’re very reassured from the detailed safety analysis, which did not, did not to our minds seem to be any relationship to the drug.

Myles Minter: Right. Thanks.

Operator: One moment for the next question, and your next question comes from the line of Joseph Stringer from Needham and Co. Your line is now open.

Unidentified Analyst: Good morning. This is Barbara on for Joey, and our question is about the big picture. Can you give us a sense of what you think ONPATTRO and AMVUTTRA’s combined market penetration of the TTR polyneuropathy and mixed phenotypes is at this time?

Yvonne Greenstreet: Yes, I think, I think just kind of taking a step back and thinking about the TTR market as a whole, I mean, there’s no doubt that it’s a rapidly growing market with increased physician awareness, increased diagnosis of patients, and obviously that’s translating into increased treatment of patients. So we are very excited that we have two RNAi therapeutics participating and helping patients who have ATTR Amyloidosis at the moment with polyneuropathy, and then of course, hoping to extend the label to patients who also have cardiomyopathy. We think it’s a market that also has room for multiple players. It really is all about kind of market growth rather than market share. And I think what’s particularly pertinent with respect to our portfolio is, we really see our portfolio driving growth overall going forward for Alnylam.

So, you know, we’re very pleased with our progress thus far. And you know, we look to continue helping physicians, treat patients as appropriate with both ONPATTRO and AMVUTTRA. I mean, Tolga is there anything you want to add in terms of just stepping back and thinking about the TTR market?

Tolga Tanguler: Yes, I mean, along the same lines we’re very pleased to be able to provide now we do have a franchise. We have two products that are available in the US? and soon for the rest of the world in polyneuropathy. And what we know is despite the fact that it’s a rare disease, there is still significant unmet need, it’s a devastating disease. If the patients are not treated, they do end up with mortality care mobility in a timeframe of five to seven years. And despite that, diagnosis and treatment is still relatively low numbers. And what AMVUTTRA launch has demonstrated to us is the fact that having a very compelling product profile and with the convenience of subcutaneous injectable every quarter really does change the game in terms of how it allows us to be able to reach more patients through by actually finding more, more physicians.

So we do have this prevalence number that’s anywhere between 40,000 to 50,000 across the world, including the mixed genocide patients. We’re still scratching the surface and as Yvonne indicated with these products coming into the market will allow us to actually increase the awareness, both on the physician side as well as on the patient’s side. And the most recent launch of AMVUTTRA clearly demonstrates that.

Yvonne Greenstreet: Thanks, Tolga. So we’ve got time for one last question.

Operator: One moment for the last question, and your last question comes from the line of Eliana Merle with UBS. Your line is now open.