Alnylam Pharmaceuticals Inc at Piper Sandler Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Okay. So you guys have been done a great job growing AMVUTTRA under the current ATTR polyneuropathy label. Jeff, what's been driving this? And maybe you can remind us on sort of what the guidance is for this year?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Yes. It's a great therapy. And congrats on the HELIOS-B data. Maybe you guys can remind us of what you reported and sort of, obviously, we now have a PDUFA date. So maybe tell us sort of what your expectations are into that?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Yeah, very compelling data set.

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : So that's a great segue into my next question. How much larger is the hereditary and wild type ATTR cardiomyopathy market? And how do you guys envision competing with Pfizer's established tafamidis, the stabilizer? And also the BridgeBio's new recently approved acoramidis?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : That's really, really helpful. So I'm going to just pause and see if there's any questions. At AHA you guys reported Phase 1 data on nucresiran.

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Yes. I haven't pronounced that one yet or ALN-TTRsc04

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Yes, sc04. How do you pronounce it?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Nucresiran. There we go. Thank you. So remind us what you presented. And where does this fit into sort of product life extension for the TTR franchise?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : And I think you guys have guided that you'll provide Phase 3 design early next year at some point. So we'll look forward to that. And also, I was just thinking, but in a way, a patient I don't even think we'll know, they'll go from getting it once every four times a year to getting it to, or once a year and it'll probably be barely even know that they're on a new medicine, so that seems really logical. I want to switch gears because I think one of the biggest trends that we've seen with the safety and now obviously, the long-term durability with RNAi therapeutics is just we move from what a predominantly orphan diseases to really much broader diseases. And I think, likely with Novartis is sort of the quintessential example that kind of started that. I want to talk about zilebesiran with Roche, which I think is a really, really compelling therapy. Maybe you can kind of tell us about this target and some of the data that you've reported? And remind us why did it make sense to partner this one with Roche? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 4:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc at Piper Sandler Healthcare Conference

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Yeah. Absolutely. And you said the cost -- you're only on the hook for 30%.

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : And 50-50 in the US. That's great

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : So I definitely want to get to the pipeline and one of them you guys have this big partnership with Regeneron right now. One thing I've never really gotten a good explanation, how does sort of candidate selection go there? Like how are you guys working with Regeneron? Do you both sort of put targets in that you want to work on? Does that evolve over time? Is there a picking strategy? How do you maybe you can share with us a little bit how that works?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Excellent. That's really helpful. One of the programs that fits under this that I think is really interesting is known as CRM. And you guys just started the Phase 2 Capricorn 1 study in cerebral amyloid angiopathy. I think you have plans to start a Phase 2 in Alzheimer's disease. Maybe you can describe the why this RNAi mechanism may prove superior to antibodies going after these targets and what data have you reported today?

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : That's really exciting. I like that program a lot. Jeff, you mentioned where we've got a story here with growing top line. I think you've done a really good job containing costs, driving the profitability. But yet it's still very robust investment and the expansion of the pipeline. I think you mentioned 9 wholly owned and as many as 13 partnered INDs over the next 18 months or so. Just kind of looking at the balance sheet, you guys had cash of about $2.4 billion old debt, we think of just over $1 billion. How do you think about funding the company with all of this going on? Is this -- do you have enough cash right now to reach profitability? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 4:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc at Piper Sandler Healthcare Conference

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : Okay. Well, Jeff, Sandeep, thank you for being with us. Excited for upcoming approval, and the launch, and just continued pipeline expansion. Thanks for being here.

Question: Ted Tenthoff - Piper Sandler Companies - Analyst : February 25, '25 REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 4:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc at Piper Sandler Healthcare Conference