Alnylam Pharmaceuticals Inc Q4 2024 Earnings Call Transcript

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

Question: Tazeen Ahmad - Bank of America - Analyst : I wanted to get a sense from your team about how long you think it's going to take to establish payer policies after the approval of -- and how do you think that could impact the initial trajectory of the launch?

Question: Tazeen Ahmad - Bank of America - Analyst : Konstantinos Biliouris, BMO Capital Markets.

Question: Huidong Wang - Barclays - Analyst : I know you cannot comment on the actual price for vital into the approval day, but wondering if you can share the key factors that impact your decision on pricing adjustment for AMVUTTRA. And related question, I know a few already asked, can you show with us what percentage of the current sites already have a coating place that you can use for AMVUTTRA in ATTR cutting ops?

Question: Maurice Raycroft - Jefferies - Analyst : Congrats on the progress. Just wondering if there's any perspective you can provide on label discussion progress. And in addition to reduction of mortality and hospitalizations, are you advocating to have anything unique on the front page of the label and the indication and usage section such as wording related to preservation and quality of life and functional capacity. Pushkal Garg - Alnylam Pharmaceuticals Inc - Chief Medical Officer, Executive Vice President - Development and Medical Affairs Yes. Thanks, Maurice. Look, first of all, we're really very enthusiastic about how the FDA review is going and we remain fully on track and for an approval date by the PDUFA. So we're excited about the progress that's been made on that front. In terms of the label, look, we believe that the study was designed and clearly demonstrated the benefit on mortality and hospitalization events. And we expect that that will be reflected in the label as well as the ancillary benefits that we saw in terms of preservation of functional ability, quality of life and benefits across a series of endpoints there. So look, at the end of the day, we want a label that reflects the study that was designed and where we saw statistically significant results in predefined endpoints across a whole series of attributes that we think are a benefit and that we're able to actually then go and educate providers and patients on so we look forward to sharing that when an eventual approval occurs.

Question: Ritu Baral - TD Cowen - Analyst : My question was actually a really good follow-up to Maurice on label language. To you think about the language in the label, specifically mortality but potentially having that mortality clinical data later in the label in the clinical data section. What sort of potential advantage could that give you or not, either detailing the product or with payers?

Question: Ritu Baral - TD Cowen - Analyst : Do you still expect that to be in the actual labeling language though? Those data? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 13, 2025 / 1:30PM, ALNY.OQ - Q4 2024 Alnylam Pharmaceuticals Inc Earnings Call Pushkal Garg - Alnylam Pharmaceuticals Inc - Chief Medical Officer, Executive Vice President - Development and Medical Affairs Look, at the end of the day, we think that the label will reflect the things that we've shown in the clinical trial. That's traditionally what is done, and we would expect this, as we've said, Ritu, that we had a whole series of prespecified endpoints that show the impact of this drug on this disease. And so we would expect that to be generally reflected in the label. And again, consistent with labeling, Tolga's team, our medical teams will be able to speak about those attributes and the results of HELIOS B study to providers and patients and payers.

Question: Gary Nachman - Raymond James - Analyst : Maybe you could talk about the competitive dynamics you're seeing with -- in polyneuropathy with both products growing at this point? And how is the difference between Part B and Part D playing out? And how you think that's going to translate over to cardiomyopathy with that approval.

Question: Jessica Fye - JPMorgan - Analyst : What do you think the early acoramadis scripts imply for the TTR cardiomyopathy market dynamics, market expansion and interest in agents beyond tafamidis as it relates to AMVUTTRA? And I think folks have been kind of trying to ask this in a few different ways, but maybe just to try one more time. What's the right way to think about the evolution of AMVUTTRA's net price as you move into this much larger patient population?

Question: Paul Matteis - Stifel - Analyst : On payer dynamics and how they evolve in this category, are you expecting the average insurer to cross manage between Part B and Part D? And I think, again, the underlying question that everyone is trying to get to on pricing is you're at a significant premium right now. Does it have to go down or not. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 13, 2025 / 1:30PM, ALNY.OQ - Q4 2024 Alnylam Pharmaceuticals Inc Earnings Call There's a lot of moving parts, right, like the payer liability on Part D is one of them. But just as it relates to how insurers are going to manage their formularies in this space, like could tafamidis or acoramidis actually be a step at it? Or is that just not something that's normal in practice at this point?

Question: Salveen Richter - Goldman Sachs - Analyst : You plan to initiate a Phase III study for Nucresiran in in ATTR-CM in the first half. How are you thinking about the timing of enrollment and events, given the availability of new options? And how do you plan to conduct an interim here?

Question: Eliana Merle - UBS - Analyst : Just a couple for me. So how would you characterize the mix of growth in the TTR guidance from polyneuropathy versus cardiomyopathy and then Tolga, you mentioned that post approval, you'll be finalizing value-based agreements in cardiomyopathy, can you elaborate a bit on how these could work and what this could look like for cardiomyopathy relative to polyneuropathy?

Question: Mani Foroohar - Leerink Partners - Analyst : Congrats on great results commercially and otherwise. I know that there's probably two more questions about AMVUTTRA and pricing in the line. So I'm going to ask actual financial question. Could you clarify the driver of the tax benefit that was noted here? And would that be recurring? Is this an NOL dynamic OUS, if you could just dig into what drove that benefit and how it might recur or not flash over the course of the year?

Question: Luca Issi - RBC Capital Markets - Analyst : Great. Maybe I'll follow Mani here I guess. But Jeff, how should we think about gross margin here going forward? I mean the same of the oral TC is very meaningful at 15%, 30%, but we obviously don't know the exact hearing of the royalty fee. Do you think that modeling gross margin compressing from 80% now, I think it's a low 80s now to like low 70s, so even upper 60s. Is that a reasonable assumption in your mind. Any call there much appreciated.

Question: Luca Issi - RBC Capital Markets - Analyst : That's super helpful.