Arrowhead Pharmaceuticals at Goldman Sachs Conference: Strategic Launch Plans

On Monday, 09 June 2025, Arrowhead Pharmaceuticals (NASDAQ:ARWR) presented at the Goldman Sachs 46th Annual Global Healthcare Conference, highlighting its strategic initiatives and upcoming commercial launch plans. The company is poised to introduce plazasiran, a triglyceride-lowering drug, amidst a competitive landscape, while leveraging a robust partnership with Sarepta to ensure financial stability. The conference underscored both the opportunities and challenges as Arrowhead navigates the path to commercialization.

Key Takeaways

• Arrowhead is preparing for its first independent commercial launch with plazasiran, targeting familial chylomicronemia syndrome (FCS) initially.
• The partnership with Sarepta has fortified Arrowhead’s financial position, funding operations into 2028.
• Plazasiran offers potential advantages over existing treatments, including greater triglyceride reduction and less frequent dosing.
• The company is actively developing a diverse pipeline, including obesity and CNS-targeted programs.
• Arrowhead is focused on business development, seeking additional partnerships and licensing agreements.

Financial Results

• Arrowhead’s financial stability is significantly bolstered by the Sarepta partnership, with funding secured through multiple potential commercial launches up to 2028.
• Key upcoming catalysts include the plazasiran PDUFA date in November 2025 and the completion of Phase III enrollment for severe hypertriglyceridemia (SHTG) studies by mid-2025.
• Data readouts are anticipated later in 2025 for obesity assets and other partnered programs.

Operational Updates

• Plazasiran is set for a potential launch, with a PDUFA date scheduled for November 18, 2025, initially targeting FCS and subsequently SHTG.
• The Sarepta deal has strengthened Arrowhead’s balance sheet, facilitating early-stage pipeline development.
• Arrowhead’s obesity programs, ELK7 and Hippie, are progressing, with clinical data expected in late 2025 and early 2026.
• The company is advancing its CNS platform, with a program targeting tau expression expected to enter the clinic within the next 6-12 months.

Future Outlook

• Arrowhead is gearing up for the plazasiran launch, focusing on building the necessary commercial infrastructure and evaluating pricing strategies against competitors like Ionis.
• The SHTG market presents a multi-billion dollar opportunity, with plans for a new SKU upon launch.
• The PCSK9-APOC3 dimer is being developed to address mixed hyperlipidemia, with clinical trials anticipated before the end of 2025.
• The company is exploring combination therapy for obesity, potentially integrating with GLP-1 agonists.

Q&A Highlights

• Plazasiran is differentiated by its greater triglyceride reduction and less frequent dosing compared to competitors.
• Arrowhead is studying both genetically and phenotypically defined patients to optimize labeling.
• The SHASTA-five pancreatitis study aims to demonstrate a clear benefit in reducing pancreatitis rates, with a focus on European payers.
• Various pricing strategies are under consideration for FCS and SHTG, including parity or premium pricing.

In conclusion, Arrowhead Pharmaceuticals is strategically positioned for its upcoming commercial endeavors, supported by a strong financial foundation and a diverse pipeline. For a detailed account of the conference call, please refer to the full transcript below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Thanks, everyone, for joining us. I’m Andrea Newkirk, one of the biotech analysts here at Goldman Sachs, and I’m really pleased to be joined by James Hamilton, CMO and Head of R and D as well as Vince Ambulone, VP of Finance and IR of Arrowhead. Thanks so much, guys, for joining us.

James Hamilton, CMO and Head of R and D, Arrowhead: Thank you. Thank you.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: So, maybe we can start with a big picture question here. Over the course of the last year, you’ve done a lot to be able to augment your balance sheet, which has been one of the key priorities here. But you’re also getting much closer to a potential first commercial product. Maybe talk to us here about, as you look through the remainder of 2025, how you’re thinking about program prioritization, the near term catalysts that you see on the horizon for 2025 as well as into 2026? And what drives your confidence heading into these key events?

Vince Ambulone, VP of Finance and IR, Arrowhead: Sure. So I can take some of those and then pass it over to James. Thanks to Goldman and everybody here for

James Hamilton, CMO and Head of R and D, Arrowhead: having us today.

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes, I think that you’re right. The recent period has been really important for Arrowhead. And if you look at our Us historically, we have always we have been building to the point where we are right now, which is approaching a first commercial launch. Plazasiran has the PDUFA date in November. I think it’s November 18.

So that will be our first independent commercial launch, which is a really critical thing for us. And just to review, plazasiran is a triglyceride lowering drug. The first indication we’re seeking approval for is for FCS, which is a rare disease where we showed really great data from our PALISADE Phase III study. The next larger indication is severe hypertriglyceridemia where we have three Phase III studies ongoing, another which will start shortly. And the approval endpoint for that population is just triglyceride lowering.

We are approaching full enrollment for the Phase III studies somewhere in the middle of this year, which would enable a completion in the middle of next year and then a subsequent launch into SHGG after that. And so what have we done to kind of get ourselves prepared for that commercial launch? One, which is a really critical thing, is the deal that we announced and closed with Sarepta, which made our balance sheet very healthy. We’re now funded into 2028 and through multiple potential independent commercial launches and then subsequent to that, additional partnered program launching in the same time frame. And in the current backdrop of the current biotech environment, it’s it makes us feel very good and makes us sleep easily at night knowing that we are funded into 2028.

And beyond that, we have a really robust early stage pipeline across multiple different cell types and multiple different disease areas, both spans all the way from rare disease up to very high prevalent obesity assets. Some of these will be reading out later this year, the obesity assets in particular. Some of the programs we have partnered with Sarepta targeting rarer disease rare muscular diseases have readouts later this year. And so we feel like we are we have the most diverse pipeline we have ever had and also the most stable we’ve ever been financially. So, we feel like we’re in a really good position today.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Maybe let’s start with Flowsaster and then to your point, PDUFA, November eighteenth here. Just maybe given all the noise around everything that’s going on with the agency and policy, maybe we can first start, how have your conversations been going? Do you see any risk to the PDUFA date at all? Just would love to hear some updates there.

Vince Ambulone, VP of Finance and IR, Arrowhead: Sure. So, I’ll start that out and Jason can fill that So, there’s no way to know what’s going on with the inner workings of FDA. We feel comfortable as comfortable as we can feel because the tenor and the tone and the type of communications we had with FDA hasn’t changed and they are exactly as we would expect at this stage. And nobody in the biotech investor community likes uncertainty and neither do we, but we feel as confident as we can be with the state of the current approval for filazazaram. And our hope is that we will be able to launch on schedule.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: And maybe just based off of the breadth of the data, and James, feel free to jump in here on how you see this being differentiated from trimmed gold. But what are your base case expectations for what a label might look like here?

James Hamilton, CMO and Head of R and D, Arrowhead: Yes, sure. So I can’t comment specifically on label expectations, but we think that the data that we presented in SCS, particularly the change from baseline triglyceride data has an edge, has a larger magnitude compared to what was shown with olanzarcen. Of course, there’s also the dose frequency advantage, whereas we dose every three months and Tranglosa is dosed monthly. And then we think that there may be some safety advantages potentially also that we’ve not seen any platelet signals or any hypersensitivity or anaphylaxis signals that is of those are on the Trinoluza label.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: How important is the distinction that you studied both genetically as well as phenotypically defined patients? Will that matter to the agency and to the physician?

James Hamilton, CMO and Head of R and D, Arrowhead: I think so. I mean, just as a reminder, that was something that the FDA had asked us to study was to include both of those, both the genetically confirmed and the clinically diagnosed FCS population in the study. We’ll see how that impacts labeling. But I do think that that is potentially important to clinicians who manage both of those types of patients.

Vince Ambulone, VP of Finance and IR, Arrowhead: I think that also that might be an important distinction ex U. S. In European markets where you have to prove value. And I think that whether it’s on the label or not, the fact that we studied that population and saw very similar results is helpful when we’re coming up with these value dossiers for national payers.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Are you surprised at all that CHIN GOLDES appears to be receiving coverage for both subsets of patients, even without the fact that they only studied it in genetically defined patients?

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes. I think the jury is still out on that. I think we’ve started to hear some of the some excerpts from policy documents. Think it’s not that’s not true across the board. And but it’s early.

The are rooting for olezarsen and for Ionis. This is a patient population that has had nothing, no treatment. And they’ve lived a very restrictive lifestyle with a restrictive diet and they’ve had recurrent bouts of pancreatitis and hospitalization. So this is good. This is good for the field.

And we are also encouraged that physicians are using this, that they’re putting patients on commercial drug. And I know that the first quarter, we some of the a lot of the use would have been with their Quick Start program, and so it wouldn’t have shown up in revenue. But the fact that physicians are using this and are finding patients to put on this is really helpful. And I think that for FCS, it might not be as readily obvious, but for SHTG, we want to have multiple voices in the marketplace. For SHTG, there haven’t been very effective treatments and there’s three million to four million people with triglycerides over 500 and almost one million people with triglycerides over eight eighty.

So it’s a very underserved population. Having two companies doing pharmaceutical marketing and promotion is really helpful in doing disease state education. So, we are happy that Ionis and olicarsen is in the market. This is good for both of us.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Maybe more specifically on that point, what will it mean to have a second player in FCS? When you come to market and you become the second approved agent there, how do you and Trangolda coexist?

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes. I think that James’ earlier point is we think that there are meaningful points of differentiation with posazaran versus olazarcin. I think that we are having very fulsome discussions with payers. We have been interacting with payers since it was November of last year. So we’ll have about a year of those interactions once we launch, provided we get approval in November.

And I think that it’s a it will be a competitive market, but it’s a short term market. Let’s remember olezarsen will be completing their Phase three studies for SHTG later this year. We will be completing ours in next year. And that’s really the big commercial opportunity that we want to maximize for. That’s the potential multibillion dollar a year opportunity that we both can grow.

Our two companies can coexist and grow this market and help many more patients than if there was just one of us in the market. So there is a way. And again, James did mention these, but there is meaningful differentiation, we believe, from clozacaran versus Olazars.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Maybe given that point that there is meaningful differentiation, how do you think about pricing Clozasiran relative to TrinGolda? If there is this added benefit, can you price at a premium?

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes. I want to be kind of guarded on pricing forecast for now, because that’s something we’re right in the middle of. There’s many different scenarios here, both with FCS pricing as well as SHTG pricing. And we want to see a little more on how FCS the uptake of how FCS goes. Also what the data look like from the olicersen Phase three studies in SHTG.

And then also we’re doing a lot of work on that now. So I don’t want to necessarily talk about what we’re going to do. But there would be an argument to be made for the pricing at parity or pricing at a premium and maybe even giving price concessions if there’s more access. Now again, it depends on the backdrop of which patient population you’re talking about. But the good thing about coming being second in a market and quick a very fast follower, we are not that far behind olanzarcen with either launch, is that we get to react to the way the market reacts.

And payers will have a little more familiarity with the marketplace than they would have if you’re the first to launch.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: And you and Ionis have both talked about potentially taking a price cut once the label expands to SHTG beyond just FCF. Presumably, Ionis gets there first and drops their price. Would you also need to drop the price of clozastrin at the same time as when Ionis enters the market for SHTG? Or is this something where you can maintain your FCS level pricing until you’re specifically on the market for SHTG?

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes. Again, I don’t want to talk too specifically about what our plans would be there. But our base case would be that we would have there would be a new product per se, a new SKU when we launch an SHTG. And then patients would slowly move over from the FCS SKU to the SHTG SKU. But the exact pricing level, we’re still working on that.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Okay. Maybe for SHTG, you have Shasta three, four and then also five, which is pretty unique relative to your competitor. Maybe talk to us about the rationale and the thinking behind running a separate trial for Shasta five and how important that is both to physicians, patients as well as payers?

Vince Ambulone, VP of Finance and IR, Arrowhead: You want to cover that one, James?

James Hamilton, CMO and Head of R and D, Arrowhead: Sure. So, SHA-five is the pancreatitis study that enrolls a population that is at high risk for having pancreatitis events. And it’s a smaller study. It’s not nearly the same size as either of the Shasta studies, but these are patients that have had events in the past and are high risk for having further events and still have severe hypertriglyceridemia. Our view on that study is it’s really a study for the payers, particularly in Europe.

It’s not something that is required for approval. But we do think that showing a material benefit there in terms of rates of acute pancreatitis will help with reimbursement down the road.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: I guess I’d be curious your thoughts. Ionis is not taking that approach. And presumably, out of Shasta three and four, you might be able to pull out a signal. If Ionis is able to show it in their core studies, is this something that you could you can maybe change your strategy, your regulatory strategy, or your thinking around these trials to be able to similarly show this type of Yes.

James Hamilton, CMO and Head of R and D, Arrowhead: I think we really wanted to guarantee that we were set up to show that difference, right? And this study allows the SHASTA-five study allows us to do it. We didn’t want to bank on either our studies, SHASTA-three and SHASTA-four, maybe showing pancreatitis improvement or the Ionis core studies may be showing a benefit. We wanted to kind of make it definitive that we are going to study this and hopefully show that there is a benefit.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Okay. Just to clarify there, can Shasta three and four, can they be amended in any way to have that be either to power the study more to be able to detect that or to have that be an endpoint that you could then use the regulatory agencies?

James Hamilton, CMO and Head of R and D, Arrowhead: Well, I think we could pool data from those studies, certainly from the Shasta studies and take a look.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Got it.

Vince Ambulone, VP of Finance and IR, Arrowhead: I think one critical thing here is that the population that we are studying in the Shasta studies and that Ionis is studying in the core studies, it’s not enriched for patients that are at high risk of acute pancreatitis. And so it’s a pretty big ask to show a statistically significant reduction even when pooling in a one year period. The one big benefit of SHASTA-five is that it’s an event driven study. And so we’re not restricted to treating and then assessing at one year. So we treat and enroll patients until we get a certain number of events, and then we unblind it and look at the differentiation there.

And that I think that’s important because the lower risk the population, longer you should watch them to see enough events on placebo. And you probably know this better than I do, but my understanding is that there’s not an expectation of a statistically significant difference in the core and core two studies with AP. It’s just that there might be a trend that they see. And so again, to James’ point, we wanted to give ourselves the best chance of showing a statistically significant change or difference between the two groups.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: How important is that to show us a benefit on acute pancreatitis? Not just for the payers, but

Vince Ambulone, VP of Finance and IR, Arrowhead: Yes. Well, I mean, I think that’s the goal when you’re talking about especially when you go further spectrum, that’s the goal is reducing acute pancreatitis risk. And how you define that could be with adjudicated pancreatitis, it could be some patient reported outcomes, but that’s the goal with this. And so I think that the further you go from U. S.

Commercial payers, the more you’re going to want to show value. Value can be defined in many ways, but an obvious clinical outcome and reduction in hospitalizations and pancreatitis events and hard clinical benefit, the better. And so I think the trade off would be without having that, you to gain broader access in ex U. S. Markets, you might have to take pretty extreme price concessions.

And so, I think that you have a lot more pricing power, the stronger your value proposition is.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Got it. Maybe one more from the cardiometabolic portfolio here. The dimer that you have, your PCSK9APOC3 dimer. Maybe talk to us a little bit more about that and when we can expect to see some updates?

James Hamilton, CMO and Head of R and D, Arrowhead: Sure. So I can touch on the dimer. We just shared some of the dimer data as a poster at NLA last week. And this one’s pretty close. I think we haven’t given specifics on when it will be in the clinic.

Probably before end of this year, we should be in a position to at least file a Phase I and would anticipate studying that. This is two linked siRNAs, one that targets APOC3, another that targets PCSK9. So the intent is to have an LDL effect as well as an APOC3 driven triglyceride effect. And the perfect population for that would be the mixed hyperlipidemia population, which is of course one of the largest populations out there that’s still pretty unaddressed in terms of available treatments. And we can study that population early on and kind of get right into that group in a Phase I study and see how we’re able to impact both LDL and triglycerides.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: And as you think about maybe this mixed hyperlipidemia population where you have talked in the past about cardiovascular outcomes trial, potentially running it with clozastrin. Does this become an attractive alternative? Maybe the dimer becomes the asset to bring forward.

James Hamilton, CMO and Head of R and D, Arrowhead: It could be. I think we have to wait and see how the data look. I mean, we still think that posazaran could potentially address that population as well with large reductions in triglycerides and large reductions in non HDL cholesterol and really not no meaningful changes in LDL per se, but still significant reductions in ApoB that could be beneficial when studied in an outcome study in that population. We’ll have to see how all of those different lipid parameters look with the dimer.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Got it. Okay. So, CVOT potentially on hold until you understand the profile of the dimer.

James Hamilton, CMO and Head of R and D, Arrowhead: I think

Vince Ambulone, VP of Finance and IR, Arrowhead: that’s fair. Yes. And I think that’s the right way to put it as that at the outset, I just mentioned our balance sheet is very healthy. We’re funded into 2028, starting a large cardiovascular outcome study. And then the specter of multiple large cardiovascular outcome studies makes it not that healthy anymore.

And so we don’t want to get to a point where we are needing to raise money or whether we’re starting a study that we just don’t have the capital to see through. Keep in mind that the pure triglyceride SHTG market is really attractive. It’s a very large underserved market. And so to the extent that posazaram is a pure play triglyceride drug that actually makes a lot of sense to us. And we are really excited about the possibility of showing LDL reduction with PCSK9 component of that dimer and the triglyceride lowering with the APOC3 component.

I think those two mechanisms together are really powerful and ones that haven’t been studied together. Now the genetics are pretty clear and we have health records and clinical trial data with LDL lowering going back decades and assessing the ability of triglyceride lowering and LDL together to reduce cardiovascular events in a specific population that James was talking about, mixed hyperlipidemia, which is many millions of patients, twenty million patients or it is a sizable population, is really interesting. We have approached our executive board and external KOLs who are very enthusiastic about the possibility of this being a meaningful treatment into the future. And I think it also talks to how siRNA is really, we think, perfectly tailored to long term management in preventive cardiology. It’s very tailored and it’s very precise.

It does one thing. It down regulates the expression of one gene. And with this dimer, we can hit two genes that potentially at that point are both clinically validated as having a benefit. And so, think it’s a lower risk proposition.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Maybe with the last ten minutes, let’s turn to obesity, since those are some of your biggest readouts that are coming later this year and then early next. ELK7 and Hippie, maybe James, I’ll start with you here. Talk about the mechanistic rationale for both of those targets, one being, I think, being studied across multiple different companies, ELK7 relatively newer.

James Hamilton, CMO and Head of R and D, Arrowhead: Yes. So they’re both part of the same access that starts in the liver, at least with inhibit E and the inhibit E being the gene, activin E being the gene product and ends in the adipocyte. Active E binds to a receptor on the surface of the adipocyte called ALK7 that also has other ligands that can bind to it. And the end result is essentially instructing the adipocyte to store triglycerides as fat in adipose tissue. Our thought was we had siRNAs against both of these targets, both inhibin E and ALK7.

The inhibin E targeting technology or the so called GalNAc technology is pretty well vetted. We and others have used that technology to silence genes in hepatocytes in numerous different genes, numerous different trials. The safety profile is pretty well understood. So we view that as low risk. When we studied these two different targets in animals, in mice, there seemed to be a little bit of a potency difference, meaning that the differential in weight gain with ALK7 targeting was greater than what we saw with inhibin E.

So maybe the pathway or hitting ALK7 is a little more potent. The technology we use to silence expression in the adipocytes is an adipocyte siRNA targeting platform that has not been in humans yet. We actually just dosed the first patients within the last week or so. So it’s just getting started in a Phase I. There’s a little more risk there because it’s first in human.

So that’s the rationale for taking both into the clinic. They both hit the same access. ALKS7 may have some potency advantages, but it’s first in human. There’s some added risk just because it’s not as well tested yet, at least the platform. And then inhibin E uses the GalNAc technology that’s, of course, been in numerous clinical trials.

And so it’s more derisked. So that’s the rationale for taking them both into the studies. And we’ll kind of wait and see which one seems to look better.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Based off of your preclinical work, I guess, maybe what differences do you see in terms of the safety profile from ELK7 versus in HITNEY?

James Hamilton, CMO and Head of R and D, Arrowhead: There’s really no difference in terms of the safety profile. Of course, both have been through non GLP and GLP tox studies in rodents and in monkeys. And the safety profile has not been a differentiator based on non clinical data. So, don’t think that will be the tiebreaker. It will likely be something either efficacy or safety data in the clinic.

Okay.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: I guess maybe as you look to the first clinical data for both programs, if you could frame your expectations for what would be a positive outcome from each program?

James Hamilton, CMO and Head of R and D, Arrowhead: Yes. For both of these, we’re not really setting an expectation in terms of weight loss effects. These are both Phase I studies, so they’re safety studies. Our goal here is to establish a dose range and use the pharmacology, the PD data that we get out of both studies to select a dose for later stage studies for Phase II. So we’ll have knockdown, we’ll have PK and we’ll have safety.

Of course, we’re looking at a whole host of other biomarkers like weight loss. We’re doing full body MRIs, so we’ll get a good look at changes in body composition and then some other blood based biomarkers. I think in aggregate that will help us make choices about is one better than the other or more appropriate for us

Vince Ambulone, VP of Finance and IR, Arrowhead: to take forward. And I would add one thing that the genetics have been very compelling about people that are loss of function mutations of these genes. There seems to be an advantage there. We’ve taken that into animal studies. And it looks like if you can harness that pathway with siRNA, you can show that you can reduce fat and spare muscle in animal models.

So we’ve translated genetics into preclinical studies. The next step is to see if we can translate those into humans. And so that’s really our main goal. James mentioned, we need to establish safety, we need to select a dose, we need to select an interval. And then lastly, we need to see at least a few signals that we get that translation, that the pathway is actually we can pathway with siRNA in humans to get a benefit.

And then efficacy really comes at Phase II. So, we do want to see signals, but the efficacy is we think will be a Phase II event.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Understood. Maybe just at a high level, I mean, you’re seeing data come out very frequently, right, about different GLP-one agents, amylin agents, like how do you think about what you have here and the potential to be differentiated or maybe even complementary to some of these other agents that are out there?

James Hamilton, CMO and Head of R and D, Arrowhead: Sure. We see a few potential ways to win here. I think there’s one of the things that we’re studying is, of course, combination therapy. So either ALK7 on top of tirzepatide or inhibin E on top of tirzepatide, we’re running both of those in the clinical trials, looking for any synergistic effects or additive weight loss. That might be one use case.

Another could be a scenario where, well, maybe you don’t need to lose 20% of body weight, but you could have something that has a more benign side effect profile, but you lose 7% to 10% of body weight and you can do infrequent dosing with not much in the way of side effects. That might be attractive for some patients as well. And then the third potential use is with maintenance therapy. If someone wants to start and be on a GLP-one agonist for a period of time and then come off of that medication, perhaps this lipolytic enhancement pathway could be beneficial to maintain weight loss or slow the rate of weight gain post GLP therapy.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: And then as you think about I guess maybe you have these cohorts that are examining the combination. What type of maybe based off of the work you’ve done here, but what type of AEs do you think could potentially pop up as you combine these types of agents?

James Hamilton, CMO and Head of R and D, Arrowhead: I mean, one of the things that we’ll certainly be looking at will be the rates of the GI side effects that are seen with the GLPs commonly. How does that look with the combination cohorts? I think that’s probably the most important AE that’s known to that class. We, of course, will look for any change in glycemic parameters, how does combination therapy impact HbA1c or blood glucose? Also, I mentioned we’re doing the full body MRIs.

We’ll get a good look at liver fat in the study to see if there’s a change one way or the other.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Maybe with the last two minutes, we’ve only talked really about two of your programs here. But in the last two minutes, updates or things you’d like to share about the rest of your pipeline?

James Hamilton, CMO and Head of R and D, Arrowhead: Well, one of the things that you’ll probably be hearing more about in the coming year involves our CNS platform. So we’ve been we have a program in the clinic now targeting ATAXN2 that uses an intrathecal route of administration, and intrathecal We’ve been working over the last few years on a platform to facilitate delivery of siRNA across the blood brain barrier. And we have a program that we’ve only shared preclinical data that targets tau expression in the CNS. And so look for that to be entering the clinic over the next six to twelve months.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Vince, maybe one last question for you here. We’ve talked about the SREFTA deal that very nicely bolstered your balance sheet. But as you think going forward, how are you thinking about additional partnerships or collaborations or licensing agreements? What is Arrowhead open to?

Vince Ambulone, VP of Finance and IR, Arrowhead: Sure. So, big benefit of having this extraordinarily productive discovery engine is that we are capable of bringing new candidates into preclinical studies and then ultimately into the clinic much faster than we will be able to exploit ourselves. So there will always be more programs that we have developed or started to develop than we’re going to commercialize ourselves. So, I think that the business development is going to remain a core part of our strategy and certainly a core part of our financing strategy. In between now and 2028, where I said we’re funded into, we do anticipate doing more deals.

I think the size and scope of the Sarepta deal are likely not something that we’re going to recreate, certainly not multiple times, but there are opportunities to do product as well as some discovery partnerships. We have the bandwidth to support those. And I think that the size of our pipeline is supports more deals. And also, I said this at the beginning that the next step for us is starting to commercialize our own therapies, which means that we need to figure out how we build commercial to support more than one product. And so to the extent there are agents within that cardiometabolic space, obesity may be part of that, They have a higher strategic value for us.

So, would say we are more likely to hold on to something that we could put into the bag of an existing sales force than we would be for therapeutic areas where we would have to rebuild commercial to support. But I would say that we do intend to do additional deals with our discovery engine.

Andrea Newkirk, Biotech Analyst, Goldman Sachs: Great. Well, with that, thank you guys so much. Thank you, everyone. Thanks, Vince, and thanks, Gene.

James Hamilton, CMO and Head of R and D, Arrowhead: Thank you.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.