Autolus Therapeutics plc (AUTL) Earnings Call Transcript & Summary

Good afternoon, everyone, and thank you for joining. My name is Rajan Sharma, European Pharma and Biotech analyst here at Goldman Sachs. I'm very pleased that we have Rob Dolski, Chief Financial Officer at Autolus. Rob, thank you very much for joining us.

Great to be here.

Maybe kind of just to start for people who maybe less familiar with the Autolus story. Could you just kind of give us an overview of the company, key focus areas and core competencies?

Sure, happy to. So again, Rob Dolski, I'm the Chief Financial Officer at Autolus. The company is a little over 10 years old, and the company has been focused from a cell therapy perspective in the autologous CAR T space. Our lead program called obe-cel, brand name now called AUCATZYL, was recently approved in the U.S. This was last year in November. And so we are very much in the launch process in the U.S. for AUCATZYL. We are also in the middle of some of the regulatory processes that you typically would go through both in the U.K. with the MHRA as well as with EMA on the European side and can get into more details, but those processes are playing out. I'd point to the key differentiation that at least we believe obe-cel has over other competitive therapies and CAR Ts really boils down to its unique design. That design, at least from a clinical perspective, and this was tested in, what we call, the FELIX study, really resulted in 2 areas of benefit. First, from a safety perspective, saw very low grades or low levels of high-grade adverse events, specifically ICANS and CRS that have typically been an issue in the space. The design of obe-cel enables it to act in a more natural way in terms of how T cells will quickly engage with the target cell and then quickly release as opposed to getting stuck. So we call it kind of a fast-on, fast-off rate. When they get stuck, there's an overactivation of the cells, and that has resulted in some of the adverse events that we have typically seen play out in the space. And again, happy to go into more details on that front. The second piece really on the efficacy side. So what that design does kind of internally is it provides -- and what we've seen in the clinical studies is a much higher in vivo expansion of the CAR T product as well as a persistence that remains consistently putting pressure on the disease. What that has really resulted in, big picture in the studies is, when you look at our event-free survival curves or overall survival curves, we see a plateau occurring about 40% of the patients that responded. So for those patients, they've remained remission-free without any other therapy. And so for a portion of patients, there is the hope that this can be more of a stand-alone therapy, and this has created certainly some excitement in the community, the physician community. So that is kind of where obe-cel sits from a U.S. perspective, and this is all in the indication for relapsed/refractory adult ALL. I guess, along with that, in supporting that business, it's worth noting that we've also internally not only built the commercial infrastructure and systems, et cetera, that are required for CAR T delivery, we also have built a rather unique manufacturing facility north of London. This is providing currently all the commercial capacity, and we'll continue to do so as the launch progresses. We think that's an extremely unique aspect of Autolus in terms of the competitive advantage to leverage that same infrastructure as we now think about our real second priority for the year beyond the launch, which is focused on expanding the obe-cel opportunity. So we've recently presented some data as well as some plans at an R&D Day, but at a very high level. We think there's opportunities with obe-cel to expand both on the hem/onc side as well in some of the new areas with autoimmune. Again, happy to get into more of those in detail. But with the recent R&D Day, we shared some of the early lupus study data that we have. And we also laid out for not only pursuing a registrational or kind of pivotal path in lupus nephritis, we also are going to be initiating an exploratory study Phase I in MS. In addition to obe-cel and clearly, that's where the focus of the company is, both in terms of launch execution as well as pipeline expansion. We do have other novel CAR Ts in the pipeline, and these are being kind of looked at and tested with a nice collaboration we have with the University College of London or UCL in the U.K. And there, we get to generate early Phase I data, clinical experience on those programs, and we have a few of those that we'll be reading out over time as well.

Okay. That's a great overview. One thing on the manufacturing is you kind of talked to it there. And I think it's something that you're proud of as a company in terms of the way that you've been able to execute there. One of the things, obviously, just bringing that into one of the sector issues that we're facing at the minute is tariffs. So of course, that manufacturing as great as it is, it's based in the U.K. So could you just kind of talk to potential impacts of tariffs in a scenario that they are applied. And those obviously been a trade negotiation point with the U.K. To what extent is that a benefit?

Yes. I mean, maybe where I'll start with the Nucleus and that's the name of the plant that we have north of London. Again, it was a unique design built to validation within 2 years, all the way to approval within 3 years, very modular and a different approach that we took. During the conduct of the pivotal program, we actually ran that study during the pandemic. So it really wasn't an issue. We were able to reliably make and deliver product into the U.S. So just from a logistics standpoint, we certainly don't see that as a disadvantage. Now to your point on the tariffs, it's very early days. And what we know is there had been an announcement around a U.K., U.S. deal for what we know today that excluded pharmaceuticals. I think we're still waiting a bit on that. I think what we would comment on with respect to tariffs and maybe prior regimes where tariffs were in place in the pharmaceutical industry, they typically didn't apply to blood-derived products. And so -- and that is something that certainly obe-cel would be considered. So that's certainly a consideration. I think the other piece that would play out if tariffs were to come into play is typically they would be levied on the custom value coming into the country. And so that is going to be more linked for us to the manufacturing cost, not an exact one to one, but it's going to be more linked to that than, say, the selling price or the commercial value of the product in the U.S. So again, we are actively monitoring and staying very close to this. And as you can imagine, scenario planning, internal to the company, those are the considerations, I think we put out there now. We'll just have to wait and see.

Okay. And then the other one, and then we'll kind of move on from policy and on to the company specifically is just the potential threat of most favored nation pricing in the U.S. You're in a position where you're still kind of, as you pointed out, negotiating pricing. How do you think about potential impact there? And what could you need to mitigate?

Yes. It's not an easy question. I mean, I think, number one, very similar to tariffs. What we would say is it's very early and without specific, it's very hard to react to. I think for many companies like Autolus, what something like this would mean is we'd have to just carefully evaluate how -- what makes sense and what doesn't make sense economically as we look at the different markets and where to go and maybe where not to go. What I would say about that is it's not a whole lot different than how we're approaching things today, right? Because it's the same story where our initial launch is in the U.S. We've got subsequent markets that we're in process with. But as we said, in particular, with respect to EU, it really is going to be a country-by-country evaluation. This would be probably another factor that we'd have to throw into the mix as we go through that.

Okay. And just a final point, I promise on this one. Just given that you're at the point of negotiating prices in Europe, one of the things that we've been hearing from some of the larger companies through the course of the week is the fact that Europe. They kind of agree with the administration's positioning that Europe has to pay more for innovative drugs. So again, from that vantage point of being in the in the negotiating room, do you get the sense that there is capacity within that European health care systems to actually pay more?

I think it's hard to comment on that. Certainly, I don't think that we would go into any specifics in terms of just the more interim meetings and discussions that we're having. So I might leave it there. I don't think that we can talk too specifically on pricing.

Okay. Let's move to AUCATZYL then and talk about the launch. So Q1, you had EUR 9 million in revenue with some of that, obviously, a meaningful proportion of that logged as deferred revenue. So before we get into the details, could you just talk about initial launch momentum, how that's tracking relative to your expectations as a company and the feedback that you're getting from your key centers on the ground?

So one of the things that we've guided to in terms of performance and kind of to watch and the launch has been the center onboarding. That has been going very well. And in fact, certainly, as we started the year, maybe started out of the [indiscernible] little bit ahead of plan. We had 30 centers ready to go. With the approval, we at JPMorgan earlier in the year had said that would take us probably through the first quarter to get fully activated until those sites were enrolling patients that came in a little early, probably towards the end of February or mid-quarter. So things are progressing there. A lot of that does sit on the center side. So it's somewhat within our control, but it also relies on them going through their internal processes. So we're tracking now just over 40 centers. I think it's about 41, 42 centers, still expect to hit 60 by the end of the year, that giving us a 90% kind of coverage in terms of the addressable patients. I think the other big piece that we've pointed to in terms of Q1 performance and the launch progress has been around access and coverage. And so feel very good. We've got a very experienced team on that front working with all the various payers. And at this stage, we're at kind of a 90-plus percent level in terms of covered total medical lives. So have not seen any impediments on that front in terms of the rollout and the launch. I think general feedback, and this is something that will play out through the course of the year, going back to kind of obe-cel's properties and what's unique, I think early on, what is the most immediately experienceable event with the obe-cel treatment is more on the safety side. These are events that typically happen days after treatment. And so that has been a very kind of positive, certainly, what we've seen playing out so far in commercial like we experienced in the clinic and very positive feedback and recognition that there's a very different dynamic in terms of hospital resources and utilization that has to go into managing patients that don't end up in that severe ICANS or severe CRS category. I think we'll have to wait and see. We're kind of now getting to the point, certainly with patients that are treated in the first quarter. But the longer-term outcome and see how that plays out. Again, we're not expecting any difference from the clinical studies. And I mentioned that physicians are very excited about this idea of being able to have a stand-alone therapy that could really put a portion of the patients in long-term remission, but that's going to take some time to play out. I think the other piece that we're also just kind of really focused on and then wanting to kind of see how we track after, say, 2 or 3 quarters, is really kind of the physician adoption curves. The centers have been coming on and it's probably not going to be a perfect pattern through the rest of the year, but very well on our way to the 60 centers. Physicians, we have a very kind of tailored approach depending on the profile. And we have some physicians that are very experienced CAR-T users, experience with the existing CAR-Ts and now have obe-cel as a tool for them to use. And we've seen some of them make some very quick changes in conversions over to obe-cel. I think you've got other physicians where there may be a bit more of a trial period, specifically kind of on the spectrum. At the other end, you've got those that might be traditionally stem cell transplants, that's kind of been the way to get to a longer-term cure up until we think with obe-cel's arrival. And that longer term kind of play out -- they need to first, and we had this happen in our clinical trial for transplant in eligible patients might be where they trial obe-cel, build some experience and then expand from there. And like I said, we saw this in the FELIX study where eventually we had traditional transplant physicians that were even taking transplant-eligible patients and moving them on to obe-cel. So that is why we haven't given specific guidance for this year. What we said is we really want to see 2 to 3 quarters play out with these various kind of pieces that are moving and variables that are playing out for the year. But we do think, certainly, going into the end of the year with the centers on board and hopefully also at that point, starting to see momentum building around some of the outcomes that folks are seeing, it will provide very positive momentum going to 2026.

Okay. And I think you made the comment that kind of first quarter was probably a little bit ahead of expectations in terms of -- from an internal perspective. I think the numbers certainly came in ahead of my model and where consensus is. So from your perspective, what went well to kind of drive that?

Yes, I think it was all the pieces that I hit on. So from a market access perspective, I think also from a center onboarding perspective, I would say, the other thing in this space, in particular, that Autolus has been very focused on and maybe more so as a small company trying to not necessarily stumble out of the gate as we've seen some and limit the launch is really the collaboration that we have with the centers. And so it's everything from managing the supply, production and logistics, but it's all the other services. And as you can imagine, first company turning on the engine for a commercial launch, you can plan and prepare as much as possible, but you're always going to have to be ready to adjust, make some improvements, and we've very actively listened to the centers and have tried to respond and have real time made those updates. So I think that, that engagement has been very positive through at least the first quarter so far.

Okay. Makes sense. And then just from the center onboarding perspective. So you said you're looking to have around 60 on board. And I think you made the comment, it's not -- should we expect that's not going to be linear given that you're at more than halfway at about the halfway point of the year?

Yes. Yes, it's hard to say because like the reality is we have a role in those activations, but a lot of it also sits on the center side. And depending on center, it will vary in terms of the time it takes to put the drug on formulary, get things up in their own systems, charge masters, et cetera. So yes, I mean, I think that we're on a reasonable path to get to our target. We feel good about that. How that exactly plays out quarter by quarter, I think it's hard to say.

Okay. And then if you get to 60%, how many -- like what proportion of the U.S. patient population will be addressed through that 60 centers?

Yes. Well, what we've done in the center rollout is we -- so we did start with an initial 30. What we said about that 30 is it gets us about 65% plus of the addressable patients. We were certainly focused out of the gates on the larger centers, the higher enrolling centers. And that's why you get a little bit more with that 30. The next 30, we believe, with 60 centers in total will get us to about 90% -- 90-plus percent coverage in terms of the addressable market in the U.S.

Okay. And of those centers that are onboarded at present, what proportion have actually kind of treated patients?

Yes. So what I would say is -- a vast majority of the centers have treated. I think what I would caveat or maybe note on this front is that there is a lag time, right? And so what we've seen, for example, is as we started the year, we were in kind of almost 2 dozen centers that were ready to go, activating and enrolling patients. Those were really the centers that were contributing the vast majority of Q1 administration of product. And now we find ourselves at kind of the 40-plus range. And so yes, we've got a lot of centers that are registering patients, the vast majority that are registering. By the time that you get to actually treating, there is this lag effect. So certainly, as we ended Q1 with around 30 centers, the build that we're seeing from there now at 40 wouldn't expect to be driving the vast majority of Q2 sales. That will really start to build with Q3.

Okay. That makes sense. And then the other thing on Q1 was just there was a bit of focus around gross margin. So those sort of COGS recognition and gross margin were 2 pieces that people were focused on. How should we think about that kind of evolving through the year? And at what point do you get to a steady state?

Yes, it's a good question. And admittedly, a lot of the, I would call, technical accounting around some of this isn't always easy to follow. And so we spent some time kind of explaining this. So let me start by saying, one of the big drivers near term in terms of improvements in the gross margin is purely going to be driven by volume and utilization in the plant. So November 8 rolled around in terms of the approval and what that meant was there's a portion of our facility that is commercially validated and approved for commercial production that includes all of the facility overhead costs, the staff, et cetera. That no longer can be treated as R&D expense. It needs to be viewed as cost of supply or cost. So even if you go back to our Q4 number from last year, while we didn't report any sales, we did have a cost of sales number. What that represented was effectively underutilized capacity in the plant or idle plant charges that no longer could go down to R&D. That continued to play out where we are on the curve in Q1, right? And so you saw more cost hitting there certainly than you might expect longer term and with targets that we've talked about with respect to gross margin, and it's largely driven by that kind of dynamic. I think the other piece that we got into with Q1 is there's not a complete matching. So you talked about the deferred revenue versus the patients that landed in product sales, both of those buckets end up in our cost of sales for the period because that material has effectively gone out from our control. The ones that land in the deferred bucket are just not yet administered and that's where we take the revenue recognition. So there was that little bit of disconnect. Longer term, what we said is, we want to target a gross margin that is about a 15% to 20% of the U.S. sales or the U.S. price. When we get there is going to depend on the trajectory, and we haven't guided specifically, but this is going to be much more as you ramp up well on the curve towards peak sales as opposed to near-term quarters.

Okay. Okay. That makes sense. And then on all AUCATZYL, let's just kind of touch on Europe. I know we talked about the pricing there. But just more broadly, where are you in terms of discussions? And at what point, if at all, should we expect Europe to be kind of potentially contribute to the top line?

Yes. So let me take the 2 pieces that we have going on there. So in the U.K., with the MHRA, we did get the conditional approval and now we're in the process with NICE to work through more of the reimbursement side of things. Both are required to launch. That process is ongoing. And we won't comment on kind of the interim meetings, et cetera, but there will be -- we did have the meeting in May that we talked about with NICE, and there'll be minutes following that in their initial opinion that will come out and be public. On the EU side, we also -- we have the CHMP positive opinion, ultimately then going to wait for the European Commission on the full approval. We've said in Europe, in particular, we are focused on the German market initially. And so we're doing a lot of work on the access front and reimbursement front there, preparing for that market. And then following that, it would again be really on a country-by-country basis, really built around assessing the economics on the reimbursement and access side and where it makes sense to go.

Okay. And then staying with obe-cel kind of additional indications that you talked to in terms of one of the focus for this year. You've got the PY01 trial, which is pediatric ALL is expected to read out later this year. Could you just kind of talk us through what the -- what you're hoping to show and what the commercialization strategy might be?

Yes, certainly. So I think it's important to maybe note, the overall development for obe-cel began in pediatric ALL. The acute need or unmet need was much more in the adult population, and that's what drove some of the early decision making with the program. But what we are currently conducting is really a commitment from a regulatory perspective, both with the FDA and with EMO around the pediatric population. And so we are conducting a study that is right around 2 dozen patients. That will be the data that we that we show in the second half of the year. Our hope -- and we still have to have some of these discussions and we haven't fully committed to plans here yet, but our hope is to find a reasonable path to a label expansion with the agency in the U.S. The thought would be to build on that data set by some increment that makes sense for the agency, that makes sense for us from an economic perspective. It's a very -- obviously, what we're hearing from the field is there is still a need for additional options for this patient population. So we're trying to be responsive to that. This is an incremental add to a lot of the infrastructure that we've built, which would be very good from kind of leveraging the utilization of the plant and the commercial infrastructure, but it also has to make sense economically. This is not as big of a market as the adult. And so that's kind of the discussion that we're having. But the idea is to hopefully find an efficient path forward to provide that to the pediatric population.

Okay. And then maybe switching gears to autoimmune. So we saw the CARLYSLE trial where you presented some data at the R&D event earlier this year. Could you just kind of provide a quick overview of that data set and specifically call out what you think are the other key takeaways?

Yes. I think starting at a really high level, for us, the key takeaway is it didn't really tell us -- it was very much in line with our expectations. But again, this is a 6 patient study. So in the field, in general, what we've been seeing is a lot of small data sets. And our initial data was no different than that in terms of size. Generally speaking, you see some variation with the different CAR T products. We've seen some differences on safety and persistence or expansion. But with respect to obe-cel, I think we were very comfortable with what we saw on that front, relating it back to our experience on the oncology side. What we presented for those 6 patients? So this was a dose confirmation study. What we effectively were doing was converting the pediatric weight-based dosing into a fixed dose for the lupus setting with adults. I think that makes a lot more sense from kind of a practical goal administration perspective. And we used 50 million cells, so that translated into 50 million cells in this cohort of 6 patients. What we were looking for was obviously, safety, some of the biomarker kind of clinical scores or seeing how they [indiscernible] and some of the B-cell aplasia, et cetera, that you would expect early on in the treatment cycle. Of the 6 patients that we treated, so there were no severe high-grade ICANS or CRS. We had 3 out of the 6 patients who did experience Grade 1 CRS. So no ICAN. When you looked at kind of the clinical scores, the biomarkers, B-cell activity, they were moving in the trends that you would expect. Three of the 6 patients got to a complete renal response. But again, very early too, the 6 patients had only really gone to their day-28 assessment. So what it did for us is kind of confirm, I think, at a high level, what we've seen from the field in terms of a very immediate impact to some of the clinical scores and biomarker scores. We were obviously pleased to see a very clean albeit in 6 patients, but safety profile and gave us confidence then to certainly have the discussions we've had with the agency and really lay out plans to move into a pivotal study. The pivotal study will be focused more in lupus nephritis. But it's a very compact study, about 30 patients, really focused on the relapsed/refractory very severe end of SLE lupus nephritis. And with that, a very efficient path in terms of development and time to market.

Okay. And then one of the other updates at the R&D event was the fact that you're focusing on severe or refractory lupus nephritis as opposed to SLE more broadly. What's the rationale there?

Yes. Well, I'd say more broadly, as we thought about CAR T and Autolus positioning in the autoimmune space, we've kind of looked at it from 2 angles. We've looked at it from a spectrum of unmet need. So if you look at the end, kind of the far unmet need around MS or even lupus, whether it's SLE or lupus nephritis, kind of those severe cases at that end whereas at the other end, you might have an indication like rheumatoid arthritis. For any of those indications, you're going to have a spectrum of severity, low mild or medium severe. And certainly, from even a health care economic perspective, we view that the severe end of the spectrum is going to be more where CAR T therapy and all that goes around CAR T therapy will make the most sense. Through the discussions with KOLs as well as the engagement with the agency, I think what was important to us was to leverage a lot of what we've built, create a very efficient path and I think what we've done with this focus is given a chance or an opportunity for us to be very quick and first to market in lupus nephritis. What we heard from the KOLs and from the agency certainly confirmed the unmet need with these patients. So the LN study will focus on refractory patients, both to any B-cell depleting biologic agents as well as the calcineurin inhibitors. And so there's a very clear unmet need. There's really no standard of care, which gave us the opportunity to keep this a compact study. It's got a very clear endpoint as well. the CRR versus the SLEDAI score that is a little bit more subjective in the overall broader SLE population and gives us a chance to run this very compact study.

Okay. And then very quickly on commercial opportunity, how does that compare in lupus nephritis versus the broader SLE opportunity?

Well, I don't think even when we -- as we started to think about SLE, I don't think we ever really envisioned the broad, let's call it, 350,000 to 400,000 patients prevalent in the U.S. We certainly always looked at that severe end of the spectrum. And many of the patients that are ultimately SLE patients will go on to have some kind of kidney involvement and progress to lupus nephritis. Now our study is going to be focused on, again, the more severe refractory kind of Stage 3 through 5 category lupus nephritis. I think that covered....

Yes. And then in the last minute, I just wanted to kind of touch on the investment thesis. So obviously, you kind of talked about your progress with the approval, the launch. The stock hasn't necessarily reflected that progress and I get a question a lot from investors. What is your kind of pitch for the investment case for Autolus here? And assuming that the sector comes back, why is Autolus the right place to be?

Yes. I think that -- look, the sector in general has had its difficulties and there's still a lot of uncertainties around some of the topics that you mentioned, whether it's tariffs or pricing, et cetera. I think the way that we also view things is, I mentioned before, we're a smaller CAR-T biotech that's going into a space that other much larger players have launched and have had some challenges in terms of whether it's manufacturing or out of spec or product profile kind of characteristics. So we view very much this year's focus. First priority is execution of the launch and really delivering the product and the slots to the physicians and patients reliably. And I think with that, demonstrate quarter-over-quarter sales performance as we get to the end of the year, hopefully launching or at least enrolling some of the first patients in our autoimmune plans with MS and lupus nephritis that we talked about and then really start to see some momentum after that. But we do feel that part of the story for Autolus is show us that you can do all these things. We executed very well through the clinical phase, both on a production standpoint as well as kind of the how obe-cel ultimately performed in the clinic. We need to see that now play out commercially.

Got it. Very clear. Thank you for your time, Rob.

All right. Thank you.