Autolus at Jefferies Conference: Strategic Growth in Healthcare

On Thursday, 05 June 2025, Autolus Therapeutics (NASDAQ:AUTL) presented at the Jefferies Global Healthcare Conference 2025. The company showcased its strategic focus on expanding the reach of its lead product, Ocatsil, while addressing both opportunities and challenges in the competitive CAR T-cell therapy market. The discussion highlighted Autolus’ commitment to quality execution and innovative treatment solutions.

Key Takeaways

• Autolus plans to activate 60 centers by year-end, aiming for 90% US patient access.
• Ocatsil’s safety profile is a key differentiator, with lower rates of severe side effects.
• Expansion into pediatric ALL and autoimmune diseases like lupus nephritis is underway.
• Q1 sales reached $9 million, but no revenue guidance for 2025 was provided.
• The UK-based manufacturing facility offers logistical advantages for US distribution.

Financial Results

• Q1 2025 sales amounted to $9 million.
• The company refrained from providing revenue guidance for the rest of the year, citing dynamic launch factors.

Operational Updates

• Launch Progress:

- 40 active centers currently; targeting 60 by year-end.

- Approximately 90% of US patient access anticipated.

• Manufacturing:

- UK-based facility ensures high control and efficiency.

- Out-of-specification product rate of 5-6% observed, consistent with pivotal study results.

• Expansion Plans:

- Pediatric ALL: Data expected at ASH, potential label expansion.

- Autoimmune Diseases:

- Lupus nephritis pivotal study to start in H2 2025.

- Exploratory trial for multiple sclerosis ongoing.

Future Outlook

• Autolus aims to demonstrate successful execution of the Ocatsil launch.
• Key data presentations planned at DHA and ASH for leukemia programs.
• Updated data submission from SLE Phase I study for ACR consideration.
• Engagement with regulatory agencies for pediatric ALL expansion.

Q&A Highlights

• Safety Profile:

- Ocatsil presents a low incidence of high-grade cytokine release syndrome (2%) and neurological toxicities (7%).

- Dosing strategies are tailored to patient tumor burden to minimize adverse events.

• Regulatory Insights:

- Single-arm trials require significant treatment effects, often twice as effective as existing therapies.

- Safety data from approximately 100 patients typically necessary for such trials.

• Manufacturing Logistics:

- UK manufacturing facilitates direct shipments to major US cities without significant delays.

- Products are shipped while final release testing is completed.

For more detailed insights, readers can refer to the full conference call transcript.

Full transcript - Jefferies Global Healthcare Conference 2025:

Kelly Xu, Senior Analyst, Biotech Team, JeffA: For attending JeffA’s Global Healthcare Conference. My name is Kelly Xu, a senior analyst on biotech team here. For this fireside chat session, we are very pleased to have doctor Kristen Iting, Chief Executive Officer from Autolus Therapeutics. Welcome, Kristin.

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Well, thanks for having us.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: And maybe to start off, could you please give us an overview of autologous therapeutics as of today? And compared to one year ago, what have been the achievements?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yeah, happy to do that. Well, first of all, welcome everybody. And thanks for your stamina towards the end of the conference, which I know is a tough thing after a few days of activity. We got a very intense year behind us. We got our lead product approved during the course of last year at November 8.

Product is called Ocatsil. The original name, obviously, ObiCell. The label we got was in patients with relapsedrefractory ALL. It’s the adult population. And what was unusual about the label was that it was the first CAR T that got approved without a REMS obligation, which is reflective of the safety profile that the product has, which clearly sets us apart from sort of the competing products in the space.

So we’re in the process. We’re prepared, obviously, for launch. We’re launching have been initiating the launch at the end of last year and are going now, I think, very strongly as we go through the course of this year. We have, at this point, about 40 centers that are active. We expect to have 60 centers active by the end of the year, which gives us approximately 90% of patient access across The US.

Within the centers, we have about 90% of the lives covered. So the market access team has done a fantastic job and made sure that every patient who was interested in getting actually on therapy actually also had an opportunity to do so and had the reimbursement required. So that was obviously been quite remarkable. One of the important things as you launch these types of products is that it’s not just actually that you deliver the product to the clinic and the clinic does the job. But you actually have to qualify the centers.

And in addition, you’re literally providing a set of services to the center, supporting the arguments on the reimbursement side, supporting the physicians. There’s training involved, product management, all the way to patient support. So it’s quite a range of activities there. All of that actually needs to be established, those services. And the systems behind it need to be established.

So as you start up, there is quite a lot of systems and processes you literally turn the switch on and you have to get going. And obviously one of the key parameters in addition to the commercial systems is of course the manufacturing side. And what we’ve done obviously through the course of last year is go through a very rigorous set of inspections by the MHRA and by the FDA. And have our own facility which is located in The UK. And gives us obviously a high degree of control over manufacturing, which is critical.

So when you think about what can go wrong in these types of launches, and what we’ve seen go wrong over the last few launches, is there are issues related to the quality of the product, the ability to supply at scale, the systems to book. And when you think about that, that also has a huge impact for the centers in their ability to deliver care, creates a lot of work, and also has created, in many of the launches, quite challenging situations for patients who obviously had high hopes, may not have gotten access to the therapy, or did not get actually productables within specifications. So that’s sort of the backdrop. So our focus for the last few months clearly was on making sure we’re delivering to the quality. We’re looking to support our customers as best as we can and really focus on execution.

Because that’s been a real challenge in the space. And there is a lot of complexity involved there. So far, knock on wood, we’re moving very, very well. And we’re excited about the level of interest that we’re seeing at the centers, the physicians, the patients to get access to product. And we’re excited for the next quarters as we go through the latter part of this year, but very pleased with the momentum that we actually have gained.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Fantastic. Super helpful. Maybe since you have launched for several months, what are the key productive features that a physician community mostly appreciated in adult ALL? And if they make a switch from another CAR T already in the market for several years, what would be the top reasons?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yeah, one of the challenges that you have with adult patients in acute leukemia is that they tend to actually have a lot of comorbidities. They tend to be frail. Part of that is due to the fact that the disease itself actually makes you immune compromised. But then to make matters worse, you obviously get treated with very intense therapies, In particular high dose chemotherapy, stem cell transplants, and so on. So these patients are quite beaten up.

So the problem that you have when you’re at that stage is that it’s actually difficult to sustain and sort of manage through adverse events. And that has been one of the fundamental challenges that we had in this space. Because you need on the one hand extremely high level of activity to cope with quite often the explosive growth of the disease. And at the same time you have to sure that the adverse event level actually stays low so that the patients can really tolerate the therapy and really manage it. And so one of the key, I think, attributes that are very quickly experienceable for the physicians and the patients is the safety profile.

And we had two elements there in terms of the design of the product and the design of the dosing that we’re using. Design of the product is such that we have an ability to engage the target cells in a way that delivers to kill but avoids over activation. And that’s at the heart. And that’s inbuilt in the design of receptor itself. But the second aspect was particularly for adult patients with acute leukemia, and actually only for adult patients we’re doing a different approach with everyone else is to actually adjust the level on how we dose to the level of tumor burden that the patient has.

And that actually has been really important because what you can do with that, by patients with high tumor burden, you dose at a lower level so that as the therapy burns through the disease, it doesn’t do that in an extremely fast way. And by actually moving through that somewhat slower, you reduce the overall adverse event level in the patients. And it gives the physicians a lot of control. And the other aspect is that before you dose, you already determine the level of tumor burden at that time. And it already tells you with a high degree of precision what the patient is likely to experience.

Overall, when you look at the profile, we have about a little bit more than two percent of the patients can experience high grade cytokine release syndrome, and about seven percent high grade neurological toxicities, or ICANS. That is very low compared to any other T cell mediated approach, whether this is through T cell engagers or CAR Ts. And that is immediately experienceable. So when you dose within a short period of time with other types of approaches, you get very quickly these adverse events. So the centers very quickly within a day or two know how different the product actually is.

That creates a lot of confidence and creates a lot of confidence with the physicians. But also, it’s a remarkable experience for the patients because most of our patients do come from prior transplant. So going through the conditioning regimen for transplant. Transplant is quite a horrific trip that you go through with a lot of adverse events and quite a lot of drama quite often. And so to be able to go on a very active therapy where you, from your sensation perspective, have very little sensation of that therapy is obviously a huge differentiator.

And then as you sort of start to look at three, four, five months out, you start to get a good feel for the impact the product had. And obviously you also see that when the product also persists over time, that there’s a high probability for longer term outcome. And I think that’s sort of as we see the engagement with the centers. We see kind of a lot of very positive momentum building based on those experiences.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Very insightful. And while we’re thinking about launch trajectory, in Q1, you booked $9,000,000 sales. And how should we think about the rest of the quarters in 2025?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Right. So we’ve been very careful not to give guidance on revenue. And the reason for that is that the launch actually has several elements where you would expect an element of acceleration. Obviously the first one is it’s the number of centers that are open. The more centers you have open, the more patient flow you can generate.

That’s an obvious one. Within each center, you tend to actually start with one or two physicians that provide the therapy. But quite often, if it’s a larger center, you might have five to 10 physicians actually treating and caring for these patients. So you actually have also more and more of those physicians over time that start using your product. And then the third element is that when you have a new therapeutic approach that you offer in an indication, you tend to first actually use that therapeutic approach for patients that are in very poor condition.

You have nothing else to give. That’s how you often start. And then as you gain information and evidence, you then actually start to actually expand and go in better and better patients. So even with that, you actually increase the range of patients you consider for the therapy. And we’ve seen that actually play out in the pivotal study where one of our actually first physician who treated a patient on the pivotal study was a physician in his 60s, a few decades of stem cell transplant experience.

And he started actually using the product for a patient that was transplant ineligible. Because there’s nothing else he could offer. He had, it was an 80 year old patient, he had made a very good experience. Safety was good. The outcome was fantastic for that patient.

So then he started to actually move more and more into younger patients, fitter patients, etcetera, until he basically covered the entire range. That is quite a typical journey. These physicians are used for their patients to pass away. So they’re really, they’re kind of very, they have to be very hard nosed about data, about facts. And so they actually, that experience is a critical parameter.

So those are several elements in the dynamic that we have. And that makes it actually nontrivial to project the actual launch itself. So internally, we think we need at least three quarters to, even for ourselves inside the company, get a sense and narrow the range of the trajectory for the launch. What we can say is we’re clearly out of the gate in a very, I think, very good way. We see very positive momentum.

And it’s sort of reflected also with sort of the consensus amongst analysts as well. We’re performing actually, we’re performing very well against that. So we’re very positive. We’re very encouraged by what we’re seeing. And we’ll keep you updated as we’re sort of getting our next borders under our belt.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: I guess you would not agree with some speculative thoughts in the past since most of the CAR T therapies actually launched by pharma. And so there was a speculation that maybe it will be very challenging for biotech to launch a cell therapy product.

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yeah. I mean, that was certainly an overhang that we were dealing with is because even the larger organization had challenges to deliver the complexity that I explained upfront and what you need to do to deliver. And so very much a show me type of situation. So we’re very pleased with where we are and how things are going.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Maybe if you could share some manufacturing metrics of performance. So in real world, what is success rate? And since you have a manufacturing in UK, so logistically, maybe how much longer time you would add for the vent to vent time compared to manufacturing actually in the same location for the infusion centers?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yeah, so the experience that we had in our pivotal study was that we have about five to 6% of the products that we were manufacturing were out of specification. So it was very low. We see, obviously, early days on the launch, but we see that we’re tracking very nicely along those lines. So we believe that we have very consistent quality and are reproducing our experience that we had in the clinical development very nicely. The turnaround time is an interesting thing.

There is a sense that if you’re sitting on the other side of that water between The UK and The US, that it would take an awful lot of time to get there and would add an awful lot of time on the logistics. It actually does not. One of the interesting things when you think about what adds time and logistics is if you have to run relays. And most of the places in The US, you will have to run relays no matter where your manufacturing site is. And you will relay through other cities or other airports to come in, which adds substantial time.

What’s very important is reliability of the transfer. So most of the largest cities in The US have multiple direct flights to London Heathrow per day. So that gives us actually ample opportunity for the shipments. It’s one leg for most of these flights. And reliability and predictability when the product comes is actually at least as important or whether you might have an hour plus or minus in terms of transit time.

What we are doing on the way back is we have an opportunity to actually ship product into The US while we’re finishing the release for the products. And then actually have the product go from a depot, which is with our partner Cardinal Health, then actually go basically the final miles to hospital. So the logistics actually doesn’t add any significant amount of time here. And so far, also with the movements that we’re seeing on the political side, we don’t actually see an impact yet from a practical perspective at the sites of entry in The US. And we’re obviously monitoring that very carefully.

But at this point in time, we don’t think we have a difference in the turnaround time compared to a situation when we were to actually run the operation here in The US.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Super helpful. And I think now we can say Albicella is a truly differentiated CD19 CAR, especially on safety front. So where do you see the opportunity to expand indications? Maybe you can also comment on the other ongoing trials in pediatric ALL, for example.

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yes. So one of the things that obviously, as we talked about, is very obvious with ObiCell or a capsule is the safety profile, that immediate experience. But what drives a lot of excitement with the physicians is the fact that we’re having a significant proportion of patients in long term remission without subsequent therapy. And that is qualitatively a different outcome. And that is what resonates probably most for the physicians, that opportunity to get to long term outcomes.

Now we’re going to provide an update at in give or take ten days on longer term follow-up with close to three years of median follow-up. And obviously at that point, level of follow-up gives you a very clear indication of how indeed the outcomes are actually tracking. And I think it will be very positive presentations that we’re going to have. We have two orals and two posters at EHA. So that’s where a lot of the excitement comes from.

And you may remember we also showed that actually adding stem cell transplant post OB cell didn’t improve the outcome for patients. Which was also first actually in the field. And so they’re indicative that maybe that’s something you shouldn’t be doing. So that’s kind of where we are in terms of that side of the equation. As part of the approval, we also have an obligation to develop in pediatric patients.

And that trial is progressing well. It’s a smaller trial. We’re going to have the data from that trial at ASH at the end of this year planned to present. And what we’re looking to do is, and what we’re in conversation with, is to see whether there’s an opportunity to actually move more forward in the pediatric setting to actually expand the label and go for a range that goes from kids all the way up to old age as sort of the age range that we could actually tackle. This is still in progress.

The conversation is ongoing with the agency, and we’ll obviously keep you updated on the progress there. Where we have made very nice progress is on the autoimmune side. We have initial set of patients that we treated that had systemic lupus. Most of these all of these patients had kidney involvement. These are very advanced patients that we were treating, quite different from some of the early academic experiences.

And we see very significant levels of activity, very quick impact on the overall condition of these patients. And what we also see that in a proportion of the patient, we also see improvement of their kidney function over time. So we had half the patients show, even with very limited follow-up, show actually a complete remission, renal complete remission. Which obviously is very encouraging where we keep monitoring these patients. So the path forward is going to be to focus on patients with lupus nephritis, which is the majority of SLE patients with advanced stage disease have actually kidney involvement.

Focus on lupus nephritis and go into a patient population that actually has exhausted the current therapeutic options. So these are patients that have gone through the B cell targeting agents. These are set of monoclonal antibodies from Belysta, Cefnello, and then the CD20 monoclonals. And then also have failed on calcineurin inhibitors. So now you’re in a situation where you’ve kind of exhausted the therapeutic options.

And at that point you have an opportunity to actually run the trial as a single arm trial. Now by focusing on lupus nephritis, it also gives us an ability to focus on a hard endpoint. This is actual filtration function of the kidney. That’s a biochemical measure that you can actually do. And the beauty of that is it’s an objective endpoint.

And that also allows you to then actually keep the study size relatively compact. So we’re currently planning to treat 30 patients into that study. So that’s a very unusual approach in that regard. And what’s obviously important is 30 patients wouldn’t be enough to discharge the safety considerations. What we obviously do have, because we have an approved product, we have a large safety database from our leukemia work.

And that safety database actually supports the activities obviously on autoimmune, Which is why we don’t have an obligation to go beyond of that trial to actually have an appropriate level of data to then consider moving forward. So a very important, I think, opportunity here. We’re also obviously going to record secondary endpoints, SLED I scores, etcetera, which look at other manifestations of the autoimmune disease. A lot of these patients have rheumatoid arthritis, have skin issues, have other organs impacted. And obviously you’ll record that as well.

So that’s what we’re doing in lupus nephritis. Obviously moving into a pivotal study, planned start of that trial second half of this year. We’re also running an exploratory trial in patients with progressive multiple sclerosis. And the hypothesis there is that B cells that are involved and drive the disease may not only be resident in your bone marrow and in the lymphatic tissue, But it also may be present in the brain itself, behind the blood brain barrier. And one of the remarkable things about CAR T cells is that they actually manage to cross the blood brain barrier very efficiently.

This is not a hypothetical for our product because we have treated patients with primary CNS lymphoma. And we’re able to show that indeed in primary CNS lymphoma we can actually get very significant levels of activity. And also on the acute leukemia side, you do actually have a lot of patients that have extramedullary disease in the CNS. And we can also show that we’re active against that disease. So we know the product is very active.

It has obviously a very good safety profile. And so we are looking forward to running this exploratory study and to see whether indeed that type of a reset could actually transform the outcome for these patients. And that’s clearly a question we’re asking. It’s also a scientific and clinical question. But if answered positively, I think could open up a very significant opportunity.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Very exciting. Maybe can you also comment, when do you plan to talk to regulatory agencies and thinking about a pivotal trial design for both lupus nephritis and in the future multiple sclerosis?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Yes. So the conversations for the pivotal study in lupus nephritis, that happened. So what I’m talking about is actually based on conversations that we have with the agency and input that we have received from the agency. Multiple sclerosis, obviously we need data. And then with the data we’re going to definitely have those conversations.

But that’s data driven interaction.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: We already see some feedback from regulatory agencies in this space. Curious your thoughts on do you see accelerated path and the single arm trial actually across different indications? Or it’s also determined by unmet needs and also available therapies across different autoimmune indications?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Right. Look, the regulators take a very rational approach. And it’s actually almost independent of the actual disease setting. So the first thing is if you want to if you think that a single arm trial is what would be appropriate, obviously you have to have a very high medical need. You have to have exhausted all other treatment options.

And you have to have a very high treatment effect. So in very simplistic terms, you have to think that you’re going to be at least twice as good as anything else that could possibly be given to these patients. Very simplistically. And that’s true for, if you go through oncology and you go through all these single arm trials, that’s sort of where the primary endpoint were pegged at. So that’s kind of the requirement.

If all of that is true, then I think running a single arm trial is sufficient to demonstrate that indeed you have an adequate level of activity that gives you the level of confidence that indeed the outcome you’re looking at is real. That’s what that is. That’s the stats behind it. If you have obviously a limited level of activity, as we’ve seen in lupus with monoclonal antibodies where you eke out four point improvement in a SLED I score. Well, at that point you need a randomized controlled study because that’s just not enough of a signal.

And then you need a large sample size to actually be able to show that there is a statistical difference between the curves. Or at least you can hope it does. At times that was true, at times it was not. So it’s very much down to the actual treatment effect that you actually can get. So if you fulfill all those requirements with high treatment effect, etcetera, then that is a possibility.

And I think there’s an openness to then actually consider that. You would still have enough, you still need to have enough safety data to sort of support that. Typically what we’ve seen across the board for high medical need settings, etcetera, is at least 100 patients worth of data, which has been quite consistent across the last fifteen to twenty years, kind of what was required. So those stats behind that are pretty steady. And I think the agency has been very consistent in the way they’ve been looking at that.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Yeah, pretty easy to follow. And maybe lastly, could we reiterate the key catalyst and milestones in the next twelve months from our to us?

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Right. So first off, obviously, we’re in a as I mentioned before, we’re sort of in a show me place. We have to prove that we can actually sell product. Okay. So that’s kind of the primary focus that we have to make sure that indeed we can deliver on launch.

That’s the first. The second is obviously we have data at DHA and then at the end of the year at ASH related to the leukemia program. And we’re planning to have updated data from our SLE Phase I study that we’re looking to submit for ACR. And then obviously as we go into next year, I think there’s obviously that’s when the pivotal study should be underway. And we’re looking forward to keeping you updated on that.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Fantastic. And we’re going to wrap up here. And thanks, Christian, again for a super insightful discussion, as always. And thanks, everyone, for staying with us.

Kristen Iting, Chief Executive Officer, Autolus Therapeutics: Thank you.

Kelly Xu, Senior Analyst, Biotech Team, JeffA: Thank you.

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