Autolus Therapeutics Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 8, 2025

There are 10 speakers on the call.

Operator

Good day, and thank you for standing by. Welcome to the Autolus First Quarter twenty twenty five Financial Results Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press 11 on your telephone.

Operator

You will then hear an automated message advising your hand is raised. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Amanda Cray, Executive Director of Investor Relations. Please go ahead.

Speaker 1

Thank you, Gigi. Good morning or good afternoon, everyone, and thank you for joining us on today's call. With me are Chief Executive Officer, Doctor. Christian Eitan and Chief Financial Officer, Rob Dolesky. I'd like to remind you that during today's call, we will make statements related to our business that are forward looking under federal securities laws and the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.

Speaker 1

These may include, but are not limited to, statements regarding status of the ongoing commercial launch of Ocassel in The U. S, Autolus' manufacturing, sales and marketing plans for Ocassel, the market potential for Ocassel and the status of clinical trials, development and or regulatory timelines and market opportunities for OviCel and our other product candidates. These statements are subject to a variety of risks and uncertainties that could cause actual results to differ materially from expectations and reflect our views only as of today. We assume no obligation to update any such forward looking statements. For a discussion of the material risks and uncertainties that could affect our actual results, please refer to the risks identified in today's press release and in our SEC filings, both available on the Investors section of our website.

Speaker 1

Turning to Slide three, you'll see the agenda for today's call. As usual, Christian will provide an overview of our operational highlights. Rob will then discuss the financial results, and Christian will conclude with upcoming milestones and closing remarks. We'll then take questions. With that, I'll turn it over to Christian.

Speaker 2

Thank you very much, Amanda, and welcome, everybody, to our first quarter twenty twenty five, results. First off, we had a great first quarter. The launch has been off to a really good start. We have seen a substantial, level of interest by the physicians in the product profile. Clearly, there is a significant unmet need, for the patients with relapsedrefractory acute lymphoblastic leukemia.

Speaker 2

And we're very pleased to report 9,000,000 in recognized revenue in the first quarter. Obviously, foundational to the ability to really reach the patients is that we have to be present in the respective clinical centers. And as of yesterday, we have 39 centers that are authorized to deliver or cancel. This is a great accomplishment, from our onboarding and market access team, and we're currently at a level of about ninety percent of total US medical lives covered, which is a great position, this early in a product launch. As of April 1, CMS also published the codes for in and outpatient use for patients on government programs.

Speaker 2

And, with that, we have now an ability to get a capsule also, be eligible for reimbursement for patients on government programs, in addition, obviously, to the patients who have been covered and are covered through the commercial programs. So this sets us up in a really good way, leading into the second quarter. And we're excited about the, initial momentum that we've seen in Q1 and see that very nicely carried over, into the second quarter. With that, I'd like to move to slide number five. When we look at the opportunities for growth, obviously the first opportunity is in The US.

Speaker 2

And, one of the key things that we're planning to do from here on to the year end is really increase the number of centers that are activated and in a position to deliver Ocassel therapy. We're gonna go from what's now 39 centers to approximately 60 centers, which will give us approximately 90% access to patients across The US. So that's also critical. And one of the key things that we're going to see as we go through the course of this year is also that we're looking to build additional momentum, and, drive and support the launch going forward, also with additional data updates. A key update is planned for this quarter, based on long term follow-up from the FELIC study, which I believe will be very encouraging and very helpful to understand the impact that, ocapsule can have as a single agent in this relapsed and refractory patient population.

Speaker 2

So first push, obviously, in The US, also actually driving, certainly as we go into next year, to start expanding the market share for CAR T products, and then gradually build from there. Now, the second layer of expansion that we're looking at is geographic expansion. We reported about a good week ago that we have received conditional marketing authorization from the MHRA in The UK, and are now in the process of engaging with NICE and the other relevant bodies in The UK to get the product actually, through the reimbursement and access process, and then into the launch in The UK. In Europe, we're progressing well, and we expect, to receive a decision by the European agency in the second half of this year. From a European perspective then, we will initially are planning, to launch in Germany, and then actually move gradually from there, obviously with various different processes that we have in Europe related to pricing and reimbursement, which has a big impact on obviously the sequence and how to actually progress in Europe.

Speaker 2

Now looking beyond the immediate launch and the geographic expansion, we also believe that the product has additional opportunities in acute lymphoblastic leukemia. Right now, we have the data set in the relapsedrefractory population. This is obviously, these are obviously patients that have already gone through a very significant amount of exposure. They've been typically up to three years of frontline therapy, have gone through high dose chemotherapy. Many of them have received other agents, targeting agents, as well as might have received stem cell transplants.

Speaker 2

So this is a very advanced population. What we do know from ObiCell and or Castle is that the product, when used in patients that are in the earlier stages of relapse, and also patients that have lower tumor burden at the time of therapy, that patients do better, with those profiles. And we believe one of the key areas that I think is important to explore is the utility of the product, ultimately in a frontline consolidation setting. We will start to explore, see exploration of frontline settings through investigator sponsored trials, and we'll certainly consider our steps, in addition to those. In addition, when we look at the pediatric population, we're currently treating pediatric patients, and we're planning an update and results to be presented by the end of the year.

Speaker 2

And we also will review whether there's an opportunity for us to move ObiCell, also forward in the pediatric setting, and hopefully find a way to make the product accessible to patients in this setting as well. Now moving to slide number six. Obviously, what we do have with O B cell is an ability to target the entire B cell compartment. And there's quite a significant additional set of opportunities that we do have with the product that we can see beyond acute lymphoblastic leukemia. So moving to slide seven, briefly summarizing our current experience with obi cel across a range of indications.

Speaker 2

Obviously, you're all aware, and you're very well familiar with the product profile in the relapsedrefractory adult population, obviously as presented, as well and published in the New England Journal, for the FELIC study. But we also obviously have earlier data in pediatric patients as well. What we do see across all of these trials that we've conducted, both with our academic partners at UCL, as well as in the subsequent trials, we do see consistently that the product actually achieves very deep MRD negative responses. Or to put it differently, the product has an ability to reset the compartment in a very profound way. That profound way is also shown with the experience we had in the old CAR nineteen study initially where we have more than five years of follow-up, as well as the Felix study, where we're gonna, as mentioned, give an update on long term outcome later this quarter.

Speaker 2

We're also obviously seeing the ability of the product to get a proportion of the patients into long term remission, clearly indicating that we had an ability to make an extremely deep cut into the compartment and completely remove the acute leukemia cells. Now, this is the experience in leukemia. We have very similar outcomes that we have observed in patients with non Hodgkin's lymphoma, which is part of the old CAR nineteen extension study. And what we've seen in that setting is very high levels of metabolic complete remissions. And when we look at the large B cell lymphoma patients in those cohorts, those patients do actually have, also the majority of those patients have long term outcomes as well.

Speaker 2

So quite clearly, a very deep activity. What we also see is that the safety profile that we have in acute leukemia is very favorable. Remember, we do not have an obligation for a REMS, for a capsule, in relapsedrefractory adult ALL. But when we then look into the lymphoma patients, the profile actually improves further. We have no high grade cytokine release syndrome in those patients, and we have not actually observed, neurological toxicities or ICANs in that population as well.

Speaker 2

So when we think about this profile, and where we can actually, where a deep cut in the compartment can make a big impact on outcomes, there's sort of two key areas to focus on. One, I already mentioned, is the frontline consolidation in aggressive B cell malignancies, where we aim for long term outcomes while avoiding overtreatment of these patients. What we've seen so far is just add on strategies, and we keep adding more and more toxicity in these patients. However, we start to see sort of diminishing returns, and in fact, at times, negative outcomes from adding additional toxicity. So getting to much more compact treatment, shortening treatment, and get to long term outcomes has to be an objective going forward.

Speaker 2

The second area is obviously the wide range of B cell mediated autoimmunity, where we've seen obviously some quite remarkable data from Georg Chet's team in Erlangen, indicating that indeed if you do have a deep cut in the compartment, you can actually transform the outcomes for these patients, and those outcomes appear to be sustainable. So the goal in those indications is to really get sustained effects, with a one time intervention. And that obviously is a very attractive proposition in those disease settings. So with that, I'd like to just, on slide eight, briefly just talk about autoimmune disease, and the fact that what we're looking at when we look at particularly advanced patients, we have on the one hand an inflammatory process where you have antigen and B cell engagement, as well as T cell engagement that sort of form a loop of activity that actually forms a very visual cycle, and continuously actually drives an inflammatory process in these patients. The autoreactive antibodies obviously have then an ability to recruit immune cells and complement onto tissue and into organs, and actually start to create damage on that tissue.

Speaker 2

If that prolongs, you start to actually get scarring in the tissue, you get scarring, and loss of function in those organs, and you can have very dramatic effects and issues that these patients do build up over time. The treatment is basically various forms, of immune suppression that you run-in these patients, and that also in their own right actually drive significant adverse events. And unfortunately, for many of these patients, do not actually address the underlying disease in a way that allows these patients to live normal lives. So when we look at the ability for B cells to really target, this type of therapeutic intervention, it gives you an ability to really block that cycle, crack it open, remove the B cell component that carries memory, remove the plasma blasts which express the autoantibodies, and with that, actually stop this vicious cycle of continuous activation and continuous damage in these patients. Now the patients that we're looking for, particularly from a CAR T perspective, are patients that do actually have disease that is advanced, disease that already actually shows impact on organs, particularly organs like the kidney.

Speaker 2

And so these are patients that do have an element of structural damage. Now when we think about the impact of the therapy, clearly we expect the therapy to have an immediate impact on the inflammatory process and the inflammatory parameters, as well as a lot of the manifestations that we see in these diseases, and I'll talk on the next slide about the various types of forms of disease that these patients experience. And you also would expect that gradually, either have a stabilization of the underlying organ that's impacted, and if it is early enough in the progression of the disease, an ability to actually reverse the negative outcome on that organ. So at least stabilize, and at the upside, improve the organ function. So with that as sort of a backdrop, what I'd like to do is really talk now briefly about the outcome that we have presented at the R and D event from our phase I CARLITAL study in patients with systemic lupus cerebrosome.

Speaker 2

So when we look at our patient groups that we have, and this is now on slide number nine, when we look at our patients, we do see that these patients actually are quite more advanced compared to the patients that were treated at the University of Erlangen by Garochevzty. These patients are older, they're 19 to 50 years of age, and they also had quite long disease histories. So the disease history of these patients was at least three years up to twenty three years. So these are patients that have been battling with the disease for a long period of time. And when you look at the SLEDI scores, which are disease scores in these patients, you see them range from 16 to 28.

Speaker 2

So this is a very severe population. And in fact, all of those patients had kidney involvement. So all of them actually had already signs of kidney damage when we looked at these patients closely. So when we look at the patients, we do see that five of six patients had class IV disease, and four of the six also had class V components. And these are patients that have gone through the standard of care, have also, already gone through the challenges with regards to B cell depleting agents, as well as calcineurinhibitors.

Speaker 2

And what we're seeing is that, in many of these patients, four out of six, we already had significantly impaired kidney function. Now, when we look at the, overall adverse event profile in these patients, we do see that the patients obviously had quite a lot of impact related to kidney damage, leading to hypertension, etcetera. But when we look at the immunological toxicity, we do see that the CRS that we're observing in these patients is that three out of the six patients have observed grade I cytokine release syndrome, but none of the patients has experienced neurological toxicity or eye cancer. This is relevant because every, approach that was tested, CAR T approach that was tested, outside of the experience at the University of Rheerlangen in patients with lupus nephritis have reported ICANS in their small data sets. So with that, moving to the next slide.

Speaker 2

This is slide number 10. And we're now looking at the individual profile that we're seeing in the patients, based on the SLAD I2K scores. So the SLAD I scores are scores that actually look at a composite of parameters related to the function of organs, autoantibodies, in these patients, complement, but also looking at the experience related to other forms of the inflammatory diseases that these patients may see. Now, I'll focus first on patient number one. Now what you can see is this patient had a SLED I score of 28 at the start.

Speaker 2

And in addition to obviously having very significantly impaired kidney function, and obviously, double strand DNA antibodies and, low levels of complement, the patient actually had other manifestations of disease, including rash, arthritis, and alopecia. Now when you think about which ones of those types of parameters the patient actually can experience, those are in fact, rash, arthritis, and alopecia. The lab parameters you can't sense, and the kidney function you can't really sense either. So the primary experience of the disease is around those inflammatory processes. What you can see is that very quickly, within a short period of time, all of these symptoms with these additional inflammatory processes actually have been removed.

Speaker 2

And you see then a gradual improvement also on the kidney side. When you then look at the other patients, you see combinations of those, various forms of manifestations of inflammation. But you also see that all of these patients improve on the top line. We have three months of follow-up at least. And on the bottom line, we have only one month of follow-up.

Speaker 2

So with that, what I think is important to realize is that although this is a limited amount of time in terms of the follow-up, we had very quick, improvement on those inflammatory processes. And we also started to already see that three of the six patients did actually achieve, renal complete remissions. And that is obviously very meaningful in this population, and we expect those patients to sort of continue to improve over time. So a very strong start. Gives us a very strong set of indications in terms of the ability of the therapy to actually improve the outcomes for these patients, obviously combined with a very good safety profile.

Speaker 2

When we look at the properties of the product, the properties actually are very, very consistent with what we have observed in the oncology setting. We see comparable level of peak expansion. What we do see is shorter persistence. So the persistence here is around three months, maybe for a few patients a little bit longer. And when you see loss of persistence, you also do see B cells coming back, and the B cells that are coming back are predominantly naive B cells, as you would expect.

Speaker 2

But then over time, for those patients where we have that data at this point, start to differentiate into more differentiated forms of B cells, as we would have postulated. So with that, we're going to move to slide number 11. Slide number 11 actually looks at the opportunity that we have in lupus in general, with a particular focus on lupus nephritis. What we're looking to do here is really focus on the patient population with the highest medical need, which are the patients that are refractory to standard of care in the lupus nephritis, group of patients. And that's around twenty five thousand to thirty five thousand patients, or about, give or take, ten percent of the overall SLE population in The US.

Speaker 2

The reason why we're focusing on lupus nephritis is on the one hand, the medical need, but on the other hand, it is the importance of having an objective endpoint that you can follow. We just looked on the slide before on SLED I scores, and you could see the complexity of the scores. What you haven't seen is that some of those scores are actually based on either physician assessment or patient assessment. So in other words, there is an element that actually, is a much softer type of data that goes into these scores, relevant to describe the disease, but much harder to actually think about statistical outcomes, and be able to actually get to relevant data in a smaller data set. Going after lupus nephritis allows you to look at renal complete remissions, and with that have a very clear endpoint that it can actually follow.

Speaker 2

Now moving to, slide number 12. What we see in slide number 12 is basically the trajectory that we're planning for our development in lupus nephritis. What we're doing with our current study, with the Carlisle study, is actually establish a fixed dose for adult and adolescent patients. With that fixed dose, remember these were SLE patients with kidney manifestation factor all categorized as lupus nephritis patients. Those patients actually, when we look, at them, are the right kind backdrop that we would also expect to include into the single arm phase II study that has a pivotal character.

Speaker 2

This particular, population are patients that also have been already exposed to B cell depleting agents. So these are typically antibody based therapies, as well as a calcineurin inhibitor, and are past those two therapies. So it's a proper refractory lupus nephritis population. So the approach here is to have an opportunity for a compact study with an objective endpoint, and with that, have an ability to be first to market in this indication with a CD19 CAR T product. We're then actually envisaging that from there on forward, there's an opportunity to consider moving into a somewhat earlier stage of disease, where we're going to look at a comparative or randomized trial against standard of care.

Speaker 2

But clearly, the core of the approach is a fast to market strategy based on the refractory population with an objective endpoint. So this study obviously is already, getting started. We have gone through the regulatory process in The US, and interacted with the FDA on the design of the product, and also have an open IND for lupus nephritis. And we're in the process, obviously, of going through the regulatory steps in other jurisdictions that, we're planning to include in this clinical trial. With that, we're moving, to slide number 13, and going to look at one of the opportunities we're very excited about, which is the opportunity in progressive multiple sclerosis.

Speaker 2

Now this is the, part of the disease that really has the highest medical need. It's about thirty percent of the relapse remitting, of the total population. You have relapse remitting, about seven hundred thousand patients, and you have progressive MS in about three hundred thousand patients. That is where we're planning to go. These are patients that have gone through, obviously standard of care, and progressed despite exposure to standard of care for at least six months.

Speaker 2

Now, when we think about MS, it's a disease that we know has a significant B cell component. They're obviously a disease where CD20 targeting monoclonal antibodies, are effective. However, the disease itself obviously is driven not just by T cells that are in the periphery and are reachable with a soluble agent that you infuse into the blood, But there are also B cells that sit on the other side of the blood brain barrier in the brain itself, and obviously those cells are not, reachable for conventional therapeutic approaches that are based on either proteins, T cell engagers, etcetera. Now, what we do know about OB cell is that OB cell actually works very well across the block brain barrier, and we've explored that in primary CNS lymphoma in collaboration with our colleagues at UCL, and could show that indeed the product had an ability when given systemically, so infused into the bloodstream, had an ability to cross the blood brain barrier and actually lead to tumor reductions in the brain. And that obviously is what exactly the type of activity you would need in these MS patients.

Speaker 2

So getting at those, B cells, malignant B cells, or B cells that are sitting behind the blood brain barrier is at the core of what we're looking to do. We believe obi cell is exceptionally well positioned to actually have that type of an activity. So when we look at the approach that we're taking here, is we're starting up a study, in progressive MS. And in fact, those are patients where we're going to run through a dose escalation. We know in the CNS lymphoma that two hundred million cell dose was the cell dose that was active, and gave us proper activity in the brain.

Speaker 2

But going to run through a dose escalation here, obviously higher levels of doses than the fifty million cell dose that we have used so far in the SLE patients. We're going to look at a range of biomarkers, as well as imaging, And we're going to follow these patients to understand the clinical impact and outcomes that we could see in these patients, and to see whether we can see an impact on the clinical visibility progression of these patients. With positive data, we obviously would then move into randomized, phase IIIII study, to drive towards, a registration for this type of an indication. Very exciting program, and we're looking forward to updating you, as we're making progress in this setting. So with that, I would like to, hand over to Rob, who will walk us through the financial results.

Speaker 3

Thanks, Christian, and good morning or good afternoon to everyone. It's my pleasure to review our financial results for the first quarter of twenty twenty five, and I'll be referring to Slide 16 in the presentation. For our first quarter of product sales, net product revenue for the three months ended 03/31/2025 was $9,000,000 We're off to a strong start with The U. S. Launch of a capsule.

Speaker 3

As Christian noted, as well as, included in the press release, earlier today, on April 1, CMS included Ecatsol in their published coding determinations and outpatient payment rates, formalizing reimbursement for patients on government programs. The CMS hospital outpatient perspective payment system or OPPS splits the therapeutic dose of a capsule into two administrations for coding and billing purposes. As a result, we are working with our treatment centers on implementing the coding and payment policy from CMS. As well, we are internally assessing any potential impact on the timing of our revenue recognition moving forward. Moving on to cost of sales.

Speaker 3

In the first quarter of twenty twenty five, cost of sales totaled 18,000,000. This amount includes the cost of all commercial product delivered to the authorized treatment centers, including product delivered but yet not yet administered to patients. So it's worth noting here that the sales value of these products is not yet recorded as product revenue in the P and L but is reflected as deferred revenue on the balance sheet. Additionally, cost of sales includes any canceled orders in the period, patient access program, product, and third party royalties for certain technology license. Our research and development expenses decreased to 26,700,000.0 for the three months ending 03/31/2025 compared to 30,700,000.0 during the same period in 2024.

Speaker 3

This change was primarily driven by commercial manufacturing related employee and infrastructure cost shifting from r and d to cost of sales and inventory following the approval of a capsule and the associated accounting change, that occurs at that time. This was partially offset by an increase in OB cell clinical trial activity and supply costs. Our selling, general, and administrative expenses increased to 29,500,000.0 for the three months ended 03/31/2025 compared to 18,200,000.0 in the same period in 2024. This increase was primarily due to salaries and other employee related costs driven by increased headcount supporting US commercialization activities associated with the launch of a capsule. Our loss from operations for the three months ended 03/31/2025 was 65,200,000.0 as compared to 38,800,000.0 for the same period in 2024.

Speaker 3

And finally, net loss was 70,200,000.0 for the three months ending 03/31/2025 compared to 52,700,000.0 for the same period in '24. Our cash, cash equivalents, and marketable securities at the end of at the end of q one twenty twenty five totaled 516,600,000.0 as compared to 588,000,000 at the December, 2024. The decrease was primarily driven by net cash used in operating and investing activities, but also impacted by a delayed cash receipt of approximately 20,000,000 in r and d tax credit that we expect from the UK HMRC. We continue to believe that with our current cash, cash equivalents, and marketable securities, we are well capitalized to drive the launch and commercialization of OB cell in relapsedrefractory B ALL and to obtain data in the loop lupus nephritis pivotal trial and MS phase one trials that Christian just talked about. I'll now hand back to Christian to wrap up with a brief outlook on expected milestones.

Speaker 3

Christian?

Speaker 2

Thanks, Rob. Moving to Slide 18. First off, in terms of the activities that we're expecting on the hemonc side, first off, we're excited, obviously to update you on the longer term follow-up, from the PHELEC study, which we expect to be able to do, towards the end of the second quarter. We also expect that for the second half of the year, the notification from the EU regarding, the market authorization application decision, for the patients with relapsedrefractory adult ALL. And we are planning towards the end of the year, to present initial data from our pediatric study, PY1.

Speaker 2

When we look at the autoimmune side, we're planning for an update and data presentation for the Carlyle study, in the fourth quarter. We also expect to, be reporting first patient dosed, for the phase II lupus nephritis study, as well as having first patient dosed for the progressive MS phase I study towards the second half, and actually towards the end of twenty twenty five. And then finally, we also are moving forward with our program AUTO8, the dual targeting CD19 BCMA approach, where we're evaluating that product, in light chain amyloidosis in a phase one trial together with our collaborators at University College in London. With that, I'll get to slide number 19, which is a summary from what we covered today. First of all, we believe that Autolus is well positioned for value creation, and we're building on a very strong foundation with Ovicell.

Speaker 2

Obviously, we talked about the Ocalcifer launch in The US. We had a strong first quarter. We're building towards 60 centers, which give us the vast majority of patients access in The US. We obviously have established infrastructure You remember the nucleus that's shown in the picture on the right hand side, as well as obviously our commercial infrastructure, which is executing very well.

Speaker 2

And we're starting to move into other jurisdictions with the MHRA authorization authorization that we have received, and we're preparing for launch in The UK. And we're also gearing up, for the activities in Europe with an expected decision, from the EU in the second half of the year. When we look at Ovicell in terms of the opportunity beyond the initial opportunity in adult ALL, we obviously are building on the unique mode of action, with a fast off rate CD19 CAR T, which gives us, obviously a very high level of activity, as well as a very well manageable, and tolerable profile for the product. Remember, the product was the first one, the CD19 CAR T product, that was approved without a REMS obligation. And, as indicated, we're having long term follow-up data coming relatively shortly, and then data sets from PY1 study and the Carlyle study, towards the end of the year.

Speaker 2

All of this is built on a strong cash position of $516,000,000 at the end of Q1, and I think sets us up very well from an execution perspective. So with that, I think we've reached the end of the presentation, and we'd like to open up for questions.

Operator

Thank Your first question comes from the line of James Shin from Deutsche Bank.

Speaker 4

Hey, good morning guys. Thank you for taking my questions. I have a couple. Firstly, I guess for the April 1 coding update for OCATSL and the $18,000,000 of COGS and those associated deferred revenues, can you say whether the 9,000,000 of revenue recognition for 1Q twenty five was triggered upon initial dosing, or is revenue recognized only upon patients receiving their full split dose? And then going forward, it sounds like there may be some impact from the April 1 coding update.

Speaker 4

And I have follow ups.

Speaker 2

All right, James. First of all, thanks for joining. Before we actually, Rob will walk you through the Rebrik answer in detail, I think just to get started, all patients that we had in q one received both doses, so it didn't matter from a practical perspective. But I'll have Rob talk you through the rev rec, approach that we're taking. Rob?

Speaker 3

Yeah. Hi, Jace. Thanks for the question. So let's start maybe with the the current guidance and approach that we had discussed previously was, in fact, kind of that full recognition with confirmation on the first administration, and that is what's reflected in q one. As Christian said, we've confirmed that all those patients, in fact, have received both the first and second dose as well, but we were we were really following the the policy that we've kind of discussed before.

Speaker 3

As noted, CMS, published, and this is effective April 1, on the OPPS side. So this is for outpatients, a split coding and billing into two administrations. So as I noted, we're working with the centers to adjust things on their end. We are evaluating the implications on our own revenue recognition policy as well. So, unfortunately, we don't have a final answer here, but I think it it might be important maybe to put a a few data points here and and and put it into some perspective.

Speaker 3

When we look at the, experience that we have in particular in the Felix study with respect to patients that completed or didn't complete both administrations, five point five percent of the patients did not receive the second dose. Right? And that's before considering any inpatient, outpatient setting in the commercial space. But so from a total number perspective, you know, it's it's a it's a smaller portion of the overall patient population. These also tended to be much more severe patients with median tumor burden at eighty percent or more.

Speaker 3

The next piece is to think about the timing corridor that we have here. And so if you look at the timing corridor between first and second administration currently, in the USPI, it's ten days plus or minus two days. And from a commercial experience year to date, it's early, but that average has been about nine days. And so given the the the number of patients that we're talking about here and certainly the timing between the first and second administration, while we're still evaluating the overall implications for kinda technical revenue recognition, we don't expect it to have a material impact on the full year sales. So hopefully, helps on that question.

Speaker 4

Thank you, Rob. Secondly, I guess, Christian, I have one on tariffs. I totally understand CAR T products were spared from the initial, retaliatory tariffs via Annex two. And I guess, coincidentally, I believe 10:00 today, President Trump is expected to announce a tariff agreement with The UK. We're also expecting pharma specific tariffs maybe over the next week or so.

Speaker 4

I guess, long winded way of asking, any early color on OCAT's potential exposure to UK or pharma specific tariffs?

Speaker 2

First of all, I understand the question. I think we're operating on about as much information as you do. I think the bottom line is, maybe a few points here. First of all, there's a good reason why blood products tend to be exempt from tariffs, because literally, it's a single individual patient's cells that you're actually going to tariff. So you're to tariff a US and American patient's cells, before that patient can receive the therapy.

Speaker 2

So that in of itself is an interesting thing to think about. So, there's a good reason why typically those were excluded. We don't know where the administration comes out on this. We also don't know yet, obviously, what the deal is between The UK and The US at this point in time. What is worthwhile to remember, though, is that when you think about tariffs, what is relevant in terms of the value that's sort of used to actually, calculate the tariff from is not the price of a product, as the product is sold, but it is the customs value, which is typically linked to the manufacturing of the product itself and the manufacturing related costs of the product.

Speaker 2

And then it has a few additional finer points to it, but basically that's kind of at the core of it. So it is a limited amount. It's not the full amount that then actually would be the basis for the calculation of the tariff. We need to see where we end up. Obviously, there was initially talk of a 10% tariff corridor for The UK.

Speaker 2

You know, we'll all know more in probably about forty five minutes from now.

Speaker 4

Great. And then finally, and this is quite fresh, any chance Doctor. Vinay Prasad taking the RAINS of CBR may result in the FDA revisiting any already agreed upon pivotal trial design such as the phase two you have for lupus?

Speaker 2

I mean, we don't know at this point in time kind of how the, what's the impact on the FDA going forward of any of frantic denominations and the changes that are in the works. Obviously we had a full conversation with the FDA on the trial design, the patient population, the description of the patient, etcetera. So this is not a of a loose sort of conversation. It's a very detailed conversation on the approach. So that said, we don't know where there's going to be, what changes might look like.

Speaker 2

I think in general, there's been sort of two kind of areas that I think we've seen. One is I think a clear statement, from the administration to look to accelerate access, and also to accelerate the approval processes. So that's one dimension that we're hearing. And then there's obviously, various degrees of views on the individual measures and approaches that could be considered here. I think we'll need to wait and see.

Speaker 2

I don't think there's a good answer for this, at this point in time. Obviously, from where we are at this point with regards to the product, I think the trial will give us a very clear answer on the utility of the product in this setting. These are very severe patients. These are not patients that are, you know, where you have actually other options that give them a good outlook. And I think medical need will matter and will continue to matter going forward in those assessments.

Speaker 4

Thank you so much, Christian. I'll yield to the floor. Thank you.

Speaker 2

Thank you.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Matthew Phipps from William Blair.

Speaker 5

Hi. Thanks for taking my questions. Congrats on a nice start out of the gate. Rob, could you give us any more breakdown of the COGS in the quarter as far as just maybe how much came from canceled orders or patient access programs? And then, you know, going back to some of the data from the R and D day, I know it's not a ton of patients, but it does look like you're seeing greater persistence of Ovi cell than what others have seen in the autoimmune disease settings.

Speaker 5

I know that it's kind of consistent with maybe what you guys have obviously shown in in adult ALL and and other hematology indications. But do you think that's a differentiated autoimmune disease, or, you know, doesn't matter in terms of driving that kind of reset?

Speaker 2

Okay. Rob, do you wanna go first?

Speaker 3

Yeah. I'll start on the cost of goods. Thanks for your question, Matt. Maybe stepping back a little bit just to to clarify. In terms of, you know, overall high level, what's going through cost of sales, obviously, contains the the cost of the product manufactured, also includes things like third party royalty license and some other period expenses, distribution fees, for example, it kind of flow through there, as well as period costs associated with idle capacity, and and we talked a little bit about, that at the end of last year even.

Speaker 3

When we dig into the product cost itself in in kind of the components there, the two majority and most significant drivers here is really the cost of the product that was sold and recorded as sales in the period as well as what I refer to those orders that were delivered to the treatment center but not yet booked as product sales, they're showing up in deferred revenue on the balance sheet. That's gonna make up the majority of the product costs related to patients. There are typical, you know, smaller pieces that are gonna be associated with patient assistance programs or or canceled or kinda scrap batches, for lack of a better word. But we expect those to be know, they're with, you know, kind of practice and and and what you would kind of put through cost of sales, but certainly not driving the vast majority of those costs. I don't think we'll get into specifics, you know, kind of patient by patient, on that front.

Speaker 3

I think the other piece here too is just, again, to note the cost associated with all that is, in particular, for the first quarter here, at a relatively inefficient level of utilization on the plant, so there are idle charges as we even saw come through in the fourth quarter of last year when we kind of had to make that change on the accounting front, following the approval of, basically, capacity charges that are coming through on a period expense basis in cost of sales.

Speaker 2

So I'll leave it there.

Speaker 5

To disclose the deferred revenues because it's not broken out in the consolidated balance sheet on the income statement. I assume it'll come in the 10 q.

Speaker 3

Yes. That will be broken out. It's yeah. The amount was about 4,700,000.0, Matt. It it'll be broken out on the

Speaker 6

Thanks, Ron.

Speaker 5

Yep.

Speaker 2

Alright. And then the second question was related to the, profile that we've seen for, obi cel in the Carlyle study. One of the key things that we're really pleased with from the the study is the consistency on how the product did perform. And there's two key parameters to sort of understand performance. One is, the expansion that you do get with the product, which gives you a lot of information about the quality of the product and the consistency of the product.

Speaker 2

And what we're seeing is a very consistent expansion, and then also very consistent, persistence, as Matt was pointing out, somewhat longer than I think what we have seen with some of the other CAR T products. I think what is obviously very encouraging is that what we're seeing is very consistent with what our experience is for OB cell across all indications that we actually evaluated to date. And it is that consistency in the quality, higher levels of expansion than what others have been reporting, obviously in acute leukemia, exceptional persistence, but also here, somewhat longer persistence that was observed, with other programs. We think that is very important, because I would say you need, it's a cell based therapy, in order to get a deep cut into the compartment. The cells actually have to stick around, they have to move around, they have to make contact, and they have to eliminate physically each one of the B cells that, are in that body.

Speaker 2

And so that is, a job that takes a certain amount of time, and so you do need to have a certain amount of minimum persistence to actually complete the job. And we'll leave the fact that we have a somewhat higher or longer persistence than what others have observed, I think is a good thing, and I think should give us a very deep, very consistent, depletion of the B cell compartment in these patients.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Astika Goonewardene from Truist.

Speaker 7

This is Karina for Astika. Congrats on a great quarter. I have a question on the twenty percent quarter over quarter drop for Takartis, which was reported by Gilead in 1Q. Can you quantify how many patients treated would have been eligible for Takartis? Yeah.

Speaker 2

Hi, Karina. Thanks for joining. Interesting question. Obviously, one that we cannot really have a good resolution for, because when you think about the labels that go into the Ticarzus sales, they include both the mantle cell lymphoma label as well as the ALL label. We do know that Briennezy, obviously, has started to expand its use in mantle cell lymphoma, which is sort of actually quite a bit ahead of our own launch.

Speaker 2

So it's very difficult to actually try to triangulate, I think, what might be a contribution on the ALL side versus the mantle cell side. I think it's reasonable to assume that the majority is probably coming from the mantle cell side. That sort of contributed to the somewhat reduced level of sales that were reported.

Speaker 7

And can you also comment on the median turnaround time so far in the manufacturing success rate?

Speaker 2

Yeah, so the turnaround time is very much in line of what we were targeting. So our target is to get to sixteen days. Remember our experience in the PHELIX trial was around twenty one days, and so we're tracking very nicely towards the sixteen. So that's, I think, kind of what we were hoping for, kind of where we are. And in terms of the success rate, that's something that I think is premature to talk about.

Speaker 2

Overall, I would say the fact that we have, or out of the gate the way that we actually got out of the gate indicates that we've been firing on all cylinders with good quality products.

Speaker 7

Okay, thank you.

Speaker 2

Thank you.

Operator

Thank you. Our next question one moment for our next question. Our next question comes from the line of Jill Bloom from Needham and Company.

Speaker 8

Good morning, everyone, and allow me to add my congratulations for a strong first quarter. So maybe addressing the same topic in a slightly different manner. Is it fair to assume that every single patient that was treated in the first quarter, would have otherwise received a competing CAR T, or do you think you're starting to see some levels of expanding on the potential patient pool?

Speaker 2

It is a really good question, Gil, and thanks for joining. It feels like, particularly you know, quite often when you launch a product, and depending on the experience of a center, if there's a center that doesn't have yet experience with your product, you typically get patients that are in pretty poor condition. And so there is a sense that there are probably patients in there that might not have been considered for a CAR T therapy before. So I think sort of on the worst end of the spectrum, I think we have certainly patients in there that were certainly part of that, really difficult to treat category. Whether we had, we're starting to see sort of patients on the upside, so with low tumor burden or early in relapse, I think that's too early to call.

Speaker 2

But I think we're clearly having patients that probably would have not been considered for CAR T therapy before in in some of the centers that we had seen in q one.

Speaker 8

Okay. And as it relates to your update that's upcoming for autoimmune disease, Just remind us what the data is gonna probably include here, you know, number of patients, follow-up Mhmm. And the increased dose as well.

Speaker 2

Right. So obviously, this point, we have six patients that are somewhere between one and eight months of follow-up. So all of these patients, these six will all be, I think, well beyond sort of the five to six month range, all of those. So I think that gives us sort of a good understanding of the behavior of the product from a longer term, and the recovery for the B cell compartment in those patients. In addition, what we're currently doing is we're dosing three patients at one hundred million cell dose, so that's twice of what we have used, in the first six.

Speaker 2

And we're planning to also treat, three adolescent patients at the fifty million dose level. And hopefully, we have, those patients included in the update by the end of the year.

Speaker 8

Great. Thanks for taking our questions.

Speaker 2

Thanks a lot, Gil.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Yanan Zhu from Wells Fargo.

Speaker 6

Great. Thanks taking our questions, and congrats on the very strong launch of TUCASL. Sorry, Elcaso. I was wondering, can you remind us of the, you know, of the TALKATA's current revenue? What percentage, by your estimate, could be coming from B ALL?

Speaker 6

It feels as though that, you know, just in the first quarter of launch, you have achieved very significant market share, in B ALL, given that, you know, Gilead's current sales is only 80 roughly 80,000,000, and it at peak, it was only a hundred million. And, yeah, give us a sense of the proportion of B ALL in there, the proportion of that that's B ALL. Thanks.

Speaker 2

Thanks, Yunnan. Very difficult question to answer, actually, because it's never been properly broken out. And with the dynamic we're seeing with Brienzi moving in, it's kind of hard to tell where that split is between the indications. When we look back probably a year, two years ago, it might have been somewhere between twenty percent and forty percent being ALL patients, but it's a hard thing to actually estimate, because there's not a real clear dataset that you could actually delineate it from. So the majority of what we would have expected to be mantle cell patients, a minority to be ALL, but how that has moved now with the change, of Breezy moving into the MCL side, very difficult to estimate.

Speaker 2

So I don't think we can give a good answer there.

Speaker 6

Got it. And then secondarily, you know, given the information that 4,700,000.0 is in a in a deferred revenue, line would be products that's delivered, but not yet infused. So two clarifying questions. One, so this 4,700,000.0 will represent the cost and not the actual, price that you will re receive from the product if that whether that's the correct impression. And two, typically, what is the, time between receipt, reception of the product and then, infusion?

Speaker 6

I I'd imagine it's a pretty short turnaround. And if that's the case, then this 4,700,000.0 could already be translating to 9,000,000. We're only early in the quarter. So just want to get a sense of those two questions. Thanks.

Speaker 2

So I'll answer the second question and then hand over to Rob for the first question. The time of dosing is quite variable in the commercial setting for these patients. So there is a with a lot of these patients, you have quite a lot of variability in terms of state, the condition that they're in. So some of these patients may have picked up an infection, and that was actually something you have to kind of go through. You have to treat.

Speaker 2

You have to get them back to a reasonable state before you dose. Other patients are in good shape, can be brought in, and can be dosed quickly. So it is actually quite a wide range, that we're seeing, and I don't think we have yet a good feel of where kind of the bulk of the patients will be landing on. But it is variable, based on sort of the underlying comorbidities of the patients, and frankly, the issues that they're encountering as a consequence of the advanced stage of their disease. So it is variable, and it's more variable than what we have seen during the clinical trial.

Speaker 2

There's more variability there. So I think as we sort of get more experience and probably have a few more quarters in, I think we start to probably have a better sense of kind of what the actual, numerical ranges will be. But at this point, clearly, it's somewhat larger than what we have observed, in the PHELIC study. With that, handing over to Rob for the first part of the question.

Speaker 3

Hi, Ynon. So, yeah, let me clarify on the deferred revenue piece. So the deferred revenue represents the sales value. It's not the cost of the product. So the way to think about product that was delivered to the treatment centers that got administered reflected in the P and L on the 9,000,000, the value of that product or the the the revenue value of that product, not yet recognized in the P and L, sits as deferred revenue.

Speaker 3

So that's kind of the the total piece to think about that. Now the the the piece is a little bit, maybe counterintuitive is when you do start to think about the cost for that, all of the cost for both the deferred revenue amount and patients as well as the P and L recognized amount do sit in our q one cost of sales line. And the reason for that largely is once we deliver product to the center, we no longer really can hold that as inventory and becomes kind of an immediate expense item once it's delivered. And so Christian, you know, alluded to there is some time and some variability around that. You also, for example, could have patients where we've delivered the product reflected in deferred revenue, and, unfortunately, a patient passes away.

Speaker 3

And and so that would actually still wind up, have been accounted for in your cost of sales, but would be deferred revenue that you may not realize.

Speaker 6

Got it. This is all super helpful. Thank you for Yep. The explanation. Yeah.

Speaker 6

If I may, one last question, perhaps for Christians. You know, can you give us a sense in terms of your positioning in, the competitive landscape for lupus nephritis with your planned dosing of first patient by year end twenty five. Where are you, vis a vis other companies who also is interested or are already taking on lupus nephritis, for autologous CAR T? Thank you.

Speaker 2

Thanks, Hidan. I think we're very well positioned. And the combination, obviously, is of having a very compact study design, as well as obviously being well set up in many of the clinical centers in The US, and we're obviously, are gearing up in The UK as well from a commercial perspective, I think should set us up well, in our ability to deliver the trial. So we believe we're very well positioned, with that. Many of the trials that have been in the lupus space more generally, that have been sort of considered or are underway are randomized controlled studies with substantially larger patient numbers.

Speaker 2

And those types of studies have been notoriously challenging to recruit. If you look at Benlysta, cefnello, etcetera, experience in the space, the patient numbers are one to two patients per cent per year, that were seen, and so conducting these larger studies will take, or can take, a good amount of time.

Speaker 6

Very helpful. Thanks, and congrats again on the quarter.

Speaker 2

Thanks a lot. Appreciate it.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Kelly Hsieh from Jefferies.

Speaker 9

Hi. This is Yifan from Jefferies. On behalf of Kelly, thank you for taking my question, and congratulations on a very strong Q1. I have a question on the lupus phase two trial. I'm wondering, like, how many patients you expected to enroll in the phase two?

Speaker 9

And more importantly, probably, like, what's your expected patient follow-up to like balance your strategy to like fast to market, and in the meantime, it can show the cell therapy's potential in induced, like durable drug free remission? Thank you.

Speaker 2

Well, thanks for joining. We had indicated at the R and D Day that we're expecting the study to be at around 30 patients. It's a relatively compact study, as indicated. And in terms of follow-up, when you look at other studies in the space, you do see that you have meaningful follow-up in these patients with a year follow-up. Also, can also follow these patients further, but you get a pretty good sense within a year from a value perspective.

Speaker 2

The actual readout that we expect to be able to go for could be shorter than that, given the time and dynamic that we've seen in terms of the response development in these patients. But I think one year is a reasonable period of time to consider.

Speaker 9

Thank you.

Operator

Thank you. This concludes today's Q and A session. I would now like to turn the conference back over to Christian Eiten, CEO, for closing remarks.

Speaker 2

Thanks a lot. Well, thanks, everybody, for joining. Fantastic to sort of give you the update. I know you're very keen to hear how we're doing for the first quarter. It was a great quarter.

Speaker 2

We're looking forward to keeping you updated, as we go through the rest of the year. But we're very excited about where we are, and we're going to push hard. Thank you very much.

Operator

This concludes today's conference call. Thank you for participating. You may now disconnect.

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Key Takeaways

  • Autolus recognized $9 million in first-quarter revenue from the US launch of Ocasel, activating 39 treatment centers and achieving coverage of approximately 90 % of US B-ALL patients.
  • The company plans to expand US access to ~60 centers by year-end and has secured conditional MHRA approval in the UK, with an EU decision for relapsed/refractory ALL expected in H2 2025.
  • In a Phase I CAR-T study for refractory systemic lupus erythematosus, all six dosed patients showed rapid symptom resolution and no high-grade CRS or neurotoxicities, supporting a planned pivotal lupus nephritis trial.
  • Key upcoming milestones include long-term follow-up data from the FELIC study, end-of-year pediatric ALL data, and first patients dosed in Phase II lupus nephritis and Phase I progressive multiple sclerosis trials.
  • Financially, Q1 operating loss was $65.2 million (net loss $70.2 million), R&D expenses of $26.7 million, SG&A of $29.5 million, and cash reserves of $516.6 million to fund ongoing launches and clinical programs.
A.I. generated. May contain errors.
Earnings Conference Call
Autolus Therapeutics Q1 2025
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