管理層發言
Good day, everyone, and welcome to Autolus Therapeutics' First Quarter 2026 Financial Results Conference Call. Please be advised that today's conference is being recorded. I would now like to turn the call over to the Executive Director of Investor Relations, Amanda Cray. Please go ahead.
Thank you, Carmen. Good morning or good afternoon, everyone, and thank you for joining us on today's call. With me are Chief Executive Officer, Dr. Christian Itin; and Chief Financial Officer, Rob Dolski. 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. These may include, but are not limited to, statements regarding status of the ongoing commercial launch of AUCATZYL in the U.S. and U.K.; Autolus manufacturing, sales and marketing plans for AUCATZYL; the market potential for AUCATZYL and the status of clinical trials, development and/or regulatory timelines and market opportunities for obe-cel. 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. On Slide 3, 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 will turn it over to Christian.
Thanks, Amanda, and welcome, everyone, to our first quarter update. Moving to Slide 4. We had a very good first quarter, and we see nice traction building both in the U.S. and in our early launch in the U.K. as well. In Q1, we had $26.2 million in revenue booked, and we do see a very nice penetration and deepening with the positive physician experience that was highlighted at the Tandem meeting as part of the ROCCA Consortium presentation, where we had approximately 60% of our commercial patients represented in that data set. We had discussed the data at the full year update, which was given a few weeks ago and was further analyzed and reviewed in the context of a KOL call we conducted in early April. When we look into the U.K., we see a very good start. We have more than 10 centers already active. We're going very strong in the U.K. with regards to not only center onboarding, but also starting to see early patients come in and actually be treated under the NHS access program. Now when we look into the 2026 overall outlook, we do see very strong continued momentum. From a U.S. center perspective, we're currently at around 73 centers, and we're increasing beyond 80 centers by the end of the year. Our year-end guidance is unchanged at $120 million to $135 million. For the first time this quarter, we did see a shift to positive gross margin, which is important because this is one of the key metrics that we're watching this year to demonstrate that the overall commercial base and our ability to drive proper cash flow into the company are increasing over time. This first-time positive gross margin was impacted, on the one hand, by an increase in volume, but also by operational improvements we made to the overall operation of the company, particularly the operation of the manufacturing plant that we're running. So this is where we are from a commercial perspective for AUCATZYL: very good dynamics, and we believe very strong momentum as we go into Q2. Moving to Slide 5. I would like to start on the right-hand side and briefly talk about the journey we're on to move the product to a place where we have a profitable ALL business. The foundation was laid during the first year of launch. What we had to establish during that first year is consistent and high-quality product supply and services so that we have a strong foundation, we serve our patients properly, and we can generate very strong outcomes. You've seen all of that presented as part of the ROCCA Consortium's experience, where almost all patients intended to be treated actually received treatment. We also saw that treatment translate into a very positive safety profile with no high-grade CRS, only 3% high-grade ICANS and more than 90% overall response rate in the patients. So a remarkable outcome in the hands of physicians in the real-world setting. We're also starting to see branching out of the patient pool into patients who are older, have more comorbidities and are typically considered difficult to treat. That's one expansion we've seen, and we're also seeing expansion into patients who have very limited tumor burden. Based on our prior studies, there is good opportunity to see a more pronounced benefit over time in those patients. So we've seen an expansion already in patient pools. That is very important because based on that behavior and the good safety profile, we expect that to form a strong foundation, and we are already seeing that translate in the first few months of 2026. The next stage, now that we have established consistent high-quality product supply and services, is to switch gears and drive optimization, improve efficiency and at the same time increase volumes through the operation to get our overall cost down for the products. We expect the peak ALL business to achieve around 65% to 70% gross profit margin, which would be a very healthy business for the ALL side of AUCATZYL. The optimization we ran was not only in processes but also included a reduction in force at the company of 13%. At the same time, we have driven up operational efficiency. One clear metric is that we expect to produce twice as much product this year compared to last year with overall staffing at or below the levels we had last year. That gives us a clear understanding of why gross margins are moving positive and will continue to improve through the year. Once the one-time effect of the restructuring is taken care of, and Rob will speak to that briefly, we expect for 2027 a net impact of savings of $15 million on an annualized basis. On the development side, leveraging the exceptional profile we see in clinical trials and in the real-world setting, we're building into a range of additional indications. The first is to extend treatment from the adult population into the pediatric population. The Phase II expansion of the CATULUS study is ongoing. Data is expected toward the end of 2027, and we have aligned with the FDA on the protocol design and support for potential registration. The CARLYSLE study is progressing well; we expect additional data toward the end of this year where we've added additional patients and will have substantially more follow-up in that patient population, which will give us a better understanding of the type of benefit CARLYSLE or obe-cel can induce beyond oncology, in this case in a refractory form of systemic lupus. The LUMINA study, which is in lupus nephritis, is actively enrolling. This Phase II study has agreement with the agency to aim for a registration in refractory lupus nephritis. The study is active in certain countries in Europe and the U.S., and we expect data in 2028. Finally, the BOBCAT progressive multiple sclerosis study is continuing to enroll. We expect initial data at the end of this year and then full data during the course of next year. More broadly, the ROCCA Consortium is continuing to collect real-world information from patients treated with commercial product and is looking at a range of questions related to product performance and properties. Also, as discussed on our KOL call, we're seeing investigator-sponsored studies looking at obe-cel in frontline consolidation settings to explore whether an abbreviated frontline treatment might be possible, and we expect investigators to report at upcoming conferences. With that, I will hand over to Rob for the financial results.
Thanks, Christian, and good morning or good afternoon to everyone. It's my pleasure to review our financial results for the first quarter of 2026, and I'll be referring to Slide 8 in the presentation. Total net product revenue for the first quarter of 2026 was $26.2 million compared with $9 million in the first quarter of 2025. This quarter reflects sales in the U.S. and our first quarter in the U.K. market. Given it is early in the launch, contribution from the U.K. was minimal. As Christian noted, we're pleased to shift to a positive gross margin during the quarter with $1.6 million compared to losses in prior quarters in 2025. With the combination of increasing product revenue and our cost reduction initiatives that Christian mentioned, we expect margin expansion to continue. Underlying that gross margin, cost of sales in the first quarter totaled $24.6 million compared to $18 million for the same period in 2025. The increase primarily reflects costs related to higher AUCATZYL sales period-over-period. Our research and development expenses decreased to $21.2 million for the first quarter of 2026 from $26.7 million during the same period in 2025. This change was primarily due to a decrease in development activities, including clinical trial and clinical manufacturing supply costs as well as capacity mobilization costs, mostly related to obe-cel. Our selling, general and administrative expenses increased to $39.9 million for the first quarter of 2026 compared to $29.5 million in the same period of 2025. This increase was primarily due to salaries, other employment-related costs and professional fees supporting commercialization activities in both the U.S. and U.K. In addition, this quarter also included one-time termination-related expenses related to the operational efficiency and cost reduction initiatives we announced last month in April. Loss from operations for the three months ending March 31, 2026, was $59.5 million as compared to $65.2 million for the same period in 2025. Net loss was $71.6 million for the three months ended March 31, 2026, compared to $70.2 million for the same period in 2025. Our cash, cash equivalents and marketable securities at March 31, 2026, totaled $229.4 million as compared to $300.7 million at December 31, 2025. This decrease was primarily driven by net cash used in our operating activities. As Christian noted, we are reiterating financial guidance issued in January that we expect between $120 million and $135 million in AUCATZYL net product revenue in 2026, including contribution from both the U.S. and U.K. markets. Finally, based on our current operating plans, including the anticipated AUCATZYL net revenues, we expect that current and projected cash, cash equivalents and marketable securities will be sufficient to fund our operations into Q4 2027. I'll now hand back to Christian to wrap up with a brief outlook on expected milestones.
Thanks, Rob. So when we look toward the end of this year, we have updates from three of our programs. We have the longer-term follow-up from the CARLYSLE study. We have initial clinical data from the BOBCAT Phase I in progressive MS. And we expect to have initial clinical data from the ALARIC Phase I study, the AUTO8 trial in light chain amyloidosis, which we're collaborating on with UCL, also by year-end of this year. Full data is then expected for the BOBCAT study during the course of 2027. The pediatric Phase II data readout is expected by year-end 2027 and the LUMINA Phase II data in lupus nephritis is expected in 2028. Those are the key updates and key milestones. With that, we're opening up to questions.
分析師問答
One moment for our first question, it comes from Gil Blum with Needham & Company.
Congrats on the progress. So you're saying you're seeing some level of market expansion, older patients, maybe patients for consolidation, that sort of thing. It didn't seem to change the guidance for this year. So I'd appreciate your commentary on that. And then I have a follow-up.
Yes. First of all, thanks a lot for joining, Gil. The dynamic we're seeing in the market is very positive. But obviously, we're still early in the year. I want to see the dynamic play out into a fuller aspect as we go through the course of the year. The guidance at this point we believe is reasonable, and there's honestly no reason for us to change the guidance at this point. We'll keep monitoring the development and, if we see a different trajectory, we will update the market.
Very helpful. As it relates to the LUMINA study, we just saw another registrational study come up, again, in lupus nephritis in a similar line of therapy. Are you guys seeing any potential challenges in enrollment? I mean, is it a little crowded?
Thanks, Gil. The population we're going after is a refractory population that is post the standard of care, which is based on B-cell depleting antibodies as well as calcineurin inhibitors. It's a pool that's currently not served by the standard of care beyond that. The trial size we need to recruit is very limited. We're looking at a 30-patient trial. Given that and the fact the study is running both in the U.S. and outside the U.S., we believe we're in a good position to enroll the study. So we're not worried about that.
Excellent. And maybe a last one on multiple sclerosis. Can you remind us what level of data you think you may have in 2026?
The BOBCAT study is a dose escalation study. We're starting at 100 million cells with the ability to go higher. That's the basic design. We expect to have initial information on the behavior of the product with regards to safety and the cellular dynamics that we're seeing, which is important. You want to understand the ability of the product to expand in the periphery and also look at the presence of the product in the CSF on the other side of the blood-brain barrier. We'll record markers that monitor inflammation and potential tissue damage both with biochemical markers and imaging. So we'll have early data in terms of behavior and safety, pharmacodynamic and pharmacokinetic markers this year. As we look into next year, we'll have more data points that assess disease scores and any potential impact on disease scores. For those measures, you want several data points and a longer observation period to ensure you are not observing placebo effects or other influences, since some assessments are physician- or patient-reported. So the hard measures this year are mostly on product behavior and safety, with disease score impacts becoming clearer next year.
Our next question comes from Simon Baker with Rothschild & Company Redburn.
Two, if I may, please. Christian, I just wonder if you could give us a little more detail on the mechanics of the U.K. rollout. I believe there are 20 CAR-T centers that, in principle, could administer AUCATZYL. What's the cadence of rolling those out in terms of getting centers on board? How does this compare with the experience in the U.S.? Any color would be helpful. And then on the R&D expense, it was lower than expected in the quarter because of clinical trial activity. I just wonder if you could give us any more color on the evolution of R&D spend as the year goes on.
Thank you. With regards to the U.K. rollout, we see a dynamic at least as quick as the initial U.S. phase and it might actually be quicker. One positive aspect of the U.K. system is that the decision whether a patient receives a CAR-T therapy is centrally made, which accelerates decision-making. That's a benefit compared with individual center- or physician-level decision-making typical of other health care systems. Elements of the U.K. organization allow us to move even faster than in the U.S. We see very positive dynamics and messaging from the NHS, including public awareness activities that have increased recognition of the product. So from an awareness perspective and because the initial decision-making goes through a central body, we expect a good rollout in the U.K. With regards to R&D expense, I'll start and then hand over to Rob. R&D expense can fluctuate as activity transitions between trial phases. When you move from one phase to another, enrollment and activity can slow temporarily. Last year there was substantial work related to long-term follow-up of the FELIX study, which is now winding down, and those activities will be reduced over time as we ramp other clinical activities. Another area was mobilization of additional capacity at The Nucleus to ramp U.S. demand; those mobilization costs are substantially reduced or coming to an end. So there are trade-offs as clinical trial costs increase in some areas and decrease in others. Overall, we expect to keep R&D at a fairly steady level. Rob, do you want to add anything?
Yes. Christian covered it really well. Another example is our pediatric study, where we had the initial cohort enrolled and active at sites, and that contributed to data presented at the end of last year. We're now in a bit of a valley while we enroll the expansion cohort. As we start treating more patients in clinical programs and investigator-sponsored trials Christian mentioned, you'll see more clinical supply-related costs come in. This will move up and down a bit. We're not projecting any significant infrastructure additions on the R&D side.
One moment for our next question that comes from Salim Syed with Mizuho.
Congrats on the quarter. Just one for us on the data we're expecting this year. Christian and Rob, it sounds like for MS, the more important data set is going to be coming in 2027. I don't know if you interpret the same for ALARIC. I'm trying to balance how important you deem these data sets coming at year-end. Are we expected to get any sort of bogey or how you deem what is the successful outcome on these year-end measures? I'm trying to balance that with the cash runway into Q4 '27.
When we look at the MS study, we collect disease score data but need a certain observation period to understand trajectory. We'll have an initial view at year-end, but a stronger statement requires longer follow-up to ensure stability of the data. For ALARIC, we expect an initial good understanding of the level of activity in this disease setting; we haven't reported on that before, so that will give us helpful interpretability. For the MS program, the immediate importance is to demonstrate safety in these patients and show that the product is active in the CSF and central nervous system. That is key to the rationale for a cell-based product because it supports the ability to cross the blood-brain barrier and have activity. As a reminder from our KOL call, Lori Muffly presented a patient with extensive leukemia intruding into the brain and CSF who had paraplegia. She treated the patient with AUCATZYL, which she felt was the only safe option, and within 28 days the patient had MRD-negative disease in the periphery, clearance in the CNS and recovered mobility. That demonstrates the product's ability to cross the blood-brain barrier and clear CD19-positive cells in the brain. That's a foundational feature we're building on for BOBCAT in progressive MS. So year-end data for MS will be insightful on safety and central activity, while more robust efficacy readouts will come with longer follow-up into 2027. Regarding cash runway, we are managing priorities and will continue to assess based on data and business development opportunities.
One moment for our next question. It comes from the line of James Shin with Deutsche Bank.
First, I appreciate AUCATZYL achieving positive gross margin this quarter. Pending top-line growth and with the RIF in place, could we see Autolus becoming profitable on a company level basis by, say, late '27? Second, between Besponsa and Blincyto, is Blincyto the main bridge before patients receive AUCATZYL? Does the bridging tend to align with AUCATZYL's manufacturing turnaround time?
Thanks, James. On the trajectory for the ALL business, we expect to cross the line to profitability in the ALL business in 2028. Whether the company as a whole will be profitable by that time depends on our reinvestment rate over the next 1.5 years. That's the key driver for company-level profitability. Regarding bridging therapies, typical bridging is chemotherapy or inotuzumab (Besponsa). We do not generally use Blincyto for bridging with our product because Blincyto is a continuous IV infusion over four weeks, which is longer than the manufacturing turnaround time for our product and therefore doesn’t line up well. So bridging is mostly driven by chemotherapy and/or inotuzumab.
And we continue to the next question, it comes from Matthew Phipps with William Blair.
Could you characterize the growth you saw this quarter? Is it greater utilization at current centers? You mentioned expansion in older patients—was it mainly expansion into new centers? What percent of the market do you think you'll cover when you reach 80 centers by the end of the year?
Thanks, Matt. When we look at growth, given we had 73 centers compared with about 65 at the beginning of the year, the majority of growth comes from centers already active. That's where you see repeat use and build-up. New centers gradually build activity, but initial activity is limited—often one or two patients to gain initial experience, which then translates into more activity later. One key metric we track is not just active centers but the number of physicians within a center who are using the product. Initially, a small number of physicians at a center might use it; over time you want broader physician adoption within the center. We see nice progression in the number of physicians dosing patients. That expansion is primarily within centers we're already active in, and we're starting to see broader adoption, which is how you ultimately build market share. Overall, we see very good dynamics increasing market share based on our guidance for 2026.
One moment for our next question please, it comes from Emily Bodnar with H.C. Wainwright.
A follow-up on the prior question. Can you comment on what contribution U.K. revenue had to growth in the first quarter and how you're expecting that to play out for the remainder of 2026? On the data update for SLE and LN later this year, what level of durability are you hoping to see in the updated data and what response rates for the 100 million dose?
For U.K. sales, because the U.K. is a fraction of the size of the U.S., we decided not to break out U.K. sales numbers initially. It's too early in the process, and we expect to break it out likely toward the end of the year. For the first quarter, it doesn't make much sense given early product flow and patient roll-in. Regarding the LN update, the initial cohort will have about 12 months or more of follow-up, and that will be meaningful because it tells us not just about initial safety and response but also about how the B-cell compartment reconstitutes over time and the ability to see sustained response in these patients. For the other patients, which include a higher dose cohort and adolescent patients, follow-up will be more limited, so those readouts will focus on initial response, safety and initial recovery of the B-cell compartment. The first cohort will have longer follow-up beyond one year.
One moment for our next question that comes from Jacob Mekhael with KBC Securities.
First, on the gross margin: you mentioned 65% to 70% at peak. Do you plan to provide peak sales guidance in the future? When would be the right time to do that? Second, regarding your agreement with Cellares for manufacturing: is there a timeline by which you need a formal agreement in order to incorporate that into manufacturing for a pivotal autoimmune program?
Thanks, Jacob. On peak sales and gross margin: for the relapsed/refractory setting in the U.S., we estimate roughly 1,600 to 1,800 patients. With a good profile, we've seen other agents reach about 60% penetration in that setting, which is the upper end of what we expect for market share. To provide formal peak sales guidance now would be premature, but that gives a sense of market size and opportunity. Regarding Cellares, we're in the midst of a feasibility study to understand how the product produced on their platform compares with the platform we use at The Nucleus today. That's the key analysis we are running this year. If feasibility data is positive, that would be the time to take next steps. If we need to scale production substantially—potentially for an indication like progressive MS—having options to scale economically is important. So this year is focused on the feasibility and assessing comparability of product quality on the Cellares platform.
Our next question comes from Roger Song with Jefferies.
This is Fiona on for Roger. A couple questions. On AUCATZYL revenue, how should we think about near-term and long-term growth drivers? Any active measures you're taking to further penetration in the U.S. versus ex-U.S. dynamics? Second, understanding you switched to positive gross margin this quarter, do you see further optimization needed going forward, or do you want to see the impact of the cost reduction play out?
Thanks, Fiona. On revenue drivers, there are two key elements. One is coverage—getting centers enabled to treat the patients we can serve. At 80-plus centers we believe we reach a high percentage of patients we can cover. The primary driver of market share growth is physician adoption within each center: physicians gaining experience and confidence to use the product more widely across the label. We've seen this play out in clinical trials and now in the marketplace; physicians who see a safe and effective product will apply it to patients they previously could not treat. That physician conviction drives growth across jurisdictions. On ex-U.S. expansion, we're active in the U.K. and continue conversations in Europe, navigating market access and MFN topics. We're not guiding on European sales at this point; those discussions are ongoing and somewhat sensitive to policy changes. On optimization, manufacturing optimization is continuous. We've implemented significant operating model changes already and will continue to improve every step of the manufacturing process. Our focus is minimizing work hours per product and reducing fixed cost allocation through higher volumes. Those two metrics—lower hours per product and higher throughput—drive cost per product down. We'll see a steady impact over the next two years.
And our last question comes from Shyam Kotadia with Goldman Sachs.
Two questions. First, gross margin reached positive this quarter—how should we think about cadence for the rest of this year? Should we expect steady improvement? What is your target gross margin for full year 2026? And when are you expecting to reach that peak margin? Second, on multiple sclerosis commercialization: are you planning to commercialize that yourself or would you look to partner?
On gross margin, we expect continuous improvement as we move through the year. Significant operating improvements implemented in the first months will start to impact cost for the remainder of the year, and we expect volume increases to be supportive as well. We haven't given a specific year-end gross margin target but will report metrics quarterly so you can track the dynamic. Reaching peak sales in indications like ALL typically takes four to five years. From a gross margin perspective, much of the improvement comes earlier once you achieve sufficient volume to reduce fixed-cost allocation and improve productivity. The biggest gains in labor-hour reductions are expected this year, and smaller, discrete technology improvements over time could add step changes, though they take longer to implement. On MS commercialization, the MS patient population is much larger and more distributed than ALL. Patients are highly motivated, and the sheer volume suggests it may make sense to consider a commercial approach in collaboration with a partner. That's one of the areas we're exploring.
Thank you. And this concludes our Q&A session for today.
Very good.
Thank you so much.
Thanks, everybody, and have a great...