Immix Biopharma, Inc. (IMMX) Earnings Call Transcript & Summary

Hello, everyone. Thank you for joining us, and welcome to the Immix Biopharma Inc. clinical data release. After today's prepared remarks, we will host a question-and-answer session. [Operator Instructions] I will now hand the conference over to Dr. Ilya Rachman, Chief Executive Officer. Please go ahead.

Thank you, Hilary, and thank you, everyone, for joining us today. We're happy to share our clinical and commercial updates today.

I'll be joined today by my colleague, Gabriel Morris, President and Chief Financial Officer; as well as Mike Grabo, Chief Commercial Officer. Here's the agenda for this morning's call. We will begin with the clinical update followed by a commercial update, and then we'll proceed to question-and-answer session. We will be updating folks today on the progress we made in a disease of light chain amyloidosis is a disease where normally protective plasma cells residing in all of our bone marrows, produce protective antibodies to protect from flus, cold, emerging cancers and other insults. In this disease, instead, they begin to produce toxic antibody fragrance called light chains. These light chains circulate throughout the body and they go up and destroy every vital organ starting with the heart, kidney, liver, nerves and others. And unfortunately, patients progress to organ failures subsequently leading to death. Here's an example of a heart that was so destroyed by these light chains, a patient had to undergo a hard transplant to cope with it. Because it's the light chains, the cause of this organ damage in this disease, clinically, we follow and monitor the levels of wide chains in patient blood. If the light chains stay persistently elevated, we know that patients are on the path to organ failure. If we're able to remove these light chains from patients' blood, we know that they will going to remission and live completely different lives. So in the next handful of slides, we will share with you the current standard of, care the progress we're making in this disease, and we'll take it to the commercial outlook as well. So if you were to look at the current lay of the land in 2026, he is a recent report from amyloidosis center of axles in the United States. It's a 12-patient report that shows that out of 12 patients in 11, the disease was not able to be controlled. Just 1 patient was able to be put in a complete remission. So in 2026, not only there are no FDA approvals in the relapsed/refractory space, the complete response frequently is less than 10%. With a single drug approval only in the newly diagnosed setting, this remains a very poorly served patient population. In addition, given that 50% will not respond to the frontline therapy. And over 1/3 of patients will progress. There's a significant unmet need in this setting. So Gabriel, perhaps, we can provide folks with the update on the recently announced data.

Thank you, Dr. Akman. I'm Gabriel Morris, President of mix -- we have in the same format on the next page, so disease burden on the top and magnitude of response on the bottom, our data. We are thrilled to report that at this moment, we have our entire 45 patients, of which 44 have today normalized their toxic light chains. And we have 95% downstream organ responses as well, which provides us with a 89% complete response rate, which we believe will increase to 98% after these 4 patients that would be 44 out of 45 have converted to complete response as folks may know from our prior disclosures, we have had patients who have -- all patients who have been minimum residual disease negative in the past have converted to CR with subsequent follow-up. And the company is thrilled with these results. For the new 25 patients that we are providing data on today, all are either in complete response or minimum residual disease negative. And we believe that at this time, this data not only provides hope to folks, but shows consistency among what we've disclosed before. With that, I'll hand off to Dr. Rachman to talk about safety organ responses that we will touch on the landscape briefly and where we are as we move towards a planned BLA next year. Dr. Rachman, anything to add on efficacy before we move to safety?

No, absolutely. Absolutely. Given the magnitude and duration for the efficacy and the complete responses, folks crossing 2 a number of folks across the 2-year mark. It remains extremely gratified to see this. And obviously, as folks would like to know and it is important to focus on the safety results as well, given this is CAR T and what we'd like to highlight is the consistency of the data set across all patients now more than doubled from our prior data release and it remains that the median duration of CRS across all patients is still -- all patients is still 1 day. There is no great -- no high-grade CRS observed no neurotoxicity of any kind, no enterocolitis observed and remains mild and well-tolerated therapy across all patients. In addition, taking a deeper look at the organ responses, folks can see that there's a rapid and sustained non-transient deep organ responses, both cardiac in renal, 100% cardiac, 92% renal and the effects are observed within days. So you can see the cardiac responses were absorbed in 26 days with the renal following in the next 59 days post dosing. So there's a predictable sequence that we observe the responses in. Post dosing at the end of the month on patients turn MRD-negative, minimal residual disease negative, which means that there is no plasma cell clone that is the producer of these toxic light chain, followed by complete responses and organ responses. And that is what we'd like to see in the clinic. Further, to build on the unique tolerability profile of NX1, we'd like to highlight the unique advantage versus other CAR Ts, for example, that advantage that provides the foundation for the leadership of CET1 in light chain amyloidosis. The median duration of CR has been 1 day that is 3 to 7x shorter than any other CAR-T both approved or in development. And the reason why that is critically important is because patients with light chain amyloidosis present a unique management clinical challenge. They frequently have coexisting heart and kidney failure present in the same patient. So CRS being a rapid hard beat, i.e., hot stress coupled with fever, which is dehydration and kidney stress presents sometimes an untenable management challenge, where for heart failure, you have to deuterated the patient and for kidney file, you have to hydrate the patient. So we believe that the unique tolerability of XE201 provides the opportunity to serve this patient population uniquely and better likely than any other CAR-T. and we'd like to expand our leadership now and put it in perspective when it comes to bispecifics as well.

Thank you, Dr. Rachman. We believe that the data that we have presented compares favorably -- we can see with other classes of therapies that are not in XC 201, we do see in data sets presented at European Hematology Association Conference, toxicity and infection challenges across the entire set as well as I can. Three points. One, we are advanced in our registrational design trial. Point number two, we believe our clinical is great. We have more prior lines of therapy prior to NXC201 than other trials, we have more stem cell transplants. Notably, we have an independent review committee assessing complete responses. We have fast and deep organ responses. And we are able to do have this effect in 24 hours, not 24 months. Therefore, for patients that are coming off of a 4 drug combo in the frontline with monthly dosing where there are side effects for 2 years, which is the recommended dosing for the frontline therapy, DARZALEX, 4-drug combo with cyclophosphamide, as you all know, steroid and protease inhibitor, we believe that a onetime therapy option, 24 hours with, as Dr. Rachman touched on, extended responses now going beyond 2 years will be a compelling option for these patients when other options remain extended dosing and time near the hospital and very careful close management. Dr. Rachman, anything to add here before we move to a time line and then back to you to wrap up clinical section.

Thank you, Gabriel. I believe this slide is truly -- this slide truly highlights the unique offering and uniquely suitable opportunity to provide one-and-done approach that would offer patients potentially transformatively effective, convenient treatment option.

Okay. Thank you, Dr. Rachman. So as we proceed -- we're happy to have everyone on the line today. We have completed a 45-patient readout here. In September, we believe the results, current results are in 40 CRs, as we sit here today, if we can incorporate MRD negativity, we're going to 44 out of 45. And that will take us to 98%, we believe, a complete response rate as we move toward a BLA. We plan to lock the database with follow-up from our completion of enrollment in March 2026. And folks can also look forward to trial initiation of our randomized controlled trial against daratumumab CyBorD coming as well with breakthrough therapy designation as well as RMAT designation, we could not be more thrilled with the consistency that we're showing here. We believe that for patients who have had 1 drug approval and 100 years and that 4-drug combo in the frontline contains some medicines that have been around for a while. We believe it's time for a possible second option when folks look at the -- on the shelf for the array of options in this disease, rare disease, progressive disease. Back to Dr. Rachman to wrap the clinical section, and then we will hand off to our Chief Commercial Officer, Mike Grab.

Look, for the disease that affects currently over 30,000 patients as Gabriel mentioned, the innovation is long overdue. And we are cautiously optimistic that better answers are just around the corner. We further would like to highlight with the next slide on the increased awareness, improved diagnosis in speedier diagnosis of light chain amyloidosis, in part driven by greater awareness of other types of amyloidosis, including ATTR. So with recent availability of transformatively more effective therapies for ATTR, there's been increased push to earlier diagnosis and awareness. And I want to point out that the diagnosed TTR folks have to rule out AL amyloidosis. So the diagnosis of -- the AL amyloidosis have actually parallel the increased awareness and diagnosis rates of ATTR as ATTR remains a diagnosis of exclusion. So we believe that this further highlights the upcoming increase in an ongoing increase in recognition, diagnosis rates and the improved awareness. So patients could get into treatment sooner without incurring the further heavier burden of organ dysfunction. Further go ahead. Please go ahead and give the you look at.

Also, to build on what Dr. Rachman mentioned, we'll add that -- we believe that AL remains underdiagnosed a recent study with Optum health records reported at ASH showed a tripling of the prevalence of this disease between 2017 and 2021 as well as an increase in incidents since 2019 of 8% per year. So as Dr. Ragan mentioned, this driver as folks present with heart failure symptoms, then ruling out with a simple blood test first AL is the ATTR diagnosis path. So we believe that, that awareness with additional approved drugs in TTR as well as general test volume of the simple blood test will continue to drive awareness and additional instance and prevalence in this disease deadly, deadly disease.

Okay. Dr. Have 1 more slide. Anything to add on ATTR and then -- or are we good? No, thank you for adding these additional details. I think this provides a great perspective. And in large part, the sort of gross parables where increased awareness really drives clinical benefits in earlier diagnosis. And next, we'd like to share with folks the nationwide footprint of clinical trial sites that participated in our trial. We will obviously continue to build on this as we expand further in anticipation of our frontline trial and subsequent commercial launch that will obviously provide the basis for all these activities. And as we mentioned, amyloidosis remains an underdiagnosed indication that we believe that's going to continue to grow. It's a large indication. We estimate that currently provides a runway of over $2 billion of sales in the front line alone. And now, I'm thrilled to introduce and hand off to our Chief Commercial Officer. Michael Grabo.

Thank you, Dr. Rachman. As this is my first investor call with Immix, I'd like to provide some highlights of my macround. So I have over 26 years of experience in the biopharma industry, half with large pharma at Amgen and half in emerging biotech with a focus in rare disease. In this time, I have played a primary role in more than 10 commercial launches, most recently with Modco, a treatment for a rare form of brain cancer initially leading commercial readiness with Cimarex and ultimately with Jazz Pharmaceuticals post acquisition. With that, I'm really excited to now be a part of the mix leadership team. Today represents an important milestone for mix in the development of NXC201. We're really encouraged by this data as it demonstrates initiates with relapsed/refractory AL amyloidosis. As we continue to develop this important program in March towards our anticipated BLA filing and potential FDA approval, I want to share with you a glimpse into our commercial strategy and plans. To start, we've been very diligent in our approach to understanding the degree of unmet medical need and potential receptivity to NXC201 in the relapsed/refractory AL amyloidosis space. Consistent with my past experience to help us gauge our commercial opportunity with NXC201, we've completed an opportunity assessment with an industry-leading consulting firm. That assessment included a robust example of hematologist/oncologist with experience treating relapsed/refractory AL amyloidosis. As you can see from this slide, our market research is demonstrating that not only does a significant unmet medical need exists, which you can see from the graphic on the left side, that in sharing our product profile using the data released at ASH 2025, the majority of respondents indicated a strong likelihood to prescribe, which you can see on the right. This gives us confidence that a clear commercial opportunity exists with a benefit to patients suffering with relapsed/refractory AL amyloidosis today. With regards to our commercial strategy in this rare disease, we're planning to build a commercial organization that is fit for purpose agile and scalable for future opportunities. To start, we're going to be highly focused on where the concentration of patients are today. As you can see from the slide, in the evaluation of claims data, there are approximately 2,800 hematology oncologists who represent 90% of all AL amyloidosis claims. Importantly, the majority of these treaters are connected to fact accredited institutions with hands-on experience administering CAR-T therapies, at which we are going to initially look to onboard about 60 to 70 treatment centers strategically located across the country to ensure patients have access upon launch. This high concentration suggests to us that we can launch with a lean and efficient team, including 35 to 45 CAR-T account managers with the appropriate mix of personal and nonpersonal promotion in line with other rare disease launches. From my experience in launching multiple rare disease brands, I've learned that to be successful in rare disease, it takes a team built around dedication, agility and discipline. Today, I've already begun building out the commercial leadership team with the right experience and mindset who fully embody these principles. Collectively, my team and I fully understand the responsibility that we have to patients suffering with relapsed/refractory AL amyloidosis, and we're ready to bring NXC201 to as many appropriate patients as possible upon our FDA approval. I look forward to sharing more updates with you as we approach our BLA filing and potential approval. With that, I'd like to turn the call over to our operator.

[Operator Instructions] Your first question comes from the line of Judah Frommer from Morgan Stanley.

Congrats on the update. Just a couple from us. Maybe first, just remind us that all MRD-negative patients historically convert to CR and what's the median follow-up for those 4 current MRD-negative patients that are not in CR yet? And then just maybe on the commercial side, remind us what will patients need to be in the hospital overnight, -- could this potentially be outpatient at some point? And does the DARZALEX patent export in the coming years affect the market in any way in your view.

I'll happy to take this question. Thank you, Julia. Well, this question. So yes, historically, every patient who reached MRD negativity status, proceeded to become a complete response. Media got response time point is approximately 2 months after MRD negativity, but it could take up to 320 days to reach CR -- with respect to tolerability and the outpatient setting. At present, you're exactly correct. Given the tolerability profile and the short CRS duration, we anticipate not needing more than a day or 2 follow-up post administration. But not unlike CD19 CAR-Ts that are currently being administered on an outpatient basis enough to 50% of cases. We believe that an outpatient transition is only a matter of time. as clinicians gain familiarity with this therapy. With anyone on my team might add any comments.

The MRD-negative patients that have not yet flipped to CR have less than 6 months of follow up those 4 Juda, we believe, as we've had 7 patients in the past that did flip from MRD to CR, we believe that they will flip prior to BLA submission.

Your next question.

I guess we'll be moving on to the next question. Jude, do you have a follow-up or how is it working?

Judah, I believe that I heard you say thank you. If you could please let us know if you have any follow-up questions.

We just had DARZALEX patent expiry.

Yes, please go ahead.

So the DARZALEX Fast Pro patent, and again, we're not representatives of other companies, but has a handful of years to go is our understanding.

Your next question comes from the line of Ted from BofA.

Congratulations on a really strong data. So I just want to have a couple of questions clarified. Can you share anything on organ responses that you're seeing so far? And then secondly, just on patient background and baseline characteristics. For the 25 new patients that you've shown today, how does that compare to the previous 20 patients -- and then lastly, data that you presented this morning, a top line is quite impressive with the 89% CR in all 45 patients and 84% in the new 25 patients. So given the quality of the data that you have so far, is there a way that potentially your time line for application and approval could be moved forward I know that you've talked about waiting for the full data set, but given that this is an area of unmet need, I'm just curious how you're thinking about it today if you have any updated thoughts. So those 3 questions.

Thank you, Tazeen. Good to hear from you today. Thank you for joining. So to your 3 questions. Number one, I would expect to the organ responses since so far, you're right. So we've demonstrated 100% cardiac or response and 92% kidney organ responses. The latest additional patients dose, share the baseline characteristics with the first 20 patients. So consistency is the theme. And to your last question, with respect to BLA timing. Obviously, like everyone understands the urgency for this patient population, and we intend to move as expeditiously forward as possible. We did set out to provide the adequate follow-up period post dosing. But stay tuned and rest assured that we will do everything possible to bring this to patients as quickly as possible.

And to build on Dr. Rachman's comment, we have shown in today's presentation to your first question, sustained and deepening organ responses over time that deepen all the way out to month -- 12-month 1. And we plan on timing of the submission after sufficient follow-up to have complete response rate in its steady state. So myself, Belia, as investors in the company, we believe it makes sense to get that 1-year follow-up for submission. Does that answer your question? Any follow-ups?