Immix Biopharma, Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference
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Ilya and Gabriel, thanks again for being here. I thought we could start with a couple of minutes of background on the company and your lead asset, maybe a bit on company formation and where the lead asset came from.
Jud, great to be with you. Thank you for the invitation. Thank you all for joining us today. Immix Biopharma was founded on a simple principle, that we could not accept the fact that the standards of care meant months of suffering with no hope in sight. Gabriel and I asked ourselves the question, can a groundbreaking modality like CAR T be applied outside of cancer? We conducted a global search, and we came across a CAR T that behaved anything but CAR T. With the mild benign tolerability profile, we licensed this technology out of Hadassah Medical Center, and a few years later, we are on the precipice of submitting a BLA in the relapsed refractory AL amyloidosis, where there are no medications that are approved today.
Yeah. Very exciting. Maybe a little bit more about this structure of your CAR T, NXC-201, how that leads to clinical differentiation from other BCMA-directed CAR Ts.
Sure. Our scientist team hailed back from, I guess, one of the cradles of CAR T at NCI, at Dr. Rosenberg's lab. Over the ensuing 20-plus years, they were able to incorporate all the learnings that have been derived from this field and incorporate construct elements- that produces distinguishing benign tolerability profile, including CD3 domain, stiffer CD8 hinge region, as well as a proprietary binder domain that combined together into biological filter-like effect that we see in the clinic today, which reduces the non-specific CRS, non-specific activation, and produces the vastly differentiated safety and tolerability profile.
Another nuance I want to point out in this case is the unique clinical profile of AL amyloidosis patients, light chain amyloidosis patients. They actually are unfortunate enough to have a combination of heart and kidney failure present in the same patient.
which presents quite a significant clinical challenge in terms of managing. That is where the CRS duration really comes into play. Our CAR T having a single day, 1-day CRS duration.
significantly reduces the clinical challenge of managing CRS, which is cytokine release syndrome, which is protracted rapid heartbeat, i.e., heart stress, combined with fever, which is dehydration. You have to really manage these patients in an opposite way. For heart failure, you want to take away water, which you can't do because they're getting dehydrated.
Right but you can't hydrate the patient to flush the kidney either.
That is why traditionally it has been challenging to apply this modality to these patients. So having 4-8 times longer CRS durations of hours.
Right has limited the adoption of this modality.
I guess one of the biggest questions we get is the risk of already commercialized CAR T's competing in AL amyloidosis. I guess, what can you tell the audience to allay fears that that's coming down the pipe?
Yeah. Despite the breakthrough promise of this technology, it has not made its way into amyloidosis for that very reason, for the duration of CRS that is intrinsic to both approved CAR T's, which know AL amyloidosis very well, as well as CAR T's in development. We really lead on the very short and mild CRS.
Okay, great. Then maybe just briefly describe AL amyloidosis, not an indication that everybody's necessarily familiar with, a bit of the epidemiology and the symptomatology, standard of care, unmet need. I think whatever you think would be helpful for background here.
Sure. I'll start with a brief background. AL amyloidosis, light chain amyloidosis, is a devastating rare disease where a patient's own immune system turns against the patient. Normally, plasma cells that reside in our bone marrows produce protective immune molecules called antibodies, which protect us from viruses, bacteria, cancers, et cetera. In this case, plasma cells turn on and they stay on all the time, and they keep producing these toxic antibody fragments, light chains, which circulate all over the body, gum up every vital organ, starting with the heart, kidney, liver, nerves, and others, and patients go into organ failures and pass away from them, starting with heart failure and death. It's a very morbid disease that's difficult to manage. NXC-201 goes to the source of production and removes the very production source of this toxic, damaging light chains.
There's about 5,000 patients in the U.S. being diagnosed on an annual basis. For the longest time, there was only one approval.
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