CABA’s Phase 1/2 RESET Clinical Trial Safety and Efficacy Results Abstracts to Be Presented at EULAR Conference in London Published Here Two Weeks Early - Lupus, Scleroderma and Myositis - RESE-CEL Effective against SLE, Lupus Nephritis and One Form of Myositis - Two ICANS Events Reported
CABA abstracts for the upcoming EULAR conference are now published and online.
Screenshots of all the abstracts here.
This morning, I found CABA’s abstracts it will present in 2 weeks at the EULAR conference in London published on EULAR’s website in a 2500 page compendium of abstracts for the conference. Link below.
The two big most important ones concern the Phase 1/2 results of safety and efficacy of RESE-CEL in severe refractory SLE and lupus nephritis and in severe refractory systemic sclerosis (scleroderma). Results for lupus and lupus nephritis were excellent. Same with scleroderma, where all 6 patients showed clinical improvement. The myositis results showed RESE-CEL very effective against one of the disease’s forms. Less so against others.
There was one Grade 4 ICANS event (a serious neurological complication) in a Lupus Nephritis patient (1 of 9 Lupus patients and 1 of 5 LN patients) that was traced to a hidden infection and that was resolved and one Grade 3 ICANS event in one of the six SSc patients. Both were resolved. No serious adverse events among the 13 myositis patients.
What follows below is my longer layman’s explanation that I prepared for friends and family as many are invested in CABA.
My takeaway from the results of the RESET trial on SLE and Lupus Nephritis.
Results were very good for severe refractory lupus and lupus nephritis. 9 patients were treated, 5 with non-kidney lupus and 4 with lupus nephritis. Among the non-kidney lupus patients, 3 of 4 patients achieved DORIS remission, meaning their lupus became clinically quiet by a strict remission standard. Disease activity scores improved meaningfully, and key lupus blood markers also moved in the right direction.
The kidney results were also solid: 3 of 4 lupus nephritis patients showed renal responses, including one complete renal response, one partial response with major proteinuria (protein in urine) reduction, and one patient with biopsy evidence of kidney improvement. Importantly, patients improved after stopping their usual lupus immune drugs, supporting the idea that RESE-CEL may “reset” part of the immune system rather than just suppress it.
CABA may take on the result of the RESET Phase 1/2 trial for scleroderma-great results on efficacy.
All 6 patients showed meaningful improvement on a standard scleroderma response scale. Several had stronger responses showing improvement in skin and overall disease activity. Some patients also showed stabilization or improvement in lung function tests, important because lung disease is one of the most serious complications of scleroderma.
The study also found biological evidence that the therapy was doing what it was designed to do. The engineered CAR-T cells expanded in the body and wiped out most circulating B cells. When B cells later returned, they mostly looked like “new” immature B cells rather than the older, potentially disease-driving ones.
Safety in the SSc trial was mixed. The patients were all pretty sick to begin with and all issues were resolved. There was 1 grade 3 ICANS,
1 case of neutropenic fever and 1 case of hypercapnic respiratory failure with encephalopathy in the setting of multiple sedating medicines. 3 cases of CRS.
Neutropenic fever is a known complication of CAR-T especially where, as here, the patients received chemotherapy preconditioning which suppresses the immune system. In layman’s term’s neutropenic fever means the patient’s immune defenses are temporarily wiped down to very low levels, and they developed a fever that doctors assume could represent a serious infection until proven otherwise. Here the patient’s condition was resolved.
In layman’s terms, hypercapnic respiratory failure with encephalopathy in the setting of multiple sedating medicines means the patient was was likely over-sedated, their breathing became inadequate, carbon dioxide built up, and that excess CO₂ impaired their brain function. Again this patient’s condition was resolved.
My takeaway from the RESET Myositis Phase 1/2 Results
The CABA myositis study tested RESET-CEL in 13 patients with severe autoimmune muscle diseases that had not responded well to standard treatments.
These diseases included dermatomyositis, antisynthetase syndrome, and immune-mediated necrotizing myopathy.
In plain English, these are conditions where the immune system attacks the muscles (and sometimes the skin or lungs) causing weakness, inflammation, fatigue, pain, and potentially serious organ complications.
The study demonstrated that RESE-CEL performed very well against dermatomyositis. Most patients with dermatomyositis or antisynthetase syndrome improved even after stopping many of their regular immune-suppressing medications, and all of the dermatomyositis patients maintained their responses through the latest follow-up. Some patients were able to stay on little or no steroid treatment.
The study also showed strong biological evidence that the RESE-CEL therapy was working as intended. After infusion, the engineered CAR-T cells expanded in the body and sharply reduced B cells, which are immune cells believed to help drive autoimmune disease. When B cells later returned, they mostly looked like newly formed “naive” B cells rather than older immune cells that may have been programmed to attack the body. In layman’s terms, the treatment appeared to partially reboot the immune system. Safety results were encouraging overall, with only mild cytokine release syndrome (CRS) seen in a few patients and no major neurologic toxicity in this study. However, the responses were not equally strong across all myositis types. Dermatomyositis looked best, while antisynthetase syndrome and necrotizing myopathy showed more mixed or less durable results.
Overall these results are excellent and show that RESE-CEL appears to be effective in treating a number of very serious and often fatal autoimmune conditions that often lack effective treatments.
Should be very good for the stock although I expect some questions over the 2 ICANS events (which are common in CAR-T in cancer treatment so doctors know how to treat them).
Note that this board only allows you to post one picture and none in the comments so I can’t post all of the screenshots of the results. Besides the link, you can also find them by googling 2026 EULAR abstracts. Once you are in the 2500 page compendium of abstracts, you can word search. I found these by using the term Cabaletta Bio.