Slide 1: Initial Safety and Efficacy Data from Nexicart-2 in R/R AL Amyloidosis
Initial Safety and Efficacy Data from Nexicart- 2, the First U.S. Trial of a CAR-T (NXC-201) in Relapsed or Refractory (R/R) Light Chain (AL) Heather Landau1, Shahzad Raza2, Aaron Rosenberg3, Jeffrey Zonder4, Raymond Comenzo5, Vaishali Sanchorawala6, Michaela Liedtke7, Amandeep Godara8, Michael Rose
Slide 2: Relapsed/Refractory AL Amyloidosis: ~32,500 U.S. Patients with No FDA Approved Drugs
Relapsed/Refractory AL Amyloidosis: ~32,500 U.S. Patients with No FDA Approved Drugs1 Light chains deposit in organs, damaging organs Light chains produced by Light chains misfold dysfunctional plasma Heart and aggregate cells in bone marrow • Heart failure • Dangerous, irregular heart rhythms Bone
Slide 3: NXC-201: Sterically-Optimized CAR-T construct
NXC-201: Sterically-Optimized CAR-T construct N-GENIUS PLATFORM “Digital Filter”…. …decreases nonspecific Sterically-optimized key construct activation modifications 1 Proprietary Optimized 2 Proprietary Optimized 3 Proprietary Optimized CD3 – “CD3ζγ” CD8 Hinge Flexibility COBRA Binder ✓ Delivers “D
Slide 4: First CAR-T Trial Designed For R/R AL Amyloidosis (NCT06097832)
NEXICART-2: First CAR-T Trial Designed For R/R AL Amyloidosis (NCT06097832) Study design • Open-label, single-arm, multi-site phase 1/2 study • n=40 patients Key criteria • Exposed to at least 1 line of therapy, including CD38 monoclonal antibody + proteosome inhibitor • Measurable hematologic disea
Slide 5: NEXICART-2: Trial Design
NEXICART-2: Trial Design Open to accrual June 2024 Phase 1 ✓ Phase 2 Ongoing Dose Dose FDA Escalation Expansion Submission 150 x 106 450 x 106 (n=3) (n=30) 450 x 106 (n=7) Data available as of cut-off November 13, 2025. No DLTs Palladini G et al. JCO 2012. Palladini G et al. Amyloid 2012. Milani et
Slide 6: NEXICART-2: Patient Characteristics (N=20)
NEXICART-2: Patient Characteristics (N=20) Median NX2-001 NX2-002 NX2-003 NX2-004 NX2-005 NX2-006 NX2-007 NX2-008 NX2-009 NX2-010 NX2-011 NX2-012 NX2-013 NX2-014 NX2-015 NX2-016 NX2-017 NX2-018 NX2-019 NX2-020 (range) Age 56 67 82 64 62 72 77 66 63 80 65 65 59 49 73 59 71 71 82 64 66 (49-82) Gender
Slide 7: NEXICART-2: Safety
NEXICART-2: Safety Only low-grade, brief CRS No neurotoxicity Median Subject NX2-001 NX2-002 NX2-003 NX2-004 NX2-005 NX2-006 NX2-007 NX2-008 NX2-009 NX2-010 NX2-011 NX2-012 NX2-013 NX2-014 NX2-015 NX2-016 NX2-017 NX2-018 NX2-019 NX2-020 (Range) CART Cell Dose Dose 150 150 150 450 450 450 450 450 450
Slide 8: NEXICART-2 Efficacy: Rapid normalization of pathologic paraprotein
NEXICART-2 Efficacy: Rapid normalization of pathologic paraprotein * Cut-off November 13, 2025. Median follow up 235 days (7.8 months) (range (days) 90-505) 19/20 (95%) patients’ disease marker normalized Immunofixation may persist for longer Palladini G et al. JCO 2012. * IgG sub-type (NX2-011) hav
Slide 9: NEXICART-2 Efficacy
• 19/20 (95%) early and deep hematologic responses NEXICART-2 Efficacy • Organ responses in 70% (7/10) evaluable patients (75% , 60% , 100% ) • Median time to response 33 and 113 days Patient # Palladini G et al. JCO 2012. Palladini G et al. Amyloid 2012. Milani et al. Blood 2017.
Slide 10: In Patient's Own Words (day +15)
… In Patient’s Own Words (day +15) “Hi Dr Landau! Here we are a week tomorrow since I left the hospital (At day 10 after those magic CAR-T cells came on board)! Just had to tell you I’ve been very happily & comfortably walking 2-3+ miles each day & doing great on the inclines (even the cross overs o
Slide 11: 75% complete response rate as reviewed by an independent review committee
NEXICART-2: 75% complete response rate as reviewed by an independent review committee n=20. Cut-off November 13, 2025. Median follow up 235 days (7.8 months) (range (days) 90-505) Subject #: NX2- 001 002 003 004 005 006 007 008 009 010 011 012 013 014 015 016 017 018 019 020 Time to response (days)
Slide 12: Conclusion
Conclusion • NXC-201 a novel BCMA-directed CAR-T cell can be administered safely & efficiently to patients with R/R AL amyloidosis – population without a single FDA-approved treatment available – who have a true unmet medical need • 100% treated with a vein-to-vein time 14 days • Low grade CRS and n
Slide 13: Sarasota, FL Montauk, NY Ireland
Sarasota, FL Montauk, NY Ireland Peru Saratoga, NY Japan Miami, FL Alaska
Slide 14: Grateful for…. Thank you for your attention!
Grateful for…. ▪ The research staff, clinical teams, apheresis units, cell therapy labs and investigators at each participating site ▪ The patients and their families Thank you for your attention! Study was funded by: NIH/NCI Cancer Center Support Grant P30 CA008748.