Slide 1: Initial Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of a CAR-T (NXC-201) in R/R AL
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, Raymond Comenzo4, Amandeep Godara5, Jeffrey Zonder6, Vaishali Sanchorawala7, Jonathan Kaufman8, Al-Ola Abdall
Slide 2: Disclosure
Disclosure Heather Landau Advisory role/honoraria: Pfizer, Nexcella, Abbvie, BMS, Alexion, Arcellx, Karyopharm, Janssen Research funding: Janssen, Nexcella, Abbvie, Alexion, Prothena, Protego 2
Slide 3: 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 4: 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 5: NEXICART-2: 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 6: 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 6
Slide 7: NEXICART-2: Patient Characteristics (N=23)
NEXICART-2: Patient Characteristics (N=23) 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 NX2-021 NX2-022 NX2-023 (range) Age 56 67 82 64 62 72 77 66 63 80 65 65 59 49 73 59 71 71
Slide 8: Time from apheresis to NXC-201 infusion
Time from apheresis to NXC-201 infusion NX2-023 NX2-022 NX2-021 NX2-020 NX2-019 • 100% of doses manufactured NX2-018 NX2-017 successfully NX2-016 NX2-015 NX2-014 • No bridging therapy required NX2-013 NX2-012 • median vein-to-vein time = 14 days (range 13-15) NX2-011 NX2-010 NX2-009 NX2-008 NX2-007
Slide 9: NEXICART-2: Safety
NEXICART-2: Safety 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 NX2-021 NX2-022 NX2-023 (Range) CART Cell Dose 150 150 150 450 450 450 450 450 450 450 450 450 450 450 450
Slide 10: NEXICART-2 Efficacy
NEXICART-2 Efficacy Rapid normalization of pathologic paraprotein dFLC M-Spike Best Response 0% -10% Percentage change in dFLC/M-Spike from baseline -20% -30% -40% -50% -60% -70% -80% -90% -91.13% -93.36% -100% -98.46% -99.48% -99.69% -100.00% -100.00% -100.00% -100.00% -100.00% -100.00% -100.00% -1
Slide 11: Rapid reduction in paraprotein results in organ responses
Rapid reduction in paraprotein results in organ responses NXC-201 dosing Organ Responses NX2-002 5 420 NYHA stage II → I NX2-003 4.5 Renal 4 360 NX2-006 Cardiac 3.5 m-spike (g/dL) 300 NX2-008 dFLC (mg/L) Cardiac + Liver 3 NX2-010 240 Renal 2.5 NX2-011 180 Cardiac 2 NX2-012 NYHA stage II → I 1.5 120
Slide 12: … 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 13: Minimal residual disease (MRD) at day 25, 6 & 12 months
Minimal residual disease (MRD) at day 25, 6 & 12 months by flow cytometry or Clonoseq (10-5 or 10-6) D + 25 D + 25 Method / Sensitivity D + 6M D + 6M Method/ Sensitivity D + 12M D + 12M Method/ Sensitivity NX2-023 Negative ClonoSEQ 10-5 NR NR NR NR NX2-022 Negative Flow / 10-5 NR NR NR NR NX2-021 Ne
Slide 14: NEXICART-2: Results
NEXICART-2: Results 74% complete hematologic response rate (independent review committee) n=23. Cut-off November 13, 2025. Median follow up 210 days (7.0 months) (range (days) 90-505) Subject #: NX2- 001 002 003 004 005 006 007 008 009 010 011 012 013 014 015 016 017 018 019 020 021 022 023 Time to
Slide 15: 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% manufactured and treated; median vein-to-vein time 14 days • L
Slide 16: Sarasota, FL Montauk, NY Ireland
Sarasota, FL Montauk, NY Ireland Peru Saratoga, NY Japan Miami, FL Alaska 16
Slide 17: 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! 17