Slide 1: Initial Safety and Efficacy Data from Nexicart-2, the First U.S. CAR-T Trial 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 J. Landau, MD 1, Charlotte Hug1hes, MD 1, Aaron Seth Rosenberg, MD 2 , Mehrdad Abedi , MD 2, Shahzad Raza, MD 3 , Jeffrey A. Zonder, MD4 , Eugene Brai
Slide 2: Overview / Conclusion
Overview / Conclusion • Initial results from Nexicart-2 suggest NXC-201 can be administered safely & efficiently to patients with relapsed/refractory (R/R) AL amyloidosis • mild + manageable CRS and no neurotoxicity • 100% experienced rapid and deep hematologic responses • Organ responses in 80% eva
Slide 3: Relapsed/Refractory AL Amyloidosis: ~23,000 U.S. Patients with No FDA Approved Drugs
Relapsed/Refractory AL Amyloidosis: "'23,000 U.S. Patients with No FDA Approved Drugs 1 Light chains deposit in organs, damaging organs Light chains produced by I Light chains m isfold dysfunctional plasma Heart and aggregate cells in bone marrow • Hurt failure r- • Danprous, lrrecul•r h ..art rhyth
Slide 4: NXC-201: Sterically-Optimized CAR-T construct
NXC-201: Sterically-Optimized CAR-T construct □ Sterically-optimized key construct modifications 0 CD3 Proprietary Optimized - "CD3~y" ' 0 CDS Proprietary Optimized Hinge Flexibility 0 COBRA Proprietary Optimized Binder I ✓ Delivers " Digital" ✓ Enhances Plasma Cell Binding ✓ Reduces cytokin e relea
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) • Open-label, single-arm, multi-site phase 1/2 study • n=40 patients • Exposed to at least 1 line of therapy, including CD38 monoclonal antibody+ proteosome inhibitor • Measurable hematologic disease, defined by one of the f
Slide 6: NEXICART-2: Status
NEXICART-2: Status o ·pen to accrual ,June 2,024 Phase 1 ✓ Phase .2 I Ongoing ➔ 25 more patients planned 45.0 X 106 (n:S'*) 4S0 .x l06 * 12 patients dosed at 450 x 106 in cludirng 7 + 5 from i( n=7*) Phase 1 and 2, respectively No DLTs Data available as of cut-off April 11, 2025. Pafladini G et al.
Slide 7: NEXICART-2: Patient Characteristics
NEXICART-2: Patient Characteristics Median 67 56-82 emae ae emae em ae ae ae ae ae ae Prior lines of 4'" 6·• 2 4 4• 3 12• 4* 4" 3• 4 (2-12) thera L 65 24 86 41 26 47 121 84 56 24-121 0.79 0.65 Organ Heart/GI/ Kidney/ Heart Kidney Heart/GI Kidney Heart Nerve Heart Heart Involvem ent NYHAsta e II II N
Slide 8: NEXICART-2: Safety
NEXICART-2: Safety CRS and ICANS reported according to ASTCT Consensus Grading CART Cell Dose 1S0 1S0 150 450 450 450 450 450 450 450 (x10 6 ) CRS None None Grade 2 Grade 1 Grade 1 Grade 1 Grade 1 Grade 1 Grade 1 Grade 1 1 (1-2) CRS Onset None None 3 3 1 1 1 1 1 3 None None 1 1 1 1 1 4 1 2 1 (1-4 No
Slide 9: NEXICART-2: Results
NEXICART-2: Results Data available as of cut-off April 11, 2025. Median follow up 121 days (range 29-289). Rapid normalization of pathologic paraprotein associated with organ responses ~ °" Best Response ... NXC-201 dosing 0.9 •10% 250 ..,! · 20% NYHA reduction 0.8 E II ➔ I 0 0.7 .:: 200 ■ dFLC .,,•
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
Slide 11: NEXICART-2: Hematologic responses reviewed by independent review committee
NEXICART-2: hematologic responses as reviewed by an independent review committee Data available as of cut-off April 11, 2025. Median follow up 121 days (range 29-289). Subject # NX2-001 NX2·002 NX2-003 NX2-004 NX2·00S NX2-006 NX2-007 NX 2-008 NX2-009 NX2-010 NXC·201 Dos@ (million CAR+ T ,ell,) 150 1
Slide 12: Conclusion
Conclusion • NXC-201 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 • Al (100%) received treatment with a vein-to-vein time 14 days • Low grade CRS and no neurotoxicity o
Slide 13: A Giant Thank You
A Giant Thank You .... • 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: - ~ • IMMIX •?. B OPHARMA ~ NEXCELLA NIH/ NCI Cancer Center Support Grant