VCAR33AUTO / Vor Biopharma - LARVOL DELTA

17-CD33CART: Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=52 | Recruiting | Sponsor: Center for International Blood and Marrow Transplant Research | Active, not recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

17-CD33CART: Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=52 | Active, not recruiting | Sponsor: Center for International Blood and Marrow Transplant Research | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=52 | Recruiting | Sponsor: Center for International Blood and Marrow Transplant Research | Active, not recruiting ➔ Recruiting | Trial primary completion date: Dec 2024 ➔ Dec 2029

Enrollment open • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=52 | Active, not recruiting | Sponsor: Center for International Blood and Marrow Transplant Research | Recruiting ➔ Active, not recruiting | N=37 ➔ 52

Enrollment change • Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Multiple Clinical and Preclinical Presentations at ASH 2023 Highlight Vor Bio’s Novel eHSC and CAR-T Platform (GlobeNewswire) - P1/2 | N=24 | NCT05984199 | Sponsor: Vor Biopharma | "This data shows that as of the cutoff date of June 1, 2023, 2 of 5 (40%) evaluable patients treated at the highest dose level (DL4, 1 x 10⁷ CAR⁺ cells/kg) achieved complete remission. Transient CD33CART expansion was detected in 11 (58%) subjects across all doses tested and in all 6 (100%) subjects evaluated at DL4, as of the cutoff date. Four out of 19 evauable patients treated had cytokine release syndrome (CRS) ≥ Grade 3."

Cytokine release syndrome • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Multiple Clinical and Preclinical Presentations at ASH 2023 Highlight Vor Bio’s Novel eHSC and CAR-T Platform (GlobeNewswire) - "Vor Bio...today announced that preclinical and clinical data supporting the Company’s novel platform and approach for treating acute myeloid leukemia (AML), will be presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, being held from December 9-12, 2023, in San Diego, CA."

P1/2 data • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Vor Bio Reports Second Quarter 2022 Financial Results and Provides Company Update (GlobeNewswire) - "Upcoming Milestones: Initial VOR33 clinical data on-track for Q4 of 2022; VCAR33ALLO IND submission expected in the first half of 2023; VOR33 + VCAR33 Treatment System IND submission expected following initial clinical data from the VBP101 clinical trial and the VCAR33ALLO program."

IND • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Vor Bio Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Company Update (GlobeNewswire) - "VCAR33^AUTO uses autologous cells from each patient, and is being studied in an ongoing Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (NMDP) in young adult and pediatric patients with relapsed/refractory AML in a bridge-to-transplant study. The Company anticipates that NMDP will share data from this study in 2022....VCAR33^ALLO is a newly announced program in which Vor Bio plans to submit an investigational new drug (IND) application in the first half of 2023 to support a Phase 1/2 clinical trial for patients with relapsed/refractory AML."

IND • New P1/2 trial • P1/2 data

Vor Reports Third Quarter 2021 Financial Results and Provides Company Update (GlobeNewswire) - "Upcoming Milestones: (i) Initial VCAR33 monotherapy clinical data expected in 2022; (ii) In-house manufacturing facility operational in 2022; (iii) File an investigational new drug (IND) application for VOR33/VCAR33 Treatment System in the second half of 2022."

Clinical data • Commercial • IND • Oncology