iCell Gene Therapeutics Presents First-in-Human Data of CLL1-CD33 Compound CAR T in Refractory Acute Myeloid Leukemia

NEW YORK–(BUSINESS WIRE)–iCell Gene Therapeutics, LLC announced results from a study ongoing at
Chengdu Military General Hospital of ICG144, the first CLL1-CD33
Compound CAR T-cell (cCAR) in clinical study, in patients with
particularly difficult to treat Acute Myeloid Leukemia (AML). Patients 1
and 2 both failed multiple previous cycles of therapy and presented with
complex conditions limiting further options. Treatment with CLL1-CD33
cCAR led both patients to complete response and engraftment of
haploidentical stem cell transplantation (allo-HSCT) without
myeloablative conditioning.

“Patient response seen to date is encouraging for refractory AML
patients, and opens the potential of this novel therapy as bridge to
transplant, a supplement to chemotherapy, or as a standalone therapy for
patients with acute myeloid leukemia.” stated Dr. Fang Liu, MD, PhD, the
Principal Investigator of the study who presented the results at the 60th
American Society of Hematology (ASH) Annual Meeting in San Diego. Dr.
Yupo Ma, MD, PhD, Chairman of iCell Gene Therapeutics added, “Initial
patient experience highlights the potential importance of iCell’s
proprietary multiple antigen targeting and enhancing technologies to
overcome antigen escape and improve treatment outcomes.”

Upon enrollment, patients receive a lymphodepletion regimen consisting
of fludarabine and cyclophasphamide followed by 1×106 – 2×106
CAR T cells/kg, nonmyeloablative conditioning and Haplo-HSCT

  • Patient 1 is a 6-year-old originally diagnosed with Franconi anemia
    transformed JMML and eventually to AML-M5 with more than 90% blasts in
    the marrow, complex karyotype and FLT3-ITD mutation.
  • Patient 2 is a 23-year-old, failed to TKIs, AP-CML (basophils20%,
    plt1000X109/L), T315I mutation.
  • Complete response and Haplo-HSCT engraftment was observed in both
    patients.
  • Grade 1 CRS and pancytopenia was observed in both patients.
  • Grade 3 neurotoxicity was observed in Patient 1.

About CLL1-CD33 cCAR T cell therapy

CLL1-CD33 cCAR is a compound Chimeric Antigen Receptor (cCAR)
immunotherapy with two distinct functional CAR molecules expressed on a
T-cell, directed against the surface proteins CLL1 and CD33. The
diseases treated by CLL1-CD33 cCAR could include acute myeloid leukemia,
myelodysplastic syndromes, chronic myeloid leukemia and chronic
myeloproliferative neoplasms. CLL1 is associated with leukemia stem
cells and disease relapse, while CD33 is expressed on bulky AML disease.
Treatment of AML is a challenge due to heterogeneity of AML bearing
cells, which renders single antigen targeting CAR T-cell therapy
ineffective. ICG144 cCAR is designed to target the mechanisms of
single-CAR relapse, specifically antigen escape and leukemic stem cells.

About AML

Acute myeloid leukemia (AML) is the abnormal proliferation of immature
myeloid cells and the most common leukemia in adults. Prognosis is
dismal when AML relapses or is refractory to chemotherapy. Mortality
associated with this disease is high, with approximately 10,000 deaths
in 2018 in the US.

About iCell Gene Therapeutics

iCell Gene Therapeutics, located in Stony Brook, New York, is a
clinical-stage biotechnology company developing first-in-class chimeric
antigen receptor engineered cells. Clinical studies on our CARvac,
T-cell targeted CARs, Compound CARs and Non-gene edited universal CARs
are ongoing in the US and in China. For more information, please visit www.icellgene.com

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