INA03 / Inatherys - LARVOL DELTA

Final results of a first-in-human Phase I study of INA03, a novel CD71/TfR1-targeted antibody-drug conjugate, in patients with relapsed or refractory acute leukemia. (ASH 2025) - P1 | "INA03, first in class CD71/TfR1-targeted ADC, demonstrated a manageable safety profile and favorablepharmacokinetics in patients with relapsed or refractory acute leukemia. The MTD was defined at 2mg/kg. Early signs of biological and clinical activity above benchmark of this novel targeted asset supportcontinued investigation in Phase II studies and rational combination strategies."

Clinical • First-in-human • P1 data • Acute Myelogenous Leukemia • Dental Disorders • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Immunology • Leukemia • Stomatitis • TFRC

Preliminary Results of a Phase 1, First-in-Human Study of INA03, an Anti-CD71 Antibody-Drug Conjugate in Patients with Relapsed or Refractory (R/R) Acute Leukemias (ASH 2023) - P1 | "Eligibility criteria are age >18y, ECOG50%, Creatinine Clearance >30ml/min, normal liver function. The preliminary results of this phase 1 study indicate that INA03 is well tolerated for doses up to 2.5 mg/kg. Induction of transient erythroblastopenia indicates effective targeting of CD71 in vivo and early signs of antileukemic efficacy have been observed in patients with refractory AL. Inclusion in the trial are continuing and additional data will be presented."

Clinical • First-in-human • P1 data • Dermatology • Hematological Malignancies • Hepatology • Leukemia • Mucositis • Oncology • Renal Disease • TFRC

Results of a Phase 1, First-in-Human Study of INA03, an Anti-CD71 Antibody-Drug Conjugate in Patients with Relapsed or Refractory (R/R) Acute Leukemias (ASH 2024) - P1 | "Eligibility criteria are age >18y, ECOG50%, Creatinine Clearance >30ml/min, normal liver function. Induction of transient erythroblastopenia and blast reduction showed effective targeting of CD71 in vivo and early signs of antileukemic efficacy have been observed in patients with refractory AL. Overall, study results indicate a RP2D of 2 mg/kg."

Clinical • First-in-human • P1 data • Dermatology • Gastrointestinal Disorder • Hematological Malignancies • Hepatology • Leukemia • Mucositis • Oncology • Renal Disease • TFRC

Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia (clinicaltrials.gov) - P1 | N=33 | Completed | Sponsor: Institut Paoli-Calmettes | Active, not recruiting ➔ Completed | Trial completion date: Jul 2025 ➔ Feb 2025

Trial completion • Trial completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • T Acute Lymphoblastic Leukemia

Preclinical evaluation of an anti-transferrin receptor antibody drug conjugate targeting triple-negative breast cancer (ESMO-TAT 2025) - P1 | "A clinical trial evaluating INA03 ADC in patients with refractory/relapsed acute myeloid leukemia (NCT03957915) is ongoing, with the recommended phase 2 dose under investigation for hematologic malignancies. But given its potent activity in TNBC preclinical models, INA03 is intended for future development also in solid tumors."

Preclinical • Acute Myelogenous Leukemia • Breast Cancer • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • Triple Negative Breast Cancer • TFRC

Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia (clinicaltrials.gov) - P1 | N=33 | Active, not recruiting | Sponsor: Institut Paoli-Calmettes | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2023 ➔ Jul 2025 | Trial primary completion date: Dec 2023 ➔ Jul 2024

Enrollment closed • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • T Acute Lymphoblastic Leukemia

INA03, a potent transferrin-competitive antibody-drug conjugate against CD71, for a safer acute leukemia treatment. (PubMed, Mol Cancer Ther) - "INA03 administration in hCD71/hTf mice did not reveal, even at high doses, any improper toxicities. Hence, these data demonstrate promising pre-clinical efficacy and safety of INA03 and support its development as a novel acute leukemia treatment."

Journal • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • CTSB • TFRC

PRELIMINARY RESULTS OF A PHASE 1, FIRST-IN-HUMAN STUDY OF INA03, AN ANTI-CD71 ANTIBODY-DRUG CONJUGATE IN PATIENTS WITH RELAPSED OR REFRACTORY (R/R) ACUTE LEUKEMIAS (EHA 2023) - P1 | "Eligibility criteria are: R/R AL after at least one line of treatment including high dose chemotherapy and/or Vidaza and Venetoclax, AlloSCT >18y, with FEVG>50%, Creatinine Clearance >30ml/min, normal liver function, ECOG<2. INA03 is well tolerated up to the dose of 2 mg/kg with some efficacy signals in R/R AL. The trial continues to accrue patients."

Clinical • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Renal Disease • TFRC

Preliminary results of a phase 1, first-in-human study of INA03, an anti-CD71 antibody-drug conjugate, in patients with relapsed or refractory (R/R) acute leukemias. (ASCO 2023) - P1 | "Eligibility criteria are: R/R AL after at least one line of treatment including high dose chemotherapy and/or Vidaza and Venetoclax, AlloSCT > 18y, with FEVG > 50%, Creatinine Clearance > 30ml/min, normal liver function, ECOG < 2. INA03 is well tolerated up to the dose of 2 mg/kg with some efficacy signals in R/R AL. The trial continues to accrue patients. Clinical trial information: NCT03957915."

Clinical • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Renal Disease • TFRC

Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia (clinicaltrials.gov) - P1 | N=34 | Recruiting | Sponsor: Institut Paoli-Calmettes | Unknown status ➔ Recruiting | Trial completion date: Feb 2022 ➔ Dec 2023 | Trial primary completion date: Jan 2022 ➔ Dec 2023

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

Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia (clinicaltrials.gov) - P1; N=34; Recruiting; Sponsor: Institut Paoli-Calmettes; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia (clinicaltrials.gov) - P1; N=34; Not yet recruiting; Sponsor: Institut Paoli-Calmettes; Trial completion date: Nov 2021 ➔ Feb 2022; Initiation date: Sep 2019 ➔ Jan 2020; Trial primary completion date: Sep 2021 ➔ Jan 2022

Clinical • Trial completion date • Trial initiation date • Trial primary completion date