FF-10501-01 / Fujifilm Holdings, Strategia Therap - LARVOL DELTA

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov) - P1/2 | N=55 | Completed | Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc. | N=41 ➔ 55

Enrollment change • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Results of a Phase 1, Dose-Escalation Study of FF-10501-01 in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML) or Hypomethylating Agent (HMA)-Resistant Myelodysplastic Syndrome (MDS) (ASH 2018) - "FF-10501-01 had predictable pharmacokinetics and pharmacodynamic testing verified its mechanism of action as an IMPDH inhibitor. FF-10501-01 in combination with other agents, is currently undergoing additional clinical testing."

Clinical • P1 data • Acute Myelogenous Leukemia • Biosimilar • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation

A Study of FF-10501-01 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome (clinicaltrials.gov) - P2; N=0; Withdrawn; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; Trial completion date: Dec 2023 ➔ Oct 2019; Trial primary completion date: Oct 2022 ➔ Oct 2019

Clinical • Combination therapy • Trial completion date • Trial primary completion date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Anti-Leukemia Effect of FF-10501-01, a Novel Inosine 5'-Monophosphate Dehydrogenase Inhibitor, in Acute Myeloid Leukemia (ASH 2016) - "High-performance liquid chromatography (HPLC) was performed to detect the intracellular concentration of guanine nucleotides, with mycophenolic acid (MPA) treated cells used as positive control. The IMPDH inhibitor FF-10501-01 can produce potent anti-proliferative and apoptotic induction effects on AML cell lines, including HMA-resistant cell lines, through inhibition of de novo guanine nucleotide synthesis. These results indicate that FF-10501-01 might be a promising new therapeutic agent for AML."

Acute Myelogenous Leukemia • Biosimilar • Hematological Malignancies • Leukemia • Oncology

Phase 1 results of FF-10501-01, a novel inosine 5'-monophosphate dehydrogenase inhibitor, in advanced acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), including hypomethylating agent (HMA) failures (ASH 2016) - Ambassador: Hagop M. Kantarjian; Elias J. Jabbour; Naveen Pemmaraju; P1, N=29; "The median number of FF-10501-01 cycles received to date is 2 (range 1 – 24). Partial remissions have occurred in 2 AML patients (50 and 100 mg/m2 BID) after 3 cycles, lasting for 5 and 24 cycles, respectively, with the higher dose patient still on study after 24 cycles."

P1 data • Hematology KOL Tracker

Results of a Phase 1/2a dose-escalation study of FF-10501-01, an IMPDH inhibitor, in patients with acute myeloid leukemia or myelodysplastic syndromes. (PubMed, Leuk Lymphoma) - P1/2 | "2 of 20 (10%) evaluable MDS/CMML patients (Phase 1 and 2a) attained marrow complete remission, one continuing treatment for 17 months. While FF-10501-01 demonstrated clinical activity and target inhibition in heavily pretreated patients with AML and MDS/CMML, increased mucositis events led to Phase 2a closure (ClinTrials.gov#NCT02193958)."

Clinical • Journal • P1/2 data

FF-10501 induces caspase-8-mediated apoptotic and endoplasmic reticulum stress-mediated necrotic cell death in hematological malignant cells. (PubMed, Int J Hematol) - "FF-10501 induced necrotic cell death via endoplasmic reticulum stress in OCI-AML3 cells. The present study is the first to identify intracellular pathways involved in FF-10501-induced cell death."

Journal

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov) - P1/2; N=41; Completed; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; Active, not recruiting ➔ Completed

Clinical • Trial completion

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov) - P1/2; N=41; Active, not recruiting; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; Trial primary completion date: Jun 2019 ➔ Nov 2019

Clinical • Trial primary completion date

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov) - P1/2; N=41; Active, not recruiting; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; Trial primary completion date: Jan 2019 ➔ Jun 2019

Clinical • Trial primary completion date

A Study of FF-10501-01 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome (clinicaltrials.gov) - P2; N=0; Withdrawn; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; N=42 ➔ 0; Trial completion date: Dec 2020 ➔ Dec 2023; Initiation date: Oct 2018 ➔ Oct 2019; Not yet recruiting ➔ Withdrawn; Trial primary completion date: Oct 2020 ➔ Oct 2022

Clinical • Combination therapy • Enrollment change • Trial completion date • Trial initiation date • Trial primary completion date • Trial withdrawal

Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies (clinicaltrials.gov) - P1/2; N=41; Active, not recruiting; Sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.; Recruiting ➔ Active, not recruiting; N=68 ➔ 41; Trial primary completion date: Jun 2019 ➔ Jan 2019

Clinical • Enrollment change • Enrollment closed • Trial primary completion date