Fujifilm : initiates a Phase I clinical trial of Anti-Cancer Drug FF-10101 in the United States in patients with Relapsed or Refractory Acute Myeloid Leukemia (AML).

August 8, 2017

FUJIFILM Corporation (President: Kenji Sukeno) announced the commencement of a Phase I clinical trial of its anti-cancer drug FF-10101 in the Unites States in patients with relapsed or refractory acute myeloid leukemia (AML).

AML is a type of hematological malignancy associated with the malignant transformation of hematopoietic stem cells, which generate blood cells, into leukemia cells. These leukemia cells then exhibit abnormal growth in the bone marrow and it prevents the generation of blood cells. Furthermore, the leukemia cells with aberrant growth invade other organs outside the bone marrow, resulting in various functional disorders. The most of the patients become refractory (5-year survival rate of approximately 30% *). Currently, the number of estimated new AML patients is around 20,000 in the United States each year.
Approximately 30% of AML patients harbor mutations related to FLT3**, a protein involved in the proliferation of hematopoietic stem cells. These mutations include internal tandem duplications (ITD)***and tyrosine kinase domain (TKD)*4, which induce abnormal growth of leukemia cells and are associated with poor prognosis. This is why there is a growing focus on FLT3 inhibitors which can suppress the replication of leukemia cells by inhibiting the activity of the mutated FLT3.

FF-10101 is a FLT3 inhibitor discovered by Fujifilm. It taps into the advanced technology for synthesizing and designing chemical compounds nurtured through Fujifilm's photographic film business*5.
FF-10101 inhibits FLT3 activity by binding irreversibly with an amino acid in FLT3, suppressing the abnormal growth of leukemia cells. In cellular experiments, FF-10101 showed strong inhibitory activities against the proliferation of leukemia cells expressing TKD mutations conferring resistance to other FLT3 inhibitors. FF-10101 also demonstrated high efficacy in preclinical mouse models using primary cells from leukemia patients with the ITD or TKD mutation, and therefore promising efficacy in clinical trials is expected.

Fujifilm is defining oncology as its focal area and promoting the R&D of anti-cancer drugs by combining the technologies and experiences including chemical synthesis capacity, design ability, analysis technology accumulated through the development and production of photographic film. Clinical trials of multiple anti-cancer drugs are already underway in Japan and overseas.

The company will keep focusing on unmet medical needs in fields including oncology and actively promote R&D to expand business deployment and supply of innovative pharmaceutical products so as to contribute to resolving social issues.

• *Approximately 30% of all patients diagnosed with AML remain alive five years after their diagnosis.
• **FLT3 is expressed on the cell membranes of hematopoietic cells and progenitor cells, and is involved in the differentiation and proliferation of blood cells.
• ***When this mutation occurs, juxtamembrane coding regions ranging in size from 3 to hundreds of nucleotides of the FLT3 gene are replicated.
• *4When this mutation occurs, some of the amino acids in FLT3 are substituted to other amino acids.
• *5The development of FF-10101 was adopted as Next generation Technology Program (NexTEP) by the Japan Science and Technology Agency(JST).

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