ExCellThera Inc. (ExCellThera), an advanced clinical-stage biotechnology company delivering molecules and bioengineering solutions to expand and engineer various cell lines for use in next generation cell and gene therapies, announced today that it has submitted a Type II Drug Master File (DMF) with the U.S. Food and Drug Administration (FDA) for UM171.
MONTRÉAL, June 17, 2022 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), an advanced clinical-stage biotechnology company delivering molecules and bioengineering solutions to expand and engineer various cell lines for use in next generation cell and gene therapies, announced today that it has submitted a Type II Drug Master File (DMF) with the U.S. Food and Drug Administration (FDA) for UM171, a proprietary molecule being studied for use in the expansion and rejuvenation of hematopoietic stem cells.
UM171 is a small molecule that leads to specific epigenetic modifications which have a profound impact on hematopoietic stem cell self-renewal. UM171 has been cited in several scientific publications, and its mechanism of action was published in 2021 in the peer-reviewed journal, Cell Stem Cell. UM171 is also a first-in-class activator of a novel E3 ligase complex which plays a role in the degradation of several proteins, including LSD1, a well-known target in cancer therapy.
ExCellThera has already shown the immense clinical potential of UM171 through ECT-001 Cell Therapy, which has been evaluated in over 90 adult and pediatric patients with severe blood disorders in several clinical trials in the United States and Canada (with additional clinical trials in Europe expected soon). In addition, last year, ExCellThera announced it had entered into a licensing agreement with Astellas Pharma Inc. through its U.S. subsidiary, Astellas Institute for Regenerative Medicine, for the use of UM171 in the separate field of pluripotent stem cells (PSCs), providing further opportunities to explore the potential of UM171 to make an impact in stem cell research and therapies.
ExCellThera has developed pre-formulated commercial-grade UM171 single-use vials that can be integrated into the manufacturing process of various cell and gene therapy products as a reagent.
“While ExCellThera is focusing on developing ECT-001 Cell Therapy to treat certain blood cancers, we believe this DMF will assist our current and future partners and collaborators in further advancing the immense clinical and commercial potential of UM171 in additional fields of development,” said Guy Sauvageau, ExCellThera’s Co-CEO and Chief Scientific Officer.
“With this DMF, we are even better positioned to licence and supply UM171 to our current and future partners and collaborators in the field of cell and gene therapy, where the quantity and functionality of stem cells and other cell lines is often a limiting factor,” said David Millette, ExCellThera’s Co-CEO and Chief Operating Officer.
About a Drug Master File (DMF)
A Drug Master File (DMF) is a submission to the Food and Drug Administration (FDA) that may be used to provide confidential detailed information about facilities, processes or materials used in the manufacturing, processing, packaging, and storing of human drugs and biologics. The information contained in a DMF is used to support, among other applications, an Investigational New Drug Application (IND).
About ExCellThera
ExCellThera is a clinical-stage cell expansion and engineering company delivering molecules and bioengineering solutions to expand and engineer various cell lines for use in novel one-time curative therapies for patients with hematologic malignancies and other diseases. excellthera.com
Contact: David Desjardins Vice President, Business and Corporate Development david.desjardins@excellthera.com
