ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, has announced that the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to its lead technology, ECT-001, in the treatment of hematologic malignancies. The RMAT designation is based on strong data from Phase I/II clinical trials using ECT-001 to expand stem and immune cells for the treatment of blood cancers.
The company was founded in 2015 and is located in Montréal, Canada. ExCellThera’s lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials.
Various clinical studies using ECT-001 are currently ongoing in the treatment of multiple myeloma, high-risk leukemia and other hematologic malignancies. In addition, ExCellThera plans to initiate additional clinical trials, including a pivotal trial in the United States and Canada, in the coming months. ECT-001 has also received FDA orphan drug designation for the prevention of graft-versus-host disease.
RMAT designation is granted by the FDA under the 21st Century Cures Act for cell therapies, tissue-engineered or similar products intended to treat or cure a serious disease, and which demonstrate preliminary evidence to address an unmet clinical need. It accords all the benefits of the FDA’s fast track and breakthrough therapy designation programs, including an ability to interact with the agency to discuss the potential acceleration of regulatory approval. Under the auspices of the RMAT designation, FDA will work closely with ExCellThera and provide advice on generating the evidence needed to support approval of ECT-001 in an efficient manner.
“The FDA’s RMAT designation is a clear signal of confidence in the potential of our lead cell therapy drug product, ECT-001, to treat patients with hematologic malignancies.” … “We look forward to working with the FDA within the RMAT framework to advance ECT-001 through the final phases of clinical development in an expedited manner.” – Dr. Guy Sauvageau, CEO and founder of ExCellThera