On April 23, 2019 ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported 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 (Press release, ExCellThera, APR 23, 2019, View Source [SID1234535340]). 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.

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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," said Dr. Guy Sauvageau, CEO and founder of ExCellThera. "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."

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.

About ECT-001
The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology, capable of expanding the number of stem and immune cells exponentially in as little as seven days, is used in novel curative cord blood transplant therapies for patients with blood cancers, allowing rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients.