Montreal, QC (Press Release) – ExCellThera Inc., an ad­vanced clin­i­cal stage bio­technology com­pany delivering molecules and bio­engineer­ing solu­tions to expand stem and immune cells for thera­peutic use, announced today 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.

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.

About ExCellThera Inc.

ExCellThera is an ad­vanced clin­i­cal stage bio­technology com­pany delivering molecules and bio­engineer­ing solu­tions to expand stem and immune cells for use in novel one-time curative ther­a­pies for patients with hema­to­logic malig­nan­cies, auto­immune and other diseases. 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. excellthera.com

Source: ExCellThera.