MONTREAL, May 14, 2018 – ExCellThera Inc., a biotechnology company delivering molecules and bioengineering solutions to expand blood stem cells for therapeutic use, announced today that its lead platform, ECT-001, will be used as part of a new Phase I/II clinical trial in patients with multiple myeloma. The study will enroll up to 20 high-risk, multiple myeloma patients from the Hôpital Maisonneuve-Rosemont in Montreal, Canada.

ECT-001 will be used in this trial to expand blood stem cells from the umbilical cord, which have been shown to have higher tolerance for HLA mismatches than stem cells in an allogeneic transplant (from a family member or unrelated donor). This higher tolerance greatly reduces the risk of chronic graft-vs-host-disease, a serious complication of allogeneic transplantation, associated with infections, poor quality of life and even mortality. In addition, cord blood appears to have better anti-tumor activity with less chance of relapse of the underlying cancer when compared to other stem cell sources. However, cord blood use has been limited by its low cell dose, causing a prolonged period of blood cell recovery during which time a patient is susceptible to infection. ECT-001 allows safe and rapid expansion of cord blood units to enable rapid blood cell recovery for patients, thereby making cord blood better suited for transplant.

“Even though cord blood is known to have several advantages, the challenge that has prevented its widespread use as a curative therapy for illnesses like multiple myeloma is the small number of viable stem cells available in a single cord blood unit,” says Dr. Guy Sauvageau, CEO and Chief Scientific Officer of ExCellThera. “Our cell expansion platform can expand these life-giving cells more than 35 times, providing sufficient quantities for treatment with better HLA-matched cord blood that would otherwise not have enough stem cells for treatment purposes. We’re very excited by the prospect of ECT-001 being further validated in this study.”

The clinical trial is supported by ExCellThera, along with the Stem Cell Network and the Université de Montréal Maryse and William Brock Chair in Applied Research into Stem Cell Transplantation.

This is the second of a series of clinical trials using ECT-001 to be initiated in patients with severe blood disorders. In an ongoing first in-human trial using cord blood expanded with the ECT-001 platform, 22 patients with blood cancers from the Hôpital Maisonneuve-Rosemont have received a transplant and results to date have shown the procedure to be safe and feasible.

About multiple myeloma
The second most common blood cancer in Canada, multiple myeloma is considered incurable, with an average life expectancy of 5-6 years. In multiple myeloma, the cancer attacks a specific type of white blood cell called plasma and eventually targets several organs including bone, the bone marrow and kidney. Currently, the only curative option for this disease is an allogeneic stem cell transplant (from a family member or unrelated donor), a procedure that is associated with serious side effects, the most significant being chronic graft-vs-host disease, affecting 80% of patients. In addition, patients also face a 50% chance of disease relapse.

About ECT-001
The ECT-001 platform is a combination of a small molecule, UM171, and an optimized culture system. The platform is capable of expanding the number of stem and immune cells in a single unit of cord blood for therapeutic purposes in as little as seven days, twice as fast as any other solution. ExCellThera holds the exclusive rights to molecule UM171, initially discovered at the Institute of Research in Immunology and Cancer at the Université de Montréal.

About ExCellThera Inc.
ExCellThera is a biotechnology company delivering molecules and bioengineering solutions to expand blood stem cells for therapeutic use. ExCellThera’s lead solution, ECT-001, 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.

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