Rare Daily Staff

The U.S. Food and Drug Administration granted Fast-Track designation under its Expedited Program for Serious Condition to Smart Immune’s experimental allogeneic T cell progenitor product SMART 101 for patients with the rare cancers acute lymphocytic leukemia and acute myelocytic leukemia who are undergoing hematopoietic stem cell transplantation.

The agency also accepted the company’s Investigational New Drug application SMART 101, or ProTcell, the first of any T cell progenitor product in the United States, according to the company. The agency previously granted Orphan Drug designation for ProTcell as a treatment to enhance cell engraftment in patients receiving hematopoietic stem cell transplant (HSCT). The orphan drug status encompasses various conditions such as hematologic malignancies like AML and ALL, along with all forms of primary immunodeficiencies including, but not limited to severe combined immunodeficiency (SCID).

Smart Immune expects to start its clinical development program in the United States in the fall of 2021 to investigate ProTcell as an allogeneic, non-engineered T cell progenitor medicine in diseases where overall survival and outcome is highly dependent on immediate immune reconstitution after HSCTs, and potentially as engineered T cell therapy.

The ALL/AML IND encompasses a clinical trial across adult and pediatric leukemia patients receiving T cell depleted allo-HSCT and will initially enroll up to 36 patients. In this phase 1/2 clinical trial, safety and efficacy of ProTcell to rapidly give rise to T cell compartment in order to reduce infections, graft versus host disease, and eventually also 1-year non relapse mortality, will be assessed. If successful in clinical development, ProTcells could make human leucocyte antigen (HLA) mismatched transplants as successful as HLA-identical, with the benefit of rapid immune-reconstitution and patient recovery and discharge.

The U.S. Smart Immune sponsored adult and pediatric leukemia studies will run in addition to the ongoing European studies of allogeneic ProTcell for the rare, pediatric indication of severe-combined immunodeficiency (SCID) where babies are born without T cells and the ProTcell therapy in combination with a haploidentical HSCT will allow a rapid and durable, long-term immune-reconstitution for patients lacking an HLA-identical donor; and for relapsed/refractory AML where ProTcell will be derived from umbilical cord blood (UCB) – CD34+ hematopoietic stem cells  and administered together with umbilical cord blood with the aim of demonstrating that the company’s biomimetic thymus is independent of the source of such CD34+ HSCs in its ability to reliably generate therapeutic ProTcell.

““As we treat very sick patients and hope to durably reconstitute their fragile immune systems, we plan to be cautious and measured in our development path starting with establishing unequivocal clinical proof of the efficacy and safety of our ProTcell that are devoid of any genetic engineering in this first phase of our development,” said Marina Cavazzana, physician and co-founder of Smart-Immune. “Such proof can then pave the way for a more expedited clinical development of genetic engineered ProTcell in the future.”

Photo: Marina Cavazzana, physician and co-founder of Smart-Immune

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