Investigational new drug status and fast track designation granted by FDA to SMART 101, a progenitor T-cell product for AML and ALL

The U.S. Food and Drug Administration (FDA) announced on May 10, 2021, that SMART 101, an allogeneic T-cell progenitor product for the treatment of patients with acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), had been accepted as an investigational new drug (IND). SMART 101 is the first T-cell progenitor product to received IND status in the U.S. In addition, SMART 101 was given fast track designation under the FDA’s Expedited Program for Serious Conditions.¹

SMART 101 has been previously granted orphan drug designation by the FDA for the treatment of patients with hematologic malignancies such as AML, ALL, and all primary immunodeficiencies, including severe combined immunodeficiency (SCID).

As part of the IND status, a phase I/II clinical trial with adult and pediatric patients with AML and ALL who are receiving T-cell depleted allogeneic hematopoietic stem cell transplantation will be set up in the U.S. This trial will enroll up to 36 patients and hopes to commence by fall 2021. The primary goal of this study will be to assess the safety and efficacy of SMART 101 at increasing the T-cell population within patients. SMART 101 aims to reconstitute T-cell populations preventing infections and graft-versus-host disease, which should in turn have a positive impact on 1-year non-relapse mortality.

SMART 101 is also currently being tested in European studies for:

• A form of SCID, in which infants are born without T cells.
• Relapsed/refractory AML, in which umbilical cord blood CD34⁺ hematopoietic stem cells will be used to derive SMART 101.

SMART 101 has the potential to make human leucocyte antigen (HLA)-mismatched transplants as successful as HLA-matched transplants, which would allow for rapid immune reconstitution and patient recovery.