Bellicum Pharmaceuticals Inc. (NASDAQ: BLCM) shares saw a solid gain early Tuesday after the company announced interim clinical data of BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies.

BPX-501 is an adjunct T cell therapy incorporating CaspaCIDe administered after haploidentical hematopoietic stem cell transplant (haplo-HSCT) for the treatment of hematologic cancers and inherited blood diseases.

Data from the ongoing BP-004 trial suggest that BPX-501 T cells may contribute to a durable anti-leukemic effect in patients with AML.

In the study, 38 pediatric AML patients in their first or second complete response underwent a haplo-HSCT followed by treatment with BPX-501. With a median follow-up of one year, rates of relapse-free survival and overall survival were 91.5% and 97.3%, respectively. This compares to one-year rates reported in the literature in pediatric AML patients undergoing alternate-donor HSCT of 60% to 80%.

Going forward, Bellicum will be working with the investigators and the U.S. Food and Drug Administration (FDA) to develop a protocol for a potential U.S. registration study in pediatric patients. Pending regulatory clearances, Bellicum expects to initiate the study by the end of 2018.

Neena Kapoor, M.D., director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and an investigator in the BP-004 trial, commented:

The recurrence of cancer is one of the most serious risks to AML patients receiving a stem cell transplant. These impressive results in children with AML suggest that BPX-501 may be effectively reducing or eradicating residual cancer cells following a haplo-transplant procedure.

Shares of Bellicum were last seen up about 4% at $8.36, with a consensus analyst price target of $16.50 and a 52-week range of $5.02 to $15.55.

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