PGEN - Precigen posts Phase 1 data for CAR-T therapy in blood cancer

Precigen, Inc. ( NASDAQ: PGEN ), a biotech focused on gene and cell therapies, has announced Phase 1 data from its Phase 1/1b clinical study for its autologous CAR-T therapy PRGN-3006 in patients with relapsed or refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (MDS).

The data set presented at the ongoing American Society of Hematology (ASH) Annual Meeting included full results from the Phase 1 dose escalation portion of the study, which involved 26 patients (N=10 non-lymphodepletion; N=16 with lymphodepletion).

Highlights of the findings indicate that the experimental therapy was well tolerated by patients in both non-lymphodepletion and lymphodepletion cohorts with no dose-limiting toxicities. Most treatment-emergent adverse events (TEAEs) were either Grade 1 or 2.

Each cohort had one Grade 3 event of cytokine release syndrome (CRS). One in the non-lymphodepletion resolved in 24 hours and the other in the lymphodepletion cohort was later downgraded by the investigator to Grade 1.

Based on 15 evaluable patients in the lymphodepletion cohort, a single infusion of PRGN-3006 led to a decline in bone marrow blasts in 60% (9 out of 15) heavily pre-treated patients. In the non-lymphodepletion cohort, 3 out of 10 patients had Stable Disease (SD), lasting for more than three months, including more than seven months of SD for one patient.

The lymphodepletion cohort indicated an objective response rate (ORR) of 27% in patients who had a median of four prior treatments (range: 1-9).

PRGN-3006 is currently undergoing a multicenter Phase 1b dose expansion phase of the study after lymphodepletion in patients.

Precigen ( PGEN ) shares surged in April after the company announced that the FDA issued its fast-track designation for PRGN-3006 as a treatment for R/R AML.

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