FDA Approves Autolus' Obecabtagene Autoleucel (Aucatzyl) for R/R B-cell ALL

Key Insights

• The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL).
• Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months.
• Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.

The FDA has granted approval to Autolus Therapeutics' obecabtagene autoleucel (Aucatzyl), also known as obe-cel, a CD19-directed genetically modified autologous T-cell immunotherapy. This approval marks a significant advancement in the treatment of adult patients with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).

The approval was supported by data from the pivotal Phase 1b/2 FELIX clinical trial (NCT04404660). The trial demonstrated promising efficacy, with overall complete remission (CR) rates exceeding 60%. Furthermore, patients who achieved complete remission within three months exhibited a median duration of remission beyond 12 months, highlighting the potential for durable responses with obe-cel.

CARsgen's CAR-T Therapies Advance

The FDA has lifted clinical holds on multiple CARsgen Therapeutics' CAR-T therapies, including zevorcabtagene autoleucel (zevor-cel), a BCMA-directed CAR-T for relapsed/refractory multiple myeloma (MM), and satricabtagene autoleucel (satri-cel), a Claudin18.2-directed CAR-T for gastric/pancreatic cancers. This decision allows CARsgen to proceed with clinical trials evaluating these therapies, offering potential new treatment options for patients with these cancers.

Other Cell and Gene Therapy Updates

• A patient death occurred in Beam Therapeutics' BEACON trial (NCT05456880) evaluating BEAM-101 for sickle cell disease (SCD). The death was attributed to the busulfan-conditioning regimen, not the BEAM-101 therapy itself.
• Neurogene's NGN-401 for Rett syndrome showed positive efficacy in a Phase 1/2 trial (NCT05898620), but a serious adverse event was reported in the high-dose cohort.
• Abeona Therapeutics' BLA for prademagene zamikeracel (pz-cel; EB-101) for recessive dystrophic epidermolysis bullosa has been accepted by the FDA, with a PDUFA date of April 29, 2025.
• Atamyo Therapeutics' ATA-200 for LGMD2C/R5 has received IND clearance, enabling a Phase 1b trial (NCT05973630).
• Nippon Shinyaku has acquired commercialization rights for Atsena Therapeutics’ ATSN-101 for Leber Congenital Amaurosis (LCA1) in the US and Japan.