On December 12, 2022 AvenCell Therapeutics, Inc. reported updated safety and efficacy data from its lead CD-123-directed Universal CAR T Cell Candidate ("UniCAR") (Press release, AvenCell Therapeutics, DEC 12, 2022, View Source [SID1234625187]). These data, based on AvenCell’s Universal Targeting platform, were presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in New Orleans, Louisiana.
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The oral presentation (#979) included updated data from the first-in-human phase I dose escalation study of AVC-101 in patients with relapsed/refractory acute myeloid leukemia (r/r AML) designed to assess safety and tolerability and identify an MTD. Secondary and exploratory objectives include efficacy, biological activity, and PK.
As of October 13, 2022, of 16 patients treated with AVC-101, five patients (31%) achieved complete response with incomplete count recovery (CRi) or MRD negative conversion and the overall response rate was 56% (n=9). Of evaluable patients with minimal residual disease (MRD+; n=2), both converted to MRD negative status after the treatment.
"The AVC-101 [UniCAR CD123] program shows extremely promising results in the most challenging AML patients who have exhausted all options," said Prof. Dr. med. Martin Wermke, head of Early Clinical Trial Unit at University Hospital Carl Gustav Carus in Dresden, Germany. "This study shows preliminary evidence of clinical efficacy including multiple CRs in the dose escalation stage, manageable toxicity, and proof of principle that the switchable technology can be turned on and off to manage toxicities thereby enabling targets too challenging for traditional CAR Ts. We are hopeful for additional trials of this platform to build on this encouraging data."
The presentation reports results from 16 heavily pre-treated R/R AML patients with a median of six prior lines of therapies (min-max: 2-9 lines), with nine patients having received a prior allogeneic hematopoietic stem cell transplantation. Median age of patients was 64.5 (range 18-80), with four patients over the age of 70.
"We are excited to be at ASH (Free ASH Whitepaper) for the first time as AvenCell and to showcase the Universal Targeting platform, which has the potential to address some of the most difficult efficacy and safety challenges with cell therapies seen to date," said Andrew Schiermeier, Ph.D., chief executive officer, AvenCell Therapeutics. "The data presented by Dr. Gerhard Ehninger is indicative of the promise of the platform to treat a multitude of difficult cancer targets, where the ability to precisely and reliably control CAR T cell activity in vivo after administration is critical."
About AVC-101
AVC-101 is an investigational CD-123-directed cell therapy targeting acute myeloid leukemia, that utilizes AvenCell’s proprietary Universal Targeting platform, a regulatable CAR T cell technology that can turn CAR T cells "OFF" and "ON" by means of a separately infused Targeting Module. With AVC-101, AvenCell is aiming to create a solution to address the heterogeneity and aggressive nature of acute myeloid leukemia. CD123 is a target in acute myeloid leukemia, but its on-target off-tumor toxicity makes a conventional CD-123-directed CAR very challenging.
AvenCell’s proprietary Universal Targeting Platform is part of a new paradigm in cell therapy through one-time engraftment of Universal CAR T Cells, which can then be controlled in vivo with the separate administration of Targeting Modules that direct the Universal CAR T cells to cancer cells for activation and killing. The platform’s ability to control CAR T activity after infusion via repeatable, titratable, and switchable Targeting Module infusion provides extensive flexibility to address such issues as avoiding T cell exhaustion, antigen escape, and reacting rapidly to any potential adverse events.
AVC-101 is not approved for any indication in any geography and has not been demonstrated safe or effective for any use.