AvenCell Presents Safety and Efficacy Data from Lead CD123-Directed Universal CAR T Cell Therapy Program at ASH 2022 Meeting

By: AvenCell Therapeutics, Inc. via Business Wire

December 12, 2022 at 17:44 PM EST

Oral presentation describing clinical proof of concept of AVC-101, a CD123-directed Universal CAR T in relapsed/refractory acute myeloid leukemia, demonstrating favorable side effect profile and rapid on/off control

Early efficacy signal with complete remissions and MRD negative conversions observed in heavily treated r/r AML patients

AVC-101 utilizes AvenCell’s autologous Universal CAR T Platform and is also being further investigated with allogeneic cells

AvenCell Therapeutics, Inc. today announced updated safety and efficacy data from its lead CD-123-directed Universal CAR T Cell Candidate (“UniCAR”). These data, based on AvenCell’s Universal Targeting platform, were presented at the 64^th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans, Louisiana.

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 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.

About AvenCell

AvenCell derives its name from the French word avenir to reflect that we are the FUTURE of cell therapy. We are building a truly transformative cell therapy company that focuses difficult-to-treat cancers, with our lead programs focusing on acute myeloid leukemia (AML) and prostate cancer, and additional programs targeting other hematological malignancies and solid tumors. We formed AvenCell with the goal of 1) the creation of truly allogeneic cells that persist as long or longer than autologous therapies and 2) a universal and switchable construct that allows complete control and target redirection of T cells after they are infused into a patient. Integration of these two platforms allows for complete separation of the manufacturing of cells from ultimate patient and cancer target, thus providing significant scalability potential at orders of magnitude more efficient than current cell therapy approaches.

AvenCell Therapeutics, Inc. was launched in 2021 by Blackstone Life Sciences, Cellex Cell Professionals, and Intellia Therapeutics and incorporated the clinical-stage biopharmaceutical company GEMoaB GmbH. AvenCell is headquartered in Cambridge, Massachusetts with additional R&D and manufacturing operations in Dresden, Germany.

For more information, visit www.avencell.com.

Follow AvenCell on social media: LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements including AvenCell’s beliefs and expectations regarding the potential safety and efficacy of AVC-101, the potential of its platform technologies to treat acute myeloid leukemia and other types of hard-to-treat cancer, and the functioning, scalability and efficiency of its platform technologies.

Forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including AvenCell’s ability to initiate, progress or complete pre-clinical or clinical studies within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing or additional pre-clinical or clinical studies; uncertainties relating to regulatory applications and related filing and approval timelines; risks related to AvenCell’s relationships and collaborations with third parties; risks related to the maintenance and protection of AvenCell’s intellectual property; risks related to the potential successful development and commercialization of AvenCell’s product candidates; and risks associated with AvenCell’s understanding of cell therapy and its application to the treatment of cancers.

All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to AvenCell, and AvenCell assumes no obligation and disclaims any intent to update any such forward-looking statements.