AvenCell to Present New Data from Lead Universal CAR T Cell Therapy Program at 64th American Society of Hematology Annual Meeting and Exposition

Oral presentation of updated safety and efficacy data for AVC-101 in relapsed/refractory acute myeloid leukemia

Achieves clinical proof of concept with efficacy signal in late-line r/r AML patients and demonstrates rapid CAR T Cell on-off control mechanism of Universal Targeting platform

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AvenCell Therapeutics, Inc. announced today that expanded data from its leading Universal CAR T Cell (“UniCAR”) Therapy program will be revealed in an oral presentation at the 64^th American Society of Hematology (ASH) Annual Meeting and Exposition on December 10-13, 2022, in New Orleans, Louisiana.

Expanded safety and efficacy data on AVC-101 (UniCAR CD123), an investigational, controllable CD123-targeted chimeric antigen receptor (CAR) T cell product in relapsed/refractory acute myeloid leukemia, will be presented as clinical proof of concept of our Universal Targeting platform that allows for rapid on-off control of CAR T cells.

Oral Presentation [#979]: Phase 1 Dose Escalation Study of the Rapidly Switchable Universal CAR-T Therapy Unicar-T-CD123 in Relapsed/Refractory AML

Presenting Author: Dr. Gerhard Ehninger, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany

Date/Time: Monday, December 12, 2022, 4:30 PM CST

This presentation will include updated data from the first-in-human phase 1 dose escalation study of AVC-101 in patients with R/R AML, designed to assess safety and tolerability and identify an MTD. Secondary and exploratory objectives include efficacy, biological activity and PK.

The abstract reports results as of July 7, 2022 for 14 heavily pre-treated R/R AML patients with a median of three prior lines of therapies (min-max: 1-7 lines), and four patients had received a prior allogeneic hematopoietic stem cell transplantation.

Abstract outlining AvenCell’s accepted data at ASH are available on the ASH conference website.

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 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 Targeting Module infusion provides extensive flexibility to address such issues as avoiding T cell exhaustion and reacting quickly to any potential adverse events.

AVC-101 is not approved for any indication in any geography.

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 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.

Contacts

Helen Kepets

[email protected]