- AB001 is an engineered high-affinity soluble BCMA decoy receptor that traps APRIL and BAFF, critical cytokines controlling B cell development and survival

- AB001 effectively inhibits the development of Multiple Myeloma and Diffuse Large B Cell Lymphoma via modulating protein translation machinery

- AB001 demonstrated impressive safety profile and on-target mechanism of action in non-human primates

LOS ANGELES & LONDON & BEIJING--(BUSINESS WIRE)--AKSO Biopharmaceutical, Inc. (�AKSO�), a global biopharmaceutical company with an innovative and comprehensive approach to treat cancer, autoimmune disease, and genetic disorder, announced the publication in the Journal of Experimental Medicine of preclinical data demonstrating that AKSO�s late preclinical stage molecule AB001 effectively inhibits growth of Multiple Myeloma (�MM�) and Diffuse Large B Cell Lymphoma (�DLBCL�) in mice. Importantly, AB001 was also found to be nontoxic in non-human primates.

�AB001 is a first-in-class and potentially best-in-class ligand trap targeting APRIL and BAFF, which are known to be difficult to sequester due to high native binding affinities to their receptors, but also critical regulators of B cell development and survival,� said Professor Amato Giaccia, AKSO�s Chairman and Senior Scientific Strategist. �Our study demonstrated the novel mechanism of APRIL and BAFF in modulating protein translation; and suggest that engineered soluble BCMA could be a superior alternative to therapeutics either approved or in development for MM and DLBCL.�

�Safe and effective targeted therapies are urgently needed for MM and DLBCL patients who exhaust currently available treatment options,� said Dr. Rebecca Miao, AKSO�s Chief Scientific Officer. �Collectively, our data support AB001 as a clinically viable candidate for the treatment of MM and DLBCL. Furthermore, the biological functions of BAFF and APRIL are not limited to B cell malignancies but extend to autoimmune disorders and other diseases triggered by pathological B cells, indicating a broad clinical application of AB001.�

Key findings from the study performed under the guidance of Professor Giaccia and Dr. Miao, reported in the publication titled �Developing High-Affinity Decoy Receptor Optimized for Multiple Myeloma and Diffuse Large B Cell Lymphoma Treatment�, include:

• APRIL and BAFF mediated signalling is required for MM progression;
• When compared to the wild-type soluble BCMA receptor, AB001 shows 500-fold stronger binding toward BAFF and 4-fold stronger binding toward APRIL;
• AB001 is capable of neutralizing APRIL and BAFF and their receptor activities, impacting protein translation and production in MM and DLBCL cells;
• Treatment with AB001 led to enhanced antitumor activity in a number of MM and DLBCL mouse models;
• AB001 also demonstrated an adequate toxicity profile and on-target mechanism of action in nonhuman primate studies.

ABOUT AB001

AB001 is a late preclinical stage first-in-class and best-in-class engineered soluble BCMA decoy receptor. AB001 traps APRIL and BAFF with ultra-high binding affinities thereby inhibiting ligand mediated activation of receptors BCMA, BAFF Receptor and TACI.

ABOUT AKSO

AKSO Biopharmaceutical, Inc., is a late preclinical stage global biopharmaceutical company focused on the discovery and development of first-in-class therapeutics for the treatment of cancer, autoimmune diseases, and genetic disorder. Leveraging the proprietary DEEP engineering platform and deep knowledge in disease biology, AKSO has developed a portfolio of innovative drug candidates covering a wide range of modalities including decoy receptors, bispecific fusion proteins, antibody-drug conjugates, and precision gene editing therapies. For additional information, please visit www.aksobio.com.

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