Preclinical programs for the treatment of type 2 diabetes and heart failure are based on Ambrx’ amino acid-based protein modification platform.
Bristol-Myers Squibb (BMS) is paying Ambrx $24 million up front for exclusive worldwide rights to develop and commercialize biologics based on the latter’s fibroblast growth factor 21 (FGF-21) protein for the treatment of type 2 diabetes, and relaxin hormone for potential applications in treating heart failure. Under terms of the deal the firms have initiated a research collaboration for both programs. Ambrx could also receive development milestones and royalty payments.
Ambrx claims preclinical studies suggest relaxin may aid in the treatment of heart failure by improving cardiac function. FGF-21, meanwhile, is a naturally occurring metabolic regulator, which preclinical studies have shown lowers blood glucose, elevates good cholesterol, and promotes weight loss. Lead FGF-21 candidate, ARX618 (PEG-FGF-21), is in the final stages of preclinical development.
Both the FGF-21 derivatives and relaxin product have been developed using Ambrx’ ReCODE™ (reconstituting chemically orthogonal directed engineering) platform for the site-specific placement of chemically-specified amino acids into proteins. The platform is being exploited both for the development of novel proteins and for the design of improved versions of already marketed products.
The firm’s pipeline includes both in-house and partnered protein and antibody-drug conjugate candidates. Lead program, to which EMD Serono has exclusive worldwide commercialization rights, is ARX201, a long-acting human growth hormone (hGH) currently in Phase II clinical trials. EMD Serono also has worldwide exclusive rights to a second candidate, ARX424, which is in preclinical development as a potential treatment for multiple sclerosis.
Ambrx in addition has preclinical-stage partnered programs in place with Pfizer and Lilly. Ambrx’ in-house pipeline includes preclinical candidates for the treatment of obesity and cancer.