Escala Therapeutics (previously known as Altamira Biosciences, Inc.), is a development stage company focused on the clinical development and commercialization of N-acetyl-D-mannosamine (ManNAc) and other therapies for orphan/ultra-orphan, rare or neglected disorders.

In July 2015, Escala Therapeutics acquired a NIH issued patent license from New Zealand Pharmaceuticals, Ltd. (NZP), for the development of ManNAc as a potential treatment of GNE Myopathy and other hyposialylation disorders. NZP, which manufactures ManNAc, will remain the exclusive global supplier of ManNAc to Escala Therapeutics. Subsequent Cooperative Research and Development Agreements were established between Escala Therapeutics and NIH.

Hyposialylation disorders are a group of pathologies associated with abnormal sialylation of tissues, characteristic of GNE Myopathy—a rare genetic disorder which causes progressive muscle-wasting and weakness.

ManNAc is an intermediary in cellular sialic acid biosynthesis generated by the GNE protein known to be involved in GNE Myopathy. Preclinical data suggest that supplementation of ManNAc to cellular models with dysfunctional GNE protein bypasses the need for GNE function and enables protein sialylation. Studies in mutant mouse models demonstrated that oral ManNAc therapy improved pathologic features of both GNE Myopathy and glomerular diseases.

The National Center for Advancing Translational Sciences (NCATS) and National Humann Genome Research Institute (NHGRI), completed two pivotal animal toxicology studies to demonstrate drug safety, and allowed successful filing of the Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). In addition, the FDA has provided orphan designation for ManNAc in GNE Myopathy. A Phase 1 clinical study was completed at the NIH Clinical Center, assessing pharmacokinetic properties and safety of ManNAc in GNE Myopathy patients ( Identifier: NCT01634750). These clinical studies demonstrated that ManNAc is safe and well-tolerated by patients and that twice-daily oral ManNAc supplementation led to significant and sustained increases in the circulating levels of sialic acid. In collaboration with NCATS and NHGRI researchers, we are currently investigating ManNAc in an open-label Phase 2 study for the treatment of GNE Myopathy ( Identifier: NCT02346461).

An IND and clinical protocol for a Phase 1 study to investigate ManNAc safety and tolerability in patients with a range of glomerular disorders (primary podocyte disorders) associated with hyposialylation has been approved by the FDA, and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Institutional Review Board (IRB). This study is currently ongoing and recruiting ( Identifier: NCT02639260).

Escala Therapeutics is also seeking continued academic and industry partnerships for the development of therapies for other orphan/ultra-orphan or rare or neglected disorders.

Escala Therapeutics