Proclara Biosciences Presents Preclinical Data Supporting the Development of GAIM-Based Therapies for Peripheral Amyloidoses at the 16th International Symposium on Amyloidosis

• GAIM-lg fusions, including NPT189, specifically and potently bind recombinant and patient-derived LC and TTR amyloid fibers;
• Systemic administration of NPT189 results in co-localization to amyloid organ deposits in animal models of AL amyloidosis;
• GAIM-Ig fusions, including Proclara’s research molecule NPT288, mediates disruption of amyloidoma tissue consisting of human amyloid LC patient-derived fibers;
• Together, these data support the development of GAIM fusions, including NPT189, as therapeutic candidates for treating peripheral amyloidoses.

About Orphan Amyloidoses

Peripheral amyloidoses are life threatening diseases caused by the systemic buildup of amyloid protein fibers in organs and nerves. Over time, this build-up results in dysfunction that leads to significant morbidity and increased mortality. Current treatments are limited, and new, more efficacious treatments are badly needed. Peripheral amyloidoses are classified according to the type of deposited misfolded protein. Amyloid light chain (AL) amyloidosis is caused by an accumulation of misfolded immunoglobulin protein resulting in vital organ dysfunction, with up to 4,000 new cases diagnosed in the U.S. each year.¹ Hereditary transthyretin (ATTR) amyloidosis, which is caused by mutant transthyretin (TTR) amyloid deposits in multiple organs, causes significant morbidity and mortality within two to 15 years from symptom onset and affects approximately 50,000 people worldwide.^2,3 Proclara is developing GAIM therapies that target LC and TTR deposits for the treatment of these two orphan peripheral amyloidoses.

1. Amyloidosis Research Consortium, Guidance for Industry – AL Amyloidosis – Developing Drugs for Treatment, Dec 2016 (www.arci.org)
2. Ando et al., Orphanet J Rare Dis, 2013
3. Ruberg et al., Circulation, 2012

About Proclara Biosciences

Proclara Biosciences is a clinical stage biotechnology company advancing product candidates based on its proprietary GAIM technology, which is capable of simultaneously targeting multiple toxic misfolded protein aggregates. The broad applicability of GAIM technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and orphan systemic amyloidoses. Proclara has developed a pipeline of drug candidates that use GAIM to target the common amyloid protein conformation, dissociating and preventing the formation of misfolded protein assemblies, and blocking the cell-to-cell transmission of toxic protein aggregates. The company’s lead program NPT088 is currently being evaluated in a Phase 1b trial for Alzheimer’s disease.

For more information, please visit proclarabio.com.

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