CAMBRIDGE, Mass.--(BUSINESS WIRE)--Proclara Biosciences, a clinical stage biotechnology company developing novel therapies for diseases caused by protein misfolding, today announced that preclinical data supporting the development of General Amyloid Interaction Motif (GAIM)-based therapies for the treatment of peripheral amyloidoses were presented at the 16th International Symposium on Amyloidosis (ISA) in Kumamoto, Japan. The data demonstrate that GAIM-based therapies, including NPT189, specifically target light chain (LC) and transthyretin (TTR) amyloid deposits in animal models and from systemic amyloidosis patient tissues.
NPT189, a second generation GAIM fusion protein, utilizes Proclara’s proprietary GAIM technology to target multiple toxic protein aggregates found in peripheral amyloidoses, including amyloid light chain (AL) amyloidosis and hereditary transthyretin amyloidosis (ATTR). Proclara leveraged structure-activity studies to optimize NPT189’s pharmaceutical properties. Proclara expects to initiate a Phase 1 clinical study of NPT189 in mid-2018.
“The results presented at ISA highlight the breadth of the Proclara platform and its potential to address a broad range of diseases caused by aberrant protein misfolding,” said Suzanne Bruhn, Ph.D., president and chief executive officer of Proclara. “These new data, coupled with earlier preclinical studies that demonstrate NPT189’s ability to reduce the accumulation and cell-to-cell transmission of toxic protein aggregates, suggest that NPT189 may offer a differentiated, first-in-class therapy to patients with orphan amyloidoses, for whom there are limited available options.”
Presentation Highlights:
GAIM Fusions are Therapeutic Candidates for Peripheral 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.
