CAMBRIDGE, Mass.--(BUSINESS WIRE)--Proclara Biosciences, a biotechnology company developing novel therapies for diseases caused by protein misfolding, today announced that new preclinical data supporting the development of General Amyloid Interaction Motif (GAIM)-based therapies were presented at the annual meeting of the Society for Neuroscience in Washington, D.C. The data demonstrate that GAIM-based therapies, including Proclara’s lead candidate, NPT088, and second generation candidate, NPT189, can reduce the accumulation and cell-to-cell transmission of multiple types of toxic protein aggregates in diseases caused by toxic protein buildup. NPT088 is currently in a Phase 1b trial for the treatment of patients with Alzheimer’s disease. Proclara plans to file an Investigational New Drug Application with the U.S. Food and Drug Administration for NPT189 for the treatment of orphan peripheral amyloidoses in the first half of 2018.
“We are pleased to present these results, which demonstrate that our GAIM-based therapies simultaneously target multiple types of toxic protein aggregates and prevent their cell-to-cell spread,” said Richard Fisher, Ph.D., chief scientific officer of Proclara. “This suggests that our development candidates have the potential to address the mixed pathologies that affect patients with Alzheimer’s, Parkinson’s and orphan amyloidoses across a broad clinical spectrum, improving outcomes and offering patients a meaningful new treatment option.”
Session Number: 479
Session Time: 11/14/2017 8 a.m. - 12 p.m. ET
Presentation Number: 479.01
- Over 100 GAIM variants were analyzed for improved amyloid-targeting activities as part of a structure-activity relationship program.
- New GAIM variants with greater activity fall into two categories: (1) variants with reduced interdomain interactions; and (2) variants with greater domain stability.
- Combining both structural variant categories creates optimized GAIM fusion candidates that exhibit greater potency, structural stability, and targeting specificity.
- NPT189, a second generation GAIM fusion, broadly targets light chain antibody deposits from systemic amyloidosis (AL) patient tissues, as well as specifically co-localizing in vivo to antibody light chain deposits in an AL disease mouse model.
- These data support the potential for GAIM fusions as therapeutics for a variety of protein misfolding diseases.
Session Number: 210
Session Time: 11/12/2017 1 p.m. - 5 p.m. ET
Presentation Number: 210.29
- GAIM-Ig fusion variants generated to optimize amyloid-targeting properties were tested for potency of blocking alpha-synuclein aggregate formation in a neuronal cell-to-cell transmission assay.
- GAIM-Ig fusion variant potencies in the transmission assay correlate with amyloid targeting potency, confirming the mechanism of action of GAIM.
- One fusion variant, NPT189, significantly inhibited alpha-synuclein inclusion formation that correlated with mitigating behavior deficits in a Parkinson’s disease mouse model.
- NPT189 and other selected GAIM-Ig fusions are in preclinical development.
About Proclara Biosciences
Proclara Biosciences is a biotechnology company advancing product candidates developed based on proprietary GAIM technology, which is capable of simultaneously targeting multiple toxic misfolded proteins. The broad applicability of the Proclara technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and several rare systemic amyloidoses. The lead GAIM drug candidate, NPT088, is in clinical development for the treatment of Alzheimer’s disease. For more information, please visit proclarabio.com.