ICBII Announces the Filing of Orphan Drug Designation Application for the Use of α-Synuclein-SMART Molecule in the Treatment of Multiple System Atrophy

By December 6, 2017Press Releases

La Jolla, California, USA, December 4, 2017 (PR Newswire): Innovative California Biosciences International, Inc., (“ICBII”), announced on November 20th the submission of their Orphan Drug Designation application to the US Food and Drug Administration (FDA) for the use of α-synuclein-SMART Molecule (α-Syn-SM), a novel antibody mimic, in the treatment of Multiple System Atrophy (MSA).

MSA is a rapidly progressive neurodegenerative disease with a mean survival of 6-10 years post diagnosis. This disease arises from the misfolding and accumulation of the protein α-synuclein in oligodendrocytes, where it forms glial cytoplasmic inclusions killing neurons and glial cells. Based on current census, prevalence, and National Institute of Health (NIH) data, there are estimated to be fewer than 15,000 to 50,000 MSA patients in the US. In the US and Europe, 66% to 82% of MSA patients have parkinsonian features (MSA-P). MSA-P has been designated an orphan disease.  According, ICBII has requested that the FDA grant the α-Syn-SM for MSA Orphan Drug Status.

MSA is a seriously debilitating disease for which there is no cure. It is characterized by a variable combination of Parkinsonism, cerebellar impairment, and autonomic dysfunction. It encompasses three presentations of a single disease, specifically Shy-Drager syndrome (which emphasized autonomic dysfunction), striatonigral degeneration (which emphasized parkinsonian symptoms), and sporadic olivopontocerebellar atrophy (which emphasized cerebellar symptoms).

About SMART Molecules

SMART Molecules (SMs) are novel antibody mimics that cross the blood-brain barrier (BBB) into the central nervous system (CNS) where they selectively target and inhibit the expression of pathological proteins in animals. SMART is an acronym that stands for Specific Molecular Architecture for Recognition and Therapy. The Company has developed a SMART Molecule for α-synuclein, which, when administered intravenously, allows quantitation of aberrant/misfolded α-synuclein in the brain of treated mice by a brain scan and halts its expression in a dose dependent manner.

“With this Orphan Drug Designation, hopefully the FDA will fast track the development and approval process of   α-Syn-SM in the treatment of MSA-P and related Parkinson’s disorders. It will be a major advancement in moving the SMART Molecules technology platform forward towards treating Parkinson’s, MSA, Alzheimer’s, Glioblastoma, and other central nervous system Diseases,” stated Ram Bhatt PhD, CEO/CSO of ICBII.

ABOUT ICBII The Company was founded in October of 2008 for the sole purpose of addressing unmet medical needs related to the diseases of the central nervous system.  The blood brain barrier has effectively excluded the brain from the entire biotech revolution.  With its SMs technology ICBII’s mission is to impact the lives of millions of patients worldwide by ameliorating their suffering. ICBII has currently two patents issued and seven patents pending with several more ready to be filed.

Forward Looking Statements

This press release may contain forward-looking statements, including ICBII’s development of SMART Molecules™ for altering the course of MSA and other α-synucleinopathies.  Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from those, express or implied, in these forward-looking statements. ICBII expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in ICBII expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based for any reason, except as required by law, even as new information becomes available or other events occur in the future. All forward-looking statements in this press release are qualified in their entirety by this cautionary statement.

Contact Information: info@icbii.com

Investor Relations: Ken Evans, kevans@icbii.com

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