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ICBII submits orphan drug designation application to US FDA for use of a-synuclein-SMART molecule to treat multiple system atrophy

La Jolla, California
Thursday, December 7, 2017, 14:00 Hrs  [IST]

Innovative California Biosciences International Inc. (ICBII) has announced on November 20th the submission of their Orphan Drug Designation application to the US Food and Drug Administration (FDA) for the use of a-synuclein-SMART Molecule (a-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 a-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.  Accordingly, ICBII has requested that the FDA grant the a-Synuclein-SM for MSA Orphan Drug Status.

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 aberrant proteins in animals. (SMART* stands for Specific Molecular Architecture for Recognition and Therapy. The Company has developed a SMART Molecule for a-synuclein, which, when administered intravenously, allows quantitation of aberrant a-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 approval process of a-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 MSA, Parkinson's, Alzheimer's, Glioblastoma, and other CNS diseases," stated Ram Bhatt PhD, CEO/CSO of ICBII.

ICBII was founded for the sole purpose of addressing unmet medical needs for CNS diseases.  The BBB 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.


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