ROAD TO THE CURE UPDATE JANUARY 2018 · Parkinson's Resource Organization

ROAD TO THE CURE UPDATE JANUARY 2018

Category: Road to the Cure

ICB International, Inc., (“ICBII”) Files Orphan Drug Designation Application for Multiple System Atrophy (MSA) with the FDA

MSA is a rapidly progressive neurodegenerative disease with a mean survival of 6-10 years post diagnosis. Disease onset is usually in the sixth decade with an annual worldwide prevalence of 1.9 to 4.9 in every 100,000 people. Together with progressive supranuclear palsy (PSP), dementia with Lewy bodies (DLB) and corticobasal degeneration (CBD), MSA is an atypical Parkinsonian disorder.

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. Two phenotypes, MSA-P and MSA-C, have been described clinically by the underlying motor dysfunction. In the US and Europe, 66% to 82% of MSA patients have Parkinsonian features (MSA-P) with neurodegenerative lesions commonly occurred in the striatonigral (SN) and basal ganglia, whereas cerebellar ataxia (MSA-C) due to olivopontcerebellar atrophy is more prominent (67%) in Japanese populations.

MSA Clinical Presentation: MSA 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). These patients respond poorly to L-DOPA and have a poor prognosis as compared to Parkinson’s disease (PD) patients.

MSA-P is characterized by bradykinesia, muscle rigidity, irregular jerky tremor and postural instability (Parkinsonism) and autonomic nervous system failures in the form of bladder dysfunction, orthostatic hypotension, rapid eye movement (REM) sleep disorder (RBD), constipation and sexual dysfunction. Symptoms of MSA may vary depending upon which form of MSA predominates. The disorder can cause the progressive loss of motor skills and results in more than 50% of MSA-P patients wheelchair-bound within 5-6 years of the onset of motor symptoms. Eventually, affected individuals may become bedridden and experience life-threatening complications.

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.

Diagnosis and Treatment of MSA Patients: There are currently no diagnoses and/or treatments available for MSA because the blood-brain barrier (BBB) has long stymied the entrance of most pharmaceuticals into the central nervous system (CNS) making it impossible to diagnose and modify brain diseases such as Alzheimer’s (AD), Parkinson’s (PD), Multiple System Atrophy (MSA), Glioblastoma (GB), and many others. Realizing that brain diseases cannot be cured and/or modified unless the drug reaches the CNS, the scientists at ICB International, Inc., (ICBII), made it a priority to solve the problem of the impermeability of the BBB, thereby creating an opportunity to meet the unmet medical need for CNS diseases. ICBII has developed proprietary antibody mimics, referred to as SMART Molecules (SMs), which have been in animal models to:

Detect and quantitate the levels of pathological protein in the brain of Alzheimer’s and Parkinson’s mice;

Inhibit expression of the pathological protein in the brain of Alzheimer’s and Parkinson’s animals in a dose dependent manner.

Compared to the current drugs available for treating AD and PD that provide short-term symptomatic relief, ICBII’s SMs have the potential to diagnose and alter (stop disease progression) the course of the CNS diseases.

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 its α-synuclein-SMART Molecule (α-Syn-SM), a novel antibody mimic, in the treatment of Multiple System Atrophy (MSA). MSA-P has been designated an orphan disease. Accordingly, ICBII has requested that the FDA grant the α-Synuclein-SM for MSA Orphan Drug Status.

“With this Orphan Drug Designation, hopefully the FDA will fast track the 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 MSA, Parkinson’s, Alzheimer’s, Glioblastoma, and other CNS diseases,” says Ram Bhatt PhD, CEO/CSO of ICBII.

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Updated: August 16, 2017