Slide 1: Neural Stem Cell Technology Platform: Cell Therapy and Pharmaceuticals
Neural Stem Cell Technology Platform Cell Therapy and Pharmaceuticals September 2014 NYSE MKT: CUR
Slide 2: Safe Harbor Statement
Safe Harbor statements under the Private Securities Litigation Reform Act of 1995 : This presentation contains forward - looking statements as defined in Section 27 A of the Securities Act of 1933 as amended, and section 21 E of the Securities Exchange Act of 1934 , as amended . Such forward - looki
Slide 3: Neuralstem Technology – Cells Nurturing, Supporting and Rescuing Remaining Neurons
Neuralstem Technology – Cells Nurturing, Supporting and Rescuing Remaining Neurons • Regionally specific CNS neural stem cells: brain and spinal cord • P atents worldwide, • cGMP manufacturing • Numerous cell therapy stem cell products • Fully characterized – Expanded under defined conditions: no an
Slide 4: Single Platform Technology, Two Programs, Multiple Indications
Single Platform Technology, Two Programs, Multiple Indications Ischemic Spastic Paraplegia Amyotrophic Lateral Sclerosis Optic Neuritis Lysosomal Diseases Ischemic Stroke Alzheimer’s disease Parkinson’s disease Spinal Cord Injury Traumatic Brain Injury Huntington’s disease Glioblastoma (Brain Cancer
Slide 5: Patents
Neuralstem Patents □ U. S. Pat. No. 8,236,299 (August 2012) □ Transplantation of human n eural c ells for treatment of neurodegenerative conditions □ U.S . Pat. No. 8,058,434 (November 2011) □ Compositions to effect neuronal growth □ U.S . Pat. No. 8,030,492 (October 2011 ) □ Compositions to effect
Slide 6: Small Molecule Drug Program: First-in-class Neurogenic Small Molecule Drugs
Neuralstem Small Molecule Drug Program First - in - class Neurogenic Small Molecule Drugs □ Initially funded by U.S. DOD and NIH grants □ Patented neurogenic small molecule compounds: the ability to recruit endogenous neural stem cells to generate more new neurons in the adult brain □ Lead Drug in C
Slide 7: MDD 1b trial
MDD 1b trial • ASCP annual meeting: June 17 th • A Phase 1B, Randomized, Double - Blind, Placebo - Controlled, Multiple - dose Escalation Study Evaluating the Effects of NSI - 189 Phosphate, a Neurogenic Compound, in Patients with Major Depressive Disorder (MDD) • Marlene Freeman, Massachusetts Gene
Slide 8: NSI-189/MDD Phase Ib: All Depression Measures Show Clinically Meaningful Improvement
Neurogenic NSI - 189/MDD – Phase Ib : All Depression Measures Show Clinically Meaningful Improvement For Duration of Trial • Data Shows First - time Depression Field Findings: large and significant effect size of antidepressant activity, which continued eight weeks after treatment stopped • Large ef
Slide 9: NSI-189/MDD Phase Ib: All Cognitive Measures Show Clinically Meaningful Improvement
Neurogenic NSI - 189/MDD – Phase Ib : All Cognitive Measures Show Clinically Meaningful Improvement for Duration of Trial • In addition to its antidepressive effects, NSI - 189 showed a significant effect size in cognitive function improvement; cognitive improvement also continued for eight weeks af
Slide 10: NSI-189/MDD Phase Ib: Depressive and Cognitive Measures, qEEG
Neurogenic NSI - 189/MDD – Phase Ib : All Depressive and Cognitive Measures Show Clinically Meaningful Improvement for Duration of Trial • Quantitative EEG ( qEEG ) measurements, an electrophysical biomarker of depression, showed: – Significantly increased brain wave patterns in the hippocampal regi
Slide 11: Cell Therapy Surgical Devices: Exclusive Worldwide Licenses
Neuralstem Cell Therapy Surgical Devices Exclusive Worldwide Licenses □ Spinal Platform and Floating Cannula □ Proprietary breakthrough medical devices □ Expected to be the standard in industry and research community for intraspinal procedures □ Neuralstem holds exclusive worldwide licenses □ Design
Slide 12: NSI-566/ALS Phase I Success: Proven Safe, Signs of Efficacy
NSI - 566/ALS Phase I Success: Proven Safe, Signs of Efficacy Five of six ambulatory patients showed improvement or very slow progression of the disease post surgery ALS Functional Rating Scale – Revised ( ALSFRS - R) monitors changes in a patient over time with ten measures including speech , swall
Slide 13: Non responders
Non responders ALSFRS-R in Gen mars enna HS Orne
Slide 14: Near Virtual Model
Neuralstem, Inc. ‘Near Virtual” model Core management functions in house □ Multiple overlap, multiple positions □ Outsource everything that makes sense □ Reduced overhead and fiscal flexibility, higher quality/less control over the calendar Financing □ Strong balance sheet □ SOX compliant □ Money go
Slide 15: Collaborators: World-Class Clinical Partners
Neuralstem , Inc. Collaborators World - Class Clinical P artners □ ALS Trial Clinical Investigators : □ Principal Investigator: Eva L. Feldman, M.D., Ph.D. , Professor of Neurology & Director A. Alfred Taubman Medical Research Inst. of the University of Michigan Medical School; President of American
Slide 16: Strengths and Future Value Drivers
Neuralstem – Strengths and Future Value Drivers 23 Scientific Strength, Out In Front • Leader: neural stem cell science • Repeated firsts in both platforms, including FDA approval: ◦ ALS & SCI cell therapy trials ◦ MDD lead novel neurogenic compound • Technology platform R&D developed at NIH • Broad