Cell & Gene Therapy Investor Day
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April 17, 2018
Cell & Gene Therapy Investor Day April 17, 2018 1 Safe Harbor - - PowerPoint PPT Presentation
Cell & Gene Therapy Investor Day April 17, 2018 1 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 27A of
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April 17, 2018
2 Safe Harbor statements under the Private Securities Litigation Reform Act of 1995: This presentation contains forward-looking statements as defined in Section 27A of the Securities Act of 1933 as amended, and section 21E of the Securities Exchange Act of 1934, as amended. Such forward-looking statements are based upon Neuralstem, Inc.’s management’s current expectations, estimates, beliefs, assumptions, and projections about Neuralstem’s business and industry. Words such as “anticipates,” “expects,” “intends,” “plans,” “predicts,” “believes,” “seeks,” “estimates,” “may,” “will,” “should,” “would,” “potential,” “continue,” and variations of these words (or negatives of these words) or similar expressions, are intended to identify forward-looking statements. In addition, any statements that refer to expectations, projections, or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. These forward-looking statements are not guarantees of future performance and are subject to certain risks, uncertainties, and assumptions that are difficult to predict. Therefore, our actual results could differ materially and adversely from those expressed in any forward-looking statements as a result of various risk factors. These risks and uncertainties include the risks associated with the effect of changing economic conditions, trends in the products markets, variations in Neuralstem’s cash flow, market acceptance risks, technical development risks and other risk factors detailed in Neuralstem’s Securities and Exchange Commission filings. For links to SEC documents please visit the company’s Web site: neuralstem.com. Although we believe that we have a reasonable basis for each forward-looking statement contained in this presentation, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this presentation as a result of, among other factors, the factors referenced in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2017 filed with the Securities and Exchange Commission on April 2, 2018, Form 10-K for the period ended September 30, 2017, and in other reports filed with the SEC. In addition, even if our results of
looking statements contained in this presentation, they may not be predictive of results or developments in future periods. Any forward- looking statements that we make in this presentation speak only as of the date of such statement, and we undertake no obligation to update such statements to reflect events or circumstances after the date of this presentation, except as required bylaw.
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NSI-566: ALS, c-Spinal Cord Injury, c-Stroke – Neural stem cell therapy – Targeting paralysis with biological activity in across 3indications – Functional restorative effects shown in primate model
– Favorable regulatory environment: US, Japan, China – Partnering efforts underway for continuingdevelopment NSI-189: MDD – Novel neurogenic small molecule approach – Positive, randomized placebo-controlled Phase 1b in MDD – Phase II Exploratory Study in Major Depressive Disorder(MDD)
– Montgomery-Asberg Depression Rating Scale (MADRS) primary endpoint – Secondary endpoints: – Physician and Patient reported – Cognition
MARKET CONSIDERATIONS
prognosis
Century Cures Act) PROGRAM OVERVIEW
patients
safety studies completed
Data
with historic data set (PROACT) insightful KEYTAKEAWAYS
tolerated
proven at autopsy MARKET CONSIDERATIONS
cord injury in the US*
PROGRAM OVERVIEW
AISA-A complete spinal cord injury
A complete spinal cord injury – First subject enrolled KEYTAKEAWAYS
tolerated
muscles below the level of injury in
confirmed via clinical and electrophysiological follow-up examinations
ALS
MARKET CONSIDERATIONS
the US*
stroke until well after the event
hours of event PROGRAM OVERVIEW
escalation, feasibility & safety study for the treatment of paralysis from chronic motorstroke
months post-event with stable hemi- paralysis
recovery KEYTAKEAWAYS
injectioncannula) system developed
Ischemic Stroke Chronic Spinal CordInjury
*Prevalence and Causes of Paralysis—United States, 2013. Armour, B.S. et al. (2016) Am J Public Health. 106: 1855-1857.
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Graft
a b
Rosenzweig et al., Nat Med. 2018 Feb 26. doi: 10.1038/nm.4502
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NSI-566: ALS Phase I & II: 2-Year Follow-Up vs Historical Data
ALSFRS-R Ph1/2 PRO-ACT N Mean (SD) N Mean (SD) t-test Baseline 21 36·5±5·4 1108 38·1±4·7 0·17 6 months 18 30·6±6·5 974 32·5±7·6 0·25 12 months 14 30·5±9 655 28·3±9·3 0·37 18 months 11 31·8±8·1 165 24·6±10·4 0·016 24 months 11 30·1±8·6 86 24·0±10·2 0·048 ALS/SURV Ph1/2 PRO-ACT N Median (IQR) N Median (IQR) Wilcoxon Baseline 21 38 (31,40) 1108 39 (35,42) 0·12 6 months 20 29·5 (23,35·5) 1012 33 (27,38) 0·11 12 months 18 26·5 (16,35) 792 26 (15,35) 0·82 18 months 17 27 (1·2yr,36) 351 1·4yr (0·8yr,24) 0·028 24 months 19 23 (1·2yr,33) 306 1·2yr (0·8yr,10) 0·0038
NSI-566 therapy as a treatment for ALS is safe. Additional analysis of the efficacy is helpful for future study design and it indicates efficacy on ALSFRS beginning at 18 months.
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Meaningful Clinical Benefits: >10 points of improvement in Fugl-Meyer Motor Score
Fugl-Meyer Motor Score Post Treatment (months, 0-12)
Change from Baseline (+/- SEM)
Modified Rankin Scale
Change from Baseline (+/- SEM)
Post Treatment (months, 0-12) NIH Stroke Scale
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Post Treatment (months, 0-12)
Change from Baseline (+/- SEM)
NSI-566: Phase 1 open-label, non-GCP dose-escalation, feasibility & safety study for the treatment of paralysis from chronic motorstroke
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April 17, 2018