Palisade Bio, Inc. · NASDAQ:PALI
Palisade Bio — Neuralstem — Cell & Gene Therapy World (January 2018)
17 slides from Palisade Bio's conference presentation, Jan 24, 2018
Slide 1: Treatment of ALS with Neural Stem Cells
neuralstem.com Treatment of ALS with Neural Stem Cells Cell & Gene Therapy World 1 Karl Johe, Ph.D., CSO & Founder Neuralstem, Inc. January 24, 2018
Slide 2: Safe Harbor Statement
neuralstem.com Safe Harbor Statement 2 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 1
Slide 3: Manufacturing at commercially viable scale while maintaining cell properties
neuralstem.com 6 Manufacturing at commercially viable scale while maintaining cell properties cGMP , 3 - tiered cell banks, fully tested & validated: • MCB (~250 vials, p6), WCB (~350 vials, p9), CCB (~400 vials, p12) — 2x10 7 cells/vial • Normal karyotype – 44 + XY 1. Large - scale, reproducible ma
Slide 4: MOA: Neuroprotection of host motor neurons
neuralstem.com 2. MOA: Neuroprotection of host motor neurons 1. Xu L, et al. Neurosci Lett. 2011; 494(3): 222 - 6. 2. Xu L, et al. J Comp Neurol. 2009; 514(4):297 - 309. 3. Yan J, et al. PLoS Med. 2007 4(2): e39. 4. Xu L, et al. Transplantation. 2006; 82(7):865 - 75. 5. Yan J, et al. Stem Cells. 200
Slide 5: Development of Cell Injection Device and Cannula
neuralstem.com 3. Development of Cell Injection Device and Cannula: Precise, reproducible, accurate injections 5 28d DCX 7 wks 1. Gutierrez J, et al. Neurosurgery. 2015 Oct;77(4):604 - 12; discussion 612. 2. Federici T, et al. J Vis Exp. 2012 Dec 7;(70):e4371. 3. Riley JP, et al. Neurosurgery. 2011
Slide 6: Commercially Viable Formulation and Distribution of Drug Product
neuralstem.com Cell Viability (%) Time after preparation ( hrs ) 0 24 48 72 Viability of Drug Product Temp ( o C ) Time after shipping ( hrs ) 0 4 6 8 10 12 14 16 18 2 Shipping Temperature of Drug Product 2 - 8 o C 4. Commercially Viable Formulation and Distribution of Drug Product: Live cell suspen
Slide 7: NSI-566: ALS Phase I: Team work!
neuralstem.com 5. NSI - 566: ALS Phase I : Team work! 7 A Phase I, Open - label, First - in - human, Feasibility and Safety Study of Human Spinal Cord derived Neural Stem Cell Transplantation for the Treatment of ALS Eva L. Feldman 1 , Jonathan D. Glass 2 , Nicholas M. Boulis 2 , Thais Federici 2 ,
Slide 8: Phase I Clinical Design: Progressive Risks and Benefits
neuralstem.com 6. Phase I Clinical Design: Progressive Risks and Benefits (n=18) 8 A1: ½ million cells A2: 1 million cells L2 L3 L4 C3 C4 C5 L2 L3 L4 1 million cells ½ million cells ½ million cells 1.5 million cells B C D E C3 C4 C5 L2 L3 L4 L2 L3 L4 Compliments of Jon Glass MD A1/A2
Slide 9: Immune suppression regimen: Long-term graft survival
neuralstem.com 9 1. Feldman EL, et al. Ann Neurol. 2014 Mar;75(3):363 - 73. 2. Riley J, et al. Neurosurgery. 2014 Jan;74(1):77 - 87. 3. Tadesse T, et al. Ann Clin Transl Neurol. 2014 Nov;1(11):900 - 8. 7. Immune suppression regimen: Long - term graft survival
Slide 10: NSI-566: Long-term graft survival with transient immunosuppression
neuralstem.com NSI - 566: Long - term graft survival with transient immunosuppression 10 Tadesse T, et al. Ann Clin Transl Neurol. 2014 Nov;1(11):900 - 8. Patient number Gender # of days on FK506 # of days on MMF # of Days IM Meds Discontinued before death Survival Days % Donor DNA 1 M 177 165 216 3
Slide 11: NSI-566: Long-term graft survival with transient immunosuppression
neuralstem.com 11 NSI - 566: Long - term graft survival with transient immunosuppression
Slide 12: NSI-566 Phase I: Clear treatment-emergent improvement of function
neuralstem.com NSI - 566 Phase I 12 Feldman EL, et al. Ann Neurol. 2014 Mar;75(3):363 - 73. Clear treatment - emergent improvement of function
Slide 13: NSI-566: ALS Phase II
neuralstem.com NSI - 566: ALS Phase II 13 A Phase II, Open - label, Dose Escalation and Safety Study of Human Spinal Cord derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis Study Sites: University of Michigan, Emory University, Mass General Hospital • N=15 of
Slide 14: Phase II Clinical Design: Maximum Tolerated Dose
neuralstem.com 7. Phase II Clinical Design: Maximum Tolerated Dose (n=18) 14 2 million cells 4 million cells B 6 million cells C 8 million cells D C3 C4 C5 C3 C4 C5 16 million cells E L2 L3 L4 Compliments of Jon Glass MD
Slide 15: NSI-566: ALS Phase II Results
neuralstem.com NSI - 566: ALS Phase II Results: 15 50% of study patients with positive change of ALSFRS slope
Slide 16: NSI-566: ALS Phase I and II Follow-Up vs Historical Data
neuralstem.com NSI - 566 : ALS Ph ase I and II: 2 - Year Follow - Up vs Historical Data 10 16 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
Slide 17: NSI-566 Target Product Profile (TPP) for ALS
neuralstem.com 8. NSI - 566 Target Product Profile (TPP) for ALS 17 Mode of Action ▪ Human neural stem cells, as an allogeneic transplant, rescue motor neurons by: ▪ Expressing excitatory amino acid transporters to restore glutamate re - uptake ▪ Releasing neurotrophic growth factors that slow motor
Neuralstem — Cell & Gene Therapy World (January 2018) on 6ix