Palisade Bio, Inc. · NASDAQ:PALI
Palisade Bio — Neuralstem — Phacilitate Cell & Gene Therapy World 2016
31 slides from Palisade Bio's conference presentation, Jan 27, 2016
Slide 1: Phacilitate Cell & Gene Therapy World, January 25-27, 2016
Phacilitate Cell & Gene Therapy World January 25-27, 2016 Washington D.C.
Slide 2: Safe Harbor Statement
NEURALSTEM, INC. 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 the Securities Act of 1933 as amended, and section 21E of the Securities Exchange Act of 1934,
Slide 3: Overview
Overview Neuralstem’s proprietary technology uses regionally specific neural stem cells for the development of CNS therapies. · Neuralstem (Nasdaq: CUR) founded in 1996 · Human neural stem cell technology, discovered by its founding Scientist and Chairman, Karl Johe, Ph.D., while at NINDS at NIH · I
Slide 4: Dual Platform Pipeline
Dual Platform Pipeline Lead Small Molecule Asset Targeting Depression Cell therapy targeting high unmet medical need populations
Slide 5: Clinical Development History/Plan of HSSC Cell Line
Clinical Development History/Plan of HSSC (Human Spinal Cord-Derived Neural Stem Cells) Cell Line Indication Location Trial ROA Doses Study Status Phase NSI-566, CCB ALS USA I Intraspinal 0.5x10 6 /5 inj- Completed in Feb 061005 (n=15) 1.5x10 6 /15 inj 2013 NSI-566, CCB ALS USA II (n=15) Intraspinal
Slide 6: Leading stem cell product: NSI-566RSC
Leading stem cell product: NSI-566RSC--neural stem cells from one fetal spinal cord tissue 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/
Slide 7: SOD-1 Rat Model of ALS
NSI-566: SOD-1 Rat Model of ALS · Koliatsos et al., Johns Hopkins · Marsala et al., UCSD 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,
Slide 8: Intraspinal Injections in Mini-pigs
NSI-566: Intraspinal Injections in Mini-pigs: · Boulis et al, Emory · Marsala et al., UCSD 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 Dec;69(2 Suppl Operative):ons147-54; d
Slide 9: ALS Phase I
NSI-566: ALS Phase I 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 , MeraidaPolak 2 , C. Kelly 2 and Karl Johe
Slide 10: ROA intraspinal injections
NSI-566: ROA intraspinal injections 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.
Slide 11: Long-term graft survival with transient immunosuppression
NSI-566: Long-term graft survival with transient immunosuppression # of # of # of Days IM Meds days on days on Patient FK506 MMF Discontinued number Gender before death Survival Days % Donor DNA 1 M 177 165 216 394 0.06 – 5.40 2 M 107 503 67 572 0.18 – 0.93 3 M 259 259 0 259 0.03 – 2.39 4 M 189 192
Slide 12: Phase I: Clear treatment-emergent improvement of function
NSI-566 Phase I Clear treatment-emergent improvement of function Feldman EL, et al. Ann Neurol. 2014 Mar;75(3):363-73.
Slide 13: ALS Phase II
NSI-566: ALS Phase II 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 variable disease profil
Slide 14: ALS Phase II Results
NSI-566: ALS Phase II Results 50% of study patients with positive change of ALSFRS slope
Slide 15: ALS Phase II Results: better than historical dataset
NSI-566: ALS Phase II Results 40-80% of study patients better than historical dataset ALSFRS-R FVC (% predicted) Grip strength
Slide 16: SCI: Motor improvement, scar reduction & cavity-filling effect
NSI-566 SCI: Motor improvement, scar reduction & cavity-filling effect grafted at Day 3 post-SCI van Gorp S, et al., Stem Cell Res Ther. 2013 May 28;4(3):57.
Slide 17: SCI: Motor improvement & functional integration
NSI-566 SCI: Motor improvement & functional integration grafted at day 7 post-SCI in rat with complete spinal transection Lu P, et al., Cell. 2012 Sep 14;150(6):1264-73.
Slide 18: cSCI: Phase I Safety Trial
NSI-566 cSCI: Phase I Safety Trial 1 – 2 years after injury AIS A only Group A: 4 patients with T2-T12 (completed) Removal of instruments to enable MRI survey 1.2 x 10 6 cells in 6 bilateral injections at and below injury 6 month follow-up: No SAE 4.5 year post-study follow-up Study Site: University
Slide 19: cSCI: Phase I Safety Trial Subject #4
NSI-566 cSCI: Phase I Safety Trial Subject #4, self-reported https://www.instagram.com/p/9161Cag9vK/
Slide 20: cSCI: Phase I Safety Trial Subject #4 EMG results
NSI-566 cSCI: Phase I Safety Trial Subject #4 25 y/o male, injury 416 days prior to stem cell treatment Baseline: T5 Neurological AIS A Complete Muscle Location 12-Week Visit: EMG 6- Month Visit: EMG Voluntary Voluntary Muscle Unit Potentials Muscle Unit Potentials L, R rectus abdominis, T6 2-3 semi
Slide 21: Robust survival & neuronal differentiation in rat model of stroke
NSI-566: Robust survival &neuronal differentiation in rat model of stroke Tajiri et al., PloS One (2014) 9: e91408
Slide 22: Ameliorates stroke-induced motor deficits in rat model
NSI-566: Ameliorates Stroke – induced motor deficits in rat model of stroke Tajiri et al., PloS One (2014) 9: e91408
Slide 23: Stroke: NSI-566 engraftment in mini-pig brain
NSI-566:Stroke NSI-566 engraftment at 4 weeks post transplantation in ischemia-induced mini-pig brain K299-HuNu K299-H&E Human NF-specific staining
Slide 24: Phase I for treatment of paralysis from chronic stroke
NSI-566: Phase I for treatment of paralysis from chronic stroke Single Study Site: BaYi Hospital, Beijing PLA Open label feasibility & safety study Cohort Treatment Cell Deposits No of Status ID Number/Deposit per Track Cannula Tracks A (n=3) 1.2x10 7 40,000 5 3 Dosed cells/μL×20μL B (n=3) 2.4x10 7
Slide 25: Stroke: Phase I interim data, Subject 101
NSI-566 stroke: Phase I interim data Group A: 1.2x10 7 cells, 15 deposits Subject # 101 Gender M Age 50 Weight(kg) 78 Infarct Site Left, Subcortical Surgery 607 (days post stroke)
Slide 26: Stroke: Phase I interim data, Subject 102
NSI-566 stroke: Phase I interim data Group A: 1.2x10 7 cells, 15 deposits Subject # 102 Gender M Age 47 Weight(kg) 75 Infarct Site Right, Subcortical Surgery 424 (days post stroke)
Slide 27: Stroke: Phase I interim data Group A
NSI-566 stroke: Phase I interim data Group A: Subject 102
Slide 28: Stroke: Phase I interim data Group A
NSI-566 stroke: Phase I interim data Group A: Subject 102
Slide 29: Stroke: Phase I interim data, Subject 103
NSI-566 stroke: Phase I interim data Group A: 1.2x10 7 cells, 15 deposits Subject # 103 Gender M Age 30 Weight(kg) 90 Infarct Site Right, Subcortical Surgery 150 (days post stroke)
Slide 30: Group A: Subject
Group A: Subject 103
Slide 31: Conclusion
Conclusion Human spinal cord-derived neural stem cells (NSI-566) has demonstrated consistent biological activities across multiple disease conditions: ALS, stroke, cSCI Next steps: Pivotal trials in multiple indications to demonstrate efficacy as the primary objective. The trials will be randomized,
Neuralstem — Phacilitate Cell & Gene Therapy World 2016 on 6ix