Rock Creek Pharmaceuticals Inc
Rock Creek Pharmaceuticals — Biotech Showcase 2015
27 slides from Rock Creek Pharmaceuticals's conference presentation, Jan 12, 2015
Slide 1: Biotech Showcase 2015
January 12, 2015 Biotech Showcase 2015
Slide 2: Forward Looking Statements
Forward Looking Statements Certain statements that will be made during this presentation constitute forward - looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements identified by words such as "believe
Slide 3: Corporate Overview
Corporate Overview • Drug development company focused on the discovery, development and commercialization of new drugs to provide therapies for chronic inflammatory diseases, neurological disorders and behavioral health • Advancing into human clinical development in the UK and US • Novel lead compou
Slide 4: Anatabine: Key Attributes
Anatabine: Key Attributes • Small molecule • Exhibits anti - inflammatory pharmacological characteristics via Cholinergic Receptor Binding • Crosses blood brain barrier • Excellent bioavailability • Well tolerated as dietary supplement by several hundred thousand users 4
Slide 5: Pentameric Acetylcholine Receptor
Pentameric Acetylcholine Receptor Pentamer nAChR top view (extracellular domain) Pentamer nAChR side view 5
Slide 6: Acetylcholine Receptor Activation
Extracellular Intracellular Cations Acetylcholine Cellular function Acetylcholine Receptor Activation 6
Slide 7: Acetylcholine and Anatabine Binding at the interface of α7-nAChR
Acetylcholine Binding at the interface of α7 - nAChR Anatabine Binding at the interface of α7 - nAChR ElectroStatic Ribbon Model 7
Slide 8: Acetylcholine Anatabine Overlay
Acetylcholine Anatabine Overlay
Slide 9: Nuclear Factor Kappa B (NF-κB)
Nuclear Factor Kappa B (NF - ƙB ) • NF - κB is a protein complex that controls the transcription of DNA. • Found in almost all animal cell types and is involved in cellular responses to external stimuli. • NF - κB plays a key role in regulating cellular response to inflammation. • Specifically, NF -
Slide 10: NF-κB Signaling pathways: Anatabine
NF - ƙb Signaling pathways: 10 Anatabine Inflammatory Molecules: TNF - α, IL - 1, IL - 6, IFN - γ, Cox - 2 Amyloid, etc. α - 7 nAChR
Slide 11: Proposed Common Mechanism(s) of Action of Anatabine
Proposed Common Mechanism(s) of Action of Anatabine across Different CNS (and non - CNS) Conditions Suppression of inflammatory activity (via ህ NF - kB) Activation of alpha 7 nAchR on microglia (macrophage, monocytes, B - cells, T - cells) Reduced inflammatory cytokine release Reduced tissue damage
Slide 12: Peer Reviewed Published Studies
Anatabine Lowers Alzheimer’s Aβ Production in vitro and in vivo Publication: European Journal of Pharmacology, Sept 2011 Anti - inflammatory Activity of Anatabine via Inhibition of STAT 3 Phosphorylation Publication: European Journal of Pharmacology , Nov 2012 Anatabine Ameliorates Experimental
Slide 13: Pre-Clinical Analysis: Experimental Autoimmune Encephalomyelitis (EAE)
• Mice injected at 0 hr and 48 hrs with 300µg of Pertussis toxin • Day 1, 10 Week - old C57Bl6/J Female mice immunized with 300 µg of murine MOG35 - 55 (myelin oligodendrocyte glycoprotein) • Mice randomized in placebo group (n=15) receiving regular drinking water and Anatabine group (n=15) receivin
Slide 14: Anatabine: Preclinical Analysis
- Reduced nerve damage in Anatabine treated EAE mouse spinal cord Anatabine: Preclinical Analysis 14
Slide 15: Normalization of Inflammatory Spleen Cytokines in EAE mice by Anatabine
15 Normalization of Inflammatory Spleen Cytokines in EAE mice by Anatabine
Slide 16: Auto-Immune Thyroiditis: Human Trial
Hashimoto's thyroiditis cytology showing lymphocytes and Hurthle cell metaplasia 17 Lowell R. Schmeltz et al, (2014) Anatabine Supplementation Decreases Thyroglobulin Antibodies in Patients With Chronic Lympho cyt ic Autoimmune (Hashimoto’s) Thyroiditis: A Randomized Controlled Clinical Trial. J Cli
Slide 17: Reduced Anti-thyroglobulin antibodies in Anatabine treated Thyroiditis patients
Reduced Anti - thyroglobulin antibodies in Anatabine treated Thyroiditis patients Lowell R. Schmeltz et al, (2014) Anatabine Supplementation Decreases Thyroglobulin Antibodies in Patients With Chronic Lympho cyt ic Autoimmune (Hashimoto’s) Thyroiditis: A Randomized Controlled Clinical Trial. J Clin
Slide 18: Potential Therapeutic Indications
Potential Therapeutic Indications • Arthritic Conditions (Osteoarthritis) • Dermatology (Psoriasis) • Inflammatory Bowel Disorders (Ulcerative Colitis, Crohn’s) • Auto - Immune Disorders ( Thyroiditis ) • CNS (Alzheimer’s, Multiple Sclerosis) Smoking Cessation Initial Clinical Targets: Osteoarthriti
Slide 19: Intellectual Property
Intellectual Property 03/01/14 • Six Issued US/Foreign Anatabine related patents: 1. Anatabine Citrate - Novel Compound - US Patent 8,577,999 2. Method of Anatabine synthesis • Six (+) US and foreign patents pending for use of Anatabine, its isomers and derivatives for inflammatory conditions/smokin
Slide 20: Anatabine: UK and US Regulatory Strategy
Anatabine: UK and US Regulatory Strategy • Clinical Trial Application (CTA) filed with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK – CTA Response Received, responding to minor comments – Expect CTA approval Q1 – Engaged Quotient Clinical as CRO for Phase I: Q1 start – Fo
Slide 21: Funding Strategies
Funding Strategies 21 • $10mn ATM Facility/$50mn Shelf (S - 3) • Evaluating Alternative Strategies • Existing Warrant Exercise • Equity Line • Direct Investment • IP Lending • Venture Debt
Slide 22: Management Team
Dr. Michael Mullan, MD, PhD., Chairman and Chief Executive Officer Recognized authority on Alzheimer's disease and related disorders. Trained as a physician. Medical degree and PhD in molecular genetics, from London University. Inventor on the patents covering the first genetic mutations lin
Slide 23: Board of Directors
Benjamin M. Dent : CEO of the Dent Consulting Group LLC and an adjunct professor with the St. Louis University graduate program. Mr. Dent previously has served in senior financial management positions with several public and private companies, and also has served as a management consultant to Lehman
Slide 24: Risk Factors
FDA/EMA regulatory timeline progression change Clinical studies do not reach desired endpoints. Ongoing operating losses, access to cash, sustainable funding and growth. Inability to attract joint venture/licensing partners Risk Factors 24
Slide 25: Upcoming Milestones
Upcoming Milestones 15 Q115 CTA Approval Phase I under CTA Safety, PK, PD data Drug Formulation development Phase IIA POC studies Q 215 Q315 Q415 2016 Phase II studies CTA Phase I Complete 25 US IND Protocol Development
Slide 26: Summary
Summary • Drug development company focused on the discovery, development and commercialization of new drugs to provide therapies for chronic inflammatory disease, neurological disorders and behavioral health • Advancing into human clinical development in the UK and US • Novel lead compound Anatabine
Slide 27: Biotech Showcase 2015
January 12, 2015 Biotech Showcase 2015