Adhera Therapeutics Inc. · OTC:ATRX
Adhera Therapeutics — Marina Biotech — World Orphan Drug Congress 2015
13 slides from Adhera Therapeutics's conference presentation, Apr 27, 2015
Slide 1: Nucleic Acid Therapeutics and Rare Diseases
Nucleic Acid Therapeutics and Rare Diseases World Orphan Drug Congress – 2015 23 April 2015
Slide 2: Forward Looking Statement
Forward Looking Statement This document contains forward - looking statements about Marina Biotech, Inc . (the “Company”) that are based on current management expectations . These statements reflect the Company’s current views with respect to uncertain future events and are based on imprecise estima
Slide 3: Marina Biotech – A Leading RNA Therapeutics Company
Marina has technology capability equal to the three largest companies in the sector, combined, . . . Marina Biotech – A Leading RNA Therapeutics Company 3 Marina Antisense siRNA microRNA mimics & antagomirs . . . and a Rare Disease Pipeline with one Phase I program and two preclinical programs!
Slide 4: Novel & Proprietary Chemistries
Novel & Proprietary Chemistries » Unlocked nucleobase monomers (UNM) – non - nucleotide, acyclic monomers 4 » Conformationally restricted nucleotides (CRN) – nucleotide analogs with a “bridge” connecting the C2’ and C4’ carbons of ribose RNA CRN LNA O OH BASE OH OP - O O - O OP - O O - O O OH O BASE
Slide 5: Novel & Proprietary Delivery Systems
» SMARTICLES – Lipid - based system comprised of unique combinations of anionic and cationic lipids » DiLA2 – Lipid - based system comprised of unique combinations of naturally occurring or man - made amino acid head groups, linkers and alkyl chains » tkRNAi – Bacterial - based system utilizing n on
Slide 6: Delivery technologies in clinical development
» Two separate and distinct delivery technologies in clinical development » SMARTICLES (lipid nanoparticle) » tk RNAi (engineered, non - pathogenic bacteria) » Only orally administered RNAi - based therapeutic ( tk RNAi ) in clinical development » Most versatile delivery system (SMARTICLES) in clini
Slide 7: Clinical Program Pipeline
7 Clinical Program Pipeline Development Stage Program Myotonic Dystrophy Pre - IND Studies Phase I CEQ508 - FAP Duchenne Muscular Dystrophy IND 2017 2016 2017 2017 2018 2017 2017 2018 2019 Phase II Lead Selection
Slide 8: Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis (FAP) » Rare hereditary disease » Mutation in Adenomatous Polyposis Coli (APC) gene » Causes dysregulation and accumulation of β - catenin » Results in numerous colon polyps appearing in early adolescence with potential for rapid disease progression » Clinical drug pro
Slide 9: TransKingdom RNAi (tkRNAi)
TransKingdom RNAi ( tk RNAi ™) 9 to Dicer for processing Bacterial lysis in endosome Receptor - mediated cell entry Endosome lysis and shRNA release into cytoplasm Invasin receptor Invasin shRNA Compound is taken orally by the patient Only orally a vailable RNAi clinical c andidate
Slide 10: Nucleic Acid-based Strategies for Therapeutic Intervention in DM1
Nucleic Acid - based Strategies for Therapeutic Intervention in DM1 10 Accumulation of altered DMPK mRNA in nucleus sequesters Muscleblind (MBNL1) protein resulting in loss - of - function » Steric Blocking » Displace/disrupt the MNBL - CUG n+x interaction » mRNA Translational Inhibition » RNAi or R
Slide 11: Ability to Move Multiple Compounds into Preclinical Development
Ability to Move Multiple Compounds into Preclinical Development for a Single Indication 11 Targeting DMPK mRNA to increase degradation and elimination Targeting the CUG - repeat for degradation/elimination or compete with proteins for binding sites Targeting intron - exon s plice to bias for less -
Slide 12: Partner of Choice for Rare Diseases
Partner of Choice for Rare Diseases » Marina Biotech has multiple novel and proprietary b iochemistries and delivery technologies » Development and delivery of single - and double - stranded constructs » Marina Biotech has the broadest, nucleic acid drug discovery platform » Validated drug discovery
Slide 13: Thank You
Thank You NOP