Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — Merger or Acquisition (April 2021)
20 slides from Eloxx Pharmaceuticals's merger presentation, Apr 1, 2021
Slide 1: Creating a Leader in Ribosomal RNA-Targeted Genetic Therapy: Eloxx Acquires Zikani Therapeutics
Creating a Leader in Ribosomal RNA - Targeted Genetic Therapy Eloxx A cquires Zikani Therapeutics April 1, 2021
Slide 2: Forward-looking statements
This presentation contains forward - looking statements, which are generally statements that are not historical facts . Forward - looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions . Forwa
Slide 3: Eloxx + Zikani: Positioned to be the world leader in ribosome RNA-targeted genetic therapies
• President and CEO: Sumit Aggarwal • Head of R&D: Dr. Vijay Modur • TURBO - ZM TM : Proprietary synthetic chemistry platform to design novel macrolide - based oral small molecules • Ribosome Modulating Agents (RMAs): Potent oral ribosome modulators with favorable therapeutic index Eloxx + Zikani :
Slide 4: Zikani leadership team to join Eloxx
Zikani leadership team to join Eloxx Dr. Vijay Modur Head of Research & Development • 20+ years in translation and drug development • Led Venglustat rare disease program at Sanofi • 30+ years building companies • Closed $2B in equity and debt financings for public and private companies Daniel Geffke
Slide 5: Transaction overview
Transaction overview Eloxx issued ~ 7.6 million shares Zikani stockholders have pro forma ownership ~ 16% of Eloxx Consideration Silvia Noiman, Ph.D ., and Martijn Kleijwegt stepped down from Eloxx Board Alan Walts, Ph.D., and Raj Parekh, Ph.D., current Zikani directors, appointed to fulfill
Slide 6: Acquisition combines complementary platforms in targeting the human ribosome
Acquisition combines complementary platforms in targeting the human ribosome Genetic nonsense mutations • Premature stop codon • Less than full length protein formed = loss of function ERSG RMA Dysregulated Ribosomes • Onco - and mitoribosomes • Selective translation of oncogenic proteins • Aids hyp
Slide 7: ELX-02: Potential for transformative efficacy in Class 1 CF patients
• S welling response in Class 1 CF patient organoids • Induces CFTR activity of up to 30 % of normal; confirmed in Ussing Chamber • Active across broad range of mutations Demonstrated efficacy in clinically relevant pre - clinical models • Generally well - tolerated for chronic dosing • Consistent p
Slide 8: Deep pipeline of synergistic potential first-in-class therapies
Target Indication Discovery Early research Lead optimization IND enabling Phase 1 - First in Human Phase 2 Nonsense Readthrough : Rare Disease CFTR Class 1 CF Collagen VII A1/LAMB3 RDEB/JEB CFTR Class 1 CF PKD1, PKD2 and Oca2 ADPKD/ Inherited Retinal Diseases Nonsense Readthrough : Oncology APC FAP
Slide 9: Zikani Therapeutics Introduction
Zikani Therapeutics Introduction
Slide 10: TURBO-ZM platform fully unlocks the potential of macrolides
10 TURBO - ZM TM (Tuning the Ribosome with Zikani Molecules) platform fully unlocks the potential of macrolides Optimize for: • Readthrough • Protein translation inhibition Interact with the peptide sequence Modulate: • PK • Safety: cardiac, liver • Oral bioavailability RMA core required for ribosom
Slide 11: Strong rationale for macrolides to bind the human ribosome
11 Strong rationale for macrolides to bind the human ribosome Nascent peptide exit tunnel in E. coli vs. Human ribosomes 1 1 Nucleic Acids Res. 2019;47(8):4198 - 4210 H. sapiens E. coli Macrolide binding site in prokaryotic (bacterial) ribosome 75% of binding site conserved in the human (eukaryotic)
Slide 12: Growing library of RMAs with drug-like properties
12 Growing library of RMAs with drug - like properties Zikani RMA Library (2000+)
Slide 13: RMAs show superior readthrough to alternatives
13 RMAs show superior readthrough to alternatives Readthrough Emax of selected RMA hits relative luciferase units compared to DMSO in W134X Nanoluc reporter assay 3,005 5,613 1,791 18,188 7,420 14,811 10,157 8,597 24,330 12,614 ZKN - 3 ZKN - 1 ZKN - 5 ZKN - 2 ZKN - 4 ZKN - 6 +710% +333% ZKN - 7 G418
Slide 14: RDEB/JEB: Clinically validated path for RMAs in rare skin disease
14 RDEB/JEB: Clinically validated path for RMAs in rare skin disease targeting patients with nonsense mutations 1 Woodley, DT. J Clin Investig 2017, 127, 3028 - 3038; 2Hammersen, Johanna et al. Dermatology May 2019 RDEB and JEB • Mutations in COL7A1 gene (Collagen) and LAMB3(Laminin) • Most RDEB pat
Slide 15: RDEB: RMAs restore functional collagen protein in primary patient cells
15 RDEB: RMAs restore functional collagen protein in primary patient cells comparable to high dose gentamicin COL7 with 48 hr. exposure in RDEB patient derived primary fibroblasts* * Fibroblasts isolated from patients two and five in gentamicin clinical trial. J Clin Invest 2017, 127, 3028 - 3038 **
Slide 16: Class CF: RMA lead showed highest ever readthrough preclinical Ussing chamber assay
16 Class 1 CF: RMA lead showed highest ever readthrough preclinical Ussing chamber assay Summary of Class 1 CF data * Forskolin 10 µM/1µM VX - 770 - both chambers ** VX 809 and RMA data averaged from 2 separate Ussing chamber results • Submitted $2.5M grant to CF Foundation to support through develo
Slide 17: APC readthrough: Supported by prior clinical success of Erythromycin in FAP
17 APC readthrough: Supported by positive prior clinical success of Erythromycin in FAP 1 Kariv, R. Int J Cancer. 2019 doi: 10.1002/ijc.32557 Clinical trial success in FAP with Erythromycin Erythromycin treatment (250 mg/day po for 4 months) Change in polyp burden at 12 months 1 Change in polyp numb
Slide 18: Clear path in treating FAP supported by efficacy in APC mutant tumor grafts
18 Clear path in treating FAP supported by efficacy in APC mutant cancer patient tumor grafts Efficacy of ZN013 in colorectal cancer patient derived tumor grafts ex - vivo • Ex - vivo sensitivity assessment in tumor grafts • Potent tumor growth inhibition – GI 50 <15uM • Cancer xenograft studies pla
Slide 19: Eloxx + Zikani: Positioned to be the world leader in ribosome RNA-targeted genetic therapies
Strengthened leadership Rare disease and oncology pipeline Compelling assets in CF Complementary platforms targeting the human ribosome 20 Eloxx + Zikani : Positioned to be the world leader in ribosome RNA - targeted genetic therapies
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