Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — Cantor Global Healthcare Conference 2018
28 slides from Eloxx Pharmaceuticals's conference presentation, Oct 2, 2018
Slide 1: Unlocking protein production with translational read-through for rare genetic diseases
Unlocking protein production with translational read - through for rare genetic diseases Cantor Global Healthcare, October 2, 2018
Slide 2: Forward-Looking Statements
This press release 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. Forwar
Slide 3: Eloxx Pharmaceutical Highlights
Eloxx Pharmaceutical Highlights 3
Slide 4: Built a Highly Experienced Leadership Team
Greg Williams, PhD COO Robert Ward CHAIRMAN AND CEO Neal Sharpe, PhD VP TRANSLATIONAL SCIENCE John van Duzer, PhD VP CMC Built a Highly Experienced Leadership Team 4 Greg Weaver CFO David Snow CBO Barbara Ryan INVESTOR RELATIONS Neil Belloff, Esq. GENERAL COUNSEL
Slide 5: The Potential for Read-Through of Rare Genetic Diseases
The Potential for Read - Through of Rare Genetic Diseases Aminoglycosides first showed read - through activity in nonsense mediated diseases Aminoglycosides' tolerability profile historically limited suitability for read - through treatment of serious genetic diseases Advances in our understanding o
Slide 6: Targeting Read-Through Development Activities
Targeting Read - Through Development Activities Eloxx read - through program is pursuing product candidates with the following characteristics: Activity independent of gene size or complexity of genetic disorder Molecular scaffold with defined ribosomal effect Active at all three premature stop codo
Slide 7: Defined Ribosomal Binding Site
Defined Ribosomal Binding Site • Well defined molecular interaction with helix 44 • Role in stabilization of tRNA binding at Site A • Optimizing scaffold by altering interaction with prokaryotic and mitochondrial ribosomes • Defined tissue penetration and tolerability profile for read - through appl
Slide 8: Discovery of the Eloxx Novel Compound Library
Novel compounds derived from aminoglycoside scaffold Screened for read - through activity on known disease related nonsense mutations Reduced mitochondrial inhibition (range 12 - 140X) Reduced prokaryotic ribosomal inhibition Discovery of the Eloxx Novel Compound Library Increased Selectivit
Slide 9: Focus on High Unmet Medical Need
ELOXX Focus on High Unmet Medical Need – Nonsense mutations represent important patient segments in over 1,800 diseases – Many of these diseases have no approved therapeutics – In some diseases the nonsense patient population is appropriate size for traditional clinical development Developing No
Slide 10: Cystic Fibrosis Nonsense Mutation Patients Represent a Key Unmet Need Population
Cystic Fibrosis Nonsense Mutation Patients Represent a Key Unmet Need Population Source: Eloxx Internal Research/CFTR2 database F508del Mutations Other Mutations Nonsense Related Mutations Global Cystic Fibrosis Patients by mutation/genotype G542X W1282X R1162X R553X Other Nonsense Mutations CFTR No
Slide 11: CF swelling assay on cystic fibrosis patient organoids
A CF swelling assay on cystic fibrosis patient organoids Organoids Pre - clinical Patient Stratification Potential Use To Define Clinical Trial Populations 11 Patient Organoid without drug treatment: No Swelling of Organoids Patient Organoid with drug treatment: Swelling of Organoids
Slide 12: Homozygous Nonsense Mutation (G542X)
Homozygous Nonsense Mutation (G542X) As presented at the European Cystic Fibrosis Society 41 st Conference, Belgrade, Serbia, June 2018 100 µg/mL ELX - 02 As previously presented Cystic Fibrosis Organoid Responsive to ELX - 02, Enhanced by Combination 0 20 40 60 80 100 120 100 120 140 160 180 20
Slide 13: Complex Heterozygous Nonsense Mutation (G542X:R1066C)
Complex Heterozygous Nonsense Mutation (G542X:R1066C missense) As presented at the European Cystic Fibrosis Society 41 st Conference, Belgrade, Serbia, June 2018 100 µg/mL ELX - 02 As previously presented Cystic Fibrosis Organoid Responsive to ELX - 02, Enhanced by Combination 0 20 40 60 80 100
Slide 14: Complex Heterozygous Nonsense Mutation (R1162X:F508del)
As presented at the European Cystic Fibrosis Society 41 st Conference, Belgrade, Serbia, June 2018 100 µg/mL ELX - 02 As previously presented Cystic Fibrosis Organoid Responsive to ELX - 02 Complex Heterozygous Nonsense Mutation (R1162X nonsense:F508del) 0 20 40 60 80 100 120 100 120 140 160 Tim
Slide 15: Complex Heterozygous Nonsense Mutation (G542X:F508del)
Complex Heterozygous Nonsense Mutation (G542X:F508del) As presented at the European Cystic Fibrosis Society 41 st Conference, Belgrade, Serbia, June 2018 100 µg/mL ELX - 02 As previously presented Cystic Fibrosis Organoid Responsive to ELX - 02, Enhanced by Combination 0 20 40 60 80 100 120 100
Slide 16: Homozygous Deletion Mutation (F508del) Not Responsive to ELX-02
Homozygous Deletion Mutation (F508del) Cystic Fibrosis Organoids Without Nonsense Mutations are Not Responsive to ELX - 02 As presented at the European Cystic Fibrosis Society 41 st Conference, Belgrade, Serbia, June 2018 100 µg/mL ELX - 02 As previously presented 0 20 40 60 80 100 120 90 100 11
Slide 17: ELX-02 Clinical Development – Phase 1 Studies
CLINICALTRIALS.GOV Identifier: NCT03292302 A Phase 1a, Randomized, Double - blinded, Placebo - Controlled, Single Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ELX - 02 in Healthy Adult Volunteers CLINICALTRIALS.GOV Identifier: NCT03309605 A Phase 1, Randomized,
Slide 18: Clinical Update for ELX-02 Phase 2 in Cystic Fibrosis
√ Submitted Orphan Drug Application to EMA √ Clinical Trial Application (CTA) for Phase 2 Study received final approved by the FAMHP in Belgium Expanding MAD Study √ 4 th Cohort Completed Additional Cohorts to Evaluate Drug Concentrations Engaging with investigators on a protocol for Phase 2 t
Slide 19: Eloxx Presentation, October 18-20, 2018, Denver
19 October 18 - 20, 2018 · Denver, CO Eloxx Presentation “Measuring mRNA Levels in Cystic Fibrosis Organoids with Nonsense Mutations Following Treatment with ELX - 02” October 18
Slide 20: Clinical Update for ELX-02 Phase 2 in Cystinosis
√ FDA granted ELX - 02 orphan drug status in cystinosis √ IND for ELX - 02 in cystinosis is open in the US Engaging Investigators on Phase 2 protocol Will evaluate changes in cysteine levels in white blood cells Study will be posted on clintrials.gov Expect top line data on cysteine levels i
Slide 21: On Track for Advancing Pipeline in 2018
On Track for Advancing Pipeline in 2018 21 Eloxx holds global rights on these library compounds ELX - 02 Composition of Matter 2031 without extensions Library Composition of Matter from 2027 - 2038 or later Library Use Patents Expire 2036 or later Extensive Intellectual Property Portfolio Multiple N
Slide 22: Novel Compound Library has Demonstrated Activity across Multiple Orphan Diseases
Novel Compound Library has Demonstrated Activity across Multiple Orphan Diseases 22
Slide 23: Substantial Potential Ocular Orphan Opportunity
Substantial Potential Ocular Orphan Opportunity 23 Population values from National Organization for Rare Disorders (NORD) Han et al, Korean J Pediatr 2016.; Wilkin et al, Am J Med Genet 2000; Freund et al, Mol Genet Genomic Med 2016; Aparisi et al, Orphan J Rare Dis 2014
Slide 24: Usher Syndrome
Usher Syndrome • No drugs approved or in late - stage development for nonsense variants • Significant unmet medical need for nonsense forms • Over 4,000 patients in North America alone • Academic collaborations have demonstrated activity with Eloxx Novel Library Compounds • Read through • Protein ex
Slide 25: In Vitro Retinal Biocompatibility
In Vitro Retinal Biocompatibility • Studies in retinal culture have demonstrated distinct biocompatibility profile, ie , no change in retinal staining or structure 1 • Multiple Library Compounds 2 have been evaluated to date • Additional supportive preclinical studies ongoing 1 Goldmann et al. EMBO
Slide 26: Demonstration of Usher Protein Production
Demonstration of Usher Protein Production • Collaborations with the Ben - Yosef and Nagel - Wolfrum labs demonstrate potential: x Favorable safety profile in the retina x Readthrough of relevant eye - disorder mutations x Production of missing protein Goldmann et al. EMBO Mol Med 2012, 4(11):1186 -
Slide 27: Eloxx Pharmaceutical Highlights
Eloxx Pharmaceutical Highlights 27
Slide 28: Thank you.
Thank you. Cantor Global Healthcare, October 2, 2018
Eloxx Pharmaceuticals — Cantor Global Healthcare Conference 2018 on 6ix