Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — Investor Presentation (February 2018)
25 slides from Eloxx Pharmaceuticals's investor presentation, February 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 Investor Presentation February 2018 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements Certain statements included in this presentation are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These include statements of management’s intentions, belief, plans and future expectations and, therefore, you are ca
Slide 3: Eloxx Pharmaceuticals Highlights
Eloxx Pharmaceuticals Highlights Clinical stage biopharmaceutical company developing novel Leading Read small molecule medicines designed to treat genetic diseases Through Company by restoring the production of proteins from genes with nonsense mutations Experienced Management team with established
Slide 4: Highly Experienced Leadership Team
Highly Experienced Leadership Team Pedro Huertas, Robert Ward MD, PhD Chairman and CEO CMO John van Duzer, Gregory Weaver Ph.D. CFO VP CMC Neil Sharpe, Ph.D. Barbara Ryan Investor Relations VP Translational Sci. 4 4
Slide 5: The Promise of Read-Through
The Promise of Read-Through Aminoglycosides first showed read-through activity in nonsense mediated diseases Advances in our Aminoglycosides understanding of tolerability profile has translational read-through limited suitability for read enables design of novel through treatment of serious genetic
Slide 6: Target Profile for Read-Through
Target Profile for Read-Through 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 codons • Reduces
Slide 7: Aminoglycoside Ribosomal Interaction
Aminoglycoside Ribosomal Interaction • 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
Slide 8: Discovery of ELX-02
Discovery of ELX-02 ü Novel compounds derived from aminoglycoside scaffold ü Screened for read-through activity on known disease related nonsense mutations ü Reduced mitochondrial inhibition (range 12-140X) ü Reduced prokaryotic ribosomal inhibition Increased Selectivity towards Cytoplasmic versus M
Slide 9: ELX-02 Preclinical Development
ELX-02 Preclinical Development • IND Enabling Studies • Functional and anatomic hearing studies • No observation of ototoxicity • Histopathology and functional renal studies • Indication of improved NOAEL margin • Currently anticipate dosing without adjustment for renal impairment • On track for mid
Slide 10: ELX-02 Clinical Development
ELX-02 Clinical Development ClinicalTrials.gov Identifier: NCT03292302 Completed ClinicalTrials.gov Identifier: A Phase 1a, Randomized, Double- NCT03309605 Ongoing blinded, Placebo-Controlled, Single Dose Escalation Study to Evaluate A Phase 1, Randomized, Double- the Safety, Tolerability, and Blind
Slide 11: Cystinosis Development Program
Cystinosis Development Program • Ultra-rare lysosomal storage disease • Caused by mutations in cystinosin (CTNS) • Cysteine efflux channel • Cystine lysosomal accumulation causes manifestations of disease • It is generally recognized that the current standard of care (cysteamine administration) stim
Slide 12: ELX-02 Preclinical Cystinosis
ELX-02 Preclinical Cystinosis In vitro model indicates ELX-02 reduces nonsense in vitro model mediated decay CTNSW138X/W138X (NMD) fibroblasts In vitro model indicates ELX-02 restores Cystinosin transporter function 12
Slide 13: ELX-02 Animal Model Cystinosis
ELX-02 Animal Model Cystinosis CTNSY226X/Y226X knock-in 21 Days of Biweekly ELX-02 Administration Dr Paul Goodyer McGill University Significantly Reduced Kidney Cystine Levels 13
Slide 14: ELX-02 Cystinosis Next Steps
ELX-02 Cystinosis Next Steps Ö Dec 2017 Pre-IND FDA Discussion • On track for mid-2018 IND Submission in US • Targeting 4Q 2018 for FPFV Phase 2 Study 14
Slide 15: Cystic Fibrosis Development Program
Cystic Fibrosis Development Program • Systemic rare disease • Caused by mutations in transmembrane conductance regulator (CFTR) • Chloride channel • Mutations lead to dysregulation in multiple organ systems • Current standard of care based on molecular Zoltan Bozoky et al. PNAS chaperones for traffi
Slide 16: Cystic Fibrosis
Cystic Fibrosis CFTR Molecular Defect • Premature stop codons or nonsense mutations are Class 1 • There are currently no approved therapies for Class 1 • G542X represents approximately 5% of the total CF patient population • Eloxx’s development path for read-through therapeutics will be focused on t
Slide 17: Goals of Cystic Fibrosis Personalized Medicine Approach
Goals of Cystic Fibrosis Personalized Medicine Approach • Development path focused on individual’s genetic background (ie, CFTR mutation) • Today most patients have genetic sequence data that could enable personalized treatment 17
Slide 18: Cystic Fibrosis: First Organoid Clinical Success
Cystic Fibrosis: First Organoid Clinical Success Our first patient in 2014 First 6 patients successfully treated based on organoid diagnosis. CF patient 18 23
Slide 19: Organoids For Cystic Fibrosis Screening
Organoids For Cystic Fibrosis Screening A CF assay on cystic fibrosis patient organoids Healthy CFTR activation: CF mutated CFTR activation: Swelling of Organoids No-Swelling of Organoids 19 24 11/13/2017 Confidential
Slide 20: Organoids Pre-clinical Patient Stratification
Organoids Pre-clinical Patient Stratification Can Be Used To Define Clinical Trial Populations A CF swelling assay on cystic fibrosis patient organoids Patient Organoid without drug treatment: Patient Organoid with drug treatment: No Swelling of Organoids Swelling of Organoids 20 25 11/13/2017 Confi
Slide 21: ELX-02 Example Organoid Results
ELX-02 Example Organoid Results 21
Slide 22: ELX-02 Cystic Fibrosis Next Steps
ELX-02 Cystic Fibrosis Next Steps Ö Jan 2018 Pre-CTA (Belgium) Regulatory Meeting • On track for mid-2018 CTA (Belgium) Submission • Targeting 4Q2018 for FPFV Phase 2 Study 22
Slide 23: Our Current Development Pipeline
Our Current Development Pipeline 2017 2018 2019 2020 ELX-02 Phase 1a Phase 1b MAD Submission Cystic Fibrosis CTA (Belgium) Phase 2 Cystinosis IND (FDA) ELX Library Compounds Candidate Preclinical Mutational Profiling Nomination ELX-02 and the ELX Library Compounds are investigational agents and have
Slide 24: Eloxx Pharmaceuticals Highlights
Eloxx Pharmaceuticals Highlights March 20 FY2017 Earnings Call Suntrust 4th Annual Orphan Drug Day Feb 13, 2018 Cowen 38th Annual Health Care Conference March 12, 2018 24 24
Slide 25: Thank you!
Thank you! 25 25
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