Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — FY2017 Earnings Presentation
19 slides from Eloxx Pharmaceuticals's earnings presentation, Mar 19, 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 FY2017 Webcast & Conference Call March 20, 2018 Exhibit 99.2
Slide 2: 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 cautioned not to place undue
Slide 3: Eloxx Pharmaceuticals Highlights
Key positive organoid data in Cystic Fibrosis Heterozygous and homozygous CFTR mutations Key positive model data in Cystinosis Reduction of kidney cystine levels On track for completion of Phase 1 studies SAD completed MAD enrolling Initiation of Phase 2 studies in Cystic Fibrosis and Cystinosis (4
Slide 4: The Promise of Read-Through
The Promise of Read-Through >1,800 Genetic diseases involve nonsense mutations In every genetic disease a subset of patients have nonsense mutations that impair the production of essential proteins Translational read through is directed at restoring the production of full length proteins by overcomi
Slide 5: 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 applicat
Slide 6: 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, Doubl
Slide 7: Our Current Development Pipeline
ELX-02 Cystic Fibrosis Cystinosis ELX Library Compounds Our Current Development Pipeline Phase 1a SAD Phase 1b MAD Submission CTA (Belgium) IND (FDA) Phase 2* Preclinical Mutational Profiling Candidate Nomination ELX-02 and the ELX Library Compounds are investigational agents and have not been appro
Slide 8: Cystic Fibrosis Development Program
Most prevalent genetic disease in the western world CF is the most common fatal inherited disease in Caucasians 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 cha
Slide 9: Cystic Fibrosis: CFTR Molecular Defect
Premature stop codons or nonsense mutations are Class I Estimated that 22% of patients have Class I mutations on one or both CFTR alleles Approximately 13% of the CF patients carry a nonsense mutation on one or both CFTR alleles Eloxx’s development path for read-through therapeutics will be focused
Slide 10: 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 Patient Organoid without drug treatment: No Swelling of Organoids Patient Organoid with drug treatment: Swelling of Organoids 11/13/2017 | Used by
Slide 11: Heterozygous nonsense mutations
Heterozygous nonsense mutations First investigational read-through agent to demonstrate in vitro activity in organoid cultures
Slide 12: Homozygote nonsense mutations
Homozygote nonsense mutations First investigational read-through agent to demonstrate in vitro activity in organoid cultures G542X/G542X W1282X/W1282X-A Early-stage data involve key homozygous nonsense mutations G542X prevalence estimated at 5% of CF population W1282X prevalence est. at 4% of CF pop
Slide 13: ELX-02 Cystic Fibrosis Next Steps
ELX-02 Cystic Fibrosis Next Steps Jan 2018 Pre-CTA (Belgium) Regulatory Meeting On track for mid-2018 CTA (Belgium) Submission Targeting 4Q 2018 for FPFV Phase 2 Study
Slide 14: Cystinosis Development Program
Ultra-rare lysosomal storage disease Caused by mutations in cystinosin (CTNS) Cysteine efflux channel Cystine lysosomal accumulation causes manifestations of disease The current standard of care, Cysteamine acts within the lysosome to convert cystine into forms which can exit the lysosome via cystei
Slide 15: ELX-02 Cystinosis Next Steps
On track for mid-2018 IND Submission in US ELX-02 Cystinosis Next Steps Dec 2017 Pre-IND FDA (Written Response) Targeting 4Q2018 for FPFV Phase 2 Study
Slide 16: 2017 Summary Financial Results
2017 Summary Financial Results
Slide 17: Financial Summary
Financial Summary $24 million cash as of December 31, 2017 No debt Funded through at least the end of the first quarter 2019 Shares outstanding totals 27.5 million Traded OTC: ELOX
Slide 18: Eloxx Pharmaceuticals Highlights
Key positive organoid data in Cystic Fibrosis Heterozygous and homozygous CFTR mutations Key positive model data in Cystinosis Reduction of kidney cystine levels On track for completion of Phase 1 studies SAD completed MAD enrolling Initiation of Phase 2 studies in Cystic Fibrosis and Cystinosis (4
Slide 19: Thank you.
Thank you. FY2017 Webcast & Conference Call March 20, 2018