Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX

Eloxx Pharmaceuticals — Investor Presentation (September 2026)

33 slides from Eloxx Pharmaceuticals's investor presentation, September 2026

  1. Slide 1: A precision approach to rare kidney disease

    A precision approach to rare kidney disease Small molecule based genetic therapy Corporate Presentation | September 2026

  2. Slide 2: Legal Disclaimer

    Legal Disclaimer Information in this presentation and the accompanying oral presentation contains forward-looking statements that involve substantial risks and uncertainties about Eloxx Pharmaceuticals, Inc. and its consolidated subsidiaries (collectively, the “Company”) and the industry in which th

  3. Slide 3: Company highlights

    Company highlights Disease-modifying potential across genetic renal diseases Exaluren reads through premature termination codons to restore full-length protein Phase 2 data in Alport (NMAS) demonstrated structural improvement Phase 2b ongoing, with 16-week topline data expected in mid-2027 ADPKD pro

  4. Slide 4: Exaluren: novel synthetic aminoglycoside restoring full-length protein by PTC readthrough

    Exaluren: novel synthetic aminoglycoside restoring full-length protein in nonsense mutations by PTC readthrough Up to 9-fold greater gene-agnostic readthrough than gentamicin* No evidence of readthrough of native stop codon (NSC) Partially functional full-length proteins are expected to be clinicall

  5. Slide 5: Exaluren naturally accumulates in kidney

    Exaluren naturally accumulates in kidney Estimated human exposure in plasma and kidney at 0.75 mg/kg 43X 393,000 • Exaluren like other AUC0-24h (ng*hr/mL) aminoglycosides binds to LRP2 protein megalin • Megalin is found in the glomerulus and proximal tubules in the kidney, the inner ear and eye 9,10

  6. Slide 6: Eight weeks of exaluren restored collagen IV and improved kidney structure in all three patients

    Eight weeks of exaluren restored collagen IV and improved kidney structure in all three patients FIRST-IN-HUMAN PROOF OF CONCEPT · n=3 Phase 2a (EL-014): 3 UK patients with COL4A4 nonsense mutation · 8 weeks of exaluren 0.75 mg/kg SC daily · open-label · 12 weeks follow up 1. Structure Improved: 2.

  7. Slide 7: Extensive safety database supports development

    Extensive safety database supports development Exaluren clinical and nonclinical data: no dose-limiting toxicities and no serious AE’s were attributed to exaluren 145 No detected 9.7–10× subjects treated with readthrough of native stop exposure margin at target exaluren codons dose of 0.75 mg/kg — 8

  8. Slide 8: Two Phase 2 programs with data readouts over 24 months

    Two Phase 2 programs with data readouts over 24 months Alport Syndrome: NMAS NM-ADPKD Phase 2b ongoing. First patient Expected Phase 2 study 3Q 2026 start in 2026 Primary Endpoint: Change in Primary Endpoint: Change in FSD in kidney biopsy htTKV growth (MRI) Readout: Mid-2027 Readout: Mid-2028* Prev

  9. Slide 9: NMAS: Loss of collagen trimer leads to structural damage

    NMAS: Loss of collagen trimer leads to structural damage Healthy Kidney Alport Kidney GBM irregularities (lamellation) COL4A3, COL4A4 or COL4A5 nonsense mutation Protein loss (proteinuria), irreversible progression to ESRD Loss of Collagen 345 (IV) Foot process trimer effacement (FPE) and podocyt

  10. Slide 10: Structural damage of foot processes is an independent prognostic marker for future kidney failure

    Structural damage of foot processes (FPE) is an independent prognostic marker for future kidney failure Adjusted survival curve for association between FPE and time-to-kidney failure1 / 10 1 Wittig M, Verma A, Bellavia A, et al. The Associations of Foot Process Effacement with Kidney Histopathologic

  11. Slide 11: NMAS patients are rapid progressors with increasing UPCR trajectory

    NMAS patients are rapid progressors with increasing UPCR trajectory RaDaR natural history data Rapid progressors: Nonsense mutations are a subset of X-linked male and Autosomal recessive AS patients / 11 Wong et al. 2026

  12. Slide 12: Phase 2a: First-in-human proof of structural improvement in NMAS patients after 8 weeks

    Phase 2a: First-in-human proof of structural improvement in NMAS patients after 8 weeks exaluren treatment Foot process effacement (FPE) and GBM changes in Phase 2a trial (Representative TEM images)* 4402-01 Foot process 4401-01 4401-02 Effaced foot process Widespread FPE Widespread FPE Moderate to

  13. Slide 13: Phase 2a: Improvements in FPW and GBM width confirmed structural improvement

    Phase 2a: Improvements in FPW and GBM width confirmed structural improvement TEM Assessment using Systematic Uniform Random Sampling (analysis blinded to treatment order) FPW: Reduced in all patients GBM width: Improved relative to baseline Thin membrane increased while thicker membrane at baseline

  14. Slide 14: FPE assessed by change in Filtration Slit Density

    FPE assessed by change in Filtration Slit Density (FSD) Unbiased assessment of filtration slit density Immuno- AI based glomeruli selection in FFPE Image acquisition with 3D- FSD = Filtration fluorescence tissue Structured Illumination Slit Length/ staining* Microscopy (3D-SIM) Podocyte area Filtrat

  15. Slide 15: Phase 2a: FSD increased consistent with FPE reduction

    Phase 2a: FSD increased consistent with FPE reduction Mean and distribution of FSD in Phase 2a clinical trial patients, m-1 (% of healthy) after 8 weeks of treatment 1.00±0.60 1.50±0.57 1.54±0.74 1.75±0.46 0.80±0.56 1.73±0.63 (48.5%) (72.8%) (74.8%) (85%) (38.8%) (84%) Healthy=2.06±0.211 EOT= 1.66±

  16. Slide 16: Phase 2a: Mean 72% increase in average collagen α3 and α4

    Phase 2a: Mean 72% increase in average collagen 3 (IV) and 4 (IV) across all patients Change in collagen 4 expression in Phase 2a trial (blinded assessment) 4401-01 4401-02 4402-01 8 8 Reduced α4 intensity 8 reflects redistribution from Maximum Intensity / Mean intensity 7 7 intracellular staining

  17. Slide 17: Phase 2a: Collagen α5(IV) GBM localization consistent with collagen IV formation

    Phase 2a: Collagen 5(IV) GBM localization consistent with collagen IV formation COL4A5 immunofluorescence staining in fresh frozen tissue biopsies 4401-01 4401-02 4402-01 Reference: COL4A5 (green) globally COL4A5 (green) globally COL4A5 (green) partially Disease localized to Bowman’s capsule locali

  18. Slide 18: Phase 2a: Collagen α5(IV) localization and evidence of trimer formation after eight weeks

    Phase 2a: Collagen 5(IV) localization and evidence of trimer formation consistent after eight weeks (n=3) Collagen 5(IV) 3D-Structured Illumination Microscopy immunostaining 4402-01 (EOT) - Detail COL4A5 REF 1 µm 1 µm Collagen α5(IV) in 3D-SIM: Baseline (Top): Diffuse and low abundance non-specifi

  19. Slide 19: UPCR trends in Phase 2a trial

    UPCR during treatment period UPCR trends in Phase 2a trial, log (mg/g)* Baseline UPCR 4401-01 4401-02 4402-01 4.00 4.00 4 3.50 3.50 3.5 Log10(UPCR), mg/g Log10(UPCR), mg/g Log10(UPCR), mg/g 3.00 3.00 3 2.50 2.50 2.5 2.00 2.00 2 1.50 1.50 1.5 1.00 1.00 1 0 2 4 6 8 12 16 20 0 2 4 6 8 12 16 20 0 2 4 6

  20. Slide 20: FSD increases predicted to reduce UPCR

    FSD increases predicted to reduce UPCR Analysis of published FSD and UPCR correlations across kidney diseases / 20 Eloxx Analysis based on Data from Kim et al J. Am. Soc. Nephrol. 1995; 5: 1659-1668; Data replotted from Kim et al. Age ranges 4- 26 years. FSD assessment using TEM image analysis of ki

  21. Slide 21: FDA supported working group ASSENT launched to explore endpoints for Alport

    FDA supported working group “ASSENT” launched to explore biopsy and clinical endpoints for Alport An established FDA endpoint playbook for approvals in rare kidney disease PARASOL → FSGS ASSENT → Alport ESTABLISHED PRECEDENT OUR OPPORTUNITY 1 FDA-backed consortium (2023) 1 FDA-initiated working grou

  22. Slide 22: The EXACT Study: 2b placebo-controlled NMAS study planned with FSD primary endpoint

    The EXACT Study: 2b placebo-controlled NMAS study planned with FSD primary endpoints Randomized, double-blind, placebo-controlled, delayed-start Phase 2b study. Phase 2b Randomized, double-blind Placebo-controlled · delayed-start N = 24 · age ≥ 12 ▲ Kidney biopsy PART 1 — 16 weeks · randomized 2:1 P

  23. Slide 23: Advancing exaluren in nonsense mutation Autosomal Dominant Polycystic Kidney Disease

    Advancing exaluren in nonsense mutation Autosomal Dominant Polycystic Kidney Disease Healthy Kidney ADPKD Kidney PKD1 or PKD2 Structural consequence nonsense mutation • Ciliary pathways disrupted • Increased epithelial-cell proliferation • New cyst formation and growth • Total kidney volume growth L

  24. Slide 24: Exaluren restored 10 to 75% of PC1 and reduced cysts by ~50% in kidney organoids

    Exaluren restored 10 to 75% of PC1 and reduced cysts by ~50% in kidney organoids PC1 expression and new cyst formation in organoids treated with exaluren for 14 days PC1 expression in iPSC organoids, % WT* Cyst prevention in iPSC organoids** * Quantification of immunoblot PC1 fold change following t

  25. Slide 25: Regulus clinical data link partial PC1 restoration with reduced htTKV growth

    Regulus clinical data link partial PC1 restoration with reduced htTKV growth Reported results of farabursen (RGLS8429) anti-miRNA-17 ASO 3-month (Q2W) treatment* Changes in PC1 expression Changes in htTKV growth** (MeanDay85-133 vsBaseline) Percent ChangePC1/CD133 Percent ChangehtTKV (EOSvsBaseline)

  26. Slide 26: Eloxx is the most advanced independent ADPKD asset

    Eloxx is the most advanced independent ADPKD asset Comparison of ADPKD programs in clinical development Company / Development Addressable US Acts on ADPKD evidence asset stage population¹ Novartis Wild-type allele PKD1 and Clinical (Regulus) PKD2. Indirect miR-17 Urinary PC1/PC2 increased; htTKV gro

  27. Slide 27: Shared mechanism with clinical proof of concept supports Alport and ADPKD programs

    Shared mechanism with clinical proof of concept supports Alport and ADPKD programs Summary Evidence Nonsense Mutation Alport Syndrome Nonsense Mutation ADPKD Platform ✓ Gene-agnostic PTC readthrough ✓ Same Mechanism Protein +72% Collagen α3/α4 (IV). Functional +10% to +75% of wild-type Polycystin- r

  28. Slide 28: Experienced leadership in rare disease drug development

    Experienced leadership in rare disease drug development Key Eloxx Leadership Sumit Aggarwal Dr. Teji Singh President and CEO Head of Clinical Development • Reshaped Eloxx pipeline and executed strategic • Seven primary product first approvals across rare agreements (Cystic Fibrosis foundation and Al

  29. Slide 29: Demonstrated ability to execute high value pharma collaborations

    Demonstrated ability to execute high value pharma collaborations Overview of global license agreement with Almirall for ZKN-013 executed in 2024 ZKN-013 global Eloxx eligible to receive up to ~$470 development and Potential for Priority million in development, regulatory commercialization rights to

  30. Slide 30: Multiple high impact clinical readouts anticipated from 2026 - 2028

    Multiple high impact clinical readouts anticipated from 2026 - 2028 Anticipated key value inflection points: Phase 2b primary endpoint (2027) and ADPKD proof- of-concept (2028) Alport syndrome 16-week Primary Endpoint 32-week End of Study  Phase 2b FPI* 3Q Fully Enrolled 1Q Readout Mid-2027 Readout

  31. Slide 31: June 2026 follow-on provides expected runway to Alport and ADPKD readouts

    June 2026 follow-on provides expected runway to Alport and ADPKD readouts Balance sheet (as of June 30, 2026) • Cash of $62.0M • Runway to mid-2028 • 12.5 million fully diluted shares, including 4.04 million common stock and 7.38 million pre-funded warrants Transaction • 2,975,000 shares at $11.00 p

  32. Slide 32: Human proof-of-concept with two clinical rare kidney disease programs

    Human proof-of-concept with two clinical rare kidney disease programs Human proof of mechanism: protein restoration and structural reversal in human kidney biopsies Same drug, two rare kidney diseases: Expected mid-2027 Alport readout supports both programs. Proof-of-Concept Phase 2 in ADPKD readout

  33. Slide 33: IR contacts

    I N V E S T O R R E L AT I O N S WEB ir@eloxxpharma.com eloxxpharma.com / 33

Eloxx Pharmaceuticals — Investor Presentation (September 2026) on 6ix