Eloxx Pharmaceuticals, Inc. · NASDAQ:ELOX
Eloxx Pharmaceuticals — Investor Presentation (October 2026)
34 slides from Eloxx Pharmaceuticals's investor presentation, October 2026
Slide 1: A precision approach to rare kidney disease
A precision approach to rare kidney disease Small molecule based genetic therapy Corporate Presentation | October 2026
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
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
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
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 AUC0-24h (ng*hr/mL) • Exaluren, like other aminoglycosides, binds to the LRP2 protein megalin • Megalin is found in the glomerulus and proximal tubules in the kidney, the inner ear and th
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. P
Slide 7: Extensive safety database supports development
Extensive safety database supports development Exaluren clinical and nonclinical data: no dose-limiting toxicities and no serious AEs were attributed to exaluren 145 No detected ~10× subjects treated with readthrough of native stop exposure margin at target exaluren codons dose of 0.75 mg/kg — 89.4
Slide 8: Two Phase 2 programs with data readouts in 2027 and 2029
Two Phase 2 programs with data readouts in 2027 and 2029 Alport Syndrome: NMAS NM-ADPKD Phase 2b ongoing. First Phase 2 IND allowed. First patient 3Q 2026 patient 3Q 2027 Primary Endpoint: Change in Primary Endpoint: Change in FSD in kidney biopsy htTKV growth (MRI) Readout: Mid-2027 Readout: 1H 202
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 a3a4a5 (IV) Foot process trimer effacement (FPE) and podocyt
Slide 10: Structural damage of foot processes (FPE) is an independent prognostic marker for 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 failure 1 / 10 1 Wittig M, Verma A, Bellavia A, et al. The Associations of Foot Process Effacement with Kidney Histopathologi
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
Slide 12: Phase 2a: First-in-human proof of structural improvement in NMAS patients
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
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
Slide 14: FPE assessed by change in Filtration Slit Density (FSD)
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
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, mm-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±
Slide 16: Phase 2a: Mean 72% increase in collagen a3 (IV) and a4 (IV)
Phase 2a: Mean 72% increase in collagen a3 (IV) and a4 (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 reflects 8 7 7 redistribution from intracellular 7 Maximum Intensity / Mean intensity staining to GB
Slide 17: Phase 2a: Collagen a5(IV) GBM localization consistent with collagen IV formation
Phase 2a: Collagen a5(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
Slide 18: Phase 2a: Collagen a5(IV) localization and evidence of trimer formation
Phase 2a: Collagen a5(IV) localization and evidence of trimer formation consistent after eight weeks (n=3) Collagen a5(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-speci
Slide 19: UPCR during treatment period
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 /
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 range 4-26 years. FSD assessment using TEM image analysis of ki
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 1 FDA-backed consortium (2023) 1 FDA-initiated working group for Alport approval endpoints (Dec 2
Slide 22: The EXACT Study: ongoing Phase 2b placebo-controlled NMAS study
The EXACT Study: ongoing Phase 2b placebo-controlled NMAS study with FSD primary endpoint 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
Slide 23: Advancing exaluren in nonsense mutation ADPKD
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 Loss of polycystin • Total kid
Slide 24: Nonsense-mutation ADPKD: a meaningful commercial opportunity
Nonsense-mutation ADPKD: a meaningful commercial opportunity NONSENSE MUTATION FREQUENCY ESTIMATED US + EU GENETIC POPULATION** ≈ 1 in 4* US: 40-50K, EU: 30-50K Illustrative 100 patients Patients with a PKD1 or PKD2 nonsense mutation US Annual Total Addressable Market $9.2–$11.5B to $15.6–$28.3B Ass
Slide 25: Exaluren restored 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
Slide 26: Exaluren increased PKD1 transcript expression and corrected ciliary abnormalities
Exaluren increased PKD1 transcript expression and corrected two ciliary abnormalities in patient primary epithelial cells 72-hour treatment of primary PKD1 Tyr1599Ter patient derived HuREC cells* PKD1 mRNA expression Cilia present Abnormal cilia % of cells with an “antenna” % cilia with abnormal bul
Slide 27: Independent clinical evidence links partial PC1 restoration with reduced htTKV growth
Independent clinical evidence links 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** Percent Change PC1/CD133 (MeanDay85-133 vs Baseline) Percent Change htTKV (EO
Slide 28: EXPRESS: Designed for a robust treatment effect and potential Breakthrough Therapy Designation
EXPRESS: Designed for a robust treatment effect and potential Breakthrough Therapy Designation Global Phase 2 · US and EU orphan designation · Full-year htTKV primary endpoint PHASE 2 RANDOMIZED · 1:1 DOUBLE-BLIND N ≈ 60 ADULTS (18-65) Key Inclusion criteria Exaluren 60 mg SC daily (n≈30) • PKD1/ PK
Slide 29: Experienced leadership in rare disease drug development
Experienced leadership in rare disease drug development Key Eloxx Leadership Sumit Aggarwal Dr. T.R. Srinivas Dr. Teji Singh President and CEO SVP, Chief Medical Officer Head of Clinical Development • Reshaped Eloxx pipeline and executed • Nephrologist; led U.S. Medical Affairs for • Seven primary p
Slide 30: 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 Potential for Priority Eloxx eligible to receive up to $470 development and Review Vouchers million in development, regulatory commercializa
Slide 31: Multiple high impact clinical readouts anticipated from 2026 to 2029
Multiple high impact clinical readouts anticipated from 2026 to 2029 Key inflection points: Alport (mid-2027) and ADPKD (1H 2029) readouts Alport syndrome Phase 2b FPI* 3Q 2026 Fully Enrolled 1Q 2027 16-week Primary Endpoint Readout 32-week End of Study Readout Mid-2027 4Q 2027 2026 2027 2028 2029
Slide 32: June 2026 follow-on offering and Nasdaq uplisting
June 2026 follow-on offering and Nasdaq uplisting Balance sheet (as of June 30, 2026) • Cash of $62.0M • 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 plus pre-funded warrants for 3,025,000 shares,
Slide 33: 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. 52-week EXPRESS Phase 2 in ADPKD expected
Slide 34: Investor Relations contact
I N V E S T O R R E L AT I O N S WEB [email protected] eloxxpharma.com / 34
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