Eloxx Pharmaceuticals (ELOX) Corporate presentation summary

Investment highlights

• Exaluren is positioned as a small-molecule genetic therapy for rare kidney diseases, using premature termination codon readthrough to restore full-length protein.

• Two Phase 2 programs target nonsense-mutation Alport syndrome and ADPKD, with readouts expected in mid-2027 and 1H 2029, respectively.

• June 2026 follow-on offering generated $66M gross proceeds; shares began trading on Nasdaq Capital Market on June 9, 2026.

• Cash was $62.0M as of June 30, 2026.

Exaluren mechanism and safety

• In PTC reporter assays, exaluren showed up to 9-fold greater gene-agnostic readthrough than gentamicin; no native stop-codon readthrough was detected.

• Estimated kidney exposure at 0.75 mg/kg was 43x plasma exposure, based on allometric scaling and clinical plasma PK data.

• Across 145 treated subjects and 89.4 subject-months of clinical exposure, no dose-limiting toxicities or serious AEs were attributed to exaluren.

• Reported safety findings included unchanged renal biomarkers, no detected native stop-codon readthrough, and no kidney or hearing signal in the three-patient Phase 2a study.

Alport syndrome

• In an open-label Phase 2a study of three patients with COL4A4 nonsense mutations, 8 weeks of daily exaluren improved kidney structure in all three and increased mean collagen α3/α4 (IV) by 72%.

• Blinded biopsy analyses found reduced foot process effacement, improved GBM width variability, and increased filtration slit density after treatment.

• The ongoing EXACT Phase 2b study plans to enroll 24 patients aged 12 or older; its primary endpoint is change in biopsy filtration slit density at week 16.

• Phase 2b first patient was dosed in 3Q 2026; the 16-week primary endpoint readout is expected mid-2027, with the 32-week study readout expected in 4Q 2027.

• Nonsense-mutation Alport syndrome is estimated at ~4,200 U.S. patients; orphan drug designations are in place in the U.S. and Europe.