Eloxx Pharmaceuticals (ELOX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Pipeline and ELX-02 mechanism

• Two rare kidney disease programs target nonsense mutations: ELX-02 is being developed for Alport syndrome and ADPKD.

• ELX-02 promotes read-through of premature termination codons to restore full-length proteins; it was engineered for almost no antibiotic activity and ~100-fold lower mitochondrial toxicity than gentamicin.

• At tested doses, kidney exposure is 43x plasma exposure, supporting the PK/PD needed for efficacy in kidney programs.

• Across 145 subjects, the drug has shown a favorable safety profile, with nearly 90 patient-months of exposure.

Alport syndrome

• In three patients with collagen IV A4 nonsense mutations, eight weeks of treatment improved glomerular basement membrane structure; foot process density reached ~80% of normal versus ~50% at baseline.

• The phase II EXACT study uses change in filtration slit density (FSD) as the primary endpoint, with UPCR and other biopsy measures as secondary endpoints; a placebo group is included.

• First patients were dosed in September; recruitment and site activation are underway in the U.S., France and India, with primary-endpoint data expected mid-2027 and a recruitment update planned for January.

• The study targets a 20%-25% FSD increase; FDA discussions are planned after the primary-endpoint readout, with attention to FSD and proteinuria correlations.

• Estimated global target population is 7,000-8,000 patients across Europe, the U.S. and Japan; estimated annual pricing of $500,000-$700,000 per patient implies a ~$4B global TAM.

ADPKD program

• ADPKD is linked to PKD1 or PKD2 mutations; tolvaptan is the only approved drug described, with tolerability challenges including polyuria and liver safety issues.

• Preclinical work showed restored protein expression and reduced cyst formation, cyst volume or cyst growth in kidney organoids, patient epithelial cells and patient-derived cysts.

• Planned study includes nonsense mutations in PKD1 and PKD2 and Mayo Imaging Classification 1B patients; the one-year, 1:1 placebo-controlled study will enroll 60 patients and assess total kidney volume.

• First-patient enrollment is expected in Q3 2027, with 52-week study data expected in H1 2029; the one-year design is intended to support discussions on breakthrough designation and a potential accelerated-approval path.

• Estimated global addressable population is ~50,000 after excluding patients with eGFR below 30 and applying an upper eGFR cutoff; at an estimated $400,000 annual price, the potential addressable market is ~$20B.