Eloxx Pharmaceuticals (ELOX) investor relations material

Company overview and business model

• Clinical-stage biopharmaceutical company focused on small molecule therapies that modulate the ribosome to promote readthrough of premature stop codons (nonsense mutations) in genetic diseases.

• Lead candidate exaluren targets rare kidney diseases, notably Alport syndrome with nonsense mutations (NMAS), and is also being explored for autosomal dominant polycystic kidney disease (ADPKD).

• ZKN-013, an oral ribosome modulating agent, is licensed to Almirall for rare skin diseases (RDEB, JEB) and could yield up to $470M in milestones plus royalties.

• The company’s approach addresses a significant unmet need, as nonsense mutations account for 10–12% of patients with genetic diseases.

Financial performance and metrics

• Net losses: $6.0M (2025), $3.1M (2024), $17.1M (2023); accumulated deficit of $308.8M as of June 30, 2026.

• Cash and cash equivalents: $62.0M as of June 30, 2026, bolstered by a $58.3M public offering in June 2026.

• Research and development expenses increased to $4.5M for the six months ended June 30, 2026, up from $1.4M in the prior year period.

• The company expects to continue incurring substantial operating losses and will require additional funding to reach profitability.

Use of proceeds and capital allocation

• No proceeds from resale of shares by selling stockholders; up to $0.5M may be received from cash exercise of pre-funded warrants, to be used for general corporate purposes.

• Recent capital infusions include $15M from Coastlands and $2M from Domicilium in August 2025, and a $58.3M public offering in June 2026.