Eloxx Pharmaceuticals : Topline ELX-02 Phase 2 Results Call and Webcast

RARE Thinking for RARE Solutions

Topline ELX-02 Phase 2 Cystic Fibrosis Results

November 17, 2021

Forward-looking statements

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This presentation (and the accompanying oral discussion) contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation, statements regarding our expected cash burn and future financial results, the expected timing of trials and results from clinical studies of our product candidates and the potential of our product candidate to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words "aim," "may," "will," "would," "should," "expect," "explore," "plan," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential," "seeks," or "continue" or the negative of these terms similar expressions, although not all forward-looking statements contain these words.

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All forward-looking statements speak only as of the date of this presentation and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

This presentation also contains estimates, projections and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events, or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical, and general publications, government data and similar sources.

Opening Remarks

Sumit Aggarwal President and CEO

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Today's agenda

Topic	Speaker
Opening Remarks	Sumit Aggarwal, President & CEO
Remaining Unmet Need in Cystic Fibrosis &	Dr. Eitan Kerem
ELX-02 Topline Phase 2 Results
ELX-02 Next Steps &Program Expansion	Dr. Vijay Modur, Head of R&D
Key Takeaways & Closing Remarks	Sumit Aggarwal, President & CEO

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ELX-02 is first-in-class drug in Phase 2 development for Class I CF patients with nonsense mutations

ELX-02 designed as superior readthrough agent to Gentamicin

Compelling preclinical activity observed in highly translatable models

Fast Track Designation from FDA for treatment of CF patients

with nonsense mutations

Phase 2 monotherapy trial designed to evaluate safety and biological

activity

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