Eloxx Pharmaceuticals : July 2023 Investor Presentation

RARE Thinking for RARE Solutions

Small Molecule Gene Therapy Leader

July 2023

Forward-looking statements

This presentation contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, including: the development of the Company's readthrough technology; the approval of the Company's patent applications; the Company's ability to successfully defend its intellectual property or obtain necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company's research and development programs and collaborations; the Company's ability to obtain applicable regulatory approvals for its current and future product candidates; the acceptance by the market of the Company's products should they receive regulatory approval; the timing and success of the Company's preliminary studies, preclinical research, clinical trials, and related regulatory filings; the ability of the Company to consummate additional financings as needed; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; including successfully integrating the combined companies; as well as those discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.

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Small molecule genetic therapy biopharma poised for potential valuation creation

Small molecule genetic therapies for nonsense mutations proven to restore full-length proteins

Ready to advance ELX-02 into Alport Syndrome pivotal study based on remission in Phase 2*

ZKN-013 from TURBO-ZM™ cleared for Phase 1

start; robust preclinical efficacy in RDEB and FAP**

Potential value by expanding into other rare kidney

and genetic diseases and cMyc cancers

/ 3	*Benlysta in lupus nephritis approved with 10% remission rate over placebo; voclosporin approved with 18% remission rate over placebo
	**RDEB/JEB: Recessive Dystrophic/Junctional Epidermolysis Bullosa; FAP: Familial adenomatous polyposis

Developing genetic therapies to treat inherited diseases with nonsense mutations

Eloxx's small molecule gene therapy solution to restore truncated proteins

Truncated peptide chain = Loss of Function

Ribosome -

Large sub-unit

Nonsense Mutations

Premature stop codon (PTC)

Ribosome -

Small sub-unit

Antibiotics

like

gentamicin

and

erythromycin are proven to restore proteins

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Our Solution: Design novel drugs with potent nonsense readthrough to restore functional proteins

• Designer aminoglycosides: Eukaryotic ribosome selective glycosides (ERSGs)

ERSGs (including ELX-02) designed for nonsense mutation readthrough1

• Up to 1,000-fold more selective than Gentamicin
• Minimal to no antibiotic activity
• Suitable chronic delivery

• Designer Macrolides: Ribosome modulating agents (RMAs)

TURBO-ZM

TURBO-ZM™ (TUning the RiBOsome

with Zikani Molecules

• Library of >2,000 RMAs including ZKN-013
• Stronger human ribosome binding affinity; minimal antibiotic activity
• Oral, well-tolerated and smaller than macrolide antibiotics

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1Data adapted from: J Med Chem. 2012 Dec 13;55(23):10630-43.1;