Eloxx Pharmaceuticals : October 2023 Investor Presentation

Small Molecule Gene Therapy

October 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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Clinical stage small molecule gene therapy biopharma poised for value creation

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Small molecule genetic therapies for nonsense mutations proven

to restore full-length proteins

ELX-02: Ready for Alport	ZKN-013: Oral agent ready
Syndrome pivotal study with	for Phase 1 start; robust
biopsy confirmed disease	preclinical efficacy in RDEB and
regression. Preclinical POC in	FAP. Strategic Discussions
ADPKD*	Ongoing

* ADPKD: Autosomal Dominant Polycystic Kidney Disease

Two clinical stage drugs designed to treat inherited diseases with nonsense mutations

Nonsense mutation overview and MOA of Eloxx therapies

Truncated peptide chain		ELX-02 and
= Loss of Function		ZKN-013 have
Ribosome -		proven
ZKN-013 (designed	mechanism of
Large sub-unit	with TURBO-ZM™)	action with
Nonsense Mutations
	demonstrated
Premature stop codon		superior
(PTC)		functional
		protein
Ribosome -	ELX-02	restoration*
Small sub-unit

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* ELX-02 and ZKN-013 are rationally designed molecules with superior efficacy and safety to Gentamicin and Erythromycin

Focused on treating rare genetic nonsense mutation Alport Syndrome and other kidney diseases

Indication

Alport

Syndrome (nonsense)

Protein

restored

Collagen IV

Discovery

Lead	IND-
optimization	enabling

ELX-02 (SC)

Phase 1 -

first-in-human

Phase 2

Preparing for Phase 3

RDEB/JEB	Collagen	ZKN013 (oral)	IND Cleared
(nonsense)	VII/LAMB3
FAP	APC	ZKN013 (oral)
(nonsense)
Class 1 CF	CFTR	RMAs (oral)
ADPKD	PKD1/PKD2	ELX-02 (SC)
(nonsense)

In active strategic discussions

Expansion potential in rare kidney diseases

/ 5	Class 1 CF: Cystic fibrosis patients with class1 mutations; RDEB/JEB: Recessive Dystrophic/Junctional Epidermolysis Bullosa; FAP: Familial adenomatous polyposis; PoC: Proof of concept; SC:
	Subcutaneous; IND: Investigational New Drug