XORTX Therapeutics : Investor Presentation

Redefining the Future of Kidney Disease with

Xanthine Oxidase Reducing Technology

June 2024

:XRTX : ANUA

Forward-Looking Statements

Certain information included in this Presentation constitutes forward-looking information or forward-looking statements under applicable securities legislation ("forward-lookingstatements"). These statements relate to future events or future performance of the Company. Forward-looking statements are statements that are not historical facts and are often, but not always, identified using words or phrases such as "can", "continue", "develop", "expect", "forecast", "future", "may", "milestone", "plan", "potential", "proposed", "will" and other similar expressions. In particular, but without limiting the foregoing, this Presentation contains forward-looking statements pertaining to, among other things: the industry in which Company operates, including the value thereof; strategic plans, including the timing thereof; the Company's operations, including with respect to accelerated approval processes, licencing deals, patent filings and clinical trials, including the timing and results thereof; the effects of end-stage renal disease and onset delay of end-stage renal disease; timing and occurrence of certain milestones, including the success of preclinical studies and clinical trials; and the Company's products, services and assets, including the benefits thereof. In addition, this Presentation may contain forward-looking statements attributed to third party industry sources.

By their nature, forward-looking statements involve known and unknown risks, uncertainties, and other factors that may cause actual results or events to differ materially from those anticipated. Such forward-looking statements are provided for the purpose of providing information about management's current expectations and plans relating to the future. Readers are cautioned that reliance on such statements may not be appropriate for other purposes, such as making investment decisions. These factors and risks include, without limitation: incorrect assessments of the value of acquisitions, licenses and development programs; technical, manufacturing and processing problems; actions by governmental authorities, including increases in taxes; the availability of capital on acceptable terms; fluctuations in foreign exchange, currency, or interest rates and stock market volatility; failure to realize the anticipated benefits from licenses or acquisitions; and potential labor unrest. This list is not exhaustive of the factors that may affect any of the Company's forward-looking statements. Some of the important risks and uncertainties that could affect forward-looking statements are described further under the heading "Key Information - Risk Factors" in its annual report on Form 20-F filed with the Securities and Exchange Commission and under the heading "Risks Related to the Business" in its management's discussion and analysis filed as an Exhibit to its annual report on Form 20-F, which annual report is available on www.sec.gov.

With respect to forward-looking statements in this Presentation, the Company has made assumptions, regarding, among other things: the availability of capital to fund planned expenditures; prevailing regulatory, tax and environmental laws and regulations; the ability to secure necessary personnel, equipment, supplies and services; the Company's ability to manage the Company's growth effectively; the absence of material adverse changes in the Company's industry or the global economy; trends in the Company's industry and markets; the Company's ability to maintain good business relationships with the Company's strategic partners; the Company's ability to comply with current and future regulatory standards; the Company's ability to protect the Company's intellectual property rights; the Company's continued compliance with third-party license terms and the non-infringement of third-party intellectual property rights; the Company's ability to manage and integrate acquisitions; the Company's ability to raise sufficient debt or equity financing to support the Company's continued growth.

Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Undue reliance should not be placed on forward-looking statements because the Company can give no assurance that such expectations will prove to be correct and such statements are based on the beliefs, estimates and opinions of the Company's management on the date such statements are made. Many factors could cause the Company's actual results, performance or achievements to vary from those described herein. Should one or more of these risks or uncertainties materialize, or should assumptions underlying forward-looking statements prove incorrect, actual results may differ materially from those described in this Presentation as intended, planned, anticipated, believed, estimated or expected.

The forward-looking statements included in this Presentation are expressly qualified in their entirety by this cautionary statement. The Company cautions that the foregoing lists of assumptions, risks and uncertainties are not exhaustive. The forward-looking statements contained in this Presentation are made as of the date hereof and the Company undertakes no obligation to update publicly or revise any forward-looking statements, whether as a result of new information, future events or otherwise, unless required by applicable securities laws.

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Pioneering New Therapies for Kidney Disease

Our mission:

Improve the quality of life of patients with progressive kidney disease by developing innovative therapies.

Our lead programs are in late-stage clinical testing and are designed to manage aberrant purine metabolism, reduce high uric acid and slow the progression of kidney disease.

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Investment Highlights

• Developing oral therapies for serious progressive kidney diseases with high unmet medical needs including autosomal dominant polycystic kidney disease (ADPKD), type 2 diabetes nephropathy (T2DN) and acute kidney injury (AKI)
• Three programs including two that are derisked and clinically advanced with previous large studies showing safety and efficacy in kidney disease with oxypurinol, and a well-understood mechanism of action
• • XRx-008is poised to begin Phase 3 under an accelerated 505(b)(2) regulatory pathway in ADPKD, an orphan indication with an addressable U.S. market of $1.0 billion to $1.8 billion annually
  • XORLO™ (working drug name) has received orphan designation from the U.S. Food and Drug Administration
• Potential for global licensing deal with high expected pharma interest due to blockbuster sales potential, and limited and poor treatment options
• Three patent families protect pipeline-in-a-producttechnology with broad therapeutic claims in the U.S. and Europe, with protection extending through 2034 and pending to 2043
• CEO Allen W. Davidoff, Ph.D. developed oxypurinol in prior ventures; board and development team have a history of building shareholder value (Cynapsus Therapeutics acquired for $624 million*, Trillium Therapeutics for $2.2 billion)

*All $ in this presentation are USD

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First-in-Class XOI Ready for ADPKD Pivotal Trial

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Common ADPKD Progression and Symptoms

Pain and discomfort

Liver cysts (40%)

10x increase in neurologic aneurisms Increased cardiovascular disease High blood pressure

Kidney stones

Declining glomerular filtration rate (GFR) à ESRD

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XOI: Aberrant Purine Metabolism & Increased Uric Acid

Oxypurinol

Platform	New	Clinical	Clinical
Technology	Oral Rx	Data	Trials

>90% Inhibition of

Xanthine Oxidase (XOI)

Pipeline-in-a- product	Proprietary and	Robust data supporting	Ready to start
patented technology	highly scalable	therapeutic potential	pivotal trial
	CMC approach	of approach	Accelerated Approval
			Confirmed by FDA

Xanthine Oxidase	7

Effect of XOI on CKD Progression

Effect of 2 years XOI treatment, 100mg daily (n=113)

Uric Acid (UA) Change (mg/dL)

The Effect of XOI on Serum Uric Acid in CKD

0.8Patients p=0.000

0.4

0

-0.4

-0.8

-1.2

-1.6

-2

Pre-XOI

24 months Post-XOI

Pre-XOIPost-XOI

eGFR Rate Change ( ml/min/1.73m2)

The Effect of XOI in CKD Estimated Glomerular

Filtration Rate (eGFR) Decline

3

2p=0.018

1

0

-1				Pre-XOI
-2				Post-XOI

-3-4

-5

Treatment Time = 24 months

Goicoechea, et al, 2010

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Product Overview & Advantages

Product Candidate Summary

• XRx-008is a novel, proprietary, well-tolerated oral formulation of oxypurinol
• Oxypurinol is a past recipient of New Drug Application (NDA) Approvable Letter, with a clinical experience in more than 750 patients

Differentiation

• XRx-008is minimally metabolized and excreted unchanged
• Few liver toxicities à better patient compliance
• Combined extracellular and intracellular action of XRx-008 is fundamental
• Potential to avoid toxicity associated with tolvaptan, the only approved drug for ADPKD
• Potential to modify underlying disease pathology supported by third-party Phase 1 and 2 clinical trials in 750+ patients with no reported serious adverse events unique to oxypurinol
• Near-termmilestones: complete GMP manufacturing of DP and API, start Phase 3 in 2024/25, pending FDA approval

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Designed to Slow the Decline in Renal Function

• XRx-008in progressive kidney disease designed to decrease uric acid & attenuate the loss of kidney filtering capacity
• A potential therapeutic option to maintain and extend kidney health can redefine the future of kidney disease treatment
• JYNARQUE® effect focuses on minimizing cyst/kidney volume.; the FDA approvable endpoint is based upon slowing the loss of kidney filtering capacity

ESRD:

• • Life-AlteringEvent
• Renal dialysis 4 hours 3x per week
• Inability to work full time
• Dependence on family
• Pain and declining health are constant burdens
• Shortened lifespan (50% of patients survive 2 years)

Cumulative Survival

100

80

60

40

20

0

Onset Delay of ESRD May Improve Quality

of Life and Longevity

Source: Curr Opin Nephrol Hypertens - 22(2): 185-192, 2013

Dialysis (n=10)

Conservative (n=15)

0

250

500

750

1000

1250

Days after eGFR fell below 15 ml/min

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