XORTX Therapeutics : Investor Presentation

Developing Orphan Drug Therapies To Slow

Progressive Kidney Disease

March, 2024

:XRTX : ANU

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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Investment Highlights

• Developing drug-basedtherapies for serious progressive kidney diseases with a high unmet medical need, including Autosomal Dominant Polycystic Kidney Disease (ADPKD), Diabetes Type-2Nephropathy (T2DN) and Acute Kidney Injury (AKI).
• Three patent families derived from our proprietary pipeline-in-a-producttechnology with broad therapeutic claims. Multiple third-party studies with well-understood Oxypurinol have characterized the mechanism of action (MoA) in over 700 patients treated, including multiple Ph2 studies. Patent protection until 2034.
• Clear focus on XRx-008program for ADPKD with a $1.0-1.8Billion USD per year revenue potential for XRTX in the US alone: strong unmet medical need; only one drug approved subject to a black box warning with market exclusivity expiring in 2025; Orphan Drug Designated granted by FDA; path to accelerated approval confirmed; potential for early revenue in 2026/7 to replace Tolvaptan by Otsuka
• Short-termcatalyst for the share price: signing of a global licensing deal in 2024/2025 may provide non- dilutive funding to support the Ph3 pivotal registration clinical trial with approximately 200 patients
• Strong cash position of $5.2 Million USD - runway for 14 months
• Senior team led by CEO Allen W. Davidoff, Ph.D was responsible for Oxypurinol development in prior ventures and knows how to build shareholder value (Cynapsus Therapeutics was acquired for $624 Million USD and Trillium Therapeutics was bought for $2.2 Billion USD by Pfizer).

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First-in-class Product Candidate Ready for Ph3 Clinical Trials of Xanthine Oxidase Inhibitor for ADPKD

Pivotal Registration Trial

- 2024 -

AKI Due to

Resp Virus

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Common ADPKD Progression & Symptoms

Pain and Discomfort

Cysts put pressure on abdomen and imping of organs

Liver cysts (40%);

10X increase in Neurologic Anurisms; Increased Cardiovascular disease

Hypertension - High Blood Pressure

Kidney Stones

Declining Renal Function "GFR" à End Stage Renal Disease (ESRD)

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XORTX Technology: Xanthine Oxidase Inhibitor for Aberrant Purine Metabolism and Increased Uric Acid

Oxypurinol

Platform	New	Clinical	Clinical
Technology	Oral Rx	Data	Trials

>90% Inhibition of

Xanthine Oxidase

Proprietary pipeline-in-a	Proprietary and	Robust data supporting	Ready to Start
product technology	highly scalable	therapeutic potential	Pivotal Registration
	CMC approach	of approach	Trial - 2024/25 -
			Accelerated Approval
			Confirmed by FDA

Xanthine Oxidase	6

Designed to Slow the Decline in Renal Function

Product Candidate Summary

• XORLOTM - Novel, proprietary, well tolerated oral formulation of Oxypurinol.
• Oxypurinol is a past recipient of New Drug Application (NDA) Approvable Letter - greater than 750 patients clinical experience.

Differentiation

• Oxypurinol is minimally metabolized and excreted unchanged. Few Liver Toxicity à better compliance on drug.
• Combined extracellular and intracellular action of XRx-008 is fundamental.
• Potential to avoid toxicity problems 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 over 750 patients with no reported serious adverse events unique to oxypurinol.
• Next Milestone complete GMP manufacturing of DP and API, start Ph3 in 2024/25 - subject to 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 loss of filtering capacity of kidneys.
• A potential therapeutic option to maintain and extend kidney health can redefine kidney disease treatment in the future.
• JYNARQUE® effect focuses on minimizing cyst/kidney volume. The FDA approvable endpoint is based upon slowing loss of filtering capacity of kidneys.

End-Stage Renal Disease (ESRD)

A Life Altering Event

• 4 hour dialysis 3 times per week
• Loss of ability to work full time
• Dependence on family
• Pain and declining health are constant burden
• Shortened survival - only 50% of patients survive two 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 below15 ml/min

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ADPKD à ESTIMATED ADDRESSABLE MARKET IN THE US

Cost of Dialysis/yr

=

~$90,000 USD

Cost of

Tolvaptan/yr

=

~$156,000 USD

US Population - ~ 338,000,000

US ADPKD Population - Diagnosed

=~160,000

• of ADPKD Population - Stage 2, 3, 4
  =~96,000

• of ADPKD with Hyperuricemia

=~36,500 (~38%)

Anticipated Pricing/ year

36,500 X $90K=

36,500 X $156K=

US ADPKD Orphan

Revenue

Range

~$3.2-5.7 B USD/ yr

Minimum Est . WW Revenue @30% penetration

~$1.0-1.8 B USD/ yr

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Competitive Landscape

DRUG	STATUS
Tolvaptan	FDA-APPROVED
(oral vasopressin V2 antagonist)	Black Box Warning - Liver toxicity
Otsuka Pharma	Extremely low usage du to tolerability
Lixivaptan	ABANDONED
(oral vasopressinV2 antagonist)
Phase III
Centessa Pharma
Bardoxolone	ABANDONED
(oral Nrf2 activator)
Phase III
Reata Pharmaceuticals Inc.
GLPG2737	ABANDONED
(a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) inhibitor)
Phase II
Galapagos
(xanthine oxidase inhibitor)	IN DEVELOPMENT
Preparing to Enter Phase III
XORTX Therapeutics Inc.

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