Slides
Page 1
Travere Therapeutics Corporate Overview November 2026
Page 2
© 2026 Travere Therapeutics, Inc. Forward-Looking Statements 2 This presentation contains forward-looking statements, including but not limited to statements about: continued progress with FILSPARI in IgAN and in the commercial launch of FILSPARI in FSGS; statements regarding our products and products in development as potential best-in-class treatments, foundational treatments and/or treatment standards; estimates regarding the size of commercial opportunities; statements regarding the advancement of our pipeline throughout the year; statements and expectations regarding our pivotal Phase 3 HARMONY Study, including expectations regarding enrollment and data, and the timing and outcome thereof; statements and expectations regarding the other clinical studies and data described herein; statements regarding additional development and regulatory milestones; statements and expectations regarding civorebrutinib and our transaction with Everest Medicines, including statements regarding the characteristics and potential efficacy, safety profile, and differentiators of civorebrutinib with respect to the diseases and conditions described herein; statements regarding potential future milestone and royalty payments; statements regarding potential changes to treatment paradigms; statements and expectations regarding the activities of our partners and collaborators; statements regarding estimates of prevalence and potential addressable market sizes; and statements regarding financial metrics and expectations related thereto. These forward-looking statements may be accompanied by such words as “anticipate,” “believe,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “project,” “schedule,” “target,” “will,” and other words and terms of similar meaning. You should not place undue reliance on these statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties related to our business and finances in general, the success of our commercial products, risks and uncertainties associated with our preclinical and clinical stage pipeline, risks and uncertainties associated with the regulatory review and approval process, risks and uncertainties associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. Specifically, we face risks associated with the commercial launch of FILSPARI in FSGS and the continued commercialization of FILSPARI in IgAN, the timing and potential outcome of our and our partners’ clinical studies, market acceptance of our commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, risks related to the challenges of manufacturing scale-up, risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI, and risks and uncertainties related to the current administration, including but not limited to risks and uncertainties related to tariffs and the funding, staffing and prioritization of resources at government agencies including the FDA. We also face the risk that we will not receive some or all of the potential future milestone and/or royalty payments described herein, the risk that our cash runway might not last as long as currently anticipated and the risk that we will be unable to raise additional funding that may be required to complete development of any or all of our product candidates, including as a result of macroeconomic conditions; risks relating to our dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of our products, and technological changes that may limit demand for our products. We also face additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations, and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the SEC. These statements are based on our current beliefs and expectations and speak only as of the date of this presentation. We do not undertake any obligation to publicly update any forward-looking statements.
Page 3
© 2025 Travere Therapeutics, Inc. rare for life. At Travere Therapeutics, we are a biopharmaceutical company that comes together every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent — that is why our global team works with the rare disease community to identify, develop, and deliver life-changing therapies. We are in 3© 2026 Travere Therapeutics, Inc.
Page 4
© 2026 Travere Therapeutics, Inc. Four Strategic Growth Pillars Support Durable Long-Term Value Creation 4 CORE GROWTH DRIVER Continued growth of FILSPARI in IgAN Established leadership with continued demand in evolving treatment landscape POTENTIAL NEXT APPROVED MEDICINE Pegtibatinase as potential first disease modifying therapy for classical HCU Topline data from pivotal Phase 3 HARMONY Study expected in 2H27 NEW GROWTH PLATFORM Civorebrutinib complements the rare kidney disease portfolio Pipeline-in-a-product potential with multi-billion- dollar global opportunity A CLEAR ROADMAP WITH MULTIPLE VALUE INFLECTION POINTS Continued portfolio expansion through external innovation and disciplined investment CORE GROWTH DRIVER Successful launch of FILSPARI in FSGS First approved medicine with rapid early adoption supported by high unmet need and prescriber familiarity
Page 5
© 2026 Travere Therapeutics, Inc. Pipeline of Potential Best-in-Class Programs Targeting Rare Kidney and Metabolic Diseases Abbreviations: IgAN: IgA nephropathy; FSGS: focal segmental glomerulosclerosis; HCU: classical homocystinuria, MCD: minimal change disease; pMN: primary membranous nephropathy. 1 In September 2024, the FDA granted full approval of FILSPARI (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk for disease progression. 2 In April 2026, the FDA granted approval of FILSPARI (sparsentan) to reduce proteinuria in adult and pediatric patients aged 8 years and older with FSGS without nephrotic syndrome. CSL Vifor has exclusive commercial rights for sparsentan in Europe, Australia, New Zealand, Bahrain, Brazil, Chile, Israel, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates. Chugai Pharmaceutical has exclusive commercial rights for sparsentan in Japan, South Korea, and Taiwan. 3 Topline efficacy data from the Phase 3 HARMONY Study is anticipated in 2H 2027. 4 Current Everest Medicines study. 5 As part of the ongoing basket study conducted by Everest Medicines. 5 PROGRAM THERAPEUTIC AREA PRECLINICAL PHASE 1 PHASE 2 PHASE 3 APPROVED COMMERCIAL FILSPARI® (sparsentan)1 FILSPARI® (sparsentan)2 Thiola EC® and Thiola® (tiopronin) IgAN FSGS Cystinuria Civorebrutinib pMN4 FSGS/MCD5 Other potential immune-mediated rare kidney diseases Pegtibatinase (TVT-058)3 HCU
Page 6
© 2026 Travere Therapeutics, Inc. FILSPARI: Clear Path to Durable Growth Through Leadership in Rare Kidney Disease 6 1 Source: Independent market research, and data on file. FILSPARI is indicated to slow kidney function decline in adults with primary IgAN who are at risk for disease progression. FILSPARI is indicated to reduce proteinuria in adult and pediatric patients aged 8 years and older with FSGS without nephrotic syndrome. Multiple growth drivers support FILSPARI expansion >$3B Estimated sales opportunity in the U.S. by 2033 Large and Expanding Market Opportunity Differentiated Clinical Profile Designed for Long-Term Use Scalable Commercial Platform The only foundational nephroprotective therapy in IgAN and FSGS that can replace the historical standard of care Once-daily oral medicine with growing long-term and real-world evidence Leveraging cross-indication synergies to target ~7,000 nephrologists >100,000 addressable patients across IgAN and FSGS in the U.S.1
Page 7
© 2026 Travere Therapeutics, Inc. FILSPARI Commercial Execution Continues with New and Repeat Prescribers Driving Demand 7 Evolving IgAN landscape expanding the market High compliance and persistence rates Increasing breadth and depth of prescriber base High unmet need, urgency to treat, and familiarity with FILSPARI driving adoption in FSGS $200M U.S. net FILSPARI sales in 3Q26 1,932 New PSFs in 3Q26 120% growth vs 3Q25 Strong demand adding to a growing base of patients with IgAN and FSGS
Page 8
© 2026 Travere Therapeutics, Inc. 146 417 430 459 511 521 505 693 703 745 731 908 993 2,012 1,932 1Q23 2Q23 3Q23 4Q23 1Q24 2Q24 3Q24 4Q24 1Q25 2Q25 3Q25 4Q25 1Q26 2Q26 3Q26 Strong Commercial Momentum Supported by Significant Long-Term Growth Drivers ~10% penetration of the combined IgAN and FSGS addressable market provides significant runway for continued growth Strong execution across IgAN and FSGS is establishing FILSPARI as the foundational nephroprotective therapy in rare kidney disease Expanding clinical evidence and evolving treatment paradigms continue to strengthen FILSPARI's long- term growth trajectory 8 1 KDIGO 2025 Clinical Practice Guideline for the Management of Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV), October 2025. * As measured by quarterly FILSPARI new patient start forms (PSFs). FILSPARI streamlined REMS requirements and updated KDIGO guidelines1 FILSPARI full approval in IgAN FILSPARI label expansion with FSGS approval
Page 9
© 2025 Travere Therapeutics, Inc. FILSPARI | IgAN + FSGS © 2026 Travere Therapeutics, Inc. 01 FILSPARI in IgAN Most commonly prescribed medicine approved for IgAN in evolving treatment landscape 02 FILSPARI in FSGS Successfully launch first approved medicine in FSGS 03 Pegtibatinase First disease-modifying therapy for classical HCU, if approved 04 Civorebrutinib Pipeline-in-a-product potential with multi-billion-dollar global opportunity © 2026 Travere Therapeutics, Inc. Caitlin, living with IgAN 9
Page 10
© 2026 Travere Therapeutics, Inc. A Substantial Opportunity to Improve the Lives of Patients Living with IgAN 10 2025 KDIGO guidelines4 to drive earlier treatment and combination therapy market >70,000 addressable patients5 peak incidence age of IgAN125-39 of transplants fail due to disease recurrence3 ~11 yrs median time to kidney failure in high-risk adult patients2 30-40% $1B+ potential for FILSPARI in IgAN6 1 Nair R & Walker PD. Kidney Int 2006; 69:1455–1458. 2 Barratt J, et al., Natural History of IgA Nephropathy: Analysis of a UK National RaDaR IgA Nephropathy Cohort, ASN 2021; Poster presentation (Abstract P01577). 3 Uffing A et al., Clin J Am Soc Nephrol. 2021 Aug;16(8):1247-1255. 4 KDIGO 2025 Clinical Practice Guideline for the Management of Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV), October 2025. 5 Independent market research and data on file. 6 Company estimates. Ashley, living with IgAN
Page 11
© 2026 Travere Therapeutics, Inc. Current KDIGO Guidelines: The IgAN Treatment Paradigm is Evolving Earlier Treatment, Lower Proteinuria Targets and Simultaneous Therapy • Kidney biopsy in all adults with proteinuria ≥0.5g/d1 Earlier diagnosis 1 At risk of progressive loss of kidney function requiring treatment • At risk if proteinuria ≥0.5g/d while on or off treatment • FILSPARI included for earlier, first- line use to optimize nephroprotection • Simultaneous treatment in most patients 2 Treatment goal • Proteinuria should be maintained at <0.5g/d, and ideally at <0.3g/d 3 Proteinuria is the only validated early biomarker to help guide clinical decision-making Source: KDIGO 2025 Clinical Practice Guideline for the Management of Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV), October 2025. 1 Or equivalent. In whom IgAN is a possible diagnosis and who do not have a contraindication for kidney biopsy. 11
Page 12
© 2026 Travere Therapeutics, Inc. The Shifting IgAN Treatment Paradigm: Two Areas to Target; Two Treatment Categories Abbreviations: RASi: renin–angiotensin system inhibitor. Source: KDIGO 2025 Clinical Practice Guideline for the Management of Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV), October 2025. * Indicated to slow kidney function decline in adults with primary IgAN who are at risk for disease progression. 12 FILSPARI is the only oral non-immunosuppressive, long-term treatment positioned to replace historical standard of care for patients with IgAN* OVERACTIVATION IN THE KIDNEY TARGET THE KIDNEY INJURY TARGET THE IMMUNE SYSTEM OVERACTIVATION OF THE IMMUNE SYSTEM Foundational Treatment Immunosuppressant Treatment RASi Steroids Budesonide Other APRIL / BAFF, B-cell therapies (in development) ± SGLT2i Complement Inhibitor ETA + Supportive Care (accelerated approval) APRIL / APRIL+BAFF (accelerated approval)
Page 13
© 2026 Travere Therapeutics, Inc. FILSPARI Positioned as a First-in-Class Foundational Treatment in IgAN with Best-in-Class Features Two-year safety data comparable to maximum-labeled dose irbesartan stronger foundation for kidney preservation One pill, once daily administration Superior two-year proteinuria reduction vs active comparator irbesartan Only therapy to date to demonstrate statistically significant slowing of kidney function decline in a Phase 3 study vs active comparator irbesartan Flexibility for combination use and newly diagnosed patients with IgAN Streamlined liver monitoring 13
Page 14
© 2026 Travere Therapeutics, Inc. License to Chugai covers Japan, South Korea, and Taiwan CMA covers all 27 member states of the European Union, plus Iceland, Liechtenstein, and Norway2 United States >70k addressable patients with IgAN1 Abbreviations: EC: European Commission; CMA: conditional marketing authorization. 1 Source: independent market research, data on file. 2 License to CSL covers Europe, Australia, New Zealand, Bahrain, Brazil, Chile, Israel, Kuwait, Oman, Qatar, Saudi Arabia and the UAE, with potential to expand. 3 Potential milestone payments include achievements for both IgAN and FSGS indications, as of the execution of the license agreements with CSL and Chugai Pharmaceutical. Through June 2026, the Company has received $63.5 million in disclosed milestone payments. * Marks indicate licensed territories. Paving a Path to Global Access for FILSPARI in IgAN with Established Commercial Partners Travere eligible to receive up to $910 million in potential milestone payments3 + tiered double-digit royalties on global net sales of FILSPARI Standard approval in Europe and the UK; FILSPARI launched in Germany, Austria, Switzerland, Luxembourg, and the UK Submitted New Drug Application for sparsentan in Japan 14
Page 15
© 2024 Travere Therapeutics, Inc. FILSPARI | IgAN + FSGS © 2026 Travere Therapeutics, Inc. 01 FILSPARI in IgAN Most commonly prescribed medicine approved for IgAN in evolving treatment landscape 02 FILSPARI in FSGS Successfully launch first approved medicine in FSGS 03 Pegtibatinase First disease-modifying therapy for classical HCU, if approved 04 Civorebrutinib Pipeline-in-a-product potential with multi-billion-dollar global opportunity 15 © 2026 Travere Therapeutics, Inc.
Page 16
© 2026 Travere Therapeutics, Inc. | FSGS Represents Significant Patient and Community Burden 7-year kidney survival rate for FSGS is the lowest among primary glomerular diseases1 >4,000 people on the kidney transplant waitlist due to FSGS in the U.S.2 ~33,000 adults and children in the U.S. are currently experiencing kidney failure due to FSGS3 Swelling (edema) in legs, feet, or eyes Weight gain from fluid Foamy urine (due to excess protein) High blood pressure Fatigue Symptoms of FSGS Side effects from medications (IST) Significant toxicity Infections Hypertension Diabetes Bone loss Mental health problems ! ! ! ! ! 5-10 years median time to kidney failure for 30-60% of patients4 | up to 55% of transplant patients experience disease recurrence5 Abbreviations: FSGS: focal segmental glomerulosclerosis. Sources: 1 Moranne O., Watier L., Rossert J., Stengel B., GN-Progress Study Group Primary glomerulonephritis: an update on renal survival and determinants of progression, Qjm. 2008;101(3):215–224. 2 Organ Procurement & Transplant Network (OPTN) data accessed December 2025 from HRSA website. 3 Bensink ME, Goldschmidt D, et al., Kidney failure attributed to focal segmental glomerulosclerosis: a USRDS retrospective cohort study of epidemiology, treatment modalities, and economic burden, Kidney Med. 2023;6(2):100760. doi: 10.1016/j.xkme.2023.100760. 4 Kiffel et al. Adv Chronic Kidney Dis. 2011;18:332-338. 5 Kienzl-Wagner et al. 2019; Trachtman et al. 2015; USRDS 2020. 16
Page 17
© 2026 Travere Therapeutics, Inc. Proteinuria is the Primary Predictor of Disease Severity and Kidney Failure in FSGS Role of Elevated and Persistent Proteinuria in FSGS KIDNEY FAILURE Podocyte Loss3,4,5 Glomerular hypertrophy hyperfiltration6,7 Glomerular sclerosis1,2 Tubulointerstitial fibrosis1,2 Podocyte injury1,2 PROTEINURIA Abbreviations: FSGS: focal segmental glomerulosclerosis. Sources: 1 De Zoysa, 2024. 2 Puelles, 2019. 3 Fogo, 1991. 4 Butt, 2020. 5 Kris, 2005. 6 Wiggins 2007. 7 Matsusaka, 2011. 17
Page 18
© 2026 Travere Therapeutics, Inc. Regardless of the Underlying Etiology, All Types of FSGS Share a Similar Glomerular Lesion Initiated by Podocyte Injury Abbreviations: Ang II: angiotensin II; ET-1: endothelin-1; FSGS: focal segmental glomerulosclerosis. 1 Kidney Disease: Improving Global Outcomes (KDIGO) Glomerular Diseases Work Group. Kidney Int. 2021;100(4S):S1-S276. 2 De Vriese AS, et al. J Am Soc Nephrol. 2018;29(3):759-774. 3 Kohan DE, et al. Clin Sci. 2024;138(11):645-662. PODOCYTE INJURY mediated by upregulation of ET-1 and Ang II3 PRIMARY FSGS lesions with diffuse foot process effacement due to putative circulating factors, possibly a cytokine originating from extrarenal sources1,2 GENETIC Identified genetic variants of podocyte and basement membrane proteins as well as high risk alleles for FSGS1,2 UNDETERMINED CAUSE FSGS lesions without diffuse foot effacement accompanied by proteinuria, in the absence of a genetic or identifiable secondary cause1 SECONDARY Secondary to other disease processes. Podocyte damage caused by a known pathophysiologic process1 18
Page 19
© 2026 Travere Therapeutics, Inc. ABSENCE OF nephrotic syndrome Nephrotic-range proteinuria >3.5 g/d BUT Serum albumin >3.0 g/dl OR Proteinuria <3.5 g/d with or without low albumin • Evaluate for underlying cause, exclude secondary forms of FSGS • Consider genetic screening (where appropriate) to exclude genetic forms • Do not start immunosuppression • Supportive therapy • Monitor proteinuria and serum albumin Worsening proteinuria and reduction in serum albumin KDIGO Guidelines Recommend FSGS Management Based on Nephrotic Syndrome Status 19 1 Source: KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Diseases | Volume 100 | Issue 4S | October 2021. FILSPARI is indicated to reduce proteinuria in adult and pediatric patients aged 8 years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome Patient with FSGS lesion on kidney biopsy1 PRESENCE OF nephrotic syndrome Proteinuria >3.5 g/d AND Serum albumin <3.0 g/dl with or without edema Especially in the presence of diffuse foot process effacement • Likely primary FSGS • Treat with immunosuppression • If no response, consider genetic testing
Page 20
© 2026 Travere Therapeutics, Inc. Population growth Earlier referral to nephrologist in light of approved treatment options Increased biopsies Lower treatment targets Expanding access Addressable FSGS Population Across Disease Subtypes is Expected to Grow Over Time 20 Source: Independent market research and data on file. Future growth drivers: >30K PATIENTS ADDRESSABLE AT LAUNCH Primary Genetic ~20K Secondary and Undetermined Cause ~10K >30,000 addressable patients with clear drivers of future growth
Page 21
© 2026 Travere Therapeutics, Inc. “In FSGS, there's no definitive therapy. We use steroids and then we move on to other things... But the effect is not great, we'd like something more definitive, more focused.”1 –Nephrologist Strong Anticipation and Clinical Familiarity with FILSPARI Expected to Drive Utilization in FSGS © 2026 Travere Therapeutics, Inc. 1 Source: Spherix FSGS Market Dynamix Report, October 2025. 2 Source:Spherix 2025 Patient Chart Dynamix. >80% of nephrologists indicate novel non-immunosuppressives are highly desirable in FSGS1 ~78% of nephrologists report being “highly familiar” with FILSPARI for FSGS1 ~90% of patients are already on ACE/ARBs, positioning FILSPARI as a clear replacement option from supportive care to kidney-targeted therapy2 21
Page 22
© 2026 Travere Therapeutics, Inc. Leveraging FILSPARI's Established Commercial Platform to Accelerate FSGS Adoption Strong commercial foundation that supports uptake across both indications Patient & Physician Focus Continuous patient and physician education on approved indication Established Commercial Infrastructure Experienced field team leveraging high prescriber overlap to accelerate uptake across both indications Access & Reimbursement Established payer access in IgAN positions FILSPARI for supportive access environment in FSGS Focus on expanding policies to include FSGS Comprehensive Support Program Travere TotalCare infrastructure applied and customized to patients with FSGS Product Available Day 1 Streamlined logistics with product already in channel 22
Page 23
© 2026 Travere Therapeutics, Inc. Potential ImpactData GenerationEvidence Generation Strategic Evidence Generation Expected to Further Support FILSPARI's Long-Term Growth Opportunity 23 Supports broader adoption across the full spectrum of patients with FSGS, including patients at high risk of progression Abbreviations: eGFR: estimated glomerular filtration rate. 1 Source: Cheung CK, et al. presented at ASN 2026; October 21-25, 2026; Denver, CO. TH-PO0453. FSGS Generate evidence in patients of African ancestry with FSGS (SPARLIGHT Study) Study initiation in 2H 2026 To support broader FSGS adoption, including high-risk patients IgAN FILSPARI as first-line therapy in newly diagnosed patients (SPARTAN Study) ~77% proteinuria reduction & eGFR stabilization (week 110)¹ ~75% complete remission¹ Supports foundational therapy utilization in treatment-naïve patients IgAN & FSGS Evaluate FILSPARI in post-transplant recurrent IgAN & FSGS (SPARX Study) Data presentations expected in 2027 To support use in ~7,000 addressable post-transplant patients
Page 24
© 2024 Travere Therapeutics, Inc. FILSPARI | IgAN + FSGS © 2026 Travere Therapeutics, Inc. 01 FILSPARI in IgAN Most commonly prescribed medicine approved for IgAN in evolving treatment landscape 02 FILSPARI in FSGS Successfully launch first approved medicine in FSGS 03 Pegtibatinase First disease-modifying therapy for classical HCU, if approved 04 Civorebrutinib Pipeline-in-a-product potential with multi-billion-dollar global opportunity © 2026 Travere Therapeutics, Inc. Caitlin, living with IgAN 24 © 2026 Travere Therapeutics, Inc. Jamela, living with HCU
Page 25
© 2026 Travere Therapeutics, Inc. HCU Market Represents Significant Unmet Need with Potential for Growth with Better Diagnostics, Awareness and Effective Treatment Options Expected growth in addressable patients with HCU in U.S. 25% of HCU patients by age 16 and 50% by age 29 develop life- threatening thrombotic events, including heart attack and stroke1,2 7,000 to 10,000 patients living with HCU in U.S.; similar number in Europe3 No approved treatments to address the underlying genetic cause of HCU Pegtibatinase has the potential to become the only disease-modifying therapy in a market with significant growth expected Disease education/awareness, enhanced diagnostics and better treatment options are expected to lead to increased patient identification, earlier diagnosis, and better outcomes – driving growth in addressable market Despite newborn screening for HCU in the U.S., fewer than 50% of people with HCU are diagnosed at birth4 ~80% of patients with HCU are partially or non-responsive to B6 therapy (current standard of care)5 At-launch Future ~3k+ ~5k+ 25 Sources: 1 Mudd et al., Am J Hum Genet (1985), 2 Yap et al., J Am Heart Assoc. (2001), 3 Data on file. 4 Levy H, et al., Clin Chem. 2023;69(5):433-434, 5 Kozich V, Sokolova J, Morris AAM, et al., J Inherit Metab Dis. 2021;44(3):677-692.
Page 26
© 2026 Travere Therapeutics, Inc. Pegtibatinase is an Investigational, Modified, Recombinant CBS Human Enzyme Therapy Pegtibatinase is designed to address the underlying genetic cause of HCU Mechanism of action is designed to have broad effect across HCU population Administered subcutaneously and designed to be active and stable in plasma, unlike native CBS Designed to introduce the CBS enzyme into circulation and reduce intracellular and plasma Hcy levels PegtibatinaseVitamin B6 CBS Betaine Cystathionine Cysteine Homocysteine Methionine Pegtibatinase has been granted multiple regulatory designations for the treatment of classical HCU FDA Breakthrough Therapy designation FDA Rare Pediatric Disease designation FDA Fast Track designation Orphan Drug designation in the U.S. and Europe 26
Page 27
© 2026 Travere Therapeutics, Inc. Treatment with Pegtibatinase in the Phase 1/2 COMPOSE Study Showed Rapid and Sustained tHcy Reduction Through 12 Weeks of Treatment 27 Placebo Pegtibatinase Cohort 1 0.33 mg/kg QW Cohort 2 0.66 mg/kg QW Cohort 3 1.0 mg/kg QW Cohort 4 1.0 mg/kg BIW Cohort 5 1.5 mg/kg BIW Cohort 6 2.5 mg/kg BIW Sample Size (n) 6 3 3 3 2 3 4 Summary of relative reduction in geometric mean of total homocysteine from baseline from cohorts 1-6 in the Phase 1/2 COMPOSE Study67.1% mean relative reduction in total homocysteine from baseline All patients in highest dose cohort achieved a clinically meaningful threshold in mean tHcy over weeks 6 to 12 of treatment Methionine and cystathionine biomarkers suggest that pegtibatinase acts similar to the native CBS enzyme and can restore the metabolic dysregulation in patients with HCU Pegtibatinase was generally well-tolerated at all doses tested -70 -60 -50 -40 -30 -20 -10 0 10 Relative Change (%)* 0.6 -4.0 4.2 1.4 -25.2 -57.1 -67.1 Abbreviations: BIW: twice weekly; QW: once weekly. * The data referenced in the table above and the analysis conducted in cohort 6 assess the relative reduction in tHcy from baseline in the geometric mean by averaging tHcy over weeks 6, 8, 10, and 12. This measure improves the precision and reliability of assessment of the treatment effect and takes into account that there is some variability in tHcy depending on food intake and diurnal variation. The Company intends to use this measure moving forward.
Page 28
© 2026 Travere Therapeutics, Inc. Innovative Pegtibatinase Phase 3 Program Designed to Enable a BLA Submission Abbreviations: BIW: twice weekly, DSP: diet standardization period, LTE: long-term (open-label) extension, SC: subcutaneous, tHcy: total homocysteine, FU: follow up. * Protocol allows for ~25% of patients with tHcy ≥50 to <80μM. ** ClinicalTrials.gov ID: NCT06247085. *** In April 2026, the Company dosed the first new patient following the Phase 3 HARMONY Study restart. Topline efficacy data anticipated in 2H 2027. Phase 3 HARMONY Study Double-blind treatment 24 weeks Screening ≤4 weeks ≤6 weeks 2-week dose titration Safety FU period 4 weeks Pegtibatinase 2.5mg/kg SC BIW Roll into ENSEMBLE LTE Study or safety follow upPlacebo SC BIW (N≈70) • ≥12 to ≤65 year of age • Confirmed HCU diagnosis • Plasma tHcy ≥80 μM* Pre-treatment DSP Randomization (1:1) Primary endpoint • Change from baseline in plasma tHcy levels (averaged over weeks 6 through 12) Key secondary endpoint • The relative change from baseline in plasma tHcy levels averaged post-week 12 (weeks 16, 20, 24) Week 52 Key endpoints Open-label treatment period 52 weeks Day 1 Week 5 Week 10 Week 56 Pegtibatinase 2.5mg/kg SC BIW Safety follow-up period End of Study- HARMONY/ Day 1 of ENSEMBLE Protein Tolerance Modification Sub-study Safety FU period 4 weeks 2-week dose titration Self-admin training Phase 3b ENSEMBLE Study Concurrent Phase 3b Study to evaluate if eligible patients can increase their natural dietary protein intake while maintaining an acceptable level of metabolic control while receiving pegtibatinase Week 12 Primary endpoint Week 24 Secondary endpoints Day 1 28
Page 29
© 2026 Travere Therapeutics, Inc. On Track to Position Pegtibatinase as the First Potential Disease-Modifying Therapy in HCU 29 Phase 3 HARMONY Study enrollment continues Completed site activation Leveraging patient identification efforts to build enrollment momentum Topline data on track for 2H 2027 Patients continue to be followed in ENSEMBLE open label extension study
Page 30
© 2024 Travere Therapeutics, Inc. FILSPARI | IgAN + FSGS 01 FILSPARI in IgAN Most commonly prescribed medicine approved for IgAN in evolving treatment landscape 02 FILSPARI in FSGS Successfully launch first approved medicine in FSGS 03 Pegtibatinase First disease-modifying therapy for classical HCU, if approved 04 Civorebrutinib Pipeline-in-a-product potential with multi-billion- dollar global opportunity 30 © 2026 Travere Therapeutics, Inc.
Page 31
© 2026 Travere Therapeutics, Inc. Civorebrutinib is a potential best-in-class BTK inhibitor differentiated by oral administration, with rapid onset of action, high selectivity, strong potency, and a potentially favorable safety profile Civorebrutinib is a Potent Covalent Reversible BTK Inhibitor with Potential Across Several Immune-Mediated Rare Kidney Indications 31 1 Covalent reversible Designed for strong yet reversible BTK binding, potentially enabling more controlled immune modulation vs other approaches 2 High selectivity 3 Potent target binding 4 Potential best-in-class safety profile Highly selective against other kinases to reduce off-target toxicity Single digit nM potency and high target occupancy at clinical doses leads to sustained target inhibition Potentially favorable safety profile based on clinical and non-clinical studies to date Abbreviations: BTK: Bruton tyrosine kinase. 31
Page 32
© 2026 Travere Therapeutics, Inc. Civorebrutinib Offers Higher Selectivity and Potency Compared to Covalent Irreversible BTK inhibitors 32 Compound IC50 nM Kinase IC50/BTK IC50 (fold) BTK LCK SRC LYN EGFR ITK TEC JAK3 Civorebrutinib 1.8 3668x 4853x 1125x 714x 5178x 6x >10000x Ibrutinib 1.5 4x 17x 17x 4x 3x 5x 21x BTKi Selectivity (IC50 fold) EGFR ITK TEC Civorebrutinib 714x 5178x 6.6x Ibrutinib 4x 3x 5x Zanubrutinib 8.7x 187x 6.7x PRN1008 400x 338x 0.6x Acalabrutinib >200x >200x 18x Civorebrutinib demonstrates potent and selective inhibition of BTK without affecting EGFR, ITK, or TEC family kinases—targets associated with potential adverse effects 1 Reversible covalency 2 Excellent selectivity 3 Potent target binding 4 Potential best-in-class safety profile Source: Everest Medicines 2025 Interim Results Presentation, 8/29/2025.
Page 33
© 2026 Travere Therapeutics, Inc. Significant Patient Population Across Potential Rare Kidney Disease Opportunities 33 Estimated Prevalence Across Immune-Mediated Rare Kidney Diseases Mechanistic rationale supports further evaluation in immune-mediated FSGS and MCD >130k diagnosed patients across multiple indications pMN1 >90k Immune- mediated FSGS2 >20k MCD3 >20k Additional Indication potential © 2026 Travere Therapeutics, Inc. Abbreviations: FSGS: focal segmental glomerulosclerosis; MCD: minimal change disease; pMN: primary membranous nephropathy. 1 Source: Swaminathan S., et al. CJASN, 2006. 2 Source: Bensink M., et al. Kidney Medicine. Kidney Failure Attributed to Focal Segmental Glomerulosclerosis: A USRDS Retrospective Cohort Study of Epidemiology, Treatment Modalities, and Economic Burden, 2023, and Munis M., et al. CJASN. Incidence and Proportion of Primary Focal Segmental Glomerulosclerosis (FSGS) among a Racially and Ethnically Diverse Adult Patient Population between 2010 and 2021, 2014. 3 Source: Vivarelli M., et al. CJASN. Minimal Change Disease, 2016, and Waldman M., et al. CJASN. Adult minimal-change disease: clinical characteristics, treatment, and outcomes, 2007.
Page 34
© 2026 Travere Therapeutics, Inc. Primary Membranous Nephropathy Remains a High Unmet Need with Significant Patient Burden 34 1 Symptoms include high proteinuria (common range 3.5 g – 20 g/24h), decrease in serum albumin levels, severe swelling, microscopic hematuria. 2 Source: Couser W., Primary Membranous Nephropathy, CJASN, 2017. 3 Source: Hladunewich M., et al. The Natural History of the Non-Nephrotic Membranous Nephropathy Patient, CJASN, 2009. 4 Source: Ronco P., et al. Membranous nephropathy, Nature Reviews Disease Primers, 2021. 5 Source: National Kidney Foundation “Voice of the Patient Report”, Externally Led Patient-Focused Drug Development Meeting on Membranous Nephropathy. Public Meeting: August 27, 2021. Report Date: May 5, 2023. >70% of patients present with nephrotic syndrome1,2 ~60% of patients presenting with sub nephrotic- range proteinuria will progress to full nephrotic syndrome within 1-2 years from presentation3 40-50% of patients with persistent nephrotic syndrome will progress to kidney failure over a period of 10 years4 >70% of patients with pMN report moderate or significant impacts on their daily life5 0 approved treatments High blood pressure “Brain Fog” Symptoms of pMN Ophthalmic problems Anxiety and/or depression Swelling (edema) Muscle and joint aches and pains Skin problems Fatigue Gastrointestinal problems Recurrent infections High blood sugar/diabetes ! ! !
Page 35
© 2026 Travere Therapeutics, Inc. BTK Inhibitors Target Multiple Key Nodes in the Pathogenesis of Primary Membranous Nephropathy 35 BTK inhibitors suppress B-cell signaling, reducing B-cell activation and proliferation and limiting differentiation into autoantibody-producing plasma cells BTK inhibitors provide functional B-cell modulation without broad b-cell or immunoglobulin depletion Oral BTK inhibitors may allow for more rapid recovery of B-cell function following treatment discontinuation, if needed Civorebrutinib (BTKi) Abbreviations: anti-PLA2R: anti-phospholipase A2 receptor; anti-THSD7A: thrombospondin type-1 domain-containing protein 7A; BAFF: b-cell activating factor; BCMA: b-cell maturation antigen; BlyS: b-Lymphocyte stimulator; BTKi: Bruton tyrosine kinase inhibitor. 1 Image adopted from Ruggenenti P., et al. Nature Reviews Nephrology. Treatment of membranous nephropathy: time for a paradigm shift, 2017. 2 Image adopted from Hendriks R. New BTK inhibitor holds promise, Nat Chem Biol 7, 4–5 , 2011. Prevents differentiation into antibody-secreting cells Intracellular site of action2 Civorebrutinib 1
Page 36
© 2026 Travere Therapeutics, Inc. Phase 1b/2a Study of Civorebrutinib in Patients with pMN with Positive Anti-PLA2R Autoantibody 36 Data from at least 8 pts dosed for 10 days is required to start Cohort 2 Low Dose: civorebrutinib (EVER001) 100mg QD×4W→ 100mg BID×32W(N=13) Follow-up 36 weeks treatment 16 weeks Extended Follow-up Period Up to Week 104 High Dose: civorebrutinib (EVER001) 200mg BID (N=18) 36 weeks treatment Follow-up 16 weeks Extended Follow-up Period Up to Week 104 Part A Part B Part A Part B • Safety and tolerability • Percentage change from baseline of 24h proteinuria, anti-PLA2R autoantibody level, UPCR and eGFR • Complete or partial remission of 24h proteinuria • Remission of anti-PLA2R autoantibody Key Inclusion and Exclusion Criteria • Biopsy proven primary membranous nephropathy • Anti-PLA2R autoantibody > 20 RU/mL • Nephrotic range proteinuria >3.5 g/24h • eGFR ≥ 45 mL/min/1.73m2 • Serum albumin level ≥ 25g/L and IgG ≥ 3g/L • No use of rituximab, cyclophosphamide and CNIs in 2 years, 6 months and 3 months before study treatment, respectively • Exclude patients with positive TB test or positive HBsAg Primary endpoint Secondary endpoints Abbreviations: anti-PLA2R: anti-phospholipase A2 receptor; CNI: calcineurin inhibitor; eGFR: estimated glomerular filtration rate; HBsAg: hepatitis B surface antigen; TB: tuberculosis; UPCR: urine protein to creatinine ratio.
Page 37
© 2026 Travere Therapeutics, Inc. -62.2% -95.5% -88.3% -87.3% -95.5% -93.1% -120% -100% -80% -60% -40% -20% 0% 0 4 8 12 16 24 28 36 44 52 Geometric LS Mean of Percent Change from Baseline, % Week Cohort 1 Cohort 2 Phase 1/2 Proof of Concept: Civorebrutinib Demonstrated Rapid and Sustained Reductions in Key Disease Biomarkers 37 Abbreviations: anti-PLA2R: anti-phospholipase A2 receptor. Note: data cutoff date: September 5, 2025. Presented at 63rd ERA Congress in Glasgow, UK, on June 3-6, 2026. Cohort 1 (n) 13 13 13 11 13 11 11 11 11 Cohort 2 (n) 17 17 17 17 16 16 16 Part A Follow-up Period Percent Change in Anti-PLA2R from Baseline (FAS) ~90% reduction in anti-PLA2R by week 12 in a higher-dose cohort ~90% reduction in anti-PLA2R by week 12 in a higher-dose cohort
Page 38
© 2026 Travere Therapeutics, Inc. -56.8% -76.6% -78.9%-68.0% -80.0% -82.6% -100% -80% -60% -40% -20% 0% 20% 40% 0 4 8 12 16 20 24 28 36 44 52 Geometric LS Mean of Percent Change from Baseline, % Week Cohort 1 Cohort 2 Phase 1/2 Proof of Concept: Civorebrutinib Demonstrated Rapid and Sustained Reductions in Key Disease Biomarkers 38 Note: data cutoff date: September 5, 2025. Presented at 63 rd ERA Congress in Glasgow, UK, on June 3-6, 2026. ~80% reduction in proteinuria in both cohorts by week 36 sustained after treatment discontinuation Cohort 1 (n) 13 13 13 13 12 12 11 11 11 11 Cohort 2 (n) 17 17 17 17 17 16 16 16 16 16 Percent Change in 24h Proteinuria from Baseline (FAS) ~80% reduction in proteinuria in both cohorts by week 36 sustained after treatment discontinuation Part A Follow-up Period
Page 39
© 2026 Travere Therapeutics, Inc. Favorable Safety and Tolerability Profile Category of AEs, n (%) Cohort 1 (N=13) 100mg QD→ 100mg BID Cohort 2 (N=18) 200mg BID Total (N=31) Treatment emergent AE (TEAE) 12 (92.3) 15 (83.3) 27 (87.1) Serious TEAE 3 (23.1) 3 (16.7) 6 (19.4) ≥CTCAE Grade 3 TEAE 3 (23.1) 3 (16.7) 6 (19.4) Treatment-related TEAE 10 (76.9) 9 (50.0) 19 (61.3) Treatment-related serious TEAE 2 (15.4) 2 (11.1) 4 (12.9) ≥CTCAE Grade 3 treatment-related TEAE 2 (15.4) 2 (11.1) 4 (12.9) TEAE leading to discontinuation 0 2 (11.1) 2 (6.5) TEAE leading to death 0 0 0 Abbreviations: AE: adverse event; CTCAE: common terminology criteria for adverse events; TEAE: treatment-emergent adverse event; URTI: upper respiratory tract infection. Note: TEAEs is defined as AEs occurred or worsened in severity or relationship to study treatment from first dose of study treatment throughout the treatment period and including the follow-up period. The most common treatment related TEAEs (>= 2 subjects ) in all patients were anemia (12.9%), B-lymphocyte count decreased (9.7%), dizziness (9.7%), URTI (6.5%), hyperkalemia (6.5%), palpitations (6.5%), nausea (6.5%), and vomiting (6.5%). 39 No clinically meaningful AEs typically associated with BTK inhibitors, such as neutropenia, hemorrhage, cardiac arrythmia
Page 40
© 2026 Travere Therapeutics, Inc. Civorebrutinib With the Potential for Rapid, Controllable Immune Modulation, Civorebrutinib is a Viable Candidate to Address Key Gaps in pMN Treatment 40 Patients at high risk of progression, relapse, or inadequate response to current options need therapies with faster onset, favorable tolerability, and flexible immune modulation, representing an important opportunity for further evaluation of civorebrutinib Rapid and sustained reductions in key disease biomarkers seen in Phase 1/2 study (anti-PLA2R and proteinuria) Oral administration Convenient patient-friendly option Safety & reversibility Potential to avoid side effects traditionally associated with immunosuppression Potential to address unmet need in patients who relapse or remain inadequately controlled on current standard of care Abbreviations: anti-PLA2R: anti-phospholipase A2 receptor; pMN: primary membranous nephropathy.
Page 41
© 2026 Travere Therapeutics, Inc. BTK Inhibitor Mechanism Has Promising Potential in Several Rare Kidney Diseases, including Immune-Mediated FSGS and MCD BTK plays a functional role in immune cell types involved in FSGS pathology BTKi's have a potential role in FSGS with immune cell involvement Emerging data suggests BTK is expressed in monocytes and macrophages in kidney biopsies of FSGS patients BTK biology supports further evaluation of civorebrutinib in selected immune-mediated rare kidney diseases, including FSGS and MCD Abbreviations: BTKi: Bruton tyrosine kinase inhibitor; FSGS: focal segmental glomerulosclerosis; MCD: minimal change disease. 41 BTKi’s target overactivation in the immune system, which is distinct and complementary to mechanisms that are nephroprotective for a heterogeneous disease like FSGS
Page 42
© 2026 Travere Therapeutics, Inc. 1 Diluted share count calculation includes all outstanding equity awards but excludes convertible notes. 2 Cash, cash equivalents and marketable securities as of September 30, 2026. 42 A Strong Financial Foundation to Deliver New Treatment Standards in Rare Disease ~$220M in total U.S. net product sales in 3Q26 Represents ~95% growth year-over-year ~$472M in cash and cash equivalents2 as of September 30, 2026 ~95M basic shares outstanding as of September 30, 2026 Diluted ~108mm1 ~$95M in convertible notes due March 2029 $525M due May 2032
Page 43
Data Appendix
Page 44
© 2026 Travere Therapeutics, Inc. Treatment with FILSPARI May Potentially Delay Dialysis or Transplant Abbreviations: eGFR: estimated glomerular filtration rate; ESKD: end-stage kidney disease. 1 FILSPARI Prescribing Information. 2 Data on file. 3 United States Renal Data System. 2023 USRDS Annual Data Report: Epidemiology of kidney disease in the United States. NIH, NIDDK, Bethesda, MD, 2023. Based on extrapolation of eGFR slope data from PROTECT, FILSPARI may potentially delay dialysis or transplant by 4.5 years when compared to maximum-labeled dose irbesartan1-3 FILSPARI Irbesartan Years eGFR (mL/min/1.73 m2) 0 201 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 0 60 10 20 30 40 50 +4.5 years vs irbesartan ESKD3 (eGFR <15 mL/min/1.73 m2) FILSPARI 15.7 years Irbesartan 11.2 years Potential long-term impact of preserved eGFR slope1,2 44
Page 45
© 2026 Travere Therapeutics, Inc. Further Evidence Generation to Drive Broad Uptake: Supporting First-Line FILSPARI Use in Newly Diagnosed Patients Abbreviations: eGFR: estimated glomerular filtration rate; UPCR: urine protein-to-creatine ratio. Source: Cheung CK, et al. presented at ASN 2024; October 23-27, 2024; San Diego, CA. FR-OR63. 1 KDIGO 2025 Clinical Practice Guideline for the Management of Immunoglobulin A Nephropathy (IgAN) and Immunoglobulin A Vasculitis (IgAV), October 2025. 2 Heerspink HJL, et al. Lancet. 2023;401(10388):1584-1594. 3 Rovin BH, et al. Lancet. 2023;402(10417):2077-2090. SPARTAN Study: Preliminary clinical findings at 24-weeks in treatment-naïve patients on FILSPARI FILSPARI led to rapid and sustained reductions in proteinuria (~70% from baseline) and stabilization of eGFR at week 24 Within 24 weeks of starting FILSPARI, ~60% of patients achieved complete remission of proteinuria, a treatment goal recommended in the 2025 KDIGO guidelines1 FILSPARI was generally well tolerated over 24 weeks of treatment, with no evidence of fluid retention. Safety results were consistent with the Phase 3 PROTECT Study2,3 −100 −80 −60 −40 −20 0 0 2 4 6 8 10 12 14 16 18 20 22 24 Change in UPCR from baseline, geometric mean (SE), % n=12n=11 Week n=11 n=11 30 40 50 60 70 80 90 100 -2 0 2 4 6 8 10 12 14 16 18 20 22 24 eGFR, mean (SD), mL/min/1.73 m2 Week n=12 n=12 n=12n=11 n=11 n=11 45
Page 46
© 2026 Travere Therapeutics, Inc. SPARTAN Study: Urinary Biomarker Analysis Suggests Disease-Modifying Effects of FILSPARI in IgAN 46 −100 −80 −60 −40 −20 0 0 12 24 Change in urinary biomarkers, % (SEM) BAFF sC5b9 −68% −75% Abbreviations: α2M: alpha-2-macroglobulin; BAFF: B-cell activating factor; CH13L1: chitinase-3-like protein 1; CXCL10: C-X-C motif chemokine ligand 10; CXCL16: C-X-C motif chemokine ligand 16; GDF15: growth/differentiation factor 15; IL6: interleukin 6; MCP-1: monocyte chemoattractant protein-1; sC5b9: soluble C5b9; sCD163: soluble CD163. Source: Cheung, et al. presented at the National International Podocyte Conference & ISGD Meeting; June 10-13, 2025; Hamburg, Germany. Poster FR-11. 1 One patient discontinued after week 6 and has been excluded from all urinary biomarker analysis (n=11). 2 α2M, clusterin and plasminogen analysis was performed only at baseline and week 12. Treatment with FILSPARI resulted in rapid and sustained reductions in urinary biomarkers of inflammation and fibrosis that reveal anti-inflammatory and anti-fibrotic effects of FILSPARI1 Change in urinary biomarkers from baseline to week 24 Inflammatory and profibrotic α2M2 –83% CHI3L1 –52% clusterin2 –47% GDF15 –42% plasminogen2 –85% sCD163 –50% Chemokine and cytokine CXCL10 –28% CXCL16 –22% IL6 –23% MCP-1 –16%
Page 47
© 2026 Travere Therapeutics, Inc. SPARTACUS Study: FILSPARI Added to SGLT2i Resulted in Further Proteinuria Reduction and Was Generally Well Tolerated Source: Ayoub I., et al. presented at ERA 2025, June 4-7, 2025; Vienna, Austria. Abstract: No. 1916. * Reported in the same patient. †The incident of acute kidney injury was mild, deemed unrelated to SPAR or SGLT2i treatment, and was resolved after interruption of SPAR and SGLT2i. ‡Abnormal liver function test results met the following criteria: (1) new elevation in ALT or AST >3 × ULN with or without elevation of total serum bilirubin >2 × ULN and (2) 2-fold increase in ALT or AST above the baseline value in patients who had elevated values prior to taking study medication. § One patient each discontinued SPAR treatment due to a TEAE of vertigo, hypotension, peripheral edema, and Henoch-Schönlein purpura. TEAEs Patients (N=48) Any TEAE, n (%) 30 (63) SPAR related 10 (21) SGLT2i related 2 (4) Any TEAEs in >2 patients, n (%) Hypotension 7 (15) Headache 4 (8) Edema 4 (8) Peripheral edema 4 (8) Upper respiratory tract infection 4 (8) Dizziness 6 (6) Any severe TEAE, n (%) 2 (4) Peripheral edema 1 (2) Gout 1 (2) Any serious AE, n (%) 4 (8) Acute kidney injury*† 1 (2) Cerebrovascular accident 1 (2) Chemical burn 1 (2) Deep vein thrombosis 1 (2) Osteoarthritis* 1 (2) Any abnormal liver function test results >3×ULN, n (%)‡ 0 (0) Any TEAE leading to SPAR discontinuation, n (%) 4§ (8) Transitioning patients from RASi to FILSPARI resulted in a mean reduction in UACR of ~56% at 24 weeks BL 4 12 24 −70 −60 −50 −40 −30 −20 −10 0 10 Geometric least-squares mean change from baseline in UACR (95% CI), % Week n=45 n=42n=48 47 n=39 Abbreviations: BL: baseline; RASi: renin-angiotensin system inhibitor; SGLT2i: sodium-glucose cotransporter-2 inhibitor; SPAR: sparsentan; UACR: urine albumin-to-creatinine ratio; TEAE: treatment-emergent adverse event; AE: adverse event; ULN: upper limit of normal.
Page 48
© 2026 Travere Therapeutics, Inc. Evaluate the efficacy and safety of sparsentan vs. the active control irbesartan in patients with focal segmental glomerulosclerosis (FSGS) • Phase 3, double-blind, active-controlled global trial in patients with biopsy-proven FSGS or genetic FSGS, N=371 patients (ages 8 to 75 years)* • The only head-to-head Phase 3 study of its kind in FSGS • Surrogate efficacy endpoint: (36-week interim analysis) = proportion of patients achieving FPRE at week 36 (UPCR ≤ 1.5 g/g and ≥ 40% reduction from baseline) • Primary endpoint: eGFR total slope: From day 1 to week 108 of treatment (U.S. primary), eGFR chronic slope: From week 6 to week 108 of treatment (EU primary) Objective Trial Design The DUPLEX Study of Sparsentan is the Largest Active- Controlled Interventional Phase 3 Trial in FSGS to Date Resume SOC, including ACEi/ARBs Double-blind treatment 108 weeks, randomized 1:1 4 weeks post-cessation of randomized treatment No ACEi/ARBs Screening • Ages 8-75 years • FSGS (excluding secondary causes) • UPCR ≥1.5 g/g • eGFR ≥30 mL/min/1.73 m2 Week 112 Last double-blind assessment Week 36 Prespecified interim analysis Week 108 End of double-blind treatment BaselineDiscontinue ACEs/ARBs Irbesartan 150 mg / day → 300 mg / day at week 2 Sparsentan 400 mg / day → 800 mg / day at week 2 Washout period 2 weeks Abbreviations: ACEi: angiotensin converting enzyme inhibitors; ARBs: angiotensin receptor blockers; eGFR: estimated glomerular filtration rate; FPRE: FSGS partial remission endpoint; g/g: grams per gram; SOC: standard of care; UPCR: urine protein to creatinine ratio. * ClinicalTrials.gov ID: NCT03493685. 48
Page 49
© 2026 Travere Therapeutics, Inc. In DUPLEX, Treatment with FILSPARI Demonstrated Even Greater Reduction in Proteinuria in FSGS without Nephrotic Syndrome 49 DUPLEX Population without Nephrotic Syndrome* % reduction in UPCR from baseline by visit Overall DUPLEX Population % reduction in UPCR from baseline by visit -46% -30% -60% -50% -40% -30% -20% -10% 0% 0 6 12 24 36 48 60 72 84 96 108 LS Mean Change from Baseline UPCR, % Week FILSPARI (N=184) Irbesartan (N=187) -48% -27% -60% -50% -40% -30% -20% -10% 0% 0 6 12 24 36 48 60 72 84 96 108 LS Mean Change from Baseline UPCR, % Week FILSPARI (N=129) Irbesartan (N=125) FILSPARI vs Irbesartan1 : 22% p-value: 0.0299 FILSPARI vs Irbesartan1 : 29% p-value: 0.0075 Abbreviations: CI: confidence interval; LS: least squares; UPCR: urine protein to creatinine ratio. 1 95% CI. All p-values are nominal. * In the DUPLEX Study, nephrotic syndrome was defined as (a) documentation of nephrotic syndrome in the medical history, or (b) the concurrent presence of proteinuria >3.5 g/24 hours (adults) or UPCR >2.0 g/g (pediatric patients <18 years of age), serum albumin <3.0 g/dL, and edema at baseline. Patients without nephrotic syndrome did not meet both criteria (a) and (b).
Page 50
© 2026 Travere Therapeutics, Inc. 69.0 31.0 18.5 50.8 14.4 7.5 0 10 20 30 40 50 60 70 80 < 1.5 g/g < 0.5 g/g < 0.3 g/g Proportion of Patients Achieving UPCR Below Threshold, % FILSPARI (N=184) Irbesartan (N=187) RR (95% CI): 2.15 (1.44-3.20) In DUPLEX, FILSPARI Demonstrated Even Greater Relative Proteinuria Reduction Across Thresholds in FSGS without Nephrotic Syndrome Abbreviations: CI: confidence interval, RR: relative risk, UPCR: urine protein to creatinine ratio. 1 At any time during the double-blind period. All threshold analyses have nominal p-value <0.05. 2 Source: Rheault MN, et al., Sparsentan versus Irbesartan in Focal Segmental Glomerulosclerosis, The New England Journal of Medicine and Supplement, 2023. * In the DUPLEX Study, nephrotic syndrome was defined as (a) documentation of nephrotic syndrome in the medical history, or (b) the concurrent presence of proteinuria >3.5 g/24 hours (adults) or UPCR >2.0 g/g (pediatric patients <18 years of age), serum albumin <3.0 g/dL, and edema at baseline. Patients without nephrotic syndrome did not meet both criteria (a) and (b). 50 79.1 35.7 20.2 50.4 12.8 6.4 0 10 20 30 40 50 60 70 80 < 1.5 g/g < 0.5 g/g < 0.3 g/g Proportion of Patients Achieving UPCR Below Threshold, % FILSPARI (N=129) Irbesartan (N=125) DUPLEX Population without Nephrotic Syndrome* Patients achieving UPCR thresholds1, % Overall DUPLEX Population Patients achieving UPCR thresholds1,2, % RR (95% CI): 3.12 (1.46-6.66) RR (95% CI): 2.47 (1.37-4.45) RR (95% CI): 2.15 (1.44-3.20) RR (95% CI): 2.66 (1.61-4.40) RR (95% CI): 1.36 (1.16-1.59) >3x greater complete remission rates RR (95% CI): 1.50 (1.25-1.80) >2x greater complete remission rates
Page 51
© 2026 Travere Therapeutics, Inc. In DUPLEX, FILSPARI Demonstrated a Clinically Meaningful Effect on eGFR in FSGS without Nephrotic Syndrome FILSPARI (N=129)1 Irbesartan (N=125)1 Difference p-value5 Adjusted Mean Change in eGFR (mL/min/1.73 m2) at Week 108 from baseline2 -11.3 -12.4 1.1 NS Total eGFR slope (mL/min/1.73 m² per year)3 -4.1 -6.2 2.1 0.031 Chronic eGFR slope (mL/min/1.73 m² per year)4 -3.7 -6.2 2.5 0.015 FILSPARI (N=184)1 Irbesartan (N=187)1 Difference p-value5 Adjusted Mean Change in eGFR (mL/min/1.73 m2) at Week 108 from baseline2 -14.3 -13.3 -1.0 NS Total eGFR slope (mL/min/1.73 m² per year)3 -5.4 -5.7 0.3 NS Chronic eGFR slope (mL/min/1.73 m² per year)4 -4.8 -5.7 0.9 NS Abbreviations: CI: confidence interval; eGFR: estimated glomerular filtration rate; LS: least squares; NS: not significant; UPCR: urine protein to creatinine ratio. . 1 On treatment. 2 Adjusted mean change from baseline in eGFR is obtained from the mixed model repeated measures analysis. The comparison between FILSPARI and irbesartan was based on the difference in adjusted mean change in eGFR at Week 108 with baseline on the absolute scale. 3 LS mean of annualized slope from Day 1 to Week 108. 4 LS mean annualized slope from Week 6 to Week 108. 5 Nominal p-values. * In the DUPLEX Study, nephrotic syndrome was defined as (a) documentation of nephrotic syndrome in the medical history, or (b) the concurrent presence of proteinuria >3.5 g/24 hours (adults) or UPCR >2.0 g/g (pediatric patients <18 years of age), serum albumin <3.0 g/dL, and edema at baseline. Patients without nephrotic syndrome did not meet both criteria (a) and (b). Overall DUPLEX Population DUPLEX Population without Nephrotic Syndrome* 51
Page 52
© 2026 Travere Therapeutics, Inc. In DUPLEX, FILSPARI Demonstrated Even Stronger Benefit on Exploratory Kidney Outcome Endpoints in FSGS without Nephrotic Syndrome FILSPARI showed clinically meaningful benefit in the broad DUPLEX population, with enhanced efficacy in population without nephrotic syndrome* 0 2 4 6 8 10 12 14 Confirmed ≥50% Reduction in eGFR, ESRD, or Death Confirmed ≥50% Reduction in eGFR, ESRD, or Renal Death Proportion of Subjects Reaching KF, % FILSPARI Irbesartan (23/184) (21/184) (31/187) RR: 0.19 p-value: 0.01801 1.6 8.0 6.5 11.2 RR: 0.58 p-value: 0.07861 DUPLEX Population without Nephrotic Syndrome Overall DUPLEX Population 52 Abbreviations: CI: confidence interval; eGFR: estimated glomerular filtration rate; KF: kidney failure; RR: relative risk; UPCR: urine protein to creatinine ratio. 1 Nominal p-values for odds ratio for rates of no events. * In the DUPLEX Study, nephrotic syndrome was defined as (a) documentation of nephrotic syndrome in the medical history, or (b) the concurrent presence of proteinuria >3.5 g/24 hours (adults) or UPCR >2.0 g/g (pediatric patients <18 years of age), serum albumin <3.0 g/dL, and edema at baseline. Patients without nephrotic syndrome did not meet both criteria (a) and (b).
Page 53
© 2025 Travere Therapeutics, Inc. © 2026 Travere Therapeutics, Inc.