CSL Vifor and Travere Therapeutics announce EMA has accepted for review the Conditional Marketing Authorization application for sparsentan for the treatment of IgA Nephropathy

A review decision by the European Medicines Agency (EMA) is expected in the second half of 2023

If approved, sparsentan will be a first-in-class treatment to address the significant unmet medical need in IgA nephropathy (IgAN) in Europe

CSL Vifor and Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the EMA has accepted for review the Conditional Marketing Authorization (CMA) application for sparsentan for the treatment of IgAN, a rare kidney disorder and a leading cause of end-stage kidney disease (ESKD). The EMA will review the application under the centralized marketing authorization procedure and a review decision on a potential approval is expected in the second half of 2023.

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“The acceptance of the EU regulatory application for sparsentan marks a major milestone towards our goal of bringing this first-in-class therapy to the patients suffering from IgAN, for which there are currently no approved non-immunosuppresive therapies,” commented Klaus Henning Jensen, Chief Medical Officer of CSL Vifor. “We look forward to working closely with our partner, Travere, through the EMA review process with the aim to bring this innovative treatment option to patients living with IgAN in Europe.”

“Following U.S. FDA’s acceptance and granting of priority review of the NDA for sparsentan for IgA nephropathy in the U.S., we continue to make great progress towards our goal of enabling sparsentan to become a new treatment standard for rare kidney disorders, if approved,” said Jula Inrig, M.D. chief medical officer of Travere Therapeutics. “The acceptance of the CMA application marks an important next step on our pathway to expanding access to sparsentan as the first non-immunosuppressive treatment option for IgA nephropathy in Europe, if approved. We look forward to continuing to collaborate with our partners at CSL Vifor and with the EMA throughout the review process.”

The EMA filing is supported by positive topline interim results from the ongoing pivotal phase-III PROTECT Study of sparsentan in IgAN, as well as supportive data from additional clinical studies. The PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients. Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated to date in the study and consistent with its overall observed safety profile.

If approved, sparsentan would receive CMA in all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

Sparsentan is currently also being evaluated in the pivotal phase-III DUPLEX Study for the treatment of focal segmental glomerulosclerosis (FSGS), another rare progressive kidney disorder and leading cause of end-stage kidney disease. Sparsentan has been granted Orphan Drug Designation for the treatment of IgAN and FSGS in Europe and in the U.S.

About CSL Vifor

CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency, dialysis and nephrology & rare disease. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care).

The parent company, CSL (ASX:CSL; USOTC:CSLLY), headquartered in Melbourne, Australia, employs 30,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, www.cslvifor.com.

About Travere Therapeutics

At Travere Therapeutics, we are in rare for life. 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. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

About IgA Nephropathy

IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disorder characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure.

IgAN is the most common type of primary glomerulonephritis worldwide and a leading cause of end-stage kidney disease (ESKD). IgAN is estimated to affect more than 100,000 people in the U.S. and is one of the most common glomerular diseases in Europe and Japan. There are currently no approved non-immunosuppressive treatments indicated for IgAN.

About the PROTECT study

The ongoing PROTECT Study is one of the largest interventional studies to date in IgAN. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400mg of sparsentan, compared to 300mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite available ACE or ARB therapy. In August 2021, the Company announced the PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients (p<0.0001). The Company believes that preliminary eGFR data available at the time of the interim analysis are indicative of a potential clinically meaningful treatment effect after two years of treatment. Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated to date in the study and consistent with its overall observed safety profile. The PROTECT Study is fully enrolled and is scheduled to continue as planned on a blinded basis to assess the treatment effect on eGFR slope over 110 weeks in the confirmatory endpoint analysis. Topline results from the confirmatory endpoint analysis are expected in the second half of 2023.

About sparsentan

Sparsentan, a Dual Endothelin Angiotensin Receptor Antagonist (DEARA), is a novel investigational product candidate selectively targeting the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). Pre-clinical data have shown that blockade of both endothelin type A and angiotensin II type 1 pathways in forms of rare chronic kidney disease, protects podocytes, prevents glomerulosclerosis and mesangial cell proliferation, and reduces proteinuria.

Sparsentan is also currently being evaluated in the pivotal phase-III DUPLEX study for the treatment of FSGS. In February 2021, Travere announced that the ongoing DUPLEX study of sparsentan in FSGS achieved its pre-specified interim FSGS partial remission of proteinuria endpoint (FPRE) with statistical significance. FPRE is a clinically meaningful endpoint defined as urine protein-to-creatinine ratio (UP/C) ≤1.5 g/g and a >40 percent reduction in UP/C from baseline. After 36 weeks of treatment, 42.0 percent of patients receiving sparsentan achieved FPRE, compared to 26.0 percent of irbesartan-treated patients. Preliminary results from the interim analysis suggest that at the time of the interim assessment, sparsentan had been generally well-tolerated and shown a comparable safety profile to irbesartan. In the phase-II DUET study of sparsentan in FSGS, the combined treatment group met its primary efficacy endpoint, demonstrating a greater than two-fold reduction in proteinuria compared to irbesartan, and was generally well tolerated after the eight-week, double-blind treatment period. Irbesartan is part of a class of drugs used to manage FSGS and IgAN. If approved for both indications, sparsentan could potentially be the first medicine approved for both FSGS and IgAN.

Forward Looking Statements

This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: the EMA’s potential approval of sparsentan for IgAN in the second half of 2023 or at all; the potential for sparsentan to be the first non-immunosuppressive treatment option for IgA nephropathy approved in Europe and to be a first-in-class treatment; the goal of enabling sparsentan to become a new treatment standard for rare kidney disorders, if approved; references to the companies’ expectations of working with the EMA during the review process; references to the number of IgAN patients in Europe who may have sparsentan as a treatment option, if approved; references to the efficacy, safety and tolerability profile of sparsentan based on the preliminary data from the PROTECT Study interim analysis; the companies’ belief that preliminary eGFR data available at the time of the interim analysis from the PROTECT Study are indicative of a potential clinically meaningful treatment effect after two years of treatment; expectations regarding the future conduct of the ongoing PROTECT study and timing for topline results from the confirmatory endpoint analysis; references to the estimated number of IgAN patients in Europe and the United States; and the potential for sparsentan to become the first medicine approved for both FSGS and IgAN. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the regulatory review and approval process, including the Subpart H accelerated approval pathway in the United States and the conditional marketing authorization (CMA) pathway in the Europe Union. Specifically, the companies face the risk that the Phase 3 PROTECT Study of sparsentan in IgAN will not demonstrate that sparsentan is safe or effective or serve as the basis for accelerated approval of sparsentan as planned; risk that the Phase 3 DUPLEX Study of sparsentan in FSGS will not demonstrate that sparsentan is safe or effective or serve as a basis for approval of sparsentan as planned; and risk that sparsentan will not be approved for efficacy, safety, regulatory or other reasons, and for each of the companies’ programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. There is no guarantee that the EMA will grant conditional marketing authorization of sparsentan for IgAN or that sparsentan will be approved at all. Travere faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to its 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; risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of its products, and technological changes that may limit demand for its products. The companies face additional risks associated with the potential impacts the COVID-19 pandemic may have on their business, including, but not limited to (i) the companies’ ability to continue their ongoing development activities and clinical trials, (ii) the timing of such clinical trials and the release of data from those trials, (iii) the companies’ and their suppliers’ ability to successfully manufacture their commercial products and product candidates, and (iv) the market for and sales of their commercial products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The companies undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in Travere’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.

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