We know that many of you may have questions on the recent regulatory update from Travere Therapeutics on their sparsentan program for FSGS. We’re posting this to help you understand what it means:

• Travere reported that despite the DUPLEX Study achieving its interim endpoint, the FDA indicated in recent interactions that the data from the DUPLEX Study are not yet adequate enough to support accelerated approval
• Travere noted that FDA has acknowledged the high unmet need and the limited treatment options for people living with FSGS
• For anyone participating in or following the DUPLEX Study, there are no changes to report at this time. The study will continue as planned to the final endpoint after 108 weeks of treatment
• Travere is continuing to engage with regulators about generating additional data from DUPLEX in the first half of next year with the gold of submitting for accelerated approval in 2022
• This means that sparsentan could potentially gain accelerated approval in the U.S. in 2023
• Patient retention in the study is critical to providing the FDA with most robust data package possible to support a potential approval at the appropriate time
• Travere remains confident in the potential for sparsentan to become a new treatment standard for FSGS, if approved
• Travere also recently announced that their Phase 3 PROTECT Study in IgAN has completed enrollment and interim data from the trial are expected in August of this year
• The interim data could also lead to an accelerated approval submission for IgAN

To read Travere Therapeutics’ original press release, click here.

Still want to learn more about how a drug gets approved by the FDA? Here is an FAQ list to help you find the information you’re seeking.

How does the FDA determine which study drugs are eligible for accelerated approval and which follow the normal regulatory timeline? 

The purpose of the FDA’s accelerated approval program is to get important new drugs to patients in need earlier. Generally speaking, the drug must have an impact on factors like survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more seriousone. The drug must fulfill an unmet medical need, which the FDA defines as providing a therapy where none exists or providing a therapy which may be potentially better than an available therapy.

How does this news impact my participation in the trial?

This news doesn’t change anything about your participation in this study. In fact, it’s very important that you remain in the study. Your continued participation is the only way Travere’s sparsenten will retain the potential to be granted accelerated approval. Please continue to do everything you are currently doing and comply with all study protocol/requirements. Be sure to talk to your study doctor if you have questions.

The FDA’s response to Travere’s request for accelerated approval was, ‘the available data from the interim assessment of the DUPLEX Study would not be adequate to support an accelerated approval at this time.’ Will they be able to submit for accelerated approval at a later time?

Although Travere was not granted accelerated approval at this time, they may still be able to submit for accelerated approval once additional data accrue in the DUPLEX Study. Subject to further discussions this summer, those data may become available to FDA in the first half of 2022.

What does this mean for Europe?

Travere continues to work through the conditional marketing authorization process and anticipates meeting with European regulators this summer to discuss next steps. Subject to their upcoming discussions with EMA, Travere is still planning for a submission in Europe this year. They expect to provide an update in the coming months.

Have you or your loved one been diagnosed with FSGS? We have resources for you.

The rates of severe kidney disease are high in individuals who are African American, Hispanic black, Afro-Caribbean, or of African ancestry. This could be due to differences in the genetic makeup in the APOL1 gene found typically in individuals with recent African, Caribbean, or Latin American descent. These differences in the genetic makeup are associated with increased rates of hypertension-associated kidney failure, FSGS, HIV-associated kidney disease, and other forms of nondiabetic kidney disease.

Fast facts about APOL1 FSGS:

• Black Americans account for 32% of all kidney failure in the US
• Black Americans are four times more likely to develop kidney failure than White Americans
• Approximately 4 in 10 Black Americans on dialysis have kidney failure caused by APOL1 gene changes
• Approximately 1 in 5 people with two copies of the APOL1 gene changes will develop kidney disease
• The high-risk APOL1 genotype is present in 75% of Black patients with FSGS

Below are resources we’ve complied that might be useful for you:

This informational flyer on APOL1 FSGS breaks down the basics and helps you better understand this disease.

To download the full informational sheet, click here.

In June 2020, we hosted a NephCure U session specifically on APOL1-Associated FSGS. Dr. Jeffrey Kopp from the NIDDK lead, “Kidney Disease in Patients of African Descent: APOL1-Associated Disease” and discussed more on the diagnosis, treatment options, and clinical trials. Listen in on the hour-long educational webinar below.

NephCure co-hosted a GlomCon Clinical Trial Conference Series session on Advances in APOL1 Therapeutics on February 14, 2021, featuring Dr. Ogo Egbuna, Dr. Opeyemi Olabisi, and Dr. David J. Friedman. Click here to watch the recording of this session.

In addition to these resources, there are also clinical trials available for APOL1-Associated FSGS patients. Clinical trials look at the safety and effectiveness of potential new treatments. The main goals of clinical trials are to find new ways to prevent, detect (find) or treat diseases or health conditions, and to make sure potential new treatments or therapies work well and are safe for people.  Check out some pre-screener questions below, provided by Vertex, to see if their clinical trial could be a fit for you or your loved one.

• Eligible participants must meet the following criteria:
• Be male or female adults between the ages of 18 and 65 (inclusive)
• Female participants must not be pregnant or breast-feeding
• Be of African, Caribbean or Latin American descent
• Have had a kidney biopsy which has found focal segmental glomerulosclerosis (FSGS)
• Have not had a diagnosis of kidney disease other than FSGS
• Be willing to complete the investigational apolipoprotein L1 (APOL1) gene test
• Be willing and able to follow the study instructions

To learn more about Vertex’s clinical trial, click here.  If you meet the preliminary criteria listed above, find the location closest to you and click the “I’m Interested” button to get in touch with an investigator for additional evaluation of eligibility.

You can find a full list of clinical trials for all protein-spilling kidney diseases on KidneyHealthGateway.com.

Need an on-the-go snack? Want to switch up the breakfast routine? Try making some of Chef Sachet’s kidney-friendly granola. As a mother and caregiver to a young boy suffering from FSGS, her recipes are tried and true for those following kidney conscious diets.

Granola Recipe

Prep Time: 5 min

Cook Time: 30 min

Yield: A LOT!

Ingredients:

• 6 cups oats
• 3 cups toasted shaved almonds
• 1 1/2 cups toasted sunflower seeds
• 1 cup unsweetened toasted coconut flakes
• 1 cup plumped raisins, dried cranberries
• 1 cup pure maple syrup
• 1 cup honey
• 1/2 cup coconut oil
• 2 tsp ground cinnamon (optional)

Instructions:

1. In a large bowl, toss oats with honey, syrup and oil.
2. Lay onto a non-stick baking sheet and toast in the over at 375 degrees for 20 min (or until oats are toasted and stiff). Be careful as to not over toast the oats as they will burn and turn bitter!
3. Let oats cool completely, then toss with other ingredients.
4. Store in a sealed cereal container or mason jar.

Important tips:

• Nuts (always use unsalted) provide a great source of protein for those trying to stay away from high phosphates in meat.
• Add some m&m’s and dried apricots and you got yourself a yummy trail mix!
• Add some yogurt, fresh fruit, and a drizzle of honey and you got yourself a delicious breakfast alternative!

Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on advancing kidney precision medicine. Its mission is to deliver disease-modifying precision medicine that brings hope and renewed quality of life to people living with kidney diseases. We recently spoke with Goldfinch Bio’s Dr. Liron Walsh, Vice President Clinical and Translational Nephrology, who explains more about the company’s precision medicine approach, breaks down what podocytes are, and speaks more on its Phase 2 clinical research trial, TRACTION-2, which is currently enrolling patients.

Dr. Liron Walsh, Vice President Clinical and Translational Nephrology, Goldfinch Bio

What is precision medicine?

The causes of kidney disease are unique among groups of individuals. In diseases such as focal segmental glomerulosclerosis (FSGS), the causes of disease can vary from genetic to environmental. However, current treatments for FSGS are the same regardless of the cause.

With these “one-size-fits-all” approaches, some patients respond to treatment, while others do not. Many patients experience harmful side effects from these treatments with little to no benefits.

The goal of precision medicine is to develop treatments which are specifically designed to treat the underlying cause of an individual’s kidney disease. In this way, patients can receive the right medicine and avoid treatments which are not going to be helpful or may even be harmful.

Today, precision medicine is often used to treat cancer. By first understanding the genetic makeup of a patient’s tumor, a doctor can then choose the best medicine to specifically treat that patient’s cancer. This approach has greatly improved the lives of many people with cancer.

Precision medicine is now on the horizon for people with kidney diseases. Patients, nephrologists, and kidney researchers are working together to better understand the causes of kidney diseases. Through this deeper understanding, precision medicine will help develop treatments that target the specific causes of kidney diseases like FSGS and minimal change disease (MCD). Precision medicine holds promise to improve treatment outcomes and quality of life for many people living with kidney disease.

Goldfinch Bio and Precision Medicine

Goldfinch Bio is developing a novel experimental medicine, GFB-887, with the hope of preventing the progression of kidney disease in patients with FSGS, treatment-resistant MCD (TR-MCD), and diabetic nephropathy. While these kidney diseases may seem very different, they have something very important in common– very specialized cells of the kidneys called podocytes are damaged. GFB-887 is designed to specifically target these podocytes and prevent damage.

What are podocytes?

Podocytes are very specialized cells that wrap around the blood vessels of the kidneys. They form a network with other nearby podocytes to prevent the body from losing important proteins, such as albumin, while at the same time, filtering out waste. Normal functioning podocytes are very critical to our health.

When podocytes are damaged, gaps form in the barrier and important proteins are lost in the urine. This is commonly referred to as ‘spilling protein in the urine,’ and is an early sign of kidney disease. Patients with damaged podocytes experience swelling, weight gain, and fatigue. Unfortunately, damaged podocytes cannot heal, and they cannot be replaced by new, healthy podocytes. The kidney tries to repair the damage by creating a scar, much like the scar from a cut. Over time, more damage and more scarring will lead to kidney failure. By protecting the podocytes from being damaged in the first place and ensuring that the podocytes can function normally, it may be possible to prevent or delay kidney failure.

What makes the Goldfinch Bio team different than other pharmaceutical companies?

First, we are focused on advancing treatments for people with kidney diseases. Second, we are pioneering precision medicine for kidney diseases. While precision medicine is often used in cancer, and some rare genetic diseases as well, they are an entirely new area of drug research and development for kidney diseases. We are deeply committed to bringing innovative treatments to people with kidney diseases and working very closely with the patient community as partners.

Goldfinch Bio’s tagline is ‘Saving Kidneys, Ending Dialysis.’ How did you come up with this?

Our tagline– Saving Kidneys, Ending Dialysis–  is a shortened version of our vision, which is “We aspire to save kidneys and end dialysis.” We know that for many patients, kidney transplant and dialysis are the only treatment options. Unfortunately, kidney transplant may not be an option for many patients and is associated with significant long-term complications, and the experience of dialysis is very difficult. Through our precision medicine approach, we want to bring new, effective treatment solutions to patients so that we can give them hope and a renewed quality of life. We finalized our vision by hosting a focus group with people living with kidney disease to really understand what was important to them. A resounding theme emerged: these individuals expressed to us that they would give anything to avoid dialysis. And, from there, our new vision emerged.

Can you tell us more about your ongoing clinical trial?

The TRACTION-2 clinical research trial is evaluating our investigational precision medicine, GFB-887, for the potential treatment of FSGS, TR-MCD, and diabetic nephropathy. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. For more information on the trial, you can visit tractionclinicaltrial.com or at Kidney Health Gateway.

What are you most looking forward to at Goldfinch Bio?

We are very excited about the recent creation of our FSGS Patient Advisory Board.  We have created this board to foster a close working partnership with the patient community. For National Kidney Month, we held a virtual panel discussion with our board members. You can learn more about our Patient Advisory Board and watch the panel discussion here. We are very excited to continue to collaborate with this Board to ensure we have a patient-centric approach and mindset throughout our company.

To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities, and to find the trial best for you or your loved one, visit KidneyHealthGateway.com.

This article was developed in partnership with Goldfinch Bio, Inc.

Have you ever wondered what it might be like to walk in a kidney specialists’ shoes for a day?

Get a rare peek ‘behind the curtain’ and hear from actual nephrologists about their perspective on treating rare kidney patients, some of their daily challenges, and tips for making the most of your appointments.

Join us Sunday, May 16th from 12-4pm ET for the 2021 NephCure Patient Summit. During this FREE virtual gathering of patients from around the globe, you’ll hear about the latest in research and treatments and learn about your disease. You’ll also have the opportunity to directly ask our experts questions and participate in interactive community roundtables.

The “Day in the Life of a Nephrologist” panel will feature Drs. Jason Cobb, Michelle Rheault, and Suneel Udani. The Patient Summit will also offer you the chance to hear from other NephCure specialists, NephCure staff, and regional volunteer leaders.

Meet the Panelists:

Dr. Jason Cobb is an assistant professor of medicine in the Emory University Division of Renal Medicine. Dr. Cobb is a graduate of Morehouse College with a BS in biology. He holds a medical degree from Emory University School of Medicine and completed his internal medicine residency and nephrology fellowship at Emory University. He is board-certified in internal medicine and nephrology. He sees patients in nephrology clinic at Emory University Hospital Midtown in the Medical Office Tower seeing patients with nephrology complaints such as chronic kidney disease due to diabetes, hypertension, vascular disease, and glomerulonephritis. Also, Dr. Cobb takes care of hemodialysis patients at Emory Dialysis Greenbriar and Emory Dialysis Northside, and home dialysis patients at Emory Dialysis Northside. He also rounds on inpatient services at Emory University Hospital Midtown and Emory University Hospital. Research and teaching interests include quality improvement and nephrology fellow clinic at Grady Memorial Hospital.

Dr. Michelle Rheault is a board certified pediatric nephrologist and Associate Professor of Pediatrics. She completed her Pediatric Nephrology fellowship at the University of Minnesota in 2006 followed by a research fellowship at Mount Sinai School of Medicine in New York City. She joined the faculty at the University of Minnesota in 2008 and is currently the Director of the Division of Pediatric Nephrology and Medical Director of the Pediatric Dialysis unit. She is on the steering committee of the Pediatric Nephrology Research Consortium, a clinical research network that aims to facilitate collaboration in pediatric nephrology. Her clinical and research interests include Alport syndrome and other genetic kidney diseases, pediatric glomerular disease, and pediatric end stage kidney disease.

Dr. Suneel Udani is a native of Chicago’s western suburbs.  He joined Advanced Renal Care in July 2011. He completed his undergraduate, Master’s in Public Health, and medical school degrees at Northwestern University and went on to do his Internal Medicine training at the University of Chicago and the University of Pittsburgh. He served as a Chief Medical Resident at Cook County Hospital prior to returning to the University of Chicago for his fellowship in Nephrology. Dr. Udani is the author of multiple peer-reviewed journal articles and has presented his research at national conferences and is the author of multiple text book chapters on diagnosis and management of kidney disease. His focus is on glomerular disease and heart-kidney interactions. He is also a clinical investigator for community-based renal research program and a dedicated educator and advocate for patients with kidney disease.

To learn more about the 2021 NephCure Patient Summit, including a full list of speakers, and full event agenda, click here. 

NephCure’s Nurse Kristen Hood and Kylie Winkler discuss Travere’s Therapeutics achievement of interim proteinuria endpoint in the ongoing Phase 3 DUPLEX study of Sparsentan in Focal Segmental Glomerulosclerosis (FSGS).

Learn more about the trial currently underway, why this is such a crucial step for patients with rare kidney diseases, and what potential treatments this could lead to in just a few short years! Read the full press release here.