How would you describe Travere and your vision for the company since you joined in 2019?
Travere is a biopharmaceutical company dedicated to rare kidney, liver, and metabolic diseases. Many of our colleagues are rare disease patients, caregivers, and/or survivors like me who have a special commitment to the field. This is a critical aspect of our holistic approach, since we aim to focus not just on the drug, mechanism, and target of the disease, but also on developing a deep understanding of the individual experiences of patients and their caregivers.
Why did Travere choose to focus on kidney and metabolic diseases out of the 8,000 rare diseases out there?
If we look back at the last forty or fifty years of research in different therapeutic areas, rare glomerular diseases are some of the highest contributors to kidney failure and have had the lowest levels of clinical trial activity for decades.
Part of the reason for this is that some of the rare causes of kidney disease have been underrecognized and are challenging to study from a regulatory and clinical trial perspective. Many of our colleagues in the field, such as our chief medical officer, have worked over the last decade to help establish the “end points” for trial design that best enable innovation in drug development.
We work to develop new therapies, raise awareness about rare kidney, liver, and metabolic disease, and we are dedicated to deliver innovation in areas with high unmet need. We are setting a precedent for more rapid clinical study approaches that respect the differences in trial design for rare diseases.
What are the plans for sparsentan’s commercialization after its positive interim Phase 3 results?
We are currently carrying out Phase 3 trials on two rare kidney conditions called focal segmental glomerulosclerosis (FSGS) and IgA Nephropathy (IgAN). Both trials test proteinuria and eGFR as short- and longer-term measures of kidney function. What we have seen so far is very exciting and has allowed us to submit our first application in the U.S. and Europe for sparsentan as a treatment for IgAN. And once we read out the results of our Phase 3 DUPLEX Study in FSGS next quarter, we plan to submit another application for that indication- pending supportive data. This means that we could be the first to have a treatment option for FSGS and a non-immunosuppressive therapy for IgAN (if it is approved later this February).
Do you feel that rare diseases are receiving the support that they need from policymakers?
Current treatment options for IgAN like ACEs and ARBs, are not indicated for IgAN, and long-term steroid prescriptions, dialysis, and transplants are not enough. Patients living with rare kidney diseases have helped members of Congress understand that there is much more that needs to be done in terms of lowering the burden caused by rare kidney diseases. There is also a rare kidney disease bill under consideration that would help spur investment in innovation and address some of these patients’ fundamental needs at a community level.
In your biography we see a passionate defense of minority patients who are often not given equal access to treatment and care. How do you consider that your own life story has influenced your commitment to inclusion?
Growing up poor and coming out as gay during the AIDS epidemic in the ‘90s, I saw the little value that society, the government, and health care providers placed on the lives of the men who were dying. That was the moment when I made my decision to go into a career that would help bring about a broader societal difference. In my career, I use my position to advocate for the health of all communities, particularly to give a voice to patients who might feel like they lack access to the medical care that they need.
What are the main two objectives for Travere in the coming years?
First and foremost, pending FDA accelerated approval for IgAN, our goal is for sparsentan to have a very successful launch by reaching IgAN patients who have been waiting for a new treatment for their rare condition. In addition to being a transformative milestone for the company, the approval and launch would give Travere the opportunity to set the bar as a leader in the field. We will continue to put rare patients and families at the center of our work.