Travere Therapeutics Inc to Discuss the Topline Results from the Ongoing Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Congrats on the data. Can you give any additional details on the patient who experienced the hives? And if any other patients had any, maybe less severe infusion-related reactions? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 15, 2021 / 1:30PM, TVTX.OQ - Travere Therapeutics Inc to Discuss the Topline Results from the Ongoing Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria Call

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Great. And then I also wanted to ask about compliance with the low protein diet during the trial. If you have any information on that? And would you expect patients in the future to be able to eat a normal diet, if they're on drug? I can imagine that'd be a pretty attractive attribute for patients and their families.

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Congrats on the data. Maybe just following up on some of the questions around the long-term extension. How many patients have rolled into the long-term extension? And then have you dose escalated within the long-term extension as you've gone up cohorts? I guess I'm wondering if you see any deepening of the, I guess, intra-patient homocysteine reductions as you escalate the dose? And also, are you seeing an effect as placebo patients roll over? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 15, 2021 / 1:30PM, TVTX.OQ - Travere Therapeutics Inc to Discuss the Topline Results from the Ongoing Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria Call

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. So you mentioned that there's a patient that was dosed 3 years ago, I think in your opening remarks. So the patients -- so have patients not continued therapy, I guess, continuously since they enrolled in the study?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. And in those other dose cohorts, are you seeing that the reductions in homocysteine are sustained through the long term?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. And then you mentioned that about half of patients were on B6 when they entered the study. Did you see similarities in, I guess, the magnitude of response between patients that were on vitamin B6 and patients that were not on vitamin B6?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : And then the high dose cohort that you are evaluating, just I guess a couple of clarifying questions. The first is that you will use the lyophilized formulation in that dose cohort. Am I hearing that correct? And then the other is -- are you evaluating a different dose frequency? Or is it just a higher dose? Do you think that -- I guess I'm wondering is like twice a week going to be the frequency going forward, do you think? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 15, 2021 / 1:30PM, TVTX.OQ - Travere Therapeutics Inc to Discuss the Topline Results from the Ongoing Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria Call

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. If I can sneak one more in. Sorry. Do you have a plan for, I guess, a pivotal trial that you are planning to present to the FDA in your meetings -- in your end of Phase II meeting? Or are you having more general discussions? And I guess I'm wondering if you have an idea of what you're planning yet to bring to the FDA, or if you are just going to kind of have an initial discussion to discuss the data and get their feedback?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. Are you able to give us an idea of whether or not the study design that you're thinking of now is focused on a biomarker, and maybe if you have any idea of what the right duration might be for a pivotal design?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Okay. Do you have any idea on like how big the study might be?