Q1 2022 Travere Therapeutics Inc Earnings Call

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Please standby.

Operator: Please stand by. Good day and welcome to the Travere Therapeutics conference call. Today's conference is being recorded. At this time, I'd like to turn the conference over to the Senior Vice President of Investor Relations, Chris Cline. Please go ahead, sir.

Good day and welcome to the trivia Therapeutics Conference call. Today's conference is being recorded at this time I'd like to turn the conference over to the senior Vice President of Investor Relations, Chris Cline. Please go ahead Sir.

Great. Thank you Justin good afternoon, and welcome to severe therapeutics first quarter 2022 financial results and corporate update call. Thank you all for joining us.

Chris Cline: Great, thank you, Justin. Good afternoon, and welcome to Servere Therapeutics' first quarter 2022 financial results and corporate update call. Thank you all for joining us. Today's call will be led by our Chief Executive Officer, Dr. Eric Dube. Eric will be joined for the prepared remarks by Dr. Jula Inrig, our Chief Medical Officer; Peter Heerma, our Chief Commercial Officer; and our Chief Financial Officer, Laura Clay. Dr. Bill Rote, Senior Vice President of Research and Development, will join us for the Q&A session.

Today's call will be led by our Chief Executive Officer, Dr. Eric Today, Eric will be joined for the prepared remarks by Dr. Julie <unk>, Our Chief Medical Officer, Peter Hermann <unk>, our Chief commercial officer, or Chief Financial Officer, Laura Quake.

Bill Rote senior Vice President of research and development will join us for the Q&A session. Before we begin I would like to remind everyone that statements made during this call regarding matters that are not historical facts are forward looking statements within the safe Harbor provisions of the private Securities Litigation Reform Act of 1995.

Chris Cline: Before we begin, I would like to remind everyone that statements major in this call regarding matters that are not historical facts are forward-looking statements within the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees of performance. They involve known and unknown risks, uncertainties, and assumptions that may cause actual results, performance, and achievements to differ materially from those expressed or implied by the statement.

Forward looking statements are not guarantees of performance they involve known and unknown risks uncertainties and assumptions that may cause actual results performance and achievements to differ materially from those expressed or implied by the statement.

Please see the forward looking statement disclaimer on the company's press release issued earlier today as well as the risk factors section of our Form 10-Q, and 10-K filed with the SEC. In addition, any forward looking statements represent our views only as of the date such statements are made they said, 20% to interfere specifically disclaims any obligation to update such statements to reflect future information events or circumstances.

Chris Cline: Please see the forward-looking statement disclaimer in the company's press release issued earlier today, as well as the risk factors section in our Forms 10-Q and 10-K filed with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made, May 5, 2022, and Travere specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. I'll now turn the call over to Eric. Thank you, Chris, and good afternoon.

I'll now turn the call over to Eric Eric.

Thank you, Chris and good afternoon, I'm very pleased with our progress to start 2022 during the first quarter, we made significant headway on our key priorities of advancing our pipeline of potential first in class treatments for people living with rare disease.

Eric Dube: I'm very pleased with our progress to start 2022. During the first quarter, we made significant headway on our key priorities of advancing our pipeline of potential first-in-class treatments for people living with rare diseases and preparing our organization for potential launches of Sparcentin if approved. Most notably, during the quarter, we achieved an on-time submission of our new drug application, or NDA, for accelerated approval of Sparcentin in IgA nephropathy. We look forward to receiving notice later this month from the FDA regarding the acceptance of the NDA as well as the timeline for review. If the NDA is accepted and we are granted priority review, we would anticipate a launch in hygiene and property as early as the end of this year if Sparsantan is approved.

In preparing our organization for potential launches of sports that says if approved.

Most notably during the quarter, we achieved an all time submission of our new drug application or NDA for accelerated approval of <unk> in Iga nephropathy, we look forward to receiving notice later this month from the FDA regarding the acceptance of the NDA as well as the timeline for review.

If the NDA is accepted and we were granted priority review, we would anticipate a launch in Iga nephropathy as early as the end of this year as far as <unk> is approved.

We're also on track to submit additional Egfr data from the ongoing duplex study to the FDA this quarter consistent with our prior guidance, we will be providing an update following our regulatory interactions, but we will not be providing incremental updates before then.

Eric Dube: We are also on track to submit additional EGFR data from the ongoing duplex study to the FDA this quarter. Consistent with our prior guidance, we will be providing an update following our regulatory interactions, but we will not be providing incremental updates before then. As a reminder, we believe the potential outcomes are either the FDA is supportive of a submission of accelerated approval for FSGS, in which case, we will be preparing to submit an NDA under subpart H in the coming months, or the FDA recommends that we instead wait until full completion of the study in the first half of next year, and we would subsequently expect to submit for full approval in 2023.

As a reminder, we believe the potential outcomes that are either at the FDA is supportive of a submission of accelerated approval for <unk>.

In which case, we will be preparing to submit an NDA under subpart H in the coming months or that the FDA recommends that we instead wait until full completion of the study in the first half of next year and we would subsequently expect to submit for full approval in 2023.

Together with our European partner <unk> for pharma, we also remain prepared to submit a combined Iga nephropathy and S. G. S. MAA submission for conditional approval of <unk> in Europe pending alignment with the regulatory path in the U S.

Eric Dube: Together with our European partner, B4 Pharma, we also remain prepared to submit a combined IgA nephropathy and FSGS MAA submission for conditional approval of sparsentin in Europe, pending alignment with the regulatory path in the U.S. I am also pleased with the further positive reception of the data from our ongoing Phase 1-2 Compose study of PEG dibatinase following its presentation at SIND last month. Pegtybatinase represents an exciting potential new treatment for people living with classical homocystinuria, or HCU.

I'm also pleased with the further positive reception of the data from our ongoing phase one two composed study of pegged about Nate following his presentation at S. I M D last month.

I think about <unk> represents an exciting potential new treatment for people living with classical home assistant urea or each see you and we look forward to continuing to progress our discussions with regulators on the design of a pivotal program.

Eric Dube: And we look forward to continuing to progress our discussions with regulators on the design of a pivotal program. On the commercial side of the business, our performance for the quarter was strong and in line with expectations in a challenging environment. As many of you will recall, during our end of year update, we outlined that in 2022, we do not anticipate overall growth in net product sales due to the generic dynamics of FIOLA. Our first quarter performance is consistent with that data. Demand across all products in the quarter increased.

On the commercial side of the business our performance for the quarter was strong and inline with expectations in a challenging environment as many of you will recall during our end of year update we outlined that in 'twenty 'twenty. Two we do not anticipate overall growth in net product sales due to the generic dynamics for fire a lot are.

First quarter performance is consistent with that guidance demand across all products in the quarter increased in fact, we are seeing a return to pre pandemic levels for overall demand.

Eric Dube: In fact, we are seeing a return to pre-pandemic levels for overall demand, and the Bile Acid Portfolio continues to perform well. But, as expected, the dynamics of the evolving competitive landscape in Sistineria are beginning to put pressure on the net revenue from our thiol abyss. This shift is accounted for in our planning, and I continue to be incredibly impressed by our team's ability to maintain continuity of the business and support for the patient community despite these challenges.

And the bile acid portfolio continues to perform well.

But as expected the dynamics for the evolving competitive landscape insistent urea are beginning to put pressure on the net revenue from our Faiola business.

This shift is accounted for in our planning and I continue to be incredibly impressed by our team's ability to maintain continuity of the business.

And support for the patient community. Despite these challenges notably.

Notably Peter in the commercial organization continued to do a fantastic job of building upon the strengths and expertise of our current sales team and nephrology to prepare for a launch of sports centered in the U S. As early as the end of the Europe approved.

Eric Dube: Notably, Peter and the commercial organization continue to do a fantastic job of building upon the strengths and expertise of our current sales team in nephrology to prepare for a launch of Sparcentin in the U.S. as early as the end of the year. As a result of our progress to date, both in the clinic and from a commercial preparation perspective, I remain confident that, if approved, Sparsentin can become a new treatment standard for the many patients with rare kidney disease currently facing a rapid progression to dialysis. Finally, during the quarter, we strengthened our financial foundation through the convertible refinancing transactions enacted in March.

As a result of our progress to date, both in the clinic and from a commercial preparation perspective I remain confident that if approved <unk> can become a new treatment standard for the many patients with rare kidney disease currently facing a rapid progression to dialysis.

Finally during the quarter, we strengthened our financial foundation through the convertible refinancing transactions enacted in March these transactions resulted in us being able to effectively extend the maturity on the vast majority of our convertible notes from 2025 to 2029 and that modest incremental cash to the balance sheet.

Jula Inrig: These transactions resulted in us being able to effectively extend the maturity on the vast majority of our convertible notes from 2025 to 2029 and add modest incremental cash to the balance sheet. I will now turn the call over to Jula for a clinical update.

Let me now turn the call over to July for a clinical update sure.

Thank you Eric and good afternoon, I'm pleased to report that in the first quarter, we advanced all of our clinical programs.

Jula Inrig: Thank you, Eric, and good afternoon. I'm pleased to report that in the first quarter, we advanced all of our clinical programs. First, Barsentine, the most notable achievement during the quarter, was the submission of an NDA for accelerated approval in IgA nephropathy. In looking at the IJ nephropathy treatment paradigm, there's a desperate need for novel, non-immunosuppressive treatment.

The most notable achievement during the quarter with the submission of an NDA for accelerated approval and Iga nephropathy.

And looking at the Iga nephropathy treatment paradigm, there is a desperate need for novel non immunosuppressive treatments.

Physicians are currently treating patients with Iga nephropathy, with RAF inhibitors, and steroids and an attempt to reduce proteinuria delay the progression to dialysis and avoid the need for transplantation if possible.

Jula Inrig: Physicians are currently treating patients with IgA nephropathy with RAS inhibitors and steroids in an attempt to reduce proteinuria, delay the progression to dialysis, and avoid the need for transplantation if possible. However, what we've seen for many years is that the available treatments are limited in their effectiveness or long-term safety. Many patients are still progressing at a rapid pace to end-stage kidney failure. Xpercenton is ultimately a- It has the potential to be the first non-immunosuppressive treatment indicated for IJ nephropathy, and we believe it has the ability. Become a Foundational Therapist.

What we've seen for many years is that the available treatments are limited in their effectiveness or long term safety.

Patients are still progressing at a rapid pace to end stage kidney disease.

As far as <unk> is ultimately approved it has the potential to be the first non immunosuppressive treatment indicated for Iga nephropathy, and we believe has the ability to become foundational for the future.

NDA submission in March was a critical step on the path to potentially delivering <unk> to patients and physicians.

Jula Inrig: NDA submission in March was a critical step on the path to potentially delivering Sparcentin to patients and families. Given the significant unmet need in IJNF properties and the strength of the data from the PROTECT study, we requested priority review in our, We expect to receive a response on the acceptance of the submission and a review timeline later this month if priority review is granted. Expect that the new facts

Given the significant unmet need in Iga nephropathy, and the strength of the data from the protect study we requested priority review in our submission.

We expect to receive a response on the acceptance of the submission and the review timeline later this month.

If priority review is granted we would expect that the action date and potential approval as early as November of this year.

Jula Inrig: Possible approval, as early as November of this year. We look forward to working with the FDA throughout the review process. We also remain on track to provide the FDA with additional EGFR data from the ongoing duplex study of Firsantin and FSGS this quarter. As Eric outlined earlier, we will provide an update once we've had our regulatory interaction, consistent with our plans since our Type A meeting last year should the additional eGFR data meet the agency's requirements for accelerated approval.

We look forward to working with the FDA throughout the review process.

We also remain on track to provide the FDA with additional Egfr data from the ongoing duplex study for Santana and FSD, Yes this quarter.

As Eric outlined earlier, we will provide an update once we've had our regulatory interactions.

Consistent with our plan since our type a meeting last year, you said the additional egfr data meet the agency's requirements for accelerated approval, we will be in position to quickly submit NDA breakfast yes.

Jula Inrig: We will be in position to quickly submit an NDA for FSGS, as well as a joint MAA submission with our European partner, V4 Pharma. I am pleased that both Duplex and Protect, the largest studies to date in FSGS and IGAN, respectively, continue to progress and remain on track for their completion next year. Finally, we continue to be excited about the potential for PEG-Tobatinase to become the first... Modifying Approach, Thelma Cisneria In April, we had the opportunity to present detailed results from the first five cohorts of the Phase I-II Composed Study at the SIMD Annual Meeting in 2022.

Well as it joined MAA submission with our European partner <unk> pharma.

I am pleased that both duplex and protect two of the largest studies to date and FX, Yes, and again, respectively continued to progress and remain on track for their completion next year.

Finally, we continue to be excited about the potential for <unk> to become the first disease modifying approach Thomas a scenario.

In April we had the opportunity to present detailed results from the first five cohorts of the phase <unk> study at the <unk> annual meeting for 2022.

The data, including rapid and sustained decreases and homocysteine in methionine and achieving mean homocysteine levels below 100 micro mill demonstrate <unk> can improve overall metabolic dysfunction and support proof of concept for <unk> as a potential treatment for HCV.

Jula Inrig: The data, including rapid and sustained decreases in homocysteine and methionine and achieving mean homocysteine levels below 100 micromole, demonstrate pegtobatinase can improve overall metabolic dysfunction and support proof of concept for pegtobatinase as a potential treatment for HCU.

Throughout the presentations of the data engagement with Kols patient leaders and an AD board held in conjunction S. AMD.

Jula Inrig: Through the presentations of the data, engagement with KOLs, patient leaders, and an advertising board held in conjunction at SIMD, with uniformly positive feedback on Ped-to-Batinase Potential in HCU, and invaluable insights to incorporate into our pivotal program planning. Patients from the first five cohorts in the composed study are continuing in the open-label extension, and we're continuing enrollment activities in the sixth cohort. The continued advancement of the COMPOSE study is designed to build upon the strong initial data set as we plan for the next phase of development. In parallel, we're advancing our discussions with regulators on a pivotal program while working through ongoing global supply constraints. Scale CMC and Manufacturing for the Pivotal Phase of Development and Commercial Access

Received uniformly positive feedback on <unk> potential and Hcl.

And invaluable insights to incorporate into our pivotal program planning.

Patients from the first five cohorts in the <unk> study are continuing in the open label extension and we're continuing enrollment activities and the sixth cohort.

The continued advancement of the <unk> study is designed to build upon the strong initial dataset as we plan for the next phase of development.

In parallel we're advancing our discussions with regulators on a pivotal program, while working through ongoing global supply constraints to scale CMC and manufacturing for the pivotal phase of development and commercial access.

We are pleased with our progress on both of these initiatives and we look forward to providing additional updates later this year.

Peter Heerma: We are pleased with our progress on both of these initiatives and we look forward to providing additional updates later this year. I will now turn the call over to Peter for the commercial update.

Let me now turn the call over to Peter for the commercial update Peter.

Thank you Judah.

Peter Heerma: Thank you, Jula. We continue to see great execution from our commercial organizations, which is particularly encouraging as we move closer to the first potential launch of Sparsanton, if approved. The first quarter of the year had the highest number of new patients initiating our therapies since the first quarter of 2020. We believe this is a result of our team's continued efforts, increased access to engage with prescribers, and patients visiting their physicians again more regularly. Despite the increase in new patients initiating therapy, we did see a slight decline in net product sales compared to the same period last year.

We continue to see great execution from our commercial organization, which is particularly encouraging as we move closer to the first potential of Spartan is approved.

First quarter of the year at the highest number of new patients initiate therapy since the first was 2010.

We believe this is a result of our team's continued efforts increased access to engage with prescribers and patients visiting guests issued again more regularly.

Despite increasing new patients initiating therapy, we did see a slight decline in net product sales compared to the same period last year.

This is driven by generic dynamics affecting sale, which is consistent with our expectations and business lending.

Peter Heerma: This is driven by generic dynamics affecting FIAL law, which is consistent with our expectations and business planning. As we predicted at the beginning of the year, we expect further pressure on Biola through the balance of the year, but we will work to continue identifying new patients and continue to support the systemuria community. Notably, in the first quarter, our bile acid product experienced 14% organic growth over the same period last year, primarily driven by the team's execution with Goldbaum.

As we guided at the beginning of the year, we expect further pressure on payroll through the balance of the year.

We will work to continue identifying new patients and continue to support the system urea community.

Notably in the first quarter, our bile acid product experienced 14% organic growth over the same period last year.

Primarily driven by the team's execution in this global.

While we see quarterly performance was the bile acid products due to the ultra rare nature of the conditions. They treat this was a particularly strong quarter for cobalt, which we believe is a testament to our goldbaum students continue engagements.

Peter Heerma: While we often see uneven quality performance with the bio-acid products due to the ultra-rare nature of the conditions they treat, this was a particularly strong quarter for Cobalm, which we believe is a testament to our Cobalm team's continued engagement. We continue to expect modest growth of the bio-acid products in 2022, which is expected to partially offset the generic impact on failure loss. Most importantly, our performance is within our expectations to start the year, and it continues to demonstrate our ability to identify and treat patients with rare diseases.

We continue to expect modest growth in the bile acid products in 2022, which is expected to potentially partially offset the generic impact.

<unk>.

Most importantly, our performance is within our expectations to start the year and it continues to demonstrate our ability to identify and treat patients with rare diseases.

Furthermore, it provides added confidence in our planning and execution as we prepare for the upcoming potential launches of Spartan.

Peter Heerma: Furthermore, it provides added confidence in our planning and execution as we prepare for the upcoming potential launches of Sparcentrum in the future. As we move ahead through the balance of 2022, we will leverage our nephrology call points to develop a deeper understanding of those physicians who treat patients with IgA nephropathy and FHDS, and we will continue to focus on the three key areas we outlined at the outset of the year.

If approved.

As we move through the balance of 2022, we will leverage our nephrology goalposts to develop a deeper understanding of those physicians, who treat patients with Iga nephropathy and <unk>, yes.

And we will continue to focus on the three key areas outlined at the outset of the year.

Working with our medical team. So they can provide helpful disease State education.

Peter Heerma: Working with our medical team so they can provide helpful disease pain education, advancing the compelling value proposition to payers, and expanding our established commercial infrastructure to be ready to support the launches if SPARC's income is approved. I continue to be very pleased with our progress. For example, we have already hired senior leadership and senior sales leadership with extensive RARE and AFROLOGY experience.

Dumping with compelling value proposition to payers.

Extending our established commercial infrastructure to be ready to support the launches of stocks income is approved.

I continue to be very pleased with our progress for example, we have already hired senior leadership.

Senior sales leadership has extensive rare in the fall as the experience and we had incredibly strong demand from high quality 10 days.

Laura Clay: And we have incredibly strong demand from high-quality candidates for our field-based roles to ultimately support Spar Center. All together, we remain confident that we will be in a position to enable SPARC Centrum to become a new treatment standard in rare nephrology, if approved. I will now turn the call over to Laura for the financial updates.

Field based tools to ultimately supports Barr syndrome.

Altogether, we remain confident that we will be in a position to enable <unk> to become the new treatment standard in rare nephrology is approved.

I will now turn the call over to Laura for the financial update.

Uh huh.

Thank you Peter.

Laura Clay: Thank you, Peter. For the first quarter of 2022. We reported total revenue of $48.5 million, consisting of approximately $46.4 million in net product sales and $2 million in collaboration revenue from our European partnership with V4Pharma. This compares to $47.4 million in net product sales reported for the same period in 2021. As has been typical for us in years past, we did experience higher growth in net discounts in the first quarter, driven by insurance coverage changes at the beginning of the new year.

The first quarter of 2022.

We reported total revenue of $48 5 million.

Listing of approximately $46 4 million and net product sales and $2 million in collaboration revenue from our European partnership with V for pharma. This.

Compares to $47 4 million in net product sales reported for the same period in 2021.

As has been typical for us in years past, we did experienced higher gross to net discounts in the first quarter driven by insurance coverage changes and then beginning of the new year.

As we move through the balance of the year. The gross to net discounts are expected to narrow, but will continue to be higher than in previous years for the sale of a business.

We reported a GAAP net loss of 76 million for the first quarter of 2022.

Laura Clay: As we move through the balance of the year, the growth in net discounts is expected to narrow, but will continue to be higher than in previous years for the FIOLA bids. We reported a gap net loss of $76 million for the first quarter of 2022.

After adjusting for noncash expenses and income tax we reported a non-GAAP net loss of 51 6 million for the first quarter of 2022.

Laura Clay: After adjusting for non-cash expenses and income tax, we reported a non-GAAP net loss of $51.6 million for the first quarter of 2022. On a gap basis, R&D expenses were $56.6 million for the first quarter of 2022. The increase compared to 2021 is largely attributable to the ongoing studies of sparsentine, as well as the advancement of the pig-to-batinase program in HCU. On an adjusted basis, R&D expenses were $53.2 million for the first quarter of 2022. Relevant non-cash expenses for the fourth quarter included $3.5 million of stock-based compensation and amortization. On a gap basis, selling, general, and administrative expenses for the first quarter of 2022 were $46.8 million.

On a GAAP basis, R&D expenses were $56 6 million for the first quarter of 2022.

The increase compared to 2021 is largely attributable to the ongoing studies of spar setting as well as advancement as it take the baton needs program in HCM.

On an adjusted basis R&D expenses were $53 2 million for the first quarter of 2022.

Relevant noncash expenses for the fourth quarter included $3 5 million of stock based compensation and amortization.

On a GAAP basis, selling general and administrative expenses for the first quarter of 2022 were $46 8 million.

The increase compared to 2021 is largely attributable to increased head count and commercial launch preparation.

On an adjusted basis SG&A expenses for the first quarter of 2022 were $35 million.

Significant noncash adjustments for the quarter consisted of $11 8 million in stock based compensation and depreciation and amortization.

And Opex perspective, we anticipate that our R&D expenses will continue to run at a rate slightly higher than in the first quarter.

This is representative of the ongoing duplex and protect studies that will continue into 2023 as well as the ongoing composed study is pegged to that nice and foundational work, including scaling CMC to prepare for a pivotal program.

Consistent with our planning from the beginning of the year, we expect increases in SG&A from the first quarter levels. As we continue the commercial build out including additions to our field team for the potential launch in Iga nephropathy.

Importantly, we continued to be in a strong financial position to execute in 2022 and beyond.

We ended the first quarter was $603 4 million in cash and cash equivalents.

This balance includes approximately 19 million in net proceeds from our at the market facility prior to our convertible transaction in the first quarter and approximately $95 million from the convertible.

Refinancing transactions affected in March.

Taking into account the potential impact of further competitive pressure on cellular.

Milestone payments, we expect to pay related to regulatory achievements for <unk>, but not yet factoring in the full pivotal program for peg. The bad news is that is still being planned out we anticipate this cash balance will support our planned operations into 2024.

Let me now hand, the call back over to Eric for his opening comments Eric.

Laura Clay: The increase compared to 2021 is largely attributable to increased headcount and commercial launch preparation. On an adjusted basis, SG&A expenses for the first quarter of 2022 were $35 million. Significant non-cash adjustments for the quarter consisted of $11.8 million in stock-based compensation and depreciation and amortization.

Thank you Laura.

Laura Clay: From an OPEX perspective, we anticipate that our R&D expenses will continue to run at a rate slightly higher than in the first quarter. This is representative of the ongoing duplex and protect studies that will continue into 2023, as well as the ongoing composed study of PEG-2-Batinase and foundational work, including scaling CMC, to prepare for a pivotal program. Consistent with our planning from the beginning of the year, we expect increases in SG&A from the first quarter level as we continue the commercial build out, including additions to our field team for the potential Sparsanton launch in IGA Nefrop. Importantly, we continue to be in a strong financial position to execute in 2022 and beyond. We ended the first quarter with $603.4 million in cash and cash equivalents.

Last month, we announced that after spending more than 30 years in the biopharmaceutical industry and more than seven years as CFO of trivia Laura has decided to retire so that she can spend much deserved time with family.

I would like to take this opportunity to thank Laura for her unwavering dedication to our mission and to her contributions in making <unk> into a leader in the rare disease community.

As a result of Lora leadership, we have developed an outstanding finance team and a strong financial foundation.

We're very pleased that Chris will be taking over the CFO role in August his capital markets experience deep institutional knowledge and strategic vision will be ideally suited for the role and we look forward to continuing to work closely with the investment community as we enter this new phase of growth.

Overall, I am very pleased with our execution to start the year. Most importantly, we have continued to progress towards our goal of making <unk>, a new treatment standard for rare kidney disorders if approved.

Laura Clay: This balance includes approximately $19 million in net proceeds from our at-the-market facility prior to our convertible transaction in the first quarter and approximately $95 million from the convertible refinancing transactions affected in March, taking into account the potential impact of further competitive pressure on Thiola. Milestone Payments We Expect to Pay Related to Regulatory Achievements for Sparsentine, but not yet factoring in the full Pivotal Program for PEG-2-Batinase as that is still being planned out. We anticipate this cash balance will support our planned operations into 2024. Let me now hand the call back over to Eric for his opening comments. Okay, Eric?

Eric Dube: Thank you, Laura. Last month, we announced that after spending more than 30 years in the biopharmaceutical industry and more than seven years as CFO of Travere, Laura has decided to retire so that she can spend much-deserved time with family. I would like to take this opportunity to thank Laura for her unwavering dedication to our mission and for her contributions in making Travere into a leader in the rare disease community. As a result of her leadership, we have developed an outstanding finance team and a strong financial foundation. We are very pleased that Chris will be taking over the CFO role in August.

Eric Dube: His capital markets experience, deep institutional knowledge, and strategic vision will be ideally suited to the role, and we look forward to him continuing to work closely with the investment community as we enter this new phase of growth. Overall, I am very pleased with our execution to start the year. Most importantly, we have continued to progress towards our goal of making Sparsantin a new treatment standard for rare kidney disorders, if approved. With the on-time submission of our NDA for IJ nephropathy, we have furthered our commercial preparations to be ready for a potential launch as early as the end of this year.

With the all time submission of our NDA for Iga nephropathy, we have furthered our commercial preparations to be ready for a potential launch as early as the end of this year.

And we remain on track for our planned FDA interactions regarding the Egfr data from duplex and the accelerated approval pathway in the <unk>.

Eric Dube: And we remain on track for our planned FDA interactions regarding the eGFR data from duplex and the accelerated approval pathway in FSGS. Together with the continued advancement of our PEG-to-Batinase program and the strengthening of our balance sheet during the quarter, we are well positioned to continue our execution throughout the balance of 2022. Chris?

Together with the continued advancement of our pegged at bad <unk> program and the strengthening of our balance sheet. During the quarter. We are well positioned to continue our execution throughout the balance of 2022, Let me now turn the call over to Chris for Q&A Kris.

Alright, Thanks, Eric Justin can we go ahead and please open up the lines for Q&A.

Chris Cline: Great. Thanks, Eric. Justin, can we go ahead and please open up the lines for Q&A? Absolutely. If you would like to signal with questions, please press star one on your touchtone telephone. If you're joining us today, use a speakerphone. Please make sure your mute function is turned off to allow you to reach our equipment.

Absolutely if you would like to signal with questions. Please press star one on your Touchtone telephone. If you are joining us today use a speaker phone. Please make sure. Your mute function is turned off to allow your signal to reach our equipment again that is star. One if you would like to signal with questions. We'll pause for just a moment.

Operator: Again, that is star 1 if you would like to signal with questions. We'll pause for questions. And our first question today comes from Joseph Schwartz with SVB. Hi all, this is Will on behalf of Joe, and thank you for taking our questions today, and congratulations on the recent progress. So one question for us, in terms of the overall IGANT market, there's obviously a relatively large addressable patient population here, but we're curious who the early adopters would be. Is it something for those with higher proteinuria, or are there other segments that are important for physicians and the company to target as you approach the potential launch in the fourth quarter?

Yeah.

And our first question today comes from Joseph Schwartz with SBB Securities.

Hi, all this is will on for Joe and thank you for taking our questions today and congrats on the recent progress. So one question for us in terms of the overall again market now there's obviously a relatively large addressable patient population here, but were curious who the early adopters would be of that.

<unk>.

Something like <unk>.

Is it something with those with higher common area or are there other segments that are important that physicians and their company to target as you approach the potential launch in the fourth quarter. Thank you.

Well thanks for that Great question I will.

Operator: Well, thanks for that great question. I will ask Peter and Jula to give their thoughts on where we might assume the early uptake force bar sentence. Peter, maybe you can start, and then Jula, maybe you can add your perspective.

As for Peter and jewelry to get their thoughts on where we might assume the early uptake for sports Center.

Peter maybe you can start and then Julian maybe you can add your perspective.

Yeah, absolutely well, we've spoken about how we see the Iga nephropathy markets I think the total market is about 130 to 150000.

Peter Heerma: Yeah, absolutely. Well, we've spoken about how we see the IJ nephropathy market; I think the total market is about 130 to 150,000. Ebenezer Smith, College of Medicine, Canvas Institute of Medicine, bless my youth, that have a confirmed IgA nephropathy diagnosis through a biopsy and that have consistent high proteinuria levels.

Patients the way we are thinking about like what is the addressable patient population at launch with <unk> syndrome.

You have indicated that's about 30% to 50000 those are the patients that are seen by the nephrologist.

Confirmed.

The Iga nephropathy diagnosis biopsy, that's consistent high potent urea levels. We've seen is the patient that I wouldn't read the Fas progression. So those are the patients that we will be focused on initially.

Peter Heerma: And those are seen as the patients that are on a rapid path of progression. So those are the patients that we will be focused on initially. Great, thank you. And if I could just sneak in one quick follow-up.

Great. Thank you and if I could just sneak in one quick follow up.

Jula Inrig: We recently saw another sponsor in this space report some interim data with another ET inhibitor, also an IG nephropathy. So just wondering kind of how you're thinking about the competitive landscape moving forward with potentially multiple players in this market? Sure. I'll ask Jula to give her perspective on that. And I think, you know, it's an important caveat that those are very early interim data. But, Jula, maybe you can pick up on there and where you believe Spar-Senten will be well positioned if approved.

We recently saw another sponsor in this space report some interim data with another E. T inhibitor also in Iga nephropathy. So just wondering kind of how youre thinking about the competitive landscape moving forward with potentially multiple players in this market.

Sure I'll ask Joe to give her perspective on that.

You know, it's an important caveat that those are very early.

From data with Julie maybe you can pick up on there where you believe.

<unk> will be well positioned.

If approved.

Yeah, there remains a significant unmet need for treatment of Iga nephropathy, and we believe that nephrologist will ultimately be seeking out new treatment options.

Jula Inrig: Yeah, there remains a significant unmet need for treatment of IJ nephropathy, and we believe that nephrologists will ultimately be seeking out new treatment options. And patients remain at very high risk with the current treatment options we have, which are ACE inhibitors or ARBs. And so we believe sparsantan therapy, with the significant reduction in proteinuria that we saw with PROTECT, with a 50% reduction in proteinuria, will become foundational therapy for the treatment of IJ nephropathy.

And patients are remain at very high risk with the current treatment options, we have which is ace inhibitors or Arab beef and so we believe for suntan therapy with the significant reduction in proteinuria that we saw with protect with a 50% reduction in proteinuria at first suntan will become foundational therapy, if it's approved.

And yes, there are a number of other therapies in development that we believe Fenton will be foundational therapy in some of the other niche therapies can potentially be additive to that.

Jula Inrig: And yes, there are a number of other therapies in development that we believe for Fenton will be foundational therapy, and some of the other niche therapies can potentially be additive to that. Excellent. Thank you again and congratulations on the progress. Thanks, Paul. And our next question: Carter Gould, which Barclay, Good afternoon and best of luck to Laura and congratulations on the role.

Excellent. Thank you again and congrats on the progress.

Thanks, Paul.

And our next question will come from Carter Gould with Barclays.

Good afternoon, and best of luck to Laura and congrats to Chris on the new role.

Peter Heerma: I guess the first question is around understanding that we're not going to see any of our data from Protect anytime soon. Are there plans to share or publish the data? As shown already, and maybe an additional secondary analysis. Bratisline character, Esther. For a potential launch, you know, it's been.

I guess the first question is around.

Understanding that we're not going to see any egfr data from protect anytime soon are there plans to share or publish the data.

But you've shown already and maybe additional secondary analyses or baseline characteristics for a potential launch.

And then I guess close to 260 days since we got the top line and we still haven't seen any other kind of data and then beyond sort of the Egfr data in <unk> are there any other data youll be providing to the FDA as part of the next go round in terms of conversation. Thank you.

Eric Dube: Close to 260 days since we got the top line, and we still haven't seen any other kind of data. And then, beyond sort of the EGFR data in FSTS, are there any other data you'll be providing to the FDA as part of that next go-round? Yeah, Carter, thanks for the questions. I think the answer to both of those is that we are going to prioritize managing the blind and the integrity of the study until it is completed in the first half of next year.

Yes, thanks for the questions I think the answer to both of those is that we are going to prioritize.

Eric Dube: And any additional data beyond the interim analysis on proteinuria, which was the primary endpoint at that point; however, we do not have any additional analyses beyond that update to EGFR. We wanna make sure that we are doing as minimal a look as possible based on what FDA would like to see. Anything beyond that may actually compromise the unblinding within the company or with sites and could compromise our ability to maintain alpha for the final analysis.

Managing the blind and the integrity of the study until it is completed.

The first half of next year and any additional data beyond the interim analysis on proteinuria, which was the primary endpoint at that point.

Could potentially increase the risk of.

Blinding trial integrity, and I think as we've discussed repeatedly in the past that's really an area of focus for FDA. So we're balancing that very carefully and making sure that we do everything possible to complete the study with with the with a robust dataset and as such we do not have plans to.

Published any additional data at this point and also for the interim analysis, all Egfr that.

We're meeting with FDA later this quarter.

We do not have any additional analyses beyond that update egfr.

Eric Dube: So at this point, we do not have plans for additional analysis or disclosure of additional data beyond what we have today. All right, thank you. Thank you. Thank you. Our next question will come from Greg Harrison.

We want to make sure that we are doing is minimal look as possible based on what FDA would like to see anything beyond that may actually compromise the unwinding within the company.

Or with the sites and.

Could compromise our ability to maintain alpha for the final analysis. So at this point, we do not have plans for additional analysis for disclosure of additional data beyond what we have to date.

Alright, thank you.

Thank you.

Thank you. Our next question will come from Greg Harrison with Bank of America.

Good afternoon. This is Mary Kate on for Greg. Thank you. So much for taking our question maybe just a question in regard to the potential launch in <unk>.

Peter Heerma: Good afternoon, this is Mary Kate on behalf of Greg. Thank you so much for taking our question. Maybe just a question in regard to the potential for ascent launch and IGAN. If approved, what are your expectations for the cadence on the initial uptake and ramp up of Sparse Sentinel-NIGAN, and maybe what are your expectations regarding reimbursement? Thank you. Mary-Kate, thanks for the questions.

It's approved what are your expectations for the cadence on the initial uptake and ramp up of sports Centre My Dad, and maybe what are your expectations regarding reimbursement. Thank you.

Okay. Thanks for the questions and as you can imagine that is <unk>.

Eric Dube: And as you can imagine, that is right, the area of focus for Peter and his team. But it is a bit early in terms of us talking about that. But our goal is to make sure that we have a very strong launch and that Sparsantan does become the new standard within IGAN and FSGS after that. And in order to do so, we need to have broad access within those populations. I'll ask Peter to talk a little bit more about the early expectations that his team is working on. Yeah, very good.

Right.

The area of focus for Peter and his team.

It is a bit early in terms of us talking about that but but our goal is to make sure that we have a very strong launch and that's far sentence does become the new standard within within again and F. S. G. S. After that.

And in order to do so we need to have broad access within those populations I'll ask Peter to talk a little bit more about early expectations as his team is working on those.

Yeah very good thank you Mary Kay so.

Peter Heerma: Thank you, Mary Kay. So... It's too early indeed to give specifics how we see the last uptake, but I think what we are seeing very consistently in our own research, as well as syndicated research, as well as actually external research that is being done, also among the investor community, we see a very consistent trend that physicians feel very excited about the opportunity, the mechanistic mode of action of Spar Centum, to claim that foundational therapy, because to Jula's earlier point, like a lot of those patients remain not treated to the targets that physicians would like to see.

It's too early in these to give specifics how we see the launch uptake, but I think what we are seeing very consistently in our own research as well as our syndicated research as well as external research that is being done.

The Investor community, we see a very consistent trends that physicians feel very excited about the opportunity the mechanistic motivational spark syndrome.

To claim that foundational therapy goes to you last really a point like a lot of those patients remain not 3% to the targets that position, we'd like to see.

Peter Heerma: So with stage ARBs often failing, we think that that's really the position that Spar Centum can claim. Also, think about things like the new Cadego guidelines that were presented last fall. With regard to your other question about access, we are so far very pleased with the interactions we've had with payers and the response we're getting from payers as well. They see the unmet need, they recognize the unmet need, and there is also a high interest with regard to new modalities and innovative treatments. So I think we're making good progress.

Arps, often failing we think that that's really the position of Spartan I think and also thinking about like the new <unk> guidelines that was presented last law suit.

With regards to your other question was excess we so far and are very pleased with the interactions. We've had these figures and the response, we're getting from payers as well they see the unmet need to recognize the unmet need and there is also a high interest.

With regards to review modalities and innovative suite and so I think we're making good progress with <unk>. We are building very solid value proposition so more to come but so far I think we are very pleased with the feedback we're getting from physicians as well as from the payer community.

Peter Heerma: I think we are building a very solid value proposition, so more to come. But so far, I think we are very pleased both with the feedback we're getting from physicians as well as from the payer community. Yeah, I think that's right, Peter. Thank you for that.

I think that's right Peter Thank you for that and there the only thing that I would I would add to what Peter mentioned is that we certainly recognize that nephrologists are conservative by nature and very much focused on understanding the clinical evidence and the mechanism of the treatment and that very much is part of how we're planning.

Eric Dube: And Mary Kate, the only thing that I would add to what Peter mentioned is that we certainly recognize that nephrologists are conservative by nature and very much focused on understanding the clinical evidence and the mechanism of a treatment. And that very much is part of how we're planning to prepare to give them the information and the tools to help make the best decision for these patients once approved. Great, thank you.

Turning to prepare to give them the information and the tools to help make the best decision.

For these patients once approved.

Great. Thank you.

Thank you.

And our next question comes from Lisa <unk> with Evercore ISI.

Eric Dube: And our next question comes from Liisa Bayko with Evercore IS. First, congratulations on the progress, and my question is, how will the EGFR data and FSDS and subsequent FDA decisions affect the MAA submission in Europe? Jimmy, thanks for that question.

So first congrats on the progress and my question is how well the Egfr data F. S. Geos and subsequent hefty a profession of fact, the MAA submission in Europe .

Jamie Thanks for that question. So I think what what our focus is to make sure that we understand.

Eric Dube: So I think our focus is to make sure that we understand the position of the FDA and what our timelines would be for an FSGS submission before we, you know, change any of the plans that we have in place. Nothing has changed in our plans to date, and we are on track to provide a combined MAA in the middle of this year. The statement that I mentioned around, you know, it's going to be after, you know, there may be information that FDA provides that, you know, helps us to think through things before anything that may affect us. But at this point, we don't see any of that.

The position.

The FDA and what our timelines would be for.

And F S GFS submission beef.

Before we change any of the plans that we have in place nothing has changed in our plans to date and we are on track to provide combined MAA in the middle of this year. The statement that I mentioned around you know it's going to be after as you know there may be information that FBA provides that helps us to think through it.

Eric Dube: We've not yet met with FDA, and our plans have not changed in our timeline for MAA. Thank you. And if I can add one more question, can you give some color on your collaboration with Pharma Corispo? Like, what is your rationale and how does this fit into your portfolio?

With me for anything that.

That may effect, but at this point, we don't see any of that we have not yet met with FDA and our plans have not changed in our timeline for MAA.

Thank you and if I can ask one more question on can you give some color on your collaboration with from a crystal like what is your rationale and how does that fit into your portfolio.

Sure well, we're very pleased about this opportunity it is early.

Eric Dube: Sure. Well, we're really pleased about this opportunity. It is early, and it is an asset or program that we believe is well-suited within our rare nephrology expertise. This is an asset that, or, you know, potential asset that, you know, is pre-IND. And so, you know, we're not discussing much at this point, but it is something that, you know, over the next couple of years, we do see that, you know, we have the potential for an IND and are continuing to progress with development.

And it is a and asset or program that we believe is well suited with in our rare nephrology expertise. This is an asset that or potential asset that is.

He is pre IMD and so we're not we're not discussing much at this point, but it is something that over the next couple of years, we do see that we have the potential for an IMD and continuing to progress.

Four four development, we'll certainly share more information when we get to that point and we're excited because it's another opportunity for us to support the rare nephrology community.

Eric Dube: We'll certainly share more information when we get to that point, and we're excited because it's another opportunity for us to support the rare nephrology community. Thank you. Thank you, and our next question. Maurice Raycroft, Lift Jets. Hi, thanks for taking my questions and congratulations on the progress.

Okay. Thank you.

Thank you.

And our next question come from Maury Raycroft with Jefferies.

Hi, Thanks for taking my questions and congrats on the progress.

You disclosed some.

Eric Dube: You disclosed some patient baseline characteristics from the IGAM-Protect study in the ERA Conference Abstract. Just wondering if you can help contextualize what's disclosed in the abstract and maybe talk about what FDA is going to be focused on with comparing and contrasting your drug with Cletatosis tarpeo to determine what the label should ultimately look like. Sure, I'll ask Jula to take the first question, which is really providing context for the baseline data, and then I'll ask Bill to share what FDA may be looking for. Okay, so we are excited to share our baseline characteristics at ERA from both Duplex and PROTECT. Independently, they're both the largest interventional trials testing a novel therapy in IGAN and FSGS.

Patient baseline characteristics from the protect study in that era conference abstract just wondering if you can help contextualize, what's disclosed in the abstract and maybe talk about what FDA is going to be focused on with.

With comparing and contrasting your drug with <unk> to determine what the label should would ultimately look like.

Sure I'll ask Julie to take the first question, which is really providing context for the baseline data and then I'll ask bill to share what FDA may be looking for.

Sure. So we are excited to share our baseline characteristics at here from both duplex and protect independently.

The largest interventional trials testing, a novel therapy, and ICANN and F S. Yet.

And what we think is really important is that both studies are represented at the planned patient populations and so we are excited to share. This data with the nephrology community and each of the populations are unique and so you can see that represented when you look through the abstract and that we cover those patient populations representative Lee.

Jula Inrig: And what we think is really important is that both studies are representative of the planned patient populations. And so we are excited to share this data with the nephrology community, and each of the populations is unique. And so you can see that when you look through the abstracts and that we cover those patient populations. I'll take the second question.

I'll take the second question. Thanks for the question Maury I think the heart of it is compare and contrast sports center and versus Budesonide, and I think the <unk> and.

William Rote: Thanks for the question, Maury. I think the heart of it is, you know, to compare and contrast Sparsenthan versus Budefinite. And I think the potential impacts on that eventual labeling course are of speculation at this point. But if you look at benefit risk, which is where the reviewers will start with us on any of the steroid class. The risk profile is very different than what you see with sparsentine, where we have a high degree of tolerability such that it's a long-term therapy that other therapies would be added to. So I would, I hope that there would be a broader label than what was provided to Tarpaio.

And the potential impacts on that eventual labeling of course, it's a speculation at this point, but if you look at benefit risk, which is where the reviewers will start.

With us any of the steroid class the risk profile is very different than what you'd see with Spartan <unk>.

Where we have a high degree of Tolerability such that it's a long term therapy that other therapies would be added upon so I would.

I hope that there would be a broader label than what was provided the chart Vale certainly most of my experience with the agency has been based around the premise of you get what you study.

William Rote: Certainly, most of my experience with the agency has been based around the premise that you get what you study. So, we'll need to wait and see based on the review, but again, it's really going to come down to benefit-risk, and I'm very confident in the position that Sparstentin has in that. That's helpful. And do you think there will be some restrictions on proteinuria based on baseline characteristics, or is that tough to say at this point?

So we.

We will need to wait and see.

Based on the review, but again, its really going to come down to benefit risk and I'm very confident in the position that as far as <unk> has in that arena.

Got it that's helpful. Do you think there will be some restrictions on proteinuria based on baseline characteristics or tough to say at this point.

It's very difficult to say at this point.

William Rote: It's very difficult to say at this point. Okay, okay, maybe last quick question, just since Sparse Sentin is a first in class dual ARB and ERA blocker, can you talk about chances of getting an ADCOM and remind me what feedback you've gotten on potential for an ADCOM so far? Sure, Phil. Would you like to take that? Certainly. We don't expect to have an advisory committee.

Okay, Okay, and maybe last quick question just.

Since as far as <unk> is a first in class dual arb and DRA blocker can you talk about chances of getting an AD com and remind what feedback you've gotten on potential for an AD com so far.

Okay.

Sure Bill would you like to take.

But certainly.

We don't expect to have an advisory committee. We asked the question during our pre NDA meetings, and we ask them directly their answer was no with what we know now we don't anticipate having an advisory committee.

William Rote: We asked the question during our pre-NDA meetings, and we asked them directly. Their answer was no. With what we know now, we don't anticipate having an advisory committee.

William Rote: That could certainly change, and if it does, we'd be prepared for that. But it's a very clear picture, strong data. It's not the type of package where the agency would normally request an adcom.

That could certainly change and if it does we'd be prepared for that.

But it's a very clear picture.

Strong data, it's not the type of package, where the agency would normally.

William Rote: So we're confident in their first answer that no, they don't intend to have one. Got it. Okay. Thanks for taking my questions. And moving on to Tim Lugo with William...

Our request an AD com. So we're confident in their in their first answer that no. They don't intend to have one.

Got it okay. Thanks for taking my questions.

Thanks Mark.

And moving on to Tim Lugo with William Blair.

Thanks for the question and congratulations to Laura on the career.

Eric Dube: Thanks for the question and congratulations to Laura on her career as well as to Chris on the new role. Briefly, I guess, some questions on Peg the Bat Maze. Can you talk about these global supply chain issues? Are they impacting... The next, you know, the end of phase two meeting, or do you need to complete the fifth cohort before that? Can you also remind us how the formulation of the six cohorts is different from the formulation used in the other five?

Well as Chris on the new role.

Briefly I guess some questions on pegged at bad <unk> can you talk about the global supply chain issues are they impacting.

Yeah.

End of phase two meeting or do you need to complete the fifth cohort before.

Yeah.

And can you also remind us how the formulation with fixed cohort as well.

Difference in the formulation used.

Other five.

Yes.

Sure, Tim I will hand over to bill.

Eric Dube: Sure, Tim, I will hand those over to Bill. Thanks, Tim. With the challenges that we're seeing in the manufacturing space, I think they're not so much specific to pegtobatinase, but I think that they're endemic within all of biologics manufacture. You have a combination of a flexing of the supply chain to manage a lot of COVID vaccine Manufacturer around the world, and that was inserted into an already full contract manufacturing space. So that did two things. It took up manufacturing slots and made them more challenging to obtain, and it consumed a lot of raw materials and consumables.

Certainly thanks, Tim with the challenges that we're seeing in the manufacturing space I think they're they're not so much specific to pegged to bad news, but I think that they're endemic within all of biologics manufacturer you have a combination of a flexing of the supply chain to manage.

A lot of Covid vaccine.

Manufacture around the world and that was inserted into an already full.

Contract manufacturing space, So that did did two things.

Took up manufacturing slots and made them more challenging to obtain and it consumed a lot of raw materials and consumables. So those have been the areas, where we've had challenges we're happy with the progress that we've made working through those we continue to make progress on the manufacturing side.

William Rote: So those have been the areas where we've had challenges, and we're happy with the progress that we've made working through those. We continue to make progress on the manufacturing side, but it's important for us to, you know, let stakeholders know that these are challenges that we've been navigating. So the second half of your question was the formulation difference for Cohort 6. This is the first cohort that will be using a lyophilized formulation. Recall that Cohorts 1 through 5 used a liquid formulation that, for stability reasons, was frozen at very low temperatures.

But it's important for us to.

Let's stakeholders know that it's that these are challenges that we've been navigating so on the second half of your question was the formulation difference per cohort six.

This is the first cohorts it'll be using a lyophilize formulation recalled that cohorts one through five used a liquid formulation that for stability reasons was frozen at very low temperatures so by going to the lyophilize formulation. It makes the overall.

William Rote: So by going to the lyophilized formulation, it makes the overall handling of the material shipments and the supply of clinical trial materials much easier, both for us as the sponsor and our collaborators but also for the patients in the study. Thanks, Bill. I think the only thing that I would add, Tim, just on that question of supply challenges is that these are not new. These are ones that we've shared in the past.

Handling and the material shipments and the supply of clinical trial material.

Much easier both on us as the sponsor and our and our collaborators but also on the patients in the study sites.

Thanks Bill.

Things that I would add Tim just on that question of supply challenges and these are not new.

These are these are ones that we've shared in the past and I think you know.

Eric Dube: And I think, you know, it's one that Bill's team is making great progress on, and we don't believe it has any bearing yet on the timing of our interactions with FDA. We've made good progress with those interactions, and we expect that they will continue to be. And again, Bill and his team are working through all of those challenges as well. Okay, and maybe I could ask a broader question, kind of giving your current cash position, and what we're seeing is, Unknown Speaker, the wide overall weakness in the market, which everyone's aware of. Does that change your thinking around putting in additional assets like that? It seems to be a good transaction. I'm sure there are some other assets out there.

It's one that Bill's team is making great progress on and we don't believe has any bearing yet on that.

The timing of our.

Interactions with FDA, we've made good progress with those interactions we expect that they would continue to be and again bill and his team are working through all of those challenges at good pace.

Okay, and maybe I can ask a broader question kind of given your current cash position and what we're seeing.

Kind of a wide overall weakness in the market, which everyone is aware of.

Does that change your thinking around bringing in additional assets it seems to be.

Good transaction I'm sure there are some other assets out there.

Yes, it's a great question Tim.

Eric Dube: Yeah, it's a great question, Tim. I'll start and then, you know, certainly I'll ask Laura to provide anything further. We continue to look at opportunities that are out there. You know, certainly there is a lot of weakness within the marketplace.

I'll start and then certainly I'll ask Laura to provide anything further and we.

Continue to look at opportunities that are out there.

Certainly there is a lot of weakness within the marketplace. We know that there are companies that may have either the lack of expertise and capability or cash to move their assets in rare disease forward.

Eric Dube: We know that there are companies that may have either the lack of expertise and capability or cash to move their assets in rare diseases forward. That said, we also want to be very mindful about our use of cash and be very sensitive to any potential dilution, particularly before, you know, we get Sparcentin out there and, you know, have a successful launch there. So, our number one goal remains the successful launch of Sparcentin.

That said, we also want to be very mindful about our use of cash and be very sensitive to any potential dilution, particularly before we.

Guitar center out there.

Have a successful.

Launched there. So we are number one goal remains the successful launch of <unk>, we're doing all we need to to invest in that but it's not going to slow down our diligence and monitoring of that landscape. Lord is there anything else that you'd want to add.

Eric Dube: We're doing all we need to to invest in that, but it's not going to slow down our diligence and monitoring of that landscape. Laura, is there anything else that you want to add? No, I think you've covered it well, Eric.

No I think you've covered it well Eric.

Yes.

Really our position on cash as we mentioned on the call.

Laura Clay: Really, our position on cash, as we've mentioned on the call, is we do believe our cash will be in 2024. Of course, to your point, that really isn't contemplating any additional BD work, but to Eric's point....... You seem to be a little more undervalued these days, so you never know, there could be a good fit out there, but nothing planned right at this point.

We do believe arc.

The 'twenty 'twenty four of course to your point that really.

Any additional BD works that took place.

And.

You seem to be a little more undervalued. These days. So you never know there could be a good fit out there.

But nothing.

Right at this point, Yeah, I think the only other thing I would.

Eric Dube: Yeah, I think the only other thing I would add is Pharmacristo is a good example where, with very little cash, and little investment, this could be an important area within the rare renal space. Again, it's early, but it's a reflection of what we're going to continue, but we're going to be very responsible and disciplined in the use of our cash and in any business development activities. Great, thank you, Thanks, Tim. And our next question will come from Michelle Gilson with Canoncord Genewe. Hello, this is Michael. I'm from Michelle.

That is a farmer christo.

Ah is a good example, where with.

Very little cash little investment this could be an important area within the rare renal space again, it's early but it's a reflection of we're going to continue but we're gonna be very responsible and disciplined in the use of our cash in any business development activities.

Great. Thank you.

Thanks, Tim.

And our next question will come from Michelle Gilson with Canaccord Genuity.

Hello. This is Michael on for Michelle Congrats on the quarter, just a couple more questions on types of acne.

Eric Dube: Congratulations on the quarter. Just a couple more questions on pegtybatenase. So, moving forward with pegtybatenase, how has your engagement with regulators been regarding moving forward based on a biomarker versus perhaps clinical outcomes? And what would you need to show on any secondary functional endpoints?

William Rote: Thank you, Michael, for the question. Bill, maybe you can take the question on how that's going, the engagement with regulators and Biomarker. And maybe, Jule, if there's anything else you want to add on secondary endpoints that you're hearing from the specialist community. Certainly. Thanks for the questions, Michael.

Jula Inrig: The path forward is going well. We are in what is essentially an iterative dialogue with the agency around various aspects of the program, the CMC endpoint. Thank you.

William Rote: ... and when we get to a point where we've completed enough of that, we'll update you on the overall package, including your specific question around biomarker versus clinical endpoint. This is an ideal situation for the use of a biomarker as a clinical endpoint, and we're working with the agency and following their guidance on how to qualify total homocysteine as the appropriate endpoint for that. And those efforts are underway. I think if you wanted to use a clinical endpoint in this disease space, it would be very challenging because the timescale on which these changes take place is longer than what traditional pivotal clinical trials are. There's just a mismatch in the duration of what would be required.

Alright use of a biomarker as a clinical and point and we're working with the agency.

And following their guidance on how to qualify total homeless sistine as the appropriate and point for that and and those efforts are under way I think if you wanted to use a clinical and point in this disease space. It would be very challenging because of the time scale on which these.

Changes take place are longer than what traditional pivotal clinical trials are there's just a mismatch in the in the duration of what would be required. So it really is an ideal setting and there is precedent all early Ah from the nineties, but there is precedent of a drug that was.

William Rote: So it really is an ideal setting, and there is early precedent from the 90s, but there is precedent for a drug that was approved using total homocysteine as a biomarker. So we're optimistic that we'll be able to achieve that once we've completed those interactions. Jula, do you want to take the question around? Clinical Secondary Endpoint. Yeah. This disease, as Bill mentioned, is very self-destructive.

Approved using total homeless <unk> as a biomarker so we're optimistic.

We'll be able to achieve that once we've completed those interactions.

Two or do you want to take the the question around clinic.

Clinical secondary endpoints.

[noise] [laughter] they'll mention that diseases, very slowly progressive and some of those secondary endpoint lengthy collecting as patients like be in our studies, but they develop over many years and.

Jula Inrig: As Bill mentioned, the disease is very slowly progressive, and some of those secondary endpoints we would be collecting as patients would be in our study, but they develop over many years, so those clinical endpoints, such as ocular effects or thrombotic events, would be collected over many, many years and not as part of homocysteine direct lowering over a short term. And of course, for the patients, from their perspective, eventually, their And so that would be the ultimate goal over the long term, and we'd hope to be able to capture some of that information eventually. Excellent Thank you. And just one quick one more on the type of atonement. How does the IP look?

Time said, that's clinical endpoint search ads ocular effects or thrombotic events would be collected over many many years and not as part of almost 15 direct lowering over a short term.

And of course for the patience from their perspective eventually their goal would be able to liberalize their diet and so that would be the ultimate goal over the longterm and we'd help to be able to capture some of that information eventually over time.

[noise] excellent. Thank you and just one quick one more on check with that name.

William Rote: How long do you anticipate an exclusivity period? And thank you. Yeah, so we do have a patent family for pegtobatinase. But I think that the simplest approach and the most robust would be for the exclusivity period granted for a biological orphan disease product. So you can assume 10 plus years for that. And then, you know, 12 years in Europe.

How was the how did the I T look for how long do you intend to pay an exclusivity period and thank you [noise].

Yeah. So we do have a patent family for <unk>, but I think that the simplest approach and most robust would be for the exclusivity period granted for a biologic orphan disease products. So you can assume 10 plus years for for that.

William Rote: Excellent, thank you. Thank you. And our next question? Laura Chico.

And then you know 12 years in Europe .

Excellent. Thank you.

Thank you.

And our next question will come from Laura Chico with Wedbush Securities.

Laura Chico: Wedbush Security, Good evening. Thank you very much for taking the questions. I just have two.

Good evening. Thank you very much for taking the question I I just have two so first and F. S. G. S. One of your competitors announced they'll pursue a single pivotal study any genetically defined F. S. T. S population, but the implants, they're focusing on R. E. D. F R slope and change your partner Yeah. So I'm, just wondering kind of with a broad lines could you.

Eric Dube: So first, in FSGS, one of your competitors announced they'll pursue a single pivotal study in a genetically defined FSGS population, but the endpoints they're focusing on are EGFR slope and change in protein area. So I'm just wondering, kind of with a broad lens, could you comment on the robustness of FPRE as a surrogate measure and maybe how you see the FSGS regulatory landscape And then my follow-up question is actually for Jula or Eric.

Comments on the robustness of F theory, as a surrogate measure and maybe how you see the F. S T as regulatory landscape changing changing over time and then my follow up question is actually perhaps for July Eric you made the comment earlier about the about the first set and serving as a foundational component versus other M O S.

Eric Dube: You made the comment earlier about Sparsentin serving as a foundational component versus other MOAs in development for IGAN, and I'm curious if you could perhaps elaborate a little bit more in terms of what specific features make Sparsentin well-suited to be a foundational treatment.

And development for again.

I'm curious if you could perhaps elaborate a little bit more in terms of what specific features <unk> well suited to be a foundational treatment. Thank you.

Certainly I'll take that first one F T. <unk> <unk> do you want to add anything further because for work before joining the company certainly involve a lot of pay foundational work hiding.

Jula Inrig: Thank you. Certainly, I'll take that first on FPRE. I'll ask Jula to add anything further because her work before joining the company certainly involved a lot of the foundational work tying robust measures of proteinuria to long-term renal outcomes, but that really is the foundational element of FPRE. It is a robust measure of proteinuria reduction and is using some of the largest registries of FSGS patients correlated with longer-term renal survival and EGFR.

Robust measures of proteinuria to longterm regional outcomes.

But that that really is the foundational element of F. P. Sorry. It is a robust measure croton, you're ready reduction and is in some using some of the largest registries of F. S. G S patience correlated with longer term rental survival.

M. P. GFR. So that was a strong component of why we chose F theory and are confident in that being a a high bar for four criteria function.

Jula Inrig: And so that was a strong component of why we chose FPRE and are confident in that being a high bar for proteinuria function. There is another program in clinical development. It is in a subset of FSGS patients, but I think fundamentally, it's the same focus of measuring proteinuria, which is aligned with how nephrologists think about treating these patients, as well as EGFR over the long term, which we know is very important to regulators.

There is another program in clinical development. It is a subset of F. S. G F patience, but I think fundamentally it's the same focus measuring COVID-19 urea, which is a line with how Nephrologist think about you know treating these patients as well as egfr over the longterm, which we know is very important to regulators.

Uhm Julia anything else that you want to add on F theory, and then also the kind of profile of <unk>, making it a foundational therapy.

Eric Dube: Jula, anything else that you want to add on FPRE and then also the kind of profile of Sparcentin making it a foundational therapy? Yeah, FPRE is more aligned with how we think of FSGS, which is a disease in which you're looking at remission. It is a measure of propranuria and patients getting to a certain threshold and a magnitude of reduction.

Yeah F. Perry is more aligned with how we think of F. S. Yes, which is a disease in which you're looking at remission.

You can and and it is a measure of Maria and patience getting to a certain threshold and a magnitude of reduction <unk>. So it captured that and it's Eric said it is the measure which has the strongest correlation with protecting egfr and preserving kidney function and delaying time to dialysis or transplantation. So.

Jula Inrig: So it captures that, and as Eric said, it is the measure which has the strongest correlation with protecting EGFR and preserving kidney function and delaying time to dialysis or transplantation. So it has the greatest correlation there. However, it's ultimately a measure of reduction of propranuria. So it is consistent with the other trial. So we just have the best data for that. Let me answer then a little bit further about why we believe Barsentan can be foundational therapy. So, sparsantan itself blocks endothelin and angiotensin receptors in a single molecule.

It has the greatest correlation there however, it's ultimately a major reduction area. So it is consistent with the other trial and so we just have the greatest data for that appointment.

Let me answer that a little bit further about why we believe are sent and can be foundational therapy. So smart fan-tan itself blocks endothelin, an anti attention receptor in a single molecule and so the benefit of that if you reduce proteinuria.

Jula Inrig: And so, the benefit of that is Proteinuria. And you are getting a combined mechanism in a single molecule. And so you get the safety profile with that in addition to the magnitude of the proteinuria reduction. And so, essentially, you can switch patients from their therapy of an angiotensin receptor blocker or an ACE inhibitor over to a safe and effective therapy with a significant reduction in proteinuria, which we know we've demonstrated is beneficial in reducing proteinuria. And so that's why we believe it can be foundational therapy. So far, it's safe, and it's effective. And Peter, anything you'd want to add? Yeah, no, I think, I think it's a great, great explanation.

And you are getting a combined mechanism and the single molecule and so you'll get the safety profile with that in addition to the magnitude of the <unk> reduction.

And so essentially you can switch patients from their therapy as in angiotensin receptor blocker earn ace inhibitor over to a safe and effective therapy.

With a significant reduction in <unk>, which we know we've demonstrated it's beneficial in reducing proteinuria and so that's why we believe that can be foundational therapy, so far it's safe and it's effective.

And Peter <unk> you'd want to add.

Yeah, No I think <unk> I think it's a great a great explanation. We also hear from all of this issue is that they are very comfortable with the mechanistic approach allows itself.

Peter Heerma: What we also hear from our physicians is that they are very comfortable with the mechanistic approach because it allows itself for add-on therapies if needed. And I think that's what gives confidence to the physicians we're speaking with, that they see this as frontline treatments early in the treatment paradigm, allowing for additional modalities if needed. Thank you very much and congratulations to Chris and Laura both on the transition.

And I think that this will just go from these two of decision to be speaking with that they see it as like some blind treatments clearly in the in the treatment paradigm, allowing for additional modality is needed to add.

Thank you very much and congrats to Chris and Laura built on the transition.

Thank you Laura.

Laura Chico: Thank you, Laura. Thank you, with Piper. Hi, this is Skylar on behalf of Joe Kim.

Thank you.

Moving on to do Kim with Piper Sandler.

Hi, This is skylar on for <unk>. Thank you for taking my question I was wondering if you get approval in both I again and S. Yes, how are you.

Unknown Attendee: Thank you for taking my question. If you get approval for both IGAN and FSGS, how are you thinking about pricing when you consider the different dosing levels for the indications where FSGS could be two times the dose of IGAN? I'm curious if you could provide any color around your thoughts around that.

Thinking about pricing when you consider the different dosing levels vindication for at this jazz can be two times the dose of I again, I'm curious if you could provide any color around your thoughts are on that thank you.

Sure. Thank you Scott for the question. So I I'd say largely its it's early for us to talk specifics around pricing Uhm that said, there's a lot of work that we're doing to assess the burden of illness and health economic aspects of <unk> and our overall strategy for the pricing.

Eric Dube: Thank you. Sure. Thank you, Scholar, for the question. So I'd say largely it's early for us to talk specifics around pricing. That said, there's a lot of work that we're doing to assess the burden of illness and the health economic aspects of Sparcentin, and our overall strategy for the pricing and reimbursement approach for Sparcentin is to ensure broad access within the labeled populations to make sure that patients are able to benefit from it.

And reimbursement approaches <unk> is to ensure broad access within the label population to make sure that that's patients are able to benefit from sports and sports center, but ultimately that's the way that we will be able to achieve our vision of sparse Simpson being the foundational therapy Oh.

Peter Heerma: But ultimately, that's the way that we will be able to achieve our vision of Sparcentin being the foundational therapy. I'll ask Peter to talk a little bit more about how his team is thinking about the potential price difference given the difference in dosage. I think it's well said, Eric. I think you covered the majority there. I think it's different disease states and also is generally seen as a more progressive disease.

Peters would talk a little bit more about how his team was thinking about the potential price difference given the difference in dosing.

I don't think it will set here we've got you covered.

Peter Heerma: Your point is well taken. There's a dose difference that you have to take into account from a pricing perspective, but there's also the burden of disease, to Eric's point, is different as well. And all those aspects will be taken into consideration before we make a pricing decision. Our initial launch will be in IJN, and I think we have a competitor there already, so I think that allows you some flexibility, but more to come on that, based on the data that we're gathering right now.

Yeah, I think it's it's different disease.

States and also <unk> is generally seen as a more progressive disease.

Your point is well taken days of those differences do I have to take into account from a pricing perspective, but there's also like to do is <unk> is different as well and.

Those <unk>.

Take into consideration before we make a surprising this issue.

Our initial logs.

There'll be an I N and I think you have to compare to that already so I think that allows you some flexibility with more to come on that's based on the data gathering my account.

Makes sense. Thank you.

Okay got it.

And our next question will come from Ed.

Unknown Attendee: Thank you. And our next question will come from Ed. H.C. Wainwright said, Hi, thanks for taking my questions. Let me add my congratulations to both Laura and Chris.

With H C Wainwright and company.

Hi, Thanks for taking my question.

Let me add my congrats to both Laura and Chris a couple of questions for me Firstly.

Unknown Attendee: A couple of questions for me. Firstly, on pricing. I know you just mentioned this, but I wanted to take a slightly different approach. As you think about the decision process, and of course, you still have to see the label ultimately, but I wanted to get your take on how you view pricing and that decision in the context of the value proposition. In general, but specifically in the context of being a non-immunosuppressant agent.

On pricing I know you just mentioned this but I wanted to take a slightly different approach as you think about the the decision process and of course, you still have to see the label ultimately, but wanted to get your take on.

Would you view pricing and that decision in the context of the value proposition.

In general, but specifically, we context of being a non <unk> immuno suppressant agent and.

And then I have a follow up.

Okay, great. Thank you answer the question Peter would you like to pick up.

Peter Heerma: And then I have a follow-up question. Okay, great. Thank you, Ed, for the question. Peter, would you like to take that?

Yeah.

Peter Heerma: Yeah, I think the last aspect of your question is the important part, right? And I think that's where we see the most excitement from the prescriber community because this allows them to more effectively treat those patients. And again, referring to the interim data that we had shown, 50% versus 15% is a substantial increase in efficacy, and it allows physicians to use Spar-Centered without going down the steroid path. And the reluctance to steroids is quite high, both in patients as well as with physicians.

The the last aspect of your question I think is D is the important part I didn't need nothing that's where we see most exciting from the prescriber W. Z.

Because this allows them to more effectively treat those patients and again recording to the interim data that we had children 50 per cent service 15 per cent.

A substantial increase in the system.

<unk>, Okay <unk>.

The lowest physicians to use <unk> without going to see it with that and and do lessons in steroids is quite high both of <unk> as well as with physicians and I think that's why physicians are so excited about this this modem action.

Peter Heerma: And I think that's why physicians are so excited about this mode of action. Also, in the light of the new clinical guidelines that I was mentioning earlier, where you see a higher reluctance and caution in regards to steroid use. So, to Eric's earlier point, we want a price for broad access because we think this project has the potential to become a new standard of care. The value proposition that we are building, and you have to take into consideration that kidney disease patients, in particular those patients who have progressed to dialysis, are amongst the most expensive patients for payers and society. And so, I think we have, especially for the rapid-progressing patients, we have a strong burden of disease that we can build. Right. Great. Thank you for that. And then.

So in the light.

Right. When you said that was mentioning earlier when you see a higher reluctance being cautious with regards to to to <unk>. So <unk>, we want a price for broad access because we think this book tour, which has the potential to be <unk>.

The value proposition that we are building and you have to take into consideration that.

Kidney disease patients in particular does patient have progressed to dialysis.

The most expensive patience for for so pay your since society and so I think we have especially for the rapid progress in patients we have.

The cities that we can build one.

Alright, great. Thank you for that and then.

Thinking through.

Eric Dube: Speaking through it. You know, the expense structure for the rest of the year and your expectations there. I wanted to get your take on the commercial readiness activities that are ongoing, not only now and through presumably November when you have a PDUFA day, but also what you may be leaving pending approval. Yeah, great question, Ed.

The the expense structure for the rest of the year and your your expectations there.

I wanted to get.

Excuse me.

Sure.

Your take on a commercial readiness activities that are ongoing not only now and and through presumably November when you have to do today, but also what you may be leaving pending approval.

Yeah, Great question and I think first let me, let me say that our approach is to plan for success in our large overall and to be ready for day, one of approval and that's largely what is driving a lot of the work that Peter and Jewel is T.

Eric Dube: I think first, let me say that our approach is to plan for success in our launch overall and to be ready for day one of approval. And that's largely what is driving a lot of the work that Peter and Jula's team are doing in launch readiness. So there will be, as Laura mentioned, a continued increase in our SG&A throughout the year, not just in the expansion of our commercial infrastructure but also in investing for that launch from day one.

We're doing a launch readiness. So there will be as Laura mentioned in a continued increase that R. S. G&A throughout the year not just in the expansion of our commercial infrastructure, but also in investing for that launched from day. One we recognize that that may be different from other companies, where you know you you.

Eric Dube: We recognize that that may be different from other companies where, you know, you wait until you've de-risked a lot of that. We are planning for success. And I think, you know, we'll continue to provide updates on what that looks like and how we're preparing for launch later this year. Peter or Laura, anything else that you want to add?

Wait until kind of Derisked a lot of that we are planning for success and I think we'll continue to provide updates on what that looks like and how we're preparing for lunch later this year, Peter or anything else that you wanted to add.

No I think you've covered as well Earache I think.

Peter Heerma: I think you covered it well, Eric. I think we're building upon an organization that is existing and that has shown year over year that they are successful in identifying and treating patients. I think to the point that you're making, like, how does that expense look? I think we are really focused on three aspects. One is educating the market, in particular, making sure that physicians are comfortable with that new mechanistic approach of dual angiotensin and endothelin antagonism. Two, to make sure that we educate the payers, and they understand the burden of disease and hygiene from the NFSDS. So that the hourly value proposition is well perceived.

<unk> a as an organization that is existing <unk> <unk> <unk>.

Successful in identifying and treating patients.

I think due to the point that you're making like how does that extends look like I think we are really folks since <unk> educating democracy particular, making sure that physicians are comfortable with a new mechanistic approach <unk>.

Peter Heerma: And the third one is increasing the awareness of sparsity and also the intention to treat. And also in those parameters, we see nice progress, both in our own research as well as external. Fantastic. Thanks for taking the questions. That does conclude the question and answer session. I will now turn the conference back on. Additional Thank you, Justin. And thank you all for joining us today. This concludes our first quarter update. We look forward to keeping you updated throughout the year and speaking with you again soon. Have a great rest of your evening. And that does conclude today's conference. We do thank you for your participation.

And get this in in in the ceiling and thank goodness.

To to make sure that we educate the <unk> understands the bismol disease and I enjoy the phone B M. S. S. Yes. So <unk> proposition is will received.

Third one is increasing the awareness of sparse and also the intention to treat.

<unk>, a nice rug rats.

<unk>.

Fantastic. Thanks for thanks for taking my questions.

Thank you.

That does conclude the question and answer session I'll now turn the conference back over to you for any additional foreclosing remarks.

Thank you Justin and thank you all for joining US today. This concludes our first quarter update we look forward to keeping you updated throughout the year and speaking with you again soon have a great rest of your evening.

Thank you and that does conclude today's conference with you. Thank you for your participation have an excellent day.

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