Travere Therapeutics, Inc. (TVTX) Q4 2016 Earnings Report, Transcript and Summary

Good day, ladies and gentlemen, and thank you for standing by. Welcome to the Retrophin Incorporated Fourth Quarter and Full Year 2016 Financial Results and Corporate Update. At this time, all participants are in a listen-only mode to prevent background noise. [Operator Instructions] We will have a question-and-answer session later and the instructions will be given at that time. And as a reminder, this conference is being recorded. Now, I would like to welcome and turn the call to Senior Director, Investor Relations, Mr. Chris Cline.

Thank you, Collin. Good afternoon everyone, and thank you for joining Retrophin’s corporate update and fourth quarter and full year of 2016 financial results call. Joining me on the call today 1are Steve Aselage, Chief Executive Officer; Neil McFarlane Chief Operating Officer; and Laura Clague, Chief Financial Officer. Also in the room with us is our newly appointed Head of Research & Development, Dr. Bill Rote. Before we begin, I'll provide a brief outline of how we will move throughout the call today. Steve will open the discussion by walking through us Sparsentan update, highlights from other programs. Neil will then give an overview of our operational performance, followed by an update on the financials from Laura. We will then pass the call back to Steve for his closing remarks before moving to Q&A. Prior starting, I would like to remind everyone that statement made during this call regarding matters that are not historical facts or 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 involved known and unknown risk uncertainties and assumptions that may cause actual results, performance and achievements to differ materially from those expressed or implied by statement. Please see the forward-looking statement disclaimer on the Company's press release issued today as well as the risk factors section in our Form 10-K filed with the SEC. In addition, any forward-looking statement represents our view only as of this date such statements are made March 1, 2017 and Retrophin specifically disclaims any obligation to update such statements to reflect future information, events or circumstances. With that, I'll now turn the call over to Steve. Steve?

Thanks, Chris. Good afternoon, everyone and thank you for joining us. We made substantial progress on both the development and commercial fronts in 2016. I am very proud of our many accomplishments during the year. Most recently in the fourth quarter, we presented additional positive data from the Phase 2 DUET study of sparsentan. We've reached agreement under a scheduled protocols assessment on a Phase 3 trial for RE-024, and we grew revenue by more than 20% over the same period last year. Importantly, all of our progress in the fourth quarter and throughout the balance of 2016 puts us in a great position to deliver significant sustainable value in 2017 and the years ahead. A meaningful component of that future values inside ties to sparsentan. I know much of the focus recently has been on its regulatory pathway. So, I'll walk through our update from earlier today. To do that, let me first take a step back and set the stage for interactions with the Food and Drug Administration. As most of you know in September, we announced positive top line results from our Phase 2 DUET study of sparsentan in focal segmental glomerulosclerosis or FSGS. Those results show that the overall sparsentan treatment group achieved statistical significance in the primary efficacy end point, demonstrating a greater than twofold reduction of proteinuria compared to irbesartan after the eight week double blind treatment period. In November, we followed up with additional positive data from the DUET trial, which was presented in the late breaking oral solution at the American Society of Nephrology Kidney Week. Those results included an analysis of the trial secondary end point modified partial remission of proteinuria. This endpoint is defined as proteinuria levels of less than or equal to 1.5 grams urinary protein and a greater than 40% reduction proteinuria from baseline. Importantly, modified partial remission has been associated with long-term preservation of renal function in FSGS. Our data shows that after an eight-week period, double blind treatment period, 28.1% of patients receiving sparsentan achieved modified partial remission of proteinuria, compared to 9.4% of irbesartan-treated patients. Also notable was the change and the proportion of patients achieving modified partial remission during the open label period of the study. After 48 weeks of treatment with our sparsentan, 57.7% of patients achieved modified partial remission. In addition for the group transferring from irbesartan to sparsentan at the beginning of the open label period. The proportion of patients achieving modified partial remission increased from 9.4% to 50% after receiving sparsentan for 40 weeks. Further analysis of the safety database from the initial eight week double-blind treatment period presented at the conference showed that sparsentan was generally safe and well tolerate. So heading into our end of Phase 2 meeting with the agency in late January, we build a strong body of evidence supporting potential amount of sparsentan. I’m pleased to share that we had a very constructive and insightful interaction with the agency. We came away from the meeting with a much needed clarity on how to move sparsentan forward as expeditiously as possible. Specifically, we are pleased to have alignment on an outline of a pivotal Phase 3 trial design with the FDA agrees may demonstrates sparsentan's benefit for patients with FSGS and enabled an NDA filing. Perhaps most importantly, our path to potential following along encompasses single trial with pre-and the post-marketing elements. The pre-marketing portion of the trial will focus on an interim analysis to proteinuria, which shows a substantial treatment effect which would in turn enable an NDA filing for accelerated approval under Subpart H. A key part of our dialogue with the agency was around the ability to show treatment affect on proteinuria in the interim analysis to provide confidence that the post-marketing element of the trial would be able to verify the anticipated benefit. They provided valuable feedback on an approach to determine this threshold and in fact provide a statistical guidance along the minutes we just received. We believe our definition of modified partial remission of proteinuria will be the right bar to set for the interim analysis. As many of you know, this definition was derived in conjunction with the Neptune Consortium and based on statistical analysis of more than 200 FSGS patients in multiple data bases. This work showed the better long-term outcomes were associated with FSGS patients who reach a modified partial remission of proteinuria. Given the strong related to the from our Phase 2 direct trial, we believe it does give us a great potential to expedite sparsentan path to approval. As I mentioned, we are recently in receipt of the minutes and modeling guidance. So, we need to spend some additional time with our statistical team to complete the analysis and gain agreement with FDA on this particular piece, we are confident we’ll get there in a very near future. The post-marketing portion of the trial will subsequently monitor changes and estimated glomerular filtration rate or EGFR over a longer period of time. EGFR is widely regarded as the best overall major of kidney function. So, we are pleased that its clarity and the outline of the trial as we make made further progress on the statistical plans will be in position to give more detail on the specific trial design elements including patient numbers and observation periods. We look forward to continue our discussion with the agency to finalize protocol and initiating with trial later this year. It is worth noting that since DUET results were originally announced and presented last fall, external awareness and excitement around the sparsentan program has grown significantly in the investigator patient and advocacy communities. Channeling this excitement will be a great asset for us as we look to begin enrollment of this pivotal trial. We will also utilize our experience with the DUET study and leverage our strong and growing network of FSGS stakeholders to efficiently enroll this trial far more quickly. We have identified more than 100 sites globally with interest in participating in the trial. We estimate these sites represent more than 1,600 patients who would be potentially eligible for entry into the trial. Beyond that, we will continue to work closely with the Neptune Consortium and NephCure International Organizations to ensure we fully involved and leverage the resources of the broader nephrology community. Before moving on to other product updates, I want to briefly highlight that this path forward gives us a great opportunity to finalize our valuation of additional indications for sparsentan. This could significantly increase its overall value proposition. We will have more on that later this year as we move towards key decision points. Moving on to other late-stage program RE-024 for PKAN, the stride we made in 2016 with this program will instrumental and building excitement to the program and advancing RE-024 close to the patients. In March ACMG, was a milestone for us as we shared RE-024 data for the first time in medical congress. We followed that up with further case reports and presentations at the MDS meeting in June. This upgrades a new level of excitements among investigators and the PKAN community as a whole. More recently, in the fourth quarter, we were very gratified to reach an agreement under the spot process with FDA on a Phase 3 trial to support an NDA filing for RE-024. Today, we have gained central IRB approval for this trial and have further site preparation underway. As you know, our CMO have an unforeseen manufacturing delay which resulted in us having to move first patient dose in the trial. We believe that we have resolved the issue now have multiple processes running in parallel that give us increasing confidence will be in position to dose the first patient around the mid year. Finally, I am pleased to report that all four patients receiving RE-024 treatment is part of position initiated protocols outside the U.S. remained stable on therapy today. Regarding liquid ursodeoxycholic acid, we have a bit more formulation work to go and are more than likely looking at a filing in 2018. To round up the development discussion, I want to highlight the most recent addition to our leadership team, Dr. Bill Rote, sharing the room with us today. Bill brings the well proven abilities to lead successful research and development organizations and a scientific expertise that aligns directly with our mission of delivering life changing therapies to people living with rare diseases. He joins at an exciting time and we look forward to his leadership and our efforts to advance life therapies for patients. Lastly, I want to note that made consistent progress with growing all of our commercial products closing the year with great momentum in this regard which will health curious in and the further growth in 2017. Let, me now turn the call over to Neil to walk through our performance update. Neil?

Thanks, Steve. From an operational perspective I am pleased with the value we continue to create for people living with rare diseases, and of the priors for making to support our development efforts which will help shape the future of our organization. Starting with our commercial performance, we closed the year with the strong fourth quarter. We grew revenues by 23% year-over-year and made our top line guidance by reaching a 134 million for the full year 2016. Our success in 2016 and continued organic growth is testament to the value our products provide to patients and the consistent focus of our commercial organization. Regarding, Thiola, the matter remained consistent during the quarter. Our total care hub continues to offer excellent patient support and as a result of this dedicated service compliance has remain steady. Our bile acid therapies Cholbam and Chenodal, also had meaningful growth year-over-year and solid respective patient based is expand during the quarter. For Cholbam, we continue to be excited about the potential impact of the Retrophin sponsored neonatal and adult cholestasis sequencing panel. During the quarter, we saw consistent uptick of this important diagnostic tool among physicians and it continues to evolve the vital piece of the diagnostic paradigm for patients with bile acid synthesis disorders. Regarding Chenodal, the CTX prevalent study continues to enroll subjects and be a value to better to understand the population and help raise awareness for the disorder. We’ve gleaned meaningful information from the enroll population in the study; and in 2016, it showed that patients can be identified as a result of these efforts. Looking ahead to 2017, we have a number of exciting initiatives that we expect to further our top line growth over 2016. Most notably, we are implementing a small expansion of our sales force which will allow us to have two dedicated teams, one for Thiola and one for Cholbam. Moving forward, this will enable us to efficiently maximize the value and growth potential of both products in 2017 and beyond. In 2017, the Thiola team will be looking to leverage our experience with ICD-10 code data and identify more physicians that have diagnosed patients in their practice. One thing we’ve learned over the past year is that there are still a significant number of patients diagnosed with cystinuria susceptible to stone formation and we want to ensure, we do our part to educate treating physicians. For Cholbam, the focus for bile acid synthesis disorders will be on supporting the evolving change in the diagnostic paradigm. The team will do this by raising more awareness of our genetic Cholestasis panel and increasing its use. Based on our planned efforts, we expect the use of the panel to double in 2017. For Zellweger spectrum disorders, we will be pushing forward our patient identification efforts and embarking on further education about treating liver dysfunction in this fragile population. Specifically, we want to ensure physicians and caregivers alike understand the importance of monitoring the impact of liver function overtime and the potential for Cholbam to positively impact ZSD patients. In terms of development support, we are increasingly excited about the added potential to create long-term value with sparsentan. As Steve mentioned, work on assessing additional indications is ongoing and we look forward to giving more details in the near future. Finally, I will touch on our business development efforts before turning it over to Laura to run through the financials. BD remains a key piece of our strategy and with further clarity on the sparsentan pathway we're prioritizing potential transactions to build the sustainable portfolio to serve rare disease patients. I’ll now turn the call over to Laura, to give you the financial update. Laura?

Thank you, Neil. Net product sales from our commercial portfolio were 37.3 million in the fourth quarter and 133.6 million for the full year 2016. As Neil mentioned earlier, we had growth across all of our commercial products during the quarter, which led to the 22% growth over the fourth quarter last year. We reported a GAAP net loss of 8.6 million for the fourth quarter and a net loss of 47.9 million for the full year 2016. Adjusting for extraordinary in one-time expenses resulting in a net income of 0.1 million for the quarter and a net income of 4.4 million for the full year. Significant non-cash adjustments for the quarter included 18.9 million of non-GAAP operating loss adjustments, $6.5 million related to the Company’s derivative liability resulting from share price fluctuation and an income tax benefit of 3.7 million. R&D expenses on a GAAP basis were 20.1 million for the fourth quarter and 70.9 million for the full year 2016. The increase over the fourth quarter last year is due to higher clinical expense related to sparsentan and RE-024. On an adjusted basis R&D expense for the fourth quarter was 17.6 million and 60 million for the full year 2016. Relevant non-cash expenses for the fourth quarter included 2.5 million of stock-based compensation and amortization. Selling, general and administrative expenses were 26.6 million on a GAAP basis in the fourth quarter and 92.8 million for the full year 2016. The increase over the fourth quarter of 2015 is largely attributable to further investment in sales and marketing programs and support of our commercial product. On an adjusted basis, SG&A expense for the fourth quarter was 17.9 million and 58.4 million for the full year 2016. Significant one-time and non-cash adjustments for the quarter consisted of 8.7 million related to stock-based compensation and depreciation and amortization. As of December 31, 2016, we had approximately 302.7 million in cash and cash equivalents, marketable securities, and notes receivables from the sale of our PRB. This value includes the present value of the 147.5 million payments remaining from Sanofi. Notably in the fourth quarter, we had non-recurring uses of cash totaling 19.4 million. Of this 19.4 million, 9.2 million was primarily related to income tax payments as a result of the receipt of the second installment of our PRB payment, 8 million related to Cholbam net sales milestone and 2.2 million related to our previously disclosed advancement of legal fees. In 2017, we will continue to develop the necessary resource to advance our pipeline, notably two Phase 3 programs that we expect to deliver significant future value. As such, we do expect operating expenses primarily in R&D will increase over the levels reported for 2017. I'll now turn the call back over to Steve for his closing remarks. Steve?

Thanks, Laura. In 2016, we made progress by creating value for our stakeholders in many ways. We generated value by reaching key milestones with demonstrate sparsentan could represent a significant advancement in the treatment of FSGS and clarify the regulatory path to potential approval of RE-024 and PKAN. We also created value by delivering our commercial therapies to more patients that ever before. Through focus on execution and now further clarity on regulatory pathways sparsentan will build on those achievements in 2017. We are in the incredibly strong position advancing two first-in-class Phase 3 clinical programs supported by our commercial portfolio with double digit year-over-year growth expectations. Combining that with the ability to leverage our strong financial position to allocate our capital appropriately, both internally and externally will allow us to build sustainable long-term value that will help shape the promising future of our organization. Let me now turn the call back over Chris for questions. Chris?

Thanks, Steve. Collin, can we go ahead and open up the line for Q&A please?

[Operator Instructions] And our first question is from the line of Joseph Schwartz with Leerink Partners.

Great thanks very much and congrats on all the progress and the clarity from the FDA. I was wondering if the agency explained why you couldn’t file on the existing data and how you think the next study is likely to compare to DUET in terms of size and duration of the study as well as the doses that you’re thinking of study?

That's a couple of questions in there. Let me start out with probably the easiest ones. We got a couple of takeaways from the agency and their feedback. I think they appreciated the impact is sparsentan had for patients in DUET study, but they also felt that they would like to see a longer observation period then the eight weeks, which was a controlled observation period in DUET, that was clear. One of the things we are working with them on right now is what is the exact observation period that we want to put into the confirmatory trial we know it will be longer than eight weeks, but that exact timeframe has not been set yet we are working on that and details for. The other thing they have asked us to do in which DUET did not do for us reduction in proteinuria or the achievement of modified partial response to long-term changes in EGFR, we are working through database that we have accessed to now could be able to make that connection, that connection is going to give us we believe the insight to be able to know how many patients intervention and then how long before the EGF observation. I think I covered all your questions.

And our next question is from the line of Liisa Bayko with JMP Securities.

Hi, thanks for taking the question and congratulations on the progress as well. In terms of the relationship with proteinuria and EGFR, is proteinuria thought to be a leading indicator or how can we enter the still that connection?

Yes, I think, certainly Neptune data was presented at ASN really clearly show that if you could achieve a complete or a modified partial response, as defined by the 40% reduction proteinuria and getting urinary protein down to 1.5 g per day or lower that you had improved kidney survival. EGFR, we believe there is a little bit quicker way to show the benefit that if you carry a study long enough, it would result in seeing a difference in kidneys survival. So, FDA has previously stated that EGFR is an acceptable endpoint for our approval. We can get there faster than time to dialysis and end stage disease. What we do need to do for them is go through those databases there and link up the modified partial remissions to patients change in EGFR slope and then the timeframes. And that’s what we are going to be working -- that’s where we are already working on right now, and we hope to be able to do that relatively quickly and then have final protocol back to the agency in the very near term.

I was looking at your DUET study results and there is no change in EGFR, but from what I understand that it would take significantly longer than the timeframe on your study to see a change. Is that something you’ll be looking at in your open label extension? And then also I noticed the patients in your study were sort of within the normal range maybe as you think about going forward might you one enroll a sticker population to see a better change on EGFR or what's the right way to think about that?

Yes, with regard to EGFR results in DUET, the observation period was only eight weeks and you would not expect to see any change in that sort of time period. We will definitely take a look at changes any EGFR and the extension, although at sometimes we believe we will probably be closer to two years to see the type of change and slope of EGFR that we would be comfortable with including into a protocol. And we aren't going to have enough patients two year mark in the open label to be able to pull as much out of our own data basis as we will be able to pull out the FSGS CT database and then Neptune database. So, we will give what we can from DUET, but we need other databases and fortunately have access to those databases.

And just looking at the patient population in DUET, it looks like as I mentioned they were kind of -- I think it was about 73 on average filtration rate at baseline. And then that seems to be within the normal range more or less. So, are you thinking about maybe -- do you think you will see a change in with a level like that? Or you think you might up to be looking at a different slightly maybe more severe patient population in Phase 3 to see a change on that metric?

Well, generally the patients with urinary protein around 3 are considered nephrotic and our median patient in DUET was around that nephrotic range. So, it was a relatively sick population, it was a distribution obviously. But I don’t think, we need to go through a sicker population to be able to see a meaningful difference in EGFR.

Okay and then just one last question, if I may. So, is the way you are thinking about Phase 3, I mean you did see a change of protein urine relatively short period of time that would be -- maybe wouldn't you just study that for such a long period of time you could file on that and then look for more like at least 24 months or longer to see data EGFR change and confirmatory?

Exactly, I mean that’s the great news we walked away from the meeting within. I realized that everybody or maybe not everybody, most people were hoping that we did an immediate accelerated approval, but we came out of that meeting with an agreement from FDA that proteinuria could be used as an appropriate endpoint for an accelerated approval. We came out of meeting with the agencies supportive of helping us with the other protocol that they would find acceptable. And we believe we have a path forward that gives us clarity on time, on cost and a clear opportunity to get the product through the clinical process and registration process in reasonable period of time.

And our next question comes from the line of Do Kim with BMO Capital Markets.

Hi, this is Alex for Do Kim. Thanks for taking my questions and congrats on the progress. We wanted to know. Did the FDA make any comments with regards safety and the Phase 3 study? Do they want to see more safety data with specific reference to hypertension and edema focus?

There were no issues related to safety that came up in the meeting.

And with the interim analysis protein urea, what will trigger the interim analysis exactly?

We are doing some statistically modeling right now where we want to do is to make sure that we have a long enough observation period that the agency is going to be comfortable that the number of patients or percent of patients reaching a remission modified partial or complete, is efficient to give them comfort that it is predictive of type of EGFR changes that we want to see. And then we are obviously second part of that is statistical modeling to see how many patients, we need to have reach that time point in order have the study powered to be comfortable that we want statistical significance when we take a look. And those numbers that it is not going to take us long to be able to pull that together and be able to give you details, I can’t do that today though, as we just very recently got the minutes and need a little bit of time to work through some of those statistical issues.

And then one more question, I know that some of the details you're being hammered out, but what doses will be evaluated and it would be evaluated in trial?

That’s a really good question and our plan is to start patients at 400 milligrams. All patients start with 400 milligrams in the sparsentan arm and then the physician can titrate up to 800 milligrams. So, 400 starting dose up to 800.

And our next question is from the line of Joseph Schwartz with Leerink Partners.

Hi, thanks for getting me back on. Actually my follow-up was in the doses which you just answered, but while I've got, how about the DUET study? And when do we likely to get another update on the patients then and at what point will GFR data be reported out of DUET? Thanks.

Yes, I mean that's I can’t give you affirming answer on that. We have a publication plan which will go into more detail then we were able to present in the relatively short oral session and ASN. I believe that has been submitted, but not accepted yet, so I can’t give you a firm date on that. As soon as we’ve got a firm date of publication, we will get that to you though.

Okay. And then do you anticipate using the same active comparator in the Phase 3 study?

We do yes. Yes, good question again, irbesartan is what we anticipate being active comparator.

[Operator Instructions] And I’m not showing any further questions. I’d like to turn the call back to management for final remarks.

Thanks, Collin, and thanks everybody for joining us today. We look forward to updating you on our progress in the coming months.

Ladies and gentlemen, thank you for participating in today’s conference. This concludes the program and you may all disconnect. Have a wonderful day.