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Retrophin Inc (TVTX -0.79%)
Q4Â 2018 Earnings Conference Call
Feb. 26, 2019, 4:30 p.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
See all our earnings call transcripts.
Prepared Remarks:
Operator
Good afternoon, ladies and gentlemen, and welcome to the Retrophin Incorporated Fourth Quarter and Full-Year 2018 Financial Results and Corporate Update Conference Call. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. (Operator Instructions) As a reminder this conference call is being recorded.
I would now like to turn the conference over to Chris Cline. Sir, you may begin.
Chris Cline -- Vice President, Investor Relations & Corporate Communications
Great, thank you, Tom. Good afternoon, everyone, and welcome to Retrophin's fourth quarter and full-year 2018 financial results and corporate update call. Today's call will be led by Eric Dube, our Chief Executive Officer. Eric will be joined by our Chief Medical Officer, Dr. Noah Rosenberg; and our Chief Financial Officer, Laura Clague for their prepared remarks. Neil McFarlane, our Chief Operating Officer and Dr. Bill Rote, our Senior Vice President of Research and Development will be joining 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. 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 in our Form 10-K filed with the SEC today.
In addition, any forward-looking statements represent our views only as of the date such statements are made, February 26, 2019, and Retrophin specifically disclaims any obligation to update such statements to reflect future information, events or circumstances.
With that, let me now turn the call over to Eric. Eric?
Eric Dube -- President and Chief Executive Officer
Thank you, Chris, and thank you all for joining us today. As this is my first corporate update call since joining Retrophin last month, let me start by saying how honored I am to be on board and leading an organization with such an important mission. Retrophin has a number of important opportunities to deliver innovative treatments to patients living with rare disease and the coming years will be pivotal in shaping the future of our Company and how we can help patients. Aligned with this mission and opportunities is our work to bring potential first-in-class treatments for diseases like pantothenate kinase-associated neurodegeneration or PKAN, focal segmental glomerulosclerosis or FSGS and IgA nephropathy or IgAN to communities with severe unmet needs.
Last quarter, further demonstrated our ability to execute on the key clinical objectives to advance our fosmetpantotenate and sparsentan development programs for these rare disorders. I want to commend our employees for an impressive fourth quarter and start to 2019 and to thank them for their unwavering focus and performance.
For fosmetpantotenate, we were very pleased to complete enrollment in the Phase 3 FORT Study in December. We know how difficult life is for these patients, who are living without an approved treatment. So we are very encouraged to see their dedication to the FORT Study. Completing enrollment in December put us on track for top-line readout in the third quarter of this year, which will be a major milestone for the program and potentially for PKAN patients. As you know, the study is designed in alignment with a Special Protocol Assessment agreement with FDA. And if we generate positive data, it should support both NDA and MAA filings. Our development teams are already working together on submission preparations, so that we will be in position to put a high-quality package together for review in 2020.
We are also making good progress on our sparsentan programs. Given the need for effective therapies to treat these rare nephropathy, we are advancing the two pivotal programs as quickly as possible, and working to establish our leadership in the rare nephrology community. We continue to advance our efforts to enroll the Phase 3 DUPLEX Study of sparsentan in FSGS. Our focus remains on randomizing the first 190 FSGS patients in order to enable the top-line readout from our interim proteinuria assessment in the second half of 2020. We believe a positive result from that readout would allow us to potentially gain conditional marketing approval in the US and Europe.
In parallel, we continue to make significant progress with our additional indication for sparsentan. During the fourth quarter, we achieved a key developmental milestone with the first patient dosed in the Phase 3 PROTECT Study for IgA nephropathy. IgAN represents a significant opportunity for sparsentan to potentially help hundreds of thousands of people globally, who are currently facing progression to end stage renal disease with no indicated treatment options. The teams here did an amazing job of operationalizing this pivotal study in a short period and we are looking forward to initiating additional sites around the world. Through these two pivotal programs, our goal is to contribute to the better understanding of these rare diseases and improve patient's lives with the introduction of a potential first-in-class treatment.
Another key value driver for us is the ongoing collaboration with Censa to advance the CNSA-001 for the treatment of phenylketonuria or PKU. Our partners at Censa made considerable progress in the fourth quarter and to start this year. Just recently, the Censa team had their Phase 1 dosing study published online in the Journal of Molecular Genetics and Metabolism. This is an important milestone for their program and the findings support the hypothesis that the amount of BH4 in plasma derived from CNSA-001 was significantly greater than what we're seeing with standard of care. As many of you know, Censa is currently running a Phase 2 proof-of-concept study to further evaluate CNSA-001 versus current standard of care, and we will continue to be pleased with their development.
We understand from our partners that they remain on track to deliver top-line data to Retrophin in the second quarter of this year, with a full data package to follow thereafter. We continue to believe this program has the potential to address a significant unmet need for patients and possibly BH4 pivotal program in our pipeline. We anticipate making the decision on our option to acquire Censa after we have had a chance to fully evaluate the complete data as well as the regulatory pathway and other requirements for successful product development.
The fourth quarter also marked another consecutive quarter of period-over-period growth from the commercial portfolio. Our approved products continue to play an important role in patient's lives. Thiola performed in line with expectations and we saw a nice rebound in the bile acid products during the fourth quarter. Our commercial teams have helped to deliver organic growth based on new patient starts. And we look forward to continuing that growth at a similar rate in 2019.
Also during the quarter, the NDA for the new formulation of Thiola was accepted for review by the FDA and assigned a June 30th PDUFA date. If approved, we may have the ability to improve the treatment experience for cystinuria patients and we are working to make sure that we have a solid launch in the second half of the year.
I'd now like to turn the call over to Noah for an update on our late-stage development programs.
Noah L. Rosenberg -- Chief Medical Officer
Thanks, Eric, and good afternoon to everyone. As Eric mentioned, we met key clinical milestones in the fourth quarter that have set the stage for 2019 to be a very exciting year for all of our programs in development.
I'll start with fosmetpantotenate for PKAN. This will be the first of our three pivotal programs under way to readout. In December, we announced the completion of enrollment in our Phase 3 FORT Study. FORT is our placebo-controlled pivotal trial being conducted under a SPA agreement with the FDA and is expected to support approval in both the US and Europe, if we generate positive results. I want to commend our clinical team and investigators for enabling us to reach our target of completing enrollment in this study by year-end.
Most recently, we had our fourth review of safety for our Independent Data Monitoring Committee. This review was based upon a 100% enrollment in the trial and the committee recommended we continue as planned. Based upon the randomization schedule and pre-specified 24-week observation period, we are expecting to have database lock and top-line data from this study available in the third quarter of this year. Our internal biometrics team continues to work closely with our CRO partner to ensure we have the highest quality data and we look forward to sharing those results with all of you and the PKAN community later this year.
If successful, we want to make fosmetpantotenate available to patients as quickly as possible. In that spirit, our development teams are working to be in position to submit NDA and MAA applications after we receive our data package. Sparsentan also continues to make steady progress toward becoming a potential first-in-class therapy for both FSGS and IgA nephropathy, rare and debilitating kidney disorders, which often progressed to end stage renal disease. With our two pivotal studies progressing, I am pleased with how we are continuing to make progress toward becoming the leaders in the rare nephrology community.
The Phase 3 DUPLEX Study of sparsentan in our lead indication FSGS continues to advance toward the goal of enrolling the first 190 out of 300 subjects necessary to enable our interim assessment. Our team has done a great job engaging with investigators and I'm pleased that we now have more than 100 opened study sites actively screening patients. The overall reception has been positive and we continue to believe that the community is excited about driving this landmark study to meet its enrollment goals. We expect our work over the next 10 months will enable a top-line readout of the FSGS partial remission of proteinuria endpoint during the second half of next year. If positive, we would expect to subsequently pursue an NDA filing under Subpart H in the US and conditional marketing authorization in Europe.
We also continue to make progress in our next indication for sparsentan, IgA nephropathy. As Eric mentioned, at the end of December, we reached our targeted milestone of dosing the first patient in the pivotal PROTECT Study. The PROTECT Study is our active controlled Phase 3 clinical trial designed to support registration of sparsentan for the treatment of IgA nephropathy in the US and Europe. Similar to our ongoing Phase 3 study in FSGS, the PROTECT Study utilizes a proteinuria-based endpoint after 36 weeks of treatment with sparsentan to support a Subpart H submission in the US and consideration for conditional marketing authorization in Europe.
While still early in the study rollout, we've completed three global investigator meetings and are encouraged to have site initiations progressing in line with our plan. For the balance of this year, we'll be focused on building further awareness of our study and getting additional sites opened around the world, to move toward our ultimate goal of enrolling 280 patients in the study. Based on our current projections, we expect to generate top-line data from the 36-week proteinuria assessment of 280 patients in the first half of 2022.
Looking ahead, our teams will remain focused on meeting our near-term development goals to move us closer to potentially delivering important new treatment options to the patients we serve. We are especially excited by the opportunity to advance our pipeline, enduring multiple NDA and MAA filings in the coming years starting with fosmetpantotenate in the near future.
Let me now turn the call over to Laura for financial update. Laura?
Laura M. Clague -- Chief Financial Officer
Thank you, Noah, and happy birthday to you.
Net product sales from our commercial portfolio increased to $43.8 million for the fourth quarter and $164.2 million for the full-year 2018. This represents a 6% increase over the full year, 2017. We reported a GAAP net loss of $7.5 million for the fourth quarter and a net loss of $102.7 million for the full-year 2018. After adjusting for noncash expenses, we reported a non-GAAP net loss of $8.5 million for the fourth quarter and $51.8 million for the full year 2018.
On a GAAP basis. R&D expenses were $32 million for the fourth quarter and $123.8 million for the full-year 2018. The increase over 2017 is largely attributable to higher expenses to support our clinical and product development efforts for fosmetpantotenate and sparsentan as well as funding for CNSA-001 collaboration. On an adjusted basis, R&D expenses were $30.1 million for the fourth quarter and $116.6 million for the full-year 2018. Relevant noncash expenses for the fourth quarter included $1.9 million of stock-based compensation and amortization.
Selling, general and administrative expenses were $26 million for the fourth quarter and $103.7 million for the full-year 2018. This is largely unchanged from our 2017 SG&A spending levels. On an adjusted basis, non-GAAP SG&A expenses for the fourth quarter were $18.1 million and $72.4 million for the full-year 2018. Significant noncash adjustments for the quarter consisted of $7.8 million of stock-based compensation and depreciation and amortization. As of December 31, 2018, we had $471.5 million in cash and cash equivalents and marketable securities, a very strong financial position for our Company.
Looking ahead, we expect our operating expense levels in 2019 to increase over 2018 levels. We are investing in our ongoing clinical and product development activities as we advanced our pivotal programs, and as such, we expect our year-over-year R&D expenses to increase materially over the course of 2019. While there may be some uneven quarters with the timing of manufacturing runs or CRO activities, we expect the general trend to be upward throughout the year. We anticipate that SG&A will incur smaller and more stable increases throughout the year as we invest in our product launch capabilities.
Lastly, as a result of our 2025 convertible notes issued in the third quarter of last year, you should expect that our interest expense will be higher in the future when compared to previous year.
Let me now turn the call back to Eric for his closing remarks. Eric?
Eric Dube -- President and Chief Executive Officer
Thank you, Laura. In closing, I would like to share some initial impressions from my first two months at Retrophin. I've spent much of my time with the teams to get an initial sense of the strength of our business and areas where we can continue to build our organization for future growth. I've also spent time meeting with community leaders and stakeholders to gain additional perspective on the Company. It has become clear to me that I have inherited a tremendously talented group of employees dedicated to helping patients living with rare disease and the strong foundation built upon commercial capabilities and the pipeline with the potential to create significant value for patients.
Looking ahead, we know the value creation will come from our pipeline, and as such, our number one priority will be to continue the near-term execution of these programs. I am going to leverage my background in delivering innovative treatments to ensure that we are doing everything we can to get our therapies to patients as quickly as possible.
As part of my evaluation of the organization over the first 100 days of my tenure, I am assessing all of our programs, their timelines and our readiness to deliver them to patients. What I have seen in the first 60 days gives me confidence in our product candidates and the expertise of our people. I am encouraged by our ability to execute as you most recently saw with the completion of enrollment in the FORT Study and our capabilities to ultimately get our first-in-class therapies across the finish line.
With our first pivotal readout reaching key enrollment milestones with sparsentan and the decision on the CNSA-001 program planned for this year, it is going to be an important and exciting year for our Company's advancement on this front. Simultaneously, I will be working with our leadership team to further our plans to accelerate the Company's growth for the future. We are uniquely positioned with a commercial portfolio that will continue to provide an important source of funding to pursue and execute our R&D efforts. But more importantly, it gives us a strategic advantage when bringing new therapies to market.
We expect to be launching at least three new therapies in four indications in the coming years and the strong capabilities of our commercial and medical teams will be instrumental in our successful launches. We'll also be laying additional groundwork for Retrophin's long-term sustainability and success, which will require a disciplined approach to our execution and a continued focus on business development to further diversify our business. I look forward to continuing to deliver therapies for people living with rare disease and to sharing more with all of you as we execute on our goals of bringing the first approved treatments to address the significant unmet needs of patients with PKAN, FSGS and IgAN as well as a potential improved treatment for PKU.
Okay, Chris, I think we can open up the call for questions.
Chris Cline -- Vice President, Investor Relations & Corporate Communications
Great, thanks, Eric. Tom, can we go ahead and open up the lines for Q&A, please?
Questions and Answers:
Operator
Sure. Thank you. (Operator Instructions) And our first question comes from the line of Tim Lugo from William Blair. Your line is now open.
Tim Lugo -- William Blair & Co. -- Analyst
Thanks for taking my question. And I'd also like to add a happy birthday to Noah and (inaudible) just ask Eric questions.
Noah L. Rosenberg -- Chief Medical Officer
Thank you, Tim.
Tim Lugo -- William Blair & Co. -- Analyst
No problem. For the CNSA-001, it sounds like the acquisition may not just be dependent on the upcoming data readout, but also I think you noted Eric in your commentary some comment about the regulatory path forward. Does that mean you may want to have an end of Phase 2 meeting with the agency before deciding to bring that program in-house? And I guess, are there any mechanisms for where you would go forward with that program maybe in some sort of co-developments, I guess path forward?
Eric Dube -- President and Chief Executive Officer
Yeah. Thank you, Tim, for the question. Certainly, we are working closely with Censa through the joint steering committee in looking at the path for Phase 3, what that would look like. And my comments really about the path forward and the decision to acquire the company are really based on making sure I can make the most informed decision given that we would be using a sizable portion of the equity for that acquisition. I want to make sure that we look, not just at the totality of the data, but how quickly we're able to bring this product to the FDA and ensure that we are keeping a continued keen eye on the evolving competitive landscape. So I'm not sure if that significantly changes how that decision would have been made in the past, but I certainly want to make sure that I am using the company's resources in the best way possible to deliver patient value as well as value for equity holders.
Tim Lugo -- William Blair & Co. -- Analyst
Okay, that's understood. And will you be thinking at all about maybe its impact in BH4 deficiency or I know that there is also the gastroparesis? Investigator led study ongoing or maybe that's already over with, but will you be looking at any other indications, I guess, outside of PKU and your thoughts around whether you acquire 001 or not?
Eric Dube -- President and Chief Executive Officer
Yes. So we are going to be looking at any data that is made available from Censa at the time that we get the complete data package for the PKU program. As you know, there are a number of studies like primary BH4 deficiency, we would be very excited if we see positive data where we'd be able to bring that to patients, but I certainly wouldn't want to wait until those data are completed or those programs are completed before we make a decision on the PKU data.
Tim Lugo -- William Blair & Co. -- Analyst
Okay, fair enough. So maybe one last question, you mentioned you're assessing timelines for DUPLEX, would you say that you're still on track for the 190th patient to be enrolled in the second half of this year? And I guess, Noah, you can't answer, because it's your birthday and I did mention I won't be asking you any question.
Eric Dube -- President and Chief Executive Officer
So, I'll speak on his behalf Tim. From everything that I have seen, we continue to be on track and see as we move forward as you often see with trials, a continued acceleration of screening and enrollment. Noah, anything else you want to add as a birthday gift.
Noah L. Rosenberg -- Chief Medical Officer
I think you've captured it well, Eric.
Eric Dube -- President and Chief Executive Officer
Thank you, Tim.
Tim Lugo -- William Blair & Co. -- Analyst
No, problem. Thanks for the question.
Operator
Our next question comes from the line of Maury Raycroft from Jefferies. Your line is now open.
Maury Raycroft -- Jefferies -- Analyst
Hi, everyone, and congrats on the progress. My first question is on CNSA-001, specifically on the blood-brain barrier penetration benefit. And I'm wondering, with the Phase 2 readout, it will be able to, if you're looking for any neuro benefit based on a standard measure, and if you're not looking for a benefit on efficacy, could we potentially anticipate differences in our neuro AEs from that readout?
Eric Dube -- President and Chief Executive Officer
Thank you, Maury. I'm going to pass that over to Bill.
William Rote -- Senior Vice President and Head of Research and Development
Sure. Thanks for the question, Maury. I think the short answer to your question is that the Phase 2 concept study does not have any neurocognitive assessment in protocol. So, it's really primary looking at safety and efficacy, efficacy driven by plasma phenylalanine. So with the second piece of your question around adverse events, would we be able to see if I understand it a differential on adverse events between CNSA-001 and the standard of care vis-a-vis the differential in blood-brain penetration. I think the answer is probably likely no, just based on the number of patients in the study. 24 patients we are certainly going to look at that, but I would expect in a study, traditionally a study that small you don't have a large enough signal-to-noise ratio to pick out something that would be in my eye probably subtle, and that would becoming a larger in later studies.
Maury Raycroft -- Jefferies -- Analyst
Got it. So, with this readout, we are probably not going to get a good sense of benefit based on the drug's ability to penetrate the blood-brain barrier.
William Rote -- Senior Vice President and Head of Research and Development
It's really going to be an evaluation of fee reductions really what we are going to be looking at.
Maury Raycroft -- Jefferies -- Analyst
Got it, OK. As far as that assessment on the primary endpoint, can you -- should we anticipate stat sig differences and potentially with some of the secondary endpoints where you are looking at the 10% 20% and 30% reduction rates? Should we look for any stats on that as well?
William Rote -- Senior Vice President and Head of Research and Development
I think on overall fee reduction percent reduction in phenylalanine, I think we should be seeing statistical significance. I expect that these won't be difficult to see changes between the agents, whether it breaks down when you start to get into the different buckets of less than 10%, less than 50%, less than 30%. It's going to come down to a numbers game. Certainly the trend should should be consistent across those groups. But I do think, they'll see statistical differences in the overall endpoint.
Maury Raycroft -- Jefferies -- Analyst
Got it. That's very helpful. And then just a question on FSGS, so you mentioned you've got 100 sites up and running. I guess, can you provide any general perspectives on the types of patients that you're enrolling and whether you're seeing any enrollment trends based on the regions where the sites are located in, and how that could shape your strategy in the U.S. or ex-U.S. that you're willing to comment on?
William Rote -- Senior Vice President and Head of Research and Development
Noah, do you want to take that?
Noah L. Rosenberg -- Chief Medical Officer
Yeah, so just to also -- back into -- Eric's question for us. The team has done a great job of engaging with investigators generating screening activity. We now have as we said over 100 sites up and running. And so while we haven't done a formal analysis to your question on patient demographics our real-time monitoring suggests, the patients coming in or aligning with our pre-specified protocol. It's probably still too early to look for regional differences, but it's something we will definitely monitor and pay attention to.
Maury Raycroft -- Jefferies -- Analyst
Okay, great. Thank you very much, and congrats again.
Noah L. Rosenberg -- Chief Medical Officer
Thank you.
Operator
Our next question comes from the line of Joseph Schwartz from SVB Leerink. Your line is now open.
Dae Gon Ha -- SVB Leerink -- Analyst
Hi, good Afternoon. Thanks for taking my questions. And congrats on all the progress. This is Dae Gon dialing in for Joe, and I'd like to extend our welcome to Eric for joining Retrophin. Look forward to meeting you soon. So just a couple of questions. Let's start with the commercial. Eric, you were talking about the projection on Thiola sales to be roughly in line with the 2018 year-over-year growth, if I heard you correct. So just wondering, I mean, you have a Thiola 2.0 PDUFA date at midyear and you said that the launch should commence, if approved in the second half of '19. Given the four attributes, if I remember correctly of benefit that the Thiola 2.0 delivers, are you just being conservative?
Can you walk us through sort of a logic behind getting roughly a 6% year-over-year growth there? And then in terms of I guess sparsentan and DUPLEX Study, just looking through the baseline, urine protein creatinine ratio of greater than or equal to 1.5 grams per gram. That's slightly over or higher than what DUET was inherence of inclusion. So just wondering, can you remind us in your subgroup analysis that you conducted in DUET, to what extent does baseline UPCR impact outcome whether it would be modified partial remission, complete remission or proteinuria reduction? And I guess from a commercial standpoint, what proportion does that 1.5 gram cut off represent into roughly 40,000 patient populations?
Eric Dube -- President and Chief Executive Officer
Thank you, Joe. And thank you for the welcome. Actually, why don't we start with the second question. Noah, do you want to talk about the UPC at baseline.
Noah L. Rosenberg -- Chief Medical Officer
Yeah, we -- so I don't believe specifically that we've analyzed an done that cut that you're referring to. But I think overall, what we've seen was directional improvement regardless of patients baseline UPCR. And I think the -- that's total difference, the 1 to 1.5, it's probably not that clinically meaningful. So we would expect any significant difference.
Eric Dube -- President and Chief Executive Officer
Thank you, Noah. And with regard to the commercial projections. We are expecting that our overall growth rate across the three commercialized products will be consistent with that 6% growth that we saw last year. With regard to the expected growth profile specifically and more about the four potential benefits. I'm going to turn that over to Neil.
Neil McFarlane -- Chief Operating Officer
Yeah, Dae Gon, so I want to talk about two things. One, you talked about the four attributes of the product. We've consistently said that our new more patient friendly accumulation, it's three of the four attributes of which we will continue to be consistent in that and through until last PDUFA date when we actually described those, the whole business as Eric said, in line with our previous 2018 growth rate. So on Thiola, we still see new patient adds matter of fact that our early Q1 is absolutely in line with our guidance for 2018. And maybe I'll stop there and see if there is anything else that I might've missed.
Dae Gon Ha -- SVB Leerink -- Analyst
Yeah, no, I guess that's fairly consistent, just wondering given the benefit of low pill burden and the pill is now cracking. I would assume that it would be better on the compliance side and given the new patient adds and higher retention probably the growth might be higher. So, I guess your answer is sufficient enough and we can just see what the June PDUFA date unfold. If I can ask just one more question though to your point, Eric, about BD going forward. Can you maybe elaborate on how you are thinking or approaching the BD strategies going forward?
I guess we have got kind of two scenarios or the way how you see two scenarios is one which is Censa-001 kind of becomes an in-house product and you develop it? And scenario two is where you forgo the option and look for alternatives. Just thinking will nuero or neurodegeneration continue to be a focus at the Company given that PKAN is cutting that wheelhouse? Or will you open a new organ system beyond nephropathy that you have with the sparsentan? I guess in terms of evaluation, what is the sweets spot that you are considering? Thanks. bye.
Eric Dube -- President and Chief Executive Officer
Okay. Thank you, Joe. With regard to the overall development approach, we want to continue to look at areas in rare disease that have a high unmet need. While we believe that we have strong expertise in certain therapeutic areas, we want to make sure that we are opportunistic in looking broadly areas of rare disease where there is unmet need and a potential for us to be able to accelerate potentially being first in class or potentially better in the case of Censa.
With every one of those decisions including Censa, we want to be very disciplined about how we manage our decisions. And we're continuing to be very active in business development regardless of what the ultimate data readout and decision will be on Censa. Noah, anything else that you would like to add on business development?
Noah L. Rosenberg -- Chief Medical Officer
Yeah, maybe I'll just go specific to your point and you talked about in terms of neurodegeneration. As we have continued in our PKAN program, we are going to be leveraging our footprint that we have commercially today, we will be able execute in that area. We are continuing to look externally disciplined to diversify the portfolio, but anywhere that we can actually see that we can add value today from our current footprint our areas both commercially and our late stage development capabilities that we want to be able to leverage.
Dae Gon Ha -- SVB Leerink -- Analyst
Great. Well, thanks for taking our questions. And congrats on all the progress.
Eric Dube -- President and Chief Executive Officer
Thank you.
Operator
(Operator Instructions) Our next question comes from the line of Do Kim from BMO Capital Markets. Your line is now open.
Keith Tapper -- BMO Capital Markets -- Analyst
Hi, there, and congratulation on the progress this quarter. This is Keith Tapper on for Do. I just had a quick question. You announced the enrollment completion for the FORT study. I just wanted to know if there is anything that you would be willing to comment on our in terms of what you're seeing so far either from the enrolled patients or from the compassionate used patients in terms of signs of benefit or measurable impact or anecdotal evidence from physicians or anything like that? Should we be expecting subgroup analysis? And what might that look like in terms of disease progression in context of patient age for example in the form of PKAN? And then finally or thirdly, the FORT protocol, you mentioned it was approved under the Special Protocol Assessment agreement. Is it safe to assume a similar arrangement with EMA? And then I have one more to follow up.
Eric Dube -- President and Chief Executive Officer
Okay. Thank you, Keith. I am going to turn that over to Noah to answer your first part.
Noah L. Rosenberg -- Chief Medical Officer
Yeah, so just to address, your first part of your first question. In terms of the overall what we are seeing, obviously, we are blinded to the data set. There is not much we can really say between the groups but what we can say overall is we are seeing a representative population that's tracking well with our protocol, and all the demographics based on information that we've seen to-date is in line with our expectations. It looks like we wind up with about two thirds of the study in these old cohorts with the pediatric being large enough to show an effect, if our hypothesis is correct. I just want to add, we have had a lot of dedication from patients in founding the study. We had our FORT DMC meeting between in the month that maybe rolled into your question and we got the all clear and they were unblinded to the data. So, they gave us proceed as planned so that was a very encouraging sign. In terms of the subgroup analyses, if you remember this is not a huge study, so we probably will conduct subgroup analysis pediatric result. I think when you have studies in the size you are not necessarily looking for statistical significance. You are looking for trends and some benefit in those populations. And until we have really top line results we don't anticipate any program updates there. Just in terms of spot Bill you want to handle that one.
William Rote -- Senior Vice President and Head of Research and Development
We have to respond in U.S. we don't have the similar agreement in the EU, but have alignment that on the study design and a single study to support approval, if positive. So, effectively, the same set up.
Keith Tapper -- BMO Capital Markets -- Analyst
Terrific. Okay, great. And just want to follow up on Thiola. Is there any update that you are seeing in terms of the generic entrance or competition at present? Is there anything intensifying or ramping up in terms of competition? And then, could you remind us the commercialization strategy that you are looking at in terms of transitioning patients to the new formulation? Or can you comment on recapturing the patients that would be more likely to take more patient friendly formulation that you perhaps lost because of pill burden?
Eric Dube -- President and Chief Executive Officer
So Keith, with regard to generic we have not seen anything. We obviously continued to monitor with that landscape. And we don't access to all information to predict everything that's maybe going on out there, but nothing that we see would suggest that there is a generic on the near term horizon. What I would like to do is turn it over to Neil to give a maybe a bit more detail on that and commercialization question.
Neil McFarlane -- Chief Operating Officer
Yeah. I could probably give you a little bit more flavor on that one specific to your question in regards to. We will not be creating any barriers to conversion of patients for the new friendly formulation, to our new patient friendly formulation. I think what we've done is continued to listen to patients to understand what are the needs that they are looking for to be able to better their compliance and persistency. And what we are planning to do and a partnership with our hub service providers and the others is to actually be able to go out and ensure that patients who may have discontinued product for whatever reason, have -- and their physicians we have the ability to contact those physicians and let them know that we've got a new formulation and the attributes that we've heard. So that's what we'll do. In addition to that the barriers to entry for this product, we don't plan to raise any to being able to have patients convert to the new formulation.
Keith Tapper -- BMO Capital Markets -- Analyst
Okay, perfect. That's helpful. Thank you.
Operator
Our next question comes from the line of Michelle Gilson from Canaccord Genuity. Your line is now open.
Michelle Gilson -- Canaccord Genuity -- Analyst
Hi, thanks for taking my question. I also want to offer happy birthday to Noah. I'm not as nice, so I do have questions for you.
Noah L. Rosenberg -- Chief Medical Officer
Thank you, anyway.
Michelle Gilson -- Canaccord Genuity -- Analyst
I was just hoping you could talk a little bit about the dosing of fosmetpantotenate and FORT, and how you selected this 300 TID dose? And then also a little bit on how you're managing, if you're seeing any LFT elevations in your protocol? And are you expecting patients to need to reduce their dose?
Noah L. Rosenberg -- Chief Medical Officer
Yeah, so in terms of selection of the dose, we pick the maximally tolerated dose. We do want to get the maximum benefit. I'll just correlate that back to the patients who are in compassionate use who actually saw effect even at lower doses. In terms of the LFT question, we are following -- we follow safety as you are aware very closely and specifically we are looking at any potential for increases in any lab abnormalities specifically LFTs, and we have not seen a signal or any concern with regarding to LFTs.
Michelle Gilson -- Canaccord Genuity -- Analyst
Okay. I think one of the patients in compassionate use might have gotten sick during the study?
Noah L. Rosenberg -- Chief Medical Officer
Yeah, there was the one -- yes, well, I'm referring to -- correct. There was one patient in compassionate use that saw a transient elevation to two to three folds in transaminases that was improved when the dose was cut and the patient is doing fine on the drug. So, again, that's -- we have that to your point Michelle and as usual, very diligent in picking that point up. We are following as an AEOI or adverse event of interest LFTs. And again, specifically, we are not seeing anything concerning and again we had a recent DMC meeting and everything was clear. So thanks, great question, Michelle as usual.
Michelle Gilson -- Canaccord Genuity -- Analyst
Maybe one more on PKAN diagnostics and if you're hearing I guess anecdotally from KOLs and investigators in your study, if they are seeing any uptick just I guess based on your conversation with this study now enrolling?
Eric Dube -- President and Chief Executive Officer
Neil?
Neil McFarlane -- Chief Operating Officer
Yeah, maybe I can answer some of that and you are absolutely right. The awareness of the study and some of the work that we've done with our medical affairs assets on the ground across both the U.S. and Europe have identifying more patients across than we had originally identified. And we have seen additional activity around newly diagnosed patients, as we may have mentioned on the last call, we have had a flurry of patients that came in to our clinical sites once they are aware. So, yes, it is answer to that question.
Michelle Gilson -- Canaccord Genuity -- Analyst
Okay. That's it for me. Thank you, guys.
Eric Dube -- President and Chief Executive Officer
Thank you, Michelle.
Noah L. Rosenberg -- Chief Medical Officer
Thanks, Michelle.
Operator
Our next question comes from the line of Christopher Marai from Nomura Instinet. Your line is now open.
Alan -- Nomura Instinet -- Analyst
Hi. This is Alan on for Christopher. Just one question on the PKAN. For the format of the readout or presentation maybe this year, will you be segmenting by age of onset? And can you elaborate a little more on the correlation between age of onset and disease progression and its relevance to the readout expectations? And I have got a follow-up.
Noah L. Rosenberg -- Chief Medical Officer
Yeah, so in terms of your first question -- it's Noah here. In terms of the actual readout, at a minimum, we expect to communicate the study that is primary endpoint in any key safety aspect. To know outside of that, it will really depend on the timing and relevant information we provide while retaining ability to get that data, presented at an appropriate medical meeting. So, I think that was the question around the top line data.
And in terms of predictions, there is an understanding that there are two populations. One is pediatric and then other age of onset pediatric potentially may move a little more quickly, but I don't think that's so well understood in terms of what the actual drug effect would be. So, I don't know if that's clear. And I think we have a population with again two-thirds of those are, third pediatric, so a fairly nice sampling patients well represented. So, that is much as I can say.
Alan -- Nomura Instinet -- Analyst
Got it, thanks. And how is the patient or physician interest on compassionate use for the asset?
Noah L. Rosenberg -- Chief Medical Officer
It's very high. We are obviously just in the stage now where we are moving patients over from the FORT into the long-term open-label, and we are working very hard internally to do our best to get a compassionate use program up and running, but there's definitely a high level of interest.
Alan -- Nomura Instinet -- Analyst
Great. Thanks for the color. Congrats, and happy birthday to you Noah.
Noah L. Rosenberg -- Chief Medical Officer
Thank you.
Operator
(Operator Instructions) Our next question comes from the line of Liisa Bayko from JMP Securities. Your line is now open.
Liisa Bayko -- JMP Securities -- Analyst
Hi. The vast majority of my questions have been answered, but just one quick new one. For the Censa program, how could we expect that data to be communicated? Will you share a full data set with us? Or just tell us that it's a go-no-go? Or what level of disclosure should we expect around that?
Eric Dube -- President and Chief Executive Officer
Thank you, Liisa. I'll ask Bill to cover that.
William Rote -- Senior Vice President and Head of Research and Development
Certainly, as you know, this is a partnered program and we will be working with our partners Censa Pharmaceuticals to determine where will we presenting -- where the data will be presented. So, we need to get to the point where we have top line data evaluate that, and then make an underlying decision around that. We don't have -- we haven't had that discussion yet or we haven't had its completion. And when we do have that nailed down, we will let you know.
Liisa Bayko -- JMP Securities -- Analyst
Very good, thanks.
Operator
(Operator Instructions) There are no further questions at this time. I would like to turn the conference over to Mr. Chris Cline.
Chris Cline -- Vice President, Investor Relations & Corporate Communications
Great. Thank you, Tom, and thank you everybody for joining us today for our call. We look forward to updating you in the near future on our progress.
Operator
Ladies and gentlemen, this concludes today's conference. Thank you for your participation. Have a wonderful day. You may now disconnect.
Duration: 48 minutes
Call participants:
Chris Cline -- Vice President, Investor Relations & Corporate Communications
Eric Dube -- President and Chief Executive Officer
Noah L. Rosenberg -- Chief Medical Officer
Laura M. Clague -- Chief Financial Officer
Tim Lugo -- William Blair & Co. -- Analyst
Maury Raycroft -- Jefferies -- Analyst
William Rote -- Senior Vice President and Head of Research and Development
Dae Gon Ha -- SVB Leerink -- Analyst
Neil McFarlane -- Chief Operating Officer
Keith Tapper -- BMO Capital Markets -- Analyst
Michelle Gilson -- Canaccord Genuity -- Analyst
Alan -- Nomura Instinet -- Analyst
Liisa Bayko -- JMP Securities -- Analyst
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