Q3 2021 Mirum Pharmaceuticals Inc Earnings Call

Hello, everyone and welcome to the Marathon Pharmaceuticals third quarter business update conference call. My name is Victoria and I'll be coordinating your call today.

We would like to ask a question during the presentation you might do side by pressing star followed by one in your telephone keypad. If you have joined US online. Please press the Red flag icon I'll now have NEVA Jia Hi, Ian Clemens Chief Financial Officer Ann. Please go ahead.

Thank you Julia and good afternoon, everyone.

I'd like to welcome you to minimum Pharmaceuticals third quarter 2021 conference call.

I'm joined today by our President and CEO, Chris Peetz Palm Bay had a research and development and Peter Roberts Chief operating officer.

Earlier this afternoon <unk> issued a news release announcing the Companys results for the quarter ended September 32021.

Copies of this news release and SEC filings can be found in the investors section of our website.

Before we begin I'd like to remind you that during the course of this conference call, we will be making certain forward looking statements about Maryland.

Progress on our programs based on our management's current expectations.

Any statements regarding managed business firms commercial and development programs strategies prospects market opportunities and financial forecasts and guidance.

These statements are subject to numerous risks and uncertainties and actual results could differ materially from the results anticipated by these statements.

Investors should read the risk factors set forth in <unk> 10-Q for the quarter ended September 32021, and any subsequent reports filed with the SEC.

With that said I'd like to turn the call over to Kristy Chris.

Thank you Ian and good afternoon to everyone joining us on the call today.

Third quarter was an exceptional period for <unk> marked by the FDA approval of them early for close to <unk> patients one year and older with <unk> syndrome.

The first ever approved medicine in this indication in the first FDA approved product for mirror.

<unk> launched a tremendous achievement and I'm very proud of our team for the impressive strides made this quarter.

We are all humbled and excited about what it means to be ours, You'll center community now I'd like to again extend my thanks and appreciation to the patients parents.

Caregivers investigators advocates and employees that got it done.

Hard work of the Mira team is delivering results.

Our prelaunch preparations have resulted in a strong start in market.

Peter will speak to our commercial efforts, including an update on the many partner companies multiplying our efforts to launch live barley around the world.

This quarter also witnessed several important milestones from our clinical and regulatory efforts we submitted.

Our application for <unk> in Europe for the treatment of course static liver disease in patients with ALS Youll syndrome.

And just today at the a S. R. The liver meeting the global hours. Your Alliance presented landmark data that was part of that submission.

These data showed a highly significant improvement in transplant free survival for patients and let them early studies compared to a similar untreated group.

Overall, 70% risk reduction was saying.

This presentation was the lead oral late breaking presentation today and received best of liver meeting a recognition. We believe these results are nothing short of remarkable.

Yeah.

Before turning the call over to Peter I'd like to share a couple of updates on the leadership team first I would like to thank Dr. Ed Tucker, who has served as CFO since 2019, supporting the application and approval uplift barley twist, leaving the company.

We wish him the best of luck as he embarks on his next professional endeavor.

Second I am excited to share that our chief Scientific officer, Dr. Pam Big is being promoted to head of research and development for.

She will review today is there'll be a presentation on our call today.

Now, let's turn to an update on the launch it's early days, but we're confident in the initial progress we've seen Peter.

Thanks, Chris.

Over the last 18 months, we have been quite busy building a world class commercial team.

And conducting a lot of work to prepare for the launch of lip Marlin.

It's clear that these efforts have paid off as the live Marley commercial launch is off to a strong start.

Today I'm excited to share early insights into our achievements following just over a month of commercial availability.

Given that the FDA approval of loop Marley occurred on September 29.

We're not providing specific revenue or associated commercial metrics in this third quarter earnings report as there was no product revenue recognized in the third quarter.

We will provide an update on Q4 results early next year.

That being said, we're very pleased with what has been a great start in the early weeks of commercialization.

From an access perspective, we've seen faster than expected acceptance by payers.

Previously communicated that we expected the majority of allergy will syndrome patients to have a reimbursable pathway sometime in the first half of 2022, which was based on the observed loss performance of several comparable rare disease medicines.

As of today, the majority of allergy all syndrome patients in the United States have a reimbursable pathway.

Leading our access goal by several months.

For example, the three largest commercial payers in the United States have Reimbursable pathways established with claims approved for each major player on.

On the Medicaid front, we signed our CMS rebate agreement and several states have added live Marley as a reimbursed product and their systems.

We have also been very pleased by the receptivity to the Marley by health care professionals.

Our field team has reached all of our top 125 accounts in October and we have seen prescribing from our broad base of accounts and physicians today, ranging from the top programs in the country to smaller community based centers.

Feedback from patients and caregivers as well as prescribers on the Miram access plus program or map has been very positive.

The goal of the goals of map include ensuring that patients have timely access to look marley once prescribed as well as minimizing any financial barriers.

I am pleased to say that map is delivering according to our expectations as we've seen timely defenses in the early weeks of launch.

Turning to international this past quarter, we announced that we've entered into a development and commercialization agreement with Takeda for lip Marley in Japan, leveraging their expertise in rare disease as well as Gi herpetology.

Beyond Japan, we've entered into licensing or distribution deals for live Marley and each of greater China, South Korea, Israel, the Middle East, Russia, and the Baltics, and finally central and Eastern Europe.

All of our commercialization partners in these geographies, bringing a tremendous depth of local knowledge and rare disease expertise, which nicely complements the capabilities of mirrors, one commercial team in the U S and Europe.

We are very grateful to have such a strong and dedicated group of partners working towards ensuring as many patients as possible can benefit from lip Bartlett.

Partnering discussions for additional geographies are ongoing and we'll keep you updated on our progress there.

There are tremendous opportunities ahead, both from a commercial standpoint, but also across our development programs and we'll share more about our recent data and provide an update on our pipeline progress.

Thanks Peter.

And supported with violates approval, we remain committed to ongoing research and analysis to continue to just further validate this model its utility in this debate.

To that end, we're so excited by the data presented today at the S. There'll be annual meeting in a late breaking oral presentation, demonstrating significant improvement in the net free survival with mid mildly compared to natural history control cohort.

This is a landmark data set for Allergist center and the first time in interventional drug has demonstrated improved event free and transplant free survival in this devastating disease.

The presentation is available in the publication section of our website and I will now share a few highlights with you.

For patients with <unk> syndrome, theoretically a leading indication for liver transplantation and the prognosis in this setting is quite troubling with transplant free survival among patients with <unk> syndrome of only 41% by 18 and a half years of age.

In the presentation today evaluated six years of follow up from the program Alex about studies analogy I'll syndrome, keeping in mind that this is the largest intervention dataset available in this rare disease and.

And compared it against the <unk> natural history database, which includes over 600 patients the largest natural history control cohort established for Ala Joseph.

The goal of this assessment was to compare time to first clinical events between the two groups.

The results demonstrated a striking 70% overall reduction for clinical outcomes with lyft mildly treatment and a statistically significant improvement in six year event free survival with a P value of less than 0.0001, and a hazard ratio of 0.3 or five.

In addition to event free survival. The analysis showed statistically significant improvements in transplant free survival, which evaluates transplantation and death only.

And this also demonstrated a P value of lessons 0.0001, and a hazard ratio of 0.32.

Multiple sensitivity in subgroup analyses were conducted to test the robustness of the data and consistent findings were observed across seasonality.

These data have been included in our submission to the European Medicines agency for live Miley for the treatment of Cholesteric liver disease due to our gel syndrome, and we expect a decision from the EMA in the second half of 2022.

Now in its second late breaking abstract we also analyzed event free survival. In this analysis was presented by Dr. Ron So Paul.

This work demonstrated that bilirubin serum bile acids, and pruritus, where predictors of event free and transplant free survival.

<unk> of 76 patients remained event free at the time of this analysis with up to six years of treatment.

And critically the improvement of pruritus being predictive of event free survival supports live I'm always impact on long term outcomes and these data may be helpful to inform medical management for patients treated with a mileage.

Lastly on the publications front, we're very excited that the lancet published our pivotal iconic study following four years of treatment with live Marley inpatients with allergen syndrome.

Now outside of volatile Center, Maryland, smell elixir that pipeline continues to progress with respect to <unk>, we expect to announce topline data from the phase III <unk> study the second quarter of 2022.

As a reminder, the March <unk> study enrolled all <unk> subtypes. Additionally.

Additionally, enrollment continues to gain momentum in the embark phase <unk> study of <unk> in biliary atresia.

Now a brief update on the looks about in the adult study.

We're committed to continue expanding the potential role of I bet inhibition across cholestasis.

Our religious about program builds on the learnings from our pediatric program and the adaptive designs potentially registrational bullets about studies are currently underway. As a reminder, we have launched a phase <unk> study to evaluate but looks about in adults with primary sclerosing cholangitis.

And the phase two be a hollow study to evaluate and look for that in intrahepatic cholestasis of pregnancy.

We expect interim analysis for both of these studies in 2022 and I'm excited to share that the vantage primary biliary Cholangitis study is active in currently screening patients.

But all of that said, we have a clear vision of <unk> clinical programs and we're very excited about the future of these indications.

And on that note I will turn the call over to Ian to provide an update on our financial health Ian.

Thanks, Tom.

Currency for a review of our quarterly financials is available in the press release distributed earlier this afternoon.

Form 10-Q filed with the SEC you can find both in the investors section of our corporate website at minimum pharma Dot com.

I'll provide an overview of what we believe to be the highlights from the third quarter of 2021, all of which position us well to support our current launch of live Molly and our further development pipeline for both <unk> and <unk> and their respective indications.

First from a revenue perspective, we received an upfront license payment of $5 million from GCE pharma under.

Under this exclusive licensing agreement with <unk> pharma has agreed to develop and commercialize merrell looks about in South Korea.

Operating expenses for the quarter ended September 32021, G&A expenses were $17 4 million.

G&A investments increases in the third quarter of 2021 versus the third quarter of 2020 with primarily due to increased personnel and operational costs associated with the launch of this morning, as well as expenses related to general legal and public relation activities.

R&D expenses were $35 million as compared to $16 million for the third quarter of 2020.

The increase was driven by the following collaboration program funding increases related to Alex about clinical trial expenses for PSC, PVC and ICP as well as related manufacturing activity supporting clinical supply and increases in personnel and other compensation related expenses.

Merrell remains well funded and at the close of the third quarter ended September 32021, we had cash cash equivalents and investments of $205 million.

In connection with the FDA approval of live Marty we have received a priority review voucher.

Okay, Avi, we intend to monetize the voucher and along with this expected additional cash the company has more than three years at runway.

Of note our financing arrangement with Oberland capital allows an additional $35 million of financing upon the approval of live Marty.

Given the strength of our financial position and projected financial performance of the business, we have decided to forego this tranche of the structure.

So with that update I'll turn the call over to Chris for any final comments Chris.

Great. Thanks, Dan and thank you to everyone who joined today.

To close Byram has made great strides as a company this quarter as we continued to advance the treatment of devastating liver diseases.

Our launch is off to a great start thanks to the hard work, we put into commercialization effort efforts ahead of the launch.

The excitement for our launch really stems from the convincing data and excitement of treating physicians to have a new medicine for patients living with this terrible disease.

And the data presented today shows the great potential we see in with Marley.

Before I close I wanted to briefly recap our upcoming milestones.

Our eligible syndrome application is under review in Europe, and we're preparing for a potential launch in the second half of 2022.

We plan to share top line data from the Phase III <unk> Chief X study of Merrill Lynch and <unk> in the second quarter of next year.

And Ford Melissa that are potentially Registrational program in adult cholestasis expect interim analyses.

<unk> Opana and Vista studies in Intrahepatic cholestasis of pregnancy, and primary sclerosing Cholangitis next year.

We are thrilled with our progress and the recent launch of Love Marley, We look forward to updating on continued achievements as we head into 2022.

Thank you again for joining US operator, please open the line for questions.

Great. Thank you.

On to the Q&A session, if you'd like to register a question. Please press star followed by one of your telephone keypad. If you change your mind. Please press star followed by Jim.

Those who have joined US online please press the red flag icon.

And our first question comes from Jessica <unk> from J P. Morgan Jessica. Please go ahead. Your line is open.

Hey, guys. Good afternoon, thanks for taking my questions.

First when you guys said you will provide an update on <unk> results. Early next year are you, suggesting that you might pre announced <unk> results or was that just a reference to the typical timeline when your results when you report for Q.

And second can you remind us maybe when in 'twenty two we could expect to hear more from the interims for the Bullock spot of Hana investors trials and what the communication to the street will look like for each of those.

Yes, thanks for the question.

So on start up the first one on <unk>.

The update for the fourth quarter as we get into next year.

To be.

Totally transparent about it we haven't decided yet on pre announcement versus the typical filing timelines. So that's something that we'll work through as we get through the balance of the year here.

Certainly could be something that makes sense to pre announce as we get towards conference season.

And the year.

And then on the <unk> studies.

The interim analyses for the Opana and Vista studies have a key difference that's worth highlighting as we talked about the potential for those analysis.

Towards the fourth quarter of next year.

For the this does PSC study. This is a closed interim so it will be an adaptive closed interim with predefined rules on continuing this study in selecting a dose all of this with an eye towards moving into a registrational portion of the study.

All of this discussed and aligned with FDA on how that analysis plan will work and how the study conduct will lead towards a potentially registrational dataset with that study alone for PSC.

The O'hara ICP study will.

Conductus interim a little bit differently. It will be an open interim and we'll have data at least headline data from that interim too to Sharon announce once we conduct that analysis.

Great. Thank you.

Great. Thank you Jessica.

And our next question comes from Mani <unk> from SVP Leerink. Please go ahead.

Hey, guys. Thanks for taking the question I apologize for any background noise Im standing on the Street in New York City at the moment, maybe on the military.

You made some comments on the majority of patients have a path to reimbursement.

Actually around.

How to think about discounting.

Should we think about the scale of where discounting fall was looking similar to the Medicaid minimums.

So look more like a commercial rare disease assets or is this the kind of thing or is this or.

Or is this an asset for which discounting is likely to be a really small part of the story.

Going to transplant centers specifically.

Hi, and thanks for the question Danielle I'll look to Peter to give some color on our expectations. There yes. Thanks for the question Manny.

It's really the answer is it really more the latter of your comments there.

The discounting here that you should expect is really in line with the statutory requirements to participate in Medicaid program.

The vast majority of what will drive the gross to net there is no strategy beyond that internally.

Great. Thanks.

Follow up okay.

But it <unk> reimbursement.

How should we think about that.

So we anticipate that there will be some gap from patient first diagnosis.

Through presumably a hub.

To receive them and commercialize the drug and you should be thinking about and looking kind of like the one to three months.

The world.

Hello, This photo a little bit longer just how should we be thinking about the delayed.

What's the reimbursement reimbursable value.

Yes, yes. Thanks for the question on that and we are really pleased with how the map program has performed so far and you did get the correct steps in the process of course Theres a discharge of the diagnosis in the case of ALS Youll syndrome. The best a lot of the patients are already diagnosed and then a prescription and then fill.

And what I can say at this point is we're really pleased with the timelines we've seen so far between prescription and Phil and that is one of the key goals of the map program both to get patients access to the medicine in a timely basis.

So to support the providers.

Terms of their goals.

And just just kind of an anecdote out there kind of in the early early days and we have seen we have seen is variable there is different different timelines associated different payers, but we have seen dispenses occur as quickly as you know a handful of days after receiving a complete.

Enrollment form and paperwork.

Great. Thanks, guys. Congrats again on all the progress.

Thanks Bennett.

Great. Thank you Mani and.

Our next question comes from Josh <unk> from Evercore ISI. Please go ahead.

Great. Thanks, so much for taking the questions maybe first on the <unk>.

Study I guess since you've indicated that this could be a registration enabling trial, what what is it that you ultimately need to do.

Delivered to the FDA in terms of our registration package that may also.

Yes.

Consider pregnancy outcomes.

Thanks, Josh for the question yes.

Overall, the interaction with FDA has some of the some of them are other indications has focused on pruritus. So for this setting we do expect the endpoint for approval with FDA to be improvement in pruritus.

Study looked at much more so I'll actually pass it over to Pam to talk through some of the other endpoints and implications we see across the outcomes potential yeah. Thanks. Thanks for the question Josh.

For ICP, just a little bit of background that you're probably already aware of that these women present in the late second trimester of third trimester of pregnancy.

Often significant pruritus elevated serum bile acids.

And we know that when serum bile acids are about 40 and above 100 that you're at where these women are at risk of having iatrogenic delivery are spontaneous preterm birth and also in very extreme cases stillbirth Theres also consider so we'll look at all of the endpoints that are associated with poor.

It'll outcomes, including length of hospitalization stays et cetera. So all of those are in the secondary part of the key analysis that we're conducting and will be part of our filing.

Just just as a follow up to that what are what are the indications for early delivery and as you think about I.

I guess extending that gestation will chime.

Serum bile acids haven't been normalized.

Concerned that the longer.

Time to in gestation could have.

Some kind of adverse outcome on the fetus because of prolonged exposure to maybe not normal but potentially still elevated.

Yes, so we anticipate wind bullish about as with our experience with <unk> that that there is a significant reduction in serum bile acids that you see in colas static liver disease and this would expect it to be no different so you'd see reductions hopefully in serum bile acid and that helps inform.

Medical decisions.

On leaving the baby in longer and maybe not having a iatrogenic delivery and lower serum bile acids.

Also are helpful for.

Preterm spontaneous forests.

That when kids are born early that in premature children.

There is a lot of risks associated with that in and of itself. So keeping the baby and as long as possible is a goal.

And Laurie Stern bile acids, hopefully will help that.

Got it helpful. And then maybe a couple of quick questions on the merge P. FIC program now that there is a competitor drug approved in a number of markets is there any.

Risk or concern that you may lose some patients from that trial onto an approved therapy and given that there is an approved therapy.

What are you looking for in the in the Phase III results to continue to advance two filings does it doesn't need to be kind of numerically better.

A comparable dataset worth advancing and if so why.

And thanks for the question there.

From this study conduct standpoints.

So far what we're seeing there is no real meaningful impact on the overall study conduct and worth noting that we're it's an international study. So it is actually in a number of countries some of which may not have access to.

The approved newly approved agent.

At this point.

So with.

Many of these patients having enrolled over the prior time periods. They have actually some of them already onto the open label extension as well, where there's just less of an issue.

And in terms of the competitive profile for Lyft Marley and FIFA and also an allergy Olsen as well one of the big advantages. We have in our program is the longevity of the data and the ability to present data like we did earlier today.

Six year event free survival impact of Love Marley in these indications and we have some data that's been previously presented in <unk> showing similar findings in the piece of it too.

So that gets paired with results from the March Chief X study next year.

Also optimistic that we will see.

And improve response rate because of the increased dose that's involved in the ongoing study.

Okay. Thanks very much.

Great. Thank you Jeff for your question and we will now move on to Ed Arce from H C. Wainwright. Please go ahead.

Great. Thank you for taking my questions and congrats on all the recent progress including.

Today's presentation.

Wanted to start with that.

Just given.

No.

Really long.

The long effects that have now been shown.

With Marley.

How does that infer.

Inform discussions with Payors have you.

<unk>.

Have you seen hurdles.

Payers put in place.

Perhaps less than you had thought.

Maybe discuss that and have you seen any.

Declamations, yet I recognize it's very early still and a lot of them are still going through it but just wondering.

The overall sense of the payer engagement so far in the launch.

Thanks for the question.

I'll, let peter jump into that one.

Thanks for the question Ed.

With regards to the U S and then I'll pivot to Europe I think there is an important point there as well.

We invested early in our field team was out engaging with payers back in the spring.

And kind.

For many payers. This is the first time, they even heard of <unk> syndrome are they knew very little about it. So I think they've really come to appreciate what is a very high unmet medical need and that these children's children really are robbed of their childhoods.

And the safety and efficacy profile of look Marley.

As has been presented and the recent lancet publication with long term follow up really really quite persuasive to them.

And we think Thats part of what we've seen when we updated you on today or in terms of earlier than anticipated.

Reimbursable pathways.

And with regards to the late breaking abstract today in the best of ASR. The natural history comparison, the gala that actually is part of our European package and we've had a number of interactions with European payers and it's just really exciting to have that kind of data as part of the package and if the initial.

HPA health technology assessment stages.

To build a long term outcomes compared to natural history cohorts. So those those data quite frankly played very well in those conversations.

And we're excited about the European launches in the second half of next year.

Okay, Great and then just one.

One follow up.

Regarding advantage.

I know you just started screening patients here, but.

Given that there are a couple other competitive.

Programs in late stage development here wondering.

What your overall impression is.

The bar.

In terms of.

Not necessarily approval, which is well known but more about commercial acceptance in the competitive landscape. There recognize its early but just wanted to get your thoughts.

Thanks for that question Ed.

This is actually there is important.

The element of the strategy for vantage that is worth highlighting in this and the competitive set in PVC.

Youre alluding to there is.

Some competitive activity in studies for kind of a second or third line setting of PBC with agents that are focused on alkaline phosphatase as a primary endpoint for use as a surrogate marker for approval.

And our approach for <unk> and PVC is quite different so we are not using alkaline phosphatase as the primary endpoint, but using dried us again as the outcome.

For approval and the primary endpoint for the study in which.

Which we expect to have the possibility of being Registrational in the second portion of the study.

In terms of what that means from a competitive dynamic and where the looks about can fit in the treatment paradigm.

It really is line of therapy agnostic Triton can occur in PBC.

With.

Lower or elevated alkaline phosphatase levels, so think of it as a first or second line agents.

That can be kind of on a parallel track to some of the other competitive trials that are in kind of the second or third line setting.

Okay. That's helpful. Thank you.

Great. Thank you for your question, we will now move to Yasmin Rahimi from Piper Sandler.

Please go ahead your line is open.

Hey, everyone. It's Dave on the line for your ethylene and thanks for taking my question.

So on a percentage basis I was hoping you could shed some color on what percent of the 120 key accounts I would cover 80% of lgs lives.

So what percent of that have have an awareness of Marley and have started to write scripts and I have follow up questions after that.

Yeah. Thanks for the question there is no as we mentioned.

It's very early days to provide specific metrics, but.

Peter can speak to some of the color of what we're seeing out there yeah. Thanks, Chris and we've interacted with all of the $1 25.

In October.

And have been able different ways has been able to kind of gain access and raise awareness. So.

It's not a large universe out there and pleased that we've been able to get out there.

And in terms of.

Where the prescriptions are coming from I think it's early days here six weeks into launch you get into specific quantification other than to say, we're really encouraged by the sort of broad uptake we've seen from both the top centers in the country, but also the smaller regional accounts. So I think thats a good sign for where things stand and look forward to updating you.

More as we move through the launch process.

Great. Thanks for the additional color and then if we could switch gears to the abstract here with regard to the late breaking abstract presented at a S. L D. How similar worthy baseline characteristics between the patients on the Marley.

Compared to the Gallup cohort and what I've heard what excuse me what efforts are being made to use this data to educate treating physicians and how do you think that that'll that'll impact adoption like down the road.

Yeah. Thanks for the question I'll start off and then with regard to adoption I'll hand over to Peter for that.

So we're just really excited about this analysis, it's been a it's been a labor of you can imagine six years of.

Involvement from these patients and physician community to get to this database and a lot of effort by the Gala natural history study group.

And it's now since we compared with Marley to natural history Gala control cohort in the way that we ensure that there was similarity between baseline characteristics look through a prespecified very rigorous selection process in which.

Outcomes were blinded and actually.

The all of the robust methodologies, including sensitivity and subgroups were included to try to.

Ensure robustness statistical robustness of the clinical efficacy.

The cohort selection once the cohort was selected and this is based on using key entry criteria from the mileage that study.

To try to get our representative in appropriate control cohorts.

There is an assessment of comparability across the two.

The two groups and this showed that there were similar.

Disease severity between the two groups. This is looking at alkaline phosphatase. So L. T. L. P. S T GTT bilirubin.

Et cetera, and we found that both of these.

We're well aligned.

There were sensitivities on potential variable. So this is where we looked at adjustments at baseline and this included everything from Iot Bilirubin HGT sexier of birth et cetera. All of these were analyzed using different adjustment analyses.

I think it's important to note that we found that there were no statistical differences between the two groups at baseline with the exception of serum bile acid, where it was.

Higher in the Merrell looks about treatment group and I think also important to note that SBA is not commonly collected in the real world.

<unk> for the subset of these data.

But we ran subgroup analyses on that as well as several other.

Components using different index times.

When do you start the clock for the Gallup cohort.

Looking at transplant free survival and also pruning analysis to adjust for a model time bias. So all taken all of this together.

Pre specified analysis that I, just mentioned with all of these sensitivities and subgroup analysis really demonstrate the robustness of the data was highly statistically significant improvements in event pre and transplant free survival between the two cohorts.

Great. Thanks.

Oh yeah.

Sure and I'll hand to sorry go ahead.

Alright.

I'll hand to Peter for that did you have another question for me.

Well I did have a follow up question, but I mean.

I'm going to go bad or appreciate over yes, yes.

So my final question here and this actually provides a sort of a good segue that how representative in your view is the gala database to the real world setting and where there any subset analysis that you can discuss some of the data where either liver transplantation or ability of biliary diversion surgery or decompensation.

Events, perhaps patients of those that did they have a greater event, Oh, sorry, greater benefit over others with <unk>.

Yeah. So I think there is a.

Really we had another late breaker presentation, which was a poster and the first author is wrong so call.

This really goes hand in hand nicely with the gala natural history comparison and shows predictors of event free survival. So we looked at our data.

And try to identify what are the predictors of event free survival and transplant free survival and we found I think importantly for this discussion that improvement in pruritus.

One point improvement in credit specifically was predictive of that Fran transplant free survival were 88% of patients who had that improvement.

It remains of that freight and truck spot rate.

Great I appreciate your time and I appreciate you answering all my questions. Thanks, a lot.

Yeah, Thanks for the questions.

Great. Thank you very much.

And our next question comes from Brian Corning.

Oh, sorry.

Our next question comes from Steve seed House from Raymond James. Please go ahead.

I know this is Ryan deschner on for Steve <unk>.

Just wanted to ask.

How many genetic subtypes will be reported on in the March topline readout and then I have a follow up question.

Yes. Thanks for the question there. The March <unk> study includes really all genetic subtypes of <unk> and we do see.

Have seen broad representation across multiple subtypes in the study so randomized placebo controlled data for all <unk> subtypes included in the study that we'll be able to report on.

Yes.

Okay terrific.

Then also I was wondering if you can give us a little more detail on what your expectations for heightened weight Z score or in the phase III March study there were some competitor data out at <unk>.

That was interesting.

Yes, thanks for the follow up on that I'll pass over to Pam to share some of what we've seen in other studies, but worth noting that.

At six months in.

We wouldn't expect to see the full effects and some of this happens over time periods longer than just that first six months. So we do have an open label extension is part of the study that we will continue to gather some of that data on.

Yeah, I think so.

Yeah happy to speak about this for a second so in our indigo phase two study.

Presented data.

On patients who had a serum bile acid response.

And those patients who have a stern bile acid response, and we know that this is predictive of transplant free survival. This was shown by <unk>, which is the natural history control that natural history registry for <unk> and when we applied those stern bile acid threat.

Thresholds to our study we saw a 100% transplant free survival in patients who have serum bile acid responder.

And in those patients the growth data.

Was statistically significant.

As was.

L T.

Asps.

And peak quality of life and of course, ICP, scoring also pruritus recourse so across a broad base.

Improvement in clinical parameters, including growth as you're stating in patients who have serum bile acid control until we presented five years of data on this as Chris said.

We will continue to look at the March <unk> Phase III study six months, it's early but as we know patients are continuing into an open label extension, so certainly more to come across all piece, except that Thomas.

Okay. Thank you very much.

Thanks for the question.

Great. Thank you, Steve and as a reminder, if you'd like to ask a question. Please press star followed by one of your telephone keypad.

Just a follow up I want on your telephone keypad.

Great Operator, I think we can close.

There are no further questions.

Showing no problem I'll now pass over to quest piece for final remarks.

Great and thanks again to everyone for joining today's call and for your interest in Europe.

It's an exciting time with the launch of Love Marley in today's presentation of groundbreaking data showing the potential to improve long term outcomes.

We look forward to sharing further updates as the launch progresses, thank you and goodbye.

Thank you everybody for joining today's call you may now disconnect your lines.

Yes.

[music].

Okay.

Yes.

[music].

Okay.

Yeah.

Okay.

Okay.

Okay.

Yes.

Thanks.