Q1 2021 Mirum Pharmaceuticals Inc Earnings Call
[music].
Welcome to the Miriam pharmaceutical Q1 earnings call.
My name is Reese and I will be your operator for today's call.
At this time all participants are in a listen only mode.
Later, we will conduct a question and answer session during.
During the question and answer session. If you have a question. Please press Star then one on your Touchtone phone.
I will now turn the call over to Ian Clements Ian you may begin.
Welcome to the Miram Pharmaceuticals, Q1 earnings call.
My name is Reese and I will be your operator for today's call.
At this time all participants are in a listen only mode.
Later, we will conduct a question and answer session.
During the question and answer session. If you have a question. Please press Star then one on your Touchtone phone.
I will now turn the call over to Ian Clements Ian you may begin.
Please standby as we are experiencing technical difficulties again, please standby one moment please.
Okay.
Yeah.
And further strengthening our position and piece of it we recently announced and exciting new gene therapy collaboration with the vet Therapeutics, a leader and liver directed gene therapy.
While achieving incredible clinical results with <unk> about including improvements of five year transplant free survival.
We recognize that there will be peace of patients with genetic subtypes, who will not respond to <unk> inhibition.
This was the impetus for entering into the option and license agreement with your debt.
Are there two P fixed gene therapy programs.
Our vision is that Merrill Lynch, the that will be first line treatment and piece of it.
And Dts <unk> III and <unk> eight of two will be second line treatment options for <unk>, three and <unk> two.
And those patients who do not respond to <unk> inhibition.
These programs work by addressing the root cause of the disease through correction of the defective genes that cartage condition. This is cutting edge technology and while early and it's our hope that these gene therapies could some day offer of cure for patients living with victory and peaking too.
And the collaboration.
The vast experience gene therapy team will lead work until and the submission and mirror and will lead the clinical development and commercialization and globally.
And we look forward to sharing more with USD studies progress.
Of note that net has two abstracts at the upcoming American Society for gene and cell therapy on additional VTS eight of three studies showing further preclinical proof of concept.
Now shifting back to our clinical development programs underway.
We were pleased.
He used to expand the merits of that program with the initiation of the phase III embark study and biliary atresia.
And we also significantly expanded our clinical programs for the elixir that we believe this is another potentially transformative treatment for several coal of static liver diseases.
Earlier this year, we launched our phase II study for adults with primary sclerosing cholangitis and more recently, we launched the phase II. The hottest study that will evaluate <unk> about the intra paddock cholestasis of pregnancy.
And finally of Phase III study in primary biliary cholangitis is targeted for later this year.
We've incorporated and FDA feedback into the design of all of these studies all of which we believe are potentially registrational.
We expect interim analysis from the <unk> and <unk> studies to occur in 2022.
So the growing body of evidence of Merrill Lynch, and <unk> strengthens belief and their potential transformative impact on the lives of patients with call of static liver diseases and their families and.
On that note I will now turn it over to Pierre who will provide an overview of our launch readiness activities and partnership update Peter.
Thanks, Chris with our <unk> date of September 29, just over a quarter away. We are working diligently to ensure we are ready to deliver Merrill Lynch back to the allergy of syndrome community should it be approved by the FDA or.
Our goal is to establish <unk> as the standard of care and allergy of syndrome, we believe and its opportunity to transform the management of this disease offering and early treatment option that could potentially delay or prevent the need for liver transplant, which as Chris said is based on more than six years of clinical data and this patient population.
If approved Merrill Lynch that would serve as the first approval and allergy of syndrome and will represent the only pharmacologic therapy with clinical data to support its use.
We have made great progress towards being launch ready by late summer of ahead of our participate I'd like to spend a few moments highlighting some of these key activities.
And important component of our launch strategy is insurance thorough disease awareness of allergy of syndrome by health care professionals to.
So that and we launched our allergy of syndrome disease awareness campaign. The program developed for health care providers to increase awareness of the disease educate on pathophysiology and including the link between bile acid and clinical manifestation seen and patients and to improve the times diagnosis.
Our goal is also to elevate awareness around the burden of pruritus, which is often the most troubling symptom for patients with allergy of syndrome and their families.
To further ensure of launch readiness, we have achieved several critical milestones of the prime Miriam and Merrill Lynch that for success for instance, hiring the right team is of most importance our colleagues and medical affairs, we're first to be fully on board and engaging and scientific discussions with our key pediatric liver cell.
On the commercial side, we recently completed hiring of our U S based team, including our field sales Representatives and I'm, particularly pleased with the highly skilled group, we were able to bring together, which consists of broadly experienced professionals, who have a track record of success and and rare.
And pediatric disease markets.
We also hired our payer accounts team and we are still other crucial commercial roles and market access marketing trade distribution and patient services.
Together this team of high caliber and experienced professionals and work to build the capabilities to launch <unk> and allergy of syndrome and beyond.
One hallmark of our launch readiness is the development of our patient support program, which we are calling miram access plus the goal of this program is to provide a seamless and carrying experience for patients and families addressing key educational caps for patients who are prescribed narrow the spat through the develop.
Two of the deployment of of fully dedicated staff.
We have worked closely with patients families and advocates and health care professionals. The understand the needs and concerns that are of utmost importance as I think about starting a trial on a prescription medication for allergy of syndrome.
This input has been invaluable to us and helped to establish what we hope will be a program that provide exceptional customer service and one that addresses and provides support around the issues that are really critical to families that they navigate and new diagnosis and treatment.
And all of US Youll syndrome, many patients exhibit the range of symptoms and are no strangers to the prescription medications and the associated issued the often follow.
Through our payer team our goal is to ensure broad quality payer coverage are billed per team has already begun discussions with payers as the top prelaunch and priority and to date have engaged with payers covering approximately 95% of the United States slides.
Through these conversations we will introduce and Europe.
And our focus on coal static liver disease, helping to underscore the disease burden as well as to provide key clinical detail around Merrill Lynch to accelerate the formulary reviews.
Post launch and the team's goal is to gain formulary acceptance and a line any per clinical management criteria evidence based medicine.
Turning to our plans for commercializing now and that's about it and Europe.
Earlier this year, the EMA validated our marketing application, our marketing authorization application primarily of stat for patients with <unk>, which puts us on track for a launch in early 2020 true pivot occurred.
The support the launch in Europe and globally, we are building on our team in Europe and have begun to fill key roles.
We're also actively preparing for payer dossier submission and key markets. Our strategy is to have a targeted approach focused on larger European markets first while leveraging early access programs.
More broadly our goals to extend narrow the expense availability worldwide through partnerships with distributors or out licensing opportunities and the regions outside of <unk> core operations, and the United States and Europe.
We've engaged in conversations with top rare disease companies with local expertise of a proven track record of success and commercializing treatments and high unmet need rare disease settings.
With this strategy in mind, we announced last week, our first out licensing agreement with Cambridge Pharmaceuticals of leading rare disease company based in China.
We believe Cambridge to be of strong strategic partner, who is poised to accelerate the global launch of Merrill Lynch debt, and China, Taiwan, and Hong Kong Macau based on their experience developing and commercializing medications for patients and these highly populous regions.
They will also support the development of <unk> of 1 billion of nutrition.
Yes.
We look forward to sharing more about additional geographic expansion efforts and the coming months.
And I came to mirror on last year, because of my enthusiasm and confidence and what Merrell list that is proven in the clinic and I'm thrilled to be at the forefront of delivering the first medication for the allergy of syndrome community.
I also look forward to keeping you appraised of our great progress as we near launch.
I will now turn the call over to Ian to share an update on our financial health and.
Thanks Peter.
I'm not going to do a deep dive into our quarterly financials as those are available and the press release, which we distributed the of the softening on the not form 10-Q, both of which you can view and the investors and media section of our corporate website at minimum pharma dotcom.
I will have on the provide an early view of what we believe to be the highlight the demonstrate our financial strength and position us well to support the launch of <unk> and two geographies and approved as well as embarks on development pipelines of both <unk> and other expenses.
On the balance sheet perspective at the close of the first quarter ended March 31st 2021, we had cash cash equivalents and investments of $213 1 billion.
Subsequent to the quarter and in April and we received $65 million from Oberland capital related to the acceptance of our NDA the amount of X and <unk> syndrome.
As a reminder, we also have access to up to a third of $85 million from open on capital.
Speaking of comprises of $175 million upon approval of our NDA amount of X about and how much yield syndrome and to a third of $50 million. The mutually agreed upon in licensing or acquisition opportunities.
Additionally, we anticipate receiving the rare disease priority review voucher and connection with our expected FDA approval amount of X about later this year the may provide a source of funds.
With respect to operating expenses first G&A expense.
And the quarter ended March 31st 2021 of G&A expenses were $9 $5 million and this reflects increased activity around the launch readiness, including market research disease state awareness activities and increased personnel costs to support our planned launches and approved.
Second on R&D expenses were $38 1 million.
Which included $15 million milestone to Takeda.
The $5 billion, which would of been Jude had we initiated the phase III clinical trial, and the LNG of syndrome, and $10 million associated with the acceptance of the company's NDA filing the amount it's about for the treatment of Costar pruritus in patients with allergy of syndrome.
Now turning to our recent business development activities.
Regarding our option and license agreement with the vet therapeutics.
And an upfront fee of $3 5 million euros and the additional cost sharing expense for initial development work of $5 3 million euros.
We're also contributes on the ongoing cost sharing payments on development work with potential progress payments and option payments and option exercise.
And then also mid to high single digit royalties on sales of Etfs, <unk> and <unk> if approved.
Finally, turning to our recent announced license agreement with Cambridge Pharmaceuticals and speed.
And the outlines of last week, we entered into an agreement and the Cambridge and under the terms of this licensing agreement kind of bridge is obtained the exclusive rights to develop and commercialize <unk> of apps within the greater China regions. The allergy of syndrome P thick and getting the accretion.
And exchange were entitled to receive and $11 million upfront payment R&D funding and up to $109 million per the achievements of future regulatory and commercial Malik and that milestones.
And with significant double digit tiered royalties based on product net sales.
Our strong and healthy balance sheet, and a testament to strategic investments judicious spend and planning surrounding our upcoming anticipated the launches and continued pipeline development.
And with access to approximately $400 million and capital, we expect our cash position to sustain our operations for the next three years.
With that I'll turn the call over to Chris for final comments Chris.
Thanks, Dan and thanks, again to everyone, who joined the call today.
Before we go to questions I wanted to share a few final thoughts.
We are paving the way to being the leader and the treatment of rare liver diseases with clinical programs across a wide range of indications.
We take pride and the strong body of scientific evidence that we have built including more than six years of outcomes and safety data and both <unk> syndrome and <unk>.
We had numerous clinical and commercial milestones ahead, and both the U S and Europe we.
We are confident and our ability to achieve these milestones and create a remarkable impact on the lives of patients and their families.
A quick recap of these milestones.
First we plan to launch of Merrill Lynch about for the treatment of pruritus in patients with ALS Youll syndrome, and the U S and Q3.
Our European application, primarily start and piece of two was validated and we're planning for launch in Europe and early 'twenty two of its approved.
We expect top line data from our phase III <unk> <unk> study and early 2022.
And we expect interim analysis from our two bullets about the phase III programs for ICP and PSC next year as well.
And finally and the second half of this year, we will be initiating a phase <unk> study evaluating <unk> and patients with primary biliary cholangitis.
We have built a strong team and the pipeline with tremendous potential.
We are positioned for success and meaningful outcomes for our patients and families.
And also and creating significant value for our shareholders.
Thanks, again for joining us and Peter and I are happy to take your questions.
Operator will you please remind everyone of the prompt.
Absolutely. Thank you.
We will now begin the question and answer session.
If you have the question. Please press Star then one on your Touchtone phone.
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Okay. The high level of standpoint, and at this point, we don't have kind of clear metrics that we're gonna be communicated, but what I can tell you is that there is really great interest across U S and S. U S countries and the extent of access program. So we have multiple sites open multiple.
<unk> at a number of the sites and.
So that effect is happening out there that's not something that will get updates on head of the approval.
Keep in mind that we also have some of the patients from the original mirror list of that studies and knowledgeable syndrome. Those patients also and many of them still on therapy long term and.
And kind of with that is kind of setting expectations of it and it will turn to Peter to comment a little bit on how we think about managing that transition.
To really convert some of these patients to commercial supply and upon approval.
Thanks, Chris and thanks for the question many yeah.
The.
<unk> access plus program will be in touch with those patient and I think the the first and most important point as well and sure of uninterrupted supply through the approval process and there will be in touch with them around the time of the approval I think in terms of.
Being able to comment on you know kind of precise timelines, there's of heterogeneity of payers and the U S and.
Well I think what we'd expect to see the variability on formulary coverage decisions and access decisions on the commercial side as well variability on the the the state Medicaid side. So I think as we get into the launch will be able to give you more details on on how that's going on which I think will ultimately be what drives the timing for the.
Those patients coming over to reimburse commercial product.
Great that's really helpful and I guess the other question Uhm now the worst entering into a world, where we've got very uneven uneven true pandemic status hospital volume status of et cetera.
And just look and how do you as you continue of muscle stays the case to be studied et cetera. How do you think about the geographic split of enrollment and.
And your ongoing way stay of studies, especially I T P et cetera.
And and thanks for the follow up and there are a couple of comments on what we're seeing out there as soon as you have there is some.
And.
Clearly of different impacts by country as we look at the list of that program and.
And the ability of trees of study, where we have open those up first and the U S.
Not really seeing any COVID-19 impact on sites and the U S and and meaningful way at this point.
And on some of the upcoming country opening that we have on those programs a lot of those are and and countries that the the.
The early starts frankly aren't as impacted so we're not projecting.
And any headwinds from a pandemic standpoint, as we look at the list of that and billionaire and Theresa rollout.
UK for example, being one of the key countries headdress, where there's just not as much in terms of the restrictions currently.
Great that's really helpful I'm on call back and the Kia.
Mmm.
Okay. Thanks man.
Our next question is from yes mean rahimi from the paper Sandler.
19, and this is Kathy on free yes can you hear me okay.
Yeah, I hear Ya.
Thank you for taking our questions and congrats on the progress the corner Uhm I have three and did you want and take on one and a timer me for all of them off of that line.
Oh, I think one at a time sounds high risk to try and remember all strength.
Okay cool so I'm free my first question is for the Max Pubic study you got it that and it.
And it expanded her on that day too COVID-19 Uhm can you provide us some color on the room and update hike, how many site her going on with the progress and and at one at the expected to be fully enrolled.
Yeah. Thanks for the question and I think.
Helpful and important to get into the some of the the color here and from a starting point, we have approximately 50 patients randomized currently and so we've made tremendous progress.
In terms of tracking towards the largest randomized study ever conducted and the setting and.
And we're also seeing a very broad and mix of genetic subtypes, so and that enrollment to day that includes piece of types 123 and four.
And in terms of impact by sight overtime.
It was.
Kind of evolved over the course of the pandemic so far.
And continues to be that way, depending on how the beach country has been managing.
The factors locally.
At this point, we have the rods.
Rods spread in terms of geography for sites and.
And we're getting close to where we want in terms of of mix of patient subtypes as.
And as well as the the number of piece of two patients and the study.
Well, thank you and.
And then my next question is what information is needed to kick off the face to be and PDC and the second half of the year.
Yeah, so for the the the links to the PVC study.
We recently went through kind of follow ups desk with FTA finalize are protocol and I N G.
And so at this point, we are just completing some of the operational steps for startup. So that's just around the corner frankly.
And for all of the full list of that programs, we felt and important to go meet with regulators discuss the and points. So that we could incorporate everything they wanted to see for an NDA.
So that we took a full shot on the goal the studies as potentially registrational.
Oh, Thank you and then.
My last question is we're trying to equal what day and I shall we expect to see from Ireland to that and the next and I got the two months for now.
Oh, Thanks for the question there the for Connor.
Conference abstract.
And we'll have an announcement just the head of the conference in terms of what we're presenting and.
We do have abstract and covering the mirror of list of that program and the number of of analyses I think probably what the what the real exciting analysis that I think everybody's interested and and some of the longterm event free survival analysis that we've been conducting for hours you'll syndrome.
We're on the the missed the finalizing some of that work now so it is not submitted for for easel and you can look for that later this year.
Okay and thank you so much.
As a reminder, if you have a question. Please press Star then one on your Touchtone phone.
Our next question comes from answered the roof from Guggenheim partners.
Hey, this is Paul on for that Sir Thanks for taking my question.
I have of I guess of follow up on the EAP comments earlier and sort of interest there for primarily the to that and the number of countries across the globe and can you speak on any feedback you received on the drugs and physicians and patients from that program and then maybe how that can eat the winter focus on for efforts beyond U S and major you the countries.
And I think for the the question a couple of comments to make their.
First day of the feedback has been positive. So we have heard back from and it's from.
From different channels of that's overall good experience.
More from Ah kind of of concrete next step standpoint, we on.
Are incorporating.
And the first cohort of these patients and and the update as part of our NDA process of safety data from the first set of these patients and we'll also be submitted to FDA. So of further expanding the the amount of marrow of Tibet data that gets worked and ultimately worked into the filing.
Okay, and just a quick follow up and so on the gene therapy programs on your partnership on so it was kind of where are these assets currently and preclinical down and and sort of how far away might be from the clinic.
Uhm Gray and so just to clarify.
The question on timely.
Timeline for the the vet collaboration to get into clinic and I'll, let the Peter comments, a little bit on on some of the work that's going on there and what you can expect the head.
And thanks for the question.
Yeah, the that you have a platform.
And the platform and they're working on optimizing both of the spectrum right now.
The program and keep it three is further along and we think well.
Well, it's important to note. The research is difficult to the timelines around where we think that there's a I N D and I wanted to your window for the eight O three program with eight O two program falling on.
Great. Thanks, a lot.
Thank you.
Our next question comes from Brian <unk> from Bird.
Hi, Thank you guys for taking the questions. This is Jack Allen on for Brian You mentioned your disease awareness campaign and I was wondering if you can elaborate a little bit on the color or provide a little bit more color with regards of that program and are you contribute and tiny testing programs help identify patients and develop kind of patient database and you so much.
Yeah.
Sure. It's non thanks for the question so with regards to the disease awareness campaign and me just and can provide some color on the strategy and I think one of the things, we see and and rare diseases almost universally.
One of the most important things is to promote the disease to the medical community and itself not even and not even a product because awareness outside of and can we talk about the 125 key accounts and really the top half or a quarter of those are the kind of expertise T. On the state of the usual and and ask the et cetera, but outside of that there's not a lot of of.
Wariness of the symptoms and how to make the diagnosis. The journey of of these patients go through with allergy Oh.
And I don't like the other room, so we really focus with our disease awareness campaign on trying to reach the broader community of health care professionals beyond just the top centers.
Community based pediatric Gi the ox Neonatologist and you.
And some reach and to primary care of courses of not personal.
And and so this is the the idea of here really to increase awareness of the the.
Short of times diagnosis and.
Educate on the link between the the file assets themselves and the first clinical outcomes.
So this is really kind of an opportunity for Margaret shaping the the that we took ahead of the the.
Anticipated approval and.
And now and and the second half of the question and I think those tests.
Testing testing yesterday.
Yeah, we are we have a a collaboration that.
Really led by trivia.
And and we do provide support to them and it says and the industry consortium that provide support to allow for the testing.
Program that can run and I think now they're almost up to 70 jeans and putting the G of the cause T J and how is your.
For children, but west Coast day. So so we do contribute to that and have the date of the standard payment with the the premier around that so and.
And we have seen.
That that health and.
<unk> diagnoses over the years and we've been involved and very much lines off with the epidemiology, we talked about with Algeo center of being about $2020.
Three of us.
And.
Great. Thank you.
Thanks for the question.
Our next question comes from Steve seed House from Raymond James.
Good afternoon. This is Ryan dish and on for Steve seat and.
One of the ethical a bit more about the the debate the potential assets uhm, how different wood primary and secondary and points and share and Biomarkers look for clinical V. T X eight O three program and.
And P pick three vs mayor looks bad and.
And teach it too and and his expansion into other P fix the types of possibility.
Thanks I appreciate the question. So just kind of us taking a step back and think about gene therapy, and he fixed free and Piecyk too.
The approach hairs to completely restore functionality for for the transporter and these patients that have mutations that lead to a complete or almost complete lack of function and that transporter.
So and in fact, what we're trying to do is similar to the <unk> that treatment, except for it and the settings, where like the that to be effective there's some working transport of.
Sort of what that means is for the the patients that are the most severe that's where the stream therapies will be most impactful because I actually can't efficient and just not going to be able to be.
And the effective if you have no transport of function.
So that said come back to your question on what you measure we're trying to do the same thing here.
So when you look at the levels of Sarah and buy lasted.
Other liver for amateurs, the things that we saw improve and indigo and the piece of two patients.
Alright, and that we expect to see and the March study across all piece of subtypes.
You'd be looking at some of those same measures so sir and bylaws said at the end of a primary marker of cholestasis and file transport function and then the other parameters that show.
If they improve that your impact and go over all of that for health.
Okay, and did I hear that correctly that uhm, the target timeline or the target regulatory I guess positioning for exercising the licensing option would theoretically be at the around the time of IMD for these assets.
That's correct, yeah and the.
It's address on a product of their product basis. So it is specific to the checks eight O three per piece of three P. T X eight O two so piecuch too.
And we can make a decision and all the way up through and the acceptance.
Yeah.
Okay. Thank you very much.
Thanks for the question.
There are currently no further questions and the queue.
As a reminder, please press star then one on your Touchtone phone. If you have a question we will stand by to see if anybody jumps into the queue.
And thanks operating we can go ahead, and and wrap and I'm just making it a couple of closing comments just first of all thank you to all of those who joined us today as.
As we share of this as the big ear for mirror.
Dancing, the potentially life changing therapies rare liver disease.
Our approval and lots of Merrill Lynch the bad for hours you will send the room is expected next quarter.
Our pipelines of expanded the sixth late stage indications and the gene therapy collaboration for <unk>.
And we project, our balance sheet and future access to non dilutive capital provides three plus years of runway.
And we look forward to sharing updates on the coming months. Thanks, so much.
Thank you ladies and gentlemen. This concludes today's conference. Thank you for participating you may now disconnect.
[music].
[music].
[music].
Welcome to the Miram Pharmaceuticals, Q1 earnings call.
My name is Reese and I will be your operator for today's call.
At this time all participants are in a listen only mode.
Later, we will conduct the question and answer session.
During the question and answer session. If you have the question. Please press Star then one on your Touchtone phone.
I will now turn the call over to Ian Clements and you may begin.
So what types of thanks for joining us.
I mentioned.
I'm joined today by our President and CEO, Chris <unk>, our Chief operating officer of Pizza, rather niche.
The area of the top and issued a news release announcing the company's results for the quarter ended March 31st 2021.
Copies of the Houston region.
And filings can be found and the investors section of our website.
Right.
There's always a game of I'd like to remind you that during the course of this conference call, we will be taking steps in order and statements about them and that program is based on management's current expectations and trade.
The statements regarding das business plans and development programs and strategies prospects market opportunities and financial forecasts and guidance.
These statements are subject to numerous risks and uncertainties and actual results could differ materially on the results anticipated by the statements.
Investors should read any of the rest of it should be based factors set forth in the 10-Q for the quarter ended March 31st 2021, and any subsequent reports filed with the SEC.
All of that I'd like to turn the corner of it but you at Christie's question.
Thanks, Dan.
Merit is focused on events and life changing medicines for rare liver diseases today, we'll provide updates and context around some of the exciting recent steps we've taken building bearer of the leading rare disease company.
And important time and realizing our vision.
I'll cover our regulatory and pipeline update followed by Peter on Global commercialization, and then yeah and with the financial update.
First recapping recent announcements.
Our first potential launches around the corner for Merrell looks about analogy of.
Syndrome, and the U S. A dearth of date and the third quarter.
We strengthened our IP position with the patent allowance and the U S and extend exclusivity for Merrill Lynch of back to 2040.
And we broadened our pipeline to include late stage programs and six indications and the gene therapy collaboration per feedback and entered into a partnership for Merrill Lynch and greater China to accelerate and the global watch and Merrill expense.
Today, we will talk about these update and the value we continue to build within the <unk> programs.
So let's dive in.
Merrill Lynch of that has great momentum and is on the cusp of launch if approved by the FDA.
And March our NDA was accepted for priority review by the FDA with the <unk>.
<unk> date of September 29.
We are planning to the launch ready by late summer.
The FDA also indicated that this time there are no plans for an advisory committee.
From our standpoint of regulatory review progress is on track and we've been able to accommodate all sections of today.
And if Merrill Lynch of that is approved and the U S. It would serve as the first treatment approved for patients with ALS Youll central.
Peter will share more with you about on launch readiness and the unveiling of the patient services program beyond access plus.
We are optimistic about the benefit of Merrill Lynch of that can have for patients with allergy of central.
And the iconic study, which served as our pivotal study.
Highly significant improvement at the Reits were observed with more than 80% of patients achieving a clinically meaningful reduction and itch score.
We had six years of follow up data and are seeing durable responses across all three randomized studies of Merrill Lynch that balance sheets are simple.
The program that included 86 patients and Joel.
Now were analyzing the impact of Merrill Lynch on event free survival across the studies.
Looking at times of the biliary diversion surgery, liver transplant, hepatic decompensation events or debt.
These data and basketball history of comparisons are planned to be part of our European filing per eligible syndrome anticipated next year as an indication expansion to the piece of two application.
Our piece of two application and Europe has been validated.
We are working through the review process and remain on track with our plans for launch of Merrill Lynch.
And two in Europe, and early 2022 should it be approved.
As a reminder, this filing is based on key findings from the <unk> Phase III study.
First serum bile acid response, leading to improvement in etch gross and other lipid parameters.
That's followed by improvements and transplant free survival and dose zero balance of responders.
And third our filing includes comparisons to the natural history cohort on event free survival.
Turning to our phase III <unk> study the.
And the study is advancing well with approximately 50 patients with <unk> sub types 123, and four randomized across two cohorts.
Some countries continue to be impacted by COVID-19, and competitive enrollment. So we now plan to extend the enrollment in order to achieve the target number of patients per cohort.
This study is projected to be the largest randomized study ever conducted and piece of it.
And includes a broad range of genetic subtypes.
As a reminder, March pizza could evaluate the higher dose of Merrill Lynch of about that has been shown to drive greater bile acid clearing activity.
And importantly, we remain on track to report top line results in early 2022.
And further strengthening our position and piece of it we recently announced and exciting new gene therapy collaboration with the vet Therapeutics, a leader and liver directed gene therapy.
While achieving incredible clinical results with Merrill Lynch, the bat, including improvements of five year transplant free survival.
We recognize that there will be peace of patients with genetic subtypes, who will not respond to the SPG inhibition.
This was the impetus for entering into the option and license agreement with your debt for.
Are there two of <unk> gene therapy programs.
Our vision is that Merrill Lynch of that will be first line treatment and piece of it.
And Dts <unk> III and PT X eight of two will be second line treatment options for piece of <unk> III and <unk>.
And those patients who do not respond to SPG and efficient.
These programs work by addressing the root cause of the disease through correction of the defective gene that causes condition. This is cutting edge technology and while early the dark hope that these gene therapies get some day offer of cure for patients living with <unk>.
And the collaboration.
The vast experience gene therapy team will lead work until I and the submission and merit and will lead the clinical development and commercialization of globally.
We look forward to sharing more with USD studies progress.
Of note the debt at two abstracts at the upcoming American Society for gene therapy on additional Dts eight of the three studies showing further preclinical proof of concept.
Now shifting back to our clinical development programs underway.
We were pleased to expand the violence of that program with the initiation of the phase III embark study and biliary atresia.
And we also significantly expanded our clinical programs for the likes of that we believe this is another potentially transformative treatment for several cola static liver diseases.
Earlier this year, we launched our safety of the study for adults with the primary sclerosing cholangitis and more recently, we launched the phase two of the hottest study that will evaluate the likes about intra Abbott close statements of pregnancy.
And finally of Phase III study in primary biliary cholangitis is targeted for later this year.
We've incorporated the FDA feedback into the design of all of these studies all of which we believe are potentially registrational.
We expect interim analyses for the Vista and the harvest studies to occur in 2022.
So the growing body of evidence of the Maryland, and the likes that strengthens belief and their potential transformative impact on the lives of patients with call of static liver diseases and their families on.
I will now turn it over to Peter who will provide an overview of our launch readiness activities and partnership update Peter.
Thanks, Chris with our <unk> date of September 29, just over a quarter away. We are working diligently to ensure we are ready to deliver of Merrill Lynch back to the allergy of syndrome and community should it be approved by the FDA.
Our goal is to establish the outlets at the standard of care and <unk> syndrome.
We believe and its opportunity to transform the management of the disease offering and early treatment option that could potentially delay.
And the need for liver transplant, which as Chris said, it's based on more than six years of clinical data and this patient population.
If approved Merrill Lynch that would serve as the first approval and allergy of syndrome and will represent the only pharmacologic therapy with the clinical data to support its use.
We have made great progress toward being launch ready by late summer of ahead of our pictures of the dates.
Like to spend a few moments highlighting some of these key activities.
And important component of our launch strategy is insurance thorough disease awareness of allergy of syndrome by health care professionals to that and we launched our allergy of syndrome disease awareness campaign. The program developed for health care providers to increase awareness of the disease educate on pathophysiology of putting the link between.
Bile acid and clinical manifestation seen and patients and to improve the types of diagnosis.
Our goal is also to elevate awareness around the burden of pruritus, which is often the most troubling symptom for patients with allergy of syndrome and that balance.
To further ensure of launch readiness, we have achieved several critical milestones of the prime Marilyn and Merrill Lynch that for success for instance, hiring the right team is most importance our colleagues and medical affairs, we're first to be fully on board and engaging and scientific discussions with our key pediatric liver centers.
On the commercial side, we recently completed.
<unk> and hiring of our U S based team, including our field sales representatives and the.
Particularly pleased with the highly skilled group, we were able to bring together, which consists of broadly experienced professionals, who have a track record of success and and rare and pediatric disease markets.
We also hired our payer accounts team and we are still other crucial commercial roles and market access marketing trade distribution and patient services.
The other this team of high caliber and experienced professionals have worked to build the capabilities to launch Merrill Lynch debt and allergy Olson and beyond.
One hallmark of our launch readiness and the development of our patient support program, which we're calling mirror of access plus the goal of this program is to provide a seamless and carrying experience for patients and families addressing key educational caps for patients who are prescribed narrow the spread to the developer.
And the deployment of a fully dedicated staff.
We have worked closely with patients families and advocates and health care professionals the understand the needs and concerns that are of utmost importance as they think about starting the trial on of prescription medications for allergy of central.
This and put has been and valuable to us and helped to establish what we hope will be a program that provide exceptional customer service and one that addresses and provided support around the issues that are really critical to families as they navigate the new diagnosis and treatment.
And the allergy Olsen and many patients exhibit a range of symptoms and are no strangers to the prescription medications and the associated issued the often volatile.
Through our payer team our goal is to ensure broad quality payer coverage of our field per team has already begun discussions with payers at the top prelaunch and priority and to date have engaged with payers covering approximately 95% of the United States slides.
Through these conversations we will introduce the air.
And our focus on coal status liver disease, and helping to underscore the disease burden as well as required and key clinical detail around Merrill Lynch.
To accelerate the formulary reviews.
Post launch and the team's goal is to gain formulary acceptance and a line any payer clinical management criteria evidenced based medicine.
Turning to our plans for commercializing now that's better in Europe.
Earlier this year, the EMA validated our marketing application, our marketing authorization application for Merrell lifts that for patients with true, which puts us on track for a launch in early 2020 true if approved.
The support the launch in Europe and globally, we are building on our team in Europe and have begun to fill key roles.
We're also actively preparing for payer dossier submission and key markets. Our strategy is to have a targeted approach focused on larger European markets first while leveraging early access programs.
More broadly our goal is to extend Merrill Lynch and availability worldwide.
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