Q1 2021 Albireo Pharma Inc Earnings Call
Good day them all kinds of the Albert <unk> pharma first quarter 2021 earnings Conference call. Today's conference is being recorded at this time I would like to turn the conference over to Mr. Paul Art. Please go ahead Sir.
Thank you operator, and good morning, everyone. Thank you for joining today's call.
This morning, <unk> issued a press release, highlighting its recent business accomplishments and reporting its financial results for the first quarter ended March 31 2021.
This press release is accessible via the company's website at Www Dot Albarino pharma dotcom.
Before proceeding we would like to note that managements comments today may include forward looking statements regarding the company's plans and expectations. These statements are being made under the private Securities Litigation Reform Act of 119, 95, and they are subject to various risks and uncertainties.
Actual results may differ materially due to various important factors, including those described in the risk factors section of the company's most recent form 10-K and the subsequent SEC filings. These.
These filings can be accessed from the media and investors section of the company's website at www Dot Albury of pharma dot com or on the SEC's website Eddie.
Any forward looking statements represent management's views as of today Thursday may six 2021, and should not be relied upon as representing the views as of any subsequent dates.
The company undertakes no obligation to update these statements publicly now it is my pleasure to turn the call over to Ron Cooper, <unk>, President and Chief Executive Officer Ron.
Great. Thank you Paul and thank you everyone for joining us. This morning for all of the rails of Q1, 2021 results and business update with you.
<unk> today are Simon Harford, our Chief Financial Officer, Dr. Pat Horn, our Chief Medical Officer, and Pamela Stephenson and our Chief commercial officer.
Our theme for today, our commercial readiness and corporate growth.
At the start of the year. We lay ahead of plan of how we would expand to a global organization to reach the large 100000 pediatric cholesteric piece of an opportunity around the world.
And realize our aspiration to exceed $1 billion in the second half of the decade.
The deliver on this aspiration, we must build a strong global commercial organization for successfully launched build a or the VIX about in people.
Expand beyond piece of it can do other rare pediatric conditions.
And progress our pipeline in adult coldest attic and viral liver diseases.
So what I'll do is I'll take you through the progress and achievements as we continue to deliver on our objectives and made plans in support of our ambitions.
In the short term our priorities remain number one bring bellway or the VIX about over the line with product approvals in the U S and Europe with the issuance of a priority review voucher and a fast start and improve markets number two.
Focus on the global opportunity in Colas, static liver disease progressing our market, leading global commercialization of approach in the top 25 markets to reach the 100000 patients with rare cole of static liver disease.
Three continue to enroll patients in our two gold standard global pivotal phase III studies in Billary Atresia Bowl and the <unk> syndrome a search.
The number for advanced E. Three 907 through phase, one and the interface too.
Moving a Q3 for two into the clinic and characterize our other novel bile acid modulators.
Commercial readiness, starting with our commercialization strategy and launch readiness.
With priority with the priority review and if the <unk> date of July 20th in the U S and accelerated assessment in Europe, we've engaged in a good dialogue with regulatory authorities and anticipating the approval and launch in the second half of the year.
Finally, we need to be ready to launch at any time and are encouraged by the ongoing discussions in progress with U S and EU regulatory bodies on the past the gaming product approvals.
Now part of line for readiness and making sure that you of a brand name that is simple and consistent around the world.
We are very pleased to announce that Bill day is the brand name for road of VIX about that has been accepted by the EMA and that's been provisionally accepted by the FDA.
Having one global name is important for brand recognition and it's just not easy to achieve the named Bill of a supports our brand positioning in this test the well with physicians and patients on multiple parameters. So we're really excited to unveil it now.
And then officially upon anticipated product approvals.
Day, one readiness is a core focus within our organization and Pamela and her team of driving ahead on both the U S and new launches in the rest of world commercial partnerships, having made great strides the significant progress in hiring Onboarding and training of people who are highly specialized in their respective geographies.
Functions and in rare and liver disease markets.
In the U S. The sales team is on boarding and our survey of U S. Hcp's on their current management of PV patients in preparation for launch at the same time, we're actively engaging with career therapeutics to operationalize the planning for our combined efforts for the U S launch.
Also on track all of our customized in the house patient support services through <unk> assist.
The entire care coordinator team in place training on alongside our commercial and medical teams and working on for affecting the customized patient support services to help families navigate their access to build the from the start and be with them when they grow.
In Europe, Germany in early launch countries and it has the largest market potential so.
We are prioritizing completed hiring of the full team in Germany, including commercial medical and operations. We're also replicating our patient support services and localized for in country needs and regulations.
So Albert Aosis, Germany will soon be of reality, providing support services to optimize patient outcomes and support long term adherence to build value.
In both the U S and Europe, we know we have of compelling billing day value story, including the <unk>, one randomized placebo controlled trial <unk> two open label data with patients on drug beyond two years nap at natural history data and the caregiver disease burden.
<unk> study.
This compelling package of evidence should be an advantage in our mission to gain broad axis of building across a wide range of patients.
We have confidence in our comprehensive submission package and country level plants that we of compile up to support the value of build day in.
And ensure patient access globally.
Outside the U S and Europe, a key component of our commercialization strategy is a solid partnership network with leading rare disease companies.
This is particularly important the regions such as Turkey, and the middle East that have an increased prevalence of the <unk>, making them top 10 markets for commercial opportunities.
So we're thrilled to share the completion of two ex U S commercial partnerships for Bill day.
The first with getting the loss or gain a leading specialty pharmaceutical company in Turkey with more than 20 years of experience partnering with global pharmaceutical companies to bring innovative therapies in rare solutions to the community yen will be responsible for regulatory filings reimbursement submissions medical.
On the commercial support for build value.
The second partnership is with Gen pharma Gen Pharma services, a privately held regional pharmaceutical company focused on rare diseases, specifically in Saudi Arabia and the Gulf.
Unlike other countries. The number one reason for pediatric liver transplant in Saudi Arabia as Vic making.
The making the region importance for of helping patients and commercial opportunities.
Similar to similar to a range of again Gen pharma will be responsible for regulatory followings reimbursement submissions medical and commercial support for <unk>.
As part of both of agreements Albarino net sales will be recognized on the sales to each partner.
Adding the again and Jen pharma agreements to the one signed in the first quarter with medicine, we now count three solid commercial distributorships in place.
Each company is a rare disease market leader in this respect the region and each of the regions have some of the highest prevalence rates.
These agreements are a demonstration of the advancement of our well laid plans as part of our global strategy for the commercialization of commercialization of Bill day.
Now we can look ahead for the longer term commercialization.
Dino and expanding beyond <unk>, we continue to progress our pediatric development programs that will enable additional indications for current unmet.
The whole of static liver disease patients.
The global pivotal phase III study of build the analogy all syndrome for search study continues to enroll and dose patients tracking to of all sites active in the first half of 2021 with top line data available next year.
The bold study, which is the first and only pivotal phase III trial of an iPad inhibitor and Billary atresia.
Continues to be on track and we're meeting on patient enrollment goals with 48 global site Activations, including <unk> in the U S and 30 sites ex U S.
Valerie are treated as the largest pediatric cholesteric liver disease with an estimated prevalence of approximately 45000 patients around the world. We anticipate top line data in 2024.
A positive data readout will lead to a major global expansion opportunity and of potential treatment that could make an impact on many families.
Corporate growth R&D looking at our early stage of preclinical work our focus remains on smart pipeline expansion efforts. Most recently solidifying progress in rare pediatric liver disease, two adult and viral liver diseases.
In March we announced the initiation of our phase one study with <unk> 907.
The first highly bio available <unk> inhibitor to enter the clinic, which we're planning to develop for out of Cholesteric liver diseases, such as primary sclerosing cholangitis, PSC and primary biliary cholangitis PBC.
Yes.
Yeah.
This is significant we.
We dosed the first patients in the first in human study to investigate the safety Tolerability pharmacokinetics and pharmacodynamics of the oral formulation the.
The unique properties of <unk> 907, especially the high systemic availability holds great promise for increased for increasing dose to gain additional efficacy, while minimizing the diarrhea dose limiting effects observed with the iPad inhibitors in this patient population.
Now that we have the phase one study in progress we're looking at the top line data later this year and the initiation of phase two in 2022.
We're also pleased with the issuance of of U S composition of matter and method of use patent which provides exclusivity into 'twenty 40, non including patent term extension.
We believe that <unk> three nano <unk> has significant commercial potential in the outer colon static liver diseases.
We're also excited about the ongoing preclinical studies and modeling for <unk> three <unk>. The first oral <unk> inhibitor for viral and cole of static diseases and TCP as the key transport for bile acids, as well as hepatitis B and the viruses.
Eight to $3 <unk> also has unique properties as it is an oral potent small molecule that blocks entry into the liver and should have really excellent combined ability potential.
<unk> X of daily sub Q Mtc P inhibitor purchase by Gilead for over 1 billion euros provides both good scientific and financial proof book.
Financial value proof of concept.
<unk> is currently in R&D, enabling studies for the phase one trial anticipate the startup in 2022.
Overall, we continue to expand our pipeline delivering on the promise of scientific leadership for context. <unk> 907 is the third compound that the breach of Illinois coal team has progressed into the clinic, which for which continues to give us great confidence at E. Three for two will also advance this plan.
There is also potential and our other bile acid bile acid modulator approaches the research team continues to work on.
Enterprise strength beyond the outstanding hiring we've completed in the U S Europe, and most specifically, Germany, UK and Italy, we are adding top level leadership strength as part of the enterprise team.
<unk> pointed John Connolly, as Chief Technology Officer, who joined the organization to oversee drug substance and product development clinical supply distribution commercial supply chain.
The quality.
Jones career spans manufacturing management regulatory filing CMC and product commercialization as well as supply chain logistics sourcing and procure most recently John led the technical operations that stem line Therapeutics, where she was responsible for taking the lead product from the R&D stage through to commercialization and before.
That John had held senior roles at <unk> systems on Bristol Myers Squibb.
So were fortunate to add Jones expertise that spans from early drug development through product launches, which is key as we prepare for launch while progressing our clinical and preclinical programs in pediatric and adult liver diseases.
On deep expertise and experience in the end to end drug development and commercialization only strengthens our capabilities as we continue to progress our pipeline and prepare for the market.
So in summary, we feel very confident as we planned for launch build rate taking us towards our ambition to achieve a $1 billion in bill of <unk> sales in the second part of the decade for.
Progressing our clinical programs analogy on biliary atresia and further characterizing our promising preclinical compounds.
We are of the right vision strategy approach and people as we continued to deliver on our corporate objectives and build out the rail into a fully commercialized organization that will deliver on the successful product launch.
Let me turn it over to Simon to give you a quick financial update Simon.
Thank you Ron let me quickly summarize our financial results for Q1, 2021 revenues with $2 million for the first quarter of this year compared to $1 5 million for the first quarter of 2020.
The higher revenue was due to the estimated royalty revenue received from EA pharma for <unk> of that for the treatment of chronic constipation. The royalty revenue as always has passed all of them to healthcare royalty partners.
R&D expenses were $19 9 million for the first quarter of 2021 compared to $16 1 million for the first quarter of last year. The higher expenses were principally due to personnel expenses as we continue to increase our head count of program activities the increase in <unk>.
Graham activities was primarily due to bill day for the regulatory submission named <unk> and the additional indications for biliary atresia and <unk> syndrome, as we continue to drive our development programs. In addition, we increased spending for 839 O seven as the drug progressing.
Phase one of these increased expenses were partially offset by the fact that we are no longer get developing <unk> and due to lower spend in preclinical programs.
General and administrative expenses were $15 3 million for the first quarter of the year compared to $8 2 million for the same quarter last year. The increase is due to head count and activities and a number of the areas such as marketing field force preparations in the U S in Europe and the.
The infrastructure to support commercialization of <unk> once anticipated approval is received.
Net loss for the first quarter of 2021 was $43 $7 million or a loss of $2 29 per share compared to a net loss of $31 $5 million or a net loss of $2 23 per share for the first quarter of 2020.
As of March the 31st 2021, we had cash and cash equivalents of $217 1 million, which compares to $251 3 million at December 31 2020.
The company has sufficient cash runway to fund the plan launch about Apixaban and perfect and its development programs into 2023. Additionally, we plan to monetize the priority review back at an appropriate time if received upon approval.
We have revised guidance for the 2021 operating cash burn, which is now anticipated to be in the range of $130 million to $135 million. The increase is primarily due to acceleration of the pipeline. For example, <unk> 907 phase one started earlier than the.
Originally planned and to further bolster the fast stop built a launch efforts 2021 revenue from Bill day is anticipated to be in the low single digit U S. Dollar of millions with that let me turn the call back over to Roy on for closing remarks.
Super Thank you Simon.
We started the year strong and marching towards the potential launch of build value, while progressing our pipeline proving our ability to deliver against guidance on.
And we need to bring bill the over the line in the U S and Europe with the issuance of prior to the voucher and a fast start in the approved markets.
So to recap of our top priority number one we're focusing on the global Cholesteric liver disease opportunities in the top 25 markets, starting with the <unk> regulatory approvals patient access and commercial distributed distribution agreements.
We will continue to enroll patients in our two global phase III studies in biliary atresia analogy of syndrome, as well as expand beyond <unk>.
Three R&D will advance with <unk> hundred seven phase one underway, while moving <unk> into the clinic and characterize our other novel biologic modulators.
Overall, our global commercial strategy is solid with <unk> robust value proposition.
Our high level of readiness and our actions to expand beyond <unk> into other diseases in the coming months, we look forward to announcing the approval of Bill day in both the U S and Europe and updating you on our launch efforts so more to come on that.
Thank you everyone for joining us and we're pleased on the call now for Q&A operator over to you.
Thank you if you would like to ask a question. Please signal by pressing star one on your telephone keypad.
If you are using a speaker phone. Please make sure. Your mute function is turned off to allow your signal to reach our equipment.
On the press Star one to ask a question and we will take our first question from Andy Yang with Jefferies. Please go ahead.
Thank you.
So couple of of question just so I think for PC in the past you mentioned debt about 500 to 700 grants for patients.
Patients from the U S.
The most of them the Halloween identified so.
And the you asked a question.
Yes.
Why would it be the kind of the.
Gating factors to reach the patients and get them on the Zac and second question is on the European pricing side. So in the U S. C U E.
The commercial day, you talked about price range.
Neil.
Just the Germany.
Well they have the highest of price, but overall, what do you think of price on bond or would it be thank you.
Hi, it's Pamela.
The answer yes, I'll take the first question.
The first around the the gating factors in the U S. With the estimated piece. The prevalence you had mentioned that we shared on commercial day, the 500 to 700 addressable patients.
In the U S.
The the gating factors to the reach this.
The market potential.
There are two fold one is ensuring that patients have access.
To therapy, and what we're doing there.
With the payers and showing that they are educated around key effects.
Secondly, with al derail assessed.
Customized the patient support program, which will help individual patients navigate through reimbursement.
To answer your question in the dose.
We will be working as quickly as possible.
And we know that payers. It takes it takes time to move through that process right through the through the reimbursement process for each individual payer. So we'll be moving moving quickly to ensure the.
Alright are addressing that in a gating factor on your second question around European pricing, we've done a lot of work.
And as you know.
Europe the re.
The <unk> process.
Including Germany.
And just on your ability to.
It showed the value of the product.
And on be able to demonstrate efficacy through your clinical data, especially robust clinical data and as we looked across Europe.
Confident.
On the pricing band.
That other rare disease analogs have been able to secure it. So if you look across the of analogs of its quite well within the range typically we see a list prices that are within 20% to 30% of what youre going to see on the left and that that's done.
For the analogs that we're looking at so.
Just closing we're excited we're ready to go and.
Both in Europe and in the U S.
Thank you and thanks for the question you just just to add on as Pamela said, probably the biggest issues for uptake is access in the Hemlo said were working actively here in the U S. But also right around the world with our submission. So what we would like to do as quickly as possible is.
Get to the 2000 estimated between 2300 2800 available patients right.
So that's where our focus is but its patent losses as access here in the U S and axis in Europe as well.
For the question for you.
Yeah.
We'll take our next question from the two barrel with Cowen.
Go ahead.
Yeah.
Good morning, guys. Thanks for taking the questions.
And I'm going to beg your forgiveness of day Okay.
Jade It analyst here has been dealing with the FDA. This last six months.
Given your upcoming to do for the day.
July has there have there been any new questions or submissions to the agency submitted that you think could drive.
A three month extension of the picking for the like we have like.
Like we've seen all around.
And then right now as far as we know.
Without disclosing anything you plan on labeling discussions plan to start.
On a relatively timely basis per usual schedules.
Ritu, thanks for the questions and I'm sure that that's been the challenge for you on multiple fronts.
You can appreciate I can't really get into the specifics of it.
With the on our dialogue with the FDA.
Nice to say that.
<unk> has high unmet medical need there is no approved drug.
Our data and the Patrick one of <unk>. Two studies are great efficacy of great Tolerability and thus far I think we've enjoyed a good dialogue with the agency.
Been engaged and timely so we feel pretty confident about our July 20th of Paducah date, and the subsequent launch for Bill day.
The U S.
Got it.
Thanks for thanks for addressing that and then I guess as you think about your hires as you mentioned the Albury of assist seems to be hired up on your field force is in place can you talk about can you just reiterate sort of the head count in both of those and then.
Who are the hires debt.
You have me.
In those positions.
It's the experience where are they from and do they have pre existing relationships with.
Pediatric hematologist or theirs.
Our specialist centers.
Okay.
Yeah. Thanks for the question Ritu.
I'll, let Pamela talk about a little bit of the background, but you know in terms of numbers. What we've said is we anticipate about 10 representatives in the U S.
In addition to the the dozen representatives.
With <unk> therapeutics for the partner, we haven't really given.
Given the details in terms of the care coordinators, but suffice to say the care coordinators there'll be enough individuals there to deal with.
Any patients all of the prescriptions and help those patients navigate through the.
The the process of getting access to build the in Panama, maybe you can talk a little bit of both the background of our teams.
Sure so on and on.
On our sales team, we had a good fortune of just being able to recruit and attract.
Hi, Bill.
High caliber experienced sales professionals, who have been.
Working and I'd say the.
The two different things, we're looking for that we've been able to achieve the first is experience in hepatology space. So to your point day, they know that oncologists, the great experience really understand and have existing relationships and then the second would be the rare disease, so on being able to understand.
Now.
And the environment, where you're going into sort of the targeted 16 centers on.
Understanding and knowing how to put in for two large scale.
So through the entire year.
The center.
So those two attributes I would say are the most important things on we've been successful on being able to have those attributes on our team and then as Ron mentioned that bolstered by our partnership with on.
On <unk>, because they have those existing relationships as well and Theyre very experienced and then the final thing is recency of experience. So we're taking people right out of the field right now we have been selling through the COVID-19 environment.
And so they have the virtual selling skills. So that that's the makeup of our sales team and then on the care coordinator side again.
Gary we have hired care coordinators, who have been doing that their entire career day had been working from the highly experienced the companies that have their own in house patient support programs, who are who are very skilled at speaking with patients and families as well as understanding.
How to navigate the complex reimbursement environment in the U S. So really pleased and excited by the caliber of people, we've been able to attract.
So the care coordinators who've had rare disease that sort of rare disease.
Perhaps the literally on his targeted rare disease company.
Super.
Sorry last question that I wanted to Squeak Squeak Squeak, one the only last allowances for the COVID-19 roll off period that youre going to be launching into we've heard from other companies that patient doctor interactions have been down.
Across the number of disease States and I'm, just wondering how much of that.
Is applicable for pediatric cold static diseases.
And do you see any trends in that sort of patient clinician interaction.
Thus far in the first half.
We find that in the series.
Serious rare diseases like this that the interactions with the between the patients and the physicians has remained quite strong.
Other through in person visits and the orb virtual visits.
So we don't anticipate on impact in terms of patients being able to see their providers, primarily because of the severity of the disease.
Got it thanks for thanks for letting me sneak that one of them taking price.
Thanks for retail.
And we will take our next question from Liana <unk> with Wedbush Securities. Please go ahead.
Thank you for taking my questions and congratulations on your progress.
For the label.
Are there any do you expect a broad label.
Or are there any bogeys.
That that could happen for.
For likely.
Hello Anna.
You can appreciate I really can't comment on any of directly.
Directly on our regulatory interactions suffice to say that we are we've submitted all five modules both for the FDA and the EMA.
We submitted the pet the.
The Patrick data, which you know is whether you hit both primary endpoints and showed good tolerability. We also submitted the pet the pick two open label data as well an interim cut.
And within that we of Patrick the <unk>, one two and three patients so.
I have all of the information we're in active dialogue with them and we're looking forward to sharing with you more details as we get past the the <unk> date of July 20.
Thank you.
And we'll take our next question.
From Brian <unk> with Baird. Please go ahead.
Okay.
Hey, good morning, everyone and thank you for taking my question just wanted to kind of get a little help in trying to think through the the.
The near term guidance on Bill day sales I'm, just kind of how to think about it a little bit.
On the medium term.
And so for the near term.
How do we kind of think about that in terms of of sort of new entirely new starts versus patients on the expanded access program and can you discuss on all of the size of the expanded access program sample right now in the sort of timeline, we should think about for converting those.
From the program.
The commercial form.
And then on the medium term side of things when you think about that 600 patient available U S market.
Can you contextualize, how many patients are there really <unk> on under our care if I look at something like the launch of a highlight of the Obs project basically at 90% of the patients on treatment about a year and there's.
It was bit of an outlier, but do we how should we be thinking about peak back in that context on the differences you see in the market that would sort of anticipate sort of a longer range to higher penetration.
Alright, great, Brian Let me try and characterize how we see how we see this year going I think the way to think about this year is there still a lot of variables that.
That arent quite clear as yet and that's why we've given guidance for sales.
The low single digit million, so what's not that we don't know exactly the date of approval right and remember that's approval for for both the U S growth. The <unk> date of July 20th, but also Europe and remember what we're trying to do with access the 'twenty 300, 2800 patients right.
From a global perspective that are available that are you know and all of these are people that are eligible for treatment right now so what so one of the things that we have to have first of all we have seven approval. So both the U S and Europe are pretty important and we're still working on our European.
Approvals that we don't know exactly when that's going to occur, but we feel confident that's going to occur in the second half of the year. The second thing is the sequencing of access right. It's Pamela.
Oaken about we've got of we've been in dialogue with insurance companies in the Europe in the U S. But it does take time for them to process as well you know we're in active dialogue with payers within Europe as well on that will take some time as well. So we've got to work our way through that so think of this year as the year of where we sort of saw.
Through the access part of the things, we sort through the regulatory process right.
And then your question about about EAP patients on open label patients part of that as these people are in different countries right. So think about those patients of sort of correlating with that time of when we get approval and we get access so as we think about this year the patients that we're talking about here of the 20.
300 2800.
Available patients. These are patients that are available.
For the therapy. They are in various stages of early diagnosis or waiting for treatment in the <unk> system as quickly as possible get access for them get them into El Dorado assists.
Put a nice white gloves around them and take care of them and help them grow it to be.
The stronger and healthier children.
Thanks, Brian.
Thank you.
And we will take our next question from Ed Arce with H C. Wainwright. Please go ahead.
Great. Thanks for taking my questions and congrats on the continued progress across the pipeline.
Couple of questions for me.
First for for Pamela.
We're just a little over two months away from the producer date now and of course you've mentioned.
Debt your reps are on boarded.
The appropriate outreach as well as.
The the team of <unk> as well as your Alberto assist it seems like everything is in place I'm wondering.
What is the focus.
Between now and produce for us.
The last few weeks from.
In terms of.
Still may need to get done for that day, one readiness.
Okay.
The high Ed. Thanks for the question Yeah, we're two months away from the Paducah day, but we're ready to go now so as mentioned.
Higher trained for Onboarding eight on.
On boarding and so on.
We're ready to go from an operational perspective from a from a team perspective and the one outstanding item. It is the is the final FDA approved label. So between now and then as we get more insight on that label, we keep training and training and training.
And fine tuning.
We're ready to go there is really nothing left.
We could launch tomorrow, we've gone through all of the critical path that needed to get done so yeah from a commercial perspective, we are ready to go we've just.
Ladies and for the approval.
Great. Thank about the debt is the team has been practicing and practicing what it looks like.
How we generate prescriptions, how we deliver drug right now we make sure that through operating of assist that the patients have a really great experience. So.
There's just some things you just can't do until you have the final label right and so once we get the final label, we will be moving very quickly.
Of course.
Alright.
So just one follow up for me.
On the commercial partnerships as you mentioned.
Can you just recently signed your second and third.
I know there is.
25 <unk>.
<unk> that youre focused on globally.
Wondering if you could help us understand.
Which of those.
Our most likely amenable to partnerships.
Either on a regional basis or country by country basis.
As you continue to March forward.
Hi, Ed This is Simon set so as we've looked at the regional partnerships. Our approach has really been to say, let's focus first and foremost on.
Those major markets, where we will be able to launch relatively quickly following U S and the European anticipated approval.
If you look at top 10 market overall.
Several countries come into the top 10.
Outside of Europe, and the U S and those include Turkey.
Saudi Arabia, and parts of the Gulf region, as well as Brazil in the.
Latin America.
So we've really tried to focus on some of those key top markets because they typically allow for Sally early access.
In and of themselves as markets as well as being a good opportunity and as we discussed on our commercial day, yes, more than 50% of revenue typically in similar around the logs in rare diseases cabos from markets.
Outside of the U S.
Then we have for the long debt.
Sort of looking at the opportunity for.
For China as.
As we said on collateral day Thats not in the top 25 markets currently of what we've sort of projected going forward, but that is a good opportunity, particularly for biliary atresia longer term, but to go to a market such as that we would want to make sure that we have the the right.
While stones and place them that way of getting appropriate value from the opportunity such as that but near term focus.
Turkey, Saudi two of the the top 10 markets.
And then Brazil and other important one.
Great.
Helpful laying out the timeline I appreciate it congrats again.
Okay.
Thank you Ed.
And once again, if you would like to ask a question. Please press star one.
Take our next question is from Tim Lugo with William Blair. Please go ahead.
Thanks for the question and I know everyone's probably excited about another regulatory question, but.
Just following up on on.
Some of the I guess anxiety from the free.
From the analyst community last year. The agency has just for.
<unk> kind of executing manufacturing inspections, because of the pandemic, but hopefully that.
Not as much of an issue now with the U S. Opening back up can you just update us on your manufacturing status and how confident you are from a regulatory perspective.
So that I know of anything that's.
I'm, sorry, Tim I know, there's anxiety out there but to be honest I'm not anxious right I think we feel really confident strength from a manufacturing perspective as I said, we submitted the full module.
The full CMC module in our phase III study, we used the planned commercial formulation in that we spoke to we had a good dialogue with the FDA of couple of years ago. When we're executing on that plan right and that seems to be on track. So we feel pretty confident again about on.
Our approval.
Around our produce the date of July 20th and as Pamela said.
The ready to go for more from all fronts.
That's great to hear the.
We look forward into 2022, and hopefully raw exits.
Given the patient dynamics from the U S.
You're pregnant trivia.
But also what should be of European approval knock on wood hopefully by then.
Do you expect a significant portion of revenues in 2022 could be coming from Europe, and maybe even a larger portion than what would be we would be seeing from the U S.
You know again at it you know as I said in my earlier response, there just some there are multiple on those in this case right. So the exact need of our U S approval, we think it's going to be the fate of the 20th of July we're in good dialogue with Europe on the exact date of that and then I'll.
So how these regional partnerships click in and how access so that's the one of those ones were going on we'll see but frankly, we're focused on the global opportunity for the VIX what were building of pediatric coldest attic liver disease drug we want to get out of the old.
On the around 100000 individuals around the world and when it comes to <unk> that range of 20 302.
Two to 2800 page on individuals, we're going to try and get to the as quickly as possible and given that there are more patients outside the U S. It's quite possible that we see.
Higher than anticipated contribution from Europe, but it's really too early to say.
Alright, great. Thank you for all of the color.
Thanks.
We will take our next question from Yasmin Rahimi. Please go ahead.
Hi team. Thank you so much for sharing and all of the update.
Couple of questions for you maybe the first one with the can you share wide into with Pete pick two of them.
You know.
Continuing to enrolling patients on have you been able to collect it.
At the end rates of Andrey, meaning you know coming off of current plantation lit.
Or no.
Ladies bank.
Is there a potential of the regulator credit.
Alright, those type of data points and.
And may even require a separate you know.
Study to be conducted so I would love to hear your thoughts on that and then maybe the second one is on the P. R V.
So you know.
What is in your view of the likelihood of being granted that.
Pier B.
All day approval on that I have the follow up.
So this is Pat and I can take the one on the <unk>. Two study. So that is our ongoing studies looking at the long term safety and efficacy of all the things of that in patients with all the types of peak day and there. We are really collecting those long term outcomes. We're looking at time to build every day.
Asian surgery time for liver transplant, whether patients can come off surgery the high.
Either from the familiarity of diversion surgery or is it for transplantation.
So that is that is the ongoing study is continuing as planned.
The plans for that study are well known for both of the regulatory agency in terms of post marketing commitment.
Certainly.
Those will be part of the discussions with ongoing regulatory agencies.
And then I guess to add to that you asked me. The answer your question about the pier B, we're in dialogue with with both regulatory agencies, the FDA and EMA.
We have confirmed at that time that head fake one.
On its own with all of that we needed to do from a submission perspective.
For approval much like the single study of all the single study of assert is needed for approval. So the additional data that we gave for pet food to give additional color.
<unk> cut and so most of the types of things we will continue to talk through in terms of post approval commitment as Pat indicated now as it relates to the priv, we'd announce.
A couple of years ago that in fact, the FDA has has deemed or the VIX about as being the <unk> eligible. So we are <unk> eligible and I think the way you need to think about it is that approval.
Pretty much equals <unk> being granted right. So we have to fulfill the requirements of the <unk>, which is doing the pet fixed study and submitting the drug there are some administrative pieces that debt that have to occur.
On the approval, but we feel pretty confident of the <unk> will be.
Issued on the approval and as you know those are quite valuable we sit in a very good cash position. So we have the luxury of and we don't need the <unk>. So we plan to monetize the more monetized at the right time.
Thank you Ron and thank you Pat for the color.
Maybe a follow up are you.
Germany, maybe one of the.
The largest market opportunity there and also you can go in with you know.
No pricing.
Able to get the market Ax faster can you help us understand sort of how much.
Any piece of the patients have been identified.
In Germany, just just to kind of help us understand more granularly on some of the larger opportunity like how many patients that are at least in Germany alone.
That could be.
Available for therapy.
Yeah. Thanks for the for the question you asked me, we're pretty excited about Germany, because as you said.
There is a fair amount of people that immigrating into Germany with the disease. So it has pretty good pretty good level of disease, we are really going to key opinion leader support.
And and as you said it has good good pricing at this time, we're not giving the country by country detail in terms in terms of the numbers, but suffice to say that in terms of patients that are available for treatment.
Ex U S. We anticipate somewhere between 70, and 100 and in 'twenty 100 patients, so a pretty significant opportunity there as well.
Thank you Ron per day of my question.
Thank you guys.
As a reminder, please press star one to ask a question and we'll take our next question from Joseph Stringer with Needham <unk> Company. Please go ahead.
Hi, everyone. Good morning, Thanks for taking our questions just the one on the kind of the competitive side of things.
Likely the first the market in the U S for piece of it Ken.
And in Europe.
If things go well likely first the market, but because the competitor.
Coming to market in the first part of 'twenty 'twenty two piece.
In Europe I, just wanted to get your thoughts on.
How you see that affecting your current.
Commercial credit.
<unk> been discussions with <unk>.
Around reimbursement of things in Europe, and maybe would there be any sort of.
Read through too for your U S <unk>.
Launch discussions thank you.
Yeah, I think what we're really excited about Joey is that we're taking a leadership approach with bill <unk> with.
With all of diseases right around the globe right. So.
When you think about the <unk> study the bold study the of search study. These are all gold standard double blind placebo controlled studies right and so they should help us gain approval if the law.
The latter two of our successful in multiple regions around the world right. So we feel really good about our leadership approach of them, but thats not only of our regulatory approval of that also was from an access perspective, right and so that.
That will actually that is level one class one type of information that we're going to supplement that with the nap of natural history database and the burden of care study and we've put together a pretty compelling access package and when you considered in the pet Big data, we have both the <unk> one two and.
Three patients.
So theres a lot of really interesting interesting data there. So as a result, we believe that we will have a leadership approach for both regulatory approvals and access across multiple indications for for Bill day and building building into what we will believe we believe will be $1 billion product day in the second.
Part of the decade.
Yes.
Great. Thanks for taking the question.
Thanks for joining.
It appears there are no further questions at this time I'd like to turn the conference back the CEO, Ron Cooper for any additional or closing remarks.
Great. Thanks, operator, well first of all of thank you all for it for attending today's conference call.
As we continue to ready for approval and will update you on our regulatory filings and launch planning as well as the advancement of our clinical programs as part of our growth strategy really appreciate your attention day, we have a lot of exciting near term milestones.
Head of us from and our strong financial position will enable us to continue to advance <unk> mission, which is to provide hope to families of patients with liver disease in the entire of liver community. Thanks for getting to all of you for your continued support of a great day.
This concludes today's call. Thank you for your participation you may now disconnect.
Yeah.
Yeah.
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Okay.
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