Q3 2019 Earnings Call
Laura Flake: Our Medical Officer, Dr. Noah Rosenberg, and our Chief Financial Officer, Laura Flake.
Unknown Executive: It involves known and unknown risks, certainties, and assumptions that may cause a cyber attack.
Chris: Thank you, Chris, and good afternoon, everyone.
Unknown Executive: We are here today to settle our four conditions of identifying, developing, and delivering life-changing therapies to people with disabilities. During the third quarter, our team called execution possible.
Momentarily.
Unknown Executive: Thank you.
Unknown Executive: Good afternoon, everyone. We are continuing to fulfill our important mission.
For session and instructions will follow at that time.
Unknown Executive: Our team has solidified changes across our physical development programs and a renal diet.
Good afternoon, ladies and gentlemen.
Since during the conference.
So since third quarter financial.
Okay cool.
As a reminder.
<unk>.
This conference call is being recorded.
Unknown Executive: We made progress towards our goal of creating transformational programs in the coming years. For example, patients with FFGS and IgG nephropathy...
You bet arena listen only mode.
Over to your host for today.
He.
Uh huh.
Indeed answer session and instructions will follow at that time.
Thank you everyone welcome to <unk> third quarter 2019 financial results in corporate update call.
Hey, Colby led by our Chief Executive Officer, Dr., Eric today.
Unknown Executive: are at a high risk of end-stage terminal disease with no approved treatment option, so we continue to act with a great sense of urgency. I am pleased that the institution
We'll be join for prepared remarks bar, Chief Medical Officer, Dr. <unk> No Rosenberg in our Chief financial Officer looks like.
Kirby.
Right.
Hello, everyone welcome to <unk> third quarter 2019 financial results in corporate update call.
I'd like to remind everyone that statements made during this call the party matters that are not.
Yeah.
We'll be join for prepared remarks, Burke, Chief Medical Officer, Dr. <unk> No Rosenberg in our Chief financial Officer looks like.
Unknown Executive: And on track, most importantly, our site engagement.
Payments are not guarantees are performing.
Research and development will join us for the Q and.
Unknown Executive: On our last quarterly call, we talked about the implementation of initiatives to
It's an assumption that may cause actual results.
When they made during this call already matters that are not historical.
Unknown Executive: [inaudible]
Unknown Executive: Most importantly, our site engagement and recruitment efforts have resulted in positive momentum for enrollment.
By by the.
Events within the Safe Harbor provisions of the private Securities Litigation Reform Act 1995.
Yes.
Forward looking statements are not guarantees performance.
Okay filed with the FCC.
Unknown Executive: And we are seeing a continuation of a positive change in projectory prescription.
Risks uncertainties and assumptions that may cause actual results.
As of today.
Vince to differ materially from those expressed or implied by the.
Unknown Executive: [inaudible]
Unknown Executive: Transcripts provided by Transcription Outsourcing, LLC. Transcription Outsourcing, LLC. All rights reserved. Transcription Outsourcing, Inc. Transcription Outsourcing, Inc.
Typically disclaims any obligation to update such statements to reflect future information event in.
What's the risk factor section, our Form 10-Q , and 10-K filed with the FCC.
Good afternoon, everyone.
Looking statements represent our views only as good as of the date such statements are made October 32019, and Retrophin, specifically disclaims any obligation to update such statements to reflect future information events or circumstances with that I mean, nothing to call over the air.
Unknown Executive: On the commercial side of things, we continue to track records of year-over-year organic growth in net product sales.
Program.
Unknown Executive: If approved,
And the commercial portfolio.
Unknown Executive: to more than 100,000 patients in the U.S. and Europe. On the commercial side of the business, we continue our track record of delivering year-over-year organic growth and net product sales.
Turning to everyone.
Continued advancement of the spar sense in phase three.
It's been of identifying.
We made progress towards our goal of creating transformational growth in the coming years.
During the third quarter, our teams had solid execution across our pivotal development programs.
Nope.
Portfolio.
Prove treatment.
Indeed advancement of the spar sense in phase three.
Unknown Executive: We saw a new patient today.
Currency.
Yes.
Unknown Executive: Importantly, our group reflects...
I'm pleased with the execution.
Towards our goal of creating transformational growth in the coming years.
Protect studies remain on track.
Unknown Executive: is a great example of this; our belief was that if we could offer a challenge that provided additional
And I actually in the property or at a high risk at the end stage renal disease.
Paul we talked about the implementation of initiatives to support enrollment.
Unknown Executive: And our organizational burden is successful because we live and work together.
Urgency.
D. centers of excellence for pivotal duplex study NFS, yes.
Unknown Executive: For more information, visit www.cdc.gov
Our phase three programs and the pivotal duplex and protect studies remain on track.
Unknown Executive: And we must act. We are seeing clear evidence that there is strong access and demand.
Importantly, our site engagement.
Okay.
And recruitment efforts have resulted in positive momentum for enrollment in the study.
Should is to support enrollment beyond the key centers of excellence for our pivotal duplex study in Fscs.
Unknown Executive: And in the first three months of the war,
Unknown Executive: This is in line with our expectations for a successful launch and a demonstration of our ability to effectively engage payers. We are also seeing a broad uptick in the first three months of the launch. At this point, more than 80% of Viola EC prescriptions are being covered by payers, and the potential for reduced hillbillies.
Indicator for a moment gives us confidence in our ability to achieve our.
Yes.
Most importantly, our site engagement.
Okay.
Recruitment efforts have resulted in positive momentum for enrollment in the study.
We're seeing a continuation of strong enrollment trends there as well.
Factory for screening.
Oh Sparsentan has the potential to expand the addressable populations for treatment.
Our targets.
It's 100000 patients in the U.S. in.
The is maintaining its robust support from the community.
Our record of delivering year over year organic.
Trends there as well.
Unknown Executive: This is in line with our expectations for a successful launch and a demonstration of our ability to effectively engage payers. We are also thinking about a new way to engage payers.
Sales.
If approved as far south and has the potential to expand the addressable populations for our treatments.
The more than 100000 patients in the western.
Consistently identify new.
We continued our track record of delivering year over year organic growth.
<unk> continued growth.
Product sales.
Organization.
Saw new patients initiate treatment and our portfolio is performing to plan.
Importantly, our growth reflects our team's ability to consistently identify new.
Unknown Executive: including those who discontinued the original.
Unknown Executive: We have seen a number of these patients try Thiola EC in the hopes of gaining additional flexibility and convenience that was not previously available with the original.
We could offer a treatment option to provide additional freedom of administration.
Can you.
And potential for reduced pill burden.
It's successful because we listen to patients and physicians.
Unknown Executive: All of these early signs from the BioECE launch give us confidence that this product will be a meaningful option for secondary patients. The bile acid portfolio also continues to perform as expected and show year-over-year growth. Overall, our commercial team delivered a strong curve forward that keeps us on track to reach our guidance for the year. Peter's appointment directly aligns with our priorities of optimizing future launches, including first intent. Finally, before I hand the call over to Noah for the clinical update, I want to highlight our three years of experience launching Best in Class therapies in the U.S.
Choice.
And we act.
You are seeing clear evidence of this with strong access and demand in the first three months of the launch.
The offer a treatment option that provides additional freedom of administration.
Covered by payers.
So for reduced pill burden.
Our expectations for a successful launch.
The a meaningful treatment choice.
To effectively engage payers.
Going into this with strong access and demand in the first three months of the launch.
But you discontinued the original formulation.
I'll, let you see prescriptions are being covered by payers.
He see.
And with our expectations for a successful launch.
The stability and convenience.
Philly to effectively engage payers.
With the original.
Fraud uptake amongst patients, including those who discontinued the original formulation.
This product will be a meaningful option for cystinuria patients.
Unknown Executive: Peter's appointment directly aligns with our priorities of optimizing a barcentine's value for patients and building upon
In the hopes of gaining additional flexibility and convenience.
As expected and showed year over year.
Unknown Executive: A highly accomplished leader with more than 20 years of experience.
Frictional.
All our commercial team delivered a strong third quarter that keeps us on track.
Unknown Executive: in Launching Best-in-Class Therapies in the U.S. and Abroad
You must confidence that this product will be a meaningful option for cystinuria patients.
Unknown Executive: Our clinical development team has made solid progress, advancing our typical trials to shape the treatment paradigm.
I spoke with future launches.
I'll asset portfolio also continues to perform as expected and showed year over year growth.
The clinical update.
Paul our commercial team delivered a strong third quarter that keeps us on track.
Unknown Executive: and F.S.G.S. and I.G.A.
Noah: nephropathy. Now, let me turn the call over to Noah. Noah? Thank you, Eric, and good afternoon. Our clinical development teams have made solid progress advancing our pivotal trials, evaluating SporScan10 in every field.
Total officer.
Guidance for the year.
I mean, it directly aligns with our priorities of optimizing sparsentan value for patients.
And building upon our strong commercial abilities.
Over to Noah for the clinical update I want to highlight the recent appointment of Peter Heerema. The Companys first chief commercial officer.
Unknown Executive: and a function on ureter tissue or a dialysis defect in the right side of the front teeth.
Peter's appointment directly aligns with our priorities of optimizing sparsentan value for patients and building upon our strong commercial abilities.
Unknown Executive: [inaudible]
Unknown Executive: and the F.S.C.S.
Unknown Executive: Our treatment indicated that for these disorders, there is a significant unmet need.
Which will be instrumental as we position sparsentan to shape the treatment.
Unknown Executive: to shape the treatment paradigm for patients with these rare kidney disorders.
Yes.
Yes, yes.
Hi therapies in the us and abroad.
Unknown Executive: experienced a more than doubling of endothelial receptors, which when stimulated are believed to lead to...
Now, let me turn the call over to Noah.
She will teams at top organizations.
Good afternoon.
So has.
Unknown Executive: with FSTS.
Clinical development teams have made solid progress.
Unknown Executive: This outcome gave us great confidence in our robust Phase II data set and the ability to demonstrate a clinically meaningful reduction in risk.
Yeah.
Sensing.
Instrumental as we position sparsentan to shape the treatment paradigm.
Yes, I Shana property.
Shifting to propagate.
Unknown Executive: and a reduction of protein production for textiles.
For kidney disorders.
The call over to Noah.
I progressive loss.
Unknown Executive: I am pleased to report that our
Thank you Eric and good afternoon.
Okay.
Our clinical development teams have made solid progress advancing our pivotal trials evaluating sparsentan.
Unknown Executive: have made meaningful progress in support centers to drive enrollment and continue to demonstrate a clinically meaningful reduction in student enrollment.
At 40% to 50% of these patients.
Okay.
No says Fscs I'd unit property.
Unknown Executive: We have a 36-week treatment, and our...
Renal failure within 10.
A functional familiar tissue.
Al.
Unknown Executive: The Independent Data Monitoring Committee meeting
And you will fill to filter function.
Second renal function ultimately leads to a remarkable.
Unknown Executive: All of which you have recommended has made meaningful progress.
40% to 50% in these patients.
Unknown Executive: Plan, Unearned Issues,
In fact.
And those with Fscs Egina prophecy.
Unknown Executive: For the complex study and both studies, we now have more than 200 sites for this meeting.
To me approve pharmacologic treatments indicated for these disorders.
Unknown Executive: More than 200 sites opened this week, which is an important milestone for the Board
Third.
There is a significant unmet need.
Patient.
Decline in renal function ultimately leads to a remarkable reduction.
Goal.
While the of life.
Unknown Executive: Meetings for both duplex and pursuit.
I'm going pivotal programs.
After she asks RG nephropathy.
Unknown Executive: And the DMC recommended to our press to proceed as planned.
Yes may approved pharmacologic treatments indicated for these disorders.
Unknown Executive: Proceed as planned.
Unknown Executive: This is an important milestone for broadening...
Class therapy.
Nitpicking unmet need to improve outcomes.
Patients with these rare kidney.
Unknown Executive: are continued cyber-siegement activities.
Ill disease for patients.
So unique dual mechanism.
Unknown Executive: We have a highly knowledgeable team that drives scientific...
With the ongoing pivotal programs Fscs in Idina property.
And we'll fill in receptors.
Unknown Executive: Operational Site Engagement, and we remain on track with a primary focus...
For Centene as a first.
I believe to lead.
Pete.
Detrimental effects on renal.
For nine.
So with these rare kidney disorders.
Tens unique dual mechanism and selectively profile.
She is.
Both.
Variance in more than doubling.
And we'll fill and receptors.
Urea.
When stimulated.
Resort and treated.
Good to detrimental effects on renal structure structure and function.
Robust phase two data set from the duet study.
It demonstrates a clinically meaningful reduction in proteinuria.
Unknown Executive: for Enrollment in Policy.
Unknown Executive: The interactions continue to have a positive impact on 2020.
Experienced a more than doubling.
And our phase III duplex and protect.
Irbesartan treated.
We support.
Fscs.
Our goal and operations teams.
Unknown Executive: of
Unknown Executive: to have top study data for the first 190 patients.
Yeah.
We had made meaningful progress.
Sparsentan potential any ability.
Unknown Executive: and We've been very pleased with the program.
Hi, good quality in both studies.
Full reduction in proteinuria after 36 weeks of treatment.
Unknown Executive: We now have approximately 150 sites open for training.
Actual.
Phase III duplex and protect.
Unknown Executive: And as we have been open and enrolling patients for about 10 months, we've been very busy.
Yeah.
Meeting.
Im pleased to report.
For.
Clinical and operations teams.
Had made meaningful progress.
Unknown Executive: We've been calling patients for about 10 months, and we've been very pleased with this progress. We now have approximately 150 sites open for screening.
Initiatives to drive enrollment and continued quality in both studies.
And we now have more than 200 sites open for screening.
I can scheduled.
And then data monitoring committee meeting.
And outside.
Flex and.
In the U.S. and.
Recommended.
Unknown Executive: [inaudible]
Proceed as planned.
Yeah that both studies.
Unknown Executive: The trajectory remains strong.
We are seeing.
For the duplex studying fscs.
Unknown Executive: Thank you for watching.
I'll have more than 200 sites open for screening, which is an important milestone.
Unknown Executive: In the first half of 2022, we look forward to engaging with an evolving community.
Clinical.
Turning our reach to patients outside.
Unknown Executive: Building upon the growing support...
Centers of excellence in us and expanding our presence.
Unknown Executive: are in addition to raising the
Enrollment and quality.
Interactions.
Unknown Executive: of our ongoing equitable
We are seeing.
Could impact.
And you'd site engagement activities.
Action.
Unknown Executive: We'll be highlighting some of the new research.
We have a highly knowledgeable team.
Unknown Executive: [inaudible]
Paul.
That drives scientific clinical.
Unknown Executive: So far, this is the other supported piece.
Having the intended.
Yeah, well site engagement.
And on track.
Primary focus on ensuring.
Unknown Executive: And if you want it, And if you...
From the first 190 patients.
Our interactions continue to have a positive impact.
2021.
Unknown Executive: Alport Syndrome. This is a presentation by the National Center for Disease Control and Prevention.
For traction.
Productivity.
For accelerated approval submission.
Initiatives for duplex or having the intended impact.
Unknown Executive: and other supportive people that will discuss SME and build upon the previously recorded material.
And seeing a property program.
The protect study.
Yes.
Unknown Executive: ...
Unknown Executive: And while early, there are also pre-clinical times.
From the first 190 patients.
For about 10.
Path.
And we've been very pleased with.
2021.
We now have approximately 150 sites open.
Celebrated approval submissions.
Unknown Executive: which is pre-clinical work that will be discussed at the meeting from
Our benefiting.
In Europe .
<unk> footprint with.
Machine a property program.
Unknown Executive: You're continuing to elevate
Three remains strong.
And as a.
And enrolling patients for about 10 months.
Unknown Executive: [inaudible]
Been very pleased with this progress.
The first half of 20 to 22.
Unknown Executive: and their physicians to address this aspect of the disease. We are continuing to develop the treatment.
60 sites open.
To engaging within a project community.
Fitting.
Upcoming Aesynt kidney week meeting next.
Thanks.
Our enrollment trajectory remains strong.
Support.
Sentence.
In the ability for.
Unknown Executive: and we'll share more in the future as we progress with our assessment.
From the future new.
The first half of 2022.
We look forward to engaging with the nephrology community.
Thanks.
At the upcoming Aesynt kidney week meeting next week.
Letting some of the new research.
Building upon the growing support.
As you've reported at previous Sn.
Future new.
Okay and ability to reduce proteinuria.
And I Janus profits.
In addition to raising awareness.
For ongoing pivotal studies.
We will be highlighting some of the new research.
But new findings from the open label extension.
So.
Since receiving sparsentan.
Previous ASEAN.
Errors.
Percentand has demonstrated an ability to reduce proteinuria.
A progressive disease.
Far.
This.
84 weeks of treatment.
Did you get open label study.
Four cents inherited fscs.
About new findings from the open label extension.
As Joe patients receiving sparsentan.
Thanks for sentence potential in rare nephrology.
The of life.
May be to exceed four cents cents potential alport syndrome.
This is in other supported.
Clinical.
The growing sparsentan narrative fscs.
Previously reported.
And.
Potential for Sparsentan.
As part of our efforts to maximize for a sentence potential in rare and apology.
Also preclinical side.
Since potential Alport syndrome.
So.
Our collaborators.
Plus which is encouraging.
That will be discussed at the meeting.
For patients.
On the previously reported.
This aspect of the disease.
To have a positive impact on kidney function.
Woltman pathway.
Really.
Feasibility for Sportscenter and Alport.
We will.
Jerre more in the future as we progress our.
Hearing loss, which is encouraging.
This call over to Laura.
For the financial update.
And their physicians to address this aspect of the disease.
We are continuing to elevate the development pathway.
Folio grew to 44 million, 9% increase over the same period in 2018.
Unknown Executive: Let me now turn the call over to you.
Laura: Thank you all. Now, over to Laura for the financial update.
Let me now turn the call over to Laura.
Noah: Thank you, Noah.
Then my team.
We'll update.
For did net product sales of 128 7 million and we remain on track to reach our guided growth for the full year.
Laura: During the third quarter, Net Product Sales from our Commercial Portfolio grew to 44.4 Gap Net Loss, a 9% increase over the same period in the third quarter of 2019. After a jump of 2,019, we reported Net Product Sales of 128.7 million, and we remain on track to reach our Guided Growth for the fourth quarter of 2019.
It's important.
Million, 9% increase over the same period in 2018.
19.
For the first nine months in 2019, we reported net product sales of 128 7 million and we remain on track to reach our guided growth for the full year.
The expenses were 33.2 million for the third quarter.
Million third quarter 2019.
Over the same period in 2018.
As an income tax.
Well to higher expenses to support our clinical.
Point 2 million.
Correct.
On a GAAP basis.
Okay and basis.
This is were 33.2 million for the third quarter.
Third quarter.
The team.
Little bit noncash expenses for the third quarter included 2 million of stock based compensation and amortization.
To support our clinical and product development efforts.
The quarter were 29.8 million.
R&D expenses were 31.2 million per the third quarter.
We attributable to increased compensation expense.
Clinton 2 million of stock based compensation and amortization.
non-GAAP SG.
Administrative expenses for the quarter were 29.8 million.
Significant non cash adjustments for the quarter consisted of 7.5 million and stock based compensation.
Unknown Executive: For more information, visit www.fema.gov.
Ben and higher professional fees.
On an adjusted basis non-GAAP SG any expenses for the third quarter.
Unknown Executive: On a gap basis, R&D expenses were $33.2 million for the third quarter of 2019.
Ram CNS C O one.
Significant non cash adjustments for the quarter consisted of 7.5 million and stock based compensation and depreciation and amortization.
Unknown Executive: [inaudible]
Unknown Executive: R&D expenses were $31.2 million for the third quarter.
Dissipate operating.
This concludes our previous guidance related to the discontinuation of the joint development program for CNS COO one.
Unknown Executive: Relevant non-cash expenses for the third quarter included
Accelerate.
Period, we incurred a 15 million impairment of long term investment associated with that program.
Breathing expenses will decline from current levels.
Unknown Executive: Non-GAAP selling general and administrative expenses for the quarter were $29.3 million. Significant non-GAAP changes for the quarter consisted of 7.5 million dollars.
The operating.
To see a modest decrease in ESG M&A from what we anticipate reporting for the full year 2019.
We continue to accelerate.
R&D spend in 2020 will decline incrementally from our current state.
Thanks.
And the discontinuation of the Fastnet Pan tilt in a.
Current levels.
We are maintaining a disciplined approach to our investments in our balance sheet remains quite strong.
Unknown Executive: are largely attributable to increased competition.
Unknown Executive: On an adjusted basis, non-gap estrogens...
Full year 2019.
Unknown Executive: Significant non-cash adjustments for the quarter.
Equivalents as of September 32019.
Unknown Executive: and depreciation and amortization anticipate operating expenses with our previous order.
We will decline incrementally from our current state.
Cash balance will allow us to invest in spar sentence growth potential.
And support our current level of operations beyond the planned interim readout of the duplex study.
Unknown Executive: During the period, we incurred a $15 million impairment of the long-term investment associated with them.
407 million of cash and cash equivalents as at September 32019.
Okay.
As you heard from the teams remarks.
Dissipate this cash balance will allow us to invest in our sentence growth potential.
Unknown Executive: We anticipate that operating expenses will decline from currently expected.
Part our current level of operations beyond the planned interim readout of the duplex study.
Unknown Executive: We expect that via modesty, proof, and STNA will be similar to what we reported for this quarter. Our duplex and protection activities continue to accelerate, and as we work to complete support study closure.
Program, we've realigned our organization to maximize our chances of success.
Yes.
As you heard from the teams remarks, we are building positive momentum.
And to create the greatest value for.
Our.
Answers.
And following the decision during the quarter to discontinue the Phos met tend to make program, we've realigned our organization to maximize our chances of success.
Unknown Executive: We expect to see a modest decrease in our investments, and our balance sheet remains quite strong with $407 million of cash and cash equivalents as of November 8, 2019.
Secure payment of four key priorities.
The already that are expected to create the greatest value for patients.
Unknown Executive: Declining. Incrementally.
Duplex and protect studies as quickly as possible.
Unknown Executive: We are at the third level of operation.
With clear purpose.
Strong quality and study conduct we've seen to date.
Yes.
Right.
I focused on optimizing our transformational value.
Unknown Executive: Scorch, George F B E N F G E R E M O N C H O E H E N F O N F O E N F O
Okay and opportunities for Sparsentan may have additional potential for patients.
Broad the successful enrollment of our pivotal duplex and protect studies as quickly as possible.
Unknown Executive: with $407 million in cash and cash equivalents.
Ladies.
Maintaining the strong quality and study conduct we have seen to date.
Laura: Thank you, Laura.
Go.
Yeah.
We will explore work to evaluate additional development opportunities for Sparsentan may have additional potential for patients.
This development to diversify our growth.
Upon our strong commercial teams capabilities.
We believe can create meaningful value.
Fruit product portfolio.
Our team is incredibly dedicated.
The launch of Sparsentan.
That retrophin are in position to execute and we look forward to sharing our progress on each of these priorities as we move throughout the balance of 2019.
For two entities that we believe can create meaningful value.
Unknown Executive: We anticipate...
Unknown Executive: Our balance will allow us to invest in our sentence growth potential and support our
Over to Chris.
Open it up for questions.
Our team is incredibly dedicated.
Can we go and open up the lines for Q and I believe.
And our in position to execute and we look forward to sharing our progress on each of these priorities as we move throughout the balance of 2019.
Unknown Executive: Support our current level of...
Unknown Executive: Operations Beyond the Planet
Eric: Let me now turn the call back to Eric for his closing remarks.
Stone.
Plenty.
And if your question has been answered or you wish to removed yourself from.
Unknown Executive: Maximize our chances!
Unknown Executive: As you heard from the team's remarks...
Question Chris.
Because the.
Thanks, Eric Kirby can we go and open up the lines for Kunaev. Please.
Unknown Executive: Following the decision during the quarter to discontinue the FOSMET and TOTENATE programs, we realize that we have failed.
Okay.
Absolutely.
Yes.
Have a question at this time.
For a star is in the number one on your Touchstone telephone.
Unknown Executive: [inaudible]
Unknown Executive: We focus on our strengths and the key priorities that are exceedingly important to us.
First question comes.
We have been answered or you wish to remove yourself into.
Unknown Executive: We will drive the successful enrollment of our students.
Unknown Executive: and Focus on optimizing our transformational value through the execution of four key priorities.
VB.
Hi, Chris the County.
Go ahead your line is now.
The compound the cleaning roster.
Great. Thanks, very much congrats on the progress.
Unknown Executive: We will drive the successful enrollment of our pivotal duplexes upon ourselves.
Yeah.
It looks like we now have a couple of.
If spreads and.
Unknown Executive: Transcribed by https://otter.ai
From comes from the line.
Swartz.
In terms of like there.
But that's too.
That's your line is now open.
Unknown Executive: We will explore work to evaluate additional development opportunities.
Severity or other baseline characteristics.
Thanks, Congrats on the progress.
Our being enrolled now from.
Detected any difference and.
Driven towards referral.
The patients in terms of like there.
Sites, where you were primarily getting patients earlier.
Be or other baseline characteristics.
In terms of.
Our being enrolled now from.
Non centers of excellence and.
Importantly, we see consistency across the.
The referral sites, where you were primarily getting patients earlier.
Ultimately, what we're looking to do is to replicate and strengthen the results that we saw from from the duet study in phase two.
Unknown Executive: Additional development opportunities for Sparstens and, finally...
I'll, let us know what active his thoughts on what we're seeing thus far.
If we see consistency across the.
The protocol for the studies and ultimately what we're looking to do is to replicate and strengthen the results that we saw from from the duet study in phase two unless noted give his thoughts on what we're seeing thus far.
Unknown Executive: And finally, we will prioritize business development to diversify our growth business model.
Unknown Executive: We are building upon our strong commercial team capabilities to realize the full potential value of our approved end-of-year term.
Broken the data out by community versus the.
Being centers, but that is something that Tom.
Unknown Executive: and Our team is incredibly dedicated.
Unknown Executive: And finally, we will prioritize business sharing to diversify our growth potential and remain throughout the plan in our focus on opportunities for 2020.
Our.
But we certainly will look at in the future.
Got.
Okay, and I think the important point being that.
And so.
CMC meeting recently and Theyve been able to look at a number of different parameters.
By community versus the.
Full.
In centers, but that is something that.
Unknown Executive: Open it up for questions. Our team is incredibly dedicated and talented employees at Retrofit.
Our.
Look at in the future.
The past without duet.
Thank you know the important point being that we had our DMC meeting recently and Theyve been able to look at a number of different parameters.
Unknown Executive: In a position to execute.
Unknown Executive: And we look forward to sharing our progress.
We have as well in a blinded.
Both.
The new product profile are resonating the most in the market.
Unknown Executive: We look forward to sharing our progress on each of these priorities as we move throughout the balance of 2019 and into 2020.
Any surprises or.
So I do.
Well.
As much as I can say.
Center learnings that Youve.
Unknown Executive: 2020
Pound.
Chris: Now, let me turn the call back over to Chris to open it up for questions.
And appeal of 2.0.
Board and then.
So.
You mentioned.
New product profile are resonating the most in the market is there any surprises or.
Kirby: Thanks, Eric. Kirby, can we go ahead and open up the lines for Q&A, please?
Any notable.
Would represent.
Learnings that youve.
Somehow.
Pound.
Kirby: Absolutely. Ladies and gentlemen, if you have a question at this time, please press the star and then...
Yes.
Ken and form the launch going forward and then our.
You mentioned that that some patients are coming back out of the would work.
Sure. Thanks, Thanks, Joe I think overall with regard to the aspects of file that you see.
Unknown Executive: And if your question has been answered or you wish to remove yourself from the queue, you may press the pound key. We'll pause for a moment.
Yes.
During that patients are pleased with the flexibility of being able to take.
Previously.
Unknown Executive: Great. Thanks very much. And congrats on the progress. I was wondering if
Or without food and that was a very important aspect that we heard in the development of the product as well as the market research that we did with patients and physicians before the launch. We also have seen that the average number of pills that a patient.
Hey, the therapy with or without food and that was a very important aspect that we heard in the development of the product as well as the market research that we did with patients and physicians before the launch. We also have seen that the average number of pills that a patient takes.
And with regard to.
Yeah.
Yeah.
Seven.
As we move forward. The important thing is the continued growth that we expect to see in our commercial portfolio and.
Unknown Executive: The first question comes from the line.
Unknown Executive: type of SGF
Our focus.
Thus far again.
Unknown Executive: F-S-G-S, F-S-G-S, or Severity, or other baseline characteristics. Great, thanks very much.
On the identification of new patients given that the majority of patients with cystinuria are not currently treated.
And for those patients that discontinued therapy in the past.
That we expect to see in our commercial portfolio and.
Unknown Executive: Congratulations on all the progress.
Unknown Executive: and those that are being enrolled now.
Unknown Executive: If you've detected any difference in the type of patients in terms of their type of FSGS,
Our focus.
It's a meaningful segment of patients that we want to engage.
The of patients with cystinuria are not currently treated.
And for those patients that discontinued therapy in the past.
Unknown Executive: of those that are being enrolled now from non-centers of excellence.
Teams focused on now.
It's a proportion of the tioga patients that over time, we think it's a it's a meaningful segment of patients that we want to engage.
Unknown Executive: And I think it is important that we can see consistency across the sort of core of the study.
But broadly we're focused on them and new patients to continue the growth that we've seen over the last few years and that's that's largely what our teams focused on now.
Unknown Executive: Are there any differences?
Frank Bento: Frank Bento
Okay.
Hi.
Joe: Joe, thanks for the question. I think, importantly, we see consistency across the protocol for studies and, ultimately, what we're looking to do.
Okay.
Yes.
Thanks again for taking my questions.
Two quick questions.
Okay.
Thanks.
I'm wondering how many.
Our.
Next question comes from the land leases Bacon.
JMP.
Yes.
Good.
Hey, Your line is now open.
Unknown Executive: Ultimately, what we're looking to do is to replicate and strengthen the results that we saw from the DUET study in phase 2.
John Thanks for the question. So we we've not commented on the number of patients that have enrolled.
Unknown Executive: We don't, right at this stage...
Unknown Executive: At this stage, we haven't pulled the data out yet.
Enrolled so.
What we have.
I wonder.
Due to provide guidance on is.
As the timeline for reporting of headline data.
Joe: This is a great question, Joe. The main centers, but let me just say that we are currently blinded to the data set, and we've got...
And we are very pleased with the progress of the study to be able to achieve for duplex that readout.
The number of patients that enrolled.
2021.
We have continued to provide guidance on is the timeline for reporting of headline data.
And we are very pleased with the progress of the study to be able to achieve for duplex that readout.
Unknown Executive: We haven't broken out the data yet, but we have...
Academic sites the two clinical research.
The 21, and I'll hand, it over to know what to to answer the rest of your question.
Unknown Executive: , , , , , , ,
Unknown Executive: We had our DMC meeting recently, and they were able to look at a number of different parameters.
Yes, I think the question around centers of excellence. The majority of these sites would be considered centers of excellence and when I was really referencing was.
Unknown Executive: And we can say that, overall, the data are consistent with what we've seen in the past with Duke.
There are about 300 academic sites to two clinical research in United States. Most of which are academic there are there was a percentage that arent and then there's about 8700 community nephrology.
Right.
The key really for this program one of the key learnings we've had.
Connecting up.
For the color on.
These centers of excellence over too.
<unk>.
Immunity nephrology centers on the registries to broaden out the foundation of.
I have that.
Unknown Executive: I think that's probably as much as I can say.
Of the sites is that that makes sense.
At this stage, it's something that would.
Unknown Executive: and then, um, Theola 2.0.
See accessible to the to the data monitoring committee.
Unknown Executive: And then on the deal of 2.0, what aspects are there...
In say the higher.
And.
Let me let me, let me rephrase that we actually can see that on a blinded.
We don't.
We haven't commented.
Hi.
On that.
We havent looked at that data at this stage, it's something that would.
Joe: You know, points and learnings.
Unknown Executive: Thanks Joe, I think those were all with regard to the prospect of siloed work.
Accessible to the to the data monitoring committee.
And.
Unknown Executive: We're hearing that patients are pleased with the flexion.
We I, let me, let me, let me rephrase that.
Here are you.
See that on a blinded.
Unknown Executive: S. E. C. S. R. O. S. L. A. S. E. S. R. O. S. S. E. S. R. O. S. S. E. S. R. O. S.
Jeremy.
So we have we haven't commented.
On that.
And just one quick clarification question for.
Really just talking about SGN, a in R&D that onetime impairment you kind of have to carve that out.
Unknown Executive: and the development of the
Here are you, saying.
Unknown Executive: The development of the product, as well as the market research, gave us confidence and conditions before the launch.
So Q3.
Are you.
Q4 level will be similar.
The line item SGN, a in a line item of R&D.
Thanks.
Unknown Executive: E.T.
Unknown Executive: We're hearing that patients are pleased with
Unknown Executive: Reflective
Unknown Executive: of Being Able.
Unknown Executive: The therapy can be with or without food.
Unknown Executive: [inaudible]
Unknown Executive: We also have seen that...
Unknown Executive: The average number of pills that a patient takes per day has been reduced.
Thanks, John .
Unknown Executive: by seven for those patients that are.
For what you see you mentioned, 80% being covered by payers.
Your next question comes from the line.
Unknown Executive: Biola EC, and we know that for many patients, that pill burden can be a challenge. That's why, anecdotally, our focus has been early in the launch, and with regard to...
And so the number we're referring to is the number of claims that we've received fourth bio AC prescriptions that have been adjudicated in approved by payers.
Being covered by payers.
We continued evolution at that I think what we're looking for us to ensure that Theres no difference really in the access for Faiola.
We're referring to is the number of claims that we've received four file AC prescriptions that have been adjudicated in approved by payers.
In some layer with as well as you know looking at.
Unknown Speaker: Unknown Speaker Transcripts provided by Transcription Outsourcing, LLC.
Sure that I think what we're looking for us.
To ensure that Theres no difference really in the access for Thiola.
Unknown Executive: there will be a new
Unknown Executive: most recently, we have a new patient in our commercial portfolio and our focus has been on the identification of new patients, given that the majority of patients with cystic aneuria are not currently treated. And for those patients that just continued therapy in the past, you know it's a proportion of the
Okay did in the first.
C.
90 days.
Back to be able to achieve that and.
In.
Access for patients.
Prior launches that I've been familiar with as well as looking at.
Recent assessments of launches, having 80 plus percent of lives covered or or prescriptions adjudicated in the first.
So your proteinuria or something else.
Is a very very strong signs of of access for patients.
The data that we're seeing the baseline data.
And that the data the DMC size consistent with data from do add.
From what we saw into I guess.
Are you there I'm, whether you're referring to proteinuria or something else.
In terms of the 800 milligram 40 milligram dose question.
Staying morry debt.
The data that we're seeing the baseline data.
Seeing any.
Coming in is consistent with what we've seen from a from what we saw in duet I guess.
Unknown Executive: The next question will come from the line-up.
We can get a look at that obviously on a blinded.
So say in in terms of the 800 milligram 40 milligram dose question.
I can add a little bit of color I mean I think.
Unknown Executive: Video by Microsoft Office Word Document MSWordDoc Word. Document.8
The.
Biz.
Greenlight from the data monitoring committee as.
Yes.
Significant safety issues or safety concerns.
Overall.
All flags.
And we can get to look at that obviously on a blinded basis.
So I think thats most important message at this stage in the study.
Failure.
It's also supported by the.
Unknown Executive: That's largely what our team is focused on now. Okay. Great. Thanks again.
On this front.
From the data monitoring committee as well.
The result of having more sites.
Got it that's helpful and then.
Unknown Executive: Thank you again for taking my questions.
For duplex.
Joe: Thanks, Joe. I'm wondering how many patients you have enrolled so far?
We should have.
The positive change in trajectory.
Good about the confidence in the timelines and what we're doing to improve the trajectory here. So I think thats really the Celtics your.
Unknown Executive: of JNP Security
John: We say your line is now open.
Unknown Executive: John, thanks for the question. We've not commented on the number of patients that have been enrolled.
We are and I am confident the timelines.
Yes.
As a part of that is based on.
Good question, let me sort of.
So we realize this is an underserved.
Unknown Executive: And we are very pleased with the progress that's been made.
The confidence in the timelines and what we're doing to improve the trajectory here. So I think thats really the subtext your question.
Unknown Executive: So we've not...
Noah: I'll hand it over to Noah to provide guidance on the timeline for recording that headline data. We are very pleased with the progress of the study to be able to achieve that readout for duplex in the first half of the survey.
Yeah.
We are and I am confident the timelines.
To put in place to establish.
Based on.
What is.
As leaders in Fscs as you know we realize this is an underserved.
Those challenges the way that we've addressed it I think this is why are we at the confidence we're seeing the inflection.
Well, what we learned was there really are.
Sites getting the right sites in.
Yes.
Ensuring that there is active engagement with those sites.
What is.
The physician and a community level.
This in this field so.
You know to get that engagement.
The way that we've addressed it I think this is why we have the confidence we're seeing inflection.
So we're aware of the study.
Ill foundation of sites getting the right sites in place.
Study.
Ensuring that there is active engagement with those sites.
Sure.
Fourth at a physician and a community level and that takes time morry as you know to get that engagement.
Via registries and actually what Thats led to.
It is an increasing enrollment.
Where are the study.
And.
Physicians are aware of the importance of study.
Okay.
And I think that's that's the teams on a really good job there.
Definitely seeing the.
Up to the connectivity to the community sites, we referred to and to the registries and actually what Thats led to.
We are learning.
In increasing enrollment.
Develop better.
Unknown Executive: All right.
Unknown Executive: Yeah, I think the question around centers of excellence...
You said screens.
And they get the right patients in house, So I think it's really a combination of those but.
Please see the.
As such as.
Okay, great come down.
In in recruitment.
That is probably because.
In our ability to execute in the first half of it and I get to date and.
They develop better screening techniques and they get the right patients in house. So I think it's really a combination of those but the main messages.
Unknown Executive: The question on Centers of Excellence, the majority of insights would be on the Centers of Excellence, and what I was really referencing was...
Andrew.
Priests in in recruitment.
Unknown Executive: There are about 300 academic sites that do clinical research.
Sam will win.
It's been us.
Yeah.
And our ability to execute in the first half of and I get the data and.
I think some gina.
Thank you for taking my questions.
Unknown Executive: in the United States, most of which are academic.
Got it that's very helpful. Thanks for taking my.
Follow.
Thanks.
Well then question.
Unknown Executive: There was a percentage that, all right, and then there are about 8,700 community nephrology centers.
Right.
Wondering.
Portion for 190 patients have already.
Jim will win from Barclays.
Thank you Jennifer your question, So we don't comment.
Gina.
Excellent rates and the progress that.
Unknown Executive: And the key, really, for this program, one of the key learnings we've had...
I've seen.
Maybe just one follow.
In the guidance is on the timeline for data readout. So we are absolutely on track to be able to deliver the headline data in the first half of 21.
Unknown Executive: is connecting up the centers of excellence
Unknown Executive: on the registries to broaden out the foundation of...
Unknown Executive: [inaudible]
Unknown Executive: and let me rephrase that we can actually see that on a variety of pages.
Hi, Good unit for your question. So we don't comment on the enrollment rates and the progress that we've seen.
Unknown Executive: We don't. We haven't.
Unknown Executive: We haven't looked at that data at this stage, or anything that would be accessible to the Data Monitoring Committee, and let me rephrase that.
Bye.
Again, our focus is and the guidance is on the timeline for data read out. So we are absolutely on track to be able to deliver the headline data in the first half of 21.
Unknown Executive: We can actually see that on a blinded basis, but we haven't commented on it.
Laura: And just one quick clarification question for Laura. I believe you said you expected an R&D plan.
The time to be able to clean and analyze the data.
C.
Okay.
That enrollment.
Unknown Executive: I kind of have to carve that out. Are you saying just 15 A and RG, or not?
Question regarding.
I'd say mid 2020.
In order to be able to deliver to date.
Unknown Executive: [inaudible]
How much is a from you see.
Unknown Executive: will be similar for the line item SG&A and the line item R&D.
How much is from for.
36 weeks that are required and study and then the time to be able to clean and analyze the data.
Unknown Executive: Yeah, we're really just talking about SG&A and R&D. That one-time impairment, you kind of have to carve that out. So Q3 versus Q4 levels will be similar for the line item SG&A.
Planed revenue for both of those brands, we're not breaking that out.
Oh, sorry.
At this point, it's early in the launch we may consider doing that reporting out later, but at this point it.
Yes.
It's not something that we're in a position to do.
Yes. Thank you for the question Gina So we are reporting the combined revenue for both of those brands, we're not breaking that out.
Unknown Executive: And the line item of our meeting. Hi, everyone. Congratulations on the progress, and thanks for taking my questions. Thanks, John. Thanks, John. To start, for FIOLA EC, you...
At this point, it's early in the launch we may consider doing that reporting out later, but at this point, it's not something that we're in a position to do.
Reflects only too much.
The fair to say like majority.
You know with many launches there was a ramp up period so.
You can imagine that the majority of it is with viola, but I don't want to.
That level of detail, we're not we're not related to position. The do you think about for quarter three.
That reflects only two months of the launch and as you know with many launches there was a ramp up period. So you can imagine that the majority of it is with viola, but I don't want to.
Set a precedent where we're starting to break out those.
Jeffrey: Is that a number that can be improved upon going forward?
Especially early in launch.
Jeffrey: I'm Jeffrey.
Unknown Executive: More thanks for the questions.
Then on for Doug Thanks for taking.
Well. Thank you for taking my question.
Unknown Executive: Thanks for the question. So the number we're referring to is... Hi, everyone. Congratulations on the progress, and thanks for taking my question.
So this is very broad based on general catch all question.
How do you think about.
Going forward, how do you think about.
Okay. Then comes from the line of though Ken.
And just your general thoughts about here.
Your line is now.
Thank you.
Hi, This is Jason on for Doug Thanks for taking our questions and congrats on the quarter.
C.
This is very broad.
In the priorities that that we outlined of continuing the execution of our Sparsentan development programs.
Unknown Executive: Is that a number that can be improved upon going forward? I think what we're looking for is to ensure that there's no difference between access to Biola and biology.
And just your general thoughts about here.
Development certainly is an important one as well we do want to continue to diversify our portfolio and our pipeline.
Please that that we outlined of continuing the execution of our Sparsentan development programs.
You don't feel pressure to do a deal.
Unknown Executive: And, you know, prior launches that I've been aware of, as well as, you know, looking at recent assessments and launches, having 80 plus persons in lives covered or persons adjudicated for 90 days is a very, very small number.
Certainly is an important one as well.
We do and the approach that we take is very.
By our portfolio and our pipeline.
We'll continue to look opportunistically within the rare and ultra rare spaces.
Any deal that we do.
Pressure to do a deal.
Tropin strengths and capabilities.
We feel that we do and the approach that we take is very.
So rare and ultra rare.
That there are opportunities.
Of therapeutic areas.
Unknown Executive: And you mentioned that the date of the DMT is consistent with the date of the launch.
Jan in bringing assets in.
Famous and a bit offensive, what we're thinking about for business development.
Unknown Executive: I guess. Can you provide any more clarity?
We are sensitive to the use of our stock at this current price.
Be aligned in our strategy of focusing on rare and ultra rare.
Stability in looking at assets.
Unknown Executive: Yeah, I know, I guess what I was just saying...
Our cubic areas.
And certainly in a way to not compromise our ability to execute exclusively on the sparsentan.
Unknown Executive: Thank you for your patience.
Unknown Executive: And you mentioned that the data the DMC saw is consistent with data from the U.S. Can you provide any more clarity there on whether you're
We are sensitive to the use of our stock at this current price and we believe that the cash position gives us the flexibility in looking at assets.
Yeah. It's a great question I mean part of what we want to do Opportunistically as look broad, but if you think about where.
Unknown Executive: And I'll also say, in terms of the 800 mg, I'll give you a little bit of color on that. I think the data that we're seeing, the baseline data that we're seeing coming in, is consistent with what we've seen from what we saw on DUET. I guess that's more of a clarification there.
And.
Sure. So this organization.
Thats Great just quick follow up then.
And commercialization.
Assets are you looking for.
Steve we have the opportunity to bring those in without.
Distracting or having to invest significantly to build new capabilities.
But if you think about where.
Our strengths of this organization lie.
Unknown Executive: We are not seeing any significant safety issues or safety concerns.
Come on.
Say, it's more in the late stage development and commercialization aspect, we have the opportunity to bring those in without.
Unknown Executive: and then.
Unknown Executive: And we can get a look at that on a trajectory basis.
Unknown Executive: for Screening Nation
Unknown Executive: It's awesome. We look forward to working with you.
Okay.
Strapping or having to invest significantly to build new capabilities and thats, probably where we will prioritize most of our.
Unknown Executive: How are you doing? You're currently on the...
Unknown Executive: [inaudible]
Unknown Executive: More sites got it, so that's helpful, and then that's really for duplex.
Thanks for taking the question Tom.
Perfect. Thank you so much.
Unknown Executive: For the positive change in trajectory for screening patients that you're seeing, are you referring...
I will let you see launch maybe could you comment on any.
Positive impact you might be see also on.
Well the scripts or.
Unknown Executive: And if so, what are you doing differently on this front, or is the screening improving?
Trends lineups, Christopher Maher right of Nomura.
I only see.
The free your line is now open.
Unknown Executive: are a nice result of having more sites.
I think I recall correctly, you guys were not looking to perform a sort of switch type range for.
Unknown Executive: Good question. Let me sort of, as you know, at a high level, talk about areas and timelines and what we're doing to improve the trajectory here.
Well, let you see launch maybe could you comment.
Cases at this point.
Might be seek also on just I will let scripts or patients below.
Strategy.
Does that potentially panning out.
By earlier.
Unknown Executive: [inaudible]
Typically.
And then secondarily I think I recall correctly, you guys were not looking to perform a sort of switch tight for.
Unknown Executive: Duet, what we learned was there really are...
Unknown Executive: You know, we are, and I am...
Unknown Executive: We are, and I am confident in the timeline that you need to put in place.
We are seeing.
The continued growth of new patients.
Yeah.
Treated.
Unknown Executive: [inaudible]
I want to.
Area, and we're very pleased with that progress.
Unknown Executive: for the largest study in community law in this field. So setting up those challenges, the way that we've addressed them...
Including new patients coming on to file that you see.
For your questions first with regard to new patients we are seeing.
Orange is is there patient demand.
Patients.
Treated for cystinuria, and we're very pleased with that progress.
Unknown Executive: [inaudible]
Unknown Executive: There is increasing enrollment and increasing screenings. I could just say that teams did a really good job there. We've definitely seen the screen fail rate come down somewhat, and I think that is probably because of bad site learning and increased enrollment.
I think patient.
Being new patients coming on to file that you see.
The profile.
I think part of what we're looking at as as early signs of the launch is is there patient demand and the patient demand.
Okay.
Come not just from new patients, who learn about the treatment options.
So for existing patients who may look too.
Position.
Aspects of the profile.
Unknown Executive: , , , , , , , , ,
Unknown Executive: In our ability to access sites, they are learning and getting trained; they are developing...
Yeah.
Those are two sources of filed E C up.
We have seen.
Ill coming back to therapy.
Likely on file that you see given the the improved profile.
Unknown Executive: better screening techniques, and they get the right patients in-house. So I think it's really a combination.
Okay.
For those patients.
Who's going to come from new patient acquisition.
So were in line with.
And it will be bolstered by patients that may have discontinued thiola coming back to therapy.
Unknown Executive: Of those, but the main message is:
Likely on file that you see given the the improved profile for for those patients.
Unknown Executive: has really given us confidence in our ability to execute in the first half.
Gs any.
And we're pleased that were in line with.
Unknown Executive: Thank you for taking my question.
Andy filings in the near term maybe.
Unknown Executive: That's very helpful. Thanks for taking my question.
And setting us up for continued growth next year.
Chris for that I'll ask.
Bill wrote to give his comments on the.
Your.
R&D collaborations the end by one.
Yes any.
Last question there in the filings in 2020 the answer is no.
Unknown Executive: for it.
Collaborations art.
Jim: And your next question comes from the line of Jim.
Adam maturities stage.
Chris for that I'll ask Bill wrote to give his comments on the.
A lot of production on July one.
Certainly thanks for the question.
And.
Broadly.
Ask question there the island filings in 2020, the answer is no.
Gina: Thank you, Gina, for your questions.
Gina: This is Gina. Thank you.
Collaborations art, Adam maturities stage.
Unknown Executive: [inaudible]
Unknown Executive: Again, our focus is, and the guidance is, on the timeline for data readout, so we are absolutely on track to be able to deliver the headline data in the first half of 2021.
The teams.
As well as the efficacy organization in Britain patients into.
Early stages and thanks for the earliest stages.
Discovery research.
We find that.
Thanks.
To be very productive.
In early stage, so we'll have more on that in the future but.
Great.
Unknown Executive: Thank you for your questions.
That's a current.
Unknown Executive: So, we don't comment on the enrollment rates and the progress that we've seen. Again, our focus is, and the guidance is, on the timeline for data readout. So, we are absolutely on track to be able to deliver the headline data in the first half of 2021. And then there is time to clean and analyze the data.
Yes, as well as the advocacy organizations in three patients into.
To be very efficient with our resources, but be able to go into areas of high unmet need and potential innovation from a scientific.
More of an early stage, so we'll have more on that with future but.
Then I guess sort of any.
Current status.
Frank E filing.
Versus a very important model for us to explore.
Material.
The very efficient with our resources, but be able to go into areas of high unmet need and potential innovation from a scientific.
12.
Yeah.
Regarding these.
However, the and then I guess sort of any.
Unknown Executive: [inaudible]
Clinical path forward.
Unknown Executive: In order to be able to deliver the data,
Randy filings do you expect any.
Unknown Executive: Yeah, yeah, I think that's...
I think with discovery research.
Gina: Thank you for the question, Gina. So we are requiring the combined revenue for both of those grants. We're not breaking that out.
Just to predict.
Due to that.
To arise from.
Over the next 12 month period, but certainly if those events occur we would come back.
Starting these programs point points that might help de risk the clinical path forward.
Great. Thank you.
Hello.
Thanks, Chris.
Well I think with discovery research, it's difficult to predict where it's going to.
The line.
Over the next 12 month period, but certainly if those events occur we would come back.
No.
Hi, Thank you for for taking.
Great. Thank you.
Can you talk a little.
Yes.
Okay.
The focus area.
For the deal.
Question comes from the line Michelle Gilson.
Of Canaccord.
Okay.
Yeah.
Correct.
Im showing your line is now.
Yeah.
And and then.
Hi, Thank you for for taking my question can you maybe talk a little.
Unknown Executive: at this point.
Unknown Executive: At this point, it's early in the launch, so we may consider doing that and reporting out later, but at this point, it's not something that we're anticipating.
Yeah.
Okay.
The key areas.
Area.
Yeah when you.
For the Dan.
Yes.
Ian you know why.
Ken.
Additional rare kidney.
Just to recommence proceeding.
Thank you Michel asking though it to take those two questions.
Unknown Executive: At this point, it's early in the launch. We may consider doing that and reporting out later, but at this point, it's not something that we're in a position to do.
Hi level.
Thanks, Dan can you just talk about.
Pete.
Some of.
It is generally focused as their primary focus.
Thanks.
Safety.
It seems person can into additional rare kidney indication.
Unknown Executive: So it is fair to say that the majority of the revenue is defunding progressives.
I didn't see.
Making sure there are no signals concerning signals.
Unknown Executive: The majority of it is his bio, but I don't know why.
Because two questions.
Unknown Executive: That level of detail we're
Since earlier they are unblinded, we don't actually.
Unknown Executive: We're not really in a position to do, you know, if you think about quarter three, you know, that reflects
It is generally focused as their primary focus.
The TMC into.
It does.
Flooring that looking carefully at adverse events CES.
Unknown Executive: Thank you for taking my question.
Unknown Executive: You know, set a precedent where we're starting to.
When you get the Green light because.
Concerning signals.
And as mentioned earlier they are unblinded, we don't actually.
Unknown Executive: ..
Unknown Executive: especially early.
Unknown Executive: early in launch.
Unknown Executive: Okay.
Unknown Executive: Well, thank you for taking my questions.
And so.
I see what they look at its really between the DMC into blinded.
Unknown Executive: Your next question...
To protect and duplex of course not surprising.
Unknown Executive: [inaudible]
Jameson: Thank you. All right. This is Jameson. On for Doe. Thank you. Yeah.
Eight and.
What data set and we've got a large.
When you get the Green light because.
To draw from and clearly have seen.
And ask for anything and everything.
Does safety.
From that study, which I know you're familiar with as well.
Unknown Executive: How do you think about going forward, and how do you think about profitability versus bar signs and development?
That was really a good signal for us for both protect and duplex of course not surprising.
Yeah.
Given that we've got to do what dataset and we've got a large.
Percent 10.
The dataset to draw from and clearly have seen.
You know some good good safety.
Not study, which I know you're familiar with as well.
Vince.
So I think thats that's.
You know, we're not ready to commit to.
DMC discussion.
We have encouraging data.
Outlook on Sparsentan, we did allude to in the script.
And at HSN, we have upcoming our alport.
Hi.
From a in additional indications.
Well this that thats something definitely look at.
Ready to commit to at this stage.
Going to the.
Encouraging data.
Its valuation.
The mouse model in terms of proven of Egypt kidney function.
When we talk to the advisors.
An important.
Retail wells.
Hum.
Pardon finding patients and they've told us that thats something definitely look at.
And angiotensin with regard to committed disease. It does open up other opportunities.
Our valuation.
Continues beyond Fscs again, and even alport.
Talk to the advisers.
We are looking at from a feasibility.
Because of the common pathway of both.
You know there are some other things were looking at too.
And with regard to committed disease. It does open up other opportunities.
John We certainly will come back and share with.
I'll port.
And these are things that we are looking at from a feasibility standpoint.
Longer term and.
Yes.
There are some other things were looking at too.
Which when we're we're comfortable.
We've got more clarity on we certainly will come back end and share with you.
Thanks for taking the questions.
Just wondering if you could talk a little on.
How you're managing.
Thanks Michelle.
So attention in the.
Just trials that are ongoing and.
And your last question comes from the line.
As Tim Lugo.
No.
William.
Monitoring can be.
Before the interim readout.
Hey, This is welcome Tim Thanks for taking the questions. I was just wondering if you could talk a little on.
Okay.
Yeah.
Sway I think to answer that question is looking at duet.
Trials that are ongoing and.
Yes.
Yeah.
A bit of fluid retention, which was managed well with direct.
Morning.
And we did not see heart failure there.
And so that was extremely encouraging because as you know I think the backdrop for the question is.
The best way I think to answer that question is looking at duet.
Unknown Executive: We do want to hear from you.
Attention with the class, we're not seeing that.
Unknown Executive: Yeah, so I'd say our strategy...
A bit of fluid retention, which was managed well with direct.
Looked at that.
And we did not see heart failure there.
Unknown Executive: We want to make sure as well that we do want to continue to diversify our portfolio and our population.
Couple of other.
Purging because as you know I think the backdrop for the question is.
Silly to look at both studies together gives the DMC greater kind of data and power to identify if there are.
Specifically on within our duet data set we looked at that.
With the progression of the study, but they're also based on.
Unknown Executive: Will continue to look opportunistically within the rare and ultra-rare spaces.
Couple of other points here Laughlin first of all.
Paul to be able to manage.
With studies together gives the DMC greater kind of data and power to identify if there are.
Unknown Executive: We want to make sure that any deal that we do and the approach that we take is very disciplined. We do think that there are opportunities in the near term for value creation.
And with these drugs.
Though it is encouraging that they're continuing with the progression of the study, but they're also based on the of the approach with duet.
Aspects of the protocol to be able to manage.
These patients and.
And retention.
This important.
The key really is you're going to see some fluid retention with these drugs.
Question is is it manageable.
Alright Committee are there any more planned before the.
Unknown Executive: And we want to make sure that any...
Thanks.
I think we've heard that direct use.
Unknown Executive: We believe that the cash position gives us flexibility in looking at therapeutic areas.
Yes, we I can say that there are planned future.
Jameson: And I hope that gives you, Jameson, a bit of a sense of what we're thinking about for business development. We're sensitive to the use of our stock at the current price, and we believe that...
Since and.
Thanks.
The them on this important therapy.
I haven't disclosed the timing of those meetings, but when they occur.
And just on the data monitoring committee are there any more planned for the full the interim.
Unknown Executive: with the
Findings are.
Yes.
Unknown Executive: And if they answer my question. Yeah, it's a great question. I mean, part of what we want to do opportunistically is look broadly, but if you think about where this transition is going,
There are yes, we I can say that there are planned future.
Question.
See meetings.
We havent disclosed the timing of those meetings.
There are no further questions.
Correct.
I would like.
Courtesy of course, we we'll certainly update this group and let you know.
Yeah.
Binding.
Thanks for joining us today. This concludes our call we look forward to updating you in near future on our progress.
Unknown Executive: Major
Unknown Executive: Unknown Speaker Yeah, it's a great question. I mean, part of what we want to do opportunistically is look broadly, but if you think about where the strengths of this organization lie, I would say...
Question.
Thanks, so much to our presenters and to everyone Cooper.
Yeah.
I would like to turn it back to Mr.
Thanks.
Great. Thank you Kirby. Thank you everybody for joining US today. This concludes our call. We look forward to updating you in near future on our progress.
Thanks, so much drove presenters and to everyone who participated.
And you have a great. Thanks.
Okay.
Unknown Executive: I have the opportunity to bring those changes in.
Unknown Executive: [inaudible]
Unknown Executive: Distractions or having to invest significantly to build new capabilities, and that's probably where we'll prioritize most of our
Unknown Executive: Perfect, thank you so much.
Unknown Executive: [inaudible]
Christopher: Christopher, you're alive as well.
Unknown Executive: I wanted to ask about the IOLA-ED launch; maybe you could comment on the indication...
Unknown Executive: Transcripts provided by Transcription Outsourcing, LLC.
Unknown Executive: First, with regard to new patients, we are seeing the continued growth of new patients treated for cystaneuria, and, you know, we're very...
Unknown Executive: Progress, including new patients coming on to Fioli.
Unknown Executive: Thank you for your questions.
Unknown Executive: We are seeing...
Unknown Executive: They are seeing the continued growth of new patients.
Unknown Executive: You know, we're very pleased with...
Unknown Executive: And, you know, I think part of what we're looking at is the early signs of...
Unknown Executive: Unknown Speaker In terms of a switch strategy, that's not the approach. You know, a strategy for a clinical portfolio is contingency.
Unknown Executive: about the treatment options but also
Unknown Executive: [inaudible]
Unknown Executive: For more information, visit www.fema.gov.
Unknown Executive: is going to come from New Patient Acquisition that, you know, we're largely...
Unknown Executive: you know likely on file what you see given the improved profile for those
William E. Rote: Thank you, Chris, for that. I'll ask Bill Rote to finish his comments on the program.
Chris: Any updates with respect to those and potential IMD filings in the near term, if you intend to publish them?
Chris: Thank you, Chris.
Unknown Executive: We have had a lot of production on this slide, one, I think this question has been the last question for me, and both collaborations are in production now. However, the collaborations aren't at a maturity stage to be to the point where we're triggering that epitome of the organization.
Unknown Executive: [inaudible]
Unknown Executive: Just getting off the ground and in more of an early stage, so we'll have more on that in the future.
Unknown Executive: is a very important model for us.
Unknown Executive: Innovation from a scientist's panel
Unknown Executive: Thank you. 12 minutes.
Unknown Executive: [inaudible]
Unknown Executive: Very researchable, difficult to predict where it's going to be over the next 12 months, but certainly, if those events occur, we would come back.
Chris: Thanks, Chris. Well, I
Unknown Executive: Where is it going to go?
Unknown Executive: from the line over the next 12-month period, but certainly, if those events occur, we will come back to them.
Unknown Executive: Hi, thank you for taking my call. Thank you.
Unknown Executive: Talk a little bit.
Unknown Executive: about the focus area for the NAD
Michele Gilson: This next question comes from the line of Michele Gilson of Canaccord, Jan.
Unknown Executive: planned and then hi, thank you
Unknown Executive: Can you maybe talk a little bit about some of the areas that we're looking at when you get a chance?
Unknown Executive: [inaudible]
Unknown Executive: ...
Unknown Executive: Can you just talk about some of the areas that you're looking at when you think of bringing them?
Unknown Executive: Adversive
Unknown Executive: [inaudible]
Unknown Executive: And, as I mentioned earlier, they are all online.
Unknown Executive: have an opportunity to see what they've looked at and what they've seen.
Unknown Executive: ...ensuring that, looking carefully at......so it's actually pretty encouraging......making sure there are no signals......concerning signals...
Unknown Executive: We don't actually have an opportunity to see what they've got. That was really a good signal for us. We're both protected.
Unknown Executive: And Duplex, of course, not surprising, given that we've got the duet data set, we've got a large... So it's actually pretty encouraging when you get the A3 data set to draw upon and fill in. They're really getting a full data set. They can ask for anything and everything, and they often do, from that point of view, which I know you've learned as well. So I think that's a good signal for us. We're both protected. And Duplex, of course, not surprising, given that we've got the duet data set. We've got
Unknown Executive: Mr. Troughproblem, Lillia Screams
Unknown Executive: [inaudible]
Unknown Executive: On this page, we certainly have a large amount of data.
Unknown Executive: And at AF10, we have an upcoming event, our outport.
Unknown Executive: As well as hearing, which is an important...
Unknown Executive: Carewell's
Unknown Executive: [inaudible]
Unknown Executive: Other opportunities; a broader opportunity.
Unknown Executive: Alport. These are things that we are looking at from a feasibility standpoint in the longer term, and you know there are some other things we're looking at too, which you know when we're you know we're comfortable, we've got more clarity on, we certainly will come back.
Timothy: Tim, thanks for taking the question. I was just wondering if you could talk a little about how you're managing the fluid retention in the Sparsentin trials that are ongoing and whether you have any more or expect any more data monitoring.
Timothy: Thanks for taking the question; I was just wondering if you could talk a little.
Timothy: Timothy
Timothy: Tim Harrison
Unknown Executive: That's the best way I can think of to answer that question.
Unknown Executive: Whether you have any more...
Unknown Executive: Whether you have any more or expect any more, I've got them all.
Unknown Executive: And so that was extremely encouraging to know, I think, the backdrop.
Unknown Executive: A bit of fluid retention.
Unknown Executive: fluid retention, which was managed well with diuretics, that we looked at that, and
Unknown Executive: And we did not see, you know, heart failure there.
Unknown Executive: [inaudible]
Unknown Executive: Heart Failure Rate Reduction with a Class. We're not seeing that. You know, specifically within our duet theaters, we looked at that.
Unknown Executive: continuing
Unknown Executive: And I think, you know, let me add a couple of other people.
Unknown Executive: First of all, the ability to look at both studies together gives the DNST greater data and power to identify if there are signals emerging, so it is encouraging that there is consistency.
Unknown Executive: with the
Unknown Executive: And I think we've heard that with diuretics there are aspects of the protocol to be able to manage, you know, actually.
Eric: I think that's right, Eric. I think the key really is, you know,
Unknown Executive: https://www.kenhub.com
Unknown Executive: http://TheBusinessProfessor.com
Unknown Executive: And I think we've heard that, you know, with diuretics, there are, yeah, I can say that there are planned future DMC meetings.
Unknown Executive: [inaudible]
Unknown Executive: Thank you.
Unknown Executive: [inaudible]
Unknown Executive: Yeah, there are. Yeah, I can say that there are.
Unknown Executive: are.
Unknown Executive: We haven't disclosed the timing of those meetings.
Unknown Executive: All this courtesy, of course, we...
Kirby: Great, thank you Kirby. Thank you everybody for joining us today. This concludes our call.
Unknown Executive: Thank you. We look forward to updating you.
Unknown Executive: Thank you.
Unknown Executive: Thank you so much to our presenters and to everyone who participated.
Kirby: And you have a great day. Good. Thank you, Kirby. Thank you, everybody, for joining us today. This concludes our call. We look forward to updating you in the near future on our progress.
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