Omeros Corporation Q1 2023 Earnings Call
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Once again, please continue to hold your conference call will begin momentarily.
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Ladies and gentlemen, please continue to hold your conference call will begin in less than a minute.
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Good afternoon, and welcome to today's earnings call for <unk> Corporation. At this time, all participants are in a listen only mode. After the company's remarks, we will conduct a question and answer session. Please be advised that this call is being recorded at the company's request and a replay will be available on the company's website for one week from today.
I'll turn over the call to Jennifer Williams Investor Relations for Maris.
Good afternoon, and thank you for joining the call today I'd like to remind you that some of the statements that will be made on the call today will be forward. Looking these statements are based on management's beliefs and expectations as of today only and are subject to change.
All forward looking statements involve risks and uncertainties that could cause the company's actual results to differ materially. Please refer to the special note regarding forward looking statements in the company's quarterly report on Form 10-Q, which was filed today with the SEC and the risk factors section of the company's most recent annual report on Form 10-K.
For a discussion of these risks and uncertainties now I would like to turn the call over to Dr. Greg Demopoulos, Chairman and CEO of Amerigas. Thank.
Thank you Jennifer and good afternoon, everyone. Joining me on today's call are Mike Jacobsen, Nordea dock, Cathy Melfi and Steve would occur.
Our respective heads of finance commercial regulatory and clinical.
I will start today with a brief overview of our first quarter 2023 financial results followed by a corporate update and Mike will then provide a more detailed financial summary, before we open up the call to questions.
So now let's look at our financial results for the first quarter.
Our net loss for the first quarter of 2023 was $33.7 million or 54 cents per share compared to net income of $128 $7 million or $2.05 per share in the fourth quarter.
Last year.
This difference is the result of the merger related 200 million dollar milestone that was achieved in the fourth quarter.
Payment of the milestone together with accrued interest was later received from Rainer surgical in February of this year.
Cash burn for the first quarter of 2023 was $23.6 million.
Down from 26 million in the prior quarter.
Net sales of Omidria at $37 million were in line with expectations for Q1, which historically is the weakest quarter of the year.
Omidria royalties for the quarter were $9 $2 million, reflecting the new 30% royalty rate on U S. Net sales of Omidria following our achievement of the milestone and receipt of the milestone payment.
The 30% royalty translates to more than 40% of the operating profit from Omidria total sales.
As of March 31, 2023, we had 371 $4 million of cash and investments available to support ongoing operations.
This amount provides <unk> the flexibility simply to pay off the $95 million of convertible debt that matures. This November while still funding operations and advancing our multiple programs well in to 2025.
We expect our royalties from Omidria to continue to grow.
As mandated by recent congressional legislation.
Omidria now has separate payment from CMS in ambulatory surgery centers until at least January 2028.
No later than January 2025, CMS will also pay separately for Omidria when used in hospital outpatient departments or <unk>.
Further we understand from Rainer that they expect to expand the U S sales force as well as to begin selling omidria internationally later this year.
Oh, one additional piece of financial information for our shareholders are the rebalancing process for the Russell U S indexes is underway.
Friday April 28th was ranked day at the close on that day index membership eligibility was determined based on market capitalization.
Multiple banking model show that Don Merril once again will be part of the Russell 3000 index, which based on those models is expected to drive index related buying in the open market of seven 5 million to 8 million shares of bone marrow stock by the 26 of <unk>.
X months.
Date, when the market opens with the newly reconstituted index.
Let's turn now to our program updates starting first with our family of agents targeting <unk> two the effector enzyme of the lectin pathway.
In our supplement is our lead antibody against mass to then our supplemental biologics license application or BLA.
For hematopoietic stem cell transplant associated thrombotic, microangiopathy or Ta TMA is pending with FDA.
Consistent with direction from Fda's office of new drugs in response to our formal dispute resolution request and working closely with our regulatory and legal advisers led by Heinemann Phelps Mcnamara, we have submitted to the division of non malignant hematology.
<unk> proposed plan.
To assess already existing clinical trial data together with external data on both response rate and survival.
We are meeting with the division this month.
To discuss the details of our proposed analysis and to confirm the information required by FDA.
To support Resubmission of the BLA for <unk> approval.
Once the required package is confirmed we expect to be able to use substantially the same package for our European regulatory submission.
As well.
The objective of course is to bring <unk> to market as quickly as possible, helping patients with life threatening ta TMA for whom there is no approved treatment.
Alexia <unk> and Astrazeneca are also pursuing this indication with their C. Five inhibitor Ravi Alyssa map.
As evidence of the difficulty in conducting a clinical trial in Ta TMA information available.
On clinical trials Dot Gov indicates that Alexia on just delayed its estimated study completion date by 16 months to June 2025, while at the same time.
Reducing the targeted number of enrolled patients by nearly 40%.
Clinician support for use of in our supplemental to treat Ta TMA continues to grow.
And a number of additional manuscripts have recently been published.
One in the thrombosis Journal describes a six year old girl, who received a stem cell transplant or severe aplastic anemia, and then developed TMA.
Treatment with the February tide was tried but failed.
She then received in our supplement and her TMA resolved.
Here also we have a convincing example of challenge re challenge.
Is that a parvo virus infection triggered a second episode of TMA and this child.
This recurrence of TMA was again successfully treated with in our supplement.
Another manuscript is in press at clinical and experimental immunology. This one examining patients from our Ta TMA pivotal trial the.
The authors of group of clinical and research investigators from Weill Cornell Medicine a.
Evaluated clinical plasma samples from our trial patients treated with <unk>.
The authors showed that increased levels of caspase eight.
Biomarker of endothelial injury are highly correlated with Ta TMA.
When looking at the patients in our pivotal trial those patients that responded to in our supplement.
A marked reduction.
And those caspase eight levels, while non responders did not.
These data might well help explain why some of our pivotal trial patients did not respond to <unk> and further strengthen our understanding of <unk> mechanism of action and Ta TMA.
Growing.
Physician support for use of our <unk> Ta TMA is also reflected in the frequent presentations at recent and upcoming scientific Congresses.
The annual meeting of the European Society for blood and marrow transplantation already BMT.
There were multiple presentations on the use of <unk> to treat Ta TMA.
And reports of both pediatric and adult patients all of whom had failed treatment with <unk>.
Or both <unk> and <unk> <unk>.
<unk> was given under compassionate use.
And TMA is resolved.
Just this week at the annual meeting of the American Society of pediatric Hematology oncology, which begins tomorrow.
Another transplant expert will detail the course of a three year old little girl with recurrent high risk cancer.
After her second stem cell transplant, she developed a refractory ta TMA here again.
<unk> attempted to treat her with first <unk> and then to February Todd.
Both failed.
And our <unk> requested under compassionate use successfully treated her ta TMA.
Let's now turn.
Two are in our supplemental <unk> phase III clinical program.
In Iga nephropathy.
We remain on track to read out nine months proteinuria data next quarter.
We expect that these data will form the basis for both a BLA in the U S and a marketing authorization application or MAA in Europe .
Our team is committed to making our <unk>. The first approved complement inhibitor and what's expected to be a multibillion dollar Iga nephropathy market.
As a N ta TMA, there's strong physician support for use of <unk> in Iga nephropathy. The disease continues to represent a significant unmet need despite recent market entries of a steroid.
And our blood pressure medication.
Our recent in our supplemental presentation at the annual meeting of the Korean Society of Nephrology was the first reported use of complement inhibition in a patient with recurrent Iga nephropathy.
<unk> stabilized, both proteinuria and Egfr and.
And these beneficial effects were further confirmed by kidney biopsy.
Also another manuscript authored by a group of international experts and recently accepted for publication in kidney International details the role of the lectin pathway across the important pathophysiological components of Iga nephropathy.
Namely glomerular injury, thrombotic microangiopathy and two below interstitial fibrosis.
As previously discussed our other and our thoughtful of Mab Phase III program in atypical hemolytic Uremic syndrome remains a low priority for us.
Turning to our <unk> and COVID-19, as well as acute respiratory distressed distressed syndrome or <unk>.
Our work continues at <unk> labs, and the University of Cambridge, where we are collaborating with multiple U K consortia in the study of acute severe and long COVID-19 Amanda.
A manuscript detailing the central role of <unk> pathway inhibition in well established in vitro and animal models of both COVID-19, and Rds has been submitted to and reviewed for peer reviewed publication and.
And we expect that it will be accepted.
Discussions are ongoing with relevant branches of the U S government, which have been public.
Their focus.
And their intention.
To fund development of agents to treat both Covid and.
Yes.
Yeah.
Now, let's look at all of them is $10 29, our long acting next generation antibody targeting masked two and the lectin pathway.
<unk> M. S. $10 29 is designed for chronic use.
Earlier this year, we successfully completed the phase one single ascending dose clinical trial and data support our plans for once quarterly subcutaneous or intravenous dosing of LMS $10 29.
There have been no safety concerns and dosing in the RMS $10 29, multiple ascending dose study in healthy subjects is scheduled to start this summer.
We're also nearly ready to select our lead drug development candidate from our orally active masked two inhibitor program.
Our strategy here of developing an orally available <unk> inhibitor together with in our supplement and our long acting follow on antibody LMS $10 29.
Could well enable <unk> to control first line therapy for most if not all lectin pathway related diseases.
Okay. So we've completed the update on our mast two and lectin pathway inhibitors lets turn now to the other major part of our complement franchise mass three <unk> 906.
Well, that's 906 is our lead antibody targeting mass three of the key activator of the alternative pathway of complement.
Based on our data and those generated around our potential competitors, we believe that masked III and <unk> 906 are the premier target and therapeutic <unk>.
For indications related to the alternative pathway.
We recently disclosed publicly Dave.
Data from our pre specified interim analysis of our clinical trial of <unk> 906 in treatment nave patients with paroxysmal nocturnal hemoglobinuria or <unk>.
A life threatening form of hemolytic anemia.
We've continued enrolling and following patients in this trial before sharing with you. Our most recent data, let's first review the expected advantages of masks III and <unk> 906 over other alternative pathway targets and drugs either on the market.
Or in development.
First mask three is the most upstream target in the alternative pathway, it's in hip its inhibition.
Leaves the infection fighting function of the classical pathway intact.
This is a meaningful advantage oversee three and <unk> inhibitors, which block the classical pathways adaptive immune response and increase infection risk.
Second masked III is known not to be an acute phase reactants and has very low native circulating levels relative to other alternative pathway targets.
Together these translate to a substantially lower risk of breakthrough occurrence of the underlying disease with LMS 906, then with C. III C five or factor B inhibitors, all of which are acute phase reactants, meaning that.
In the setting of inflammation, such as even an infection or any other inflammatory condition.
Circulating concentrations of the target can increase.
Beyond the inhibitory capability of the drug's dosing.
Leaving a patient less protected from their life threatening disease.
The third potential advantages better patient convenience and compliance dosing for <unk> 906 should be once quarterly either subcutaneously or.
Our intravenously.
This is expected to be more convenient for patients improving their compliance with treatment compared to other alternative pathway inhibitors that are marketed or in development.
Now here are some important updates since our recent press release on our trial data of <unk> 900, <unk> in treatment naive <unk> patients.
Enrollment as I said is ongoing.
And for these data and know where the number of patients ranges from three to nine depending on where the data points fall on the timeline earlier time points capturing more patients.
We'll look first at hemoglobin.
So as a reference point mean baseline hemoglobin in the study is six eight grams per deciliter, the laboratory normal hemoglobin range for women.
Is 12 to 15, six grams per deciliter, and four men 13, 5% to $17 two.
So at day 29, and our study the mean absolute increase in hemoglobin is three four grams per deciliter.
6.3 at day 85, and at day 113.
Mean absolute increase in hemoglobin. It is 9.7 grams per deciliter.
So.
<unk> hemoglobin levels have been achieved.
By day 29, following only the first dose 67% of patients increase their hemoglobin level by two grams per deciliter or more.
And after only two doses.
All patients achieved this threshold of a two gram increase with 80% of them achieving at least twice that.
Meaning an increase of four grams per deciliter or more.
My day 85, two thirds of the patients had a hemoglobin level of greater than 12 grams per deciliter and by day 113.
All patients.
Had a hemoglobin level of 15, seven grams per deciliter or higher.
So by definition, they all had been restored to well within normal hemoglobin levels.
Also at all time points, the mean reduction in absolute reticulocyte counts from baseline.
Was at least 70000 per microliter, ranging from 70 to 136000 per microliter.
Hemoglobin improvement remains statistically significant at all time points throughout the study.
And no patient.
Has had a clinical breakthrough none have had a thrombotic event and none have required a transfusion, while receiving <unk> treatment.
Now, let's examine the LDH data.
Main baseline LDH is $19 31 units per liter that.
Nearly eight fold higher than the upper limit of normal.
15 days after the first dose of <unk> 906, the main reduction in LDH from baseline is 83%.
And at day 29, the main reduction in LDH is 80%.
At the last available time point following four doses day 113, LDH reduction is 87% with.
With all patients, having either normal LDH or LDH levels less than one five times the upper limit of normal.
Just like the hemoglobin data LDH reduction remains statistically significant at all time points throughout the study.
It is important to point out here that all of these data result from the lowest dose of <unk> 900, <unk> that we plan to evaluate in this study and we're now preparing to move to higher doses and exposures to allow for a longer dosing interval.
Yet even at this lowest dose or hemoglobin in LDH results compare very favorably to the detailed and publicly available data on other alternative pathway inhibitors on the market or in development.
This comparison is even more impressive.
And that of the nine <unk> patients enrolled to date in our study in addition to the Red cell destruction caused by Pn H. Two patients also have a plastic in EMEA, which suppresses the production of red blood cells and two others have myelodysplastic syndrome.
What type of cancer that blocks, the production of mature red blood cells, and often increases LDH levels.
So all of them as 906. Despite these challenges appears to be performing extremely well in all of these <unk> patients.
Second Oems 906 clinical trial and <unk> is ongoing this trial has a switchover design enrolling <unk> patients receiving <unk> Mab, then, adding <unk> 906 to provide combination therapy with Raviolis Mab for 24 weeks and then.
Bidding <unk> 906 monotherapy in patients who demonstrate a hemoglobin response with combination therapy.
Treatment with <unk> 906 has already begun.
We also have a third clinical trial evaluating <unk> 900, <unk>. This one treating patients with complement <unk>, Mary Allopathy or <unk>, a rare kidney disease.
Here, we plan to provide data updates really on all three of our PSNH trials in the second half of this year.
And we plan to present data as well at upcoming medical Congresses.
The data to date.
<unk> really clearly demonstrate that <unk> 906 is a highly effective inhibitor of <unk> three and of the alternative pathway.
Given those data and the requirement of nearly complete inhibition of the alternative pathway to be effective in Pn, H, which <unk> 906 has met.
We're confident that our drugs should perform well across the other alternative pathway diseases and disorders.
Those other indications already have been and continue to be identified and validated by our competitors.
We're already in the field.
This should significantly accelerate our development programs given that much of the guesswork.
It has already been removed.
Our next steps are to evaluate higher doses of <unk> 906, so that we can quickly identify the optimal dose for phase III trials.
The aim is to initiate and complete phase III trials across multiple indications as soon as possible.
And the team is focused on and committed to achieving this goal.
Turning now to all in that five to seven or PD seven inhibitor program.
We have shown and published that <unk> seven inhibition blocks, both craving and relapse across substances of abuse.
With positive data in animal models of opioid cocaine nicotine and alcohol addiction.
<unk> seven inhibition in animals is also effective in compulsion, specifically, we've shown that in binge eating.
In addition to blocking both craving in relapse <unk> seven inhibitors do not appear to depress the reward system a significant advantage over current anti addiction agents, all of which do depress the reward system.
So while having only a limited effect on craving. These other agents can cause a reduction in the enjoyment of other aspects of a patient's life, making compliance with treatment quite challenging.
And all of the animal and human Phase one clinical studies run with all of them as five to seven we've seen no evidence of reward system depression.
This would be a major differentiator between <unk> five to seven.
And frankly, <unk> seven inhibitors in general.
Over other anti addiction agents on the market or in development.
Last month, we announced the award of a three year $6 7 million grant from the National Institute on drug abuse or Nader to support the continued development of our lead orally administered <unk> inhibitor <unk>.
An estimated 1.4 million people in the U S have cocaine use disorder and more than 24000 Americans died in 2021 from cocaine overdoses.
Research by our team and others shows that the mechanism of cocaine use disorder, specifically tied to the dopamine system in the brain is the same or highly similar to that of other addictions, and compulsions, including opioids nicotine alcohol and Ben.
<unk>.
One in six Americans 12 years of age or older experienced a substance use disorder in 2021.
And the societal cost of epidemic substance abuse runs well into the trillions.
We look forward to working with Knight and unexpected PD <unk> inhibition could be.
A major part of the solution.
To this massive societal challenge of addiction.
Merrell also controls broad intellectual property surrounding <unk> seven inhibition in movement disorders with our collaborators at Emory University, we're evaluating all of them as five to seven as a potential treatment for L. Dopa induced dyskinesia or lid. These.
These dyskinesia are crippling involuntary movements in Parkinson's patients caused in good part by prolonged treatment with L. Dopa.
L dopa as the most prescribed and most effective drug used to treat Parkinson's disease itself and as a result lid represents a large unmet patient need and a substantial market opportunity.
More than 10 million patients are living with Parkinson's worldwide and reportedly 50% of more of those treated with L. Dopa.
Suffer from lit only one drug extended release Amantadine is approved for the treatment of lids, but its use is limited by suboptimal efficacy as well as by numerous and significant adverse side effects.
The investigators at Emory have developed a primate model of led that is highly predictive of clinical efficacy and have evaluated RMS five to seven.
Extended release Amantadine has also been evaluated extensively in the MRE model.
We've recently received data on <unk> five to seven from memory, and we'll disclose them publicly.
After filing all appropriate patent applications.
Well end todays corporate update with our immuno oncology programs. There are two broad subsets or platforms of our Io franchise, namely cellular and biologic therapies.
And we're building expansive patent to states around each.
Our cellular platform is comprised of novel approaches to adoptive T cell and car T therapies. Both of these follow from our work on <unk> 174, and are based on our identification of specific T cell signaling pathways, which once inhibited <unk>.
Expand the body's population of tumor specific memory T cells that recognize and effectively kill.
Tumor cells.
Our adoptive T cell therapies. Unlike currently available car T approaches do not require cellular modification or engineering and are not limited to cell surface expressing antigens. This represents a potentially revolutionary advance.
Beyond currently available adoptive T cell therapies.
Increasing rapidity of treatment preparation decreasing cost and enhancing efficacy by enabling multiply rip.
Repetitive administration.
If our data to date hold in the clinic.
Both our adoptive T cell and car T programs could substantially improve response rates for cancer patients across both liquid and solid tumors.
Our biologic platform is equally or even more broad consisting of classes of novel molecules, combining tumor antigen binders with potent adjuvant <unk>.
Based on our work, we expect some to function in the body as therapeutic vaccines against a wide range of tumors.
Here again, our data suggest that we might well have overcome the challenges limiting the efficacy of currently available tumor vaccine specifically.
That the beneficial effects of the available vaccines are only transient.
Based on the data generated to date the products from our biologic platform have the potential to be long acting and to improve greatly.
The survival rates across solid and hematologic tumors.
I'll now turn the call over to Mike Jacobsen, Our Chief Accounting officer to go through a more detailed discussion of our first quarter financial results, Mike Yeah. Thanks, Greg.
Our net loss for the first quarter was $33 7 million or 54 cents per share.
<unk> to net income of $128 $7 million or $2.05 per share in the fourth quarter of last year.
The fourth quarter of last year includes the $200 million milestone we earned from Rainer in December .
Cash burn for the first quarter.
2023 was $23 $6 million.
That is exclusive of receiving the $200 million milestone payment.
As of March 31.
We had $371 million of cash and investments on hand, and $10 million in receivables, which primarily represent our midway royalties for the February and March.
Which are due 60 days after the respected month then.
Cost and expenses from continuing operations for the first quarter was $35 $7 million.
Which was a decrease of $4 $4 million from the fourth quarter of last year.
The decrease was primarily due to receiving notification in the current quarter of the $2 1 million employee retention tax credit related to the cares Act.
And annual bonuses that were accrued in the fourth quarter.
Interest expense for the first quarter was $7 $9 million, which is consistent with the fourth quarter of last year.
The primary drivers of interest expense are the 2023 and 2026 unsecured senior notes.
And the D IRI amid via royalty obligation.
Now, let's take a quick look at the amid very royalties.
From the divestiture of the imagery business to remain there until the passage of the 2023 omnibus Bill in late December of last year.
We received royalties that 50% of U S net sales.
Upon passage of the omnibus Bill Omidria royalties decreased to 30% of U S net sales.
30% royalty equates to approximately 40% of the U S. Omidria operating profit considering that we don't have any omidria operating expenses.
The 30% royalty rate extends throughout the duration of the railroad relevant patent terms.
Which we expect to be at least through 2033.
For the first quarter of 2023 or 30% royalty on range of net sales was $9 $2 million.
Royalties earned are recorded as a reduction in the omidria contract royalty asset on our balance sheet.
Income from discontinued operations in the first quarter was $6 million and included two primary components.
$3 $9 million of GAAP interest earned on the Omidria contract royalty asset and $1 $7 million of income due to remeasurement adjustments to that same omidria contract royalty asset.
Now, let's look at our expected second quarter results.
We expect overall operating costs from continuing operations in the second quarter to increase slightly due to the timing of clinical development and certain manufacturing activities.
Interest income should be nearly.
$4 million and interest expense for the second quarter should be consistent with the first quarter approximately $8 million.
Income from discontinued operations should be in the $5 million to $6 million range.
With that I'll turn the call back over to Greg Greg.
Okay. Thanks, Mike.
Operator, let's open the call to questions.
Thank you Sir as a reminder to ask a question. Please press star and then one on your telephone and wait for your name to be announced so which are your question. Please press star one again.
Please stand by while we compile the Q&A roster.
And I show. Our first question comes from the line of Steve Brozak from WPB. Please go ahead.
Yes, hi, and thanks for taking the question I've got one from what you said very very early on in the call.
Specifically on the Biomarkers that you've been looking at with the Ta TMA.
Patients how how extensive.
Do you believe these biomarkers can be because obviously in previous calls you had talked about.
Collaborations that you had you had done and shown that the Biomarkers were present with source Kobe too but is this this is now something that trends not just not just with that particular borrower viral indication, but also goes to other indications dealing with the lectern.
Pathway can you tell us.
So as much as possible about this because it's I mean, it's scientifically fascinating, but it's more than that as far as what you are looking at for the use of in our supplement going into the future.
Oh yeah.
Steve.
First the Biomarkers that I was describing.
In the prepared comments.
Were those.
Or really is that used by the group at Weill Cornell and that is cash based shade as a biomarker.
Of microvascular cell injury.
And that's been well documented so really what this does is tie our clinical data our pivotal trial data of <unk> seven to one in our supplemental in Ta TMA.
To specifically.
Caspase shaved levels and the difference between responders and non responders I think what youre referencing with respect to our work in Covid, which is ongoing as well as our work in a R. D S.
Those are biomarkers that in part we have developed and when I say, we that's the collective we of our group in Cambridge as well.
We have an assay.
Or a set of assays that we are looking to effectively put on a on a shingle multiplex.
Which identifies those patients.
Who have <unk>.
Moderately severe to severe COVID-19. We're also doing work in <unk>, but also I think as you mentioned or I think you mentioned.
We're looking at that as well and long COVID-19.
Seeing some interesting data in long COVID-19 as well.
But I'm not at a point, where I think we want to discuss that.
Today.
Yes, thanks for thanks for separating the two between the the Biomarkers at Cornell and the ones that you've got working in collaboration elsewhere, but.
I guess the point I'm getting back to.
Two is that this now shows that there is a quantifiable response, a quantifiable path for showing the effectiveness of our supplemental specifically in dealing with the the upregulation of the lectin pathway.
I would I would think that you would probably also be able to look at it for.
Theoretically a number of other disease indications that you're looking for are looking at and.
What does this mean as far as the ability for you to go into the future and say you know you you have a quote unquote designer diagnostic, but specifically references your your your drug.
Yeah, I, I see where you're going with that I think there's more work that needs to be done I would point you, though to the publications.
That are out.
One by all the it all and frontier is in immunology the other by Lynch at all.
In.
Clinical and translational medicine, I believe and these are both last year publications.
If I'm remembering correctly.
That speak directly to the biomarker, so I well, let me just point you to there I don't think going into detail on all of that now is going to be.
Useful.
The expenditure of time, given the limitations, but.
Happy to to address those offline if that would be helpful. Great.
Great Let me hop back in the queue. Thanks again for taking the questions. Thank you.
Okay.
Thank you.
And I show. Our next question comes from the line of Greg Harrison from Bank of America. Please go ahead.
Hey, good afternoon, thanks for taking the question.
Just wanted to talk about your expectations for overall Iga.
Hi, Jamie nephropathy readout for <unk>.
What would you view that as.
Successful data and then how do you think about.
Potential place in the market now that there are a couple of approved therapy and again.
Yeah with respect to what we would consider a successful read out I guess the answer to that is let's see what the data looked like and we'll tell you at that point I think that certainly we're expecting that nurse thoughtful amount.
We will work and work well in Iga nephropathy as you understand the question is also and what specific subsets are there specific subsets as Ed in high protein is it in and those that are generally across the board.
Iga nephropathy. These are all questions that I think the data will answer and I think that's really premature Greg to to put a stake in any of those the data.
The trial that we're running is.
Placebo controlled double blind. So we don't have any sense of those data now, but if you look at the publications I referenced a few in the prepared comments, but there are others I mean, if you take the time and actually read those.
Think it's clear that the lectin pathway plays a key role not only in the la merial or injury of Iga nephropathy.
But in the.
Two below interstitial.
Disease.
And that's really important because the tubular interstitial <unk> is the is sort of the point of convergence.
All of the end stage renal diseases.
<unk> stage protein, Eric renal diseases, certainly so we're really talking about something that is well beyond.
Just iga nephropathy with respect to how I think.
We will fair or how I think we fit into the market.
With these other drugs I don't see those other drugs.
Creating any impediment to our market entry.
One as you know is is a steroid.
That has.
Reportedly increased activity in the gut.
But that is really only approved for a six month course, just like any other steroids.
And you know the interesting thing is when you look at the at the use of steroids and Iga nephropathy, they can be effective.
But the testing study made it pretty clear that mortality was increased so that study actually needed to be stopped with steroids. So the <unk> recommendations, our six months only for treatment.
And those are those recommendations hold as well.
For this new steroid.
With respect to.
Any of the blood pressure medications.
<unk> blockade is a standard part.
Of all.
Of all Iga treatment, so again I don't see that.
Affecting us in any way.
But let me turn the call over to two commercial and clinical and see if they've got any other any other thoughts that that they'd like to add and whatnot. How do you want to go first.
Thanks, Greg.
The market opportunity is really significant the way we look at this as the Iga nephropathy market has been dormant.
Without anything that has been approved up until now.
The Nephrologist are open to combination use as well, which is something that they're not afraid of in doing with limited options that they have.
And certainly when they look at the profile of a drug that isn't dosed on a daily basis has sort of a shorter course theyre very excited by that so we look at this as a significant opportunity of a market that's yet to wake up.
Steve.
Excuse me I'm sorry.
If you look back at the data from the previous studies and proteinuria as well as Egfr.
The supplement has and this is all data dependent of course tremendous potential to to really help these patients and differentiate itself from from the current competitors on the market as well as others may appear.
Thank you.
Did that did that address the question Greg.
Okay.
Okay.
Okay sorry.
Sure.
And I show. Our next question comes from the line of Elliot Bosco from UBS. Please go ahead.
Hi, Elliot BASCO on for Colin Bristow from UBS. Thank you for taking our question for.
For the P. Eight for the Ta TMA Resubmission.
Last quarter, you noted the threshold for comparison would be based on an independent literature analysis.
Was curious if there are any updates on this and if so could you elaborate on the thresholds and then regarding the meeting assuming positive feedback what are the next steps and potential timeline for a submission. Thank you.
Yes, first with respect to the literature review.
I think we listed that as as.
As one of a number of options all of those being external sources of data for comparison. So I think that as we talked about chart reviews, we talked about registries and we've talked about literature abuse. So I think it's it's.
All part and parcel of the same.
The same effort.
And they're really that that will depend on our discussions with with FDA, what I would see as.
Next steps and again I'll turn this over to regulatory to comment as well, but obviously, we need to reach agreement with FDA on what needs to be included.
And the BLA, what do they want to see what do we need to do and once we know that then we can get that work done we believe pretty quickly.
And then that requires.
Pulling together most of the B L. A is that would be the same but we need to update the safety part of the BLA put the new data in and provide all of this to FDA, which would then have.
A six months timeline.
To review I would hope they wouldn't take that long, but they well could they are busy.
And so that's it.
I think from that you can establish a timeline, but let me say Kathy do you want to comment.
Yes, just to say as Greg mentioned in his prepared remarks, our proposal to FDA is consistent with the decision that we had received in response to our formal dispute resolution request and but we do have to run it by the review division before we can embark on it so.
That's what our next step is and it's.
Consistent with what we hear and so that's where we are right now.
Thank you.
Thank you yeah, yeah could I guess could I get one more in yes, sure if we have ter.
On a 906 you mentioned your intention to pursue.
Pursue geographic atrophy and was curious if you could give a little more color on this and what your thoughts are on the competitive landscape. Thank you.
Yeah, we are looking at geographic atrophy as a possible indication to pursue again, we haven't finalized any of our plans. Our focus is sort of pushing hard on pn H and C. Three G.
But clearly we are thinking about what else to start.
The potential there is obviously the potential for systemic delivery of versus intra vitriol.
<unk> III to our knowledge is not generated.
In the eye or specifically in the retina.
And so clearing masked three from circulation should be sufficient but we're also obviously looking at the commercial aspects.
And systemic versus intra vitriol and how might we want to pursue is it a is it a different molecule that we take forward. These are all.
Questions that the team is currently addressing so again I don't want to get out ahead of ourselves there. How I think we would compare I think we would compare very well when you look at the Pn H data.
You know again I think.
We've we've referenced this but may be not as clearly as we should.
<unk> really represents a pretty high bar.
An alternative pathway inhibition.
It is a life threatening indication and the inhibition of the of the alternative pathway.
<unk> to be effectively complete so you're talking about as close to a ablation as you can get to be effective clearly.
<unk> 906 is showing that capability, even at this low dose and so we're pushing.
We're going to push the dose we have the headroom to do that in terms of our safety studies. So we can push dose and exposure.
So we think we compare very well, but I should after having said all that I want to make sure that our clinical is an agreement. So Steve can you feel free to comment on that.
Thanks, Greg.
Geographic atrophy is.
A really attractive indication for a lot of reasons commercial as well as Greg said the differentiation. If we can provided systemic Lee.
We're obviously focused on P and agency three G and pushing those as hard as we can but.
I can say that we are looking into resource not just strategy, but resourcing to get a G. A program going as quickly as we can because it represents such a substantial opportunity.
Thanks Jay.
Okay.
Thank you.
Thanks Kelly.
Okay.
And I show. Our next question comes from the line of Brandon Folkes from Cantor Fitzgerald. Please go ahead.
Hi, Brian Thanks for taking my questions and congratulations on all the progress and I just want to come back to <unk> 96, so as the data looks really good there so maybe just.
Two questions from my side one anyway.
Any way to speed up development given your strong balance sheet you have now and then secondly.
Given the good data you are seeing at the lower dose.
Do you think you can get quarterly dosing with the smaller dairies, where do we need to go to the high doses to achieve quarterly dosing. Thank you.
Okay, I think I caught most of that you were trailing off at the end, but first with respect to can we accelerate it.
We are absolutely looking at that we.
We want to put the throttle all the way down on this program I mean that the data are clear the end points are objective.
These are not subjective endpoints. These are endpoints that have been used for approval. So we know where FDA stands on those so yes, we are going to push very hard on the 906 program. We think that it has tremendous value.
And I think.
That I think that that's abundantly clear when you look at potential competitors and then you look at the advantages of our potential advantages of 906 over those but are the.
The other question that you had yes, absolutely we believe that we can reach.
We're quite comfortable that we can reach once quarterly dosing, which would be.
A tremendous advantage over.
Either the sub Q or oral age.
<unk> already approved or in development.
Okay.
Great. Thank you very much.
Thank you Brendan.
Yeah.
Thank you.
Showing no further questions in the queue at this time I'd like to turn the conference back to Dr. Demopoulos for closing remarks.
Alright again, everyone. Thank you for joining US today, we hope that the call was helpful and that we've made clear the team's substantial and steady progress. The remainder of 2023 holds a good number of important milestones and we look forward to <unk>.
Erring more information with you over the coming months.
As always we appreciate your continued support and have a good evening.
This concludes today's conference call. Thank you for participating you may now disconnect.
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