Q2 2023 Omeros Corporation Earnings Call
Okay.
Good morning, and welcome to today's earnings call. Our marriage Corporation. At this time all participants are in a listen only mode. After the company's remarks, we'll conduct a question and answer session. Please be advised that today's call is being recorded at the company's request and a replay will be available on the <unk>.
Its website for one week from today.
Turn the call over to Jennifer Williams Investor Relations for Marin.
Good morning, and thank you for joining the call today.
I knew 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 Humira.
Thank you Jennifer and good morning, everyone. Joining me here, Mike Jacobsen, Nordea Doctor and Cathy Melfi, our respective heads of finance commercial and regulatory.
I will start today with a brief overview of our financial results for the second quarter, followed by a corporate update.
Mike will then provide a more detailed financial summary, before we open the call to questions.
Now, let's look at our financial results.
Our GAAP net loss for the second quarter of 2023 was $37.3 million or 59 cents per share compared to a net loss of $33 $7 million or 54 cents per share in the first quarter of this year.
The increase was primarily due to research and development costs.
Cash burn for the second quarter of 2023 was $31 million, which as Mike will explain later includes an artificial accounting driven component of $3 $4 million.
Omidria royalties for the second quarter were $10.7 million or $1 5 million increase over first quarter royalties.
As of June 32023 to support ongoing operations and that service, we had $341.3 million of cash and investments on hand, and an additional $11.2 million in receivables, primarily consisting of Omidria ROI.
<unk>.
Maryland has $95 million of convertible debt maturing in November .
Our available cash and investments enable us to pay off these notes at maturity, while continuing to fund operations and advancing our multiple programs well into 2025.
As mandated by congressional legislation late last year Omidria secured separate payment from CMS in ambulatory surgery centers until at least January 2028.
The legislation further mandates that beginning no later than January 2025, CMS will also pay separately for Omidria when used in a hospital outpatient departments.
Last month CMS issued its proposed 2024 rule for the outpatient prospective payment system and consistent with the legislation called for ongoing separate payment of Omidria.
Let's turn now to our program updates starting first with our family of agents targeting masks to the effector enzyme of the lectin pathway of complement.
In our supplement as our lead antibody against masks to our biologics license application or BLA for in our supplement.
Hematopoietic stem cell transplant associated thrombotic microangiopathy or Ta TMA.
Is pending with FDA.
Following our recent meeting with FDA in which the agency reiterated its commitment to work with her barrels toward a resubmission.
And provided helpful guidance on our proposal to collect and analyze external survival data.
We expect to submit to FDA early next month, a detailed plan of how we intend to analyze those survival data from already identified external sources.
This proposal will be submitted as a type b meeting request with Fda's response expected within 60 days.
After receiving FDA feedback on our detailed plan, we intend to conduct the analysis and together with additional they're supportive data.
Plan to resubmit the BLA.
Assuming the full duration of relevant FDA review periods, we're targeting an approval decision by FDA in mid 2024.
We'll update you when we have resubmitted the BLA.
We continue to make in our supplement available to both pediatric and adult patients worldwide under our expanded access or compassionate use program.
We've treated more than 125 compassionate use patients.
Interestingly a good number of patients have responded to in our sample of map, despite failing treatment with Hercules a mab raviolis amount.
Or <unk>.
Ta TMA patients treated with in our supplement under compassionate use have been the subject of numerous presentations by investigators at international Congresses.
Recently, a group of investigators in Italy submitted an abstract to the annual meeting of the American Society of hematology detailing the clinical and survival benefits are in our supplement and 15 adult and pediatric compassionate use patients with severe ta TMA.
I'll turn now to our phase III clinical program evaluating <unk> in Iga nephropathy.
We remain on track to read out 36 week proteinuria data from our phase III Artemis <unk> trial later this quarter.
Despite the recent market entry of a steroid and a blood pressure medication.
Significant unmet need persists and Iga nephropathy.
The lectin pathway of complement is increasingly recognized as a key immunologic driver of kidney injury in Iga nephropathy.
A review article authored by an international group of experts and published in the most recent issue of kidney International describes kidney biopsies from Iga nephropathy patients.
These biopsies consistently showed preliminary OLED deposition of man and binding lectin or M. B L. Together with Iga, one and up to 50% of patients with Iga nephropathy.
Preliminary alert deposition of lectin pathway pattern recognition molecules like M. B L is known to be associated with more severe galore, merrell or damage as well as more severe proteinuria hematuria all of this meaning more severe.
Iga nephropathy.
Studies also show that the lectin pathway contributes to tubular interstitial fibrosis in Iga nephropathy.
The lectin pathway is the only complement pathway linked to tubular interstitial damage, which is thought to be the common road to a wide range of end stage kidney diseases.
Not just Iga nephropathy.
The primary endpoint in our phase III Artemis <unk> trial is a reduction in proteinuria at 36 weeks in a population of patients at high risk for progressive worsening to end stage renal disease and dialysis.
Meaning those patients with baseline proteinuria greater than two grams per day.
These high proteinuria patients represent about 50% to 60% of all Iga patients globally and to our knowledge <unk> is the only drug.
Being evaluated in this specific population.
Underscoring the importance of proteinuria reduction and our focus on high proteinuria patients a poster presented by international experts at the 60, if Congress of the European Renal Association in June .
<unk> described and analysis of data from <unk> patients along with quality of life measures associated with kidney disease.
<unk> showed that higher urine protein excretion was associated with higher symptom burden.
And worse quality of life overall.
Artemis Hi, again is a double blind placebo controlled trial, assuming positive data.
We will submit both a BLA in the U S and a marketing authorization application or MAA in Europe .
Iga nephropathy is a multibillion dollar market opportunity worldwide.
And there is no approved complement inhibitor for this disease.
Our other <unk> phase III program in atypical hemolytic Uremic syndrome remains a low priority as we have noted previously.
Turning to our thoughtful of Mab in Covid, 19, and acute respiratory distress syndrome or <unk>.
Our work continues at the <unk> labs at the University of Cambridge in patients with acute severe and long COVID-19.
We have been collaborating with multiple U K consortia.
We expect a number of important publications from this work in.
In addition, a manuscript detailing the beneficial effects of mast two inhibition on both symptoms and survival in chemically induced <unk> was published at the end of May and frontier is in immunology.
Another manuscript has been submitted for publication, describing the pulmonary and central nervous system benefits of mast two blockade on symptoms and survival and well established animals of COVID-19, a rds.
Animal studies evaluating a mass two inhibitor an H one N. One driven rds are now underway.
And discussions are ongoing with BARDA, which has declared its interest and helping to develop agents to treat both covet.
Rds.
Following behind <unk> in the clinic is our next generation long acting masked two inhibitor Oh I'm asked 10 29.
Designed to be complementary to and our thoughtful map data from our successfully completed phase one single ascending dose clinical trial support both subcutaneous and intravenous dosing just once quarterly.
So very well suited.
For chronic use.
Dosing in the multiple ascending dose study of <unk> $10 29 in healthy subjects began last month.
In our phase III program is slated to begin next summer.
We've also made good progress in our orally administered <unk> inhibitor program together with intravenous <unk>, our long acting subcutaneous inhibitor, Oh I'm at $10 29.
We expect our oral masked two blocker to complete a franchise of antibody in small molecule <unk> two inhibitors, enabling them arrows to control first line therapy for lectin pathway related diseases.
Okay.
And <unk> complement franchise, just continues to expand and strengthen.
Our lead antibody targeting <unk> three the key activator of the alternative pathway of complement continues to prove its prominence as an alternative pathway inhibitor.
We're currently advancing Oh I'm at 906 across three clinical trials designed to build rapidly on the clinical efficacy data already in hand for Oems 906.
Two of these trials are evaluating <unk> 906 for the treatment of paroxysmal nocturnal hemoglobinuria or <unk> the.
The first is evaluating all of them at 906, <unk> patients who have not previously been treated with a complement inhibitor. The second trial has a switchover design.
Enrolling <unk> patients receiving the C. Five inhibitor Ravi Elisa map, adding RMS 906 to provide combination therapy with Raviolis Mab for 24 weeks.
And then providing Oems 906 monotherapy in patients who demonstrate a hemoglobin response with the combination therapy.
A third <unk> 906 clinical program is underway in patients with complement <unk> Mary Allopathy R. C III G.
A rare kidney disease.
We are amending the dose in this trial due to information already learned in the <unk> program.
In June data from a prespecified interim analysis in our ongoing trial of <unk> 906 in treatment naive <unk> patients were presented at a late breaker session of the 2023 Congress of the European Hematology Association or <unk>.
Sure.
The presentation identified by EHS Scientific program Committee as one of the top five lakey late breaking submissions and delivered in a special oral session is.
It is available on the Investor Relations page of our website.
<unk> 906 is the only drug.
And that has been reported to be able to restore gender normal hemoglobin levels and PSNH patient gender normal meaning.
Hemoglobin levels that based on gender are normal for women and men respectively.
This is important because normal hemoglobin levels are meaningfully higher in men than in women.
Other drug companies in the <unk> space had been using the lower limit of normal.
For women.
Is the threshold for normal hemoglobin for all patients.
At this level is substantially below.
The normal level for Matt.
<unk> 906 has restored not just women.
But also men to truly normal gender specific hemoglobin levels.
The trial evaluating <unk> 906 in treatment naive patients is over enrolled and patient treatment and data collection are ongoing.
Although enrollment has been completed we have been informed by investigators that additional patients are requesting participation.
We are evaluating a protocol amendment that could increase enrollment, but not delay study completion.
At the end of July we performed another analysis of the data in hand.
The results are robust and impressive and we plan to present them at the upcoming Congress of the American Society of Hematology in December .
The second <unk> trial, the switchover trial has already been amended to a phase III study.
And the first low dose cohort has been enrolled at study entry. These patients had all received Ravi Iliza map, but had an inadequate response with hemoglobin levels remaining below 10, five grams per deciliter.
We're targeting 12 patients in this study and already have dosed seven with others and screening.
These patients are receiving combination therapy of <unk> 906, and the first will begin receiving all of them at 906 monotherapy shortly.
We anticipate providing data on this cohort late this year.
Or early next.
It's important to remember that all of them as 906 data already made public result from the lowest dose of <unk> 906 that we plan to evaluate in this program.
And we are now moving to higher doses and exposures to allow for a longer dosing interval.
Yet even at this lowest dose.
Hemoglobin LDH results compare very favorably to the detailed on publicly available data on other alternative pathway inhibitors on the market or in development.
This.
<unk> is even more impressive given that of the 10 Oems 906 treated patients publicly reported in addition to the hemolytic anemia or red blood cell destruction caused by PSNH to patients also have a plastic anemia.
And two others have myelodysplastic syndrome, both of which suppress bone marrow production of.
Of mature red blood cells, despite not having a formal diagnosis.
Other patients in the trial.
And these four also had evidence of bone marrow failure at study entry.
Those other patients similarly have shown a strong response.
<unk> 906 treatment.
The RMS 906 data to date underscore the potential of all of them at 906 as a premier therapy for Pn edge.
But beyond that.
They also demonstrate the expected utility of <unk> 900, <unk> across a broad range of diseases and disorders involving the alternative pathway.
Clinical data have demonstrated that <unk> 900, <unk> not only inhibits mask III, but that masks <unk> inhibition provides a marked level of alternative pathway suppression sufficient to inhibit complement driven hemolysis and <unk> a high bar.
For efficacy.
Given that our competitors in the field have already validated the alternative pathway inhibition is effective.
And multiple other diseases and disorders are data and <unk> provide us with good reason to expect that masked III inhibition with <unk> 906 will also be effective across these other indications.
So how do we expect to differentiate <unk> from its potential competitors.
And both the target masked III in the drug arms 906.
Have multiple expected advantages over other alternative pathway targets and drugs already on the market or in development.
<unk> mask III blockers do not inhibit the infection fighting function of the classical pathway by contrast, both <unk> three and <unk> five inhibitors 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.
This should allow <unk> to maintain inhibition of masks III, allowing more effective blockade of alternative pathway activation.
Importantly, it is expected that these mass III characteristics will translate to a substantially lower risk of breakthrough.
The underlying disease with inhibition of mask III than with C. III C five and factor B.
All of which are acute phase reactants, whose concentrations increase in the setting of inflammation, such as infection or any other inflammatory conditions.
When these increased target concentrations occur they can exceed the inhibitory capability of the respective drugs dosing.
Leaving patients less protected from their life threatening disease.
The third important advantages better patient convenience and compliance we expect that masked three's favorable target characteristics together with the pharmacokinetics and pharmacodynamics of all M. S. 906 will allow once quarterly subcutaneous and intravenous administration of <unk>.
Drug <unk>.
These are expected to enhance patient convenience and compliance compared to other alternative pathway inhibitors on the market or in development.
Based on our substantial data in hand, and the significant expected advantages of our masks III target and drug our objective is to make <unk> 906. The first line standard of care for the treatment of <unk> and a large number of other alternatives.
The way of diseases and disorders.
Okay, let's now move onto all of them as five to seven or <unk> inhibitor program. We have shown <unk> published that PD <unk> inhibition in animal models blocks both craving.
And relapse across multiple substances of abuse, including opioids cocaine nicotine and alcohol.
<unk> seven inhibition has also been shown in animals to be effective in treating compulsive disorders, specifically <unk>.
Current anti addiction agents depress the reward system significantly diminishing enjoyment of other aspects of a patient's life, while having only a limited effect on trading.
In contrast, PD <unk> inhibitors block craving and relapse and importantly, do not appear to depress the reward system. So patients treated with our PD <unk> inhibitors would be expected to avoid the negative effects on life enjoyment seen with other anti.
Addiction drugs.
M S five to seven treated patients would simply lose their craving.
For the substance abuse or for the compulsion.
Now with funding support from the National Institute on drug abuse. We are moving ahead at night as request and in collaboration with them.
To develop our lead orally administered PD seven inhibitor to treat cocaine use disorder. The three year $6 7 million grant from Nida is intended to support both preclinical and clinical work, including a randomized double blind inpatient study comparing the safety and effectiveness of our <unk>.
<unk> seven inhibitor.
To placebo in the treatment of adults with cocaine use disorder, who receive concurrent intravenous cocaine.
<unk> also controls broad patents surrounding <unk>, seven inhibition and movement disorders with our collaborators at Emory University, we are evaluating all of them as five to seven as a potential treatment for levodopa induced dyskinesia or.
<unk> L I D.
Allied manifests as crippling involuntary movements in Parkinson's patients caused.
Primarily by prolonged treatment with levodopa.
Levodopa, though is the most widely prescribed and most effective drug used to treat Parkinson's disease as a result <unk>.
<unk> represents a large unmet patient need and a large market opportunity more than 10 million patients are living with Parkinson's worldwide, and reportedly 50% or more of those with at least five years of L. Dopa treatment suffer from allied.
Okay.
If treated long enough with levodopa, it's thought that effectively all Parkinson's patients will develop I'd only one drug extended release amantadine is approved for the treatment of L. I D.
In addition to its only marginal efficacy amantadine is fraught with multiple significant adverse side effects.
A more effective and safe her treatment is needed.
Our collaborators and Emory have developed a primate model of <unk>, which is highly predictive of clinical efficacy.
Extended release Amantadine has been evaluated in the Emory model.
The MRE and investigators have also use this model to a SaaS <unk> five to seven and allied.
We're evaluating the data and we'll file patent applications as appropriate.
We'll wrap up today's corporate review with our immuno oncology programs.
There are two broad arms.
Of our I O franchise, our cellular platforms and our molecular platforms.
An hour to cellular platforms. We have developed novel approaches to both adoptive T cell therapy in chimeric antigen receptor or car T cell therapy.
Both of these proprietary platforms are based on the novel identification.
Specific T cell signaling pathways, which one's inhibited.
Significantly and preferentially.
Potentiate and enhance the expansion of tumor specific memory T cells.
Distinctively recognized and efficiently kill tumor cells.
Our adoptive T cell platform is designed to target both the cell surface.
And intra cellular cancer antigen significantly broadening its range of indications.
Further unlike existing car T therapies are adoptive T cell technology does not require cellular modification or engineering.
This represents a potentially major advance over currently available adoptive T cell therapies markedly decreasing costs and the time required for treatment preparation, while enhancing efficacy by enabling multiple repetitive administer.
<unk>.
The result, we expect will be a better and sustained anti tumor response.
Also in our modified car T technology, we've incorporated an immuno modulator of T cell signaling, which protects T cells from the immunosuppressive environment promoted by cancer cells, another important benefit over existing car T therapies.
Our car T modifications significantly potentiate, the efficacy and sustained response of <unk> car T therapy.
Our team is validating these novel cellular adoptive T cell and car T platforms and is establishing a broad patent to stay at around them. We believe that these proprietary technologies could meaningfully and substantially improve response rates for cancer patients receiving.
Either engineered or native T cell therapies for not just liquid tumors.
Like existing cellular therapies, but for both liquid and solid tumors.
Our other Io platforms, our molecular therapy platforms, our biologics designed to be injected directly into the patient and include therapeutic cancer vaccines.
Modulators and modified toxins are what we term our oncotype <unk> platform.
Successful development of therapeutic cancer vaccines, though widely pursued remains difficult to achieve.
With the current approaches inducing only transient and ineffective immune responses. We believe that we've discovered a way to overcome this challenge having now generated novel molecules that combined tumor antigens with a potent adjuvant.
These molecules activate antigen presenting cells, which in turn lead to amplification of cancer specific T N b cells.
When injected into the body of these novel Biologics should result in not only elimination of currently present tumor cells, but importantly <unk>.
<unk> memory against future cancer relapse.
Our immuno modulator platform is designed to target and activate immune cells to convert cold tumors into hot.
A cold tumor is one that is refractory to immune therapy, because its microenvironment suppresses the activation and function of therapeutic immune cells.
<unk> modulators are designed to make the immune cells resistant to these suppressive conditions and restore the functionality of the therapeutic immune cells.
As a consequence lymphocytes macrophages antigen presenting and other immune cells can infiltrate the tumor converting it from a cold to a hot tumor.
Allowing the tumor to be destroyed.
Finally, our Oncotype <unk> platform uses engineered toxins to kill only cancer cells that are actively proliferating.
While not affecting oregon's or any healthy cells.
We expect that this novel approach will avoid the deleterious side effects, resulting from currently available toxin carrying therapeutics, especially damage to endothelial cells vascular leak as well as serious complications, including death have severely hindered the use.
Of currently marketed toxins, we expect our Oncotype <unk> platform to have a significantly higher safety margin.
And broader applications than those currently marketed approaches.
Based on the preclinical data generated to date, all three of our molecular platforms, namely cancer vaccines modulators and <unk> have the potential to be long acting and to improve survival rates significantly.
Across both solid and hematologic or liquid tumors.
I'll now turn the call over to Mike Jacobsen, our Chief Accounting Officer.
Go through a more detailed discussion of our second quarter financial results, Mike, Yes. Thanks, Greg.
Our net loss for the second quarter was $37 $3 million or <unk> 59 per share compared to a net loss of $33 $7 million or <unk> 54 per share in the first quarter of this year.
Cash burn for the second quarter of 2023 was $30 $1 million.
Royalties are generally paid 60 days after the month. They are earned although the $3 $4 million payment due on June 30 was received on July 3rd.
This late payment artificially increased our second quarter cash burn.
By $3 4 million.
As of June 32023, we had $341 million of cash and investments on hand, and $11 million in receivables, which primarily consist of the imagery of royalties.
Cost and expenses from continuing operations for the second quarter was $40 $9 million, an increase of $5 $2 million from the first quarter of this year.
The increase was primarily due to additional research and development costs related primarily to drug manufacturing and.
In two clinical costs associated with our phase III clinical trial of <unk> in Iga nephropathy as well as site startup expenses for our 906 studies.
Excuse me.
Interest expense for the second quarter was $7 $9 million, which is consistent with the first quarter of this year.
I'm Mary drivers of interest expense or the 2023 and 2026.
<unk> convertible senior notes and the DRA omidria royalty obligation.
Now, let's look at our midway royalties.
Under our current contract with the trainer immediate royalties decreased from 50% to 30% of U S net sales.
Earning the $200 million milestone payment at the end of last year.
Well I'll separate payment for Omidria is in effect, the 30% royalty rate will apply throughout the duration of the relevant patent terms, which we expect to be at least through 2033.
For the second quarter of 2023, our royalties on Omidria net sales were $10 $7 million.
Royalties earned are recorded as a reduction in the major contact Raleigh royalty asset on the balance sheet.
Income from discontinued operations in the second quarter was $7 million and includes two primary components.
<unk> hundred $28 million of interest earned on the imager contract royalty asset.
And $3 $1 million of income due to re measurement adjustments.
On our Omidria contact royalty asset.
Now let's take.
A minute and talk about expected third quarter results.
We expect overall operating costs from continuing operations in the third quarter to increase by approximately $5 million to $6 million.
The increase is primarily due to our planned payment of a $5 million.
Our milestone obligations tied to advancing clinical development in our <unk> program and the timing of certain manufacturing activities.
Interest income in the third quarter should be nearly $4 million and interest expense for Q3 should be consistent with the second quarter at approximately $8 million.
Income from discontinued operations for the third quarter should be approximately $6 million.
With that I'll turn the call back over to Greg Greg.
Thanks, Mike.
Operator, let's open the call to questions.
Thank you.
As a reminder to ask a question you will need to press star one on your telephone.
Your question. Please press Star one again, please wait for your name to be announced.
Please standby, while we compile the Q&A roster.
One moment for your first question. Please.
Our first question comes from the line of Colin Bristow with UBS. Your line is now open.
Hey, good morning, Thanks for all the helpful color here.
First on the supplement in the Ta TMA can you give a little more color.
The agencies key concerns.
In the May meeting and then any detail on the specifics of the survival assets plant, but also would be helpful. And then just secondly, I guess.
What are you hoping to see in the <unk> data.
The components of this update that you would highlight stage. Thank you.
Sure I think having we're having a little trouble hearing you here.
But I think the first question was are there any concerns or what if any concerns does FDA have.
With respect to our BLA resubmission process and.
I think that.
The answer to that would be I don't think there are specific concerns I think that FDA is wanting to make sure.
That the data and we provide a robust.
That the data we provide.
Demonstrate a survival benefit.
In the <unk> treated group versus.
The external control sources, so I think really.
Onus is.
On us at <unk> to make sure that we satisfy.
All of those.
Requests.
By FDA.
So I'll look to Kathy who is here and our head of regulatory to to add anything to that.
That she'd like.
Yeah, and as Greg mentioned in the call FDA has indicated that they're committed to working with us in terms of the resubmission of the BLA and we believe the data that we have are well will be strong in the approach consistent with the path forward that was presented to us and so again, we're just continuing.
You're going to work with FCA.
Okay, Thanks, Cathie and Colin I am sorry.
Really I think everyone in the room sort of did not quite hear your second question I know it was tied to <unk>.
But it all sort of trailed off and we could not hear it here would you mind just repeating your second question.
Yeah on just on the upcoming items.
I was just wondering what what are you hoping to see in the <unk> data.
Any specific thresholds and any other components.
Any update that you would highlight to us or have a focus on thank you.
Okay no. Thank you for repeating that understood.
Don't have a specific threshold that we're targeting and I guess I mean I.
I think it's important to look at how other agents that have been approved have performed.
And <unk>.
Think those numbers are ranging as a delta over placebo.
At around high twenties.
And I think that clearly.
Indicates.
What is approvable.
As you know there is no approved complement inhibitor. So we really don't see our competitors.
As either a steroid.
Or additional Ras blockade.
It always is.
Iga patients you are going to want to optimize Ras blockade.
And steroids could Diego has.
Has recommended when you can't went went in.
When a clinician cannot get a handle on the Iga that a six month course of steroids is warranted, but thats really only six months.
And the Budesonide that has been approved.
Uh huh.
It has been approved really for only one nine months period.
So.
We don't see either of those.
As as competitors.
What we need to do is is.
See the data.
Understand the data and go forward from there I mean, we expect that data to be positive of course, we can't guarantee that but if we look at all the data we have generated with our supplement as well as the mechanism of action as I said earlier.
Masked two inhibition not only.
Is important in the glomerulus, but also in the tube yellow interstitial, which is why in patients with long standing Iga nephropathy patients, whose disease and Nicola Mariel. This should have a long ago burned out.
We saw a marked reduction in proteinuria and stabilization even improvement in some patients in egfr.
So yes.
As to.
To the best of our knowledge.
Mass <unk> inhibition is.
The only mechanism that works at both those sites. So we're looking forward to the data.
We look forward to sharing the data.
Think with respect to the question is there anything else.
That we want to see.
Look were looking specifically at high protein spillers patients with two or more grams of protein area per day, we're doing that because thats of.
Of high interest to FDA these are patients who rapidly.
And continue to progressively advance to end stage renal disease and dialysis, we've selected that group for a specific reason and as I've just laid out. So I think we're eager to see the data we're eager to share.
The data.
Once we have them in hand, we'll have a lot we'll have a lot more to say.
That's great. Thank you.
Thank you Colin.
Moment for our next question please.
Our next question comes from the line of Steve Brozak with Debbie BBB Securities. Your line is now open.
Yes, Thank you and good morning.
One quick question and one housekeeping question, but let me go to the question on the compassionate use that you mentioned early in the call can you give us any details on what's taking place there and specifically around.
Any changes any increase what the clinicians are telling you anything you can do to help that obviously, specifically in the Ta TMA space. Please.
Yeah sure Steve Thanks.
Uh huh.
With respect to compassionate use and Ta TMA four in our supplement as I said, we have provided drug under compassionate use for over 125 patients. These are both adult and pediatric patients worldwide.
We don't have full case report forms or case report forms really.
To speak of for these patients given that its an extended use our compassionate use program.
But we do get feedback and that feedback is also made public through the multiple presentations at the international Congress by the investigators who request.
Then use.
<unk> to treat <unk>.
Our patients.
It is I think.
Notable that we receive multiple requests from the same institutions.
And this is not a small set of institutions, obviously with $125 plus patients, but we across that 125 plus patient number.
We're receiving.
Repeated requests.
From institutions and obviously.
Our conclusion from that is.
The drug must be doing something good otherwise.
They would not be repeatedly requesting it for their patients both adult and pediatric as I mentioned in the prepared comments Interestingly also we are seeing.
Patients who have failed.
Other therapies <unk> inhibitors, <unk> Mab Raviolis mab.
The February tied as well who.
Are then treated with <unk>, so effectively at that point, we're catching a falling knife. These are patients who are very very sick.
Have gone.
For a while with the Ta TMA progressing moving to end stage.
Organ failure, or having Oregon failure, and yet we we treat them because we do not want to deny these patients we treat them and and they recover if you will.
See that once you see that twice.
You might say well Gee, that's just coincidence or an act of God, you see that enough times.
And I think it becomes pretty clear that the drug.
As effective.
The latter is what we're seeing.
So let.
Let me see if that answers your question.
Thank you for that answer and obviously these patients. Thank you for the answer and what you've done.
So that is that as succinct and explanation as you can ask for.
On the housekeeping side, and then I'll jump back in the queue earlier in the call you reiterated and I looked online and you basically stated that the loss was 59, but I'm seeing some other places reporting a 70% loss.
I believe thats, a typo, but I just wanted to make sure that you're re iterate that number at 59 and there is no other.
No other issues around around.
We're seeing elsewhere.
No there's not.
The <unk> 59 per share losses, the correct GAAP number I think what is happening and we've tried to correct. This.
Multiple times on our earnings calls, but I think what's happening is the <unk> is being lifted from continuing operations.
We are required by accounting rules to.
To account for our royalties in non continuing operations.
So that whole piece is clearly being missed I think when when many report our our EPS. So it's looking at just continuing operations, ignoring non continuing operations and within the non continuing operations are.
All of our royalties that we receive but it's just it's a function of accounting.
And accounting and accounting requirements, let me see if Mike Mike do you have any anything to add to that or make that more clear.
Yes, I think the key to know is our discontinued operations is going to continue as long as we get them imagery of royalties.
A lot of times discontinued operations as a year or two and it's gone but in our case the way the accounting made US do this we will continue to have discontinued operations at <unk>.
<unk> royalties are obviously, a significant positive cash flow for us.
Sure.
Using just continuing operations misses a big part of our findings.
Got it thanks for that explanation.
Thank you.
One moment for our next question please.
And our next question comes from the line of Greg Harrison with Bank of America. Your line is open.
Hey, good morning, Thanks for taking the question.
Is there any color you can provide about your latest interim analysis on <unk> you mentioned in.
In the press release.
And how consistent those results more with what you presented at <unk>.
Sure. Thanks.
Thanks, Greg.
We want to be careful here that we don't preempt.
What we have submitted for presentation.
At at Ash, but I think we can speak to it in general terms.
We are seeing very consistent.
Results.
And as we move forward.
Further out in time, one might expect that we are seeing continued improvement.
So we're very encouraged by what we're seeing.
The latest cut of the data.
Only reinforces I think our confidence.
In the target.
Our confidence in the drug.
But again not just in PSNH, but more broadly.
Across really a very wide range.
Alternative pathway related disease.
Diseases and disorders Pn H is a good litmus test.
Because you really do need to.
Effectively ablate.
Alternative pathway activity and if you don't do that you see it pretty quickly in your LDH levels and and shortly thereafter in your hemoglobin levels.
So it's a very good litmus test on how effective.
Drug is.
And inhibiting.
Alternative pathway activation.
And we're clearly seeing that.
Is that <unk> is highly effective and then that should be translatable to the other alternative pathway indications and really with the other therapeutics in the space, It's pretty easy for us to identify what is an alternative path.
<unk> related disease or disorder, and what is not.
And we can then follow.
Those.
Those other agents.
What will be.
With an agent that we expect.
Has significant advantages as well as the target having significant advantages over other drugs and other targets.
I'll see if not any idea.
Additional comments.
I think one of the things I'll add not only for 906 that our portfolio is also the advantage of lessening treatment burden on these patients and <unk>.
Having having patients taking daily pill, so are very often injections and things like that is another key differentiator and so we're excited across the entire portfolio, including 96 for this additional advantage.
Thank you Nadia so so as you see I mean, we've got.
Relatively short acting IV, we have long acting IV or sub Q and now and mask too.
We're moving pretty quickly on our oral and Thats looking that's looking good as well. So we have the landscape cover and we control the effector enzyme of the lectin pathway.
Which effectively means we control the lectin pathway and we also control the key activator of the alternative pathway.
With all of the advantages that <unk> had just mentioned in that that I went through in the prepared comments. So we really do believe that.
We can make a very strong case that we have the premier complement franchise in the industry full stop.
That's great to hear thanks for taking the question.
One moment for our next question please.
Our next question comes from Sergey <unk> Mellinger with Needham Your line is now open.
Hi, Serge.
Hey, good morning, Greg Thanks for taking our questions.
A couple on the <unk>.
<unk>.
Program, maybe can you talk about the statistical powering of the trial, that's going to readout later this quarter.
And secondly.
Do you believe the arguments trial is the only phase III trial required to support a BLA and MAA filing in Europe .
And then my second question I guess third question.
Regarding the no payment coverage.
In Omidria.
Omidria had they've had some good traction in the ASC setting just curious how the no pay and coverage that takes effect in 2025.
We will change the overall coverage of the product and whether.
It will see increased usage beyond the ASC setting thanks.
Sure Thanks, Serge and answer to your first question.
Again powering.
Our statisticians believe that and.
We are quite confident that we are overpowered.
So I think we're in good shape there.
Yes.
We expect that the one phase III clinical trial will be sufficient.
To support our BLA and MAA and Thats consistent with other therapeutics that have received approval recently, so we see no difference there.
With respect to <unk>.
Omidria.
And <unk> look we we know what happens in the <unk> represent about <unk>.
20%.
Of the total procedures.
Once we again receive our Omidria again received separate payment and the H O P DS.
We expect to see.
About a 20% boost.
In an hour.
Total revenues.
Net sales from Omidria at least.
And again I think the key here and when I say that.
20.
Percent boost.
Yes that would really be about a 25% increase over where we were we are given that it's 20% of total net sales.
I think that the key here is also.
In the amount of time.
That we now have secured.
So long term separate payment how quickly Ken Rainer <unk>.
<unk> down med advantage or med part C separate payment reimbursement that's the key to this because right now I think still.
Physicians and centers.
Mostly call their patients they will look for patients who are med part b.
And the reason they do that is the concern that if they use.
Use omidria on a med part C patient of med advantage patient and are not paid.
That is a meaningful cost to that.
But there becomes a threshold.
Beyond which if you can access med advantage for doesn't need to be a 100% of the patients it just needs to be.
<unk> of the patients where physicians and centers then are willing to use it broadly.
And once it it has that kind of med advantage coverage. Then you really you don't even need to increase the number of sites or number of customers. You simply are our drilling down on those customers and on those sites and moving from <unk>.
Part B to med advantage, which by definition then effectively includes commercial.
The payers are the same for med advantage and commercial and all of a sudden.
You've opened this up and you've made the drug available to not only med part b, but med advantage and commercial patients as well, which is really what should happen right.
The drug clearly works it works well the benefit risk ratio is so heavily weighted to the benefit I know that when we had omidria. We did not have a single safety signal of concern zero.
Associated with the drug.
Nothing came to us there.
So yes. This is pretty clear, but it is really a it is really a reimbursement issue.
And I think I know that Rainer is working on that.
And we look forward to their success, which I think will be.
Not only good for Rainer and by extension <unk>, but I think most importantly, it's going to be good for patients and we need to get that so let me see not again anything you want to add to that.
And the only thing I will add I agree with everything you just said is even.
Going back to the hospital outpatient Department question. They request for imagery of continue even without no.
<unk> previously so we know there is demand and there is significant opportunity that we're excited about.
Thank you you are not here, yet I would underscore what <unk> just said I mean, there are a number of H O P DS.
That are using the product really at their cost.
Because they believe that strongly in it and they believe that patients should have it so.
So I think once that H O PD separate payment begins as well I.
I think it has an amplifying effect.
Across <unk> and <unk>.
Thank you.
One moment for our next question please.
Our next question comes from the line of Olivia Brayer with Cantor Fitzgerald. Your line is now open.
Hi, Good morning, this is Google and Roger Almond.
Yes, thanks for taking my question.
Could you give us any more color on what type of survival analysis is included in the proposal you're submitting next months, our Ta TMA and once you're in agreement with the FDA on those how quickly do you think you can turnaround the survival data for the submission package.
Sure Hi, Thank you.
Sure.
First with respect to what we are proposing these are just specific analysis.
That we.
And.
To run.
That will allow a robust and a meaningful comparison.
The survival data from our.
Clinical trial.
The Ta TMA trial.
To survival in the external sources, so there's not really anything magical about this.
We just want to submit a detailed proposal so that we have.
Hopefully have alignment with FDA.
After having done that submitted that proposal as I said this will be part of a type B meeting we would expect a response from FDA within 60 days.
Then it is largely for us.
Pushing a button.
And I don't want to minimize this I'm sure that our statisticians and data management group might not want me.
Representing it that way, but again. These these analyses will already have been set and it is pushing the button getting the readout.
What it will take for us to submit.
It's going to be work, but we are resubmitting a BLA. So it's updating safety, it's adding the new information into the BLA and then resubmitting and I think as we said our objective here is even with the full.
<unk> another six months.
Review cycle by FDA.
Our objective or our target is to have a decision from FDA.
In mid 2024, but let me turn to Kathy and see Kathy what would you like to add to that yes.
Yes.
Say that.
For for part of the Resubmission that don't count on the analysis that we still have to do we are already doing the work and preparing the documents that we need for it. So we're trying to do everything we can sort of in advance and in parallel so that as Greg said, we minimize the amount that we.
We need to do once we get the feedback from FDA that we're working very hard on it right now.
The objective of course on our side and thanks Cassie the objective of course is to get the.
La resubmitted as quickly as possible.
I mean this has been an.
And our Jewish task.
And we want to and expect to get in our supplement over the finish line.
In the relatively near term.
So we've laid out the timelines we think that those are are reasonable.
And I can tell you internally, it's really all hands on deck pushing.
To get this to get this completed.
We had an earlier question about compassionate use.
Clearly a need and a desire for the product and we get these compassionate use requests.
I would say weekly I mean, we just had I think Cathy we just had.
101 or more this week already.
And when we get those.
We don't ask 10 day pay.
We ask particularly if these are children.
A question. We ask is how quickly can we get it there how quickly can we get the drug there.
So.
We're trying we're making this available and.
And we do make it available because we are very confident it works and obviously the requests that we're getting I think indicate our confidence within the transplant community that it works. So our objective as fast as possible get it over.
Finish line.
And make this make this available broadly to patients hopefully adult and pediatric patients make this available.
So that we can continue.
To save lives, which I can tell you with complete certainty we're doing right now.
Yeah, I'll add something as well in terms of the needs out there and the demand. So we've recently attended a couple of payer conferences and continued interactions with the transplant centers and the number. One question is when are we going to have this because they are eager to.
Have a product that is specifically indicated for Ta TMA.
So that they can add it to their formularies they can get it to patients and not have to deal with off label use that unfortunately is.
What is the standard of care and all that's available now so that is the top question that we have to answer repeatedly.
Alright.
Hope that.
Hope that answered your question, if not let us know.
Thank you and at this time I'd like to hand, the conference back over to Dr.
Gregory Demopoulos for closing remarks.
Alright, Thank you operator, and thanks to all of you for joining this morning as you can see.
Across our programs, we have a handful of value driving milestones this quarter and over the next six months.
All of us at <unk> are looking forward.
Seeing how they play out.
So with that enjoy the rest of your summer and as always we appreciate your continued support have a good day.
This concludes today's conference call. Thank you for your participation you may now disconnect everyone have a wonderful day.
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