Q3 2024 Omeros Corp Earnings Call
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Speaker Change: Good afternoon, and welcome to today's conference call from Harris 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 to ask a question. During the session you will need to press star one on your telephone.
Didn't hear an automated message advising you. Your hand is raised to withdraw your question. Please press star. One again, 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 will now turn the call to Jennifer Williams Investor Relations for minerals.
Jennifer Williams: 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.
Jennifer Williams: Statements are based on management's beliefs and expectations as of today, only and are subject to change.
Jennifer Williams: 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, and subsequently filed.
Jennifer Williams: Early reports for a discussion of these risks and uncertainties.
Speaker Change: Now I would like to turn the call over to Dr. Greg Demopoulos, Chairman and CEO was on there.
Speaker Change: Thank you Jennifer and good afternoon, everyone I'm joined on today's call by our Chief Accounting Officer, David barges.
Speaker Change: Nadia Dock, our Chief commercial officer, Andreas Grauer, our Chief Medical Officer, our Chief Regulatory Officer, Cathy Melfi, and our vice President of clinical Steve would occur.
Speaker Change: We'll start today with an overview and discussion of our third quarter 2024 financial results.
Speaker Change: Followed by an update on our ongoing development programs.
Speaker Change: David will then provide a more detailed financial summary, before we open the call to questions.
Speaker Change: Now, let's look at our financial results for the third quarter, our net loss for the third quarter of 2024.
Speaker Change: Well were $32 $2 million or 56 cents per share compared to a net loss of $56 million or 97 cents per share in the second quarter of this year.
Speaker Change: The $23 $8 million quarter over quarter decrease in the net loss was primarily driven by $17 $6 million of nurse thoughtful of Mab drug substance recorded as an expense in the second quarter.
Speaker Change: As of September 32024, we had $123 $2 million of cash and investments on hand, a decrease of $35.8 million from June 32024.
Speaker Change: In addition, as relayed in earlier calls our recent sale of Omidria royalties to D. R. I healthcare carries with it to sales contingent milestones payable by D. R. I too am arrows, each up to $27 $5 million with payment dates in January of 'twenty.
Speaker Change: 26 in January 2028.
Speaker Change: Earlier this year the centers for Medicare and Medicaid services issued their outpatient prospective payment system final rule confirming ongoing separate payment for Omidria and ambulatory surgery centers and for the first time beginning on January one.
2025, expanding.
Speaker Change: Expanding separate payment to hospital outpatient departments or <unk>.
Speaker Change: The initiation of <unk> sales in January is expected to grow omidria sales meaningfully in the U S.
Speaker Change: Ex U S sales should also begin next year with home arrows, receiving 15% of those net revenues.
Additionally, our term loan agreement from June 2024 includes a commitment by the lenders to fund a $25 million delayed draw facility. Upon our request prior to June three 2025 contingent on receipt of FDA approval of <unk>.
Speaker Change: And Ta TMA within 30 days of the funding request.
Speaker Change: The additional term loan commitment provides <unk> with a ready source of capital to fuel the early commercialization of <unk>.
Speaker Change: Let's move on to an update on our development programs, starting with our complement franchise and <unk>. Our first in class masked two inhibitor targeting the effector enzyme of the lectin pathway of complement.
As described in previous updates we have been working closely with FDA regarding the anticipated resubmission of our biologics license application or BLA for <unk> in hematopoietic stem cell transplant associated thrombotic microangiopathy.
Speaker Change: Or Ta TMA.
Speaker Change: Over the last several quarters, we have had a series of interactions with the agency focused on our proposed plan for Resubmission and.
Speaker Change: In September we held a pre submission meeting with FDA.
Speaker Change: The meeting was collaborative and as part of the meeting we received additional minor feedback on our proposed statistical analysis plan for the primary endpoint.
Speaker Change: That being patient survival in our pivotal <unk> trial compared to that in an external registry of patients with Ta TMA.
Speaker Change: FDA had previously reviewed the plan and all FDA comments had been incorporated the additional feedback was limited to requesting a few more sensitivity analyses we quickly incorporated additional sensitivity analysis into the plan.
Speaker Change: And incentives back to the agency.
Speaker Change: We are awaiting fda's reply and expected to arrive imminently.
Speaker Change: No other pending information requests and are not aware of any other impediments to resubmitting, our in our supplemental BLA.
Speaker Change: Once Fda's response is in hand, and assuming general alignment on the revised analysis plan, our independent external statistics group will conduct the pre specified analyses directed to the primary endpoint.
Speaker Change: In addition to the primary analysis, the external statistics group will perform supplementary analysis.
Speaker Change: The <unk> expanded access program or EAP together with sensitivity analyses and descriptive statistics on both the <unk> pivotal trial results.
Speaker Change: And then our <unk> EAP data.
Speaker Change: The key statistical programs for these analyses are already written so results from the analysis should be available in a matter of days after their initiation.
Speaker Change: Following completion of the analyses, we look forward to sharing the results publicly.
Speaker Change: To accelerate the timeline to resubmission preparations, including drafting and revising sections of the Resubmission began months ago.
Speaker Change: Overall favorable analysis results, we will dedicate the necessary internal and external resources to resubmit, the BLA as quickly as possible.
Our recent interactions with FDA.
Speaker Change: Our occurring against a backdrop of heightened awareness and focus within both the agency and Congress on the need for expanding treatments for rare diseases, including increasing regulatory flexibility in Fda's review and approval.
Speaker Change: Process for these treatments.
Speaker Change: This is evidenced by public statements and congressional testimony by FDA leadership.
Speaker Change: The congressional rare disease, Kaka says communications with FDA.
Speaker Change: As well as published editorials authored by the caucus is bipartisan chairs and significant stakeholder engagement among highly credible rare disease community organizations.
Speaker Change: Perhaps most impactful and widely noted.
Speaker Change: Fda's recent and well received establishment of its rare disease innovation hub.
Speaker Change: The hub co led by the directors of FDA centers for drug and for Biologics evaluation and research is designed to provide drug sponsors and other stakeholders in the rare disease community with a single point of connection and engagement.
Speaker Change: Within the FDA.
The objective is to ensure greater consistency in the review process and to overcome and mitigate the unique challenges associated with developing therapeutics for rare diseases.
Speaker Change: Separate from our interactions with FDA, we are preparing a European marketing authorization application or MAA for <unk> in Ta TMA, which we expect to submit in the first half of 2025.
Speaker Change: In parallel with our efforts to submit both in our supplemental BLA and MAA panels of international experts are preparing two manuscripts for publication in top tier peer reviewed journals.
Speaker Change: <unk>.
Speaker Change: In keeping with our primary endpoint for regulatory approval will compare survival of patients in our pivotal Ta TMA trial with.
With the same rigorous external control used for our BLA resubmission.
The second details the survival data.
Speaker Change: 140, <unk> treated adult and pediatric patients.
Speaker Change: From our global expanded access program.
Speaker Change: As we have moved closer to resolving the status and direction of R&R supplemental program and Ta TMA, we have in parallel.
Speaker Change: <unk> to advance rapidly towards the initiation of phase III clinical programs <unk> <unk> also known as <unk> 906.
Speaker Change: Our first in class masks three inhibitor targeting the key activator of the alternative pathway of complement.
Speaker Change: Let's turn now to an update on our progress across our south kind of our development programs.
Speaker Change: <unk> focus for us all tenant Bard has been on multiple phase II studies assessing the drug as a potential treatment for two chronic rare disease indications paroxysmal nocturnal hemoglobinuria or <unk>, a life threatening hematologic disorder.
Speaker Change: C III Calamary Allopathy RC three G, a debilitating and potentially life threatening kidney disease.
Speaker Change: Let's first discuss our PSNH program, which continues advancing rapidly and generating compelling data.
Speaker Change: Our phase III switchover study evaluated <unk> in <unk> patients with suboptimal response to Ravi Elisa map.
Speaker Change: As planned and on schedule all patients have now completed the trial with the last patient visit having occurred in October.
Speaker Change: These completed study patients have rolled into the long term open label extension study further building our safety database to be included in the planned BLA for resolve kind of art and Pn H.
Speaker Change: Patients in the switchover study began by receiving both <unk> and Raviolis Mab a C. Five inhibitor with those patients demonstrating a response to the combination therapy, then switching to <unk> monotherapy the tree.
Speaker Change: While evaluated to sell 10 of our dose levels results from the adjunctive therapy portion of the trial were presented in June at the annual Congress of the European Hematology Association.
Speaker Change: Patients, receiving <unk> and <unk> combination therapy demonstrated a statistically significant improvement both in mean hemoglobin compared to baseline and then absolute reticulocyte count.
Speaker Change: Now data from those Altana Bart monotherapy stage of the trial will be presented next month in San Diego at the annual Congress of the American Society of Hematology.
Speaker Change: We are pleased with our results.
Speaker Change: So now I'll turn about monotherapy prevented intra vascular as well as extra vascular hemolysis and showed sustained and clinically meaningful improvements in both hemoglobin levels and absolute ridiculous side counts.
Speaker Change: So Panama also continues to demonstrate a favorable safety profile with no observed safety signal of concern.
Speaker Change: The second <unk> study is ongoing this one evaluating <unk> in <unk> patients who have not been previously treated with a complement inhibitor.
Speaker Change: As discussed during last quarter's earnings call. We amended this study's protocol to allow <unk> concentrations and patience to decrease until breakthrough hemolysis occurred.
Speaker Change: <unk> generated additional data on the level of mastery inhibition and the correlating plasma concentrations of Saltano Bart required to inhibit that breakthrough hemolysis.
Speaker Change: Based on our analysis of these and other data generated from ours, all 10 of our clinical program and having agreed with both FDA and European regulators on our dose finding methodology. We have now selected those all 10 of our dose for our phase III clinical trials.
Speaker Change: Both <unk> studies in Pn H, the switchover trial and the trial in treatment nave patients have resulted in impressive efficacy and safety data.
Speaker Change: These data support our expectation that's all tenant Bart will be a successful competitor in the market of alternative pathway of therapeutics and our phase III program and design is well aligned with that of our our already successful phase III program.
Speaker Change: Specifically, our phase III program consists of two trials one switchover trial in patients with an inadequate response to <unk> inhibitors and the other trial on patients who are not receiving complement inhibitor treatment.
Speaker Change: The endpoint measures are the same as those used by novartis in its pivotal trials part of hub halter.
Speaker Change: And the results of our already conducted all 10 of our <unk> edge trials show robust efficacy against those measures.
Speaker Change: So we're pleased to say that's all 10 of our phase III program in <unk> is underway.
Speaker Change: Earlier this quarter, we completed successful end of phase II meetings, with both FDA and European regulators.
Speaker Change: Both agencies agreed with the designs of our studies.
Speaker Change: <unk> had a few comments, which we incorporated in the revised protocols will be returned to FDA within the next few weeks.
Based on our discussions with both FDA and European regulators, we now have a clear path to opening enrollment, which given the upcoming December holidays, we expect in early 2025.
Speaker Change: Okay.
Speaker Change: In preparation for commercialization. We also held a successful meeting with the German Federal Joint Committee or GBA.
Speaker Change: The decision, making body in the German healthcare system that specifies which medical treatments are reimbursed by the statutory health insurance funds.
Speaker Change: The GBA provided us with productive feedback on the patient reported outcome member measures that we plan to incorporate in our trials.
We also continue to solicit input from physicians and patients through market research and advisory boards to define the data that will meaningfully differentiate <unk> from other complement inhibitors.
Speaker Change: We are collecting these data in our phase III trials and other non trial activities.
Speaker Change: Based on the insights collected to date <unk> clinical profile.
Speaker Change: Together with its low treatment burden of only four to six times per year is clearly differentiated from treatment options on the market or in development.
Speaker Change: The market size for <unk> is large and growing.
Speaker Change: Part of it to be $3 $9 billion in 2023 and projected at over $10 billion in 2032.
Speaker Change: We expect that after approval.
Speaker Change: All tenant Bart will command a substantial portion of that market given its differentiated profile that resonates well with prescribers and patients.
Speaker Change: Let's turn now to sell 10 of BARDA, and <unk> and ultra rare kidney disease, commonly affecting children.
Speaker Change: Late last month, we announced that FDA had awarded <unk> with a rare pediatric disease designation for the treatment of <unk>.
Speaker Change: Companies holding a drug with this designation receive a rare pediatric disease priority review voucher from FDA.
Speaker Change: When the designated drugs first approval is for the associated indication in the pediatric population.
Speaker Change: The voucher allows the recipient company to obtain FDA priority review of either a BLA or a new drug application.
Speaker Change: For a different product or indication.
Speaker Change: This priority review is commercially valuable in that it reduces the review time and can accelerate any subsequent market entry by at least four months.
The voucher may be used by the original recipient or can be sold to another company for us.
Speaker Change: Our so all 10 of our phase III <unk> study is enrolling and assuming positive data phase III program in <unk> is planned for initiation in the first half of 2025.
Speaker Change: As discussed at our last quarterly call. All is all 10 of our drug product needed for our phase III programs.
Speaker Change: Has been manufactured and is ready and waiting for the start of enrollment in the phase III PSNH and <unk> trials.
Speaker Change: As noted earlier <unk> will have a sizable presence at the annual meeting of the American Society of Hematology next month.
Speaker Change: <unk> Zhao <unk> abstract presentations advisory Board and medical affairs activities and meetings with experts and corporate groups across the relevant therapeutic areas for alternative pathway inhibition.
Speaker Change: So where do we go next with Zao <unk> well, we continue to follow closely the clinical trial results of other alternative pathway inhibitors to identify additional indications, where we believe <unk> would have substantial.
Speaker Change: Vantages over other agents.
Speaker Change: We also are exploring diseases for which the pathology might well be primarily masked 30, driven all of this is to say that we remain keenly focused on the wide applicability of mass very inhibition and the significant advantages that <unk> could hold.
Speaker Change: Over other alternative pathway targeting therapeutics on the market or in development.
Speaker Change: Specifically more consistent efficacy.
Speaker Change: Better safety and superior dosing compliance and convenience with significantly lower treatment burden.
Speaker Change: <unk> when compared to twice or thrice daily oral medications that are often difficult for patients to remember to take.
Closing out our discussion on our <unk> franchise of complement therapeutics, let's look at <unk> $10 29, our next generation long acting <unk> two inhibitor.
Speaker Change: Having successfully completed both the single and multiple ascending dose phase one studies of <unk> $10 29, and we are confident that the drug can effectively ablate mast two concentrations in humans with once quarterly low volume dose.
Speaker Change: Whether administered subcutaneously or intravenously.
Speaker Change: We also believe that the safety profile of RMS $10 29 is compelling.
Speaker Change: <unk> seen no safety concerns with Oems $10 29.
Speaker Change: Nor across any of the clinical trials with its shorter acting relative the mast two inhibitor and our supplement.
Speaker Change: We continue our assessment of attractive indications for LMS 10, 29, as well as of additional indications for in our supplement dependent of course.
Speaker Change: On resource availability.
Speaker Change: We also are continuing our work with <unk> two inhibition in both severe acute and long COVID-19 as well as in acute respiratory distress syndrome, or rds, including H, one N one and H five N. One.
Speaker Change: Here again manuscripts detailing the study results are being prepared for submission to top tier peer reviewed journals.
In addition to our complement antibodies in our <unk> and LMS $10 29.
Speaker Change: We continue making headway on our small molecule orally available masked two in mass <unk> inhibitor programs for use in large market chronic indications.
Speaker Change: Let's turn briefly now to almost $5 seven or <unk> inhibitor program aimed at treating addictions and compulsions.
Speaker Change: As well as movement disorders.
Speaker Change: Our ongoing work to develop all of them as five to seven as the first treatment for cocaine use disorder remains fully funded by the National Institute on drug abuse or NEDA.
We continue to expect results later this year from our toxicology study in primates in rodents exposed to both cocaine and all of them as five to seven to assess potential drug drug interactions assuming satisfactory results for next step is to begin in 2025 and.
Speaker Change: <unk>, a randomized double blind inpatient clinical trial evaluating <unk> $5 7 million individuals with cocaine use disorder.
Speaker Change: This clinical trial will also be fully funded by Nader.
Speaker Change: Also as previously discussed we are exploring the potential use of all of them as five to seven in movement disorders, specifically levodopa induced dyskinesia or allied.
Speaker Change: This is a profound and highly prevalent problem in patients with Parkinson's disease caused not by the disease.
Speaker Change: Rather by its treatment.
Speaker Change: We are pursuing substantial external funding for the further development of <unk>, 5% to 7% idea of large and essentially under unaddressed market.
Speaker Change: Finally, we will conclude today's program review with our first in class therapeutic platform of molecular and cellular programs targeting a wide range of therapeutic areas, including cancer.
Speaker Change: Building on our understanding of immunity, both innate or complement mediated and adaptive.
Speaker Change: Meaning b cells as well as CD, four and CDA T cells. Our objective is to move beyond existing targeted biologics such as antibody drug conjugate Center radio ligands.
Speaker Change: And also beyond Immunotherapies like checkpoint inhibitors and car T.
Speaker Change: To achieve this we are developing a portfolio of signaling driven immuno modulators <unk>.
And in adoptive T cell technology combined with an immuno stimulator.
Speaker Change: The T cell therapy, and immuno stimulator combination.
Speaker Change: Unlike other cellular therapy approaches requires.
Speaker Change: No cellular engineering.
Speaker Change: It reduces manufacturing costs and timelines and maintains an enhanced anti cancer immune response by means of simple and repetitive therapeutic administrations.
Speaker Change: Our growing volume of in vitro and in vivo data support the potential to deliver broadly effective and safe cancer therapies that can overcome the shortcomings of currently marketed therapeutics by treating both.
Both hematological and solid tumors.
Speaker Change: Targeting both the cell surface and intra cellular cancer antigens.
Speaker Change: And increasing levels of CD, four and CDA cancer antigen specific factor and memory cells.
Speaker Change: We are rapidly advancing our oncology programs in stealth development, while we continue to expand our intellectual property position.
Speaker Change: Over the past several months, we have sought the input and guidance of therapeutic area experts and advisers under confidentiality with uniformly positive response.
Speaker Change: Over the past few days, we began raising the visibility of these programs that international Congresses, holding non confidential discussions with academic experts in corporate groups in Houston at the annual meeting of the society of immunotherapy of cancer.
Speaker Change: As previously mentioned our team will also be attending the upcoming meeting of the American Society of Hematology.
Speaker Change: We plan to share further information on these programs publicly in the coming months.
I'll now turn the call over to David barges, our Chief Accounting officer to go through a more detailed discussion of our financial results David.
David barges: Thanks, Greg.
David barges: Our net loss for the third quarter of 2024 was $32 $2 million.
David barges: Or <unk> 56 per share compared to a net loss of $56 million or <unk> 97 per share in the second quarter of 2024.
David barges: As of September 32024, we had $123 $2 million of cash and investments on hand.
David barges: Decrease of $35 8 million from June 32024.
David barges: Costs and expenses from continuing operations for the third quarter were $35 $4 million, which was a decrease of $23 $8 million from the second quarter of this year.
David barges: The decrease was primarily driven by $17 $6 million of R&D expense related to the manufacture of <unk> drug substance lots that commenced in October 2023, and which were delivered in expense in the second quarter of this year.
David barges: Recall that our accounting policy is to expense all manufacturing cost related to drug candidates until regulatory approval is reasonably assured in either the U S or the European Union.
David barges: Manufacturing costs for <unk> were lower by $3 $7 million in the third quarter as a result of drug substance produced an expense in the second quarter.
In addition, there were $1 $9 million for term loan related transaction cost that were expensed in the second quarter of 2024.
David barges: Interest expense for the third quarter was $4 $1 million, which was $5 $2 million lower than the second quarter of this year.
David barges: The primary drivers of interest expense are the 2026 notes.
David barges: The tiara in Madrid, and a royalty obligation and the newly issued secured term loan entered into in June 2024.
David barges: In the third quarter, we recorded a $3 4 million noncash credit lowering interest expense, reflecting changes made to the omidria royalty obligation.
David barges: The remaining third quarter decrease was due to newly issued secured term loan agreement, which replaced a portion of our 'twenty six notes.
David barges: Interest and other income for the third quarter was $2 $3 million. This is lower than the second quarter of this year due to lower cash balances to invest.
David barges: And income from discontinued operations in the third quarter of this year was $4 $9 million and includes two primary components.
David barges: First it's $4 2 million of interest earned on our Mydriatic contract royalty assets and second $700000 of Remeasurement adjustments to the imagery at contract royalty asset.
As we have previously discussed royalties earned are recorded as a reduction in the omidria contact growth asset on our balance sheet and not in our income statement.
David barges: <unk> royalties for the third quarter were $9 $3 million on Omidria net sales of $31 million.
David barges: This is compared to royalties of $10 $9 million on second quarter net sales of $36 4 million a decrease of $1 6 million in omidria royalties or $5 4 million in net sales from the second quarter of 2024.
David barges: <unk> royalties decreased $700000 or $2 3 million in net sales from the third quarter of 2023.
David barges: As we discussed in prior earnings calls in February 2024, we entered into an amended agreement with Eri by which they acquired the right to receive all U S. Mydriatic royalties payable by Rainer through December 31, 2031.
David barges: We continue to hold all royalty rights to <unk> ex U S sales of imagery and.
David barges: And after December 31, 2031, all U S royalty rights returned tomorrows, resulting in all global royalty payments subsequently occurring to Americas.
David barges: Now, let's look at our fourth quarter expected results, we expect overall operating costs from continuing operations in the fourth quarter to be similar to the third quarter.
David barges: Interest income for the fourth quarter should be nearly $1 2 million in interest.
David barges: <unk> expense.
David barges: Any adjustments related to the omidria royalty obligation to be approximately $7 $2 million, which is a noncash increase of $3 1 million from the third quarter, reflecting the absence of any large noncash adjustment related to the <unk> royalty obligation.
David barges: And as you may recall that term loan transaction. We completed in June of this year had an embedded gain of $29 $8 million, which was deferred and is being amortized as a reduction to interest expense.
David barges: The amortization results in an effective interest rate of.
David barges: One, 5% or $400000 on the term debt for financial statement purposes, and finally income front.
David barges: From discontinued operations should be in the $7 million to $8 million range.
Speaker Change: With that I'll turn the call back over to Greg.
Greg Demopoulos: Thank you.
Greg Demopoulos: Thank you David.
Greg Demopoulos: Greater now, let's please open the call to questions.
Greg Demopoulos: Thank you as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
Greg Demopoulos: Your question. Please press Star one again, one moment for our first question.
Speaker Change: Our first question is going to come from the line of Steve Brozak with <unk>. Your line is open. Please go ahead.
Steve Brozak: Yes, hi, and thank you for taking the questions I only have one.
Steve Brozak: From what I've read and from what you've just stated.
Steve Brozak: Given the brevity of the requests from FDA of <unk> for nurse supplement and given the fact that.
Steve Brozak: Im sure Youre going to turn to very quickly on the resubmission of the BLA.
Steve Brozak: I would assume it's reasonable to say that youre going to be.
Steve Brozak: Selling commercially.
<unk> in 2025 can you.
Steve Brozak: Comment on that please.
Steve Brozak: Well I think that certainly.
Steve Brozak: The hope and expectation, Steve I think.
Steve Brozak: 2025, we'd actually be not.
Steve Brozak: Not looking into the latter part of it.
Steve Brozak: We'd be looking for turning the BLA around quickly again, assuming alignment on really what's left to discuss on the SAP.
Speaker Change: As I think we.
Speaker Change: Noted in the initial comments and as you just referenced.
Speaker Change: The request was really for additional sensitivity analysis.
Speaker Change: So we would expect the alignment but were waiting to receive.
Speaker Change: Comments on the SAP <unk> once we have those.
Speaker Change: We will quickly.
Speaker Change: Run the analysis or I should say our external consultants.
Speaker Change: Statistics group will run those share those results with us assuming those look good and we would certainly expect that looking at.
Speaker Change: The data that are available to us.
Speaker Change: We would then move quickly to resubmit.
Speaker Change: Alright.
Speaker Change: So yes.
Speaker Change: Yes, I think 2025.
Speaker Change: Certainly where we are targeting.
Speaker Change: Got it okay. Thank you, let me hop back in the queue.
Speaker Change: Thank you one moment sorry next question.
Speaker Change: Our next question comes from the line of.
Greater with Cantor. Your line is open. Please go ahead.
Speaker Change: Hi, good afternoon, and thank you for the question for your mast three inhibitor, Greg what more can you tell us at this point about the phase III trial designs for both indications.
Speaker Change: I know you've made a few comments on pn H, but any additional color would be helpful or anything that you can tell us around FDA or EMA feedback on those on that trial design, specifically I guess for Pn H.
Speaker Change: I also wanted to ask about specific patient segments that you are planning to enroll or if theres anything that you can do to enrich the populations in those trials.
Speaker Change: Yes, let me answer generally and then I'll turn that over to through our clinical team that's here as well.
Speaker Change: Through our representatives, but.
With respect to the phase III trial design.
Speaker Change: They will be what we discussed in the opening comments, we're going to have one trial, which is a switchover trial.
Speaker Change: Where patients are treated with <unk> those patients not responding to <unk> mab or not optimally responding to a <unk> inhibitor. They go on combination therapy, and then and then those that do respond to combination are rolled into March.
Speaker Change: Monotherapy with Zao <unk>.
Speaker Change: Alone.
And again patients who have not been exposed.
Speaker Change: Or not being treated with <unk>.
Speaker Change: Complement inhibitors.
The designs I think more specifically I'll, let Andreas and Steve Steve address I'll, just make one more comment on the FDA.
Speaker Change: And the meetings with European regulators.
Speaker Change: Those meetings.
Speaker Change: Went very well I think the response.
Speaker Change: To our data.
Speaker Change: And frankly to the drug.
Speaker Change: And both of those settings.
Speaker Change: <unk> was quite positive.
Speaker Change: And I think collaborative and that we're all working towards the same thing, which is moving <unk> through phase III studies, effectively and and making it available to patients that was certainly my takeaway.
Speaker Change: From both of those meetings with respect to population enrichment again.
Speaker Change: Let me turn this over to Andreas and then Steve you as well.
Speaker Change: Yeah. So just with regard to the design the design of these for sugar.
Speaker Change: It's really following <unk>.
Speaker Change: Established precedent.
Speaker Change: Similar to the successful.
Speaker Change: Perfect.
Speaker Change: <unk>.
Speaker Change: Yes, Greg.
Speaker Change: Greg was planning out patients that are <unk>.
Speaker Change: Having an insufficient or unsatisfactory response to either add Q.
Speaker Change: And then be available to be randomized to receive either a sub tenant Bart or continue on treatment.
Second trial is a trial in a.
Speaker Change: Population of patients not currently treated with a <unk> inhibitor, either totally naive or with.
Speaker Change: A long hiatus.
Speaker Change: Is there any kind of previous.
Speaker Change: Inhibitor treatments that theyre functionary nave.
Speaker Change: And that trial, we're planning also randomized trial.
Speaker Change: Randomized these patients to receive either sultana barge or a C five inhibitor.
Speaker Change: With regard to.
Speaker Change: Enrichment.
Speaker Change: The population that we're going to.
Speaker Change: In both trials are patients.
Speaker Change: Do not.
Speaker Change: New treatments that have a.
The hemoglobin.
Speaker Change: Dave.
Speaker Change: Not satisfactory.
Speaker Change: Typically below 10.
Speaker Change: Rest of the year.
Speaker Change: Obviously are very confident that we're going to improve that.
Speaker Change: Okay, Great. That's helpful and then if so.
Speaker Change: Anything that you guys do you ultimately get awarded a priority review voucher for Pediatrics <unk> can you just talk through what your strategy might be and whether selling the voucher is something that you are really considering.
Speaker Change: It's a good question I think at this point Olivia it's premature to discuss what our strategy would be I think we would have to we would have to assess the landscape.
Speaker Change: Out there what what is the interest.
Speaker Change: The voucher, which historically is high but I mean, we may well have risen and the good reason too to keep it for ourselves.
Speaker Change: As well I mean, I think it all depends on how the studies play out how our programs move forward.
Speaker Change: What the landscape looks like.
Speaker Change: That time.
Speaker Change: Okay, great very helpful. Thank you.
Speaker Change: Thank you. Thank you and one moment, Sir our next question.
Speaker Change: Our next question is going to come from the line of Serge Belanger with Needham. Your line is open. Please go ahead.
Serge Belanger: Hi, good afternoon.
I guess a question on the Oems an annual six phase III program.
Serge Belanger: Can you just give us a sense of the potential size and cost estimate of this program and whether your current cash balance provides you the bandwidth to.
Serge Belanger: To move it forward or you would seek a partner too.
Serge Belanger: To complete that phase III program.
Serge Belanger: Thank you Serge.
Speaker Change: Again, I'll answer at a high level, and then turn it turn it to clinical to address the specific.
Speaker Change: The sizes of the studies.
Certainly we're looking to move the programs forward ahead independently. We are confident that we can do that data look really good.
Speaker Change: The safety profile of the drug as I mentioned.
Speaker Change: Really quite compelling. So we think we have significant differentiators between <unk> and other alternative pathway inhibitors on the market or in development now having said that.
Speaker Change: Certainly we always have ongoing.
Partnering discussions we don't speak specifically to those.
Speaker Change: But as one might expect.
Speaker Change: With a phase III asset that looks like <unk> looks there there would be expected.
Speaker Change: The significant interest so let me stop there and turn to I don't know Andreas or Steve who would like to address the size of these studies.
They signed the percentages were.
Speaker Change: Again, both of these studies.
Speaker Change: Random mindset.
Speaker Change: We expect these studies.
Two.
Speaker Change: Yes slightly.
Speaker Change: Slightly below 100 patients per trial.
Speaker Change: Okay.
Speaker Change: So these are relatively small studies and readily managed.
Steve Brozak: Steve anything you want to add to that.
Steve Brozak: Yes.
Steve Brozak: And when I put just a little more color around it we.
Steve Brozak: We saw really good efficacy.
Steve Brozak: Earlier studies.
Steve Brozak: So that really helps us with our power and so we don't have to get large.
Speaker Change: <unk> and <unk>.
Speaker Change: I think another issue here as we look at the duration of the studies and we've already been look and we've already been finding pockets of patients so where we should be.
Speaker Change: We should hit the ground running in many places and be able to get this enrolled.
Steve Brozak: Recently quick period of time, that's great Steve. Thank you and thank you Andreas and just do.
Speaker Change: Just to underscore what you are saying.
Speaker Change: The fixed monthly costs.
Speaker Change: But running a drug trial, we expect will be.
Speaker Change: We will be.
Speaker Change: Sure.
Speaker Change: Affected positively by the enrollment that we that we foresee for both of these studies and frankly the ground work the clinical operations team has done in identifying those patients lining up those patients.
Speaker Change: Our ability to enroll them and run them through the trial quickly is that is that a fair statement.
Speaker Change: Absolutely right.
Okay.
Speaker Change: No. Thank you.
Thanks Serge.
Speaker Change: Thank you and I would now like to hand, the conference back over to Dr. Demopoulos for closing remarks.
Greg Demopoulos: Thank you operator.
Greg Demopoulos: In closing, we'd like to thank all of you for joining the call.
Obviously, we are encouraged by our recent meeting with FDA and expect that our.
Greg Demopoulos: Today's lengthy engagement with the agency is nearing its conclusion, which should result in the resubmission of our BLA and that we have the additional evidence and analysis to support approval. We will of course keep you updated.
As we proceed.
Greg Demopoulos: Again, all of US out <unk> appreciate your continued interest and your continued support.
Greg Demopoulos: Have a good afternoon.
Speaker Change: This concludes today's conference call. Thank you for participating you may now disconnect.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Yes.
Speaker Change: Sure.
Speaker Change: Okay.