OMER - NASDAQ

Good afternoon, and welcome to today's Earnings Call for Omeros Corporation. At this time, all participants are in 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 Omeros.

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 notes and the Risk Factors section regarding forward-looking statements in the company's annual report on Form 10-K, which was filed today with the SEC. Now, I would like to turn the call over to Dr. Greg Demopulos, Chairman and CEO of Omeros.

Thank you, Jennifer, and good afternoon, everyone. I'm joined on today's call by David Borges, our Chief Accounting Officer; Nadia Dac, our Chief Commercial Officer; Andreas Grauer; our Chief Medical Officer; Cathy Melfi, our Chief Regulatory Officer; and Steve Whitaker, our Vice President of Clinical. Today, I'll start with an overview of our 2024 financial results and provide updates across our development programs. David will go through our financials in more detail, and then we'll open the call for questions. Let's begin with the financials. Our net loss for the fourth quarter of 2024 was $31.4 million, or $0.54 per share, compared to a net loss of $32.2 million, or $0.56 per share in the third quarter of 2024. For the full year 2024, our net loss was $156.8 million or $2.70 per share. As of December 31, we had over $90 million in cash and investments on hand. Now that we have resubmitted our BLA for narsoplimab in TA-TMA, which we'll discuss in just a bit. Our focus is on restructuring the balance sheet. Having substantially reduced the amount of the 2026 convertible notes last June, we now are in preliminary discussions with certain remaining holders of the 26 notes to retire and/or refinance them to extend their maturity. As part of this debt restructuring, we intend to add substantial capital for operations. In addition, discussions are underway regarding potential partnerships, primarily around our complement franchise, which includes narsoplimab, zaltenibart, and OMS1029, with additional discussions directed to our deeper pipeline. Other possible options, of course, include royalty monetization or debt or equity transactions, including through the use of our existing at the market offering facility, which provides for the sale of up to $150 million of our common stock at prevailing market prices. We're confident in our ability to leverage these opportunities to strengthen our financial position, while driving long-term growth and near-term shareholder value. We will provide an update on our progress at such time as a definitive announcement can be made. With that, let's move on to an update on our development programs, starting with our complement franchise and narsoplimab, our first-in-class inhibitor targeting MASP-2, the effector enzyme of the lectin pathway of complement. As we announced today, there has been a tremendous amount of activity moving us toward approval of narsoplimab for hematopoietic stem cell transplant associated thrombotic microangiopathy or TA-TMA, both in the U.S. and in Europe. We expect that our first approved indication for narsoplimab will be TA-TMA, a serious, often rapidly fatal condition, that occurs too frequently in children and adults who undergo stem cell transplantation. Earlier this month, we resubmitted to FDA our Biologics License Application or BLA for narsoplimab in the treatment of TA-TMA. Over the past year, we've been working closely with FDA to complete the resubmission. This includes our meeting with the agency last September to finalize the content to be included in the resubmission and the subsequent receipt in November of FDA's final comments on both the protocol and statistical analysis plan to compare survival in narsoplimab treated TA-TMA patients to that of an external control population. The Independent Statistics Group then incorporated all of FDA's comments and ran the analyses. Results of these analyses, which we have shared publicly and are included in the BLA, demonstrate narsoplimab's significant and clinically meaningful survival benefit. Resubmissions have no filing period with the six month review clock starting on the date sent to FDA, setting in this case a PDUFA date in September of this year. We do not expect that recent or anticipated changes at FDA will affect the review of our resubmission. Let's spend a little more time on those results of the narsoplimab analysis which I expect will be instructive. As previously reported, the primary and all primary related sensitivity analyses were agreed with FDA and include propensity matching, which further enhances the matching between the patients and the narsoplimab pivotal trial and the controlled patients. At FDA's request, the analyses were also adjusted for potential immortal time bias. The primary analysis shows a more than three-fold improvement in survival associated with narsoplimab treatment with a p-value of less than 0.00001. The related sensitivity analyses recommended by FDA were also conducted with results strongly and consistently supporting that same survival benefit. FDA further requested that we conduct analyses to assess even the effect of unmeasured confounders on our results. Recommending that we use a measure called an expectation value or e-value. The e-value for our primary analysis was 5.7, demonstrating that the possibility of our results being affected by unmeasured confounders is vanishingly small. In other words, the results of the statistical comparison of narsoplimab versus a well-matched and rigorous external control group are robust, consistent, statistically significant, and highly clinically meaningful. Uniformly demonstrating compelling evidence of improved survival for narsoplimab in TA-TMA. The BLA resubmission also presents analyses of survival in our global Expanded Access Program or EAP, often referred to as compassionate use with 136 adult and pediatric TA-TMA patients at time of database lock. This includes a comparison of survival in the EAP versus survival in the same external control group used for the primary analyses and the related sensitivity analyses. The results of these EAP comparisons were consistent with those of the pivotal trial primary analyses. Hazard ratios, p-values and e-values all consistent. The EAP data also assess survival in adults and children with TA-TMA, who had failed treatment with other complement inhibitors, namely eculizumab, ravulizumab and pegcetacoplan as well as defibrotide prior to receiving narsoplimab. These refractory TA-TMA patients showed one year survival in excess of 50%, more than 2.5 fold higher than the historically referenced survival in these refractory patients of less than 20%. Two months prior to resubmitting our BLA, we had shared with FDA all data from the primary analysis and the primary related sensitivity analyses, and over the following several weeks, also sent in completed portions of the resubmission. These submissions were provided at the agency's request to allow FDA's reviewers to begin their review prior to receipt of the full resubmission, but FDA made clear that the review clock would not start until the full submission was received, which again occurred earlier this month. Concurrent with preparing and submitting the BLA, we've been compiling our Marketing Authorization Application, or MAA, for submission to the European Medicines Agency, or EMA, a plan for the coming quarter. In anticipation of our upcoming submission, the EMA has appointed rapporteurs to shepherd our application through EMA's Committee for Medicinal Products for Human Use, or CHMP. The narsoplimab rapporteur country is Germany and the co-rapporteur is Austria. Omeros met previously with regulatory authorities in multiple EMA member countries, including Germany, regarding narsoplimab for TA-TMA, and scientific advice from these meetings has been incorporated into the upcoming MAA. Upcoming meetings are scheduled between Omeros and representatives from both rapporteur countries, at which we will orient the representatives to the AM -- to the MAA included data. Rather than requiring separate national approvals across Europe, the narsoplimab MAA is eligible for submission under EMA's centralized review procedure. This allows us to submit a single MAA that if approved, authorized as marketing of the product in all 27 European Union member states, plus Iceland, Norway, and Liechtenstein. Narsoplimab has orphan drug designation from EMA for treatment in hematopoietic stem cell transplantation. In preparation with our efforts on BLA and MAA submissions, two manuscripts authored by groups of international transplant experts have been drafted and are under review. Once finalized, both manuscripts will be submitted for publication in premier peer reviewed journals. The first consistent with our primary endpoint for regulatory approval, compares survival of patients in the narsoplimab pivotal, TA-TMA trial, with the same external control used for our submissions. The second manuscript details the survival data from our global Expanded Access Program in the 50 narsoplimab treated children. Last month, two presentations reporting real world outcomes from TA-TMA patients treated under the narsoplimab EAP were featured at the 2025 tandem meetings in Honolulu. The first, a podium presentation, reported on the impressive survival in both adult and pediatric high-risk TA-TMA patients, including those who had failed another TA-TMA-directed therapy prior to receiving narsoplimab. The second reported on high-risk TA-TMA patients, who had failed equilizumab and were subsequently treated with narsoplimab, achieving an over three-fold increase in one year survival compared to the reported less than 20% survival in eculizumab-refractory patients. Preparations for the market launch of narsoplimab are going well, and our commercial team is well prepared and eager to launch the drug. Transplant experts continue to express their patients' need for narsoplimab, asking how quickly it will be approved. As an increasing volume of data are made public regarding the safety concerns surrounding the currently used off-label therapies for TA-TMA. The need becomes increasingly acute. There are 174 transplant centers in the U.S., 40 of those centers perform 60% of the transplants and 80 sites perform -- 80%. We effectively have relationships across these centers, and anticipation for the launch of narsoplimab is high. Recall also that we in collaboration with leading U.S. transplant specialists and professional associations were responsible for establishing an ICD10 diagnostic code and a CPT procedural code essential for billing in the treatment of TA-TMA. Once approved, narsoplimab would be the only drug specifically linked to these codes, facilitating reimbursement for use of narsoplimab while creating reimbursement barriers for off-label treatments not approved for TA-TMA. Now let's turn to our

Thanks, Greg. Our net loss for the fourth quarter of 2024 was $31.4 million or $0.54 per share compared to a net loss of $32.2 million or $0.56 per share in the third quarter of 2024. For the full year 2024, our net loss was $156.8 million or $2.70 per share. As of December 31, 2024, we had over $90 million of cash and investments on hand. Costs and expenses from continuing operations for the fourth quarter before interest and other income were $35.7 million, which was an increase of just $253,000 from the third quarter of this year. Research and development expenses in the fourth quarter were heavily focused on narsoplimab and

Thanks, David. Operator, would you open the call to questions, please?

[Operator Instructions] Our first question comes from the line of Steve Brozak from WBB Securities. Steve Brozak, your line is now open. Please check your mute button.

Sorry about that. Can you hear me? Hello?

Now, -- yes.

Hey. Congrats on the submission of the BLA, and thanks for taking the questions. Simply put, one question, you've given us details on the resubmission. Can you tell us why you believe in going to as much detail as possible, that this submission is that strong, and what are the implications? And anything else you want to add on that? And I'll hop back on the queue. Thank you.

Okay. That could take a while. Let me do this at a high level. Look, what we did in the analysis, and remember that the statistical analysis plan and associated protocol were created with FDA's agreement. So what we really did was what FDA asked us to do. And the result of those analyses, I think can be characterized as nothing else, but impressively strong, right, right down the line, whether it's the primary, or the primary related sensitivity analyses or the analyses that we conducted with the expanded access program on not only descriptive analyses on the EAP but also taking the EAP population and comparing it to the same external control group, which -- frankly, I don't believe we were even asked to do. We took it one step further and ran those analyses as well. And I would -- it would be wonderful to be able to share our forest plots from all of those analyses because it tells -- those really tell the story in one quick glance, but really everything is favoring in our supplement all the way down the line. And with the p-values and the hazard ratios that we generated, and I'll just -- I know everybody heard it, but I'll just remind that the primary hazard ratio was 0.32, meaning the likelihood of survival in the narsoplimab-treated group was more than threefold higher than in the control group. And then you look -- and that had a p-value of well less than 0.00001. Any way that you look at these, squeeze these, push on these, the data are impressive, they are statistically significant, and they're clinically meaningful. And all of those numbers that I just gave you are really aligned and supported by those same comparisons we did with the EAP population in comparison to the external control. It's all consistent. So when you're asking why we're confident about the data, I mean, the data speaks for themselves. We've put the data out publicly, folks, I'm sure, you have seen those. We've tried to make those very available. And I think, despite all I've just said, the data really speak for themselves. Let me ask Cathy if you have anything you'd like to add to that.

Yeah. I feel very strongly that we've got a solid package. We had a meeting with FDA back in September. It was an in-person meeting, very productive. As Greg mentioned, we've got a well-matched external control group. We, with FDA's input and agreement, adjusted for moral time bias, use propensity matching, and then pushed on the model every which way to see if it would budge, and consistently we're getting very strong results. And so, both with the FDA collaboration and the strength of the results, I think we've got a strong package.

Does that answer the question, Steve?

Yeah. And I'm sure everyone appreciates the detail you just went into. So obviously, good luck on the PDUFA. And I will hop back in the queue. Thank you.

Thank you.

Thank you.

Thank you. One moment for our next question. Our next question comes from the line of Olivia Brayer from Cantor.

Hey, good afternoon. Thanks for taking the questions.

Hi, Olivia. How are you?

I'm good, Greg. How are you doing? And can you comment at all on how you're thinking about pricing for narsoplimab now that you are officially in launch-prep mode? Or at least anything directional would be really helpful. And then on PNH, can you just talk about the level of confidence you have in the clinical profile we've seen to date, and whether there's anything you are doing in the Phase 3 to help mitigate for liver toxicity? And then I've got one follow-up.

Sure. First, let me answer about the pricing, and then I'll hand that over to Nadia. But I think we have not disclosed pricing plans for narsoplimab. But I think one would be safe to assume that there is a range within that pricing would fall, and it's probably similar to other complement inhibitors that are used in TA-TMA. But at present, we have not yet disclosed, but let me see. Nadia, do you want to comment on that as well?

Yeah. I agree with you, Greg. And the way I look at it is -- pricing is never final until we're literally publishing it and launching. We're looking at it from every angle. The uniqueness here is that nothing is approved, and narsoplimab will be the first approved TA-TMA treatment. And as Greg just explained, the efficacy, a threefold benefit in overall survival is drive significant value. And so we're taking that into consideration as payers will look at this along with physicians. And the last aspect that's really critical is that we believe that it will be administered. It has the potential to be administered both in the inpatient and outpatient setting, and so there could be numerous payers there. So we look at it as costs within the inpatient setting, costs within the outpatient setting. So those are all the parameters. But the bottom line is we believe that narsoplimab will deliver significant value, and we're taking that into consideration with pricing determination.

Okay. Thank you, Nadia. And then with respect to your second question around how we characterize

Thanks, Greg. We haven't seen anything that we think is -- it's a liver safety signal with narso -- I'm sorry, not narsoplimab --

Okay. Got it. Thanks, guys. That's helpful. And then, last question is just around cash runway and managing balance sheet from here. So I guess more broadly, what's the strategy for funding? You guys have a lot going on this year with the commercial launch and multiple Phase 3 trials kicking off. So just how are you thinking about that broader kind of balance sheet management strategy? And then specifically, how you're thinking about your current cash runway from here? Thank you, guys.

Yeah. Sure. Thanks, Olivia. Obviously, we're very aware of that. We're on top of it. We commented about discussions that are ongoing. Our objective here would be, as I think I made clear to restructure the converts and bring in additional capital in so doing. There are a lot of other levers that we have to pull, whether those be partnering, whether those be other debt instruments, whether that'd be royalty monetization or even equity, all of those are levers that we can pull. But we're quite aware of what needs to be done. And we wanted to turn some of these cards over, which I think we've now done, perhaps more coming. And what we will see is how to first, let's take care of what's right in front of us and move on from there. So we're quite confident that we've got this well in hand.

Okay. Great. Thank you guys.

Thank you. One moment for our next question. Our next question comes from the line of Serge Belanger from Needham & Company.

Hi. Good afternoon. This is John on for Serge today, and thanks for taking my question.

Hi, John.

So, pending a potential approval in September for narsoplimab, just curious where the company stands on any manufacturing scalability required to adequately support market demand? And secondly, as the potentially first product approved in this category, have you begun or plan to begin making any inroads on increasing awareness for narsoplimab with treating physicians? Thanks.

Yes. Let me answer first, and then I'll hand it to Nadia. In terms of manufacturing, we have all the drug supply necessary to launch and to support narsoplimab through the three years -- through the first several years of -- two to three years of utilization. So we're well set there, John. That drug product, drug substance and drug product are available. With -- and remember that we would expect to be launching that product very soon. After approval, our commercial team -- one of the few benefits of having this approval that we expect take so long from the initial CRL is that certainly our commercial team has been able to interact with the experts and there has been a tremendous amount of education in identification of TA-TMA patients and identification of high-risk TA-TMA patients and also with respect to their treatment. So let me hand that over to Nadia and see -- Nadia provide any additional color.

Yeah. I'll build on Greg's comments. We have a small but very targeted team in the field that has been working, as Greg said earlier, with not only our top 40 accounts that drive 60% of the allogeneic transplant volume, but all the way up to those 80 accounts that drive 80% of the allogeneic transplant volume. And they're building what we consider the first phase of the pre-launch and then launch, it's that awareness of TA-TMA. There is over -- if you rewind, historically, there has not been a lot of harmonization of understanding of how to diagnose and then treat TA-TMA. That awareness has increased significantly, a lot due to the efforts in the field, also with our MSLs on the medical side. And at a recent conference, the most important transplant conference in the U.S. in February, we have been engaging -- we engage with over 100 physicians, and the TA-TMA topic is mainstream. That's being raised by the physicians, and they want a solution. So there is a very strong relationship, a strong foundation of awareness, but those efforts have to continue. So we're very excited to be able to provide a solution in short order here.

Thanks, Nadia. Any other question, Olivia -- John, sorry.

No, sir. Thanks for all the color.

Great.

Thank you. One moment for our next question. Our next question comes from the line of Brandon Folkes from Rodman & Renshaw.

Hi. Thanks for taking my question, and congratulations on the progress in the resubmission. Greg, maybe for you. Can you just talk sort of at high level, how do you think about Omeros strategically longer-term now with narsoplimab being resubmitted and the ability to obviously self-commercialize there? But a number of these opportunities are large market opportunities, and you have a very deep and broad pipeline. How do you think about partnering commercially in the future? Should we think of Omeros as sort of taking its pipeline largely forward commercially itself? A combination of seeking partnerships where that makes sense, or -- I'd just love to get your view, especially given sort of your history right with OMIDRIA, and kind of having seen both sides of it all play out? Thank you.

Thanks, Brandon. Look, I think certainly in our supplement, we are planning to launch in the U.S. independently. We have the team ready to do that. We have the relationships with the transplant centers, with the transplanters. And as Nadia said, a tremendous amount of work has gone in on the front-end of that to make sure that we have a very successful launch. With respect to ex-U.S., absolutely. We are expecting to partner narsoplimab ex-U.S., similarly

Great. Thanks very much, Greg.

Yeah. And I would not forget the pipeline programs. So I would pay very much attention to what's happening in our oncology programs and in the other programs that we mentioned.

Thank you. I would now like to turn the conference back over to Dr. Demopulos for closing remarks.

Okay. Thank you, operator. I'd like to thank everyone for joining us today. Before we close, I just want to acknowledge the entire Omeros team. That continues to build a series of truly cutting-edge programs that will likely change the landscape in their respective fields. We look forward to sharing more with you in the very near future. All of us at Omeros appreciate your continued support. Have a good evening. Thank you.