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  Title	Q4 2024 Omeros Corp Earnings Call
  ID	8121017
  External ID	132710731
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  Published Date	April 1, 2025, 1:27 p.m.
  Summary	LIVE Trump announces new tariffs from the Rose Garden. Watch here. Q4 2024 Omeros Corp Earnings Call Thomson Reuters StreetEvents Tue, Apr 1, 2025, 9:27 AM 38 min read In This Article: OMER +1.26% Par
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  Content	LIVE Trump announces new tariffs from the Rose Garden. Watch here. Q4 2024 Omeros Corp Earnings Call Thomson Reuters StreetEvents Tue, Apr 1, 2025, 9:27 AM 38 min read In This Article: OMER +1.26% Participants Jennifer Williams; Investor Relations; Omeros Corp Gregory Demopulos; Chairman of the Board, President, Chief Executive Officer; Omeros Corp David Borges; Chief Accounting Officer; Omeros Corp Catherine Melfi; Vice President - Regulatory Affairs and Quality Systems, Chief Regulatory Officer; Omeros Corp Nadia Dac; Vice President, Chief Commercial Officer; Omeros Corp J. Steven Whitaker; Vice President - Clinical Development; Omeros Corp Steve Brozak; Analyst; WBB Securities, LLC Olivia Brayer; Analyst; Cantor Fitzgerald Brandon Folkes; Analyst; Rodman & Renshaw, LLC Presentation Operator Good afternoon, and welcome to today's earnings call for Omeros Corporation. (Operator Instructions) 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. 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. 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 and the Risk Factor 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. Gregory Demopulos 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 compliment 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, 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 US 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 biologic 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 analyses, 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 in the narsoplimab pivotal trial and the control patients. At FDA's request, the analyses were also adjusted for potential immortal time bias. The primary analysis shows a more than threefold 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 analysis 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 analysis 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 analysis 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 have 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, planned 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 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, authorizes 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 are 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 their supplement. The second reported on high-risk TA-TMA patients who had failed eculizumab and were subsequently treated with narsoplimab, achieving an over threefold 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 by 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 US, 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 US transplant specialists and professional associations, were responsible for establishing an ICD-10 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 zaltenibart program. Zaltenibart, also known as OMS906, is our lead MASP-3 inhibitor shown to block activation of the alternative pathway of complement, a pathway that is therapeutically validated and involved in a wide range of diseases. Zaltenibart treatment has several important characteristics that positively differentiate it from treatment with other complement inhibitors. These differentiators, including enhanced efficacy, convenient and reliable dosing, and potential safety advantages are related both to the zaltenibart molecule and its target, MASP-3. We continue making good progress in developing data demonstrating zaltenibart's potential to be the preferred choice for the treatment of alternative pathway-related diseases. Zaltenibart is now in Phase 3 development for paroxysmal nocturnal hemoglobinuria, or PNH, a life-threatening hematologic disorder. Our PNH Phase 3 program is comprised of two studies: one in PNH patients who are not receiving treatment with a complement inhibitor, and the other following a switchover design in which zaltenibart is administered to PNH patients who have a suboptimal response to treatment with a C5 inhibitor, either eculizumab or ravulizumab. These study designs and populations are very similar to those of our successful Phase 2 trials, substantially de-risking our ongoing Phase 3 program. Both of our Phase 3 clinical trials directly compare the efficacy and safety of Zaltenibart monotherapy to that of the C5 inhibitors eculizumab and ravulizumab, and both FDA and European regulators have agreed with the trial designs. This is the first time that both trials evaluating an alternative pathway inhibitor and a Phase 3 program are being run with an active comparator. So both of our trial designs provide head-to-head comparisons with eculizumab and ravulizumab and should provide data demonstrating superiority of zaltenibart over the therapeutic class of C5 inhibitors. These head-to-head comparisons are important for several reasons. First, these data could form the basis for comparative superiority claims for promotion. This would also allow our sales and marketing teams to discuss zaltenibart's superiority over C5 inhibitors with treating physicians, greatly accelerating healthcare provider awareness of zaltenibart's advantages. The comparative data can also enhance market access and support pricing appropriately reflective of zaltenibart's advantages over other agents. Also to support market access and pricing, we met with the German Federal Joint Committee. The committee determines availability of reimbursement from German statutory health insurance funds and has specialized expertise in patient reported outcome measures. Their recommended measures were incorporated into the zaltenibart Phase 3 design and are expected to help further secure appropriate pricing. Looking at trial logistics and execution, we have completed all zaltenibart manufacturing needed for our Phase 3 program. We've also identified and sourced active comparator drug. The trials include a total of 120 clinical investigative sites across 30 countries, which were carefully evaluated and chosen for clinical trial participation based in large part on their respective abilities to conduct well-run clinical trials and to contribute substantial enrollment. Pools of PNH patients ready to participate in the two trials have been identified, and efforts continue to locate additional patients. We anticipate all data needed for submission of the BLA and global approval dossiers for zaltenibart in PNH to be available in the fourth quarter of next year. The market size for PNH is large and growing, reported to be $3.9 billion in 2023 and projected at over $10 billion in 2032. We believe that zaltenibart clinical program will position Omeros to capture a substantial portion of that market. In addition to the Phase 3 clinical program, we have two other ongoing PNH clinical trials. One trial is an extension study to provide long-term safety and efficacy data. This trial enrolls patients who have completed other Omeros PNH clinical trials. The other clinical trial is the previously reported evaluation of zaltenibart in patients who are naïve to complement inhibitor treatment. Data from this trial have previously been presented at the American Society of Hematology and European Hematology Association annual meetings. This study has been amended to treat patients with our Phase 3 dose, 8 milligrams per kilogram of intravenous zaltenibart treatment once every eight weeks, further de-risking our Phase 3 program. The study shows strong efficacy. At the last available time point, all patients achieved an increase in hemoglobin of at least 2 grams per deciliter from their study entry baseline, one component of our Phase 3 primary endpoint. 12 of 13 patients without myelodysplastic syndrome, which is a disease that suppresses bone marrow function and the generation of blood cells on which you would not expect an alternative pathway inhibit to be effective, have achieved an absolute hemoglobin of greater than 12 grams per deciliter, the other component of our primary endpoint. These data are very encouraging for our Phase 3 outcomes. No safety signal of concern has been observed with zaltenibart. We've submitted two abstracts to the upcoming conference of the International PNH Interest Group. Both were accepted and will be presented at the conference occurring in mid-May in Paris. We also have submitted abstracts to the 30th European Hematology Association Annual Congress. Acceptance notifications will be sent by the Congress organizers by April 24. Given the strength of our PNH data and the established validation of the alternative pathway in PNH, our commercial team has already begun planning for market launch. We conducted an advisory board in parallel with the American Society of Hematology annual meeting in December. The advisors were PNH experts from academic and community settings. And we've learned that both are critical, meaning academic and community physicians in a PNH patient's diagnosis and treatment journey. The experts uniformly underscored the unmet treatment needs remaining in PNH, specifically the patient problems resulting from extravascular hemolysis caused by C5 inhibitors and compliance concerns with newer oral therapy. There was significant discussion about the experts' concerns regarding robust compliance mechanisms required with current oral therapies to prevent treatment gaps that could lead to breakthrough hemolysis and life-threatening thrombotic events. They characterized compliance as multifactorial, not just remembering to take a pill or a subcutaneous injection, both which physicians cannot monitor. But with pills, self-administered injections, and other primary pharmacy benefit products, a significant issue is whether the patient can even afford to pick up their next prescription or instead choose to delay the refill due to other bills that need to be paid. This type of delay could be quickly life threatening. These experts are convinced that zaltenibart offers unique benefits improved hemolysis control with convenient dosing that healthcare providers administer only four to six times per year, giving both the physician and the patient confidence that effective treatment is on board. They also emphasized zaltenibart's expected improvement in the patient's experience and life, limiting the time that the patients are required to think about treatment with zaltenibart to only a few occasions each year. Really giving the patients a sense of empowerment over their disease by reducing PNH visibility and enabling those patients to almost forget about their PNH between infrequent zaltenibart treatments. We're also advancing zaltenibart for C3 glomerulopathy, or C3G, an ultra-rare kidney disease commonly affecting children. Dosing is ongoing in our Phase 2 study in this indication. We recently amended the protocol to expand the study population. We've added a treatment cohort of patients with normal plasma C3 levels, a group that is a substantial portion of the total C3G population. The Phase 2 C3G trial requires enrollment of a relatively small number of patients and assuming strong evidence of efficacy, we look forward to initiating our Phase 3 program in C3G. As a reminder, zaltenibart received her pediatric disease designation from FDA for the treatment of C3G. This designation can allow us to request a priority review voucher that can be used for a different product, cutting its review time to six months. The vouchers can also be transferred or sold and have had a recent market value of over $100 million per voucher. We're very excited about the potential for MASP-3 inhibition in PNH ,C3G, and a number of other indications involving the alternative pathway. And we are confident that zaltenibart's anticipated advantages, specifically more consistent efficacy, better safety, and superior dosing compliance and convenience with significantly lower treatment burden, can effectively differentiate zaltenibart from other alternative pathway targeting therapeutics on the market, or in development. Let's now look quickly at OMS1029, our next-generation long-acting MASP-2 inhibitor. Phase 1 clinical trial data make clear that OMS1029 can readily be dosed subcutaneously once every three months, providing a very convenient dosing regimen for chronic indications administered either in healthcare centers or at home. Our recent focus has been on narsoplimab and zaltenibart and deciding on next indications for each, we are also honing in on the first indication for Phase 2 development of OMS1029. The product has already been manufactured, and the quantity stored should be more than sufficient to support a Phase 2 trial. In addition to our antibody inhibitors of MASP-2 and MASP-3, progress continues on the development of potential drug candidates in our small molecule orally available MASP-2 and MASP-3 inhibitor programs. To conclude our discussion on our franchise of complement therapeutics, our studies continue with MASP-2 inhibition in acute respiratory distress syndrome, or ARDS. We've generated compelling data and established animal models across all forms of severe ARDS: bacterial, viral, and chemical ARDS and are awaiting completion of one additional study ongoing in animals infected with H5N1 avian influenza, or bird flu, which many experts believe will become the next life-threatening global pandemic. Once the results from the H5N1 study are available, we'll submit the already drafted manuscript broadly directed to narsoplimab's benefits in ARDS for publication in a high-level peer reviewed journal. Another publication we expect will be coming soon from Weill Cornell, which is focused on MASP-2 inhibitions effects in long COVID. I'll turn now briefly to OMS527, our PDE7 inhibitor program, aimed at treating addictions, compulsions, and movement disorders. OMS527 is being developed for the treatment of cocaine use disorder at the request of and with funding from the National Institute on Drug Abuse, or NIDA. As recently announced, we successfully completed drug-drug interaction safety studies, where OMS527 showed no enhancement of cocaine's detrimental effects. Instead, OMS527 was beneficial to cocaine administered animals. In view of this success, NIDA committed to fund $4 million for the upcoming year, which will fund the planned randomized double-blind parallel group inpatient Phase 1b clinical trial comparing the safety and efficacy of OMS527 to placebo in the treatment of adults with cocaine use disorder. The preclinical, clinical, and mechanistic data generated to date suggests that our PDE7 inhibitor program could be effective in treating not just cocaine use disorder, but a broad range of addictions and compulsions. The work on the Phase 1b program is underway, and a preliminary data readout is targeted for year-end. We'll conclude today's review with our first-in-class therapeutic platform of molecular and cellular programs targeting a wide range of therapeutic areas including cancer. A building on our understanding of immunity both innate and complement mediated and adaptive, meaning B cells as well as CD4 and CD8 T cells, our objective is to move beyond existing targeted biologics such as antibody drug conjugates, which have small therapeutic indexes and limited tissue penetrance. And to advance engineered cellular therapies such as our CAR-T cells, which are expensive and time consuming, these, again, we are planning to move beyond. To achieve this, we're developing a portfolio of next-generation biologics to treat cancer consists of new modalities of targeted drug conjugates with better therapeutic indexes and better tissue penetrates, which we expect will eventually sideline the current ADC technology. Our portfolio includes an addition to adoptive T cell technology combined with an immuno stimulator that is easier, faster, and cheaper than current cellular therapy approaches. Our technology also maintains an enhanced anti-cancer immune response through subsequent, repeat, and simple therapeutic administrations. We're rapidly advancing our oncology programs and stealth development while we continue to build our intellectual property position. Over the past several months, we've sought the input and guidance of therapeutic area experts and advisors under confidentiality and have received a uniformly positive response. Our oncology platform is not our only stealth program. We're also developing and have created broad intellectual property around a program targeting the increasingly life-threatening challenges in infectious disease. We expect to be able to share our success publicly across both our oncology and infectious disease programs in the very near future. So with that, I will turn the call over to David. Story Continues David Borges 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 30, 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 zaltenibart. Interest expense for the fourth quarter was $3.2 million which was $1 million lower than in the third quarter of this year. The primary components of interest expense are the 2026 notes, the OMIDRIA royalty obligation, and the secured term loan. In the fourth quarter, we recorded a $4.1 million non-cash remeasurement adjustment to interest expense. Related to changes made to the OMIDRIA royalty obligation. This credit was $700,000 higher than the similar adjustment recorded in the third quarter and is a primary driver of the decrease in interest expense for the fourth quarter. Interest and other income totaled $2.3 million in both the fourth and third quarters. Income from discontinued operations in the fourth quarter was $5.2 million, down just $300,000 from the third quarter. The fourth-quarter total includes two primary components. First, a $4.1 million of interest earned on OMIDRIA contract royalty assets and $600,000 of remeasurement adjustments to the OMIDRIA contract royalty assets. As previously discussed, royalties earned are recorded as a reduction of the OMIDRIA contact royalty asset on our balance sheet rather than recognized in our income statement. OMIDRIA royalties for the fourth quarter totaled $10.1 million based on OMIDRIA net sales of $33.6 million. This compares to royalties of $9.3 million on third-quarter net sales of $31 million, representing an increase of $762,000 in royalties and $2.5 million in net sales quarter over quarter. Compared to the fourth quarter of 2023, fourth quarter of 2024 OMIDRIA royalties decreased by $631,000 corresponding to a $2.1 million decline in net sales. As a reminder, in February 2024, we entered into an amended agreement with DRI under which they acquired the right to receive all US OMIDRIA royalties payable by Rayner through December 31, 2031. Omeros retains all royalty rights to ex US sales of OMIDRIA, and we are entitled to receive all US royalties on OMIDRIA sales from and after January 1, 2032. In other words, all global royalty payments will accrue to Omeros beginning January 1, 2032. Now, let's take a look at our expected first-quarter 2025 results. We anticipate that overall operating expenses from continuing operations in the first quarter of '25 will be comparable to the fourth quarter of 2024. Interest and other income for the first quarter is expected to be approximately $1.1 million. Interest expense, including any non-cash adjustments related to the OMIDRIA royalty obligation, should be around $7.2 million. This represents a non-cash increase of $4.1 million from the fourth quarter, primarily reflecting the absence of a significant non-cash adjustment tied to the OMIDRIA royalty obligation. And as a reminder, the senior term loan transaction we closed in June 2024 included a $29.8 million gain resulting from repurchasing a portion of our 2026 convertible notes. Under GAAP, we're unable to recognize that gain immediately. The $29.8 million gained is deferred and advertised over the term of the senior loan, reducing interest expense. Inclusive of the deferred gain, we calculate the annual effect of interest rate to be 1.5%, and we expect to incur roughly $400,000 in interest expense for the first quarter of '25 related to the senior loan. And finally, income from discontinued operations is expected to be in the $7 million to $8 million range, excluding any non-cash remeasurement adjustments to the OMIDRIA contract asset. With that, I'll turn it back over to Greg. Gregory Demopulos Thanks, David. Operator, would you open the call to questions, please? Question and Answer Session Operator (Operator Instructions) Steve Brozak, WBB Securities. Steve Brozak, your line is now open. Please check your mute button. Steve Brozak Sorry about that. Can you hear me? Gregory Demopulos Hello. Steve Brozak 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 into 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. Gregory Demopulos Okay, that could take a while. Let me do this at a high level. Look, what we did in the analyses, 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 down the line, whether it's the primary or the primary-related sensitivity analysis or the analyses that we conducted with the expanded access program. Not only descriptive analysis 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 narsoplimab 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 treatment 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 speak for themselves. We've put the data out publicly. Folks, I'm sure 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. Catherine Melfi 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. Gregory Demopulos That answer the question, Steve? Steve Brozak 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. Gregory Demopulos Thank you. Operator Olivia Brayer, Cantor. Olivia Brayer Hey, good afternoon, thanks for taking the questions. Gregory Demopulos Hi, Olivia, how are you? Olivia Brayer I'm good, Greg. How you doing? 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. Gregory Demopulos 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's 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? Nadia Dac 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 threefold benefit and overall survival 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 cost within the inpatient setting, cost 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. Gregory Demopulos Okay. Thank you, Nadia. And then with respect to your second question around how we characterize zaltenibart. As I mentioned in the prepared statements, we have not seen a safety signal of concern with zaltenibart. That includes LFTs or lipid profile changes. I have not seen it, and I think that's what you're referencing since those have been identified with other agents. I'll hand this over to Steve Whitaker who's here, but I also would expect that very likely those changes that people have seen around LFTs and lipid profiles very well may not be target related, but I'll let Steve comment on that. J. Steven Whitaker Thanks, Greg. We haven't seen anything that we think is a liver safety signal with narsoplimab -- sorry, zaltenibart. And obviously, we look at this just like every other company looks at potential liver toxicity with every drug. We have done a couple of things to mitigate confounders, and let me emphasize that, that was a confounder, not a liver toxicity issue. But as you know, PNH patients can have elevated liver function tests because they can get biliary disease from the homolysis. They get a lot of transfusions, and they can get hemochromatosis. So we have excluded prospectively patients who have significant liver disease, pre-existing liver disease, so we don't have to worry about that confounding interpretation of the clinical trials, but we do not expect to see evidence of liver toxicity in the trials, and we don't think we've seen it to date. Olivia Brayer 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. Gregory Demopulos 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 and 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 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. Olivia Brayer Okay, great. Thank you, guys. Operator Serge Belanger, Needham & Company. Hi, good afternoon. This is John on for Serge today, and thanks for taking our questions. Gregory Demopulos Hi, John. So pending a potential approval in September for narsoplimab, just curious where the company stands on any manufacturing scalabilities 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. Gregory Demopulos Yeah. So 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 first 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 to 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. Nadia Dac 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 US in February, we've been engaging -- we engaged 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 relationships, 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. Gregory Demopulos Thanks, Nadia. Any other question, Olivia -- John? Sorry. No, I'm all set. Thanks for all the color. Operator Brandon Folkes, Rodman & Renshaw. Brandon Folkes Hi, thanks for taking my question, and congratulations on the progress and 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 future? Should we think of Omeros as sort of taking its pipeline largely forward commercially itself, a combination of seeking partnerships where it makes sense, or? I'd just like to get your views, especially given sort of your history with OMIDRIA and kind of having seen both sides of it all play out. Thank you. Gregory Demopulos Thanks, Brandon. Look, I think certainly in narsoplimab, we are planning to launch in the US 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 US, absolutely, we are expecting to partner narsoplimab ex US similarly zaltenibart. That can be regional, that could be ex US writ large. There are a lot of opportunities, and we just need to figure out what makes the most sense for us. When you say long term, there are a number of products coming behind narsoplimab and zaltenibart. And certainly, at some point in the future, we would love to be able to manage ex US commercialization independently as well. I don't see that currently for either narsoplimab or zaltenibart, so very much focused on partnering those programs, again, regionally or internationally, ex US broadly. But that is the plan. You do see a deep pipeline. I think there's going to be a lot coming. I really feel like, look, with the resubmission of the BLA, things changed. I think the tenor of the company, the complexion of the company has significantly changed. We're moving back to being a commercial entity. We've done it once with OMIDRIA. And remember, we were able to commercialize that, I think extremely well. There were a lot of folks questioning what we could do with that program. OMIDRIA brought in net revenues to Omeros north of $1 billion through the sales and through the partnerships and royalty sales that we did there. So we're very excited about this next phase of Omeros, which means commercial narsoplimab, and then coming up really right behind that, zaltenibart. And when you look at the data from zaltenibart -- and the difference here is with narsoplimab, we're the first. There was no playbook for us to follow for the development and the regulatory strategy around narsoplimab. That's a very different set of circumstances for zaltenibart. We're really able to follow where others are going, doing very similar things to what they've done. But with what we believe is a meaningfully better molecule and a meaningfully better target. And out of that, we expect that -- we're very eager to get zaltenibart in the market and be generating revenues not only from narsoplimab, but from zaltenibart as well. And you know, boy, that we all look forward to. We expect very much that we will get there. You can't look at the zaltenibart data and really, I think, rationally come to the conclusion that, that product is going to have a lot of difficulty getting approved. I think conversely, we are very confident that zaltenibart will be approved as we are for narsoplimab. With narsoplimab, we had to go through a lot of extra steps, but we've completed those, and the data look really strong. So we're looking forward to September and our launch very soon thereafter and revenue generation and being right back as a commercial company. Brandon Folkes Great, thanks very much, Greg. Gregory Demopulos 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. Operator Thank you. I would now like to turn the conference back over to Dr. Demopulos for closing remarks. Gregory Demopulos Okay, thank you, operator. I'd like to thank everyone for joining us today. Well, 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. Operator Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect. View Comments Terms and Privacy Policy Privacy Dashboard More Info Recommended Stories
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