CYGB

Researchers have discovered that a certain kind of protein similar to hemoglobin, called cytoglobin, plays an important role in the development of the heart. Specifically, it affects the correct left-right pattern of the heart and other asymmetric organs. The findings could eventually lead to the development of new therapeutic interventions to alter the processes that lead to these defects. In a landmark study led by the University of Maryland School of Medicine, researchers discovered for the first time that a certain kind of protein similar to hemoglobin, called cytoglobin, plays an important role in the development of the heart. Specifically, it affects the correct left-right pattern of the heart and other asymmetric organs. The findings, published today in the journal Nature Communications, could eventually lead to the development of new therapeutic interventions to alter the processes that lead to these defects. The team used CRISPR gene editing technologies to knock out the cytoglobin gene in zebrafish. The lack of cytoglobin caused the development of embryos with a mirrored heart, meaning the heart had a reversed left-right pattern. In humans, cytoglobin is involved in processes involving nitric oxide, a compound that helps regulate healthy blood flow to organs. Study co-senior author Mark T. Gladwin, MD, the John Z. and Akiko K. Bowers Distinguished Professor and Dean, University of Maryland School of Medicine, and Vice President for Medical Affairs, University of Maryland, Baltimore, has been researching the effects of nitric oxide on blood vessels for more than 20 years including in this recent study finding. "Since its discovery two decades ago, cytoglobin has been found to be expressed in nearly all human tissues, but the mechanisms of how this protein functions were largely unknown," said Dr. Gladwin. "We know that cytoglobin can play a role in modulating and maintaining nitric oxide levels, but our new finding indicates that it positively regulates NO production to ensure proper cilia function and its absence can lead to major laterality abnormalities of organs." To conduct the study, the research team knocked out the gene for cytoglobin in zebrafish and were amazed to see that it led to dramatic defects in the structure and location of organs in developing embryos. The heart, for example, was located on the right side of the fish instead of the left with a looping to the left instead of the right. "We found that cytoglobin plays a vital role in the structure and function of tiny hair-like structures called cilia, which determine the asymmetry and proper development of organs," said study senior author Paola Corti, PhD, Assistant Professor of Biochemistry and Molecular Biology at UMSOM. This is the first time cytoglobin -- or any of the globin proteins like hemoglobin -- has been found to be involved in fetal development and that a paucity could be linked to birth defects. It's also the first time that cytoglobin has been linked to cilia function. Such a finding could open the door for the development of therapeutics for rare birth defects that affect the movement of cilia. About 1 in every 10,000 to 30,000 people are born with Primary Ciliary Dykinesia (PCD), a rare disease that affects the cilia and can cause breathing issues from thickened mucus clogging airways. "Kartagener's syndrome is a form of PCD and is known to cause the type of heart defects seen in the zebrafish where the heart is abnormally positioned to the right and rotated," said Dr. Corti. "There is no cure for this condition, just surgery to fix any heart defects and treatments to manage symptoms." While certain genes have been identified that are known to cause about 70 percent of PCD cases, cytoglobin could play a key role in the 30 percent of cases with no known genetic cause. "We found the phenotype and connected the dots to cilia. In the presence of cytoglobin, we could track the function of the protein and how if led to proper cilia function and organ development. In the absence, we saw these defects," said Elizabeth Rochon, PhD, first author of the study and Assistant Professor of Medicine at UMSOM. Funding for the study was from the National Institutes of Health, the American Heart Association, and the Institute for Transfusion Medicine and the Hemophilia Center. UMSOM faculty co-authors include Anthony W. DeMartino, PhD, Assistant Professor of Medicine, and Qinzi Xu, MD, Assistant Professor of Medicine. Other co-authors included faculty at the University of Pittsburgh School of Medicine and the University of Copenhagen in Denmark.

An Interim analysis of 18 patients, completing 24/25 weeks of treatment in a phase 2 study in complement-naïve PNH patients demonstrates that KP104 is safe, well-tolerated, and has successfully met all primary and secondary endpoints. KP104 effectively managed both intravascular and extravascular hemolysis, with all 18 patients achieving a 2g/dL increase of hemoglobin from baseline in the absence of RBC transfusions and the majority of patients achieving normalization range of hemoglobin and LDH levels. The interim results support the advancement of KP104 to Phase 3 studies as an optimal and safe treatment for PNH patients to address currently unmet medical needs. CAMBRIDGE, Mass., Dec. 14, 2023 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, presented the interim safety and efficacy results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with PNH at the 2023 American Society of Hematology (ASH) Annual Meeting held in San Diego, on December 10th in an oral presentation. The presentation has also been selected to be featured in the 2024 Highlights of ASH. KP104 is a first-in-class bifunctional biologic engineered to inhibit both alternative and terminal complement pathways. Continue Reading Figure 1 Figure 2 Figure 3 This open-label Phase 2 clinical trial (NCT05476887) aims to assess the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in patients diagnosed with PNH who have not received prior complement inhibitors. The primary objectives of the study are to evaluate key clinical markers including lactate dehydrogenase (LDH) and hemoglobin (Hgb) levels, transfusion requirements, and FACIT-fatigue scores. The interim analysis of the Phase 2 study for KP104 demonstrated positive outcomes from 18 patients who have completed 24/25 weeks of treatment across three escalating doses: Patients showed rapid and sustained improvements in hemoglobin levels (Figures 1 and 2). At Week 24/25: 100% (18/18) achieved a ≥2g/dL increase from baseline; 56% (10/18) attained hemoglobin normalization (≥12g/dL) in the absence of RBC transfusions, with the remaining patients approaching near-normal level; Average Hgb levels for Cohort 1, 2 and 3 are 11.7 g/dL (SD 1.0), 14.2 g/dL (SD 1.9) and 12.5 g/dL (SD 1.9), respectively. Patients demonstrated rapid reduction and sustained control of LDH levels (Figure 3). At Week 24/25: 83% (15/18) sustained levels below 1.5x ULN; 72% (13/18) sustained levels below 1x ULN All patients remained free from RBC transfusion between Day 1 and Week 24/25 of KP104 treatment. Rapid and clinically significant improvements in absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores were consistently observed in all three cohorts through Week 24/25. KP104 was safe and well-tolerated without treatment-emergent adverse events (TEAEs) at or above Grade 3. Common AEs included transient injection site induration, headache, and COVID19 infection, all of which were promptly or duly resolved. Convenient dosing regimen with SC administration every 2 weeks. Kira Pharmaceuticals is committed to advancing KP104 as an innovative therapy for patients with PNH and other complement-mediated diseases. The interim results of the Phase 2 study in complement-naïve PNH patients study represent a significant step forward in developing KP104 as an optimal and safe treatment for PNH patients to address currently unmet medical needs. These data also provide a proof-of concept and strong foundation for future clinical trials in other complement-mediated diseases, such as IgA nephropathy (IgAN), complement 3 glomerulopathy (C3G), and thrombotic microangiopathies secondary to systemic lupus erythematosus (SLE-TMA). Title: KP104, a bifunctional C5 antibody/factor H fusion protein, effectively controls both intravascular and extravascular hemolysis: interim results from a phase 2 study in complement inhibitor-naïve PNH patients Authors: Fengkui Zhang, Bing Han, Li Zhang, Chen Yang, Chunrong Wang, Changhe Yue, Hui Yan, Jay Ma, Helen Fu, Chaomei He, Ping Tsui, Jingtao Wu, Richard Lee, Wenru Song Session: 508. Bone Marrow Failure: Unraveling the Future of PNH Therapy from Clinical Trials Oral Presentation Time: December 10th, 2023, 4:45pm PT About KP104 KP104 is a first-in-class bifunctional biologic designed to simultaneously block both the alternative and terminal complement pathways, providing a powerful and synergistic method of targeting validated drivers of complement-mediated disease. This dual-target mechanism of action uniquely positions KP104 to address complement-mediated diseases and potentially provide greater benefits than single-target complement agents. Engineered to have an extended half-life and enhanced potency, KP104 has a formulation suitable for both intravenous and subcutaneous administrations. KP104 is entering Phase 2 POC trials across multiple renal disease and hematologic indications and has been granted Orphan Drug Designation by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria. Phase 2 trials will be conducted globally, including in the U.S., China, and Australia. KP104 is an investigational agent not yet approved for any indication by any health authority. About Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disease that is characterized by the destruction of red blood cells, formation of blood clots, and impairment of bone marrow function. PNH affects between 1 and 5 people per million and is almost always caused by genetic mutations that result in production of aberrant hematopoietic stem cells. These stem cells produce irregular red blood cells that are highly susceptible to destruction via complement activation. Current therapies include C5 inhibitors, which do not address extravascular hemolysis (EVH) related to the alternative pathway or a C3 inhibitor, which may address EVH but may not adequately block C5 downstream, leading to life-threatening breakthrough hemolysis (Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition, N Engl J Med, July 14, 2022). About Kira Pharmaceuticals Kira Pharmaceuticals is a clinical-stage biotechnology company pioneering complement-targeted therapies to treat immune-mediated diseases. Enabled by its LOGIC platform, the company has developed a robust pipeline of novel assets against validated complement targets. Headquartered in Cambridge, Massachusetts and with facilities in China and Australia, Kira Pharmaceuticals has established a global team committed to advancing life-changing therapies to patients around the world. More information on Kira can be found at and on LinkedIn. Contact: [email protected] SOURCE Kira Pharmaceuticals

– Conference Call Today at 4:30 p.m. ET – SEATTLE--(BUSINESS WIRE)-- Omeros Corporation (Nasdaq: OMER), a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, cancers, and addictive and compulsive disorders, today announced recent highlights and developments as well as financial results for the first quarter ended March 31, 2023, which include: Net loss was $33.7 million in the first quarter of 2023, or $0.54 per share, compared to a net loss in the first quarter of 2022 of $33.0 million, or $0.53 per share; our first quarter’s net loss from continuing operations was $39.7 million compared to $39.5 million in the first quarter of 2022, and $0.63 per share for both periods. Cash burn for the first quarter of 2023 was $23.6 million, exclusive of a $200 million milestone payment received in the quarter, a decrease from $26.0 million in the prior quarter. In February 2023 we received from Rayner Surgical, Inc. (“Rayner”) the $200 million milestone payment due under the Asset Purchase Agreement by which we sold our former ophthalmology product OMIDRIA® to Rayner in late 2021. The payment became due upon the achievement of the milestone event in December 2022 and was recorded as a receivable in the fourth quarter of 2022. For the quarter ended March 31, 2023, we earned OMIDRIA royalties of $9.2 million on Rayner’s U.S. net sales of $30.7 million. This compares to earned royalties of $13.8 million during the first quarter of the prior year on U.S. net sales of $27.7 million. The above-referenced milestone event triggered a reduction of our U.S. base royalty rate from 50 percent to 30 percent. At March 31, 2023, we had $371.4 million of cash, cash equivalents and short-term investments available. In addition, we had $10.0 million of accounts receivable. We intend to resubmit our Biologics License Application (“BLA”) for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (“TA-TMA”) and, consistent with direction from FDA’s Office of New Drugs, have submitted to FDA a proposed plan for assessment of both response rate and survival based on already existing clinical trial data and external data. A meeting with the review division is scheduled for this month to discuss the details of our proposed analyses and to confirm the information required by FDA to support resubmission of the BLA for narsoplimab’s approval. In April 2023, we announced positive results from a pre-specified interim analysis of our ongoing Phase 1b clinical trial of OMS906 in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (“PNH”), and updated interim results are provided later in this release. In April 2023, we were awarded a three-year $6.69 million grant from the National Institute on Drug Abuse (“NIDA”), part of the National Institutes of Health, to develop the lead orally administered PDE7 inhibitor compound in our OMS527 program for the treatment of cocaine use disorder (“CUD”). “We are pleased with our progress during the first quarter as our team continues to deliver on important milestones,” said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. “For our MASP-2 inhibitor narsoplimab, we are meeting with FDA this month to discuss our proposed analysis plan and confirm the specifics to be included in a BLA resubmission for TA-TMA, and our Phase 3 IgA nephropathy trial remains on track for data read-out next quarter. Our long-acting MASP-2 inhibitor is set to begin enrolling its multiple-ascending-dose clinical trial this summer in the U.S. Enrollment is marching ahead in our OMS906 clinical program. The results to date in treatment-naïve PNH patients are impressive, making clear that our MASP-3 inhibitor OMS906 blocks alternative pathway activation and should be effective across AP-related diseases while potentially offering significant safety, efficacy and compliance advantages over other AP-targeting drugs. Now collaborating with and having received a $6.69 million award from NIDA, we are moving forward to assess our PDE7 inhibitor OMS527 in patients with cocaine use disorder. Our immuno-oncology franchise is also rapidly progressing across both our cellular and biologic platforms. Helping to fund the ongoing achievements of these and our other programs is the non-dilutive $200 million milestone payment received in February, forecast to extend our cash runway into 2025. This year has started well for Omeros, and we expect that momentum to continue.” First Quarter and Recent Clinical Developments Recent developments regarding narsoplimab, our lead monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (“MASP-2”) in advanced clinical programs for the treatment of TA-TMA and immunoglobulin A (“IgA”) nephropathy, include: We are preparing to meet with FDA’s Division of Nonmalignant Hematology later this month to confirm the information required by FDA to support resubmission of the BLA for narsoplimab’s approval in TA-TMA. Consistent with direction from FDA’s Office of New Drugs, provided as part of its denial of our appeal of the complete response letter previously issued by FDA, we have proposed a plan to assess already existing clinical trial data and external data on both response rate and survival. In our Phase 3 ARTEMIS-IGAN trial evaluating narsoplimab for the treatment of IgA nephropathy, we remain on track to read out 9-month data on the proteinuria endpoint next quarter. Narsoplimab is the focus of two recent peer-reviewed publications. One, in Thrombosis Journal, describes a 6-year-old girl with TA-TMA following stem cell transplantation for severe aplastic anemia who had failed treatment with defibrotide. Treated with narsoplimab under compassionate use, her TMA resolved. A recurrence of TMA triggered by parvovirus again was successfully treated with narsoplimab. A second manuscript authored by investigators at Weill Cornell Medicine is in press at Clinical and Experimental Immunology. The manuscript reports a high correlation between elevation of a biomarker of endothelial injury and TA-TMA. Using plasma samples from patients in the narsoplimab pivotal trial, the study also showed that, in those trial patients classified as “responders,” narsoplimab restored biomarker levels to those of stem-cell transplant patients without TMA while biomarker levels in narsoplimab “non-responders” remained high. The 49th annual meeting of the European Society for Blood and Marrow Transplantation (EBMT), which recently concluded in Paris, featured three presentations regarding narsoplimab in TA-TMA. The first described the design of our ongoing multi-center Phase 2 study evaluating the efficacy and safety of narsoplimab in high-risk pediatric patients with TA-TMA. The second was a case report of a patient treated with narsoplimab under our compassionate use program. The patient had worsening TA-TMA following withdrawal of calcineurin inhibitors, which resolved following narsoplimab treatment. The third was a report of five cases of high-risk pediatric TA-TMA. Three of five failed treatment with the C5 inhibitor eculizumab. Two of these three patients also failed treatment with defibrotide and were then treated with narsoplimab, provided under compassionate use. Following narsoplimab treatment, TA-TMA resolved. At the annual meeting of the American Society of Pediatric Hematology/Oncology, being held this week in Fort Worth, Texas, clinical investigators will present the course of a 3-year-old girl with relapsed high-risk neuroblastoma who developed refractory TA-TMA following a second stem cell transplant. After failing treatment with each of eculizumab and defibrotide, the patient’s TA-TMA resolved following treatment with narsoplimab. A case report on narsoplimab treatment of a patient with recurrent IgA nephropathy was featured at the recent annual meeting of the Korean Society of Nephrology. Treatment with narsoplimab stabilized both proteinuria and estimated glomerular filtration rate (“eGFR”). Kidney biopsy results confirmed the beneficial effects of narsoplimab in this patient. This was the first report of complement inhibitor treatment of a patient with recurrent IgA nephropathy. A manuscript authored by a group of international experts detailing the role of the lectin pathway in the pathophysiology of IgA nephropathy, including glomerular injury, thrombotic microangiopathy, and tubulointerstitial fibrosis, was recently accepted for publication in Kidney International. Recent developments regarding OMS1029, our long-acting, next-generation MASP-2 inhibitor, include: In April 2023, we announced results from a pre-specified interim analysis of our ongoing Phase 1b clinical trial of OMS906 in treatment-naïve adults with PNH. Updated data from this open label trial continue to show statistically significant and clinically meaningful improvements in all measured markers of hemolysis, including hemoglobin (“Hgb”) and lactate dehydrogenase (“LDH”). Despite all patients having received only the lowest of doses planned for the trial, data compare very favorably to those publicly available for marketed and in-development alternative pathway inhibitors. Enrollment is ongoing. Updated data are provided below (“n”, or the number of patients, at each time point ranges from 3 to 9 depending on each patient’s respective date of enrollment): Hemoglobin Mean baseline Hgb is 6.78 g/dL (normal: 12.0–15.6 g/dL for women, 13.5–17.2 g/dL for men) Mean absolute increase in Hgb is 3.4 g/dL at Day 29, 6.3 g/dL at Day 85, and 9.7 g/dL at Day 113 After only 1 dose (by Day 29), Hgb increased ≥ 2 g/dL in 67% of patients After only 2 doses (by Day 57), Hgb increased ≥ 2g/dL in 100% and by ≥ 4 g/dL in 80% of patients By Day 85, 67% of patients had Hgb ≥ 12 g/dL; by Day 113, 100% of patients had Hgb ≥ 15.7 g/dL Mean reduction in absolute reticulocyte counts from baseline were ≥ 70,000/µL (range 70,000 – 136,000/µL) at all time points No patients have had a clinical breakthrough or thrombotic event, and none have required a transfusion while receiving OMS906 LDH Mean baseline LDH is 1931 U/L (~8-fold higher than the upper limit of normal) Mean reduction in LDH is 83% at Day 15, 80% at Day 29, and 87% at Day 113 At Day 113, LDH for all patients is normal or < 1.5 times normal Both Hgb and LDH improvements are statistically significant at all time points in the trial; OMS906 has been well tolerated and there have been no safety signals of concern. Based on PK/PD data from a successful Phase 1 single-ascending-dose study of OMS906 in healthy subjects and the interim data from our Phase 1b clinical trial in treatment-naïve PNH patients, we are planning a dosing frequency of once quarterly, either intravenously or subcutaneously. We are also enrolling a Phase 1b clinical trial evaluating OMS906 in PNH patients who have had an unsatisfactory response to the C5 inhibitor ravulizumab. This study has a “switch-over” design and enrolls PNH patients receiving ravulizumab, adds OMS906 to provide combination therapy with ravulizumab for 24 weeks, and then provides OMS906 monotherapy in patients who demonstrate a hemoglobin response with combination therapy. Patient treatment with OMS906 is underway. A Phase 1b clinical program evaluating OMS906 in patients with complement 3 glomerulopathy (“C3G”) is also ongoing. Additional updates on data from our three OMS906 trials in progress are planned for the second half of this year. The Annual Meeting of the European Hematology Association to be held this June in Frankfurt, Germany, will feature a presentation on the study design of our two ongoing clinical trials of OMS906 in patients with PNH, along with results from our single-ascending-dose Phase 1 study evaluating both intravenous and subcutaneous administration of OMS906 in healthy subjects. Recent developments regarding OMS527, our phosphodiesterase 7 (“PDE7”) inhibitor program focused on addiction and movement disorders, include: In April 2023, we were awarded a grant from the National Institute on Drug Abuse, part of the National Institutes of Health to develop our lead orally administered PDE7 inhibitor compound for the treatment of cocaine use disorder (“CUD”). The grant – $6.69 million over three years – is intended to support a preclinical cocaine interaction study and a randomized, placebo-controlled, inpatient clinical study evaluating the safety and effectiveness of OMS527 in patients with CUD. A Phase 1 clinical trial of the study drug in healthy subjects was successfully completed. OMS527 is also being evaluated by investigators at Emory University as a potential treatment for levodopa-induced dyskinesias (“LID”), which are crippling, involuntary movements reportedly affecting 50 percent or more of levodopa-treated patients with Parkinson’s disease. LID is caused by prolonged treatment with levodopa, the most prescribed treatment for the over 10 million patients with Parkinson’s disease worldwide. Data on OMS527 in LID will be publicly disclosed following the filing of appropriate patent applications. Financial Results Net loss for the first quarter of 2023 was $33.7 million, or $0.54 per share. This compares to a net loss in the first quarter of 2022 of $33.0 million, or $0.53 per share. Net loss from continuing operations for the first quarter of 2023 was $39.7 million compared to $39.5 million in the first quarter of 2022, and $0.63 per share for both periods. Cash burn for the first quarter of 2023 was $23.6 million, exclusive of the $200 million milestone payment from Rayner, compared to $26.0 million in the fourth quarter of 2022. During the first quarter of 2023, we earned royalties of $9.2 million on $30.7 million of Rayner’s U.S. net sales of OMIDRIA. This compares to earned royalties of $13.8 million during the first quarter of the prior year on U.S. net sales of $27.7 million. These royalties were recorded as a reduction of the OMIDRIA contract royalty asset. Omeros received a $200 million milestone payment from Rayner in the first quarter of 2023 in connection with the Asset Purchase Agreement under which Rayner purchased our OMIDRIA business. The achievement of the milestone event in late December 2022 triggered a reduction of our U.S. base royalty rate from 50 percent to 30 percent. Total costs and expenses for the first quarter of 2023 were $35.7 million compared to $35.0 million for the first quarter of 2022. Interest expense during the first quarter of 2023 was $7.9 million compared to $4.9 million during the first quarter of 2022. The increase was due to interest on our OMIDRIA contract royalty obligation associated with the sale of a portion of our OMIDRIA royalty receivables, which we entered into during the third quarter of 2022. During the first quarter of 2023, we earned $4.0 million in interest and other income compared to $0.5 million in the prior year quarter. The increase was due to higher average balances available to invest and higher market interest rates in the current year quarter. Net income from discontinued operations, net of tax was $6.0 million, or $0.09 per share, in the first quarter of 2023 compared to $6.5 million, or $0.10 per share, in the first quarter of 2022. As of March 31, 2023, we had $371.4 million of cash and short-term investments, all of which are held in our name, available for operations and debt service. In addition, we had $10.0 million in accounts receivable. Our cash provided by operations during the first quarter of 2023 was $174.5 million and included the $200.0 million collection of the Rayner milestone payment. Conference Call Details To access the live conference call via phone, please dial (800) 715-9871 from the United States and Canada or (646) 307-1963 internationally and ask to be placed into the Omeros earnings call. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (800) 770-2030 from the United States or Canada or (609) 800-9909 internationally. The replay access code is 8266699. For online access to the live or subsequently archived webcast of the conference call, go to Omeros’ website at . About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, cancers, and addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, COVID-19, and atypical hemolytic uremic syndrome. Omeros’ long-acting MASP-2 inhibitor OMS1029 is currently in a Phase 1 clinical trial. OMS906, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is advancing across multiple clinical programs for alternative pathway-related diseases, including paroxysmal nocturnal hemoglobinuria (PNH) and complement 3 (C3) glomerulopathy. For more information about Omeros and its programs, visit . Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the “safe harbor” created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “likely,” “look forward to,” “may,” “objective,” “plan,” “potential,” “predict,” “project,” “should,” “slate,” “target,” “will,” “would” and similar expressions and variations thereof. Forward-looking statements, including statements regarding prospects for obtaining FDA approval of narsoplimab in TA-TMA and anticipated next steps in relation to the biologics license application for narsoplimab, expectations regarding the initiation or continuation of clinical trials evaluating Omeros’ drug candidates and the anticipated availability of data therefrom, and expectations regarding growth in royalty-generating sales of OMIDRIA, are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. Omeros’ actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, unanticipated or unexpected outcomes of regulatory processes in relevant jurisdictions, unproven preclinical and clinical development activities, financial condition and results of operations, regulatory processes and oversight, challenges associated with manufacture or supply of our investigational or clinical products, changes in reimbursement and payment policies by government and commercial payers or the application of such policies, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading “Risk Factors” in the company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 13, 2023. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. OMEROS CORPORATION UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share data) Three Months Ended March 31, 2023 2022 Costs and expenses: Research and development $ 24,610 $ 24,087 Selling, general and administrative 11,103 10,959 Total costs and expenses 35,713 35,046 Loss from continuing operations (35,713 ) (35,046 ) Interest expense (7,933 ) (4,941 ) Interest and other income 3,963 493 Net loss from continuing operations (39,683 ) (39,494 ) Net income from discontinued operations, net of tax 5,982 6,483 Net loss $ (33,701 ) $ (33,011 ) Basic and diluted net income (loss) per share Net loss from continuing operations $ (0.63 ) $ (0.63 ) Net income from discontinued operations 0.09 0.10 Net loss $ (0.54 ) $ (0.53 ) Weighted-average shares used to compute basic and diluted net income (loss) per share 62,828,765 62,724,775 OMEROS CORPORATION UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEET (In thousands) March 31, December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $ 3,829 $ 11,009 Short-term investments 367,527 183,909 OMIDRIA contract royalty asset, short-term 28,940 28,797 Receivables 10,033 213,221 Prepaid expense and other assets 6,708 6,300 Total current assets 417,037 443,236 OMIDRIA contract royalty asset 119,681 123,425 Right of use assets 21,025 21,762 Property and equipment, net 1,335 1,492 Restricted investments 1,054 1,054 Total assets $ 560,132 $ 590,969 Liabilities and shareholders’ equity Current liabilities: Accounts payable $ 6,446 $ 5,989 Accrued expenses 30,821 30,551 Current portion of unsecured convertible senior notes, net 94,554 94,381 Current portion of OMIDRIA royalty obligation 2,943 1,152 Current portion of lease liabilities 4,427 4,310 Total current liabilities 139,191 136,383 Unsecured convertible senior notes, net 221,209 220,906 OMIDRIA royalty obligation 123,057 125,126 Lease liabilities, non-current 21,287 22,426 Other accrued liabilities, non-current 452 444 Shareholders’ equity: Common stock and additional paid-in capital 724,354 721,401 Accumulated deficit (669,418 ) (635,717 ) Total shareholders’ equity 54,936 85,684 Total liabilities and shareholders’ equity $ 560,132 $ 590,969