Hemoglobinuria, Paroxysmal

The pairing of two Bristol Myers Squibb immunotherapies is now permitted for the first-line treatment of two types of gastrointestinal cancers, after regulatory decisions handed out this past week that convert the accelerated approval status of the drug combination to full FDA approval in both indications. The drugs, Opdivo and Yervoy, belong to the class of medicines called checkpoint inhibitors. On April 8, the FDA approved use of this drug combination for adults and children age 12 and older whose colorectal cancer has metastasized or cannot be removed by surgery. The cancer must also be microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), references to biomarkers that indicate the stability of DNA in a tumor. The regulatory submission was based on the results of an open-label Phase 3 test that compared the Opdivo/Yervoy combination to either Opdivo alone or chemotherapy. Results showed the immunotherapy combination led to a 38% reduction in the risk of disease progression or death compared to Opdivo monotherapy, and by 79% compared to chemo, both in the first-line setting. The study is ongoing to assess secondary measures, including overall survival. BMS said it will continue working with study investigators to present these data and longer-term follow-up results in the future. Sponsored Post Changes in Nurse Staffing Answer Clinician Demands The ongoing nursing shortage facilitates high turnover rates since nurses know they won’t have difficulties finding new jobs. In order to retain and attract staff, it’s in a facility’s best interest to understand what nurses want. By Tomasz Rezik On April 11, the FDA approved the combination of Opdivo and Yervoy for treating advanced cases of hepatocellular carcinoma. In the pivotal test in this indication, the drug combination led to median overall survival of 23.7 months. The comparator arm, in which patients received standard of care drugs sorafenib or levantinib, median overall survival was 20.6 months. At three years, the drug combination showed an overall survival rate of 38% versus 24% in the comparator arm. FDA approval of Opdivo plus Yervoy in hepatocellular carcinoma follows the European Commission’s early March approval of the drug combination in this indication. There’s plenty of news on the regulatory front. Here’s a recap of other recent highlights: New and Expanded Regulatory Approvals —Argenx drug Vyvgart Hytrulo received an additional approval for dosing of the product via a prefilled syringe. The drug, supplied in vials, previously received FDA approvals for the autoimmune conditions chronic inflammatory demyelinating polyneuropathy and generalized myasthenia gravis. Analysts say prefilled syringes bring patients a more convenient dosing option while giving Argenx the opportunity to get more market share by penetrating into earlier lines of therapy. Sponsored Post Understanding EGPA: The Role of Eosinophils and Advancements in Treatment Options FASENRA® (benralizumab) injection, for subcutaneous use, 30 mg is indicated for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). FASENRA provides a treatment option for HCPs to consider when managing this challenging disease. By FASENRA, Sponsored Content —Amgen’s Uplizna expanded its label to include the treatment of immunoglobulin G4-related disease (IgG4-RD), an immune-mediated inflammatory disorder that can affect multiple organs. The regulatory decision makes the intravenously infused antibody the first FDA-approved treatment for this rare and chronic disease. Uplizna was first approved in 2020 as a treatment for neuromyelitis optica spectrum disorder. —Bayer’s Viktrakvi now has full FDA approval for the treatment of NTRK gene fusion-positive solid tumors in adults and children without a known acquired resistance mutation. The drug received accelerated approval in this indication in 2018. —AstraZeneca and Daiichi Sankyo welcomed European Union approval of Datroway as a treatment for metastatic breast cancer that is HR positive and HER2 negative. Datroway received FDA approval in the same indication in January. The drug is an antibody drug conjugate designed to target the cancer protein TROP2. —Novartis drug atrasentan, brand name Vanrafia, was awarded accelerated approval as a new treatment for the kidney disorder immunoglobulin A nephropathy (IgAN). The drug is an oral small molecule designed to block the endothelin A receptor. It’s one of two IgAN nephropathy drugs that came via the $3.2 billion acquisition of Chinook Therapeutics in 2023. Novartis’s internal IgAN research yielded the complement inhibitor Fabhalta, which received accelerated approval in this indication last summer. —Speaking of Fabhalta, that Novartis drug expanded its label to include C3 glomerulopathy, making the twice-daily pill the first approved treatment for this rare kidney disorder. Fabhalta is a small molecule designed to block the complement protein C3. It was first approved in 2023 as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria. —Sanofi received FDA approval for fitusiran, brand name Qfitlia, as a treatment for both hemophilia A and B. The drug leverages RNA interference to lower levels of AT, a protein that inhibits blood clotting. The approval covers the treatment of adults and children age 12 and older with or without inhibitors, which are antibodies that can develop in hemophilia patients, making it more difficult to control bleeding. By contrast, new Pfizer drug Hympavzi is approved only for hemophilia A and B patients without inhibitors while Sanofi’s Alhemo is approved only for hemophilia A and B patients with inhibitors. —Blockbuster AstraZeneca cancer immunotherapy Imfinzi landed European Union approval for the treatment of limited-stage small cell lung cancer. This new approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. The FDA approved Imfinzi for this indication last December. —Imfinzi also added perioperative treatment of muscle invasive bladder cancer to its list of FDA-approved indications. The newest FDA nod covers use of the drug in combination with chemotherapy before surgery to remove the bladder, followed by Imfinzi as an adjuvant monotherapy. In the pivotal study supporting this new indication, results showed a 32% reduction in the risk of cancer recurrence and a 25% reduction in the risk of death compared to neoadjuvant chemotherapy alone. —The European Commission approved Pfizer vaccine Abrysvo for protection against respiratory syncytial virus (RSV). The regulatory decision expands the vaccine’s approval to adults ages 18 to 59. European regulators initially approved the vaccine in 2023 for preventing lower respiratory tract disease in adults age 60 and older. Abrysvo won FDA approval the same year. —The FDA expanded approval of Novartis’s Pluvicto to include use as an earlier line of treatment — after anti-androgen drugs and before chemotherapy — for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Pluvicto was initially approved in 2022 for PSMA-positive mCRPC that has already been treated with an anti-androgen drug and chemotherapy. Novartis said this expansion is important because about half of patients do not live long enough to receive a second-line treatment. —Exelixis cancer drug Cabometyx expanded its FDA-approved uses to advanced cases of pancreatic neuroendocrine tumors (pNET) and extra-pancreatic neuroendocrine tumors (epNET). The approval covers adults and children age 12 and older. Cabometyx is a small molecule designed to block enzymes that support cancer growth. The drug was first approved in 2016 for advanced renal cell carcinoma. —GSK landed FDA approval for Blujepa, an oral antibiotic developed to treat uncomplicated urinary tract infections. While drugs are already available for these infections, they can become resistant to treatment. Blujepa is designed to block two enzyme targets key to bacterial DNA replication, an approach intended to lower the potential for drug resistance. —The European Commission approved Capvaxive, a pneumococcal vaccine developed by Merck to protect against the 21 Streptococcus pneumoniae serotypes that lead to the majority of cases of invasive pneumococcal disease including eight that are not covered by any other available pneumococcal vaccine. The European regulatory decision covers adults age 18 and older. Capvaxive received FDA approval last July. —Alynlam Pharmaceuticals drug Amvuttra received FDA approval for treating cardiomyopathy caused by transthyretin amyloidosis (ATTR). The RNA interference drug was first approved in 2022 for the treatment of polyneuropathy caused by ATTR. In the cardiomyopathy indication, it will compete with Pfizer’s Vyndaqel and Vyndamax, which are established as the standard of care, as well as Attruby, a BridgeBio Pharma drug that received FDA approval last fall. —Bristol Myers Squibb’s Breyanzi received European Commission approval for relapsed or refractory follicular lymphoma. The CAR T-therapy received accelerated FDA approval in this indication last May. —Geron Corporation’s Rytelo received European Commission approval for the treatment of transfusion-dependent anemia caused by lower-risk myelodysplastic syndrome, a progressive type of blood cancer. The intravenously infused drug is the first telomerase inhibitor to reach the market. Rytelo landed FDA approval last June. —Neffy, an epinephrine nasal spray that ARS Pharmaceuticals developed to treat allergic reactions to insect bites, medications, and food, expanded its FDA approval to include patients weighing 15 to 30 kilograms (about 33 to 60 pounds). The product’s initial approval last year covered its use in patients weighing 30 kg or more. —Roche subsidiary Genentech received FDA approval for tenecteplase, brand name TNKase, as a treatment for acute ischemic stroke in adults. Genentech said TNKase is the first stroke medicine approved by the FDA in nearly 30 years. It’s the second approved indication for the clot-busting drug. The FDA approved it in 2000 for treating acute myocardial infarction. —A Neurotech Pharmaceuticals cell and gene therapy administered via a surgical implant received FDA approval for the treatment of macular telangectasia type 2. The therapy, brand name Encelto, is the first approved treatment for this rare vision-loss disorder. Regulatory Setbacks —The FDA missed the April 1 regulatory decision target date for Novavax’s Covid-19 vaccine. Politico reported the holdup was due to the intervention of Sara Brenner, the FDA’s principal deputy commissioner, who asked for more data. Novavax said it has responded to all of the FDA’s information requests and believes the application is ready for approval based on the submitted Phase 3 data. Novavax’s protein-based vaccine initially received FDA emergency use authorization in 2022, a status that it retained under amended authorizations in 2023 and last year, both times for new formulas that covered the prevalent variants at the time. Full FDA approval would put the vaccine on par with the approved messenger RNA vaccines marketed by Pfizer and Moderna. Novavax’s Covid-19 vaccine will be co-commercialized with Sanofi under a partnership announced last year. —Roche paused European clinical tests of the Duchenne muscular dystrophy gene therapy Elevidys, the pharmaceutical giant said in a letter to the World Duchenne Organization. The move follows Sarepta Therapeutics’ recent report of a liver failure-associated patient death. Roche said the European Medicines Agency asked for a clinical hold on four studies until the inquiry into the cause of death is complete. Under a 2019 deal with Sarepta, Roche holds rights to Elevidys outside of the U.S. —For the second time, the FDA has rejected Aldeyra Therapeutics drug reproxalap as a treatment for dry eye disease. The agency turned down an application for the drug in 2023. According to Aldeyra, the FDA’s latest complete response letter said the new drug application failed to demonstrate efficacy. The regulator asked the company to run at least one more clinical trial. Among the concerns raised by the FDA is differences in baseline scores, which may have affected interpretation of trial results. Aldeyra said results from two ongoing studies are expected later in the current quarter, potentially paving the way for a mid-year resubmission of the application. —The European Medicines Agency refused marketing authorization for Eli Lilly Alzheimer’s disease drug Kisunla. Safety was the main reason cited, particularly the brain inflammation and bleeding that is a known complication risk associated with all drugs in the same class of Alzheimer’s medicines. The agency said the drug’s benefits were not enough to outweigh the potentially fatal safety risks. The FDA approved Kisunla last July. —In other Alzheimer’s drug news, Eisai again failed to persuade Australia’s drugs regulator to register Leqembi. The pharma company had asked the Therapeutics Goods Administration (TGA) to reconsider its October 2024 decision to not register the drug in Australia. Eisai’s request for reconsideration covered a narrower indication, but Eisai said the TGA did not agree that safety has been established for these patients.

– RLYB212 Phase 2 PK Results Did Not Achieve Target Concentrations, Including Minimum Target Concentration Required for Efficacy – – RLYB116 Confirmatory PK/PD Study to Initiate in 2Q 2025, with Data in 2H 2025 – NEW HAVEN, CT, USA I April 08, 2025 I Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, today announced the discontinuation of the RLYB212 program for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT). The Company’s decision to discontinue RLYB212 development was based on pharmacokinetic (PK) data from the Phase 2 clinical trial demonstrating the inability of the RLYB212 dose regimen to achieve predicted target concentrations, as well as the minimum target concentration required for efficacy. Rallybio remains focused on advancing RLYB116, a once-weekly low volume C5 inhibitor for the treatment of complement-driven diseases, as well as its emerging preclinical programs. “We are disappointed by the PK results of the RLYB212 Phase 2 trial,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “Given that the results significantly deviated from the predicted range and the absence of empiric data to further inform dose adjustment, the risk/benefit no longer supports continued dosing, and we will discontinue RLYB212 development. We are grateful to the participants, investigators, and study staff for their partnership and dedication to this program.” Dr. Uden continued, “Rallybio remains steadfast in our mission to develop transformative therapies. We are focused on creating shareholder value by advancing our portfolio of potentially best-in-class assets for patients with rare diseases, which includes RLYB116 and REV102, an ENPP1 inhibitor for patients with hypophosphatasia, as well as RLYB332, a long-acting matriptase-2 antibody for diseases of iron overload.” RLYB212 Phase 2 Trial The single-arm Phase 2 dose confirmation trial was designed to assess the PK and safety of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. Secondary objectives included the assessment of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization. Second trimester PK results from the sentinel participant demonstrated an inability of RLYB212 to achieve predicted target concentrations of 6 ng/mL to 10 ng/mL, as well as the minimum target concentration required for efficacy of 3 ng/mL, with values near or below the assay’s lower limit of quantitation. Dose adjustment is not deemed feasible given that PK levels are meaningfully outside the predicted range and the absence of empiric data to inform an adjustment. It is hypothesized that HPA-1a antigen expression on the placenta may be impacting plasma concentrations of RLYB212. No further enrollment in the trial is planned and all screening of participants has been stopped. The Company will continue safety follow-up of the sentinel participant as specified in the clinical trial protocol. Promising Rare Disease Pipeline to Deliver Data in 2H 2025 RLYB116 Program Rallybio remains on track to initiate dosing in the RLYB116 confirmatory clinical pharmacokinetic/ pharmacodynamic (PK/PD) study in the second quarter of 2025, with data readouts from Cohorts 1 and 2 expected in the third and fourth quarter of 2025, respectively. The study is expected to demonstrate complete and sustained complement inhibition with improved tolerability of RLYB116. RLYB116 is a novel antibody mimetic fusion protein designed to inhibit C5 and to provide a once-weekly, small volume, subcutaneously injected therapy to meet patient demand for a convenient, self-administered at-home solution. RLYB116 has the potential to address significant unmet need for patients across a number of complement mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), antiphospholipid syndrome (APS), and generalized myasthenia gravis (gMG), which together represent a commercial opportunity of more than $6 billion. Importantly, this strategic focus on complement-mediated diseases presents a unique opportunity for Rallybio to utilize its team’s deep domain experience in successfully designing, developing, and delivering complement inhibitors for patients with rare diseases. Preclinical Programs REV102, an ENPP1 inhibitor for the treatment of patients with HPP under development through a joint venture with Recursion Pharmaceuticals, entered investigational new drug application (IND)-enabling studies in the first quarter of 2025 to support the initiation of a Phase 1 study in 2026. Data evaluating REV102 in a preclinical model of later-onset HPP is expected in the second half of 2025. Rallybio’s portfolio also includes RLYB332, a long-acting, monoclonal anti-matriptase-2 antibody that has the potential to be a best-in-class treatment for diseases of iron overload. Preclinical data has demonstrated superior impact on PD parameters relative to comparator molecules, including on serum iron, unsaturated iron binding capacity (UIBC), and transferrin saturation (TSAT). The Company is preparing plans for future development of RLYB332. About Rallybio Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of complement dysregulation, hematology, and metabolic disorders. The Company’s lead program, RLYB116, is a differentiated C5 inhibitor with the potential to treat diseases of complement dysregulation. Rallybio also has two programs in preclinical development, including REV102, an ENPP1 inhibitor for the treatment of patients with hypophosphatasia (HPP), and RLYB332, a long-acting matriptase-2 antibody for the treatment of diseases of iron overload. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn and Twitter . SOURCE: Rallybio

– The Steering Committee brings together distinguished clinical experts in AML from leading National Comprehensive Cancer Network Centers – SEATTLE--(BUSINESS WIRE)--Apr. 10, 2025-- Omeros Corporation (Nasdaq: OMER) today announced the establishment of the Omeros Oncology Clinical Steering Committee to advance Omeros’ OncotoX biologics program focused on acute myeloid leukemia (AML). AML is the most fatal form of leukemia and accounts for approximately 80 percent of acute leukemias in adults and one-third of all cancers affecting the blood/bone marrow, representing a high unmet need. Omeros’ OncotoX program for AML consists of proprietary targeted, engineered molecules (about half the size of an antibody) that deliver a toxic payload within the cancer cells, thereby killing them. Members of the Omeros Oncology Clinical Steering Committee include: Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center (Chair of the Omeros Oncology Clinical Steering Committee) Aref Al-Kali, M.D., Chair of the Acute Myeloid Group and Research Chair of the Acute Leukemia and Myeloid Neoplasms Disease Group at Mayo Clinic Jessica K. Altman, M.D., Professor of Medicine in the Division of Hematology and Oncology and Director of the Leukemia Program of Robert H. Lurie Comprehensive Cancer Center at Northwestern University Jayastu Senapati, M.B.B.S., M.D., D.M., Assistant Professor in the Department of Leukemia at MD Anderson Cancer Center Mithun Shah, M.D., Ph.D., Assistant Professor of Medicine and Oncology, and Consultant in the Division of Hematology at Mayo Clinic Anthony S. Stein, M.D., Co-director of the Gehr Family Center for Leukemia Research within the Hematologic Malignancies and Stem Cell Transplantation Institute at City of Hope Eytan M. Stein, M.D., Chief of the Leukemia Service and Director of the Program for Drug Development in Leukemia in the Division of Hematologic Malignancies at Memorial Sloan Kettering Cancer Center Roland B. Walter, M.D., Ph.D., M.S., Professor in the Translational Science and Therapeutics Division and José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research at the Fred Hutchinson Cancer Center The steering committee will, among other activities, assist Omeros with development of the OncotoX-AML program, clinical trial design and interactions with institutional review boards and will participate in OncotoX-AML clinical trials. “AML is a devastating disease, and these patients have difficulty tolerating the side effects of chemotherapeutic agents and current antibody-drug conjugates, which generally have a narrow therapeutic index,” stated Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center and Chair of the Omeros Oncology Clinical Steering Committee. “The OncotoX platform is designed to kill only dividing cancer cells while sparing normal cells. In animal models using cell lines derived from AML patients, the OncotoX-AML molecule has shown superior efficacy to current standard of care in AML treatment, and its potential utility extends broadly beyond AML to other types of leukemia. Together with my esteemed physician-scientist colleagues on the steering committee, I look forward to advancing the Omeros therapeutic for AML and bringing this novel treatment to patients as quickly as possible.” Across extensive in vivo and ex vivo studies, the OncotoX-AML therapeutic has shown to be highly effective even at very low doses, providing a significant survival benefit over currently approved combination therapy (venetoclax and azacytidine). These studies, conducted with human cell lines in well-established animal models and considered predictive of clinical response, have targeted AML tumors with mutational backgrounds commonly found in nearly 90 percent of AML patients (TP53, NPM1, KMT2a, and FLT3) indicating that the OncotoX-AML therapeutic could be “mutation-agnostic,” a currently large unmet need for oncologists and their patients. In studies using primary AML derived from patients, the OncotoX molecule preferentially and efficiently kills AML blasts (abnormal myeloid cells). OncotoX-AML also targets leukemia stem cells (LSC), which are often refractory to chemotherapy and represent another major challenge in the treatment of AML. The OncotoX-AML therapeutic is designed specifically to kill both AML blasts and LSCs that can lead to relapse. Preliminary in vivo tolerability studies demonstrate that the OncotoX therapeutic is well tolerated at doses substantially greater than one order of magnitude above efficacious doses without causing neutropenia or meaningful changes in blood chemistry values. Omeros is initiating IND-enabling work for its OncotoX-AML therapeutic. Clinical efficacy in AML, considered one of the most aggressive leukemias, is thought to bode well for a therapeutic agent’s broad applicability across leukemias. In addition to OncotoX-AML, Omeros’ portfolio of oncology platforms include signaling and antigen-driven immunomodulators, immune memory-enhancing immunostimulators, and an adoptive T-cell technology that, unlike other cell therapy approaches, does not require cellular engineering. “We are very pleased to be working with all the members of our oncology steering committee,” said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief Executive Officer. “Each is an internationally recognized leader in the fields of leukemia and AML and has been integrally involved in the development of new treatments for their patients. Their willingness to invest time and effort in our OncotoX-AML program is a strong endorsement of our molecule’s potential and, with their help, we look forward to assessing this next-generation therapeutic in AML patients.” In the U.S. alone, AML is diagnosed in over 20,000 patients annually and is responsible each year for more than 11,000 deaths. In 2030, the global AML therapeutic market size is projected to be over $6 billion with the leukemia therapeutic market forecast at $29 billion. About Omeros’ Oncology Programs Omeros’ oncology programs are informed by a deep understanding of both the innate (complement-mediated) and adaptive (CD4 & CD8 T-cells) immune systems to derive highly differentiated, first-in-class therapeutic programs to treat a wide range of cancers. While significant progress has been made within oncology through targeted therapies, advanced biologics and immuno-therapies, toxicity and efficacy shortcomings remain significant. Omeros is focused on moving beyond these approaches with a family of wholly novel molecular and cellular platforms designed to be more effective, targeting both cell-surface and intracellular cancer antigens, with less toxicity. Our oncology franchise involves three distinct platforms – OncotoX, immunomodulators, and immunostimulators in combination with adoptive T-cell therapy. IND-enabling work has been initiated to advance to clinic our OncotoX-AML program comprised of engineered molecules designed to target and kill cancer cells with precision. Our immunomodulator platform is based on our identifying and evidencing the importance of certain molecules that selectively target T cells to regulate and potentiate immune responses. Our adoptive T-cell technology, unlike other cell therapy approaches, does not require cellular engineering and is being developed in concert with our immunostimulator platform to advance beyond the challenges of current vaccine approaches, which induce only transient and ineffective immune responses. By combining tumor antigens with a potent adjuvant, these novel, engineered biologics are designed specifically to target and activate antigen-presenting cells, leading to amplification of cancer-specific T and B cells to eliminate existing tumor cells and, importantly, to generate immune memory against future cancer relapse. Collectively, our portfolio of oncology platforms has the potential to deliver substantially more effective and safer treatments broadly across hematological malignancies and solid tumors. About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing first-in-class small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders, including complement-mediated diseases and cancers, as well as 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. Omeros’ long-acting MASP-2 inhibitor OMS1029 has successfully completed Phase 1 single- and multiple-ascending dose clinical studies. Zaltenibart, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in Phase 3 clinical development for paroxysmal nocturnal hemoglobinuria and is being evaluated in an ongoing Phase 2 clinical trial for complement 3 glomerulopathy. Funded by the National Institute on Drug Abuse, Omeros’ lead phosphodiesterase 7 inhibitor OMS527 is in clinical development for the treatment of cocaine use disorder. Omeros also is advancing a broad portfolio of novel cellular and molecular immuno-oncology programs. For more information about Omeros and its programs, visit www.omeros.com. 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 “aim,” “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 the anticipated safety and therapeutic benefits of Omeros’ drug candidates and plans for future development thereof 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, our financial condition and results of operations, inability to raise capital when required to fund our operations and development plans, unfavorable, unexpected or inconclusive results of our preclinical and clinical development activities, unexpected outcomes of regulatory processes in relevant jurisdictions, including those associated with other Omeros’ development programs, challenges associated with manufacture or supply of our drug candidates or other materials needed to support preclinical and clinical development, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading “Risk Factors” in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 31, 2025. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20250410587782/en/ Jennifer Cook Williams Cook Williams Communications, Inc. Investor and Media Relations IR@omeros.com Source: Omeros Corporation