Omeros Corporation Provides Update of Ongoing Zaltenibart Phase 3 PNH Clinical Trial Program
Omeros (OMER) has initiated site activation for its Phase 3 program evaluating zaltenibart in paroxysmal nocturnal hemoglobinuria (PNH). The program spans 120 clinical sites across 30 countries, with data for BLA submission expected in Q4 2026.
Zaltenibart, administered intravenously once every eight weeks, demonstrates advantages over current treatments that require more frequent dosing. The Phase 3 program includes two trials comparing zaltenibart to C5 inhibitors eculizumab and ravulizumab, targeting both treatment-naive patients and those with inadequate response to existing therapies.
Phase 2 data showed zaltenibart effectively inhibits both intravascular and extravascular hemolysis, achieving normal hemoglobin levels with no significant safety concerns. The global PNH market is valued at $3.8 billion in 2023, with projections reaching $11.7 billion by 2034.
Omeros (OMER) ha avviato l'attivazione dei siti per il suo programma di Fase 3 che valuta zaltenibart nella parossistica emoglobinuria notturna (PNH). Il programma si estende su 120 siti clinici in 30 paesi, con dati per la presentazione della BLA attesi nel quarto trimestre del 2026.
Zaltenibart, somministrato per via endovenosa ogni otto settimane, dimostra vantaggi rispetto ai trattamenti attuali che richiedono dosaggi più frequenti. Il programma di Fase 3 include due studi che confrontano zaltenibart con gli inibitori C5 eculizumab e ravulizumab, mirando sia ai pazienti naïve al trattamento che a quelli con risposta inadeguata alle terapie esistenti.
I dati della Fase 2 hanno mostrato che zaltenibart inibisce efficacemente sia l'emolisi intravascolare che quella extravascolare, raggiungendo livelli normali di emoglobina senza preoccupazioni significative per la sicurezza. Il mercato globale della PNH è valutato a 3,8 miliardi di dollari nel 2023, con proiezioni che raggiungono 11,7 miliardi di dollari entro il 2034.
Omeros (OMER) ha iniciado la activación de sitios para su programa de Fase 3 que evalúa zaltenibart en la hemoglobinuria nocturna paroxística (PNH). El programa abarca 120 sitios clínicos en 30 países, con datos para la presentación de la BLA esperados en el cuarto trimestre de 2026.
Zaltenibart, administrado por vía intravenosa cada ocho semanas, muestra ventajas sobre los tratamientos actuales que requieren dosis más frecuentes. El programa de Fase 3 incluye dos ensayos que comparan zaltenibart con los inhibidores de C5 eculizumab y ravulizumab, dirigidos tanto a pacientes naïve al tratamiento como a aquellos con respuesta inadecuada a las terapias existentes.
Los datos de Fase 2 mostraron que zaltenibart inhibe eficazmente tanto la hemólisis intravascular como la extravascular, logrando niveles normales de hemoglobina sin preocupaciones significativas de seguridad. El mercado global de PNH está valorado en 3.8 mil millones de dólares en 2023, con proyecciones que alcanzan los 11.7 mil millones de dólares para 2034.
Omeros (OMER)는 zaltenibart의 야간 발작성 헤모글로빈뇨(PNH) 치료를 평가하는 3상 프로그램의 사이트 활성화를 시작했습니다. 이 프로그램은 30개국에 걸쳐 120개의 임상 사이트로 구성되어 있으며, BLA 제출을 위한 데이터는 2026년 4분기에 예상됩니다.
Zaltenibart는 8주마다 정맥 주사로 투여되며, 더 자주 투여해야 하는 현재 치료법에 비해 장점을 보여줍니다. 3상 프로그램은 zaltenibart와 C5 억제제인 eculizumab 및 ravulizumab을 비교하는 두 가지 시험을 포함하며, 치료 경험이 없는 환자와 기존 치료에 대한 반응이 불충분한 환자를 대상으로 합니다.
2상 데이터는 zaltenibart가 혈관 내 및 혈관 외 용혈을 효과적으로 억제하며, 안전성에 대한 중요한 우려 없이 정상 헤모글로빈 수치를 달성함을 보여주었습니다. 2023년 세계 PNH 시장은 38억 달러로 평가되며, 2034년까지 117억 달러에 이를 것으로 예상됩니다.
Omeros (OMER) a lancé l'activation des sites pour son programme de Phase 3 évaluant zaltenibart dans l'hémoglobinurie nocturne paroxystique (PNH). Le programme s'étend sur 120 sites cliniques dans 30 pays, avec des données pour la soumission de la BLA attendues au quatrième trimestre 2026.
Zaltenibart, administré par voie intraveineuse toutes les huit semaines, présente des avantages par rapport aux traitements actuels nécessitant des doses plus fréquentes. Le programme de Phase 3 comprend deux essais comparant zaltenibart aux inhibiteurs de C5 eculizumab et ravulizumab, ciblant à la fois les patients naïfs de traitement et ceux ayant une réponse insuffisante aux thérapies existantes.
Les données de Phase 2 ont montré que zaltenibart inhibe efficacement l'hémolyse intravasculaire et extravasculaire, atteignant des niveaux normaux d'hémoglobine sans préoccupations significatives en matière de sécurité. Le marché mondial de la PNH est évalué à 3,8 milliards de dollars en 2023, avec des projections atteignant 11,7 milliards de dollars d'ici 2034.
Omeros (OMER) hat die Aktivierung von Standorten für sein Phase-3-Programm zur Bewertung von zaltenibart bei paroxysmaler nächtlicher Hämoglobinurie (PNH) eingeleitet. Das Programm erstreckt sich über 120 klinische Standorte in 30 Ländern, wobei die Daten für die BLA-Einreichung im vierten Quartal 2026 erwartet werden.
Zaltenibart, das alle acht Wochen intravenös verabreicht wird, zeigt Vorteile gegenüber aktuellen Behandlungen, die häufigere Dosierungen erfordern. Das Phase-3-Programm umfasst zwei Studien, die zaltenibart mit den C5-Inhibitoren Eculizumab und Ravulizumab vergleichen und sowohl behandlungsnaive Patienten als auch solche mit unzureichender Reaktion auf bestehende Therapien ansprechen.
Die Phase-2-Daten zeigten, dass zaltenibart sowohl intravasale als auch extravasale Hämolyse effektiv hemmt und normale Hämoglobinwerte ohne signifikante Sicherheitsbedenken erreicht. Der globale PNH-Markt wird im Jahr 2023 auf 3,8 Milliarden US-Dollar geschätzt, mit Prognosen, die bis 2034 11,7 Milliarden US-Dollar erreichen.
- Less frequent dosing schedule (once every 8 weeks) compared to current treatments
- Phase 2 data showed effective treatment with normal hemoglobin levels
- No safety concerns observed in clinical trials
- Large market opportunity ($3.8B in 2023, projected $11.7B by 2034)
- All drug product for Phase 3 trials already manufactured
- BLA submission timeline extends to Q4 2026
- Faces competition from established treatments in market
- Requires intravenous administration versus oral options
Insights
Omeros' Phase 3 program for zaltenibart in PNH is progressing as planned, with site activation underway across 120 clinical sites in 30 countries. The pivotal data needed for BLA submission remains on schedule for Q4 2026, representing a significant but distant milestone.
Zaltenibart's differentiating features include its infrequent dosing schedule (IV once every 8 weeks versus daily oral or twice-weekly subcutaneous for competitors) and dual mechanism inhibiting both intravascular and extravascular hemolysis. The Phase 2 data demonstrated achievement of gender-normal hemoglobin levels without safety concerns.
The comparative trial design, approved by both FDA and European regulators, positions zaltenibart for potential superiority claims against standard-of-care C5 inhibitors (eculizumab and ravulizumab). This head-to-head comparison strategy reflects confidence in the drug's profile and could support premium pricing.
The inclusion of patient-reported outcomes based on German payer recommendations demonstrates commercial foresight in addressing European reimbursement requirements. The company has completed manufacturing all clinical trial material, removing one execution risk.
With the global PNH market valued at
-- Phase 3 Data Needed for BLA Submission Expected in the Fourth Quarter 2026 --
A total of 120 clinical investigative sites across 30 countries have been chosen for clinical trial participation in the zaltenibart Phase 3 program in PNH, a good number of which have already identified pools of PNH patients ready to participate in the zaltenibart trials, and Omeros continues collaborating with sites to identify additional eligible and already available PNH patients. Data needed for submission of the biologics licensing application (BLA) and global approval dossiers for zaltenibart in PNH remain on track for the fourth quarter of 2026.
The Phase 3 clinical trials will evaluate intravenous zaltenibart dosed once every eight weeks. Currently marketed upstream complement inhibitors require dosing orally twice daily, orally three times daily in conjunction with C5 inhibitor treatment, or subcutaneous infusions twice weekly. Zaltenibart’s conveniently infrequent dosing together with its ability to inhibit both intravascular and extravascular hemolysis provide a meaningful differentiation from the other PNH therapies. Phase 2 data have previously been presented at the American Society of Hematology and European Hematology Association Annual Meetings and demonstrate that zaltenibart effectively inhibits both intravascular and extravascular hemolysis while achieving gender-normal hemoglobin levels in both men and women. No safety signal of concern has been observed with zaltenibart.
The zaltenibart Phase 3 program includes two clinical trials, one in patients who are not receiving complement-inhibitor treatment at the time of study entry and another in patients who have an inadequate response to treatment with either ravulizumab or eculizumab. Both clinical trials 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. All zaltenibart drug product required for the Phase 3 program has been manufactured, and comparator C5 inhibitors have been sourced. These study designs provide head-to-head comparisons and should provide data to demonstrate superiority of zaltenibart over the C5 inhibitors in these patient populations. These comparison data may form the basis for comparative superiority claims for promotion, enhanced market access, and pricing reflective of zaltenibart’s advantages. In further preparation for potential commercialization of zaltenibart, recommendations regarding patient-reported-outcome (PRO) measures were sought and received from the German Federal Joint Committee, the decision-making body in the German healthcare system that specifies which medical treatments are reimbursed by the statutory health insurance funds and specialized in PRO measures. Recommended PRO measures were incorporated into the zaltenibart Phase 3 design and are expected to be helpful in securing appropriate pricing.
“All of us at Omeros are pleased that the Phase 3 clinical program for zaltenibart is well underway,” said Gregory A. Demopulos, M.D., Omeros’ Chairman and CEO. “The zaltenibart Phase 2 data have demonstrated important differentiators from currently marketed agents, and we expect those same advantages to be evidenced in the Phase 3 trials, which are similar in design to our Phase 2 trials. We continue to refine and optimize the potential commercial impact of the zaltenibart advantages, and we look forward to our Phase 3 readout late next year and to bringing a better treatment option to PNH patients and their physicians. Based on the data to date, zaltenibart is a premier alternative pathway inhibitor, and we plan to continue expanding alternative pathway indications for our drug.”
The global market size for PNH is reported at
About Zaltenibart (OMS906)
Zaltenibart is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key and most proximal activator of the complement system’s alternative pathway. The complement system is a critical part of innate immunity and plays a central role in host homeostasis and defense against pathogens. Responsible for the conversion of pro-complement factor D to complement factor D, MASP-3 is believed to be the premier target in the alternative pathway – it has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Also, unlike other components of the alternative pathway, MASP-3 is believed not to be an acute phase reactant, which could provide a significant advantage to MASP-3 inhibitors, like zaltenibart, over other alternative pathway inhibitors. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including paroxysmal nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, geographic atrophy or “dry” macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders.
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 advancing toward Phase 3 clinical trials for paroxysmal nocturnal hemoglobinuria and 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 enrollment of patients in clinical trials, statements regarding the availability and timing of data readouts from clinical trials, and statements regarding the anticipated therapeutic benefit or commercial prospects of Omeros’ drug candidates 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, unfavorable results of clinical development activities, unavailability of capital resources to support planned development activities, regulatory processes and oversight, challenges associated with conducting clinical trials, 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 April 1, 2024, and in our subsequently filed Quarterly Reports on Form 10-Q. 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.
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Jennifer Cook Williams
Cook Williams Communications, Inc.
Investor and Media Relations
IR@omeros.com
Source: Omeros Corporation
FAQ
When will Omeros (OMER) complete the Phase 3 trials for zaltenibart in PNH treatment?
How is zaltenibart's dosing schedule different from current PNH treatments?
What is the market potential for OMER's zaltenibart in PNH treatment?
How many clinical sites are participating in OMER's zaltenibart Phase 3 trials?
What advantages does zaltenibart show over existing PNH treatments in Phase 2?
