zaltenibart (OMS906) / Omeros, Novo Nordisk - LARVOL DELTA

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - P2 | N=25 | Active, not recruiting | Sponsor: Omeros Corporation | Recruiting ➔ Active, not recruiting

Enrollment closed • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

OMS906-PNH-002: Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - P1 | N=15 | Completed | Sponsor: Omeros Corporation | Recruiting ➔ Completed | Phase classification: P1b ➔ P1 | N=10 ➔ 15 | Trial completion date: Jun 2024 ➔ Nov 2025 | Trial primary completion date: Oct 2023 ➔ Nov 2025

Enrollment change • Phase classification • Trial completion • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Safety and Efficacy Study of OMS906 in Patients With C3G and ICGN (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Omeros Corporation | Completed ➔ Recruiting

Enrollment open • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology

Safety and Efficacy Study of OMS906 in Patients With C3G and ICGN (clinicaltrials.gov) - P2 | N=20 | Completed | Sponsor: Omeros Corporation | Active, not recruiting ➔ Completed

Trial completion • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology

Omeros Corporation Announces Closing of Asset Purchase and License Agreement with Novo Nordisk for Omeros’ Clinical-Stage MASP-3 Inhibitor Zaltenibart (OMS906) (Businesswire) - "Omeros is eligible to receive upfront and near-term milestone payments up to $340.0 million, of which the upfront amount of $240.0 million was paid in cash at closing. In total, Omeros is eligible to receive up to $2.1 billion including potential development and commercial milestones, plus tiered royalties on net sales....Novo Nordisk is strongly positioned to develop zaltenibart into a differentiated and potentially best-in-class treatment approach for a number of rare blood and kidney disorders, maximizing the opportunity to help people living with these diseases in the future and supporting the company’s leadership ambition in this space."

Commercial • Glomerulonephritis • Nephrology • Paroxysmal Nocturnal Hemoglobinuria

Alternative Pathway Masp-3 Inhibitor OMS906 Effectively and Potently Inhibits Complement-Mediated Hemolysis in Preclinical Models Mechanistically Similar to Paroxysmal Nocturnal Hemoglobinuria (ASH 2023) - "OMS906 inhibited RBC lysis more potently than a C5 inhibitor, providing evidence that MASP-3 inhibition blocks downstream terminal activity and thus intravascular hemolysis. In addition, OMS906 improved survival of Crry-/- RBCs comparably to a CFB inhibitor, indicating that MASP-3 inhibition prevents the AP-mediated destruction of RBCs predictive of extravascular hemolysis. OMS906 is currently in clinical development for the treatment of PNH."

Preclinical • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CD55 • CD59 • CFB • PRSS3

OMS906, a Novel Alternative Pathway MASP-3 Inhibitor, Normalizes Hemoglobin Levels and Increases Clone Size in Treatment-Naïve PNH Patients (ASH 2023) - P1b | "In this interim analysis, MASP-3 inhibitor OMS906 was well tolerated with no safety signals of concern. Once-monthly SC OMS906 resulted in clinically meaningful, beneficial effects on Hgb, LDH, and RBC clone size in treatment-naïve PNH patients. OMS906 dose escalation guided by occurrence of subclinical hemolysis is being evaluated with the goal of achieving quarterly dosing."

Clinical • Anemia • Aplastic Anemia • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Pruritus • Rare Diseases • Renal Disease • Thrombocytopenia • Thrombosis • CD55 • CD59 • PRSS3

Population Pharmacokinetics/Pharmacodynamics and Clinical Pharmacology of Zaltenibart (OMS906) in Healthy Subjects and Patients with PNH (ASH 2024) - "Clinically, zaltenibart improved hematological markers in pts with PNH, including LDH, ARC, and Hb, in a dose- and exposure-related manner, with greater magnitude and duration of effects seen at the highest dose (5mg/kg) administered Q8W to patients inadequately treated with ravulizumab. The latter is directly observed to protect against both intravascular and extravascular hemolysis in PNH pts. Taken together, these data sources will enable threshold concentrations to be identified and applied to dose/dose regimen simulations to identify the optimal dose providing durable control of hemolysis in pts with PNH."

Clinical • PK/PD data • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • PRSS3

Monotherapy Treatment with Zaltenibart (OMS906), an Alternative Pathway Masp-3 Inhibitor, Improved Key Hematologic Parameters in Patients with PNH with a Suboptimal Response to Ravulizumab: Interim Results from a Phase 2 Proof-of-Concept Study (ASH 2024) - P2 | "Zaltenibart monotherapy resulted in sustained clinically meaningful improvements in Hb and ARC and prevented both IVH and EVH. Data from this interim analysis support the potential for zaltenibart as monotherapy for treating PNH in pts with suboptimal response to C5 inhibitors."

Clinical • Monotherapy • P2 data • Anemia • Cardiovascular • Cough • Fatigue • Hematological Disorders • Infectious Disease • Meningococcal Infections • Musculoskeletal Pain • Novel Coronavirus Disease • Otorhinolaryngology • Pain • Respiratory Diseases • Thrombocytopenia • Thrombosis • PRSS3

Novo Nordisk and Omeros announce asset purchase and license agreement for Omeros’ clinical-stage MASP-3 inhibitor zaltenibart (OMS906) (GlobeNewswire) - "Under the terms of the agreement, Novo Nordisk will be granted exclusive global rights to develop and commercialise zaltenibart in all indications. Omeros is eligible to receive 340 million US dollars in upfront and near-term milestone payments, up to a total of 2.1 billion dollars including potential development and commercial milestones, plus tiered royalties on net sales....The transaction is subject to certain customary closing conditions, including applicable regulatory approvals, and is expected to close in the fourth quarter of 2025."

Licensing / partnership • Paroxysmal Nocturnal Hemoglobinuria

OMS906-PNH-001: Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab (clinicaltrials.gov) - P2 | N=12 | Completed | Sponsor: Omeros Corporation | Active, not recruiting ➔ Completed | Trial completion date: Jul 2025 ➔ Oct 2024 | Trial primary completion date: May 2025 ➔ Oct 2024

Trial completion • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Safety and Efficacy Study of OMS906 in Patients With C3G and ICGN (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Omeros Corporation | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology

DOSE DETERMINATION FOR A RARE INDICATION: PHASE 3 DOSE OF ZALTENIBART, AN INVESTIGATIONAL MASP-3 INHIBITOR, FOR TREATMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) (EHA 2025) - "Data sources: 1) PK/PD data from two Phase 1 trials in healthy subjects and 2) PK/PD and clinical data (hemoglobin (Hgb); absolute reticulocyte count (ARC)) from two Phase 1b/2 studies in pts with PNH: PNH-001 (pts with anemia on ravulizumab who switched to zaltenibart monotherapy) and PNH-002 (complement inhibitor-naïve pts who underwent individualized dose-interval assessments following stabilization on monotherapy). Zaltenibart shows potential as a therapeutic option for pts with PNH. The safety and efficacy of the 8 mg/kg dose, identified by the learning-confirming approach as protective against both IVH and EVH, is being assessed in pts with PNH. The safety of a 12 mg/kg dose, which is expected to allow once-quarterly administration, is being assessed in healthy subjects."

P3 data • Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

VALIDATION OF MASP-3 AS A NOVEL PROXIMAL TARGET FOR COMPLEMENT-MEDIATED DISEASES: LEARNINGS FROM NONCLINICAL AND CLINICAL PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) STUDIES (EHA 2025) - "These nonclinical and clinical data demonstrate that MASP-3, the most upstream target and key activator of the AP, is a viable therapeutic target for PNH. Notably, clinical data in pts with PNH highlight that inhibiting MASP-3 translates to clinical improvements, providing protection from complement-mediated hemolysis. Efficacy of MASP-3 inhibition using zaltenibart at a dose of 8 mg/kg is being evaluated in Phase 3 clinical trials in pts with PNH."

Clinical • Cognitive Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • PRSS3

ZALTENIBART, AN ALTERNATIVE PATHWAY OF COMPLEMENT MASP-3 INHIBITOR, IMPROVED HEMATOLOGIC PARAMETERS IN PNH PATIENTS WITH PERSISTENT ANEMIA ON RAVULIZUMAB: ADJUNCTIVE AND MONOTHERAPY TREATMENT PHASES (EHA 2025) - P1b, P2 | "MASP-3 inhibitor zaltenibart 5 mg/kg IV Q8W mono tx was well tolerated in pts with PNH with no safety signals of concern and resulted in sustained, clinically meaningful improvements in Hb and ARC. Treatment response was maintained following withdrawal of ravu and pts have continued on mono tx treatment in a long-term open-label extension trial (NCT06298955)."

Clinical • Monotherapy • Anemia • Back Pain • Cough • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Meningococcal Infections • Musculoskeletal Pain • Neutropenia • Novel Coronavirus Disease • Pain • Rare Diseases • Respiratory Diseases • Thrombocytopenia

Omeros Corporation Announces Presentation of Positive Zaltenibart Data at ASH Annual Meeting (Businesswire) - P2 | N=12 | NCT05972967 | P1 | N=10 | NCT05889299 | Sponsor: Omeros Corporation | "Enrollment for the zaltenibart Phase 3 clinical trials in PNH is expected to open in early 2025....In PNH patients experiencing substantial extravascular hemolysis while receiving ravulizumab, zaltenibart monotherapy resulted in sustained clinically meaningful improvements in both hemoglobin and absolute reticulocyte count and prevented both intravascular and extravascular hemolysis. Zaltenibart monotherapy was well tolerated with no safety signals of concern....These PK/PD data support the selection of 8mg/kg as the optimal dose for intravenous administration every 8 weeks to achieve complete suppression (> 98 percent) of alternative pathway activation – the dosing regimen that will be used in the Phase 3 PNH clinical trials."

New P3 trial • P1 data • P2 data • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria

OMS906-PNH-001: Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab (clinicaltrials.gov) - P2 | N=12 | Active, not recruiting | Sponsor: Omeros Corporation | Phase classification: P1b ➔ P2 | Recruiting ➔ Active, not recruiting

Enrollment closed • Phase classification • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Omeros Corporation Announces Upcoming Presentations at ASH Annual Meeting (Businesswire) - "Omeros Corporation...today announced that two abstracts directed to zaltenibart (OMS906), Omeros’ investigational inhibitor of MASP-3, the key activator of the alternative pathway of complement, will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH), to be held December 7-10, 2024 in San Diego. The zaltenibart abstracts are directed to the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening hematological disorder. Enrollment for the zaltenibart Phase 3 clinical trials in PNH is expected to open in early 2025."

Enrollment status • P2 data • Paroxysmal Nocturnal Hemoglobinuria

Omeros Corporation Reports Third Quarter 2024 Financial Results (Businesswire) - "Zaltenibart....In September and October 2024, we met with FDA and European regulators to discuss further details of our planned Phase 3 program for zaltenibart in PNH. With both regulatory agencies, we discussed data developed from our clinical and nonclinical programs to date as well as our plans for Phase 3 development of zaltenibart in PNH. Both regulatory agencies agreed with the design of our proposed studies as well as our dose-finding strategy and provided other valuable feedback to inform our development plans. We now have a clear path to opening Phase 3 enrollment, which we expect in early 2025."

European regulatory • FDA event • New P3 trial • Immunology • Paroxysmal Nocturnal Hemoglobinuria

OMS906, A NOVEL ALTERNATIVE PATHWAY MASP-3 INHIBITOR, IMPROVED HEMATOLOGIC PARAMETERS IN PNH PATIENTS WITH SUBOPTIMAL RESPONSE TO RAVULIZUMAB TREATMENT: PHASE 2 DOSE-FINDING STUDY INTERIM RESULTS (EHA 2024) - P1b | "MASP-3 inhibitor OMS906 added to ravu was well tolerated with no safety signals ofconcern in PNH pts who were experiencing substantial EVH on ravu monotherapy. Rapid improvement in Hband reduced ARC demonstrate OMS906 prevents EVH. Dose range and frequency of OMS906 are being furtherexplored, including the potential of OMS906 as monotherapy for PNH."

Clinical • P2 data • Anemia • Aplastic Anemia • Back Pain • Fatigue • Hematological Disorders • Infectious Disease • Meningococcal Infections • Musculoskeletal Pain • Novel Coronavirus Disease • Pain • Thrombocytopenia • PRSS3

CLINICAL PHARMACOLOGY OF OMS906, A POTENT INHIBITOR OF MASP-3 AND THE ALTERNATIVE PATHWAY OF COMPLEMENT ACTIVATION (EHA 2024) - "Reduction in free MASP-3 levels by OMS906, indicative of a selective and potent effecton the target, is concordant with MASP-3 inhibition as measured by the change in circulating CFD status withreduction in mCFD and a concomitant increase in proCFD levels and in inhibition of AP activity. OMS906 wassafe and well tolerated, enabling further dose escalation with the potential for prolonging the dosing interval. Continued investigation in AP-mediated diseases is warranted."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • CFB • PRSS3

EXPOSURE-RESPONSE MODELING USING POPULATION PK/PD METHODS RELIABLY PREDICT POPULATION AND INDIVIDUAL RESPONSES OF COMPLEMENT MATURE FACTOR D, HEMOGLOBIN AND LDH IN PNH PATIENTS EXPOSED TO OMS906 (EHA 2024) - P1b | " OMS906 popPK/PD modeling was based on PK data from a Phase 1 trial in healthy subjects, and PK, PD andclinical data (MFD, Hb and LDH) from two studies in pts with PNH: a Phase 2 trial in pts who had inadequateresponse to ravulizumab and received 3 or 5 mg/kg IV OMS906 as adjunctive treatment at 8-wk intervals(OMS906-PNH-001; NCT05972967) and a Phase 1b study in pts who were complement-treatment naïve andreceived repeat dose of 5 mg/kg OMS906 SC at 4-wk intervals (OMS906-PNH-002; NCT05889299). A series of indirect sigmoidal Emax response models were developed to reliablypredict the effect of OMS906 on MFD, Hb and LDH levels in pts with PNH and the predicted values wereconcordant with observed values. These models will be used to simulate dose and dosing regimen profiles foroptimal PNH pt care."

Clinical • PK/PD data • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • PRSS3

Omeros Corporation Reports First Quarter 2024 Financial Results (Businesswire) - "Interim analysis results from the combination therapy portion of our ongoing Phase 2 clinical trial evaluating OMS906 in PNH patients who have had an unsatisfactory response to the C5 inhibitor ravulizumab will be featured in a podium presentation at EHA 2024, the annual congress of the European Hematology Association to be held in Madrid, Spain. The presentation, scheduled for June 15, 2024....Data from the monotherapy portion of the trial are expected in late 2024....Two additional abstracts directed to OMS906 will also be featured at EHA 2024....We expect to meet again with FDA later this year to discuss further details of the design of our Phase 3 study in this indication, which we are targeting to initiate in late 2024....Our Phase 2 clinical trial in complement 3 glomerulopathy ('C3G') has begun enrollment and is ongoing. A Phase 3 program in C3G is targeted to begin in early 2025."

New P3 trial • P2 data • Trial status • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Omeros Corporation Announces Upcoming Presentations at 2024 European Hematology Association (EHA) Hybrid Congress (Businesswire) - "Omeros Corporation...today announced that interim analysis data from its ongoing Phase 2 study of OMS906 in patients with paroxysmal nocturnal hemoglobinuria submitted to the 2024 European Hematology Association (EHA) Hybrid Congress has been selected for podium presentation. The EHA Congress will be held June 13-16, 2024, in Madrid, Spain. OMS906 is Omeros’ investigational inhibitor of MASP-3 targeting the alternative pathway of complement. Two additional poster presentations directed to OMS906 will also be featured at the congress."

Clinical • P2 data • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Safety and Efficacy Study of OMS906 in Patients With C3G and ICGN (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Omeros Corporation | Not yet recruiting ➔ Recruiting | Trial completion date: Jun 2025 ➔ Apr 2026

Enrollment open • Trial completion date • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology