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news.US-based clinical-stage biopharmaceutical firm Omeros has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) to start the Phase II clinical program for OMS721 to treat thrombotic microangiopathies (TMAs).
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OMS721 is the company’s lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system.
In the trial, the company will assess OMS721 in the treatment of TMAs, a family of orphan disorders, including atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP).
In February 2014, the company had released positive data following completion of dosing in the Phase I clinical trial of OMS721, which was carried out in the Netherlands under a Clinical Trial Application.
OMS721 was administered by subcutaneous injection or intravenous infusion in the trial.
In the Phase I trial, OMS721 was well tolerated and achieved a high degree of sustained lectin pathway inhibition, consistent with the significant efficacy of OMS721 seen in animal models of TMA, age-related macular degeneration and other lectin pathway-related disorders.
The initial Phase II clinical trial is an open-label, two-stage ascending-dose-escalation trial in adult subjects with TMA.
Objectives of the Phase II trial are to evaluate efficacy, safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of OMS721 in patients with TMAs, including aHUS, TTP and transplant-related TMA.
Design of the Phase II trial is based on the final data from the Phase I clinical trial of OMS721 and on discussions with the FDA’s Division of Hematology Products at a pre-IND meeting held earlier this year.
Omeros chairman and chief executive officer Gregory Demopulos said the company is happy to start the Phase II program for OMS721 to evaluate the molecule across the family of TMAs.
"Based on our preclinical data, the FDA awarded OMS721 Orphan Drug designation for all TMAs, broadening beyond our initial submission for aHUS only," Demopulos said.
Image: Micrograph of thrombotic microangiopathy with the characteristic onion-skin layering seen in older lesions. Photo: courtesy of Nephron