Shares of thinly-traded small-cap biopharma Omeros Corporation (NASDAQ: OMER) are advancing Wednesday following a positive readout from a pivotal Phase 3 study of its investigational asset narsoplimab.
Omeros said the pivotal study that evaluated narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy, or HCST-TMA, met the composite primary endpoint and the secondary endpoints.
Narsoplimab is Omeros' lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2.
The primary efficacy endpoint is the proportion of patients achieving a highly rigorous set of response criteria requiring both improvement in HSCT-TMA laboratory markets and in clinical status.
Why Narsoplimab Is Important
On the primary efficacy endpoint, 56% of all patients receiving at least one dose of narsoplimab achieved complete responder status, and 68% of patients receiving the protocol-specified narsoplimab treatment of at least 4 weeks of dosing achieved complete responder status.
Omeros also noted narsoplimab was found to be safe and well tolerated, with 21 patients dying during the trial due to causes common in stem cell transplant and no additional patients discontinuing for adverse effects.
Patients with severe forms of HSCT-TMA have a dismal prognosis with no treatment currently available, the company said, citing Rafael Duarte, Secretary of the European Society for Blood and Marrow Transplantation.
"I expect a treatment with this profile would be widely adopted for use in these patients and even lead to increased physician recognition of the disorder," Duarte said.
Omeros had initiated rolling BLA submission in October.
Omeros shares traded higher by 8% to $15.53 at time of publication.
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