Omeros Reports Final Efficacy and Safety Data from the Narsoplimab Pivotal Trial in HSCT-TMA
-- Webcast Scheduled for Today at
Omeros previously presented preliminary data from its pivotal HSCT-TMA trial. The final data reported today are those that are included in the BLA. In the 28-patient single-arm, open-label pivotal trial in adult HSCT-TMA patients, treatment consisted of narsoplimab administered intravenously once weekly for up to 8 weeks with an extended follow-up period. The study’s patient population was very ill, with the large majority of study patients having multiple comorbidities at baseline (e.g., graft-versus-host disease, significant infections, multi-organ dysfunction, etc.). The FDA-agreed primary endpoint (complete response) required clinical improvement in TMA markers (platelet count and lactate dehydrogenase [LDH]) and in organ function (renal, pulmonary, gastrointestinal or neurological) or freedom from transfusion. Secondary endpoints included 100-day and overall survival as well as change from baseline for individual laboratory markers (platelets, LDH, haptoglobin, hemoglobin and creatinine).
The final results on primary and key secondary endpoints include:
- 61% (95% CI: 40.6% to 78.5%) complete response rate (CRR) in the full analysis set (FAS; patient receiving at least one dose of narsoplimab; p<0.0001 compared to 15% efficacy threshold agreed with FDA)
- 74% (95% CI: 51.6% to 89.8%) CRR in the per-protocol (PP) population (patients receiving the protocol-specified narsoplimab treatment for at least 4 weeks; p<0.0001 compared to the 15% threshold)
- 100-day survival was 68% in the FAS, 83% in the PP population and 94% in complete responders
- Median overall survival was 274 days in the FAS, 361 days in the PP population and, for complete responders, was not estimable (more than half of the responders were alive at last follow-up)
Similar responses were observed across all patient subgroups defined by baseline characteristics, transplant characteristics and transplant complications. The majority of individual laboratory markers showed statistically significant improvement with the remainder numerically improving. Adverse events were typical of the post-HSCT population (e.g., fever, diarrhea, vomiting, nausea and neutropenia) and no safety signal of concern was identified. Six deaths occurred in the study, all from causes common in HSCT.
The data will be presented today at
To access the live conference call via phone, please dial (844) 831-4029 from
To access the live or subsequently archived webcast and presentation materials on the internet, click here or go to the company’s website at www.omeros.com and select “Events” under the Investors section of the website.
Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple late-stage clinical development programs focused on complement-mediated disorders, including COVID-19, and substance abuse. A rolling biologics license application for narsoplimab, the company’s lead MASP-2 inhibitor, in hematopoietic stem cell transplant-associated thrombotic microangiopathy is being completed for submission to the
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 “anticipate,” “believe,” “can,” “could,” “estimate,” “expect,” “goal,” “intend,” “likely”, “look forward to,” “may,” “on track,” “plan,” “potential,” “predict,” “project,” “prospects,” “scheduled,” “should,” “slated,” “targeting,” “will,” “would” and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros’ investigational product, 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, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading “Risk Factors” in the company’s Annual Report on Form 10-K for the year ended
Investor and Media Relations