Omeros Corporation Provides Update on Progress Toward BLA Resubmission

Rhea-AI Summary

Omeros (Nasdaq: OMER) has provided an update on its progress toward resubmitting its biologics license application (BLA) for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). The company has received FDA's response on the revised statistical analysis plan (SAP) and has no other presubmission requests pending. An independent expert statistical group will conduct data analyses, and if results support resubmission, the company plans to finalize and resubmit the BLA promptly. The FDA's recent feedback included recommendations for additional sensitivity analyses, which Omeros has accepted.

Positive

• FDA's feedback on statistical analysis plan received with only minor recommendations
• No other presubmission requests pending from FDA
• Clear path forward for BLA resubmission if analytical results are supportive

Negative

• Additional statistical analyses required before BLA resubmission
• Timeline for BLA resubmission remains uncertain pending statistical analysis results

Insights

Medical Research Analyst

The update on narsoplimab's BLA resubmission process represents a critical regulatory milestone for Omeros. FDA's acceptance of the revised Statistical Analysis Plan with minor additional recommendations significantly de-risks the resubmission pathway. The focus on TA-TMA, a serious transplant complication with high mortality and no approved treatments, positions narsoplimab as a potential first-in-class therapy.

The independent statistical analysis adds credibility to the process, though the actual efficacy data remains pending. Key catalysts ahead include:

• Primary endpoint analysis results
• Additional sensitivity analyses requested by FDA
• Final BLA resubmission timeline

The pathway appears clearer now, but success still hinges on the statistical outcomes. A positive result could unlock significant value given the unmet medical need in TA-TMA.

SEATTLE--(BUSINESS WIRE)-- Omeros Corporation (Nasdaq: OMER) today announced an update on its progress toward planned resubmission of its biologics license application (“BLA”) for narsoplimab, the company’s first-in-class antibody targeting MASP-2, the effector enzyme of the lectin pathway of complement, in hematopoietic stem cell transplant-associated thrombotic microangiopathy (“TA-TMA”). In last week’s quarterly earnings release and associated call, Omeros stated that it was awaiting feedback from the U.S. Food and Drug Administration (“FDA”) on the company’s revised statistical analysis plan (“SAP”) for the BLA. Omeros has now received FDA’s response on the revised SAP, has no other presubmission information requests pending and is not aware of any other impediment to resubmitting the narsoplimab BLA. Following data preparation, primary endpoint and other analyses will be conducted by an independent expert statistical group. If the analytical results support resubmission of the BLA, the company plans to finalize and resubmit the BLA as soon as possible.

As previously disclosed, as part of a September 2024 meeting with the FDA regarding Omeros’ proposed BLA resubmission, FDA provided minor feedback on the proposed SAP for the primary efficacy endpoint. The feedback was limited to requesting a few additional sensitivity analyses. Omeros accordingly revised and resubmitted the SAP and FDA has now responded with additional recommendations on the SAP, which are acceptable to Omeros. The independent statistical group will incorporate FDA’s additional recommendations into the final SAP, implement and validate all required modifications to the related statistical programs, and then conduct the prespecified efficacy analyses. Following validation of results, Omeros will share publicly the outcome of the primary analysis and, as completed, additional analyses.

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. OMS906, 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 five 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 “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 next steps in relation to the biologics license application for narsoplimab, the timing of regulatory events, the availability of clinical trial data and related analyses, the prospects for obtaining FDA approval of narsoplimab in any indication, expectations regarding the initiation or continuation of clinical trials evaluating Omeros’ drug candidates and the anticipated availability of data therefrom, expectations regarding future cash expenditures, and expectations regarding the sufficiency and availability of our capital resources to fund current and planned operations, 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, unexpected or inconclusive results of our statistical analyses relating to an external registry of TA-TMA patients, unanticipated or unexpected outcomes of regulatory processes in relevant jurisdictions, unproven preclinical and clinical development activities, our financial condition and results of operations, regulatory processes and oversight, challenges associated with manufacture or supply of our products to support clinical trials, regulatory process and/or commercial sale following any marketing approval, changes in reimbursement and payment policies by government and commercial payers or the application of such policies, failure by Congress to reauthorize the priority review voucher program or other legislative developments, 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, an 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241121159957/en/

Jennifer Cook Williams

Cook Williams Communications, Inc.

Investor and Media Relations

IR@omeros.com

Source: Omeros Corporation

FAQ

What is the status of Omeros (OMER) narsoplimab BLA resubmission?

Omeros has received FDA feedback on the revised statistical analysis plan and has no other pending presubmission requests. The company will proceed with data analyses by an independent statistical group before potential BLA resubmission.

What feedback did FDA provide to Omeros (OMER) regarding the statistical analysis plan?

FDA provided minor feedback requesting additional sensitivity analyses, and recently provided additional recommendations on the SAP, which Omeros has accepted.

What is the next step for Omeros (OMER) narsoplimab BLA process?

An independent expert statistical group will conduct data analyses incorporating FDA's recommendations, and if results are supportive, Omeros will proceed with BLA resubmission.