Latest news with #TA-TMA
Business Wire
06-05-2025
- Business
- Business Wire
FDA Accepts Resubmission of BLA for Narsoplimab for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (TA-TMA) and Assigns Late September PDUFA Date
SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). The resubmission was classified as a Class 2 resubmission and pursuant to the Prescription Drug User Fee Act (PDUFA) has been assigned a target action date for the FDA decision in late September 2025. The BLA resubmission includes the primary set of analyses comparing overall survival for narsoplimab-treated patients to overall survival for an external control group of TA-TMA patients. Results of those analyses showed clinically meaningful and statistically significant improvements in survival associated with narsoplimab treatment. Also included in the resubmission are data describing survival in Omeros' expanded access program in which adult and pediatric TA-TMA patients were treated with narsoplimab. Since resubmission of the BLA in late March, Omeros has received and is responding to information requests from FDA and expects an interactive review by FDA. Omeros is also preparing a marketing authorization application (MAA) for narsoplimab in TA-TMA, which is targeted for submission to the European Medicines Agency later this quarter. About Narsoplimab Narsoplimab, also known as 'OMS721,' is an investigational fully human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 has been demonstrated to leave intact the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. A biologics license application (BLA) for use of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is under review by the U.S. Food and Drug Administration (FDA) and the corresponding European marketing authorisation application (MAA) is being prepared for submission. FDA has granted narsoplimab breakthrough therapy and orphan drug designations for TA-TMA and orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in hematopoietic stem-cell transplant. About Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is a significant and often lethal complication of stem cell transplantation. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft-versus-host disease, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of TA-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an approximately 40-percent incidence of TA-TMA, and high-risk features may be present in up to 80 percent of these patients. In severe cases of TA-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae (e.g., dialysis) are common. There is no approved therapy or standard of care for TA-TMA. 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 under review by 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. Zaltenibart, Omeros' inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in clinical development for treatment of 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 novel cellular and molecular immuno-oncology programs. For more information about Omeros and its programs, visit 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 'aim,' '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 review process and timing of FDA action on the resubmitted BLA for narsoplimab in the United States, the anticipated submission of a marketing authorization application with the EMA and the timing thereof, the prospects for obtaining FDA or EMA approval of narsoplimab in any indication, and expectations regarding the sufficiency and availability of our capital resources to fund current and planned operations, including the potential commercialization of narsoplimab if it is approved by FDA or the EMA, 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 or unexpected regulatory conclusions or interpretations related to the clinical data, external registry data, statistical analyses or other information and data included in the narsoplimab BLA , inability to respond satisfactorily to information requests during regulatory review of the narsoplimab BLA or MAA, potential differences between the diagnostic criteria used in our pivotal trial and in the external registry, and whether FDA and the EMA determine the registry used in our statistical analysis is sufficiently representative of TA-TMA patients, unanticipated or unexpected outcomes or requirements of regulatory processes in relevant jurisdictions,, our financial condition and results of operations, including our ability to raise additional capital for our operations on favorable terms or at all, regulatory processes and oversight, challenges associated with manufacture or supply of our products to support clinical trials, regulatory inspections 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, 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.. 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.
Yahoo
01-04-2025
- Business
- Yahoo
Omeros Corp (OMER) Q4 2024 Earnings Call Highlights: Navigating Financial Challenges with ...
Net Loss (Q4 2024): $31.4 million or $0.54 per share. Net Loss (Full Year 2024): $156.8 million or $2.70 per share. Cash and Investments (as of Dec 31, 2024): Over $90 million. Costs and Expenses (Q4 2024): $35.7 million, a slight increase from the previous quarter. Interest Expense (Q4 2024): $3.2 million, $1 million lower than Q3 2024. OMIDRIA Royalties (Q4 2024): $10.1 million based on net sales of $33.6 million. OMIDRIA Net Sales (Q4 2024): $33.6 million, an increase from $31 million in Q3 2024. Income from Discontinued Operations (Q4 2024): $5.2 million. Warning! GuruFocus has detected 2 Warning Signs with OMER. Release Date: March 31, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Omeros Corp (NASDAQ:OMER) has resubmitted its Biologics License Application (BLA) for narsoplimab in the treatment of TA-TMA, with a PDUFA date set for September, indicating progress towards potential approval. The company has over $90 million in cash and investments, providing a financial cushion to support ongoing operations and development programs. Omeros Corp (NASDAQ:OMER) is actively pursuing partnerships and other financial strategies, such as royalty monetization and equity transactions, to strengthen its financial position. The company has a robust pipeline, including the development of zaltenibart for PNH, which is in Phase 3 trials, and OMS1029, a next-generation MASP-2 inhibitor. Omeros Corp (NASDAQ:OMER) has established strong relationships with key transplant centers in the US, positioning it well for the potential launch of narsoplimab. Omeros Corp (NASDAQ:OMER) reported a significant net loss of $156.8 million for the full year 2024, highlighting ongoing financial challenges. The company is in preliminary discussions to restructure its 2026 convertible notes, indicating potential financial strain and the need for debt management. There is uncertainty regarding the pricing strategy for narsoplimab, which could impact market acceptance and revenue generation. The company faces competition in the complement inhibitor space, which could affect the market share and success of its products. Omeros Corp (NASDAQ:OMER) has not yet disclosed specific plans for ex-US commercialization of its products, which could limit its global market reach. Q: Can you explain why you believe the resubmission of the BLA for narsoplimab is strong and what the implications are? A: Gregory Demopulos, CEO, explained that the statistical analysis plan was created with FDA's agreement, and the results are impressively strong across all analyses. The primary analysis showed a more than threefold improvement in survival with a P value of less than 0.00001, indicating statistically significant and clinically meaningful results. Catherine Melfi, Chief Regulatory Officer, added that the package is solid due to a well-matched external control group and strong results. Q: How are you thinking about pricing for narsoplimab now that you are in launch preparation mode? A: Gregory Demopulos, CEO, stated that while pricing plans have not been disclosed, it is expected to be similar to other complement inhibitors used in TA-TMA. Nadia Dac, Chief Commercial Officer, added that narsoplimab's significant value and potential administration in both inpatient and outpatient settings are being considered in pricing determination. Q: What is the strategy for funding given the commercial launch and multiple Phase 3 trials? A: Gregory Demopulos, CEO, mentioned ongoing discussions to restructure convertible notes and bring in additional capital. Options include partnering, debt instruments, royalty monetization, or equity. The company is confident in managing the balance sheet effectively. Q: Where does the company stand on manufacturing scalability for narsoplimab, and how are you increasing awareness among treating physicians? A: Gregory Demopulos, CEO, confirmed that they have sufficient drug supply to support narsoplimab for the first several years. Nadia Dac, Chief Commercial Officer, highlighted efforts to build awareness of TA-TMA among top transplant centers and physicians, emphasizing the need for a solution. Q: How does Omeros view its strategic direction in terms of commercialization and partnerships? A: Gregory Demopulos, CEO, stated that Omeros plans to launch narsoplimab independently in the US but expects to partner for ex-US commercialization. The company aims to manage ex-US commercialization independently in the future and is focused on partnering programs regionally or internationally. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
10-02-2025
- Health
- Yahoo
Omeros Corporation Announces Upcoming Presentations Detailing Outcomes of Narsoplimab Treatment for TA-TMA Under an Expanded Access Program
SEATTLE, February 10, 2025--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced two presentations that will be featured at the 2025 Tandem Meetings – the Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood and Marrow Transplant Research, to be held February 12-15, 2025 in Honolulu, Hawaii. Both presentations report real world outcomes from patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) treated with narsoplimab supplied by Omeros under an expanded access program, also referred to as compassionate use. The first reports overall survival of 128 allogeneic transplant patients with TA-TMA treated with narsoplimab under the expanded access program and will be featured as a podium presentation by Michelle Schoettler, M.D., Assistant Professor of Pediatric Oncology and Hematopoietic Cellular Therapy at Emory University School of Medicine. The second reports on a single-center cohort of adult TA-TMA patients who were treated with narsoplimab after failing eculizumab treatment. The abstract will be featured in a poster session and presented by Piyatida Chumnumsiriwath, M.D., of the Hematopoietic Stem Cell Transplantation and Cellular Therapy Program at the University of California, Irvine. The presentation abstracts are available now on the Tandem Meetings website. Details of the presentations and links to each abstract follow: Narsoplimab Treatment for Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy – Real World Outcomes from an Expanded Access Program Presentation Session: Toxicity and Supportive Care (Oral Abstract Session D)Date: Thursday, February 13, 2025Presentation Time: 3:15 - 3:30 p.m. HSTLocation: Ballroom A (HCC)Presenting Author: Michelle Schoettler, Link Narsoplimab for Refractory Transplantation-Associated Thrombotic Microangiopathy in Adult Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Poster Session: Late Effects, Quality of Life and Accelerated AgingDate: Thursday, February 13, 2025Presentation Time: 6:45 - 7:45 p.m. HSTLocation: Exhibit Hall 3 (HCC)Presenting Author: Piyatida Chumnumsiriwath, Link The poster and presentation materials are expected to be made available on Omeros' website at shortly after the meeting presentations. About Narsoplimab Narsoplimab, also known as "OMS721," is an investigational fully human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 has been demonstrated to leave intact the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. A biologics license application (BLA) is pending before the FDA for use of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Omeros will resubmit the BLA for narsoplimab in TA-TMA followed by our planned submission of the corresponding European marketing authorisation application (MAA) in 2025. FDA has granted narsoplimab breakthrough therapy and orphan drug designations for TA-TMA and orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies. The European Medicines Agency (EMA) has granted orphan drug designation to narsoplimab for treatment in hematopoietic stem-cell transplant. About Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is a significant and often lethal complication of stem cell transplantation. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft-versus-host disease, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of TA-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an approximately 40-percent incidence of TA-TMA, and high-risk features may be present in up to 80 percent of these patients. In severe cases of TA-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae (e.g., dialysis) are common. There is no approved therapy or standard of care for TA-TMA. 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. Zaltenibart, 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 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 "aim," "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 resubmission of the BLA for narsoplimab in the United States and the submission of a marketing authorization application with the EMA, the timing and outcomes of regulatory events, the availability and outcomes of additional analyses, the prospects for obtaining FDA or EMA approval of narsoplimab in any indication, expectations regarding future cash expenditures, and expectations regarding the sufficiency and availability of our capital resources to fund current and planned operations, including the potential commercialization of narsoplimab if it is approved by FDA or the EMA, 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, potential differences between the diagnostic criteria used in our pivotal trial and in the external registry, and whether FDA and the EMA determine the registry used in our statistical analysis is sufficiently representative 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 inspections 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, 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, and 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 Contacts Jennifer Cook WilliamsCook Williams Communications, and Media RelationsIR@ Sign in to access your portfolio
