Latest news with #PNH
Yahoo
19-05-2025
- Health
- Yahoo
NovelMed's Phase II Data in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Ruxoprubart Shows Best-in-Class Efficacy as Monotherapy
--- Ruxoprubart (NM8074) met all clinical endpoints, offering a safe, differentiated treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder. Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy subjects in the Phase I trial. Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint. Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period. Reduced LDH levels to baseline, meeting the primary efficacy endpoint. Significantly increased PNH Type III clone size by protecting PNH Red Blood Cells from complement-mediated lysis. Allowed normal pathogenic clearance in all treated PNH subjects, as infection cleared as expected. The regulatory agency has recently approved a once-weekly subcutaneous protocol for Ruxoprubart — allowing for self-administration and offering a distinct advantage over Apellis, which requires twice-weekly dosing. Ruxoprubart effectively inhibited the Alternative Pathway (AP) without affecting the Classical Pathway (CP) in the trial. More importantly, the FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart for treating PNH. A Breakthrough Therapy Designation (BTD) application for Ruxoprubart in PNH will soon be filed for FDA review. Ruxoprubart is set to be developed as a novel treatment for several complement-mediated disorders, including but not limited to renal, hematological, dermatological, and ocular diseases. CLEVELAND, May 19, 2025 (GLOBE NEWSWIRE) -- NovelMed is pleased to announce positive 12-week interim results from the ongoing multi-dose Phase II trial of Ruxoprubart, a novel complement-targeting immunotherapy, in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased hemoglobin levels, reduced LDH, and increased PNH clone size — key outcomes that contributed to improved disease control and enhanced quality of life for PNH patients. 'The promising interim results from our Phase II PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short. This achievement not only underscores our commitment to advancing Ruxoprubart, an immunotherapy for PNH, but also marks a significant step toward addressing critical unmet needs within this patient population," said Dr. Rekha Bansal, CEO. "I extend my sincere gratitude to the PNH subjects and investigators whose dedication has been instrumental in this success. We are eager to present our findings to regulatory authorities and proceed with the Phase III trial."Figure. NovelMed Scientist Prepares Complement Test Assays to Support Clinical Development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). Ruxoprubart works by binding to Bb and selectively inhibiting the protease activity of the Alternative Pathway C3 convertase, while preserving the Classical Pathway. By acting proximally in the Alternative Pathway, Ruxoprubart provides complete control over both intravascular and extravascular hemolysis, resulting in marked increases in hemoglobin levels observed in this trial. These findings support the drug's advancement as a novel, next-generation immunotherapy for the treatment of PNH and a broad range of hematological, renal, inflammatory, and ocular diseases. 'We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,' said Mr. Alex Kumar, Chief Strategy Advisor at NovelMed. 'These data reinforce our confidence as we look forward to working with the regulatory agency to bring Ruxoprubart to PNH patients without delay. Our mission is to transform the lives of chronically ill patients for whom an ideal treatment has yet to be discovered.' Clinical Study Overview and Efficacy Summary in PNH This Phase II trial was initiated following the successful completion of a Phase I study involving 40 (forty) healthy volunteers. Ruxoprubart was well tolerated across all dose levels, ranging from 0.3 to 20 mg/kg, with no safety concerns. The drug demonstrated selective inhibition of the Alternative Pathway through Bb binding, without affecting the Classical Pathway. These promising safety findings paved the way for regulatory approval to initiate a Phase II study in patients with PNH. This ongoing 12-week clinical trial of Ruxoprubart is an open-label, multi-dose study in treatment-naïve adult subjects with PNH. The primary objectives are to evaluate the safety, tolerability, and efficacy of Ruxoprubart as a monotherapy in the Alternative Pathway-mediated lysis of Red Blood Cells. To date, 10 out of 12 PNH subjects have completed the study. Subjects 11 & 12 have been enrolled to complete the two-cohort trial. Interim results confirm that Ruxoprubart achieved all primary efficacy endpoints, delivering sustained clinical benefit in PNH. The primary efficacy endpoints include: Transfusion Avoidance: PNH patients typically require multiple blood transfusions to maintain normal hemoglobin levels. Ruxoprubart effectively protected PNH Type III cells from Alternative Pathway-mediated destruction, resulting in complete transfusion avoidance and successfully meeting the primary efficacy endpoint. Hemoglobin Increase: PNH patients experience reduced hemoglobin levels, leading to severe anemia. Ruxoprubart increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject increases ranging from 1.5 to 4.3 g/dL, surpassing the efficacy threshold defined for the primary endpoint. An average increase of >1.6 g/dL in hemoglobin was observed across the cohort from the beginning to the end of the treatment period. LDH Reduction: PNH patients experience severe lysis of Red Blood Cells (RBCs), leading to elevated lactate dehydrogenase (LDH) levels. By controlling complement-mediated hemolysis, Ruxoprubart reduced LDH levels to baseline, achieving the primary efficacy endpoint. Preservation of PNH Cells: Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%. By effectively preventing this lysis, Ruxoprubart significantly preserved PNH cells, resulting in a substantial increase in the total PNH clone size and hemoglobin, successfully meeting the primary efficacy endpoint. Pathogenic Clearance: As expected, Ruxoprubart facilitated effective pathogenic clearance across all treated PNH subjects. In summary, the observed changes in efficacy parameters support the drug's mechanism of action and validate Ruxoprubart's potential as a highly effective monotherapy for PNH. 'We are thrilled to share these data, which demonstrate the potential of Ruxoprubart in advanced PNH, and look forward to working with regulatory authorities to bring this new immunotherapy to this patient community,' said Mr. Robert Bard, Vice President of Regulatory Affairs at NovelMed. 'It's very clear that we are on a promising path with exciting Phase II data in this serious hematological disorder.' Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare, acquired hematologic disorder in which Red Blood Cells lack protective surface proteins due to a mutation in the phosphatidylinositol glycan class A (PIGA) gene, leaving them vulnerable to complement-mediated destruction. In healthy individuals, Type I RBCs express complement regulatory proteins and are fully protected from complement-mediated destruction. In contrast, PNH patients have a significant percentage of Type III RBCs, which completely lack these protective proteins and are therefore highly susceptible to Alternative Pathway-mediated lysis. PNH is primarily an Alternative Pathway-mediated disease, with no involvement of the Classical or Lectin pathways in its pathology. Key laboratory markers indicative of disease progression include reduced Type III Red Blood Cells, low hemoglobin, elevated lactate dehydrogenase (LDH), and an increased need for blood transfusions. These markers reflect the underlying hemolytic anemia, which in turn leads to debilitating symptoms such as chronic fatigue, pain, jaundice, and organ damage. As the disease progresses, it significantly affects the patient's quality of life and can ultimately result in life-threatening complications and death if not properly managed. While Soliris® (eculizumab) is the current FDA-approved standard of care for PNH – administered via intravenous infusion to provide C5 blockade – clinical limitations persist. Up to 88% of Soliris-treated patients continue to experience persistent anemia, with more than one-third of them requiring blood transfusions at least once every year. Despite its effectiveness in some patients, Soliris does not fully address the needs of many patients, as a significant proportion remain anemic, fatigued, and require blood transfusions, highlighting the need for specific, effective, and comprehensive therapies. Nearly all FDA-approved therapies for PNH and other complement-mediated disorders, oral or intravenous, suffer from the well-documented Black Box warning label because of the broad immunosuppression of the Classical Pathway, elevating the risk of life-threatening infections. Ruxoprubart offers a selective, upstream, and targeted approach to Alternative Pathway inhibition, without compromising the Classical Pathway, offering the best-in-class novel therapy. These attributes position Ruxoprubart as a safer and more effective treatment for patients with PNH. Ruxoprubart (NM8074) Ruxoprubart, an engineered human immunoglobulin, exhibits dual specificity: 1) it blocks only the Alternative Pathway, dysregulated by the disease, and 2) it binds Bb which has no binding to Factor B. Ruxoprubart met all primary endpoints in the interim evaluation of the ongoing Phase II clinical trial in PNH, positioning it as the first immunotherapy in this indication with the potential for approval without a Black Box warning. This immunotherapy has demonstrated a safe and well-tolerated profile across all five cohorts in the Phase I trial, including the highest dose of 20 mg/kg. Our findings confirm that the drug selectively blocks the Alternative Pathway. This differentiated, novel mechanism sets Ruxoprubart apart from other complement blockers (Soliris®, Ultomiris®, Empaveli®, Voydeya®, and Fabhalta®) currently approved or in development worldwide. Voydeya®, which received FDA approval in 2024 as a combination therapy, was developed by Achillion and acquired by AstraZeneca for $930 million. Despite its limited clinical efficacy, Voydeya®'s reliance on co-administration with a C5 inhibitor restricts its potential as a standalone therapy. Unlike Fabhalta®, which targets Factor B, Ruxoprubart (NM8074) binds to Bb, which is generated only upon activation of the Alternative Pathway, offering a unique mechanism of action as monotherapy. As a novel monotherapy with a unique, upstream, and pathway-specific mechanism, Ruxoprubart is emerging as a best-in-class candidate for the treatment of PNH and numerous complement-mediated diseases. NovelMed Therapeutics ( NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative novel biologics (immunotherapies) to treat a wide range of complement-mediated diseases. NovelMed proudly leads the development of Alternative Pathway-specific antibodies (NM8074, NM5072, and NM8070) to address a wide range of chronic complement-mediated disorders. The company is actively conducting a Phase II clinical trial in treatment-naïve PNH subjects to expedite the availability of this promising treatment. Additionally, the FDA has approved Phase II studies for several rare diseases, including but not limited to Atypical Hemolytic Uremic Syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), ANCA-Associated Vasculitis (AAV), and Dermatomyositis (DM). We are committed to delivering life-changing therapies to patients across hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is currently seeking partnerships to advance the development of Ruxoprubart to approval in several chronic diseases. We are open to discussions regarding investment, out-licensing, or acquisition. For more details regarding our progress, please visit Watch our introductory Video to learn more about NovelMed. Contact: Ya Gao, MSNovelMed Communication TeamBD@ 440 2696 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
19-05-2025
- Health
- Yahoo
NovelMed's Phase II Data in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Ruxoprubart Shows Best-in-Class Efficacy as Monotherapy
--- Ruxoprubart (NM8074) met all clinical endpoints, offering a safe, differentiated treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder. Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy subjects in the Phase I trial. Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint. Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period. Reduced LDH levels to baseline, meeting the primary efficacy endpoint. Significantly increased PNH Type III clone size by protecting PNH Red Blood Cells from complement-mediated lysis. Allowed normal pathogenic clearance in all treated PNH subjects, as infection cleared as expected. The regulatory agency has recently approved a once-weekly subcutaneous protocol for Ruxoprubart — allowing for self-administration and offering a distinct advantage over Apellis, which requires twice-weekly dosing. Ruxoprubart effectively inhibited the Alternative Pathway (AP) without affecting the Classical Pathway (CP) in the trial. More importantly, the FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart for treating PNH. A Breakthrough Therapy Designation (BTD) application for Ruxoprubart in PNH will soon be filed for FDA review. Ruxoprubart is set to be developed as a novel treatment for several complement-mediated disorders, including but not limited to renal, hematological, dermatological, and ocular diseases. CLEVELAND, May 19, 2025 (GLOBE NEWSWIRE) -- NovelMed is pleased to announce positive 12-week interim results from the ongoing multi-dose Phase II trial of Ruxoprubart, a novel complement-targeting immunotherapy, in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased hemoglobin levels, reduced LDH, and increased PNH clone size — key outcomes that contributed to improved disease control and enhanced quality of life for PNH patients. 'The promising interim results from our Phase II PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short. This achievement not only underscores our commitment to advancing Ruxoprubart, an immunotherapy for PNH, but also marks a significant step toward addressing critical unmet needs within this patient population," said Dr. Rekha Bansal, CEO. "I extend my sincere gratitude to the PNH subjects and investigators whose dedication has been instrumental in this success. We are eager to present our findings to regulatory authorities and proceed with the Phase III trial."Figure. NovelMed Scientist Prepares Complement Test Assays to Support Clinical Development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). Ruxoprubart works by binding to Bb and selectively inhibiting the protease activity of the Alternative Pathway C3 convertase, while preserving the Classical Pathway. By acting proximally in the Alternative Pathway, Ruxoprubart provides complete control over both intravascular and extravascular hemolysis, resulting in marked increases in hemoglobin levels observed in this trial. These findings support the drug's advancement as a novel, next-generation immunotherapy for the treatment of PNH and a broad range of hematological, renal, inflammatory, and ocular diseases. 'We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,' said Mr. Alex Kumar, Chief Strategy Advisor at NovelMed. 'These data reinforce our confidence as we look forward to working with the regulatory agency to bring Ruxoprubart to PNH patients without delay. Our mission is to transform the lives of chronically ill patients for whom an ideal treatment has yet to be discovered.' Clinical Study Overview and Efficacy Summary in PNH This Phase II trial was initiated following the successful completion of a Phase I study involving 40 (forty) healthy volunteers. Ruxoprubart was well tolerated across all dose levels, ranging from 0.3 to 20 mg/kg, with no safety concerns. The drug demonstrated selective inhibition of the Alternative Pathway through Bb binding, without affecting the Classical Pathway. These promising safety findings paved the way for regulatory approval to initiate a Phase II study in patients with PNH. This ongoing 12-week clinical trial of Ruxoprubart is an open-label, multi-dose study in treatment-naïve adult subjects with PNH. The primary objectives are to evaluate the safety, tolerability, and efficacy of Ruxoprubart as a monotherapy in the Alternative Pathway-mediated lysis of Red Blood Cells. To date, 10 out of 12 PNH subjects have completed the study. Subjects 11 & 12 have been enrolled to complete the two-cohort trial. Interim results confirm that Ruxoprubart achieved all primary efficacy endpoints, delivering sustained clinical benefit in PNH. The primary efficacy endpoints include: Transfusion Avoidance: PNH patients typically require multiple blood transfusions to maintain normal hemoglobin levels. Ruxoprubart effectively protected PNH Type III cells from Alternative Pathway-mediated destruction, resulting in complete transfusion avoidance and successfully meeting the primary efficacy endpoint. Hemoglobin Increase: PNH patients experience reduced hemoglobin levels, leading to severe anemia. Ruxoprubart increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject increases ranging from 1.5 to 4.3 g/dL, surpassing the efficacy threshold defined for the primary endpoint. An average increase of >1.6 g/dL in hemoglobin was observed across the cohort from the beginning to the end of the treatment period. LDH Reduction: PNH patients experience severe lysis of Red Blood Cells (RBCs), leading to elevated lactate dehydrogenase (LDH) levels. By controlling complement-mediated hemolysis, Ruxoprubart reduced LDH levels to baseline, achieving the primary efficacy endpoint. Preservation of PNH Cells: Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%. By effectively preventing this lysis, Ruxoprubart significantly preserved PNH cells, resulting in a substantial increase in the total PNH clone size and hemoglobin, successfully meeting the primary efficacy endpoint. Pathogenic Clearance: As expected, Ruxoprubart facilitated effective pathogenic clearance across all treated PNH subjects. In summary, the observed changes in efficacy parameters support the drug's mechanism of action and validate Ruxoprubart's potential as a highly effective monotherapy for PNH. 'We are thrilled to share these data, which demonstrate the potential of Ruxoprubart in advanced PNH, and look forward to working with regulatory authorities to bring this new immunotherapy to this patient community,' said Mr. Robert Bard, Vice President of Regulatory Affairs at NovelMed. 'It's very clear that we are on a promising path with exciting Phase II data in this serious hematological disorder.' Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare, acquired hematologic disorder in which Red Blood Cells lack protective surface proteins due to a mutation in the phosphatidylinositol glycan class A (PIGA) gene, leaving them vulnerable to complement-mediated destruction. In healthy individuals, Type I RBCs express complement regulatory proteins and are fully protected from complement-mediated destruction. In contrast, PNH patients have a significant percentage of Type III RBCs, which completely lack these protective proteins and are therefore highly susceptible to Alternative Pathway-mediated lysis. PNH is primarily an Alternative Pathway-mediated disease, with no involvement of the Classical or Lectin pathways in its pathology. Key laboratory markers indicative of disease progression include reduced Type III Red Blood Cells, low hemoglobin, elevated lactate dehydrogenase (LDH), and an increased need for blood transfusions. These markers reflect the underlying hemolytic anemia, which in turn leads to debilitating symptoms such as chronic fatigue, pain, jaundice, and organ damage. As the disease progresses, it significantly affects the patient's quality of life and can ultimately result in life-threatening complications and death if not properly managed. While Soliris® (eculizumab) is the current FDA-approved standard of care for PNH – administered via intravenous infusion to provide C5 blockade – clinical limitations persist. Up to 88% of Soliris-treated patients continue to experience persistent anemia, with more than one-third of them requiring blood transfusions at least once every year. Despite its effectiveness in some patients, Soliris does not fully address the needs of many patients, as a significant proportion remain anemic, fatigued, and require blood transfusions, highlighting the need for specific, effective, and comprehensive therapies. Nearly all FDA-approved therapies for PNH and other complement-mediated disorders, oral or intravenous, suffer from the well-documented Black Box warning label because of the broad immunosuppression of the Classical Pathway, elevating the risk of life-threatening infections. Ruxoprubart offers a selective, upstream, and targeted approach to Alternative Pathway inhibition, without compromising the Classical Pathway, offering the best-in-class novel therapy. These attributes position Ruxoprubart as a safer and more effective treatment for patients with PNH. Ruxoprubart (NM8074) Ruxoprubart, an engineered human immunoglobulin, exhibits dual specificity: 1) it blocks only the Alternative Pathway, dysregulated by the disease, and 2) it binds Bb which has no binding to Factor B. Ruxoprubart met all primary endpoints in the interim evaluation of the ongoing Phase II clinical trial in PNH, positioning it as the first immunotherapy in this indication with the potential for approval without a Black Box warning. This immunotherapy has demonstrated a safe and well-tolerated profile across all five cohorts in the Phase I trial, including the highest dose of 20 mg/kg. Our findings confirm that the drug selectively blocks the Alternative Pathway. This differentiated, novel mechanism sets Ruxoprubart apart from other complement blockers (Soliris®, Ultomiris®, Empaveli®, Voydeya®, and Fabhalta®) currently approved or in development worldwide. Voydeya®, which received FDA approval in 2024 as a combination therapy, was developed by Achillion and acquired by AstraZeneca for $930 million. Despite its limited clinical efficacy, Voydeya®'s reliance on co-administration with a C5 inhibitor restricts its potential as a standalone therapy. Unlike Fabhalta®, which targets Factor B, Ruxoprubart (NM8074) binds to Bb, which is generated only upon activation of the Alternative Pathway, offering a unique mechanism of action as monotherapy. As a novel monotherapy with a unique, upstream, and pathway-specific mechanism, Ruxoprubart is emerging as a best-in-class candidate for the treatment of PNH and numerous complement-mediated diseases. NovelMed Therapeutics ( NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative novel biologics (immunotherapies) to treat a wide range of complement-mediated diseases. NovelMed proudly leads the development of Alternative Pathway-specific antibodies (NM8074, NM5072, and NM8070) to address a wide range of chronic complement-mediated disorders. The company is actively conducting a Phase II clinical trial in treatment-naïve PNH subjects to expedite the availability of this promising treatment. Additionally, the FDA has approved Phase II studies for several rare diseases, including but not limited to Atypical Hemolytic Uremic Syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), ANCA-Associated Vasculitis (AAV), and Dermatomyositis (DM). We are committed to delivering life-changing therapies to patients across hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is currently seeking partnerships to advance the development of Ruxoprubart to approval in several chronic diseases. We are open to discussions regarding investment, out-licensing, or acquisition. For more details regarding our progress, please visit Watch our introductory Video to learn more about NovelMed. Contact: Ya Gao, MSNovelMed Communication TeamBD@ 440 2696 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis
Business Wire
15-05-2025
- Business
- Business Wire
Omeros Corporation Reports First Quarter 2025 Financial Results
SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced recent highlights and developments as well as financial results for the first quarter ended March 31, 2025, which include: Net loss for the first quarter of 2025 was $33.5 million, or $0.58 per share, compared to a net loss of $37.2 million, or $0.63 per share for the first quarter of 2024. At March 31, 2025, we had $52.4 million of cash and short-term investments available for operations and debt servicing, a decrease of $37.7 million from December 31, 2024. In March 2025, we resubmitted to the U.S. Food and Drug Administration ('FDA') our Biologics License Application ('BLA') seeking regulatory approval for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy ('TA-TMA'). FDA accepted the resubmission for review as a class 2 resubmission and, pursuant to the Prescription Drug User Fee Act ('PDUFA'), assigned a target date for FDA action of September 25, 2025. We are also preparing a European marketing authorization application ('MAA') for narsoplimab in TA-TMA, which we expect to submit in the second quarter of 2025. On May 12, 2025, we entered into exchange agreements with holders of our 5.25% Convertible Senior Notes due 2026 (the '2026 Convertible Notes'). We exchanged $70.8 million in aggregate principal amount of our 2026 Convertible Notes for newly issued 9.50% Convertible Senior Notes due in June 2029, on a one-for-one basis. In addition, we reached an agreement with two affiliated holders to convert $10.0 million in aggregate principal amount of the 2026 Convertible Notes into shares of our common stock in three separate tranches, with the conversion of the entire principal to be completed no later than September 15, 2025. Following these transactions, the outstanding principal balance of the 2026 Convertible Notes will be reduced to approximately $17.1 million. Significantly, the reduction in the principal amount of our 2026 Convertible Notes eliminated the need to avoid an accelerated maturity of the entire balance of our term loan by making a $20.0 million prepayment and paying a $1.0 million prepayment premium on or prior to November 2025. During the first quarter we elected to temporarily suspend or pause certain activities and programs to prioritize the allocation of our currently available capital to the development of commercial infrastructure and capacities needed to ensure the successful launch of narsoplimab, assuming approval by FDA of our BLA, and to the completion of our ongoing clinical trials with enrolled patients. Last quarter we began initiating clinical trial sites for our Phase 3 program evaluating zaltenibart (formerly known as OMS906) for the treatment of paroxysmal nocturnal hemoglobinuria ('PNH'); however, based on the anticipated ramp up in spending on those trials and the need to prioritize the use of currently available capital, we determined to pause our Phase 3 PNH program temporarily. We are working with our vendors and investigators to ensure that the program is ready to be restarted with as little disruption to the timeline as possible after securing capital. We expect to complete remaining activities in our ongoing clinical trial evaluating zaltenibart for the treatment of PNH in treatment-naïve patients and to continue the long-term extension study, which enrolls zaltenibart-treated PNH patients who have completed any of our prior zaltenibart studies. Although preparations for the anticipated commercial launch of narsoplimab will continue, we have determined to suspend our expanded access program ('EAP') for narsoplimab, also known as compassionate use, to eliminate direct costs associated with drug supply and external management of the EAP program. We remain committed to supporting patients who are currently being treated under the EAP and discontinuation of the EAP will not affect these patients. Additionally, our ongoing study of narsoplimab in pediatric patients with TA-TMA will continue. Development spending on our long-acting, next generation MASP-2 inhibitor, OMS1029 has already been limited. That asset is Phase 2 ready, with drug product needed to support Phase 2 trials having been manufactured and stored, pending the selection of the first indication and the availability and allocation of resources to initiate Phase 2 studies. Spending in other areas of our complement programs, including our small-molecule MASP-2 and MASP-3 programs, is also being reduced or halted as part of our effort to focus resources on core development priorities. 'We are pleased that our BLA for narsoplimab in TA-TMA has been accepted by FDA, which is a significant milestone for our narsoplimab program and for Omeros,' said Gregory A. Demopulos, M.D., Omeros' Chairman and Chief Executive Officer. 'We have already received and are responding to FDA's information requests, and our highest priority as an organization is to obtain approval for narsoplimab. For this reason, we have taken action to reduce expenses and prioritize spending on the narsoplimab launch and other key priorities. In parallel, through the recently completed exchange of the large majority of our 2026 convertible notes for convertible notes maturing in 2029 and converting a small portion to equity, our total debt will be reduced by approximately $10.0 million and our near-term debt maturities will be lowered by over $100 million, reducing our short-term debt repayment obligations from approximately $118 million to approximately $17 million. This should position us well to raise additional capital for our operations.' First Quarter and Recent Clinical Developments Recent developments regarding OMS527, our phosphodiesterase 7 ('PDE7') inhibitor program focused on addictions and compulsive disorders as well as movement disorders, include: Work on the planned randomized, double-blind, parallel-group, inpatient Phase 1b clinical trial comparing the safety and efficacy of OMS527 to placebo in the treatment of adults with cocaine use disorder ('CUD') is ongoing with committed funding from the National Institute on Drug Abuse, a part of the National Institutes of Health, in the amount of $4.02 million for the year commencing April 1, 2025. Enrollment in the study is expected to begin later this year and a readout of data from the study is anticipated late this year or in early 2026. Recent developments regarding our oncology platform comprising signaling-driven immunomodulators, oncotoxins, and an adoptive T-cell technology combined with an immunostimulator, include: In April 2025, we established the Omeros Oncology Clinical Steering Committee to advance Omeros' OncotoX biologics program focused on acute myeloid leukemia ('AML'). The clinical steering committee is composed of leaders in AML treatment and research at the premier cancer centers across the United States. These experts in the treatment of AML are expected to help guide clinical development of our potential AML therapeutic. We continue on a limited basis to progress pre-clinical studies within our novel oncology program, including IND-enabling studies in our OncotoX-AML program. In both in vivo and in vitro models with human cell lines, our OncotoX-AML therapeutic has consistently demonstrated superior efficacy to current AML standard of care treatments. OncotoX-AML shows broad application across AML regardless of genetic mutation including TP53, NPM1, KMT2a, and FLT3. IND-enabling work is ongoing with an estimated timeline to clinical entry of 18-24 months. Financial Results Net loss for the first quarter of 2025 was $33.5 million, or $0.58 per share, compared to a net loss of $37.2 million, or $0.63 per share for the first quarter of 2024. At March 31, 2025, we had $52.4 million of cash and short-term investments available for operations and debt service, a decrease of $37.7 million from December 31, 2024. For the first quarter of 2025, we earned OMIDRIA royalties of $6.7 million on Rayner's U.S. net sales of $22.3 million. This compares to earned OMIDRIA royalties of $9.4 million during the first quarter of 2024 on U.S. net sales of $31.2 million. Per the terms of our original 2022 and amended 2024 agreements with DRI Health Acquisition LP, ('DRI'), all U.S. based royalties through 2031 are remitted from Rayner to DRI through an escrow agent. Total operating expenses for the first quarter of 2025 were $35.0 million compared to $39.0 million for the first quarter of 2024. The $4.1 million decrease was primarily due to the wind down of our clinical program developing narsoplimab for IgA nephropathy offset by increased clinical development costs with Phase 2 of our zaltenibart program. Interest expense during the first quarter of 2025 was $3.7 million compared to $8.2 million during the prior year quarter. The decrease was due to repurchasing and retiring $118.1 million of par on our 2026 Notes in June 2024 and recording a non-cash remeasurement adjustment in the prior year to increase the OMIDRIA royalty obligation to reflect the sale of expanded royalties to DRI. During the first quarter of 2025, we earned $1.1 million in interest and other income compared to $3.4 million in the first quarter of 2024. The difference is primarily due to lower cash and investments available to invest in the current quarter. Net income from discontinued operations, net of tax, was $4.1 million, or $0.07 per share, in the first quarter of 2025 compared to $6.7 million, or $0.11 per share, in the first quarter of 2024. The decrease was primarily attributable to a decrease in OMIDRIA royalties earned in the current quarter. Conference Call Details Omeros' management will host a conference call and webcast to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. For online access to the live webcast of the conference call, go to Omeros' website at To access the live conference call via phone, participants must register at the following URL to receive a unique PIN. Once registered, you will have two options: (1) Dial in to the conference line provided at the registration site using the PIN provided to you, or (2) choose the 'Call Me' option, which will instantly dial the phone number you provide. Should you lose your PIN or registration confirmation email, simply re-register to receive a new PIN. A replay of the call will be made accessible online at 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. OMS906, Omeros' inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in clinical development 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 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 '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, plans and expectations regarding the conduct of clinical trials and the availability of data therefrom, 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 March 31, 2025. 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. OMEROS CORPORATION (In thousands) March 31, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 4,261 $ 3,400 Short-term investments 48,150 86,732 OMIDRIA contract royalty asset, short-term 29,227 29,083 Receivables 7,076 7,739 Prepaid expense and other assets 7,467 7,166 Total current assets 96,181 134,120 OMIDRIA contract royalty asset 121,560 124,266 Right of use assets 13,948 14,961 Property and equipment, net 2,418 2,678 Restricted investments 1,054 1,054 Total assets $ 235,161 $ 277,079 Liabilities and shareholders' deficit Current liabilities: Accounts payable $ 4,413 $ 5,905 Accrued expenses 24,752 26,005 OMIDRIA royalty obligation 21,072 20,645 Convertible senior notes, net 26,929 — Term debt — 21,000 Lease liabilities 6,056 5,971 Total current liabilities 83,222 79,526 OMIDRIA royalty obligation 190,091 195,612 Convertible senior notes, non-current, net 70,397 97,178 Long-term debt, net 88,562 69,405 Lease liabilities, non-current 11,941 13,466 Other accrued liabilities, non-current 4,501 4,501 Shareholders' deficit: Common stock and additional paid-in capital 730,252 727,736 Accumulated deficit (943,805 ) (910,345 ) Total shareholders' deficit (213,553 ) (182,609 ) Total liabilities and shareholders' deficit $ 235,161 $ 277,079 Expand
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
24-03-2025
- Business
- Yahoo
Omeros to activate site for Phase III programme of zaltenibart for PNH
Omeros has announced that the activation of clinical trial sites for patient enrolment is now in progress for its Phase III programme of the human monoclonal antibody zaltenibart for treating paroxysmal nocturnal haemoglobinuria (PNH). Zaltenibart, also known as OMS906, is an investigational mannan-binding lectin-associated serine protease-3 (MASP-3) inhibitor. The Phase III programme will involve 120 clinical investigative sites across 30 nations, with a significant number already having detected potential PNH patient participants. Omeros is actively working with these sites to further identify eligible subjects for the trials of the antibody. The data required for the biologics licencing application (BLA) and worldwide approval dossiers are underway for the fourth quarter of 2026. The Phase III trials are set to assess the antibody's intravenous form dosed one time for every eight-week period. Omeros' comprehensive Phase III programme comprises two clinical trials: one for subjects not on complement-inhibitor treatment at study entry and another for those without an adequate response to complement component 5 (C5) inhibitors, ravulizumab or eculizumab treatment. These two trials are designed to compare the antibody's safety and efficacy as a single agent against these C5 inhibitors, with regulatory approval received from both the Food and Drug Administration (FDA) and European authorities for the trial designs. Omeros has already manufactured the antibody drug product needed for the Phase III programme and has sourced the necessary comparator C5 inhibitors. The head-to-head study designs aim to show the antibody's 'superiority' over C5 inhibitors. Omeros Corporation CEO and chairman Gregory Demopulos said: 'All of us at Omeros are pleased that the Phase III clinical programme for zaltenibart is well underway. 'The zaltenibart Phase II data have demonstrated important differentiators from currently marketed agents, and we expect those same advantages to be evidenced in the Phase III trials, which are similar in design to our Phase II trials.' In a move to prepare for the commercialisation of the antibody, the German Federal Joint Committee, a decision-making body, provided recommendations on patient-reported-outcome measures. These recommendations have been 'incorporated' into the antibody's Phase III design, which could be instrumental in achieving appropriate pricing. "Omeros to activate site for Phase III programme of zaltenibart for PNH" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
