properdin

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients. The ODD provides NM5072 with a range of benefits, including financial incentives, regulatory support, extended market exclusivity, and extended patent life, ensuring that this groundbreaking treatment can reach those who need it. NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway (AP) while maintaining the integrity of the classical pathway required for fending off infections. The drug has completed phase I trial in 48 healthy volunteers with a clean safety profile and was well-tolerated across all doses tested up to 20 mg/kg. Total AP inhibition was achieved, with its duration exhibiting a dose-dependent response. NM5072 is being positioned to enter Phase II trial for the treatment of naïve PNH patients as the first clinical indication. The drug is expected to be useful in other complement-mediated and complement-associated disorders, further expanding the patient population for multiple clinical trials. April 15, 2024 -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. PNH is a rare, chronic, inflammatory, and hemolytic disease that occurs when PNH Red Blood Cells (RBCs) breakdown within and outside the blood vessel, causing anemia, which can be fatal. In addition to PNH RBCs, other cells, including Neutrophils, Monocytes, and Platelets, also drive cellular destruction and inflammation, leading to long-term effects on the patient’s health. If the disease remains untreated or partially treated, patients will continue to have anemia and inflammation, potentially leading to chronic pain, fatigue, and other common symptoms of PNH. Dr. Rekha Bansal, Chief Executive Officer said, “PNH is a rare disease which involves a range of blood cells that contribute to debilitating symptoms for patients, including anemia, fatigue, and severe pain with shorter life span if remains untreated. The current standard of care requires further improvements. We are hopeful that NM5072, with its unique mechanism of action that targets the top of the complement cascade, could become a promising treatment to improve outcomes in these patients.” NM5072 is under regulatory review for multiple indications in the USA and globally. “We are thrilled by the FDA’s decision, which highlights the demand for groundbreaking therapies for individuals grappling with PNH,” comments Robert Bard, VP Regulatory Affairs. This Orphan Drug Designation approval represents a remarkable stride in therapeutic innovation, showcasing NM5072’s distinct capability to selectively inhibit the alternative pathway (AP) while safeguarding the classical pathway essential for combating infections in PNH patients. NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States. The ODD from the FDA is a special designation granted to facilitate the development and assessment of new medicines. The FDA’s Office of Orphan Products Development has granted ODD for NM5072 which is in development for the treatment and prevention of rare diseases or conditions affecting fewer than 200,000 people in the US. This designation offers various advantages which include the potential for market exclusivity lasting seven years upon FDA approval, eligibility for tax credits related to qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance, and the potential qualification for expedited development programs. NM5072 (Anti-Properdin Monoclonal Antibody) NM5072 is being developed by NovelMed as a potential first-in-class monoclonal antibody that selectively blocks parts of the immune system responsible for the disease, thus inhibiting alternative pathway cascades. Blocking Properdin function is expected to block the lysis of PNH RBCs, which are implicated in the pathophysiology of anemia in PNH. NM5072 is currently an investigational drug that selectively binds to Properdin, a protein of the AP. This monoclonal antibody serves as an efficacious blocker of the AP without affecting the part of the immune system required for fighting infections. NM5072 has successfully completed a phase I trial in healthy volunteers with an expected safety profile. This potent drug is in various regulatory phases of development. With its much longer half-life, the Anti-Properdin molecule is a pioneering biologic. “While the drug is currently in the form of an IV formulation, subcutaneous formulations are being planned for future trials,” said Dr. Bansal. Regulatory preparations and review of Phase II trials for multiple indications, including PNH, C3 Glomerulopathy (C3G) and Atypical Hemolytic Uremic Syndrome (aHUS) are underway. Complement-Mediated Disorders The dysregulation of the Alternative Pathway (AP) is a pivotal factor influencing the trajectory of numerous acute and chronic rare diseases, spanning disciplines such as hematology, ophthalmology, nephrology, dermatology, and neurology. Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval. In the context of PNH, the current armamentarium of treatment options remains limited and imperfect, often compromising pathogenic clearance or inducing hyperlipidemia. PNH manifests with a spectrum of clinical features, including low PNH-RBC clone size, hemolysis, hemoglobinuria, reduced hemoglobin levels, elevated lactate dehydrogenase (LDH), and a plethora of other symptoms that can precipitate chronic complications, potentially leading to premature mortality if left untreated. Chronic symptoms and multisystem organ damage are recurrent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs provide potential for symptom management in PNH treatment, they should be approached with caution due to their significant side effects and broader limitations on labels. The imperative to develop additional treatment modalities for PNH, characterized by both efficacy and safety, remains unequivocally recognized, holding the promise of enhancing disease outcomes and improving the quality of life for affected individuals. Dedicated to pioneering innovative biologics for a range of complement-mediated diseases, NovelMed stands at the forefront of conceptualizing and validating an anti-Properdin antibody for chronic complement-mediated and complement-associated disorders. Progressing its leading product candidate through clinical trials, the company primarily focuses on addressing hemolytic and renal disorders initially. “Driven by a commitment to ingenuity and compassion, NovelMed is committed to delivering groundbreaking diagnostics and therapies that redefine the treatment landscape for patients with rare diseases,” said Dr. Rekha Bansal, Chief Executive Officer. Positioned as a frontrunner in the development of therapies targeting the Alternative Pathway (AP), NovelMed is devoted to addressing debilitating diseases across hematology, ophthalmology, Nephrology, dermatology, and neurology. Our R&D team is currently engaged in the development of a new diagnostic marker aimed at identifying complement-mediated diseases. This synergistic strategy is geared towards broadening the spectrum of diseases that can be effectively and confidently treated using NM5072. NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel NM5072 through further development and approval across multiple rare disease indications in the US and globally. For more information about NM5072, please visit www.NovelMed.com/news. The content above comes from the network. if any infringement, please contact us to modify.

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients. The ODD provides NM5072 with a range of benefits, including financial incentives, regulatory support, extended market exclusivity, and extended patent life, ensuring that this groundbreaking treatment can reach those who need it. NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway (AP) while maintaining the integrity of the classical pathway required for fending off infections. The drug has completed phase I trial in 48 healthy volunteers with a clean safety pro was well-tolerated across all doses tested up to 20 mg/kg. Total AP inhibition was achieved, with its duration exhibiting a dose-dependent response. NM5072 is being positioned to enter Phase II trial for the treatment of naïve PNH patients as the first clinical indication. The drug is expected to be useful in other complement-mediated and complement-associated disorders, further expanding the patient population for multiple clinical trials. CLEVELAND, April 15, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. PNH is a rare, chronic, inflammatory, and hemolytic disease that occurs when PNH Red Blood Cells (RBCs) breakdown within and outside the blood vessel, causing anemia, which can be fatal. In addition to PNH RBCs, other cells, including Neutrophils, Monocytes, and Platelets, also drive cellular destruction and inflammation, leading to long-term effects on the patient’s health. If the disease remains untreated or partially treated, patients will continue to have anemia and inflammation, potentially leading to chronic pain, fatigue, and other common symptoms of PNH. Dr. Rekha Bansal, Chief Executive Officer said, “PNH is a rare disease which involves a range of blood cells that contribute to debilitating symptoms for patients, including anemia, fatigue, and severe pain with shorter life span if remains untreated. The current standard of care requires further improvements. We are hopeful that NM5072, with its unique mechanism of action that targets the top of the complement cascade, could become a promising treatment to improve outcomes in these patients.” NM5072 is under regulatory review for multiple indications in the USA and globally. “We are thrilled by the FDA’s decision, which highlights the demand for groundbreaking therapies for individuals grappling with PNH,” comments Robert Bard, VP Regulatory Affairs. This Orphan Drug Designation approval represents a remarkable stride in therapeutic innovation, showcasing NM5072’s distinct capability to selectively inhibit the alternative pathway (AP) while safeguarding the classical pathway essential for combating infections in PNH patients. NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States. Orphan Drug Designation (ODD) The ODD from the FDA is a special designation granted to facilitate the development and assessment of new medicines. The FDA’s Office of Orphan Products Development has granted ODD for NM5072 which is in development for the treatment and prevention of rare diseases or conditions affecting fewer than 200,000 people in the US. This designation offers various advantages which include the potential for market exclusivity lasting seven years upon FDA approval, eligibility for tax credits related to qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance, and the potential qualification for expedited development programs. NM5072 (Anti-Properdin Monoclonal Antibody) NM5072 is being developed by NovelMed as a potential first-in-class monoclonal antibody that selectively blocks parts of the immune system responsible for the disease, thus inhibiting alternative pathway cascades. Blocking Properdin function is expected to block the lysis of PNH RBCs, which are implicated in the pathophysiology of anemia in PNH. NM5072 is currently an investigational drug that selectively binds to Properdin, a protein of the AP. This monoclonal antibody serves as an efficacious blocker of the AP without affecting the part of the immune system required for fighting infections. NM5072 has successfully completed a phase I trial in healthy volunteers with an expected safety profile. This potent drug is in various regulatory phases of development. With its much longer half-life, the Anti-Properdin molecule is a pioneering biologic. “While the drug is currently in the form of an IV formulation, subcutaneous formulations are being planned for future trials,” said Dr. Bansal. Regulatory preparations and review of Phase II trials for multiple indications, including PNH, C3 Glomerulopathy (C3G) and Atypical Hemolytic Uremic Syndrome (aHUS) are underway. Complement-Mediated Disorders The dysregulation of the Alternative Pathway (AP) is a pivotal factor influencing the trajectory of numerous acute and chronic rare diseases, spanning disciplines such as hematology, ophthalmology, nephrology, dermatology, and neurology. Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval. In the context of PNH, the current armamentarium of treatment options remains limited and imperfect, often compromising pathogenic clearance or inducing hyperlipidemia. PNH manifests with a spectrum of clinical features, including low PNH-RBC clone size, hemolysis, hemoglobinuria, reduced hemoglobin levels, elevated lactate dehydrogenase (LDH), and a plethora of other symptoms that can precipitate chronic complications, potentially leading to premature mortality if left untreated. Chronic symptoms and multisystem organ damage are recurrent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs provide potential for symptom management in PNH treatment, they should be approached with caution due to their significant side effects and broader limitations on labels. The imperative to develop additional treatment modalities for PNH, characterized by both efficacy and safety, remains unequivocally recognized, holding the promise of enhancing disease outcomes and improving the quality of life for affected individuals. About NovelMed Dedicated to pioneering innovative biologics for a range of complement-mediated diseases, NovelMed stands at the forefront of conceptualizing and validating an anti-Properdin antibody for chronic complement-mediated and complement-associated disorders. Progressing its leading product candidate through clinical trials, the company primarily focuses on addressing hemolytic and renal disorders initially. “Driven by a commitment to ingenuity and compassion, NovelMed is committed to delivering groundbreaking diagnostics and therapies that redefine the treatment landscape for patients with rare diseases,” said Dr. Rekha Bansal, Chief Executive Officer. Positioned as a frontrunner in the development of therapies targeting the Alternative Pathway (AP), NovelMed is devoted to addressing debilitating diseases across hematology, ophthalmology, Nephrology, dermatology, and neurology. Our R&D team is currently engaged in the development of a new diagnostic marker aimed at identifying complement-mediated diseases. This synergistic strategy is geared towards broadening the spectrum of diseases that can be effectively and confidently treated using NM5072. Seeking Partnership to Advance the Development of Drug candidates for Rare Diseases - NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel NM5072 through further development and approval across multiple rare disease indications in the US and globally. For more information about NM5072, please visit . Contact: Ya Gao BD@novelmed.com (216) 440-2696 The expected efficacy of the NM5072 treatment A photo accompanying this announcement is available at NM5072, anti-Properdin antibody, for treating PNH and other rare diseases By blocking Properdin function, NM5072 is expected to block the PNH RBC lysis, which is implicated in the pathophysiology of anemia in PNH.

Phase I Clinical Trial in healthy subjects shows that NM3086 was safe and well tolerated at all doses (0.1 to 20 mg/kg) in six cohorts. Total AP inhibition was achieved through all cohorts and the duration of alternative pathway (AP) inhibition occurred in a dose-dependent manner. Correspondingly, the classical pathway (CP) remained unaffected, suggesting that NM3086 selectively blocks the AP while maintaining the host defense. NM3086 blocked the deposition of C3b via AP, a marker of extravascular hemolysis (EVH). The duration of AP-C3b inhibition followed the AP inhibition profile very closely in a dose dependent manner. NM3086 did not block the deposition of C3b via the CP, a marker of opsonization. There was no suppression of CP driven C3b activity across all cohorts. Results suggest that NM3086 has the potential to selectively inhibit both intravascular hemolysis (IVH) and EVH via the AP while maintaining the CP required for bacterial clearance and opsonization in Paroxysmal Nocturnal Hemoglobinuria (PNH) and other hemolytic disorders. CLEVELAND, June 05, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. announced today topline results from First-in-Human Phase I clinical trial of its complement blocker monoclonal anti-Properdin antibody, known as NM3086. The trial was a randomized, double-blind, placebo-controlled, single ascending dose study designed to assess safety and tolerability, pharmacokinetics (PK) and pharmacodynamics (PD), and immunogenicity (ADA) of NM3086 in healthy volunteers. NM3086 was administered as a single dose (ranging from 0.1 to 20 mg/kg) and the study was followed for up to 90 days. The drug was safe and well tolerated in all doses. Assays of the alternative pathway (AP), classical pathway (CP), extravascular hemolysis (EVH), and opsonization were performed. AP activation, AP-driven C3b formation and deposition were inhibited in a dose dependent manner with the highest dose of 20 mg/kg blocking AP for more than 6 weeks. Sustained targeted inhibition of the AP, without blocking the CP, is a critical aspect of NM3086 that distinguishes itself from other FDA approved anti-complement drugs. These results from Phase I trial are consistent with our in vitro studies conducted on PNH serum and in naïve and Soliris-Treated PNH sera. Early success in Phase I studies marks the first clinical milestone towards the development of NM3086 as a safe AP blocker for anemia in PNH and other complement-mediated disorders. NM3086 selectively binds the critical protein ‘properdin” of the AP responsible in part for amplification of the AP, a major advantage of NM3086 over other FDA approved complement blockers. “This is the first clinical study to demonstrate that selective blockade of the AP can be safely achieved by neutralizing Properdin, a critical protein in the AP cascade,” said Rekha Bansal, Ph.D., Chief Executive Officer of NovelMed. Because NM3086 selectively blocks AP-mediated C3b and MAC formation, it is therefore expected to treat both extra- and intra- vascular hemolysis in PNH patients. NM3086 blocks the formation of C3a/C3b, C5a/C5b, and MAC which are responsible for overall pathology of complement-mediated disorders including PNH. “Our drug, NM3086, is expected to treat disorders mediated via (Factors B, D and P), C3a, C3aR, C5a, C5aR, and MAC. Instead of targeting each one individually, our molecule would target all of them by blocking the initiation step of the AP,” says Dr. Bansal. NovelMed is moving this molecule in multiple indications as a result. “We continue to be impressed by the data from in vitro and clinical trial studies, which confirm the potential of NM3086 to outperform other drug candidates in the complement space,” says Robert Bard, VP Regulatory Affairs. About Paroxysmal Nocturnal Hemoglobinuria PNH is a rare blood disorder in which hemolysis and thrombosis, accompanied by impaired bone marrow function, cause debilitating symptoms that adversely affect patient quality of life and can lead to early death. PNH is a rare, chronic, and serious complement-mediated blood disorder with a significant unmet need despite treatment with anti-C5 therapies, as a large proportion of people with PNH remain anemic, fatigued, and dependent on blood transfusions. About NM3086 NM3086 is an anti-P humanized monoclonal antibody that binds properdin with high affinity. NM3086 blocks both C3b and membrane-attack complex (MAC) formation, thereby preventing lysis of red blood cells via extra- and intra-vascular hemolysis in PNH. Treatment with NM3086 also blocks the production of a) anaphylatoxins C3a and C5a, b) C3 and C5 convertases, c) C3b, d) MAC, and e) LDH release. This targeted approach collectively and broadly applies to numerous complement-mediated disorders. NM3086 is suitable for use in multiple disease indications e.g., complement-mediated hemolytic disorders, renal disorders, ocular disorders, neurological disorders, dermatological disorders, and inflammation. Currently available complement inhibitors indiscriminately block both the alternative and the classical complement pathways, where in the latter is required for clearing infectious microorganisms. NM3086 is expected to control hemolysis, C3b deposition, and LDH release, all while sustaining patient host defense against infections. Given its unique mechanism of action, NM3086 is expected to function as a superior therapeutic to currently FDA approved treatment options possibly without the Black-Box warning. About NovelMed NovelMed is a clinical-stage biopharmaceutical company focused on the development of novel biologics for the treatment of a broad range of complement-mediated diseases. NovelMed is the first company to have invented and validated an anti-Properdin antibody to treat chronic complement-mediated and complement-associated disorders. NovelMed is currently evaluating its lead product candidate in clinical trials, with PNH as the first indication. NovelMed is committed to leveraging creativity and compassion to deliver game-changing therapies to patients. NovelMed is positioning itself as a leader in developing AP-targeted therapies to resolve debilitating diseases in the areas of hematology, ophthalmology, nephrology, dermatology, and neurology. For more information, please visit www.NovelMed.com. Seeking Funds to Advance the Development of NM3086 for Rare Disease Platform PNH is a rare disease characterized by intravascular and extravascular hemolysis, higher LDH values, and lower hemoglobin values. In PNH patients currently treated with C5 blockers, approximately 36% of these patients still require more than one transfusion per year. In addition, almost all patients were found to be positive for opsonization markers while nearly 70% continued to have low hemoglobin levels despite treatment. NM3086 on the other hand is expected to fully control AP related disease progression in PNH patients. NovelMed is currently seeking licensing, partnership, and acquisition opportunities to drive NM3086 through further development and approval in multiple rare disease indications. For more information, please visit www.NovelMed.com/news Contact: Robert BardNovelMed Business Development Teambd@novelmed.com(216) 440 2696