NovelMed Therapeutics, Inc.

-- The United States FDA Clears Initiation of Efficacy Trial in Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV) Patients APhase II, Efficacy Trial in adult AAV patients has been approved by the FDA. Ruxoprubart is a first-in-class treatment for sustaining remission in AAV patients as a conjunctive therapy along with Standard of Care (SOC) which includes corticosteroids plus Cyclophosphamide/Azathioprine or Rituximab. Ruxoprubart demonstrated an anticipated safety pro Phase I trial in healthy subjects in 2022. TAVNEOS® (avacopan) was approved by the FDA in 2022 for treating AAV. Ruxoprubart is being positioned as the next-generation AAV treatment based on its mechanism of action. NovelMed’s Ruxoprubart drug blocks the alternative pathway (AP) without blocking the complement classical pathway (CP), an important advantage in maintaining a healthy lifestyle. Recent commencement of Ruxoprubart proof-of-concept Phase II trial through intravenous (IV) administration in naïve PNH patients shows promising results with a clean safety and efficacy profile. The Subcutaneous (SC) route of administration is under regulatory review and is expected to start soon. Ruxoprubart is a multi-indication platform. Given the role of the alternative pathway in rare diseases, the antibody drug has the potential to treat multiple diseases, including ocular, inflammatory, renal, neurological, and hematological. CLEVELAND, June 17, 2024 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. proudly announces the U.S. Food and Drug Administration's (FDA) clearance on Ruxoprubart, the investigational drug, for commencing an efficacy trial targeting patients with ANCA Associated Vasculitis (AAV), a rare autoimmune chronic ailment characterized by inflammation in small blood vessels. Currently, the standard of care (SOC) for AAV requires high doses of Glucocorticoids, along with prolonged administration of other immunosuppressive drugs. However, this approach comes with severe side effects, including general immune suppression and toxicity from glucocorticoids, leaving unmet needs in managing the disease's impact on vital organs and associated adverse events (AEs). Results from Phase I and II trials of Ruxoprubart in PNH patients demonstrate the drug's selective blocking of the alternative pathway (AP) without affecting the classical pathway (CP), which is crucial for maintaining immunity. This selectivity presents a significant advantage over existing complement blockers approved by the FDA or under development, potentially eliminating the need for a Black Box Warning. In a forthcoming twelve-patient AAV study, Ruxoprubart will be evaluated alongside the SOC, akin to the trial conducted for TAVNEOS. Ruxoprubart is anticipated to outperform TAVNEOS due to its comprehensive control over the pathway responsible for generating three major toxins contributing to the disease pathology: C3a, C5a, and MAC. While TAVNEOS only targets C5a, Ruxoprubart aims to neutralize two powerful toxins including C3a and C5a, offering a potentially superior treatment approach. "We are thrilled to have gained FDA approval to address AAV in patients with an inadequate response to standard of care. Through this study, we aim to compare Ruxoprubart with historical data on TAVNEOS, hoping to improve AAV patients' lives substantially" says Dr. Rekha Bansal, Chief Executive Officer of NovelMed. Joseph Jankowski, Ph.D., VP, Business Development of NovelMed, expressed enthusiasm regarding the FDA’s clearance, stating, "The FDA's clearance of the Phase II clinical trial for AAV marks a significant milestone for Ruxoprubart, which harbors potential as a versatile platform for multiple indications." Ruxoprubart is positioned to compete with TAVNEOS with its potential for a superior safety and efficacy profile. Acquisition of TAVNEOS by Amgen for 3.7 Billion dollars underscores the value placed on treatments for AAV patients. Ruxoprubart is expected to be a next-generation product that can replace TAVNEOS. Ruxoprubart's selectivity and specificity, based on the clinical data from ongoing trials, hold promise not only for AAV, but also for several other renal disorders, including aHUS, and IgAN. The drug's potential to offer a superior safety and efficacy pro it apart and places it in a strong position within the market. The alternative pathway (AP) plays a pivotal role in managing anemia, inflammation, and tissue damage in various complement-mediated disorders, including AAV. NovelMed is committed to advancing the development of Ruxoprubart and addressing the unmet medical needs of patients with AAV and other complement-mediated disorders. The FDA's clearance of the Phase II trial represents a significant step forward in our mission to bring innovative and effective treatments to the market. We are actively exploring licensing, partnership, and acquisition opportunities to advance Ruxoprubart's development and approval across multiple rare disease indications. For further details, please visit . About ANCA Associated Vasculitis (AAV) Anti-Neutrophil Cytoplasmic Antibody-associated vasculitis (ANCA Vasculitis or AAV), is a rare autoimmune condition causing inflammation in blood vessels, often leading to swelling, narrowing, and sometimes blockage of blood vessels. It is characterized by the presence of Anti-Neutrophil Cytoplasmic Antibodies (ANCAs), targeting certain proteins within white blood cells, particularly neutrophils. It often causes a specific type of inflammation called crescentic glomerulonephritis, which can lead to kidney failure. Diagnosis of AAV involves a comprehensive assessment of medical history, physical examination, specific laboratory tests, imaging studies, and sometimes tissue biopsies. The current treatment approach primarily focuses on immune system suppression to alleviate inflammation and prevent further organ damage, utilizing corticosteroids and immunosuppressants. Despite being a rare and complex disease, advancements in treatment strategies are a must for continued improvement in the outcome of AAV patients. The prevalence of AAV in the United States is approximately 66,600 to 133,200 patients. TAVNEOS costs $75,051 annually per patient, with the potential to generate over 4 Billion in annual sales. About Ruxoprubart Ruxoprubart is a high affinity, humanized monoclonal antibody to Bb, selectively blocking the initiation and propagation of the alternative pathway (AP). By targeting the AP and sparing the CP pathway, Ruxoprubart holds promise for treating renal disorders, including ANCA vasculitis (AAV). Furthermore, Ruxoprubart shows therapeutic potential across a broad spectrum of rare diseases and disorders, including but not limited to hematological (PNH), renal (aHUS, IgAN), neurological (NMOSD), and ocular (GA and Uveitis). Clinical trials for Ruxoprubart can be found at , showing its potential to address various complement-mediated and associated illnesses where treatment options are inadequate or absent. About NovelMed NovelMed is a clinical-stage biopharmaceutical company dedicated to developing antibody treatments for numerous complement-mediated disorders, particularly rare (orphan) diseases. We aim to innovate and develop novel biologics to address unmet needs in rare disease treatment. NovelMed boasts a robust portfolio of intellectual property with broad applications in rare and common diseases. This includes Ruxoprubart's potential to treat a range of complement-mediated disorders associated with uncontrolled AP activation. As part of its antibody platform, NovelMed also offers humanized antibodies to C3b and Factor P (Properdin), effectively targeting the AP. NovelMed actively seeks licensing, partnership, and acquisition opportunities to advance antibody development through Phase III and approval in multiple rare disease indications. For more information, please visit News (novelmed.com). Ya Gao Email: bd@novelmed.com Tel: (216) 440 2696 Follow NovelMed on LinkedIn

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.

Breakdown of PNH RBCs within and outside the blood vessels. Credit: Phonlamai Photo / Shutterstock.com. NovelMed has announced the receipt of orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug, NM5072, for treating paroxysmal nocturnal hemoglobinuria (PNH). An alternative pathway blocker anti-properdin antibody, NM5072 selectively hinders the immune system components responsible for the disease, inhibiting alternative pathway cascades and preventing the lysis of PNH red blood cells (RBCs). By blocking the function of properdin, NM5072 prevents the breakdown of PNH RBCs, which is central to the anaemia associated with PNH. The drug has concluded a Phase I clinical trial in 48 healthy volunteers with a good safety profile. It is currently being reviewed for multiple indications by regulators in the US and internationally. See Also: Qureight secures Series A funding to accelerate drug development FDA halts Neumora’s schizophrenia drug trial over convulsions in rabbits PNH is a rare disease characterised by chronic inflammation and haemolysis, in which PNH RBCs break down within and outside blood vessels, leading to anaemia and severe health implications. NM5072, with its longer half-life, represents a significant advancement in the biologic treatment of PNH to improve patient outcomes. NovelMed CEO Dr Rekha Bansal stated: “PNH is a rare disease that involves a range of blood cells that contribute to debilitating symptoms for patients, including anaemia, fatigue and severe pain with a shorter life span if it remains untreated. “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.” The FDA’s ODD provides benefits including seven years of market exclusivity upon the receipt of approval, tax credits for clinical trial costs, fee waivers, reduced annual product fees, assistance for clinical protocol development and the possibility of expedited development programmes.