NovelMed Therapeutics, Inc.

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH. Ruxoprubart is an Anti-Bb antibody that selectively blocks the alternative pathway while maintaining the intactness of the classical pathway required for clearing infection. The drug has completed phase I trial in 40 healthy volunteers with a clean safety pro was well-tolerated across all doses. Total alternative pathway inhibition was achieved, with the duration of AP inhibition showing a dose-dependent response. Ruxoprubart is currently undergoing a Phase II trial in treatment naïve PNH patients, showing promising results. Ruxoprubart is potentially useful in other clinical indications, where the complement alternative pathway is dysregulated. The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV). The ODD designation provides Ruxoprubart with a range of benefits, including financial incentives, regulatory support, and extended market exclusivity, ensuring that this groundbreaking treatment can reach those who need it. CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a Phase II trial in treatment-naïve PNH patients, which aims to evaluate the safety and efficacy of the drug and bringing us one step closer to providing an effective and innovative therapy for PNH patients in USA and around the world. "We are delighted by the FDA's decision to grant orphan drug designation to Ruxoprubart, underscoring the pressing need for innovative therapeutic solutions for patients with PNH," says Robert Bard, VP Regulatory Affairs. This ODD approval marks a significant advancement in the therapeutic landscape with Ruxoprubart 's unique ability to selectively block the alternative pathway (AP) while preserving the classical pathway required for clearing infections in PNH patients. Orphan Drug Designation (ODD) The ODD from the US FDA is a special designation granted to facilitate the development and assessment of prospective new medications. The FDA's Office of Orphan Products Development bestows orphan status upon drugs in development for the treatment, diagnosis, or prevention of rare diseases or conditions impacting fewer than 200,000 people in the US. This Designation aims to offer various advantages to drug developers in order to bolster the creation of innovative medications. These advantages 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. Ruxoprubart (NM8074) Distinguishing itself from Iptacopan (Fabhalta) by exhibiting no affinity for Factor B, our lead candidate Ruxoprubart selectively binds to protein Bb of the alternative pathway. This humanized anti-Bb monoclonal antibody serves as a potent blocker of the Alternative Pathway. Having successfully completed the phase I trial in healthy volunteers with expected outcomes, Ruxoprubart has progressed to a phase II trial, focusing on treatment-naïve PNH patients in a multi-dose regimen. Notably, the Anti-Bb molecule marks a pioneering biologic undergoing testing in treatment-naïve PNH patients. FDA has also approved the initiation of Phase II trials for multiple indications, including C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and ANCA vasculitis (AAV) with trials to be conducted in the United States. Overseas regulatory approvals cover trials for PNH for the adult population and aHUS, extending to adult and pediatric population. Paroxysmal Nocturnal Hemoglobinuria (PNH) The dysregulation of the Alternative Pathway (AP) is widely recognized as a pivotal factor influencing the course of PNH disease. Classified as an orphan disease, current PNH treatment options remain limited and imperfect. Current sanctioned treatments either hinder infection clearance or induce hyperlipidemia. PNH is characterized by low PNH-RBC clone size, hemolysis, hemoglobinuria, low hemoglobin levels, elevated LDH, and numerous other symptoms that can lead to chronic issues, including premature death if left untreated. Chronic symptoms and multiple organ damage are also prevalent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs exist for PNH treatment with potential symptom management, they are not without significant side effects. The imperative for additional treatment options for PNH, both effective and well-tolerated, remains recognized, with the potential to improve the disease outcome. About NovelMed NovelMed is dedicated to pioneering novel biologics for diverse complement-mediated diseases. As the first to conceptualize and validate an anti-Bb antibody for chronic complement-mediated and complement-associated disorders, NovelMed advances its leading product candidate through clinical trials, primarily focusing on hemolytic and renal disorders. "Rooted in a commitment to creativity and compassion, NovelMed aims to deliver transformative therapies reshaping the treatment landscape for rare disease patients," says Dr. Rekha Bansal, Chief Executive Officer. Positioned as a leader in developing Alternative Pathway (AP)-targeted therapies, NovelMed is dedicated to tackling debilitating diseases in hematology, ophthalmology, nephrology, dermatology, and neurology. Seeking Partnership to Advance the Development of Ruxoprubart for Rare Diseases PNH, a hemolytic disorder resulting in a low PNH-RBC clone size, increased RBC transfusions, elevated LDH values, and decreased hemoglobin values, remains an orphan disease with limited treatment options. NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel Ruxoprubart through further development and approval across multiple rare disease indications in various countries. For additional information, please visit . Contact: Ya Gao BD@novelmed.com (216) 440-2696

The USAN Council has officially adopted "Ruxoprubart (Ruk”soe proo' bart)" as the generic name for NM8074, marking a significant milestone in the drug development process.Ruxoprubart is an investigational drug that targets the complement protein Bb and blocks the Alternative Pathway.Phase I Clinical Trial results in healthy subjects demonstrated the safety and well-tolerance of NM8074. Across all cohorts, total Alternative Pathway (AP) inhibition was achieved, with the duration of AP inhibition showing a dose-dependent response.Ongoing Phase II Clinical Trial reveals a safe and well-tolerated biweekly multidose regimen in PNH patients. Six treatment-naïve PNH patients have been dosed thus far with promising results.Preliminary findings from the ongoing Phase II Trial suggest that Ruxoprubart holds promise in increasing PNH clone size and reducing the need for pRBC transfusions by blocking the Alternative Pathway.Both classical pathway and lipid profiles in the ongoing clinical trial appear to be normal. The trial aims to enroll a total of 12 treatment-naïve PNH patients, further advancing our understanding of Ruxoprubart's potential in complement-mediated diseases. CLEVELAND, Ohio, Jan. 08, 2024 (GLOBE NEWSWIRE) -- NovelMed, a clinical-stage biopharmaceutical company with expertise in inflammatory and complement-related diseases, is pleased to announce that the United States Adopted Name (USAN) Council has officially assigned the generic name "Ruxoprubart" to its monoclonal antibody candidate, NM8074. Currently in Phase II trial for Paroxysmal Nocturnal Hemoglobinuria (PNH), a hemolytic disease, Ruxoprubart (NM8074) represents a significant breakthrough. The USAN Council has approved the nonproprietary name "Ruxoprubart" (pronounced Ruk” soe proo’ bart) for NM8074, designating it as a first-in-class Alternative Pathway selective investigational monoclonal antibody. Administered through the USAN Program, a renowned organization for assigning distinct names to identify pharmaceutically active substances, the name Ruxoprubart reflects a novel nomenclature system for monoclonal antibodies, rooted in antibody type and mechanism of action. Dr. Rekha Bansal, Chief Executive Officer of NovelMed, expressed, "The assignment of the nonproprietary name to NM8074 represents a significant milestone as we advance this potential PNH treatment towards study completion and regulatory filings. This allows us to establish a well-recognized name for future publications, labeling, and marketing materials." Committed to scientific exploration, NovelMed continues to explore how its leading drug could address unmet needs in the field of PNH. Ruxoprubart, a potent and highly selective investigational drug candidate, targets the complement protein Bb, playing a crucial role in benefiting several complement-mediated diseases. About Ruxoprubart The USAN Council has officially designated Ruxoprubart as the generic name for NM8074. Distinguishing itself by exhibiting no affinity for Factor B, Ruxoprubart selectively binds to protein Bb. This humanized anti-Bb monoclonal antibody serves as a potent inhibitor of the Alternative Pathway. Having successfully concluded the phase I trial in healthy volunteers, Ruxoprubart has progressed to phase II trials, focusing on treatment-naïve PNH patients in a multi-dose regimen. Notably, the Anti-Bb molecule marks a pioneering biologic undergoing testing in treatment-naïve PNH patients. FDA has also approved the initiation of Phase II trials for multiple indications, including C3 Glomerulopathy (C3G) and Atypical Hemolytic Uremic Syndrome (aHUS), with trials scheduled in the United States. Overseas approvals cover trials for PNH within the adult population and aHUS, extending to a pediatric population. About Paroxysmal Nocturnal Hemoglobinuria (PNH) The dysregulation of the Alternative Pathway (AP) is widely recognized as a pivotal factor influencing the course of PNH. Classified as an orphan disease, PNH presents limited and imperfect treatment options. Current sanctioned treatments either hinder infection clearance or induce hyperlipidemia. PNH is characterized by low PNH-RBC clone size, hemolysis, hemoglobinuria, low hemoglobin levels, elevated LDH, and numerous other symptoms that can lead to premature death if left untreated. Chronic symptoms and multiple organ damage are also prevalent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs exist for PNH treatment with potential symptom management, they are not without significant side effects. The imperative for additional treatment options for PNH, both effective and well-tolerated, remains recognized, with the potential to improve the disease outcome. About NovelMed NovelMed is dedicated to pioneering novel biologics for diverse complement-mediated diseases. As the first to conceptualize and validate an anti-Bb antibody for chronic complement-mediated and complement-associated disorders, NovelMed advances its leading product candidate through clinical trials, primarily focusing on hemolytic and renal disorders. “Rooted in a commitment to creativity and compassion, NovelMed aims to deliver transformative therapies reshaping the treatment landscape for patients,” says Mr. Robert Bard, VP of Regulatory Affairs. Positioned as a leader in developing Alternative Pathway (AP)-targeted therapies, NovelMed is dedicated to tackling debilitating diseases in hematology, ophthalmology, nephrology, dermatology, and neurology. Seeking Partnership to Advance the Development of Ruxoprubart for Rare Diseases PNH, a hemolytic disorder resulting in a low PNH-RBC clone size, increased RBC transfusions, elevated LDH values, and decreased hemoglobin values, remains an orphan disease with limited treatment options. NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel Ruxoprubart through further development and approval across multiple rare disease indications in various countries. For additional information, please visit www.NovelMed.com/news. Contact:Robert Bardrobert@novelmed.com(216) 440-2696

The Phase I healthy trial revealed that NM8074 effectively blocked the alternative pathway (AP) and AP-mediated C3b deposition without affecting the classical pathway (CP). Importantly, NM8074 did not interfere with the deposition of C3b via the CP, a crucial marker of opsonization, across all cohorts in the Phase I trial.Regulatory approval for the Phase II trial in treatment-naïve PNH patients was granted based on the safety and tolerance demonstrated in the previous Phase I clinical trial involving healthy subjects. NM8074 proved to be safe and well-tolerated across various doses, ranging from 0.3 to 20 mg/kg.Subsequent dosing of NM8074 in treatment-naïve PNH patients yielded promising results. Notably, NM8074 led to significant reductions in Lactate Dehydrogenase (LDH), Reticulocytes, Free Hemoglobin, and D-dimer levels, while simultaneously increasing Hemoglobin and Haptoglobin levels.These results collectively suggest that NM8074 has the potential to selectively inhibit both intravascular hemolysis (IVH) and extravascular hemolysis (EVH) through the AP, while preserving the CP necessary for bacterial clearance and opsonization in treatment-naïve PNH patients. CLEVELAND, Oct. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics is excited to announce the commencement of its Phase II clinical trial targeting treatment-naïve Paroxysmal Nocturnal Hemoglobinuria (PNH) patients with its groundbreaking anti-Bb antibody, NM8074. This open-label, multi-dose, and multi-center study aims to assess the safety and efficacy of NM8074 in PNH patient population. To date, eight PNH patients have been enrolled in the trial, with two patients already receiving NM8074 treatment. Key efficacy parameters include increases in Hemoglobin, increase in RBC clone size, decreased Lactate Dehydrogenase (LDH) levels, reduced pRBC transfusions, and reduced D-Dimer levels. Positive outcomes on these measures will pave the way for NM8074 as a highly differentiated and innovative biologic for treatment-naïve PNH patients. The Phase II study is divided into two cohorts, featuring a biweekly dosing schedule spanning a 3-month assessment period. Notably, the preliminary data indicates that NM8074 demonstrates a favorable safety profile, with no safety concerns. NM8074 was administered to treatment-naïve PNH patients with a clone size exceeding 10%. The results demonstrate significant reductions in LDH, Reticulocytes, Free Hemoglobin, and D-dimer, along with increases in Hemoglobin and Haptoglobin levels as expected. Furthermore, the clone size increased in a time-dependent manner. In summary, these observed changes in efficacy parameters validate the drug's mechanism of action and substantiate NM8074 as an effective PNH treatment option. Dr. Rekha Bansal, CEO of NovelMed Therapeutics, commented, "Preliminary data from the Phase II clinical trial in treatment-naïve PNH patients confirms NM8074's potential to comprehensively address intravascular and extravascular hemolysis-mediated anemia and bolster patient host defense systems, thus reducing the risk of infections." Previously, a Phase I trial involving forty healthy volunteers was successfully completed without any safety concerns. NM8074 was well tolerated across all doses, ranging from 0.3 to 20 mg/kg. These results corroborate NM8074's complete inhibition of the alternative pathway (AP) while preserving the classical pathway (CP), aligning with data generated by NovelMed's expert scientists. This selective mechanism distinguishes NM8074 from other complement blockers currently approved or in development by the FDA. About Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare blood disorder characterized by the immune system's (Complement) attack on and destruction of PNH Red Blood Cells (RBC hemolysis). This leads to a decrease in hematocrit and hemoglobin levels, elevated LDH and D-Dimer, and an increased need for blood transfusions. PNH is a rare, chronic, and serious complement-mediated blood disorder with significant unmet needs. Despite the use of anti-C3 and anti-C5 therapies, many PNH patients continue to experience anemia, fatigue, and dependence on blood transfusions. About NM8074 NM8074 is a monoclonal antibody with dual specificity, differentiating it from other therapies such as Iptacopan, which binds to Factor B. NM8074 targets Bb and selectively blocks the alternative pathway (AP). This high-affinity monoclonal antibody inhibits C3b formation and deposition and membrane-attack complex (MAC) formation, offering a significant advantage in preventing the total lysis of red blood cells in PNH. Furthermore, NM8074 is being evaluated for other clinical indications, including atypical Hemolytic Uremic Syndrome (aHUS), for which it has received regulatory approval. NM8074's potential extends to complement-mediated pathologies in renal disorders, ocular disorders, neurological disorders, dermatological disorders, and inflammation, illustrating its broad application across multiple diseases. Dr. Rekha Bansal, Chief Executive Officer, emphasized, "This wide application to multiple diseases underscores our commitment to serving diverse patient populations." In contrast to other complement blockers from third parties, which indiscriminately block both the alternative and classical complement pathways, NM8074 offers a unique mechanism of action. It is expected to function as a superior therapeutic option, potentially without the Black-Box warning associated with currently FDA-approved treatments. About NovelMed Therapeutics NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative biologics for the treatment of a wide range of complement-mediated diseases. NovelMed proudly leads in the development of anti-Bb antibodies to address chronic complement-mediated and complement-associated disorders. The company is actively conducting clinical trials in treatment-naïve PNH patients to expedite the availability of this promising treatment. Moreover, NovelMed is committed to delivering life-changing therapies to patients in the fields of hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is Seeking Opportunities to Advance NM8074 for Rare Diseases. For more information, please visit https://www.novelmed.com/news Contact: Rekha BansalNovelMed Business Development Teambd@novelmed.com(216) 440 2696