NM-8070

---Single therapy for multiple forms of Age-Related Macular Degeneration (AMD) NM3086 is a highly potent Alternative Pathway (AP) blocker that does not affect the Classical Pathway (CP).Dysfunction of the Complement Alternative Pathway is a critical factor in disease onset and progression in Wet-AMD, Dry-AMD, and Geographic Atrophy (GA).The lead drug candidate NM3086 prevented hemorrhage, fibrosis, and neovascularization in a 28-day disease model in rhesus monkeys.The specificity and functional ability of NM3086 are an upgrade over other drugs approved by the FDA for the treatment of GA.NovelMed is seeking to license the Anti-Properdin program to advance the development of NM3086 for Wet-AMA, Dry-AMD, GA, and rare ocular diseases. CLEVELAND, March 06, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics announced today that NM3086, the lead clinical asset in its Properdin-associated Alternative Pathway (AP) program, demonstrated favorable efficacy in an animal model of neovascular age-related macular degeneration (AMD) in rhesus monkeys. This animal model is characterized not only by the abnormal growth of new blood vessels behind the retina, as shown in Figure 1, but also by retinal fibrosis and vascular hemorrhage, which are the critical characteristics of Wet-AMD, Dry-AMD, and GA. While it is difficult to extrapolate the visual acuity score in animals, the damage prevented by NM3086 in this model suggests the drug's efficacy in treating the disease. Figure 1: Fluoresceine angiogram data from three animals treated with NM3086 (bottom) and three that received placebo control. The picture shows reduced hemorrhage and neovascularization (Left) and Fibrosis (Right) in the drug-treated animals. Scientists of Charles River Laboratories established a preclinical Choroidal Neovascularization (CNV) efficacy model in rhesus monkeys. This study administered NM3086 (1.2 mg/eye ) intravitreally to monkeys before creating nine laser-induced retinal lesions. Following a two-week observation period, choroidal neovascularization (formation of new blood vessels), retinal fibrosis (scar tissue), and hemorrhage (vascular leakage) were quantified against a placebo control. NM3086 resulted in a significant decrease in the extent of all three damage markers. "This All-in-One approach in ophthalmic space is a significant achievement for treating multiple ocular chronic ailments with a single therapy," states Dr. Bansal of NovelMed Therapeutics. About Complement-Mediated Ocular DisordersAMD is of two main types: "Wet" and "Dry." In the Wet form, abnormal blood vessels (choroidal neovascularization or CNV) grow under the retina and macula. These new blood vessels then bleed or leak fluid which causes the macula to bulge and distort central vision, resulting in rapid and severe vision loss. Approximately 10% of the total AMD cases have new blood vessels and leakage interactions, categorized as Wet-AMD. The Dry form (non-neovascular AMD) does not involve fluids from blood vessels; cases of this form are known as geographic atrophy (GA). GA is a chronic, progressive deterioration of the macula that affects ~1.45 million people in the United States and over 8 million people worldwide. The incidence of GA is expected to rise as the age-related disease burden of developed countries increases. The AP activation is essential to AMD pathogenesis of both types. It is well-accepted that excessive activation of the AP is a significant cause of the illness in both Wet- and Dry-AMD, including GA. Preventing tissue damage and excessive inflammation requires complete shutdown of the Alternative Pathway. A therapy that can prevent excessive activation of the AP would prove to be a blockbuster treatment for Wet AMD, Dry AMD, and GA. About NM3086NM3086 selectively binds Properdin and blocks the AP activation. As a result, the formation of C3 and C5 convertases is blocked. In general, the C3 convertase converts C3 to C3a and C3b, while the C5 convertase converts C5 to C5a and C5b. C3a and C5a are potent inflammation-causing agents which activate a variety of cells. Activated cells release pro-inflammatory molecules such as tumor necrosis factor alfa (TNFα), vascular endothelial growth factor (VEGF) and other cytokines. The C5b molecule converts into a membrane attack complex (MAC) which damages cells and tissues. This cycle repeats itself until the entire tissue is damaged and gone. This vicious cycle produces excessive levels of pro-inflammatory cytokines such as TNF and VEGF, typically elevated in ocular diseases. The VEGF is known to promote the growth of new blood vessels and makes the blood vessels leaky in Wet-AMD. Anti- VEGF drugs (Lucentis and Eyelea) stop the development of these new blood vessels. These drugs prevent the loss of central vision in Wet AMD, Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR). As an investigational drug, NM3086 has been extensively studied in various in vitro and in vivo disease models, which prove that the drug: a) blocks AP-driven cellular lysis, TNF production, and VEGF production, and b) prevents neovascularization, scar formation, hemorrhage, and inflammation in animal models. Treatment with NM3086 is specific to the AP while preserving the regular activity of the classical Pathway (CP). Collectively, selective blockade of the AP without impacting the CP will allow patients to maintain host defense mechanisms responsible for preventing infections. "NM3086 is expected to be therapeutically effective across a broad range of rare and common complement-mediated disorders including ocular (Wet AMD, Dry AMD, and GA), inflammatory, renal, and hematological," states Dr. Rekha Bansal, Founder, and Chief Executive Officer of NovelMed Therapeutics. About NovelMed NovelMed is a clinical-stage biopharmaceutical company focused on developing novel biologics for treating a broad range of complement-mediated diseases caused by the activation of the Alternative Pathway. NovelMed is the first Company to have invented and validated an Anti-Bb antibody, an Anti-Properdin antibody, and an Anti-C3b antibody that selectively blocks the AP without inhibiting the classical Pathway. NovelMed has completed a Phase I trial for one of its lead product candidates (NCT05642546) and is currently evaluating this product in clinical trials for a) Paroxysmal Nocturnal Hemoglobinuria (PNH - NCT05646524, NCT05646563, NCT05731050), b) Atypical Hemolytic Uremic Syndrome (aHUS - NCT05684159), and c) Complement Glomerulopathy (C3G - NCT05647811). Irrespective of the target protein of the AP, a drug that expresses the best safety and efficacy profile for patients will be finalized for approval. NovelMed has recently selected lead albumin-linked antibodies designed to have an increased half-life in humans. NovelMed is a leader in developing AP-targeting therapies to resolve debilitating common and rare complement-mediated and complement-associated diseases. In addition to Anti-Properdin, the Company is advancing the development of Anti-Bb and Anti-C3b monoclonal antibodies that selectively block activation of the AP in a search for the best-in-class molecule with high potency and an extended half-life. As Dr. Bansal states: "NovelMed is committed to leveraging creativity and compassion to deliver game-changing therapies to patients." NovelMed seeks licensing, partnership, and acquisition opportunities to drive NM3086 through further development and approval in multiple rare disease indications. For more information or to contact NovelMed, please visit www.novelmed.com and contact us at bd@novelmed.com. Communication Teambd@novelmed.comTel: (216) 440 2696 NovelMed is on LinkedIn! Sign up to follow @NovelMed at https://www.linkedin.com/company/www.novelmedlinkedin.com. References NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone. NovelMed. January 31, 2022.NovelMed Announces Interim Positive Results from Its Phase I Clinical Trial of NM8074, a Bb Complement Alternative Pathway Blocker Antibody. NovelMed. February 21, 2022.Returning to complement – can NovelMed's next-gen antibody outperform Ultomiris? NovelMed. April 22, 2022.Alternative Pathway Blocker Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Efficacy Trial in C3 Glomerulopathy (C3G) Patients. NovelMed. August 15, 2022.Anti-Properdin Antibody (NM3086) Reduced Hemolysis, LDH, and Free Hemoglobin in an Animal Model of Paroxysmal Nocturnal Hemoglobinuria (PNH) -- a Rare Disease. NovelMed. December 12, 2022.NovelMed reports topline data with Anti-Properdin antibody NM-3086 in a rabbit model of PNH. NovelMed. December 13, 2022Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Clinical Trial in Naïve aHUS Patients (Rare Disease). January 30, 2023.Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT 05731050).Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (NCT 05646524).Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris (NCT 05646563).Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy (NCT 05684159).Study of NM8074 in Adult C3 Glomerulopathy Patients (NCT 05647811).First-in-Human (FIH) Clinical Study of NM8074 in Healthy Adult Volunteers (NCT05642546). A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f70c3b84-1cc6-4e65-8156-d8d60f573716