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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

CHAPEL HILL, N.C.--(BUSINESS WIRE)-- Areteia Therapeutics today announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) to dexpramipexole, an eosinophil lowering small molecule, that has recently entered Phase III clinical development. The Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and National Health Service (NHS) England, to chart a roadmap for regulatory and development milestones with the goal of early patient access in the U.K. “It is great to see that in granting an Innovation Passport, the MHRA is as excited as we are about the potential for dexpramipexole,” said Jorge Bartolome, Chief Executive Officer of Areteia Therapeutics. He added, “If approved, dexpramipexole would represent the first oral drug for eosinophilic asthma patients, offering an alternative to injectable biologics.” About Dexpramipexole Dexpramipexole inhibits the maturation and release of eosinophils in bone marrow, based on evidence from cell cultures and human biopsies, thereby lowering peripheral blood eosinophil levels. Most recently in a Phase II study (EXHALE-1) in patients with moderate-to-severe eosinophilic asthma, treatment with dexpramipexole resulted in a significant, dose-dependent reduction in blood absolute eosinophil count at all doses tested (dexpramipexole doses of 37.5 mg, 75 mg, or 150 mg twice daily) compared to placebo. Dexpramipexole was well tolerated in the trial, with adverse events balanced across treatment and placebo groups, no serious adverse events, and no adverse events leading to discontinuation. About Areteia Therapeutics Areteia Therapeutics (areteiatx.com) is a clinical stage biotechnology company committed to putting asthma patients in better control of their disease and lives by developing the first potential oral drug for eosinophilic asthma. Areteia’s lead drug candidate is dexpramipexole, a first-in-class oral eosinophil maturation inhibitor. Areteia was created by Population Health Partners and Knopp Biosciences. A syndicate of leading life sciences and strategic investors led by Bain Capital Life Sciences with participation from Access Biotechnology, GV, ARCH Venture Partners, Saturn Partners, Sanofi, Maverick Capital, and Population Health Partners, has committed to invest up to $350 million in Series A financing to establish Areteia and advance dexpramipexole through Phase 3 clinical trials, secure commercial supply, and pursue potential next-generation medicines. Areteia is conducting late-stage development including three Phase 3 clinical trials of dexpramipexole in partnership with Population Health Partners’ development unit, Validae Health. About Eosinophilic Asthma Asthma disrupts the lives of more than a quarter of a billion people worldwide. More than half of asthma patients have eosinophilic asthma, which is driven by an oversupply of eosinophils, a type of white blood cell, in blood and tissue. By inhibiting the maturation of eosinophils, oral dexpramipexole acts in a way similar to injectable anti-IL-5 biologic therapies. The asthma biologic market is experiencing growth of 10% per year and is valued at around $8 billion, with IL-5 biologic therapies representing greater than $3 billion of that figure. If approved as a first-to-market oral drug, dexpramipexole could provide a compelling alternative to injectable biologics, and could potentially be used earlier in the asthma treatment paradigm to prevent progression of disease. About ILAP The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) launched ILAP at the start of 2021 in order to accelerate the development and access to promising medicines in the early stages of development. The pathway, part of the UK’s plan to attract life sciences development in the post-Brexit era, features enhanced input and interactions with MHRA and other stakeholders. Other benefits of ILAP include access to a range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit risk assessment.