--- The United States FDA Clears Initiation of a Phase II Efficacy Trial in Immunoglobulin A Nephropathy (IgAN) Patients: a renal disorder

• Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis.
• Ruxoprubart is a highly selective inhibitor of the alternative complement pathway, which plays a critical role in the pathogenesis of IgAN.
• The Bb blocker is a multi-indication therapy with potential applications across a spectrum of disorders, including hemolytic, ocular, and renal diseases.
• Ruxoprubart is being positioned as the next-generation IgAN treatment based on its selective mechanism of action, where the drug does not block the classical pathway (CP), an important advantage over other complement blockers.
• Clinical data from the drug administration in naïve PNH patients shows promising results with a clean safety and efficacy profile, further supporting its potential to deliver meaningful clinical benefits.
• Unlike the traditional "one drug, one disease" model, Ruxoprubart has the potential to treat multiple chronic rare and common diseases. Ruxoprubart has also gained Phase II IND approval in AAV and aHUS by the US FDA.
• We seek strategic partners, investors, and acquisition opportunities to advance this drug to Phase II/III trials and achieve FDA approval.

CLEVELAND, Dec. 02, 2024 (GLOBE NEWSWIRE) -- NovelMed is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted clearance for Ruxoprubart, an investigational drug, to commence an efficacy trial for the treatment of Immunoglobulin A nephropathy (IgAN), a rare autoimmune chronic ailment characterized by inflammation of small blood vessels.

The current standard of care (SOC) for IgAN includes corticosteroids and immunosuppressive therapy. However, these treatments are often associated with significant side effects, including general immune suppression and toxicity from glucocorticoids, leaving unmet needs in managing the disease's impact on vital organs and associated with adverse events (AEs).

Results from the Phase I and II trials of Ruxoprubart in PNH patients have demonstrated its potential as a highly selective blocker of the alternative pathway (AP). Unlike other complement blockers, Ruxoprubart does not affect the classical pathway (CP), which is critical for maintaining immunity against infections. This unique selectivity offers a significant advantage over existing complement blockers approved by the FDA or under development, potentially eliminating the need for a Black Box Warning.

Phase II Clinical Trial in IgAN Patients

In 2023, the United States FDA approved Tarpeyo (Calliditas Therapeutics), a corticosteroid designed to treat adults with primary IgA nephropathy who are at risk of disease progression. Tarpeyo helps slow disease progression, including the reduction of proteinuria, a hallmark of IgAN. More recently, the FDA has approved a twice-daily dose of Iptacopan (Novartis) for the treatment of IgAN, which also reduces proteinuria.

Ruxoprubart, a highly selective and specific blocker of the alternative pathway (AP), stands out since it does not affect the classical pathway (CP) which is required for fending off infections in a chronic condition like IgAN. The FDA has granted the IND approval for Ruxoprubart across multiple indications, including paroxysmal nocturnal hemoglobinuria (PNH), Complement C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), and Immunoglobulin A Nephropathy (IgAN), marking the importance of Ruxoprubart in several other disorders where inflammation is the root cause of diseases. The dysregulation of the alternative pathway (AP) is recognized as a critical driver of pathological events in IgAN, making Ruxoprubart a promising candidate. "We are excited to announce the Phase II IND approval for IgAN, alongside aHUS and AAV, where Ruxoprubart will play a pivotal role in the treatment of chronic kidney conditions," says Dr. Rekha Bansal, CEO of NovelMed Therapeutics.

Food and Drug Administration (FDA) and Ruxoprubart

The FDA's clearance of the IND in Phase II trial brings us one step closer to treating several renal disorders, including IgAN. An additional Phase II trial is under regulatory review by the regulatory agency in a country with a substantial population of treatment-naïve IgAN patients.

"We are thrilled to have gained FDA approval to treat chronic IgAN in patients,” says Mr. Alex Kumar, Chief Strategy Officer of NovelMed. "This clearance marks a significant milestone for Ruxoprubart, not only for IgAN but also as a platform therapy for multiple indications" emphasizes Dr. Rekha Bansal, the CEO of NovelMed. Ruxoprubart is expected to be a next-generation product with a good safety profile.

Ruxoprubart is an innovative anti-Bb monoclonal antibody therapy that selectively blocks the initiation and propagation of the alternative pathway (AP), a key driver of disease progression. By blocking the dysregulation of the AP while sparing the classical complement pathway, Ruxoprubart holds promise for effectively treating multiple renal disorders, including IgAN. Ruxoprubart is positioned for a broad spectrum of renal disorders, including PNH, C3G, aHUS, and AAV. For this multi-indication platform, the clinical trials for Ruxoprubart can be found at www.clinicaltrials.gov.

Immunoglobulin A Nephropathy (IgAN)

In the United States, the prevalence of Immunoglobulin A nephropathy (IgAN) is estimated to range between 82,000 and 110,000 patients, highlighting the need for advanced treatment strategies. IgAN, the most prevalent form of glomerulonephritis globally, is characterized by the deposition of IgA immunoglobulins in the glomeruli, leading to inflammation, proteinuria, and potentially progressive kidney damage. Despite current treatments, including the recently approved Fabhalta (Novartis), demonstrating that complement blockers can play a role in managing the disease, further advancements in treatment strategies are important for continued improvement in the clinical outcome of IgAN patients.

About NovelMed

NovelMed is a clinical-stage biopharmaceutical company dedicated to developing antibody therapies for many diseases where an alternative pathway is activated. NovelMed exhibits 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 AP activation. As part of its antibody platform, NovelMed remains engaged in developing a robust pipeline of treatments using various forms of antibody therapies.

NovelMed actively seeks licensing, partnership, and acquisition opportunities to advance the development of clinical candidates through Phase II/III and approval in multiple rare disease indications. For more information, please visit the News section News (novelmed.com).

Ya Gao, MS
Senior Clinical Data Analyst
Email: bd@novelmed.com
Tel: (216) 440 2696

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