United States FDA Approves NM8074 (Ruxoprubart) IND for Treating Dermatomyositis (DM): A Chronic Inflammatory Disorder of the Skin

--- The United States FDA Approves the Initiation for Phase II Efficacy Trial in Dermatomyositis (DM) Patients: A Skin & Muscle Disorder

• DM is an immune-mediated myopathy characterized by chronic inflammation of the skin and muscles, progressive muscle weakness, and potential involvement of internal organs. The role of complement appears to be well-established in the disease’s pathogenesis.
• Ruxoprubart is a highly selective blocker of the alternative complement pathway, which plays a critical role in orchestrating the pathogenesis of DM.
• By targeting Factor Bb, Ruxoprubart offers a more precise and safer approach for treating DM while avoiding the Black Box Warnings associated with the most existing complement blockers.
• Ruxoprubart does not block the beneficial components of the immune system, including opsonization and bacterial clearance via the normal route.
• Phase I clinical trial data in healthy volunteers showed promising results with a clean safety profile, supporting its potential to deliver meaningful clinical benefits in DM.
• Unlike the traditional "one drug, one disease" model, Ruxoprubart has the potential to treat multiple chronic rare and common diseases, as evidenced by its recent Phase II IND approvals in multiple hematologic and renal diseases.
• NovelMed is actively seeking strategic partners, investors, and acquisition opportunities to accelerate the development of Ruxoprubart through Phase II/III trials and towards FDA approval.

CLEVELAND, Feb. 24, 2025 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, a clinical-stage biotechnology company, pioneering complement-targeting therapies, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted clearance for Ruxoprubart (NM8074), an investigational drug, to begin an efficacy Phase II trial to treat Dermatomyositis (DM), a rare autoimmune disorder.

This announcement follows the successful completion of a Phase I clinical trial involving 40 healthy volunteers, where Ruxoprubart demonstrated promising results and a clean safety profile, with no serious adverse events reported. These results highlight Ruxoprubart’s potential as a safe and effective treatment for DM and other complement-mediated diseases.

“Our team is thrilled with the early success of Ruxoprubart in clinical trials,” said Rekha Bansal, PhD, Founder and CEO of NovelMed Therapeutics. “Dermatomyositis is a condition that causes significant physical and emotional suffering for patients, and we believe Ruxoprubart has the potential to offer real hope for improved treatment outcomes. This is a pivotal step in our mission to advance therapies for rare diseases and extend our leadership in complement-based therapeutics.”

Dermatomyositis – A Rare Disease with Unmet Need

Dermatomyositis (DM) is a rare idiopathic inflammatory condition affecting multiple organ systems, with an estimated prevalence of approximately 13 per 100,000 individuals in the United States. This disease is characterized by debilitating muscle weakness and distinctive skin rashes. Notably, about half of those affected experience falls, and one-third require mobility aids such as canes, walkers, or wheelchairs.

Current treatment options for DM remain limited, with corticosteroids and immunosuppressive agents, which carry significant side effects. While the FDA has approved intravenous immunoglobulin (IVIg) in combination with corticosteroids for DM, there remains a critical need for safer and more effective therapies.

“The FDA’s IND approval for our Phase II trial in Dermatomyositis is an exciting milestone,” said Mr. Robert Bard, VP of Regulatory Affairs at NovelMed Therapeutics. “Current treatments rely heavily on corticosteroids and immunosuppressive agents, which come with significant safety risks. Ruxoprubart has the potential to redefine the standard of care by targeting a key driver of inflammation while avoiding broad immunosuppression.”

^{Figure: Discovery of Ruxoprubart (NM8074) - Ruxoprubart was selected as the lead candidate for the treatment of Dermatomyositis (DM)}

Ruxoprubart – A Targeted Alternative Pathway Blocker

Ruxoprubart is a highly selective inhibitor of the alternative complement pathway (AP), uniquely designed to preserve the classical pathway (CP), which is essential for immune defense. By specifically targeting Factor Bb, Ruxoprubart effectively modulates the immune response, reducing inflammation associated with autoimmune conditions like Dermatomyositis (DM) while maintaining critical immune functions such as opsonization and bacterial clearance.

In DM, complement-mediated inflammation leads to muscle fiber damage and distinctive skin rashes. The deposition of the membrane attack complex (MAC) on endothelial cells causes microvascular injury and muscle atrophy, with alternative complement pathway activation playing a central role in disease progression. By selectively blocking this pathway, Ruxoprubart addresses the root cause of inflammation without the safety concerns linked to broad immunosuppressive therapies.

Initial results from completed Phase I trials and ongoing Phase II studies in PNH have shown Ruxoprubart’s ability to effectively block the alternative complement pathway while preserving immune system integrity. These promising results provide further validation of Ruxoprubart's potential as a next-generation therapeutic option for autoimmune diseases like dermatomyositis, where targeted therapies are urgently needed to address both the inflammation and the underlying immune dysfunction.

Ruxoprubart – Broad Regulatory Approvals

The U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) approval for Ruxoprubart across several indications, including paroxysmal nocturnal hemoglobinuria (PNH), Complement C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), IgA nephropathy (IgAN), and Dermatomyositis (DM).

This broad approval underscores the significance of Ruxoprubart in treating multiple inflammatory diseases, where the alternative complement pathway (AP) is a key driver of pathogenesis. In DM, the dysregulation of AP is recognized as a critical factor in the disease's progression, making Ruxoprubart an interesting candidate for this rare autoimmune condition.

“We are pleased that the FDA recognizes the potential of Ruxoprubart to serve as a meaningful therapeutic option for DM patient population,” said Alex Kumar, Chief Strategy Officer. “Leveraging our novel anti-complement antibody platform, we are committed to our mission of expanding the reach of Ruxoprubart to patients with DM. We remain on track to filing Investigational New Drug applications for various complement-mediated diseases.”

About NovelMed

NovelMed Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative antibody therapies targeting the alternative complement pathway (AP), a key driver in various autoimmune and inflammatory diseases. The company has established a robust intellectual property portfolio that spans both rare and common diseases, positioning it as a leader in complement-mediated therapeutics.

The company’s lead candidate, Ruxoprubart is a highly selective AP inhibitor showing potential in treating a variety of complement-driven disorders linked to AP activation, such as dermatomyositis (DM), paroxysmal nocturnal hemoglobinuria (PNH), and atypical hemolytic uremic syndrome (aHUS).

NovelMed’s platform is centered on the development of antibody-based therapies that can selectively target the complement system with precision, offering safer and more effective treatment options for patients. The company’s pipeline includes a wide range of long-acting antibody therapies designed to address unmet needs in both rare and common diseases, with an emphasis on conditions where chronic inflammation plays a central role in disease progression. This strategic focus on complement modulation differentiates NovelMed's approach and reflects its commitment to advancing transformative therapies for autoimmune and complement-related diseases.

NovelMed actively seeks out licensing and acquisition opportunities to propel the development of its clinical candidates through Phase II/III trials and regulatory approval. For more information about the company's latest developments, please visit the News section on NovelMed's website.

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

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A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/87560068-e7bd-47ae-8337-df560b652da1