FDA Clears Initiation of Phase 2 Efficacy Trial for Ruxoprubart in IgAN

Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) has cleared the initiation of a Phase 2 efficacy clinical trial to evaluate the investigational new drug (IND) ruxoprubart for the treatment of Immunoglobulin A nephropathy (IgAN).¹

Announced by NovelMed on December 2, 2024, ​​ruxoprubart is positioned as the next generation of IgAN treatment based on its selective inhibition of the alternative pathway, with the company noting its important advantage over other complement blockers of the classical pathway.

“We are excited to announce the Phase 2 IND approval for IgAN, alongside atypical hemolytic uremic syndrome (aHUS) and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), where Ruxoprubart will play a pivotal role in the treatment of chronic kidney conditions,” Rekha Bansal, chief executive officer of NovelMed, announced in a statement. “This clearance marks a significant milestone for Ruxoprubart, not only for IgAN but also as a platform therapy for multiple indications.”

In recent years, the treatment landscape for IgAN has experienced rapid transformation, with the emergence of multiple pharmacotherapies within the IgAN pipeline. These recent FDA approvals included budesonide (Tarpeyo) delayed-release capsules for IgAN, iptacopan (Fabhalta) for the reduction of proteinuria in adults with primary IgAN, and sparsentan (Filspari) for slowing kidney function decline in adults with primary IgAN at risk of disease progression.^2,3,4

Prevalence estimates indicate that IgAN affects between 82,000 and 110,000 individuals in the United States, pointing to the need for further treatment innovations to improve the clinical outcomes of patients with IgAN.

Ruxoprubart is an anti-Bb monoclonal antibody that selectively blocks the initiation and distribution of the alternative pathway.¹ Blocking the dysregulation of this pathway, while avoiding the classical complement pathway, Ruxoprubart could effectively treat a broad range of renal disorders.

In their announcement, NovelMed indicated the IND approval for Ruxoprubart involved numerous indications, including paroxysmal nocturnal hemoglobinuria (PNH), Complement C3 Glomerulopathy (C3G), aHUS, AAV, and IgAN. Results from Ruxoprubart Phase 1 and 2 trials in PNH demonstrated its potential as a highly selective blocker of the alternative pathway.

NovelMed added its search to seek strategic partners, investors, and acquisition opportunities that will allow the company to advance ruxoprubart to Phase 2/3 trials and ultimately achieve FDA approval.

“We are thrilled to have gained FDA approval to treat chronic IgAN in patients,” Alex Kumar, chief strategy officer of NovelMed, added in a statement.

References

1. _{NovelMed Therapeutics Inc. NOVELMED receives FDA IND approval for NM8074 (Ruxoprubart), the first Anti-Bb alternative pathway blocker for treating primary immunoglobulin a nephropathy (IgAN): A renal disorder. GlobeNewswire News Room. December 2, 2024. Accessed December 2, 2024. https://www.globenewswire.com/news-release/2024/12/02/2989809/0/en/NovelMed-Receives-FDA-IND-Approval-for-NM8074-Ruxoprubart-the-First-Anti-Bb-Alternative-Pathway-Blocker-for-Treating-Primary-Immunoglobulin-A-Nephropathy-IgAN-A-Renal-Disorder.html.}
2. _{Brooks A. FDA Awards Full Approval to Budesonide (Tarpeyo) in Treatment of IgA Nephropathy. HCPLive. December 20, 2023. Accessed December 2, 2024. https://www.hcplive.com/view/fda-awards-full-approval-to-budesonide-tarpeyo-in-treatment-of-iga-nephropathy}
3. _{Campbell P. IPTACOPAN receives accelerated approval for reducing proteinuria in IGA nephropathy. HCPLive. August 8, 2024. Accessed December 2, 2024. https://www.hcplive.com/view/iptacopan-receives-accelerated-approval-for-reducing-proteinuria-in-iga-nephropathy.}
4. _{Brooks A. FDA Approves Travere Therapeutics’ Sparsentan to Slow Kidney Function Decline in Adult Primary IgAN. HCPLive. September 5, 2024. Accessed December 2, 2024. https://www.hcplive.com/view/fda-approves-travere-therapuetics-sparsentan-for-proteinuria-reduction-in-adult-primary-igan}