Sobi Unveils Key Data for Kidney Drug Pegcetacoplan at ERA 2026

STOCKHOLM – June 01, 2026 – Sobi today announced it will present a substantial body of new data on pegcetacoplan at the 63rd Congress of the European Renal Association (ERA) in Glasgow, UK. The presentations, scheduled for June 3-6, will highlight the long-term efficacy and safety of the drug in treating C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), two rare and devastating kidney diseases that often lead to kidney failure.

The forthcoming data, generated in collaboration with Apellis Pharmaceuticals (now part of Biogen), stems from the pivotal Phase 3 VALIANT study and its long-term extension, the VALE study. For the estimated 13,000 patients in Europe and the United States living with these conditions, the findings represent a significant step forward in a field that has long suffered from a lack of effective, targeted treatments.

"As the body of evidence continues to grow, we are deepening our understanding of C3G and primary IC-MPGN and the burden these rare and serious kidney diseases place on patients," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. "The data being presented at ERA 2026 continue to build the evidence supporting the sustained efficacy and well-characterised safety profile of pegcetacoplan in patients with C3G and primary IC-MPGN."

A New Era for Rare Kidney Disease Treatment

For decades, patients diagnosed with C3G or IC-MPGN faced a grim prognosis. Characterized by the excessive activation of the complement system—a part of the body's immune response—these diseases cause severe inflammation and damage to the kidneys. Approximately half of all patients progress to end-stage renal disease, requiring dialysis or a kidney transplant, within five to ten years of diagnosis. Compounding the issue, the disease recurs in about 90% of transplant recipients, often leading to the loss of the new organ.

Pegcetacoplan, marketed as Aspaveli®/Empaveli®, is a targeted C3 therapy designed to regulate the complement cascade at its source. It is the first treatment approved for C3G and IC-MPGN in patients aged 12 and older in the United States, the European Union, and other key markets. The data that supported these approvals, and which will be expanded upon at ERA 2026, has been described by leading nephrologists as groundbreaking.

Results from the initial 26-week VALIANT study demonstrated a statistically significant and clinically meaningful 68% reduction in proteinuria—a key marker of kidney damage—compared to placebo. Crucially, patients treated with pegcetacoplan also showed a stabilization of kidney function, as measured by estimated glomerular filtration rate (eGFR). The upcoming presentations will delve into one-year data from the VALE extension study, providing critical insights into the sustained clinical benefit of the therapy.

Further analyses to be presented in Glasgow will explore the treatment's impact based on genetic factors, disease chronicity, and its consistent safety profile, including a low incidence of meningococcal infections, a known risk for complement inhibitors.

Shifting Tides in the Pharmaceutical Landscape

The clinical success of pegcetacoplan is reshaping not only patient outcomes but also the strategic landscape for rare diseases. The drug's potential was a primary driver behind Biogen's recent acquisition of Apellis Pharmaceuticals in a deal valued at approximately $5.6 billion, a move designed to bolster Biogen's immunology and rare disease portfolio.

Under the longstanding collaboration, Sobi retains exclusive commercialization rights for systemic pegcetacoplan outside the United States. In a clear signal of its confidence in the drug's long-term value, Sobi amended its royalty agreement with Apellis in July 2025, making a $275 million upfront payment to significantly reduce its future royalty obligations. This strategic maneuver positions Sobi to maximize returns from the therapy's adoption across Europe and other global markets.

The field, however, is not without competition. Novartis's iptacopan (Fabhalta®), a factor B inhibitor, also recently gained approval for C3G, marking the beginning of a new competitive dynamic in targeted complement therapies for renal diseases. While both drugs represent a monumental leap from previous supportive care standards like steroids and general immunosuppressants, the robust data on pegcetacoplan—particularly its strong effect on proteinuria reduction and C3 deposit clearance across a broad patient population including adolescents and post-transplant cases—provides a compelling case for its use.

The series of oral and poster presentations at ERA 2026 will be critical for Sobi and Biogen to further differentiate pegcetacoplan and solidify its standing with clinicians.

The Human Impact Beyond the Data

Behind the clinical trial statistics and market analyses are thousands of patients and families whose lives are dictated by the progression of these chronic conditions. The journey often involves frequent hospital visits, burdensome treatments, and the constant anxiety of declining kidney function. For younger patients, a diagnosis can disrupt education, social development, and future aspirations.

The VALIANT trial was notable for being the largest study ever conducted in these populations and for its inclusion of patients as young as 12. The findings that pegcetacoplan can stabilize kidney function and dramatically reduce proteinuria are not just clinical endpoints; they translate into the potential for a more normal life, delaying or even preventing the need for dialysis.

Physicians who have long managed these patients with a limited toolkit have hailed the arrival of effective, targeted therapies as a paradigm shift. The ability to treat the underlying mechanism of the disease rather than just its symptoms offers a profound sense of hope. The data confirming the long-term safety and sustained efficacy of pegcetacoplan is essential for building the confidence of both doctors and patients in committing to a lifelong therapy.

As the nephrology community gathers in Glasgow, the presentations on pegcetacoplan will be among the most anticipated events. The data shared will provide a deeper understanding of how to optimize treatment for a complex patient population, reinforcing the dawn of a new, more hopeful era in the management of complement-driven kidney diseases.