Sobi & Apellis’ Aspaveli/Empaveli demonstrates sustained one-year efficacy for KD

Overview

Sobi, a global biopharma company, and Apellis Pharmaceuticals, Inc., a global biopharmaceutical company, presented new data from the open-label period of the phase 3 VALIANT study, investigating Aspaveli (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The data were presented as part of a late-breaking session at the European Renal Association (ERA) Congress.

About the VALIANT study

• In the VALIANT study, Aspaveli demonstrated a statistically significant 68% proteinuria reduction versus placebo at Week 26, which was sustained at one year. 
• Additionally, patients treated with Aspaveli continued to achieve stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR).

Statement from the principal investigator

• The one-year phase 3 results are very compelling, confirming Aspaveli’s sustained benefits across key markers of disease,” said Fadi Fakhouri, M.D., PhD, presenting author, co-lead principal investigator for the VALIANT study, and professor of nephrology at CHUV Lausanne, Switzerland. 
• Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. These data further underscore the potential of Aspaveli to make a meaningful difference for patients.”

Aspaveli Shows Kidney Benefits After Placebo Switch

In patients who switched from placebo to Aspaveli at the start of the open-label period, Aspaveli demonstrated a similar magnitude of benefit in proteinuria reduction and stabilization of kidney function.

From the medical executive affairs: Sobi

Nils Kinnman, MD, PhD, head of medical affairs and clinical development, Sobi said, “The results from the phase 3 VALIANT study underscore the potential of Aspaveli in addressing the urgent needs of patients living with the kidney diseases C3G and primary IC-MPGN. This study is an example of Sobi’s commitment to advance innovative therapies that make a meaningful difference in patients' lives.”

Words from the CMA: Apellis

• These data reinforce the strength of the Empaveli efficacy and safety profile across a broad population of patients with C3G and primary IC-MPGN, including adults and adolescents with native and post-transplant kidney disease,” said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare disease, Apellis. 
• With an FDA decision this summer, we look forward to bringing Empaveli to patients living with these rare and severe kidney diseases as quickly as possible.

Empaveli/Aspaveli showed favourable safety and tolerability, consistent with its established profile. There were no new safety signals.

VALIANT Study, Top 10 Abstracts Highlighted at ERA

• A total of eight presentations, including six on podium, will be highlighted at the meeting. 
• The presentations will showcase clinically meaningful results from the phase 3 VALIANT study, among other data. 
• Additionally, two abstracts were selected by congress organizers as Top 10 best ERA abstracts. 
• The “Top 10” are deemed significant studies underlining the growing field of clinical research in kidney disease.

Rare idney disease: C3G and primary IC-MPGN 

• C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure.
• Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. 
• Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis. 
• Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence. 
• The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe. 

Process of VALIANT phase 3 study

• The VALIANT phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. 
• It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients with native and post-transplant kidneys. 
• Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. 
• Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received pegcetacoplan. 
• The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.

About the Pegcetacoplan

• Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. 
• Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. 
• Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as Empaveli/Aspaveli in the United States, European Union, and other countries globally.

Apellis and Sobi Share Global Rights for Pegcetacoplan

• Apellis and Sobi have global co-development rights for systemic pegcetacoplan. 
• Sobi has exclusive ex-US commercialization rights for systemic pegcetacoplan, and Apellis has exclusive US commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

About the company: Sobi

• Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. 
• Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. 

About the company: Apellis Pharmaceuticals, Inc.

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face.

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