Swedish Orphan Biovitrum AB (Sobi) (OTC:SWOBY) (OTC:BIOVF) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) on Thursday released topline data results from Phase 3 VALIANT study of systemic pegcetacoplan for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

Excessive C3c deposits are a marker of disease activity, which can lead to kidney inflammation, damage, and failure. Additionally, two-thirds of patients who previously received a kidney transplant will experience disease recurrence. The diseases are estimated to affect 5,000 people in the U.S. and up to 8,000 in Europe.

The study met the primary endpoint, demonstrating a statistically significant and clinically meaningful 68% reduction in proteinuria in patients treated with pegcetacoplan compared to placebo, both in addition to background therapy, at Week 26.

Results were consistent across all subgroups, including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys.

Pegcetacoplan also demonstrated statistical significance on the key secondary endpoints of composite renal endpoint, which combines proteinuria reduction and estimated glomerular filtration rate (eGFR) stabilization, and proteinuria reduction of at least 50% compared to baseline, and nominal significance on the histological endpoint of reduction in C3c staining on kidney biopsy and stabilization of kidney function as measured by eGFR compared to placebo.

In the VALIANT study, pegcetacoplan showed favorable safety and tolerability, consistent with its established profile.

All patients who have completed the VALIANT study have enrolled in the VALE long-term extension study.

Sobi plans to submit a marketing application to the European Medicines Agency in 2025. Apellis also plans to submit a supplemental new drug application to the FDA in early 2025.

Pegcetacoplan is already approved in the U.S. for geographic atrophy secondary to age-related macular degeneration as Syfovre and paroxysmal nocturnal hemoglobinuria as Empaveli/Aspaveli.

Syfovre generated sales of $154.6 million, and Empaveli generated $24.5 million.

In June, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a negative opinion on the marketing application of intravitreal pegcetacoplan for geographic atrophy.

Apellis plans to seek re-examination and expects a final opinion in the fourth quarter of 2024.

Price Action: APLS stock is up 11.2% at $37.35 at last check Thursday.

Photo via Shutterstock

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Thursday, August 08, 2024

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