Today, Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) in collaboration with Sobi® disclosed successful outcomes from their Phase 3 VALIANT trial, which evaluated pegcetacoplan in treating two rare kidney diseases, C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
The study achieved its primary goal, showing a significant 68% reduction in proteinuria at Week 26, a marker of kidney disease severity, in patients receiving the drug compared to a placebo.
The study's positive results included both primary and key secondary endpoints. Notably, pegcetacoplan led to improvements in a composite renal endpoint, which assesses proteinuria reduction and kidney function stabilization, and a significant number of patients experienced a proteinuria reduction of at least 50 percent from baseline. Moreover, the drug showed promise in reducing C3c staining on kidney biopsies and maintaining stable kidney function.
Safety profiles during the trial remained consistent with previous data, showing that pegcetacoplan was well-tolerated. Rates of adverse events, serious adverse events, and discontinuations due to adverse events were comparable between the drug and placebo groups. Importantly, there were no reported cases of meningitis or serious infections related to encapsulated bacteria.
Following the completion of the VALIANT study, participants have moved on to the VALE long-term extension study. Apellis plans to file a supplemental new drug application with the U.S. Food and Drug Administration (FDA) in early 2025, and Sobi is set to submit a marketing application to the European Medicines Agency that same year. Detailed findings from the study are expected to be presented at an upcoming medical congress.
The company's forward-looking statements indicate an intention to seek regulatory approvals for pegcetacoplan for the treatment of C3G and IC-MPGN. However, they caution that approvals are not guaranteed and are subject to the evaluation of various important factors, including the drug's review by the FDA and other regulatory bodies.
This report is based on a press release statement and the content of the Form 8-K filed with the Securities and Exchange Commission.
In other recent news, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB (Sobi) announced significant findings from their Phase 3 VALIANT study, which focused on pegcetacoplan, a potential treatment for rare kidney diseases.
The trial met its primary endpoint, showcasing a 68% reduction in proteinuria, a marker of kidney damage. The safety profile of pegcetacoplan was favorable, prompting Apellis to plan a new drug application submission to the FDA in early 2025.
In financial developments, Apellis reported robust growth for the second quarter of 2024, with its drugs SYFOVRE and EMPAVELI performing well. SYFOVRE achieved over $0.5 billion in sales since its launch, and $155 million in net product revenue in Q2 2024 alone. EMPAVELI also generated substantial revenue with $24.5 million in sales.
Despite facing increased competition, Apellis maintains a strong market share, with SYFOVRE holding approximately 50% of new starts in the geographic atrophy (GA) market. The company is also advancing multiple earlier-stage programs in complement science and expects to become cash flow positive in the future. However, sales of EMPAVELI are expected to remain flat for the next 6 to 12 months due to competition.
InvestingPro Insights
As Apellis Pharmaceuticals (NASDAQ:APLS) advances its promising drug pegcetacoplan through clinical trials, the company's financial outlook presents a mixed picture according to real-time data from InvestingPro. Despite a notable 240.74% surge in revenue over the last twelve months as of Q2 2024, analysts are cautious about the company's profitability. InvestingPro Tips indicate that analysts do not expect Apellis to be profitable this year, and three analysts have recently revised their earnings estimates downwards for the upcoming period.
InvestingPro Data also reveals that Apellis operates with a moderate level of debt and that its liquid assets exceed short-term obligations, suggesting a stable financial position for handling its near-term liabilities. However, with a high Price/Book multiple of 15.47 as of Q2 2024, the stock may be considered expensive relative to the company's book value. It's also worth noting that Apellis does not pay a dividend, which could be a factor for income-focused investors to consider.
For investors tracking the performance of Apellis, the stock has experienced a significant decline over the last six months, with a 50.69% drop in price total return. Investors interested in deeper analysis and additional InvestingPro Tips can explore more on InvestingPro, where a total of 9 tips are available to help evaluate the company's prospects.
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