WALTHAM, Mass. - Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (STO:SOBI), also known as Sobi, today announced significant findings from their Phase 3 VALIANT study. The study focused on pegcetacoplan, an investigational therapy for patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), both rare kidney diseases lacking FDA-approved treatments.
The trial met its primary endpoint, showing a 68% reduction in proteinuria, a marker of kidney damage, compared to placebo after 26 weeks. This result, with a p-value of less than 0.0001, was consistent across all patient subgroups, including those differentiated by disease type, age, and kidney transplant status.
Pegcetacoplan also reached statistical significance in key secondary endpoints, including a composite of proteinuria reduction and kidney function stabilization, and a 50% or greater reduction in proteinuria from baseline. The study further noted nominal significance in histological endpoints, such as reduced kidney C3c staining, which indicates disease activity.
The safety profile of pegcetacoplan was favorable and consistent with previous data, with similar rates of adverse events between the drug and placebo groups. No serious infections related to encapsulated bacteria were reported.
The positive outcomes of the VALIANT study have spurred Apellis to plan a supplemental new drug application submission to the U.S. Food and Drug Administration in early 2025, with Sobi preparing for a concurrent marketing application to the European Medicines Agency.
Dr. Carla Nester, the study's lead principal investigator, expressed optimism about the treatment's potential to improve patient outcomes. With many patients currently facing kidney transplants or lifelong dialysis, the need for a treatment targeting the diseases' root causes is urgent.
Apellis and Sobi's collaboration on pegcetacoplan spans global co-development, with Sobi holding exclusive commercialization rights outside the U.S. Pegcetacoplan is already approved for paroxysmal nocturnal hemoglobinuria treatment and is under investigation for other rare diseases.
In other recent news, Apellis Pharmaceuticals revealed its financial results for the second quarter of 2024, demonstrating robust growth and market confidence. The company's drugs SYFOVRE and EMPAVELI have performed exceptionally well, with SYFOVRE achieving 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.
Apellis Pharmaceuticals has secured broad coverage for SYFOVRE with two large national pharmacy benefit managers and maintains a strong market share, with SYFOVRE holding approximately 50% of new starts in the geographic atrophy (GA) market. Despite facing increased competition, Apellis is optimistic about its future, focusing on the European market and expansion into new indications.
Recent developments include advancing multiple earlier-stage programs in complement science and an expectation 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. The company is also awaiting a final decision on SYFOVRE's approval in the European market, expected in Q4 2024.
InvestingPro Insights
As Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) prepares for an important regulatory submission following their promising clinical trial results, investors and stakeholders are closely monitoring the company's financial health and stock performance. According to InvestingPro data, Apellis has a market capitalization of $4.09 billion USD, signaling a significant valuation in the biopharmaceutical space. Despite not paying dividends, which is common for companies focused on growth and reinvestment, Apellis has shown an impressive revenue growth of 240.74% over the last twelve months as of Q2 2024, indicating strong sales momentum for its products.
InvestingPro Tips highlight the analysts' anticipation of sales growth in the current year, which could be further fueled by the potential approval and commercialization of pegcetacoplan for rare kidney diseases. However, it's important to note that three analysts have revised their earnings expectations downwards for the upcoming period, and the company is not expected to be profitable this year. Additionally, with a high price-to-book ratio of 15.47, the stock may be considered expensive relative to its net assets. Apellis operates with a moderate level of debt and has liquid assets that exceed its short-term obligations, suggesting a stable financial position to manage its operations and investments.
It's also worth noting that the stock has experienced a significant decline over the last six months, with a 50.69% drop in price total return. This could present an opportunity for investors who believe in the long-term potential of the company's pipeline to buy in at a lower price. For further insights, InvestingPro offers additional tips on Apellis, which can be found at their dedicated page: https://www.investing.com/pro/APLS
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