Apellis Shares Rise on NEJM Validation of Kidney Drug Empaveli
Prestigious medical journal publishes positive Phase 3 data, reinforcing the drug's efficacy for two rare kidney diseases and bolstering its commercial prospects.
Shares of Apellis Pharmaceuticals (NASDAQ: APLS) climbed in trading Wednesday after The New England Journal of Medicine (NEJM) published positive results from the Phase 3 VALIANT study of its drug Empaveli (pegcetacoplan), validating its effectiveness in treating two rare and debilitating kidney diseases.
The stock rose as much as 3% to $21.25 a share, as investors reacted to the peer-reviewed endorsement from one of the world's most prestigious medical journals. The publication provides a critical scientific backing for Empaveli, which is the first and only FDA-approved treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 and older.
The VALIANT study data, detailed in the company's official announcement, showed that Empaveli achieved a statistically significant 68% reduction in proteinuria—a key marker of kidney damage—compared to a placebo at the 26-week mark. Furthermore, the treatment helped stabilize kidney function, a crucial outcome for patients at risk of progressing to kidney failure.
"Publication in the NEJM is a major milestone that validates the robust and clinically meaningful benefits of Empaveli for these rare kidney diseases," said Caroline P. Darcy, M.D., chief medical officer at Apellis, in a statement. "For physicians, this high-level, peer-reviewed data is essential for building confidence and driving the adoption of a new standard of care."
Empaveli, a targeted C3 therapy, is a cornerstone asset for the Waltham, Massachusetts-based biotech, which has a market capitalization of approximately $2.61 billion. The drug is already approved to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, and its injectable form, marketed as Syfovre, is a leading treatment for geographic atrophy, a cause of blindness. The expansion into C3G and IC-MPGN represents a significant step in broadening the drug's therapeutic footprint.
While FDA approval for these kidney indications was secured earlier, the NEJM publication serves as a powerful marketing and educational tool for the drug's commercial rollout. It provides the rigorous, independent validation that specialists often require before broadly prescribing a new therapy, particularly for complex, rare conditions with high unmet needs.
Wall Street has maintained a broadly positive outlook on Apellis, with a consensus analyst target price of over $35, suggesting significant upside from its current trading levels. Analysts see the continued expansion of Empaveli's approved uses as a key driver for the company's growth. The drug's success across multiple complement-driven diseases underscores the potential of Apellis's C3-targeting platform, with some analysts noting future pipeline catalysts in other conditions as areas for continued investor focus.
Looking ahead, investors will be closely watching the initial sales figures for Empaveli in the C3G and IC-MPGN market. The NEJM publication provides a strong tailwind for Apellis's commercial team as it works to establish the drug as the go-to treatment, and its ability to translate this scientific win into revenue will be a key performance indicator in the upcoming quarters.