Dive Brief:
- Irish biotech Prothena Corp. plc has stopped development of its lead drug, NEOD001, closing down its trial and open label extension studies. Its stock plunged more than 68% on Monday, to a 52-week low of $11.50 per share.
- The compound failed to meet its primary or secondary endpoints in a Phase 2b PRONTO study in patients with AL amyloidosis and persistent cardiac dysfunction. Following a futility analysis of the ongoing VITAL Phase 3 study, an independent data monitoring committee recommended discontinuation of the trial.
- The company remains confident that it has the resources and scientific expertise to continue to advance its pipeline. "We are surprised by the results from these two placebo-controlled studies and will continue to analyze the resulting data to share insights with our collaborators," CEO Gene Kinney said in a statement.
Dive Insight:
The results of the study came as a shock to both Prothena and the market, and while the data will go forward for analysis, development has come to a pretty decisive stop. Michael Yee, an analyst at Jefferies, in a note to investors called it "the worst case scenario for this program."
Yet all isn't lost for Prothena. Last month, the biotech signed a preclinical stage deal with Celgene Corp., handing over a U.S. licensing option for up to three neurological candidates at investigational new drug status, with options to expand the rights after Phase 1. Prothena snagged $100 million up front and a $50 million equity investment, with milestone payments that could reach around $563 million per target. The deal provides Prothena with some cash to invest in the development of the other two drugs in its pipeline, PRX002 and PRX004.
Prothena's other clinical-stage candidate, PRX002, is in development through a collaboration with Roche AG.
Under the terms of that 2013 deal, the two companies will work together on U.S. development, with a 70-30 split of both costs and profits. Roche is responsible for the bigger share. Outside the U.S., development falls solely on the Swiss pharma's shoulders.
A monoclonal antibody, PRX002 (RG7935) moved into a Phase 2 trial of early Parkinson's disease patients in 2017. PRX004 is also a monoclonal, and is in preclinical development for ATTR amyloidosis.
"After the recent Celgene deal, Prothena has cash value plus other pipeline programs that could get some value over time," said Yee.