Rare Daily Staff

Prothena said it is discontinuing development of NEOD001, an investigational antibody that was being evaluated for the treatment of AL amyloidosis follow the failure of the experimental therapy meeting its primary or secondary endpoints in a mid-stage trial.

Shares of the company’s stock tumbled on the news as Prothena closed at $11.50 on April 23, down 69 percent from its $36.84 close on Friday.

AL amyloidosis is part of a complex group of diseases caused by tissue deposition of misfolded proteins that result in progressive organ damage. AL amyloidosis, the most common type, is a rare, progressive, and typically fatal. Soluble toxic aggregates and deposited amyloid lead to progressive failure of vital organs including the heart, kidneys and nervous system, causing significant morbidity and mortality.

The company said it made the decision to discontinue development of NEOD001 after the independent data monitoring committee conducted an analysis of its ongoing late-stage study and recommended discontinuation for futility.

“We are deeply disappointed by this outcome, particularly for patients suffering from this devastating disease,” said Gene Kinney, president and CEO of Prothena. “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 in the scientific, medical and advocacy communities.”

Lars Ekman, chairman of Prothena’s board, said the company has a deep pipeline in clinical and preclinical development, including several candidates partnered with strategic collaborators. “We have the resources and scientific expertise to continue to advance our pipeline through meaningful development milestones towards our goal of transforming patients’ lives,” he said.

April 23, 2018
Photo: Gene Kinney, president and CEO of Prothena