In a press release, Neuraltus CEO Rich Casey says the future of NP001 development in ALS is uncertain as his company reports results from its Phase 2 clinical trial failed to reach any of its endpoints. Top-line data was presented at the American Academy of Neurology (AAN) annual meeting yesterday. This trial was closely watched by many in the ALS community and the negative results today are disappointing.
According to a since removed post by AAN reporter Jamie Talon, primary investigators reported during the presentation yesterday that there was no difference in the rate at which ALSFRS-R decreased between those treated and those in the placebo arm. An abstract from the presentation at AAN was not available online at the time of this post, nor was there additional information or data present on the company’s website.
NP001 safety and tolerability were established in earlier trials. During secondary analysis of data from the Phase 2A trial the company's investigators identified a ‘responder’ group based on pretreatment levels of C-reactive protein (CRP); a general measure of systemic inflammation.
Based on those post hoc findings, the company limited enrollment to those with elevated levels of CRP. Between September 2016 and January 2018, 138 people with ALS were enrolled and participated in the clinical trial across 21 different research sites in the US. Analysis shows there was no difference between those treated and those in the placebo group, according to an initial report from AAN reporter Jamie Talon. Further, Talon reported that there was no difference in decline rate of inflammatory markers, slow vital capacity, or time to tracheotomy between the two groups. While the data is not yet available for review, based on this early report the trial missed all endpoints regardless of being optimized from the start for the proposed responder group identified in the earlier Phase 2A study.
As the data is considered and analyzed, it is crucial to remember that nearly 300 people with ALS have participated in clinical trials on NP001 over the last decade. ALS TDI would like to recognize the heroic contribution each of those individuals made.
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NOTE: The article “Hopeful ALS Treatment Fails Phase 2” appeared online Monday April 23 for a brief period of time before being removed. It was authored by AAN reporter, Jamie Talon. A member of the ALS Forum copied and pasted the full text of that article into the ALS Forum, which can be read here.