ALS
The FDA is putting Atara’s active Investigational New Drug applications on hold due to manufacturing concerns at a third-party provider while releasing Amylyx’s investigational ALS therapy from a previous pause.
Misses from amyotrophic lateral sclerosis hopefuls Denali Therapeutics and partners AbbVie and Calico Life Sciences mark the latest setbacks for the controversial platform trial, the results from which have largely mirrored the dismal success rate in ALS overall.
Denali’s failure on Monday continues biopharma’s losing streak against amyotrophic lateral sclerosis. PTC Therapeutics and Amylyx have seen similarly disappointing results.
This year saw lofty highs and devastating lows for neuroscience drug developers like Bristol Myers Squibb, Eli Lilly and AbbVie, following the predictable pattern of successes and failures that characterizes this space.
Some 90% of investigational drugs fail—and success rates are even more dire in the neuro space. Here, BioSpace looks at five clinical trial flops that stole headlines over the past 12 months.
Launched in 2020 to more quickly bring to market an effective medicine for amyotrophic lateral sclerosis, the HEALEY Platform Trial has generated disappointing results for many but also continuing programs from Clene and Prilenia.
While supportive of Amylyx’s acquisition of a GLP-1 drug, analysts say the company’s future hinges on key upcoming readouts from multiple products in its pipeline.
After a long and challenging journey for its stem cell therapy NurOwn, BrainStorm Cell Therapeutics has aligned with the FDA on the parameters of a Phase IIIb ALS trial that is expected to begin by the end of 2024.
Patient assistance programs may actually be a two-way street, providing patients with drugs and companies with data.
Disappointed with Phase I/II results for two Ionis-partnered programs, one for amyotrophic lateral sclerosis and another for Angelman syndrome, Biogen has opted to not proceed with their development.
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