AI Therapeutics' narrowed ambitions for ALS drug pay off as phase 2/3 beckons

Phase 2Clinical Result
AI Therapeutics' narrowed ambitions for ALS drug pay off as phase 2/3 beckons
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Source: FierceBiotech
AI Therapeutics reported that its lead asset knocked down concerning protein aggregates in ALS patients.
AI Therapeutics has cast a wide net around AIT-101 with indications including follicular lymphoma and COVID-19. But those ambitions have come and gone.
Now, the company is pointing to new phase 2a data in 15 patients with a form of amyotrophic lateral sclerosis (ALS), saying the results show promise. AI announced Wednesday that AIT-101—formerly LAM-002A— hit the primary endpoints of safety, tolerability and successful drug deliverability and that critical biomarkers are trending in the right direction. Specifically, the company found a 73% reduction in poly(gp) levels, a toxic protein aggregate considered to be a viable biomarker for ALS therapies.
“This study provides early clinical evidence of AIT-101’s ability to clear the toxic protein aggregates that may contribute to the adverse pathology of ALS and provides a strong rationale for further clinical studies,” said Murat Gunel, M.D., professor of genetics and neuroscience at Yale University as well as a member of AI’s scientific advisory board, in the release.
The company plans to launch a phase 2/3 trial of AIT-101 in a broader ALS population in 2024, CEO Brigette Roberts, M.D., told Fierce Biotech.
AIT-101 works by clearing toxic protein aggregates in motor neurons. It's a familiar strategy, with much of the work in Alzheimer's aimed at knocking down amyloid-beta plaques, worrisome protein buildups associated with dementia. The name of the asset’s formal compound is apilimod, a PIKfyve kinase inhibitorPIKfyve kinase inhibitor that’s previously been touted as having potential therapeutic benefits against certain cancers and COVID-19, indications that AI Therapeutics has tried to target in the past.
Yale and AI Therapeutics sprang into action in July 2020 after research pointed to apilimod as one of the most likely existing drug candidates to have a therapeutic benefit for COVID-19. The two launched a phase 2 trial testing the drug in patients with confirmed infection but the results were never reported.
Since the COVID trial launched, the company has recalibrated, electing to focus apilimod on neurodegenerative diseases. The biotech is also developing an inhaled version of sirolimus, the compound behind Pfizer’s lung disease drug Rapamune, and oncology med AIT-102.
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