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AI Therapeutics' narrowed ambitions for ALS drug pay off as phase 2/3 beckons
05 Apr 2023
Phase 2Clinical Result
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 lyALSoma and COVID-19. But those ambitions have come and gone.
AI Therapeuticsy is pointing to new phase 2AIT-101in 15 patients with a form ofollicular lymphomaal scCOVID-19(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 tamyotrophic lateral sclerosis (ALS)ntribute 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.ALS
The company plans to launch a phase 2/3 trial ofAIT-1011 in a broader ALS population in 2024, CEO Brigette Roberts, M.D., told Fierce Biotech.ALSYale University
AIT-101 works by clearing toxic protein aggregateAIT-101tor neurons. IALS 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 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.
AIT-101d 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 thamyloid-beta plaquesr COVID-19. The two launched a phase 2 trial dementiathe drug in patients with confirmed infectionapilimod resPIKfyve kinaser reported.cancersCOVID-19AI Therapeutics
Yalee theAI Therapeuticsunched, the company has recalibrated, electing to focus apiapilimod neurodegenerative diseases. The biotech is also developing an inhaled version of siroCOVID-19he compound behind Pfizer’s lung disease drug Rapamune, and oncology med AIT-10infection
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