AI Therapeutics Announces Initiation of a Phase II Clinical Trial of AIT-101 for Treatment of ALS

Promising New Approach for a Fatal Neurological Disorder

GUILFORD, Conn., Jan. 13, 2022 (GLOBE NEWSWIRE) -- AI Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today the initiation of a Phase II study for a promising new approach to treat amyotrophic lateral sclerosis (ALS). ALS, also known as Lou Gehrig’s Disease, is a fatal neurological disorder that affects approximately 30,000 patients in the United States, with an average life expectancy of three to five years. AI Therapeutics’ drug candidate AIT-101 employs a novel approach to clearing toxic protein aggregates that accumulate in the brains of ALS patients and are a hallmark of the disease.

“We are excited to have begun our first clinical trial for AIT-101 in ALS patients and are optimistic that it has the potential to change the course of this terrible disease,” said Dr. Murat Gunel, Chair of the Department of Neurosurgery and Professor of Genetics and Neuroscience at Yale University and Chairman of the Scientific Advisory Board of AI Therapeutics. “AIT-101 is a very promising molecule as it is incredibly specific for its target, PIKfyve kinase, and has been safely dosed in hundreds of patients in other clinical trials. The potential therapeutic value for PIKfyve kinase inhibition in ALS and other neurodegenerative diseases was initially predicted by both the AI Therapeutics’ Guardian Angel™ Platform and by our own observations.”

“ALS is an ultimately fatal neurodegenerative disease with few effective treatments available. AIT-101 employs a novel approach in aiming to clear the abnormal protein aggregates that lead to motor neuron degeneration,” added Dr. Katharine Nicholson, a neurologist and ALS clinical trialist at the Sean M. Healey Center for ALS at Massachusetts General Hospital in Boston. “The AI Therapeutics trial focuses on ALS patients with the C9orf72 mutation (C9ALS) in whom we can effectively measure the breakdown of proteins specific to the C9 repeat expansion as well as proteins found in all people with ALS. The study is designed to evaluate AIT-101 in this subpopulation of ALS prior to consideration of a larger trial in the broader ALS population, and importantly incorporates access to the drug for all participants.”

The 12-week, randomized, placebo-controlled study will enroll 12 patients to evaluate the safety, tolerability, pharmacokinetics, and effect of AIT-101 on biomarkers of target engagement, toxic protein aggregates and neurodegeneration. Functional measures, including the ALSFRS-R (Revised ALS Functional Rating Scale), Vital Capacity and ALS-CBSTM (ALS Cognitive Behavioral Screen), will also be assessed throughout the study. After the initial 12 weeks, all patients will be offered AIT-101 for an additional 12 weeks. Additional information is available at (NCT05163886).

About AIT-101
AIT-101 is a first in class, highly selective PIKfyve kinase inhibitor that represents a promising new approach to treat ALS. AIT-101 treatment has been demonstrated to activate the transcription factor TFEB, which drives the clearance of toxic protein aggregates. TFEB is known to be reduced in the brains of ALS patients. The Phase II clinical trial will use LAM-002A, the current formulation of the active ingredient of AIT-101.

About AI Therapeutics
AI Therapeutics was founded by Dr. Jonathan Rothberg, serial entrepreneur and Recipient of the National Medal of Technology and Innovation for inventing high speed “Next-Gen” DNA sequencing, with the goal of utilizing artificial intelligence to accelerate the clinical development of drugs for rare diseases. The company is building out an expansive rare disease pipeline with the help of its Guardian Angel™ Platform, a suite of artificial intelligence tools that use deep learning to understand complex disease biology and the action of potential new therapeutics. To learn more, visit:

About 4Catalyzer
AI Therapeutics is part of biotechnology accelerator 4Catalyzer, which has launched Butterfly Network, Quantum-Si, Hyperfine and Liminal Sciences, Detect, Tesseract, and Protein Evolution. All of the 4Catalyzer companies strive to transform 21st century medicine and improve our planet by solving today’s most challenging problems across life science research tools, medical devices, therapeutics and the environment. To learn more, visit: