ALS Association Supports the Development of Four New Therapies with 2023 Clinical Trial Awards


There is an urgent need for new and improved therapies for ALS, as there is still no cure. To help accelerate drug development and get promising treatments one step closer to regulatory evaluation, the ALS Association has committed nearly $4 million to support four early-stage clinical trials through our Clinical Trial Awards.

Because ALS is such a complex disease with multiple underlying causes, it’s unlikely there will be a single ‘silver bullet’ that will solve everything. Therefore, having a robust pipeline of diverse new therapeutic approaches is essential to making ALS a livable disease for everyone, everywhere."
Dr. Paul Larkin
Director of Research at the ALS Association

Our Clinical Trial Awards provide up to $1 million over three years to support phase 1 or phase 2a trials that generate important data on things like safety, dosing and biomarkers. These results help make the next steps in drug development, usually much larger clinical trials, less risky and more attractive to future investors. That’s important because it can cost more than $2 billion to develop a new drug. 

“For many of our Clinical Trial Awardees, this is the first time people living with ALS will receive their investigational agent or repurposed therapeutic. This is a major milestone for these programs, and we are proud to support them at such a critical time in their development,” Dr. Larkin added.

This year’s Clinical Trial Award recipients have very different approaches to treating ALS and managing the harm it causes:

Aquilus Pharmaceuticals plans to conduct a phase 1 trial of an oral experimental drug called AQU-118, which was designed to block proteins involved in inflammatory processes that are elevated in people living with ALS. The trial will assess the safety and tolerability of the drug in healthy volunteers. This work builds on previous preclinical studies of AQU-118, which we supported through a two-year Lawrence and Isabel Barnett Drug Development Program grant.

InFlectis BioScience is testing IFB-088, which was derived from an FDA-approved drug called guanabenz that is used to treat high blood pressure. The phase 2 trial will see if IFB-088 can slow disease progression for people living with bulbar-onset ALS and will also evaluate the drug’s safety.

ProJenX will conduct a phase 1c study of a new drug called prosetin that was designed to protect motor neurons from the destructive processes triggered by ALS. This study will evaluate the safety, tolerability, absorption, distribution, metabolism and elimination (pharmacokinetics) of various doses of prosetin in people living with ALS to help select the optimal dose to test in future studies.

Swathy Chandrashekhar, M.D., an assistant professor of neurology at University of Kansas Medical Center, is conducting a phase 2a/2b trial of ranolazine, an FDA-approved drug for angina, to see if it can help relieve cramps in people living with ALS. The trial will also assess the drug’s safety and tolerability.

For more information about ALS clinical trials, visit our website HERE.  

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Submitted by: Karen M. on Sat, 01/06/2024

My sister had just been diagnosed with ALS. We are all holding out hope that she will be able to live longer than most with a good quality of life.

Submitted by: Stephanie O. on Tue, 01/09/2024

Karen, I am holding out that same hope. Please refer your sister to her local care team for more support:

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