SOUTH CAROLINA – September 16, 2025 – Aquilus Pharmaceuticals is dedicated to developing a first-in-class medication for neurodegenerative diseases, primarily focusing on Amyotrophic Lateral Sclerosis (ALS). ALS is a rare, multisystem, multifactorial disease with peripheral contributions to immune-mediated loss of upper and lower motor neurons. It causes paralysis and muscular atrophy, impairing the ability to speak, move, and eventually breathe. Its mission is to make a meaningful impact on patients’ lives by advancing therapeutics in this challenging field.
Solutions
Although there are currently no cures and few treatment options for ALS, Aquilus has developed a small molecule called AQU-118, a safe, orally bioavailable, semi-selective inhibitor of MMP-9 and MMP-2. It is known to be involved in the disease processes of neurodegenerative diseases, including ALS. AQU-118 has been found to significantly reduce nerve damage in several different rodent animal models of neuropathic pain and nerve injury, as well as mitigate weight loss and reduced grip strength in the TDP-43 overexpressed mouse model of ALS.
Strengths
- AQU-118 would be a first-in-class therapeutic for ALS.
- All pre-clinical Investigational New Drug (IND) enabling studies have been completed.
- IND filed with the FDA to begin a phase 1 trial.
- Full Human Research Ethics Committee approval to conduct the phase 1 trial in Australia.
- Partial funding for phase 1 trial secured by an ALS Association grant award.
Meet the Co-Founders
From left to right:
Irving Sucholeiki, Ph.D. | President and Chief Executive Officer
Roy Sucholeiki, MD | Chief Medical Officer, Vice President of Clinical Development