Commitment to Neurotherapeutics
Aquilus Pharmaceuticals is dedicated to developing a first-in-class medication for neurodegenerative diseases, with a primary focus on Amyotrophic Lateral Sclerosis (ALS). Our mission is to make a meaningful impact on patients' lives by advancing therapeutics in this challenging field
What is ALS?
Amyotrophic Lateral Sclerosis (ALS) is a rare multisystem multifactorial disease with peripheral contributions to immune-mediated loss of upper and lower motor neurons causing paralysis and muscular atrophy impairing the ability to speak, eat, move and eventually breathe. Age at diagnosis ranges from 40-70 years but can occur as young as 20-30 years old. While most cases are sporadic, 5-10% are familial.
Life expectancy is 2-5 years after diagnosis
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ALS association.
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Maksimovic K. Evidence of Metabolic Dysfunction in ALS. Patient and Animal Models. Biomolecules. May 2023
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Mentis AFA. ALS and the Endocrine System: Are There Any Further Ties to Be Explored? Aging Brain. 11/8/2021
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Zhang S. Genome-wide Identification of the Genetic Basis of ALS. Neuron. Volume 110. Issue 6, 3/16/2022.
LATEST
RESEARCH INITIATIVES
Matrix Metalloproteinases (MMPs)
MMP's are a family of around 24 zinc-dependent endopeptidases. As a family, MMPs can cleave virtually any component of the extracellular matrix, and thereby facilitate cell migration and affect cellular signaling that regulates cell proliferation, differentiation, and cell death. In addition to cleaving the structural components of the extracellular matrix, MMPs are involved in the processing & signaling of growth factors and their receptors, cytokines, chemokines, adhesion molecules, and a variety of other enzymes such as caspases. A growing body of evidence has linked MMP-9 and MMP-2 with the severity and rate of progression of ALS. Currently, there are no FDA approved MMP inhibitors.
Aquilus' MMP inhibitor AQU-118
Aquilus has developed a small molecule called AQU-118 that is a safe, orally bioavailable semi-selective inhibitor of MMP-9 and MMP-2. It 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 over-expressed mouse model of ALS.
*Henry, M.A. Effect of a novel, orally active matrix metalloproteinease-2 and-9 inhibitor in spinal and trigeminal rat models of neuropathic pain. J. Oral Fac. Pain Headache 2015, 29, 286–296.
*Kwan, M.Y. Biomarker analysis of orally dosed, dual active, matrix metalloproteinase (MMP)-2 and MMP-9 inhibitor, AQU-118, in the spinal nerve ligation (SNL) rat model of neuropathic pain. Int. J. Mol. Sci. 2019, 20, 811.
OUR FOCUS
RESEARCH & DEVELOPMENT
Aquilus is committed to driving progress in neurodegenerative disease treatment through cutting-edge research and development efforts.
CLINICAL TRIALS
Aquilus has completed all necessary IND enabling studies for AQU-118 and currently on track towards a phase I trial in the first quarter of 2025. Plans for the recruitment for the phase II trial to begin in the fourth quarter of 2025
CONNECTING WITH THE COMMUNITY
We partner with people who are directly impacted by neurodegenerative disease to give us guidance from the patient and caregiver perspective
Senior Leadership
Our core team of experts is driven by a shared passion for making a difference in the lives of patients facing neurodegenerative diseases.
Irving Sucholeiki, Ph.D
President & Chief Executive Officer
Darrell J. Nix, Ph.D.
Vice President, R & D
Roy Sucholeiki, M.D.
CMO & Vice President, Clinical Development
Dr. Sucholeiki leads our research initiatives, leveraging his 25 + years of experience in drug discovery and development to drive our scientific endeavors forward.
With over 20 years of industry experience as a pharmacologist, Dr. Nix guides the regulatory requirements for Aquilus' goal of FDA approval
With over 25 years of clinical experience and academic research, Dr. Sucholeiki spearheads Aquilus' clinical trials path
OUR PARTNERS
OUR COLLABORATORS AND ADVISORS
Robert Bowser, Ph.D, Chairman, Dept of Neurobiology, Barrow Neurological Institute
Merit Cudkowicz, M.D., MSc., Professor, Harvard Medical School & Director, Sean M. Healey & AMG Center for ALS
Sabrina Paganoni, M.D., Associate Professor, Harvard Medical School & Co Director, MGH Neurological Clinical Research Institute
Daniela Zarnescu, Ph.D, Professor of Molecular & Cellular Biology, University of Arizona
Professor Rita Sattler, MSc., Ph.D., Associate Professor, Barrow Neurological Research Institute
NEWS
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2024 Human Research Ethics Committee Approval to Begin Phase 1 Single and Multiple Ascending Dose Clinical Trial
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2023 The Hoffman ALS Clincal Trial Grant Award ($1,000,000)
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2021 Department of Defense ALS Research Program Therapeutic Development Grant Award ($ 1,000,000)
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2021 Lawrence and Isabel Barnett Drug Development ALS Association Grant Award ($ 500,000)
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2019 National Institutes of Health Small Business Innovation Research Grant Award ($ 457,639)