Avicena’s in-depth understanding of how to modulate cellular energy levels has specific applications in the treatment of a number of debilitating neurological and neuromuscular diseases, including ALS (Lou Gehrig’s disease), Huntington’s disease and Parkinson’s disease. As such, the company is collaborating with renowned clinicians at leading academic institutions to leverage its proprietary cellular energy modulation and cellular energy transfer platforms for the development of much needed therapeutics. Avicena’s drug candidates are designed to prolong cell life and, in turn, ease the symptoms of a disease and/or slow the disease’s progression.
Amyotrophic Lateral Sclerosis (ALS)
Duchenne Muscular Dystrophy
Amyptrophic Lateral Sclerosis (ALS), or "Lou Gerig's disease," is a progressive neuro-degenerative disease that inhibits
an individual's ability to control voluntary muscle movement. Affecting the brain and spinal cord, leads to paralysis and eventually
death. About 30,000 Americans suffer from ALS.
The Avicena Group is developing three novel drug candidates for the treatment of ALS: ALS-02, ALS-05 and ALS-08. ALS-02, the company's
lead drug candidate, has received orphan drug designation from the FDA and recently completed its first Phase III clinical trial.
Findings from this trial, when combined with data from a second study of ALS-02 previously conducted by the North East ALS Consortium
(NEALS), demonstrated a positive trend toward decreased mortality in subjects receiving ALS-02. Investigators are continuing
to review data from both trials. Upon completion of this analysis, Avicena intends to discuss the findings, including the mortality trend,
with the United States Food and Drug Administration (FDA) in order to determine the appropriate next development steps for ALS-02.
The company also recently announced the initiation of a Phas II clinical trial of two combination therapies including ALS-08. Additionally,
ALS-05 is being evaluated in a pilot study at the Carolinas ALS Center.
Huntington's Disease is a progressive neurodegenerative disease that is caused by a defective gene. This genetic defect, which is
often inherited, causes the deterioration of neurons in those parts of the brain that are responsible for controlling cognitive, emotional and motor functions. In the United States, approximately 35,000 people suffer from HD.
Avicena is presently developing HD-02, a novel drug candidate for the treatment of HD which has been granted orphan drug designation by
the FDA. The company has greatly completed a Phase I/II clinical study of HD-02 at Massachusetts General Hospital. Results from this
study, which were published in the journal Neurology, showed HD-02 to be safe and well-tolerated at the administered dose.
In addittion, findings showed an increase in serum and brain creatine levels and a reduction in an oxidative marker
in the treatment group.
In preclinical studies performed by Dr. Flint Beal of Cornell Medical Center, this compound has shown significant neuroprotective effects
such as improved motor movement and increased survival rate.
Parkinson's Disease (PD) is a progressive, neurodegenerative disease that occurs when neurons within the brain that are responsible
for producing the chemical dopamine become impaired or die. Once approximately 80% of the brain's dopamine producing cells no longer
function, the symptoms of Parkinson's disease appear. Approximately 1.5 million Americans suffer from Parkinson's disease with roughly
60,000 new cases diagnosed each year.
Avicena's lead Parkinson's disease drug candidate, PD-02, recently completed Phase II efficacy trials at the University of Rochester.
Data from the study, which was published in the journal Neurology, showed PD-02 to be safe and tolerable at the dosage given.
Furthermore, findings demonstrated that the rate of disease progression for PD-02 was lower than the threshold for futility, as measured
by the United Parkinson's Disease rating Scale (UPDRS). This indicates that further study of PD-02 is warranted as a treatment for
Duchenne Muscular Dystrophy (DMD) is a genetic, degenerative muscular disease caused by a recessive defect on the X chromosome.
This genetic defect prevents the body from producing dystrophin, a protein which protects the muscle fiber membrane and is required to
maintain muscle strength. Patients with DMD, which strikes approximately one in every 3,500 boys, rarely survive beyond their early 30s.
The Avicena Group is presently developing a novel drug candidate for the treatment of DMD: DMD-02. A double-blind, randomized, cross-over
study was conducted by Dr. Mark Tarnopolsky of McMaster University. Results of this study demonstrated an increase in muscle strength and fat-free muscle mass (FMM), as well as a decrease in bone breakdown.
Charcot-Marie-Tooth Syndrome (CMT) is a progressive neuromuscular disease that causes the deterioration of nerves responsible for controlling the muscles of the hand, arm, foot and leg. Caused by specific genetic mutations, CMT is estimated to affect approximately 150,000 Americans. However, experts estimate that about 300,000 Americans may actually have the disease.
The Avicena Group is developing a novel drug candidate for the treatment of CMT: CMT-02. A pilot study has been conducted by Dr. Robert Chetlin and Dr. Laurie Gutmann, West Virginia University School of Medicine. Results demonstrated a significant increase in the concentration of creatine in muscles, as well as an increase in leg muscle size when CMT-02 was combined with resistance training.