According to a recent article in ALS News Today, the FDA’s Orphan Status designation has been granted to NeuroSense Therapeutics for its drug, PrimeC, to treat ALS.
PrimeC received the designation as a result of two pre-clinical studies. It is currently being evaluated in two newly-initiated trials studying the drug’s safety and efficacy on humans.
About PrimeC
The novel combination drug has been designed to slow or even halt the advance of amyotrophic lateral sclerosis (ALS).
PrimeC received the designation as a result of two pre-clinical studies. It is currently being studied at two newly-initiated trials.
The drug combines ciprofloxacin, an antibiotic for bacterial infection, with celecoxib, an anti-inflammatory medication.
The combination is aimed at inflammation that occurs in the nervous system of ALS patients. It also targets RNA processing errors.
The Orphan Drug Status Designation
The orphan drug status allows the manufacturer to qualify for seven years of exclusivity in the market, the FDA’s support, and fee exemptions or reductions.
About the Pre-clinical Studies
The studies were conducted using zebrafish with mutations that cause ALS. The zebrafish were administered PrimeC therapy resulting in the fish exhibiting a significant rebound. Zebrafish have been used successfully in several other medical studies.
Another positive result after PrimeC treatment was an increase in the protection of the cellular function and structure of the nervous system (neuroprotection).
Two Clinical Trials Investigating PrimeC in Humans
Phase 1 (NCT04090684) clinical trials will be conducted at two sites in the United States.
Phase 2a (NCT04165850) clinical trial will be conducted at an Israeli site in Tel-Aviv.
Both Phase 1 and Phase 2a trials will be investigating the tolerability, efficacy, and safety of PrimeC in the treatment of ALS clinical trial participants.
PrimeC will be administered to patients for fifteen months. Phase 1 subjects will take two capsules each day while Phase 2a subjects will take three capsules daily.
The Anticipated Canadian Trial
NeuroSense intends to advance its development of PrimeC through an upcoming study of healthy volunteers analyzing how the treatment affects the body. Their focus will be on distribution, metabolism, excretion and absorption. The Canadian trial is slated to begin later this year.
The Next Step
A Phase 2/3 trial will be conducted after preliminary data from the current trials are made available. In these trials PrimeC will be studied in comparison to a placebo.
The trial will involve approximately two hundred participants including patients with familial (inherited) ALS as well as patients with sporadic ALS.
ALS is usually not inherited. About ninety percent of cases involves only one member of a family (sporadic ALS).
The success of these trials may lead to the conditional approval of PrimeC in the United States. NeuroSense has proclaimed a four-year goal for PrimeC as a viable treatment.
What are your thoughts about the role of PrimeC as a treatment for ALS? Share your stories, thoughts, and hopes with the Patient Worthy community!
