PrimeC, a new drug used to treat Amyotrophic lateral sclerosis (ALS) disease by NeuroSense Therapeutics Ltd. has been successful in proving its efficacy in its clinical study Phase 2b and it’s beginning to gear up to the final Phase 3, the drug's developers announced.
The disease, ALS, also famously known as Lou Gehrig’s after the baseball player who died of the disease, is a disease that affects the nervous system. ALS affects nerve cells in the brain and spinal cord and progressively gets worse over time. This causes muscle control problems such as weakness in the limbs and slurred speech. As the disease develops, it becomes fatal, as those with the disease begin to struggle to breathe and eat.
Findings are from an 18-month Phase 2b clinical study. During Phase 2b, the objectives of the research are confirmed, determining optimal administration methods and overall therapeutic effects. The study is following PrimeC, a combination therapy focused on inhibiting the progression of ALS. The lab that discovered and developed the drug is an Israeli biotech company– Neurosense Therapeutic Ltd.
PrimeC has been given an ‘Orphan Drug’ status by the FDA (Food and Drug Administration) and EMA (European Medicines Agency). The status of ‘Orphan Drug’ is given to pharmaceutical agents that are developed to treat rare medical conditions.
PrimeC’s Development and Phase 2b
The Phase 2b trial was randomized, double blind, and placebo-controlled. This was to ensure the efficacy of PrimeC and examine changes in the ALSFRS-R’s of trial participants.
ALSFRS-R, short for the ALS Functional Rating Scale, is a series of validated rating instruments used to monitor the progression of disability with patients with ALS. Monitoring a person includes tracking changes in a person’s physical ability to walk, speak, or eat. ALSFRS-R scores correlate significantly with quality of life.
There were 68 participants involved in the trial coming from Canada, Israel and Italy. Participants of the trial were either given PrimeC or the placebo twice a day for six months.
All endpoints were reached and proved the positive outcomes of using PrimeC, showing the differences of participants who used PrimeC compared to participants who were given a placebo and then transitioned to PrimeC.
The outcome of the study showed meaningful signs of efficacy of the drug, with a 29% difference between the PrimeC group and the placebo group. An additional analysis showed a significant slowing of disease progression with a 37.4% (p=0.03) difference in ALSFRS-R, showing favor for PrimeC.
Public response
With the successful results of PrimeC, the drug has opened more opportunities for treatment and long-term results in the health of people diagnosed with ALS.
"These exciting long-term results demonstrate how study participants experienced more slowing of progression over time with PrimeC as measured against ALSFRS-R, which is the current gold-standard scale used in ALS drug development," The Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital Merit Cudkowicz, M.D., M.Sc. said.
"The need for new treatments for people living with ALS has never been greater. PrimeC has great potential based on its mode of action and the Phase 2 trial results and warrants further evaluation in a Phase 3 trial in an expeditious manner." She added.