July 18, 2016 8:54am

A P3 will validate the reality and benefit of the treatment, the shares are UP +$0.16 or 5.35% having succumbed to a $0.47 downdraft by “hyping” a pre-release “stunt”

 


 

BrainStorm Cell Therapeutics (BCLI), results from the recently pre-released U.S. randomized, double-blind, placebo-controlled P2 Study of NurOwn® in patients with ALS achieved its primary objective - safety and was well tolerated. 

 

NurOwn achieved multiple secondary efficacy endpoints, showing clear evidence of a clinically meaningful benefit.  Notably, response rates were higher for NurOwn-treated subjects compared to placebo at all time points in the study out to 24 weeks.

The P2 trial was a randomized, double-blind, placebo-controlled multi-center study designed to evaluate the safety and efficacy of NurOwn in 48 ALS patients. 

  • It was conducted at three sites in the U.S: Massachusetts General Hospital, UMass Medical School and the Mayo Clinic. Patients were randomized to receive NurOwn cells administered via combined intramuscular and intrathecal injection (n= 36), or placebo (n=12).
  • They were followed monthly for approximately three months before treatment and six months following treatment, assessed at 2, 4, 8, 12, 16 and 24 weeks.

 

The Bottom Line: The released P2 data produced a transient and clinically meaningful beneficial response in terms of both the ALS-FRS-R rating scale and CSF biomarkers. These exciting findings a longer study must be conducted with a longer study and repetitive dosing. The absolute point improvement/month in ALSFRS-R post the treatment slope compared to pre-treatment slope over time showed evidence of a NurOwn treatment effect

The increased levels of growth factors in the cerebrospinal fluid and decreased inflammatory markers observed after two weeks are encouraging evidence for a biological effect. Based on these results, repeat dosing at 8 to 12 weeks and a larger confirmatory trial are warranted.

 

BCLI closed at $2.99 after being DOWN -$0.47 or 16.01% on Friday and is UP +$0.16 or +5.35% to $3.15