The applicant submitted the BLA on September 9, 2022. FDA...

The applicant submitted the BLA on September 9, 2022. FDA conducted a filing review and determined that a substantive review could not be performed, because the BLA submission was scientifically incomplete and grossly deficient. Critical clinical and manufacturing deficiencies were identified. For clinical, the completed randomized, placebo-controlled clinical studies failed to show efficacy in their prospectively specified efficacy endpoints to demonstrate required substantial evidence of effectiveness. For manufacturing, the required Chemistry, Manufacturing, and Controls information covering several critical categories was not included in the application, and the level of information included was insufficient to perform a full assessment of product quality. Consequently, FDA issued a refuse-to-file letter to the Applicant on November 8, 2022.

The Phase 3 pivotal trial of NurOwn did not reach statistical significance on the primary or secondary endpoints, likely due to a "floor effect," which confounds measurement of disease progression in patients with more advanced disease.

Ok, NurOwn bowled out in the innings.

Whats the difference between ourselves.



1. MSC ( Mesenchymal Stem Cell therapy) half life of 24 hours with patients age related diminished limited amount of cell population doublings. It is hard to establish if protein denigration is evident.

2. CA risk due to MSC stimulatory 11effects on tumour pathogenesis including B cell Lymphoma, T cell Lymphoma, Colon , skin and some breast cancers lines.

3. Studies show increased Cancer after one year diagnosis of MND include Lymphoid Leukaemia, Non Hodgkins Lymphoma and basal cell carcinoma. ( MND and risk of cancer: A population based cohort study in Denmark 2020).Hippocratic oath : " First , do no harm".

4. Needs patient screening before administration in my thoughts.

5.It needs to be administered to patients on dietary exercised based regimes for full benefit.

6. ALSFRS rating on endpoint of study not achieved. ( was 1.25 points per month).

7. Longevity possibly not achieved.

8. No difference on CAFS score at end of trial.

9. The idea of MSC reprogramming microglial cells is beneficial.

10. Combining MSC NurOwn with an MSC CXCR6 inhibitor signalling pathway would be beneficial. Does NurOwn MSC cause an exacerbation in pulmonary fibrolasts due to overexpression of CXCR6 . MND patients are at risk of same related to age groupings.

11. Their trial did have a percentage of cohorts below 35 in the ALSFRS and as would be expected no improvement.

12. MiR-146a expression evident on MSC NurOwn administration increase the risk of polymorphism and are associated with the risk of neurological disease. Does this counteract the benefits of MSC.

13. More deaths in treatment protocol than placebo.Though these were with patients with low ALSFRS scores or low FVC %. One has to wonder why these patient were on trial as it comprises their own safety data.

14. Severe AE 29 from 95 in study. Serious AE 23 from 95 in study.

15. Bipap usage % no change placebo v MSC .

16. PD data for TGF-B1, Galectin -1 and NFL show synergistic effect for 2 weeks then steady deterioration after that. Is this due to my number 1 dot point.


Ok HC it's my notes on NurOwn scribed a while back when chatting to MT.

Main differences= adverse effects, ALSFRS score, no subsequent morphism to other conditions such a cancer with MPL, half life of MPL 72 hours v 24 hours , more deaths.


You will remember the Switzerland Symposium late last year was littered with failed studies for MND.

Anyway, fantastic news of our next cohort being 210 patients for our own MND trial.
Pharmaust getting serious now girls and guys.

I wonder which neurological condition we will attempt next ? Alzheimer's is it the favourite!
Its my pick.

Kpax