BrainStorm Cell Therapeutics Inc (BCLI)

[Fmello]

https://www.linkedin.com/feed/update/urn:li:share:6964070853491777536?utm_source=linkedin_share&utm_medium=member_ios_link_share&utm_content=post

Michelle Lorenz - 16h

Powerful News in the #ALS community today!

Brainstorm Cell ($BCLI) published an erratum in the journal Muscle & Nerve. This updated data from the 28 week #NurOwn Phase III trial PROVES that #NurOwnWorks:

• Trial data met statistical significance (.05 p-value)
• Pre-specified secondary endpoint of 35+ ALSFRS met statistical significance
• NurOwn performed significantly better than placebo for people with ALSFRS of 27+
• People's ALSFRS were 1.94-2.86 pts better on NurOwn over 28 wks trial
• Clinically meaningful improvement in how patients feel & function

This trial data confirms what people in the Phase III trial & Expanded Access have been saying for years! #NurOwnWorks to slow progression in 100% fatal #ALS. Some people who received NurOwn shortly after diagnosis actually regained function & had improved breathing -- unheard of in ALS.

NurOwn is a mesenchymal stem cell treatment. Patients have their bone marrow harvested then the stem cells are treated with NurOwn in a lab. Appx one month later, it is reinjected into the CSF where the stem cells act like a Fed Ex truck, dropping off packages of NurOwn across the CSF.

ALS is a 100% fatal disease with no disease modifying treatments; and thus, there is a critical unmet need.

NurOwn is the 1st trial in ALS history that has appx 12+ CSF biomarkers proving the biological effect matches the clinical effect above.
• #Neurodegeneration decreased
• #Neuroinflammation decreased
• #Neuroprotection increased

These biomarkers include #Neurofilament Light (NfL), VEGF, BDNF, MCP-1 and many more!

With the strength of the biomarker data (surrogate endpoints), combined with the fact that it met statistical significance on both the secondary endpoint as well as the pre-specified endpoint, along with profound patient reported outcomes of regaining function, the totality of the evidence makes a much stronger case for #FDA approval using the #AcceleratedApproval pathway.

The erratum resulted from a statistical programming error.

• Read the erratum:
https://lnkd.in/gmzh5an9

• The original publication in muscle & Nerve in Oct 2021:
https://lnkd.in/gXu7vYCZ