BrainStorm Cell Therapeutics Inc (BCLI)

[Fmello]

Saw this post from the No More Excuses Facebook page:

Michelle Lorenz
8 hrs

NurOwn Primary Endpoint
When people tell you that NurOwn doesn't work, this Table 4 proves that it does. This is from the Phase III study published in Muscle & Nerve.

To meet the FDA's threshold of "substantial evidence" that a therapy provides a "clinically meaningful" benefit, a company has to prove two things in its trial:

1) The therapy works
2) The therapy worked on enough people to trust the statistical conclusions. This is what researchers & statisticians refer to as a p-value of .05.

We measure if a therapy works by asking if the data met the trial's endpoints. The NurOwn trial primary endpoint measured if people slowed their decline by -1.25 points per month.

In a pre-specified group of people with ALSFRS-R scores of 35+, there was evidence that NurOwn worked, but there wasn't enough people to meet the p-value. At 35+ ALSFRS-R, there were only 9 of 26 who met the endpoint.

But when you look at the clinical data of the people who had ALSFRS-R scores of 26+, there is "substantial evidence" proving NurOwn works.

• Appx 35%+ of people slowed their decline by -1.25/mo
• Once the sample size was appx 100, the data was "statistically significant"

Additionally, the Phase III biomarker data presented by Bob Brown provides further support that the NurOwn trial resulted in both clinical and biological evidence of efficacy.

Other Thoughts:

• 10,500 people
We don't know how many people currently have an ALSFRS-R above 26; however, at some point everyone is. Over a 5 year period, appx 30,000 Americans are diagnosed with ALS. If we applied this NurOwn trial outcome to 30,000 people, this would mean 10,000+ people would have a -1.25 point/month slowing in their decline with just 3 doses of NurOwn. Over 10,000 lives that could be impacted... this is why we believe #SomeIsEnough

• Dose-Dependent Response.
We don't yet have published clinical data to prove if more doses means more slowing, how long that -1.25/month slowing will be sustained, or if a slowing of decline will be sustained over a longer period of time, but the "patient reported outcomes" from the people in EAP and from Matt Bellina both seem to indicate a trend toward a dose-dependent response.

• People with ALSFRS-R below 26
We've witnessed patient reported outcomes that show NurOwn works on some people with scores below 26. Plus, with 6 consecutive doses, Matt Bellina's ALSFRS-R started at 21 and improved to 27. Thus, we suspect some people below 26 will also show improvement. We need more data & more doses to see if this can meet stastitical significance.


PHASE 4 APPROVAL
All of this evidence is precisely why we believe NurOwn works. It's also why we hope the FDA will use its Accelerated Approval Pathway and approve NurOwn with what's called a Phase 4 confirmatory study.

A Phase 4 approval would mean that NurOwn is approved for use but Brainstorm has to keep collecting data from the people who use it so we can answer some of the questions above.

The FDA uses this pathway regularly for cancer therapies. We believe the people dying of ALS deserve the same humanity.