Shelly Goettl

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

Brainstorm's update about #NurOwn:

• Committed to conducting a Phase 3b trial
• Submitting proposed trial design to FDA in Feb 2024
• FDA has 45 days to respond, meaning they will hear back sometime between mid-March to mid-April.

The timing about the start of any Phase 3b trial will be dependent upon what the FDA says about the proposed clinical trial design and when Brainstorm can raise capital to fund the trial.

Many of us in the #ALS community KNOW NurOwn works. That makes the need for another trial all the more heartbreaking for so many in the community. 💔

Thank you to Brainstorm & your entire team for continuing to fight for the ALS community. We will continue to tell the world that #NurOwnWorks.

Full copy of Letter:
https://t.co/sKiwmOKVmW

#EndALS

[Shah Minokadeh, M.D. @MinoShah]

Shame on @DrCaliff_FDA & Director of the @FDACBER, Peter Marks.

There is the blood of #ALS patients on both of your hands.
#NurownWorks #NurownIsSafe #CriticalUnmetNeed #BloodOnYourHands

You killed an effective treatment for hopeless #ALS patients.

Congress, it may be you or your loved one tomorrow who is diagnosed with this DEATH SENTENCE because of this continuation of the CRUEL #FDACBER regulatory RIGIDITY for a disease WITH NO SURVIVORS since 1st identified in 1869.

@PatriciaManhar1 @ShereeWLWT @RepGuthrie @RepLarryBucshon @RepAnnaEshoo @michaelcburgess @RepGusBilirakis @RepBillJohnson @RepRichHudson @RepBuddyCarter @DrNealDunnFL2 @cathymcmorris @RepDebDingell @RepMMM @RepAnnieKuster @RepRobinKelly @RepBarragan @RepAngieCraig @RepKimSchrier @RepLoriTrahan @SenatorWicker @PattyMurray @SenBobCasey @SenatorBaldwin @timkaine @SenatorHassan @SenTinaSmith @SenMarkey @SenBillCassidy @SenMullin @lisamurkowski @SenatorBraun @RogerMarshallMD @RepJasonCrow @TeamCalvert @RepBrianFitz @ChrisCoons @RepAndyHarrisMD @DickDurbin @SenJohnThune @RepDianaDeGette @RepMikeQuigley @RepSchakowsky @rosadelauro @RepJohnCurtis @DorisMatsui @chairmang @RepDonBacon

[Shah Minokadeh, M.D. @MinoShah]

I am a board-certified anesthesiologist and pain management physician with ALS. So tired of people talking about NurOwn data without understanding the trial when their lack of understanding is killing me, thousands of my friends, and their children.

1. ALS endpoints are based on a flawed, subjective functional rating scale with 48 points total, 12 pts for gross motor function, fine motor function, bulbar & respiratory function each. Peer-reviewed data talks about flaws in the scale including lack of linearity, inability to detect changes, and floor & ceiling effects in this bounded scale. Stop treating trial endpoints in ALS like those measured by an objective MRI or blood biomarker in oncology.

2. NurOwn met statistical significance for people with scores of ≥27/48 on both primary & secondary endpoints, which was 77% of the trial population. However, because of floor effect flaws in the scale, it did not measure changes in people with scores ≤26/48. Imagine denying approval of a chemotherapy drug that worked on stage 1 and 2 cancer but not as well on stage 4. That's ludicrous, but that is essentially what the AdComm recommended yesterday.

3. No one is reporting the testimony of people who received 6 additional doses of NurOwn in Expanded Access and the clinically documented improvements in function that have never occurred in ALS history.

4. The most important thing missing from every story is the testimony of Dr. Tony Windebank, a world-renowned neurologist at Mayo with 40+ years of experience in treating people with ALS and conducting ALS clinical trials. He was a PI in the phase 2 and phase 3 trials. He said there were a "substantial number" of people for whom NurOwn worked: "As a clinician investigator, I also look closely on the aggregate basis. While it is clear that not everyone responds to the treatment, there are clearly a significant number who do, and this response is new. I have clearly seen some people STABILIZE in a way that I have NEVER seen IN ANY OTHER TRIAL. In fact, in the small number of people who participated in EAP & received 6-9 treatments, there were people who STABILIZED while on treatment in the trial. In the interval before they were in the EAP -- which was over a year or more in some cases -- these participants deteriorated... then again STABILIZED in the additional treatment period. There were also SOME WHO IMPROVED THEIR SCORE FROM BASELINE ... something that is rarely seen. I should stress that other investigators who have been working 'hands-on' with participants in the trial have seen similar responses.... For these reasons, as a physician who cares for patients, I want to see NurOwn made available for people living with ALS.

5. NurOwn had first-in-class CSF biomarkers that showed decreases in pathways of neuroinflammation and neurodegeneration, along with increases in neuroprotection. These changes only occurred in the treatment arm and not in the placebo arm. They also occurred across the disease spectrum, confirming biological changes in people with scores ≤26/48 as well.

6. @Joe_Wiley_MD, the week 20 missing data was because of COVID. The trial sites were not allowing visits for a lumbar puncture to collect CSF that wasn't involved with receiving the therapy (tx was delivered at weeks 0, 8, and 16). Similarly, the respiratory endpoint in the trial required people to blow into a spirometer to collect FVC. Are you saying neurologists at Mayo, Mass Gen, UMass, UC Irvine, CPMC and Cedars-Sinai should have violated pandemic protocols early in 2020 when COVID was on the rise and no one had masks? Please don't make it sound like data was missing because of the drug sponsor. The FDA previously advised all drug sponsors that they would make every effort so the pandemic would not impact their trial data, especially in brutal diseases with "critical unmet needs." Yet they didn't honor their promise yesterday and advise the voting members on this statement. Should we have halted a $50M+ trial that delivered its first dose in 2017 because of a global pandemic or made accommodations for the week 20 visit to minimize unnecessary exposure to patients with compromised respiratory function?

7. Trial passed all DSMB reviews, with NO HOLDS. The increased deaths were people who died of ALS, not treatment-related deaths. Their ALS scores at death were predominantly in the teens when many die of choking/aspiration, pneumonia, or respiratory failure because they decided not to get trachs. The lifespan of ALS is 2-5 years. That's why trials don't allow people to qualify past 2 years from sx onset. In 18 months, 50% of people die, and by 5 years, 80% die. The people died of ALS.

[Phil Green @pjgreen]

I will be ready to share my experiences with #NurOwn from three dosing periods (2019, 2021 & 2022).

[No More Excuses ALS Watchdogs! @als_now]

Debating whether or not to allow dying patients access to effective new #ALS drugs feels exactly like this: #NUROWNWORKS #NOADCOMNOVOICE #FDA @biospace @STAT @biogen @BrainstormCell @NMEALS @alsassociation @ALSforums @ALSTDI @ALSCanada @mndassoc @mndaustralia @ALSMNDAlliance

[Lisa's Legacy for ALS @LisaLegacy4ALS]

Thanks for the update @BrainstormCell. @US_FDA there are so many people asking, hoping, praying for an #ADComm meeting for #NurOwn. Pls give the data a chance to be reviewed publicly. When there's so little hope in #ALS pls do what you can. @DrCaliff_FDA @FDACBER

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

We've been fighting non-stop for #NurOwn

@BrainstormCell @Cylebo
🔹What's outcome of Type A meeting?
🔹Did @FDACBER agree to AdCom?
🔹Did @US_FDA agree that people w #ALS & #neurologists have a right to share #RealWorldEvidence that NurOwn improved how they feel & function?

[I AM ALS @iamalsorg]

I AM ALS is hopeful that yesterday's Type A meeting regarding NurOwn was productive and that the @US_FDA decides to hold a public, transparent Advisory Committee meeting in the near future.

[Kandy Simons @KandySimons]

My son Kade was 21 in the Ph 3 #NurOwn trial is #DyingWaiting to hear how the Type A meeting went today @Cylebo. Will @FDACBER allow those with #RealWorldEvidence share their experience that #NurOwn slows/halts paralysis & has allowed some to restore function?

#NoAdcommNoVoice

[Smithstrongmom @Smithstrongmom1]

My son Josh is 33 yrs old and is #dyingwaiting for the results of a Type A meeting @Cylebo.
@FDACBER, we can't continue waiting. There is #realworldevidence that #Nurown improved breathing, strength & restored some function for my son. @DorisMatsui @RepDianaDeGette

[Shah Minokadeh, M.D. @MinoShah]

27-year-old Megan died of #ALS.

@DrCaliff_FDA, HOW MANY MORE HAVE TO DIE before the @FDACBER treats 100% FATAL ALS with the same URGENCY/FLEXIBILITY as cancer treatments?

Congress, DEMAND an #AdComm hearing for #Nurown to allow #RealWorldEvidence of efficacy shared publicly.

[axeALSFoundation @stevens_nation]

Eric is #DyingWaiting for the results of Type A meeting. @Cylebo
@FDACBER will you let us share #RealWorldEvidence that #NurOwn halts paralysis & restores function?

[Elizabeth McCormick @emmccormick2005]

I was in the Ph 3 #NurOwn trial & I'm #DyingWaiting to hear how the Type A meeting went @Cylebo. Will @FDACBER allow those with #RealWorldEvidence to share their experience that #NurOwn slows/halts paralysis & has allowed some to restore function?

#NoAdcommNoVoice

[BrainStorm @BrainstormCell]

We understand that people living with #ALS, their loved ones and our loyal investors deserve to know how the Type A meeting went and the next steps in the process.

[BrainStorm @BrainstormCell]

We will share an update asap since there are ongoing discussions with FDA. We are thankful for all the support, and we remain committed to the entire ALS community and our investors.

[Shah Minokadeh, M.D. @MinoShah]

Scott died of #ALS this morning leaving behind a devastated wife & 2 daughters.

As a society, how do we allow this to go on?
153 years that #ALS is 100% fatal & NO URGENCY at @FDACBER & @DrCaliff_FDA.

@RepAnnaEshoo @brettguthrie @janschakowsky @SenatorBraun @rosadelauro @RepLBR

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

Hey #SteelersNation

Devyn is just 22 years old & fighting 100% fatal #ALS.
No parents should have to watch their child battle ALS when a drug that is helping some is stuck in trials.

Can you help us fight for Devyn?
https://t.co/8Ak5skBVhk

#HereWeGo #SteelersFootball #NFL

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

@AberdareNic Help us fight in the US to get #NurOwn approved. We KNOW #NurOwnWorks. Sign the petition at IAMALS to ask the FDA to give people a public AdCom meeting where neurologists & people share their stories.

https://t.co/8Ak5skBVhk

#NoAdComNoVoice

[No More Excuses ALS Watchdogs! @als_now]

@stevens_nation @RepBarragan @DrCaliff_FDA @FDACDERDirector @DrBiden @DrWoodcockFDA This picture alone should erase any doubts about #NurOwn. Those who know Eric (and have since he entered the #ALS community) know exactly what this picture means and why: SUCCESS. From fast progressor to nearly stable- this is an EPIC #ALS result. Losing NurOwn would be a crime.