#GoDawgs

STAT No 22 yr old with #ALS should die when a drug could help her live. It’s inhumane for BCBS-TX to deny coverage for Biogen’s ASO #Qalsody. 1. BCBS-TX is denying coverage alleging “clinical benefit has not been established.” But Shelby’s medical records document her ACTUAL

This is great news for the #ALS community. Thanks Secretary Pete Buttigieg #EndALS #DisabilityRights

NOBODY should have to repeatedly battle their insurance company for medical coverage of any disease. PERIOD. Do better! Blue Cross Blue Shield Association BCBSTX #Qalsody #BCBS #ALS

#ALS IS NOT RARE & is becoming more common People who have #ALS have been left to die without hope by the FDA Biologics CRUEL regulatory RIGIDITY for a disease WITH NO SURVIVORS. Marty Makary MD, MPH Robert F. Kennedy Jr Donald J. Trump Elon Musk, We need your leadership to value #ALS lives by

Spike the football a decade late all you want The ALS Association over something #NME members were yelling in 2019: #ALS progression could be HALTED. Not for 2% of all patients, but many! Explain why T-REGS was not pushed as hard as others despite $70M+ in your bank? #ALSBIAS #endals

I was Diagnosed in 2017 with so many possibilities in the pipeline, which kept me hopeful. Now, my only hope is in God.

This is horrible news for people with #ALS.

1/12 Dr. Marty Makary Secretary Kennedy FDA Biologics, As one of the (cell therapy) NurOwn Citizens' Petitioners fighting for my life—and the lives of tens of thousands with 100% fatal #ALS—I'm terrified For 156 years, this disease has killed everyone with ZERO treatments providing any hope of

2/12 NurOwn showed REAL signs of promise in the Phase 3 trial (randomized, placebo-controlled). Despite missing the known deeply flowed 12-point questionnaire primary endpoint overall (due to heterogeneity, floor effects, high placebo response), multiple signals emerged:

3/12 *Phase 3 Trial Signs of Promise:* - Statistically significant increases in neurotrophic factors (e.g., VEGF) & reductions in neuroinflammatory/neurodegenerative biomarkers (e.g., MCP-1; robust changes only in NurOwn arm). - 23 of 45 pre-specified CSF biomarkers with

4/12 *Phase 3 Trial Signs of Promise (cont.):* - Statistically significant NfL reduction (~9.4-11% delta vs. placebo in adjusted models; p=0.037; linked to slower decline—same surrogate that got tofersen accelerated approval). - In pre-specified less-advanced subgroup

5/12 *Phase 3 Trial Signs of Promise (cont.):* - Post-hoc analyses (accounting for floor effects/advanced patients): Significant slowing of functional decline on ALSFRS-R. - Independent Harvard/Dana-Farber stats (Lee-Jen Wei): Supportive p-values (0.045, 0.021, 0.007, 0.005)

6/12 *Citizens' Petition Signs of Promise (new EAP/long-term RWE, real-world evidence):* - Unprecedented survival: 100% 5-year survival from symptom onset in EAP cohort (n=10) vs. ~20% natural history (p~0.000335) - Median tracheostomy-free survival ~7 years (range 5-8.5)

7/12 *Citizens' Petition Signs of Promise (cont.):* - Extended progression-free survival: Up to 17 months (or more) during/after treatment—far beyond expected. - Up to 85% slowing of progression (from ≥1 ALSFRS-R point/month loss to ~0.15 points/month post-NurOwn). - Greater

8/12 *Citizens' Petition Signs of Promise (cont.):* - Real-world participant benefits: Many regained motor function, maintained respiratory independence years longer than expected; restored/improved breathing (e.g., reduced need for support); Stabilized/improved forced vital

9/12 •Citizens' Petition Signs of Promise (cont.):* - Respiratory gains: 5-8 year delay in non-invasive ventilation need; halting progression in breathing-critical domain (biggest survival predictor). This is the "totality of evidence" the FDA has NEVER fully reviewed under

10/12 Dr. Marty Makary Marty Makary MD, MPH Marty, I had no idea when we took this photo years ago at Johns Hopkins that you’d one day hold my life—& the lives of tens of thousands with ALS—in your hands as FDA Commissioner. I want to believe you’ll do the right thing. Your confirmation

11/12 ...when senators Murkowski & Collins asked you about using Accelerated Approval for ALS patients facing a “very, very limited time frame” with “no hope of survival,” you committed to flexible pathways, common-sense reforms, and rare disease urgency. That’s exactly

12/12 U.S. FDA FDA Biologics Secretary Kennedy Dr. Marty Makary @VPrasadMDPH: Please don't let this be another broken promise. Grant accelerated approval for NurOwn NOW—with Phase 4 confirmation, biorepository, & natural history database. People are dying daily. We can't wait. 178 days