Andes L

[Andes L @SheSawey]

@spineopine SAP 1.1’s absolute MMRM places equal mathematical weight on both patients, but in reality they decline in different trajectory

[Andes L @SheSawey]

@spineopine For a patient starting at 38, losing 2 points (5% of capacity) is barely noticeable clinically.

[Andes L @SheSawey]

@spineopine For an advanced patient with a baseline score of 14, losing 2 points (14% of their total remaining motor capacity) often means losing critical daily independence (self feed, ability to wheelchair control)

[Andes L @SheSawey]

@spineopine FDA uses SAP 1.1 as basis of SA. Absolute change measures raw point drop treats a 2-point loss in a patient starting at a score of 38 (early stage, high shoulder function) identically to a 2-point loss in a patient starting at 14 (advanced stage, low mid-level/elbow function).

[Andes L @SheSawey]

@MaryBschanck Research published in ESC confirmed that DMD patients with reduced LVEF (specifically ≤40%) experience significantly higher rates of cardiovascular events and a lower rate of overall survival compared to those who maintain higher LVEF levels.

https://t.co/Damj5lkhxZ

[Andes L @SheSawey]

@AscendingBio @retirethisway_ FDA PUL score is from SAP 1.1 which is a draft. SAP 1.1 was provided to FDA post BLA resubmission as part of IR during review cycle as FDA wanted a full draft history.

[Andes L @SheSawey]

@AscendingBio @retirethisway_ What about PUL? Are you saying it’s also not significant at 0.029?

[Andes L @SheSawey]

From FDA’s ADCOM brief document
“There were 6 serious adverse events (SAEs) reported in the randomized, double-blind portion of Study
HOPE-3, including 5 SAEs in 5 subjects who received placebo, and 1 SAE in 1 subject who received
deramiocel.”
Only 1/6 is from deramiocel. $CAPR

[Andes L @SheSawey]

@Capricor When will you release results of the minutes with FDA?