OneWay2Mars

[OneWay2Mars @oneway2mars]

@adamfeuerstein I love your Thu article. You make great points — about FDA should be or what not. Eg, “norm-busting decision from an agency that is set up to review clinical data objectively, not lob attacks against the companies it is in charge of regulating.”

[OneWay2Mars @oneway2mars]

@pinker_ste94119 $CAPR p-value for PUL 2.0 is 0.029 -- whether percentage or absolute change. See slide #14; states 0.029 with 1.2 as diff. 54% is more INFORMATIVE than 1.2pt (e.g., if placebo decline is 25, then 1.2 change, i.e., 23.8 decline of treatment arm, is clinically not meaningful)

[OneWay2Mars @oneway2mars]

@AscendingBio There’s nothing other than AA — which requires either a surrogate marker or an “intermediate endpoint”. Surrogate marker is plausible but doubtful. So, yes, I agree it will be a long road (5+ years) to BMD approval.

[OneWay2Mars @oneway2mars]

@Biotenic @avidresearch @AppleHelix $CAPR Most importantly, the treatment differences are NOT SMALL (they may seem so, but are not). The point is: Even placebo decline was "small" -- so decline with treatment will only be small! Thus, best to look at "relative" declines -- 54% and 91%.

[OneWay2Mars @oneway2mars]

@Biotenic @avidresearch @AppleHelix REALIZE that 54% improvement is more informative than saying the decline was only 1.2 pts (which seems very small -- but significant when you see that Placebo also decline "only" 2.4). $CAPR. Same idea with LVEF. 91% is just more informative than 2.4% diff.

[OneWay2Mars @oneway2mars]

@PK_Fund So, why the discrepancy—why does FDA think otherwise? $capr

[OneWay2Mars @oneway2mars]

@showmepapi FDA lifting the halt means that, unless there are deaths in the ambulatory population, Elvedys will remain on the market. FDA will likely be amenable to resolving the non-ambulatory situation as well -- $SRPT has a risk mitigation regimen in place, which may suffice.

[OneWay2Mars @oneway2mars]

$SRPT Everything depends on your conviction/assumption: Could/would there be any deaths in the ambulatory population? If NO (over the next few years), then the stock will be $100-300 in 1-2 years. If YES, then it's a very rocky ride -- it may remain in $5-30 for years.

[OneWay2Mars @oneway2mars]

$SRPT FDA is driven by media. On Fri, FDA said the LGMD trial's death was lost in bureaucracy and hence the delayed halt (obviously, BS, or worse, pure incompetence). Today, it uses Brazil kid's as a face-saving excuse (ofc, Brazil's death had nada to do with Friday's halt).

[OneWay2Mars @oneway2mars]

@lady0fcrypt0s $35? $SRPT

[OneWay2Mars @oneway2mars]

$SRPT it’s obvious that FDA got swayed by media and WS than by science (else, there is no reason for them to take all these actions on Friday — after knowing all the facts for a few weeks, and a day after agreeing to black-box warning). What a shame!! Unbelievable.

[OneWay2Mars @oneway2mars]

@TomSilver39 @US_FDA Read your self again. FDA can seize products after a *request* to halt shipments?? Really? It’s best not to speak when you are clueless.

[OneWay2Mars @oneway2mars]

@adamfeuerstein What’s heroic about it? Serious Q. IMO, IF (yes, it’s an “if”), the drug turns out to be safe for ambulatory — $SRPT ceo would be considered a hero.

[OneWay2Mars @oneway2mars]

@adamfeuerstein Do you find it offensive to be called a "Jew Boy"? Why? Being offended by it IS more of an antisemitism -- than calling someone that!

[OneWay2Mars @oneway2mars]

@davideyoungmd @bimiki @gtm920 @adamfeuerstein HOPE-2 (Phase 2) was a placebo-controlled trial with an endpoint of PLU 2.0 (i.e., limb strength). FDA asked $CAPR to file a BLA based on ejection fraction instead (for the cardiomyopathy indication of DMD). So, HOPE-2 was placebo-cntrd, but for a diff endpt. See Co 3/25 slides.

[OneWay2Mars @oneway2mars]

@AscendingBio Of course. $CAPR is going for FULL APPROVAL (as per FDA's suggestion), not AA (where surrogate marker applies).

[OneWay2Mars @oneway2mars]

@yachmod @VPrasadMDMPH @HHSGov @RobertKennedyJr @POTUS What else did you expect? I’m sure you’ll vote again for “America First”.