CPT_Saki

[The Lancet @TheLancet]

Bloodstream-delivered cell therapy slows muscle decline in young people with Duchenne muscular dystrophy, phase 3 trial finds.

Explore the research 👉 https://t.co/cCXDL550TO

[CPT_Saki @macau_w]

@eyegenedrb @TheLancet Because deramiocel works via a transient paracrine mechanism rather than sustained cell engraftment, it bypasses immunosuppression.

[Jacob @daringjacob007]

Ok everyone, we have almost 3,000 signatures in support of boys and young men with Duchenne! We need so many more to get deramiocel the attention it deserves. SHOUT IT FROM THE ROOFTOPS!!! Tell everyone - friends, family, coworkers, etc https://t.co/sMLVojez5f

How else can you help? Immediately send an email to HHS and FDA using the simple form at https://t.co/8rnsZpD3B2. It takes 30 seconds.

Let’s take a stand!

[CPT_Saki @macau_w]

@MavenMining @Elijahjstacy @Elijahjstacy you are doing god's work. patient must win that approval (At least conditional approval)!

[CPT_Saki @macau_w]

#CAPRICOR #Deramiocel should the FDA approve this?

i think so.

[CPT_Saki @macau_w]

See which group is better off. And see which one is the better treatment. A fair outcome for both companies. And mandatory finalized studies for both company. No one loses

[CPT_Saki @macau_w]

$capricor The best outcome for patients would likely be accelerated approval for deramiocel. As a condition, Capricor would need to conduct a 3-year open-label study with all participants to demonstrate a clear, observable therapeutic effect.

And compare srpt group of patients.

[CPT_Saki @macau_w]

@AscendingBio @SheSawey @retirethisway_ There is no way to invest in $capr.

Accelerated approval for p4 might be the ethical way to proceed for fda. Nothing works in the space of dmd. Capricor might have something that works. But Accelerated approval will save these patients

[CPT_Saki @macau_w]

@AscendingBio @SheSawey @retirethisway_ Hope 2 ole is a 5 year study. And those patients disease progression slow way down compared to soslow's study.

[CPT_Saki @macau_w]

@SheSawey @AscendingBio @retirethisway_ Are there any other Phase 3 studies in DMD
that have achieved statistical significance specifically using Performance of Upper Limb (PUL 2.0) as a primary or secondary outcome measure?

Hard to tell over 1 year because patients progress differently

[CPT_Saki @macau_w]

I argue we shouldn't just ignore this, and compare this with patients on exon skipper. or the best options we have currently.

[CPT_Saki @macau_w]

$CAPR Hope 2 OLE was successful overall. Although the FDA disregarded the post-12-month data due to baseline , patients still maintained their PUL score and LVEF function, i think debating about this at adcom will be much less confusing for the panel.

[CPT_Saki @macau_w]

@Da1lyGa1n @Ridingwaves69 I agree that post-hoc adjustments to an SAP compromise trial integrity, but HOPE-3 suffered from fundamental design flaws at the outset. (management fault), the SAP was bound to produce a non-significant p-value.

[CPT_Saki @macau_w]

@Da1lyGa1n @Ridingwaves69 trying to argue statistical significance in a 1-year study like HOPE-3 is an uphill battle. SAP 1.1 was attempting to measure tiny signal changes in a disease as wildly unpredictable as DMD—especially with patient dropouts is the norm.

[CPT_Saki @macau_w]

@brown_pres3185 @markland_55 @houmanhemmati @FDA @FDAadcomms but at the same time, it seems sponsor don't have much time (if any) to reply after the FDA and Panel make the discussion. there were a lot of nuance, especially when HOPE 2 OLE data for 5 years wasn't mentioned. in this adcom. because changes are too small over 1 year.

[CPT_Saki @macau_w]

$CAPR I’d love to see these panels do a deeper dive into the data to see if their opinion would change. The way the FDA structured the question almost forced a firm 'no' on LVEF. Still, there’s so much hanging on this decision; and their opinion could spurt new hope for DMD

[CPT_Saki @macau_w]

FDA should considered all things. not just SAP. i hope the Ad-com panels see this through.

[CPT_Saki @macau_w]

$CAPR

HOPE 3: Deramiocel outperformed placebo in rank-based scoring. (drug has treatment EFT)

all DMD Trials: fail to show 1-year absolute effect.

HOPE 2 / OLE: 5-year data proves long-term efficacy.

Supported by general DMD natural progression studies.

Deramiocel works.