BioBankRaider

Tracking biotech catalysts, bull-case trades driven by clinical data, FDA PDUFA plays, and FDA Adcomm momentum. Views my own. Not financial advice.

[BioBankRaider @BioBankRaider]

This is actually a really good point and why the FDA has called on manufacturers to have greater emphasis on patient reported outcomes (PROs) in trials as well as calls on payers to incorporate in coverage determinations. Seems FDA didn't want consider much of this in discussion

[FlyingEagle @FlyingEagle86]

$capr When FDA calls deramiocels benefits“fragile,”what they really mean is that the exact pvalue or effect size shifts depending on which model and assumptions you use.But needing a particular statistical model to see the effect doesn’t mean boys aren’t actually doing better 1/3

[BioBankRaider @BioBankRaider]

@levnxrae @ChefReactions

[BioBankRaider @BioBankRaider]

I have no position in $REPL but expect more discussion and likely more favorable outcome than CAPR due to some precedent around single arm trials

[BioBankRaider @BioBankRaider]

Last thought on $CAPR. A lot of missed opportunities by both the company and the FDA to align and course correct to ensure that if the drug worked there would be some objective data to see...both failed here potentially costing patients a beneficial treatment.

[BioBankRaider @BioBankRaider]

@GravityAnalyti1 $CAPR

[BioBankRaider @BioBankRaider]

Adcomms can go a lot of different ways...$URGN had a single arm and despite lack luster Adcomm still was approved. FDA could still do the same for $CAPR but the hill got bigger imo

[BioBankRaider @BioBankRaider]

Exondys 51 (eteplirsen) - 2016 Indication: Duchenne muscular dystrophyAdcomm Vote: Split/Negative (7-3 against approval on the core question of whether data showed an increase in dystrophin reasonably likely to predict clinical benefit). FDA Action: Approved under accelerated approval following intense internal agency disagreement and heavy public/patient advocacy pressure

[BioBankRaider @BioBankRaider]

@GravityAnalyti1 So?

[BioBankRaider @BioBankRaider]

Wut...literally no trial would accomplish this feat, especially in rare disease

[Gravity Analytica Capital @GravityAnalyti1]

If $CAPR's drug worked it would work no matter how you analyzed the data.

[BioPharmCatalyst @BPharmCatalyst]

$CAPR FDA AdComm takeaways: • Cardiomyopathy: Decisively negative
• Upper Limb Function: Mixed views, moderate support
• Public Hearing: Emotionally powerful patient testimony
Reminder: FDA rigor vastly outweighs peer-reviewed journals—pre-specified endpoints are everything.

3 Yes
9 No
0 Abstain

https://t.co/0Z5UK3dxhr

[BioBankRaider @BioBankRaider]

While the Adcomm has been anything but supportive of the drug...there likely is a trend to positive patient impact and the trial design just sux. If anything this was CAPR trying to avoid another trial imo.
$CAPR

[Capricor Therapeutics @Capricor]

We're pleased to share that @TheLancet has published results from Capricor's Phase 3 HOPE-3 trial evaluating Deramiocel in #Duchenne muscular dystrophy.

The independent peer-reviewed publication validates the trial's design, statistical methodology and findings. HOPE-3 met its primary endpoint, with Deramiocel slowing upper limb function decline by 54 percent versus placebo (PUL 2.0, p=0.03) and demonstrating a clinically meaningful cardiac benefit.

Read more here: https://t.co/WPBkXv0Ws9

We are grateful to the patients, families, investigators and study teams who made this research possible.

[FlyingEagle @FlyingEagle86]

$capr *credit to the individual who did all the digging* #DMD community we will rise up tomorrow💪🏻

[Anthony @anthonystaj]

$capr so they changed the SAP **twice**, once in september and again in november. FDA being pretty clear her: never agreed to change! no "implicit endorsement" no "tacit approval" as @linda_marban told everyone in multiple public calls representing @Capricor why the discrepancy?

[Anthony @anthonystaj]

$capr is still actively trying to deceive the world on their results and directly contradicting the FDAs findings. should be fireworks at the adcom

[BioBankRaider @BioBankRaider]

$CAPR comments/response prior to adcomm: https://t.co/qmLGiNrvSS

[BioBankRaider @BioBankRaider]

dead cat bounce for $CAPR still really question what the future is here without management cleaning up the situation...they have GOT TO RESPOND...FDA literally called them liars

[BioBankRaider @BioBankRaider]

Wowza what a thrashing by the FDA...$CAPR . company better come prepared because essentially they have told everyone they didn't do what they are accused of doing...FDA has receipts

[BioBankRaider @BioBankRaider]

Big day...$MNKD and $OTLK approvals

[KairosPharma @KairosPharma]

$KAPA announced a strategic collaboration with @BayerUS to evaluate ENV-105 with XOFIGO in metastatic castration-resistant #prostatecancer involving bone.

ENV-105 previously demonstrated an 86% clinical benefit rate in the Company's Phase 2 mCRPC trial.

More: https://t.co/K51soLul8S