Steinhafen

[Steinhafen @Steinhafenn]

@rikiyatakehi @a1zhang @lateinteraction @jayden_teoh_ Have you tried to get the chain of thoughts how it does it?

[Steinhafen @Steinhafenn]

$qure FDA acceptance BLA of #amt130 PDUFA on May 1, 2027, there would be an Advisory committee meeting.

[Prof. Dollar @Prof_Dollar]

✨️ $QURE Announces FDA Acceptance and Priority Review of the BLA for ifezuntirgene inilparvovec (#AMT130) for the Treatment of #Huntington’s Disease
Source: https://t.co/K1EzPM1N5s
$CLPT $XBI

[Steinhafen @Steinhafenn]

@Prof_Oak_ Congrats Cody!
How much did it cost to get the mouse done in the 18 months?

[Steinhafen @Steinhafenn]

Bought $vktx

[Steinhafen @Steinhafenn]

Sold $psnl $rvmd

[Steinhafen @Steinhafenn]

@A_May_MD @robertcoffin3 Interesting approach. $mrna combo could theoretically activate/free more/stronger anti tumor t cells with the approach here

[Steinhafen @Steinhafenn]

$capr sold with a loss, live and learn

[Wall St Engine @wallstengine]

FDA briefing documents raised significant efficacy and statistical-analysis concerns for Capricor’s $CAPR deramiocel ahead of the July 29 advisory committee meeting.

FDA says HOPE-3 failed its prespecified primary and secondary efficacy endpoints, with no statistically significant difference between deramiocel and placebo at 12 months.

The agency also flagged changes to the statistical analysis plan made after the randomized, double-blind portion ended and during the open-label extension. Those changes modified endpoint definitions, analytical methods and the data-imputation strategy.

FDA says the final BLA analyses included additional changes without an updated SAP, while SAP v3.0 was neither submitted for FDA review before the filing nor discussed or agreed upon.

[Michel Doepke @doepke_michel]

$NKTR Announces Start of Phase 3 ZENITH AD Program Evaluating Novel Regulatory T-Cell Biologic Rezpegaldesleukin in Moderate-to-Severe Atopic Dermatitis https://t.co/59TBXnXGYw

[HODOR @Maximus_Holla]

$PSNL halted! 👀

[Steinhafen @Steinhafenn]

starter on $kytx

[Steinhafen @Steinhafenn]

$qure to submit the BLA in Q3

[Bio Stocks™ @BioStocks]

$QURE Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease

FDA communicated that the 3-year analysis from the Phase I/II study would be acceptable as the primary basis of a BLA for the accelerated approval of AMT-130

https://t.co/w3dleR6GDu

[Steinhafen @Steinhafenn]

@1realFormula Oligo

[Steinhafen @Steinhafenn]

@citrini tweeting on $rvmd is the least I have expected

[Citrini @citrini]

An amazing breakthrough by $RVMD that should see the name rally not just on pancreatic cancer TAM but on the “copy paste” potential for them to use RAS being a druggable target for further things beyond pancreatic cancer.

We detailed this dynamic in our 26 Trades for 2026.

[Steinhafen @Steinhafenn]

@ZShen3427 I once bought $nvts at 5 and sold them at 7, soon after it climbed to 17. That was a lesson for me. Sometimes we are lucky and it seems like we tine the market somehow, but other times we don't.

[Bio Stocks™ @BioStocks]

$OVID $60M PIPE @ $2/share

Point72, Adage Capital, ADAR1 Capital, Affinity Asset Advisors, Ally Bridge Group, Balyasny Asset Management, Coastlands Capital, Eventide Asset Management, Janus Henderson, and RA Capital

[Steinhafen @Steinhafenn]

@anthonystaj @houndcl @X @elonmusk That's a false positive, please bring him back @nikitabier

[Steinhafen @Steinhafenn]

So the press conference early today, was VP in panic mode. He probably got pressed or knew that he would be out at the time. Anyway, good news for $qure

[Adam Feuerstein ✡️ @adamfeuerstein]

VInay Prasad is out at the FDA, per the WSJ.

[Steinhafen @Steinhafenn]

The opportunity cost of employers who need to be told what to do has gone through the roof.

[Nivi @nivi]

The opportunity cost of employees who need to be told what to do has gone through the roof.

[Steinhafen @Steinhafenn]

@ErikOtto2 @biotechsanya2 $agmb didn't really show any functional data, they mentioned 'positive signals' and 'positive trends' on several exploratory clinical endpoints such as SED-CD and MRE. The 48 wks OLE data is expected at 2H 2026.

[FBCC @BiotechPort]

$FULC
Data looks strong. See no holes in it.

~20% HbF in 12w (this is not the peak as the trajectory is still going up).
More than doubled in % in HbF sells.
Decrease in all hemolysis markers.
Hemoglobin increase of 1.1 g/dL in 12w.

The main effect of the drug will kick in later in treatment due to decreased hemolysis and healthier bone marrow.
My expectation that the hemoglobin and HbF will go even higher with longer treatment (reminder this was only 12w).

They have a decent drug in SCD in my view.