Wave Life Sciences ($WVE) presented data on its Huntington's disease drug showing promising results in mutant htt-specific lowering with the potential for less frequent dosing. The data indicated efficacy, safety, and durability, with a focus on Nfl levels. The company aims for quarterly dosing and allele-selective targeting.
$NTLA another -7% after all the positives that happened this week ( $ALNY validation of kd in ATTR-CM, additive genome editing after repeat dosing)...and I had thought nobody cared about yesterday's event beforehand.
Arrowhead Pharmaceuticals to Advance RNAi-based Plozasiran into Phase 3 CAPITAN Cardiovascular Outcomes Trial $ARWR https://t.co/Oo5jZLY2FE
$WVE data today every bit as efficacious (if not more so) and safe wrt to Nfl as $IONS/Roche tominersen. Roche also showed that Nfl comes down with less frequent dosing....which $WVE can do based on data. The big difference: $WVE is allele-selective. https://t.co/Rcd0nKiuTg
$WVE data today every bit as efficacious (if not more so) and safe wrt to Nfl as $IONS/Roche tominersen. Roche also showed that Nfl comes down with less frequent dosing....which $WVE can do based on data. https://t.co/ljsxt8TvOz
Wave, with new data, plots path forward for Huntington’s drug https://t.co/6SkFpSolkx by @NedPagliarulo $WVE - 7% $PTCT $QURE
$WVE - the NfL chart and Caudate Atrophy data (not stat sign vs Pbo) - two key obstacles w/ AA path using current data for the regulators - .. if the NfL data is just no chg vs these spikes and the CA data is stat sign vs Pbo - then... could get a wink wink shot - .. am… https://t.co/0Eu49B67Rb
$wve taking step back...they have a drug that selectively lowers mutant htt 40%+ with potential to go down on dosing frequency. From DMD program we know that PN backbone can crazily accumulate in tissue. Something to work with for horrible disease with no drug options.
$WVE 2 key slides, efficacy and safety. Efficacy: beautiful, mutant htt-specific lowering, and possibly concomitant wt increases. Durability indicates quarterly dosing possible (goal is >30% lowering) Safety: Nfl creeps up (oligos accumulating too high?). The bet:…
$WVE slides. Not sure this data is convincing enough at this point to show a different outcome from Tominersen which failed P3 studies. Seeminly no dose response in SAD in mHTT lowering, no TMS at day 197... https://t.co/uTdPIW4wf3
$WVE no ventricular volume expansion observed as was case in $IONS/Roche non-selective htt-lowering trials (now shelved).
$WVE will listen the webcast and see what they have on the HD side - most of my curiosity with this name is over the AATD and INHBE side - .. didn't have HD side on the balance - the mHTT % knockdown is interesting - probably way too short in duration to see any functional…
$WVW will listen the webcast and see what they have on the HD side - most of my curiosity with this name is over the AATD and INHBE side - .. didn't have HD side on the balance - the mHTT % knockdown is interesting - probably way too short in duration to see any functional…
First $ALNY, now $WVE. Nice for #oligonucleotides creating some biotech excitement. $STOK earlier this year, also $ARWR with clean FCS data. A very good 2024 so far.
$WVE could this be our daily biotech doubler? Looks like one in the making. Color on AA possibility in conference call key. $PTCT was also hinting at that possibility, but they are also lowering wildtype htt.
TODAY! $ARWR CVD Day. CVOT reveal, ApoCIII and/or ANGPTL3? Financing (my guess is $CYTK-type deal with $RPRX) $NTLA CRISPR repeat-administration. Hugely underfollowed event. Big implications for systemically administered extrahepatic genome editing. Also this week: $WVE HD…