#AstraZeneca# and #IonisPharmaceutical# are the first to receive FDA approval for the #antisenseoligonucleotide# (ASO) based RNA drug #eplontersen#. The only treatment for the treatment of PN transthyretin-mediated amyloidosis (hATTR-PN). . #RNAtherapeutics# .

$ALNY -5% this morning Sellside comments re: the 1.14 event rate ratio from the stabilizer (combo) arm of the CARDIO-TTRansform study: Paul Matteis, Stifel "While minimal benefit was largely expected for the eplontersen/tafamidis combo arm in CARDIO-TTRansform, it's an incremental surprise to see that the combination had a hazard ratio of 1.14--clearly worse than expected." Michael Leuchten, Jefferies: "The presentation showed a treatment effect of rate ratio (RR) 1.14 for the baseline stabiliser group vs overall RR of 0.89, with a strong enough interaction p-value (0.017) suggesting a potential differential treatment effect between patients without stabiliser at baseline. The primary endpoint curves in patients that were on stabilisers at baseline do not show an improvement over time, if anything there is a worsening rate in the combination group." Cory Kasimov, Evercore: "...the 1.14 RR in pts on baseline stabilizers was the key metric investors cared about most coming in and will likely not be viewed favorably as it relates to implications for ALNY. At first glance, the results appear less suggestive of a trial-design issue and more consistent with a potential limitation to the incremental benefit of dual therapy with silencers and stabilizers, further entrenching the competitive positioning of the stabilizer class."

Alnylam’s $ALNY heart drug troubles take center stage at a cardiology conference #ESC26# kicks off tomorrow morning, where we will get full data from the failed CARDIO-TTRansform study of eplontersen. Important Alynylam read-thru...