BIOTECH · @_B_I_O_T_E_C_H_
6th Sep 2017 from TwitLonger
JPMorgan $SRPT SRP-4053 Top-Line Beats Expectations; Quick
Thoughts On Results
This morning, Sarepta announced positive top-line dystrophin data from the phase 1/2
4053-101 study of SRP-4053 in Duchenne Muscular Dystrophy (DMD). Overall, the
dystrophin data beat Street expectations, in our view. Indeed, expression levels are
ahead of Exondys 51 label data and were generated from a large sample set (4053
Western Blot results showing a ~11-fold induction of dystrophin expression over
baseline, which was statistically significant, versus near ~3-fold induction in Exondys
51 label; the most direct comparison). Further, all patients (n=25) saw an increase in
skipping of Exon 53 over baseline levels. Of note, full data will be presented at a
medical meeting in the near-term. We see SRPT shares up substantially on the top-line
4053 data. Thinking about SRPT shares long-term, we believe data are an important
for eliminating the bear thesis on the overall technology platform and believe the
company will likely share these data with regulatory agencies, with a focus on
potential approval pathways (recall, Exondys 51 was approved on a surrogate
dystrophin endpoint). Further, with the platform increasingly de-risked, in our view,
today's update is an important step in shifting investor base long-term.