{"id":12759,"date":"2023-05-23T16:28:03","date_gmt":"2023-05-23T20:28:03","guid":{"rendered":"https:\/\/ifintechworld.com\/news\/buy-sarepta-therapeutics-srpt-for-a-likely-positive-fda-decision-date\/"},"modified":"2023-05-23T16:28:05","modified_gmt":"2023-05-23T20:28:05","slug":"buy-sarepta-therapeutics-srpt-for-a-likely-positive-fda-decision-date","status":"publish","type":"post","link":"https:\/\/ifintechworld.com\/?p=12759","title":{"rendered":"Buy Sarepta Therapeutics (SRPT) For A Likely Positive FDA Decision Date"},"content":{"rendered":"
\n

<\/picture>
<\/figcaption><\/figure>\n<\/p>\n

Sarepta Therapeutics (NASDAQ:SRPT<\/span>) is a large (~$14 billion market cap) biopharmaceutical company that markets 3 RNA-targeted products for Duchenne muscular dystrophy (DMD) patients. In previous coverage, SRPT was touted as a Strong Buy because the FDA flip-flopped and held a Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) meeting for SRP-9001, handing investors a positive catalyst. The vote <\/span>favored Sarepta as expected, sending shares to a 52-week high of $152.89 (or 16% over the price at publication). While the FDA doesn\u2019t always follow its experts\u2019 opinions, an upcoming confirmatory trial later this year bodes well for Longs\u2019 hopes that the Agency will be lenient and approve the DMD gene therapy.<\/span><\/p>\n

The Agency wasted time during Ad Comm trying to undermine the case for SRP-9001 by bringing up clinical efficacy according to the North Star Ambulatory Assessment (NSAA), which was Study 102\u2019s primary functional endpoint. All four of the <\/span>FDA\u2019s analyses were about change (\u0394) in NSAA scores. Topic 2 even directed CTGTAC to discuss the clinical significance of exploratory NSAA subgroup analyses! However, such items are NOT required for Accelerated Approval. In fact, the only question up for voting was \u201cDo the overall considerations of benefit and risk, taking into account the existing uncertainties, support Accelerated Approval of SRP-9001 \u2014 using as a surrogate endpoint, <\/span>expression of Sarepta\u2019s micro-dystrophin at Week 12 after administration of SRP-9001 \u2014 for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene?\u201d<\/span><\/p>\n

As discussed in the previous article, Study 102 met its primary biological (the surrogate) endpoint, with micro-dystrophin expression of 23.8% at Week 12 in the SRP-9001 group compared to 0.1% for placebo (p < 0.0001). The level of change from baseline (“CFB”) was magnitudes higher than the 0.28-0.8% mean CFB of normal dystrophin demonstrated by Sarepta’s exon-skipping products. Because of this large increase, the FDA questioned its validity as a surrogate endpoint reasonably likely to predict clinical benefit. Specifically, Analyses 3 and 4 tried to show a lack of clear dose-dependent expression increase or association between micro-dystrophin and \u0394NSAA Total Score. However, functional improvement with natural dystorphin may be saturable after some threshold, flattening the curve (Figure 1). For Studies 102 and 103, the direction was positive and correlation was significant.<\/p>\n

Figure 1. Association Between SRP-9001 Dystrophin and NSAA 1-Year Change<\/p>\n

\n

\"dystrophin<\/span>
\n

Sarepta<\/span><\/p>\n<\/figcaption><\/figure>\n<\/p>\n

Finances and Takeaways<\/strong><\/h2>\n

Q1 earnings were mixed. A $387.3 million debt extinguishment contributed to a net loss of $516.8 million. EPS would\u2019ve missed by $0.18 even without it, due to ramp ups in research and development ($245.7 million, +26% year-over-year) and selling, general and administrative expenses ($110.7 million, +54% YOY). Another beat in revenues saw 28% growth YOY (Table 1), although EXONDYS sales may be close to peaking. Sarepta still has a strong cash position of $1.9 billion as of March 31, 2023.<\/p>\n

Table 1. Quarterly Revenues (in $ thousands)<\/p>\n

<\/span><\/p>\n\n\n\n\n\n\n\n\n
<\/td>\n\n

Q1 2021<\/p>\n<\/td>\n

\n

Q2 2021<\/p>\n<\/td>\n

\n

Q3 2021<\/p>\n<\/td>\n

\n

Q4 2021<\/p>\n<\/td>\n

\n

Q1 2022<\/p>\n<\/td>\n

\n

Q2 2022<\/p>\n<\/td>\n

\n

Q3 2022<\/p>\n<\/td>\n

\n

Q4 2022<\/p>\n<\/td>\n

\n

Q1 2023<\/p>\n<\/td>\n<\/tr>\n

\n

EXONDYS 51<\/p>\n<\/td>\n

\n

107185<\/p>\n<\/td>\n

\n

112461<\/p>\n<\/td>\n

\n

115598<\/p>\n<\/td>\n

\n

119117<\/p>\n<\/td>\n

\n

117133<\/p>\n<\/td>\n

\n

126377<\/p>\n<\/td>\n

\n

122262<\/p>\n<\/td>\n

\n

145977<\/p>\n<\/td>\n

\n

132571<\/p>\n<\/td>\n<\/tr>\n

\n

AMONDYS 45<\/p>\n<\/td>\n

\n

193<\/p>\n<\/td>\n

\n

6936<\/p>\n<\/td>\n

\n

26655<\/p>\n<\/td>\n

\n

34745<\/p>\n<\/td>\n

\n

43614<\/p>\n<\/td>\n

\n

54676<\/p>\n<\/td>\n

\n

54893<\/p>\n<\/td>\n

\n

61399<\/p>\n<\/td>\n

\n

65912<\/p>\n<\/td>\n<\/tr>\n

\n

VYONDYS 53<\/p>\n<\/td>\n

\n

17548<\/p>\n<\/td>\n

\n

22442<\/p>\n<\/td>\n

\n

24658<\/p>\n<\/td>\n

\n

24863<\/p>\n<\/td>\n

\n

28078<\/p>\n<\/td>\n

\n

30184<\/p>\n<\/td>\n

\n

30619<\/p>\n<\/td>\n

\n

28557<\/p>\n<\/td>\n

\n

33012<\/p>\n<\/td>\n<\/tr>\n

\n

Products, net<\/p>\n<\/td>\n

\n

124926<\/p>\n<\/td>\n

\n

141839<\/p>\n<\/td>\n

\n

166911<\/p>\n<\/td>\n

\n

178725<\/p>\n<\/td>\n

\n

188825<\/p>\n<\/td>\n

\n

211237<\/p>\n<\/td>\n

\n

207774<\/p>\n<\/td>\n

\n

235933<\/p>\n<\/td>\n

\n

231495<\/p>\n<\/td>\n<\/tr>\n

\n

Collaboration<\/p>\n<\/td>\n

\n

22005<\/p>\n<\/td>\n

\n

22250<\/p>\n<\/td>\n

\n

22495<\/p>\n<\/td>\n

\n

22736<\/p>\n<\/td>\n

\n

22005<\/p>\n<\/td>\n

\n

22250<\/p>\n<\/td>\n

\n

22495<\/p>\n<\/td>\n

\n

22494<\/p>\n<\/td>\n

\n

22005<\/p>\n<\/td>\n<\/tr>\n

\n

Total revenues<\/p>\n<\/td>\n

\n

146931<\/p>\n<\/td>\n

\n

164089<\/p>\n<\/td>\n

\n

189406<\/p>\n<\/td>\n

\n

201461<\/p>\n<\/td>\n

\n

210830<\/p>\n<\/td>\n

\n

233487<\/p>\n<\/td>\n

\n

230269<\/p>\n<\/td>\n

\n

258427<\/p>\n<\/td>\n

\n

253500<\/p>\n<\/td>\n<\/tr>\n<\/table>\n

<\/span><\/span>