SRPT Sarepta Therapeutics Inc

$SRPT Some highlights from the data yesterday worth re-posting… In the Phase 4 ENDURE interim analysis, none of the 11 ELEVIDYS patients who received preventive sirolimus developed acute liver injury (0/11). By comparison, liver injury occurred in 9 of the 45 patients who did not receive preventive sirolimus. Sarepta concludes that preventive sirolimus appears to mitigate the risk of liver injury, while emphasizing that the sample is still small.

$SRPT since the beginning of 2026: increased cash balance, new ceo, most mandatory arrowhead payments done, regenxbio settlement closed, good early results for elevidys + sirolimus safety, good early results for sirna efficacy and safety, elevidys approved in Japan, Roche took on themselves retry of Elevidys studies for EMA

$SRPT This is a once-in-a-lifetime opportunity! The company’s fundamentals haven’t changed, and I strongly believe the stock has the potential to recover and return to the $150+ range. The current price could be an attractive entry point for long-term investors. 🚀 Sustained growth in ELEVIDYS sales. Favorable FDA decisions, including a potential pathway for treating non-ambulatory patients. Strong results from the DM1 and FSHD clinical programs. Improved investor confidence under the new CEO.

$SRPT It’s difficult to imagine management prominently highlighting 80% DMPK knockdown and correction of abnormal splicing this close to the MAD readout unless they were highly confident in what’s coming. Granted, the 80% was achieved in patient-derived muscle cells studied outside the body, but management likely already has substantial visibility into the human MAD data. My bet is that the results will blow Avidity’s out of the water Of course, Avidity’s failure to meet PE in Phase 3 has raised the bar. Even exceptional MAD results may not fully convince investors until sarepta demonstrates meaningful functional improvements in later-stage trials. However, with so many potentially positive catalysts on the horizon, it’s hard for me to imagine we won’t see some meaningful pops in the share price over the next 1-6 months, followed by a more sustained re-rating as siRNA and legacy platform’s potential becomes increasingly clear.

$SRPT $ARWR has just proven that their subq shot can cross the BBB, deliver siRNA and effectively knockdown tau . This just further validates SRP 1005 for Huntingtons

$SRPT ZERO instances of ALI in all 11 participants who received sirolimus prophylacticly in this report compared to a 25% occurrence of ALI in participants who did not receive Sirolimus before, at, or slightly after treatment.

$SRPT I suspect ARO MAPT will be presented next week . That will be a big deal for sarepta huntingtons program . If they can show knockdown and entry past the BBB that will further validate SRP 1005 and the platform. IMO much bigger pop than these conferences .

$SRPT We’ll get insight on the platform we got from $ARWR over the next few days. CEO is pumped on it so let’s see what this does for the kids and our investments.

$SRPT the next 6 months are some of the most important months in this company’s history IMO 2 critical siRNA readouts Cohort 8 and getting non amb back up and running PMO franchise decision Going to be spicy.

$SRPT has fallen sharply from its 2020 peak. The stock’s all-time closing high was $178.74 on December 22, 2020, with an intraday high of approximately $181.83 that same day. Its recent 52-week high was $25.32, reached on October 8, 2025. The decline has been driven mainly by ELEVIDYS safety concerns, restrictions that reduced its potential market, and setbacks in Sarepta’s clinical pipeline. Still, the company generates meaningful revenue, has an established commercial product, and retains significant long-term potential. The key question is whether ELEVIDYS can stabilize and whether upcoming pipeline data can restore investor confidence. Positive clinical or regulatory developments could provide a meaningful catalyst for the stock. SRPT remains a high-risk stock, but its depressed valuation and future pipeline opportunities make it worth watching closely. 📊 still have hope!!!!

$SRPT Yes, Sarepta Therapeutics anticipates a robust presence at the 2026 World Muscle Society (WMS) Congress (taking place September 29 – October 3, 2026).According to Sarepta's Corporate Updates and Investor Relations Filings, their scheduled data presentations and overviews at WMS 2026 include:Elevidys Data: Supportive expression and safety data in ELEVIDYS-treated patients under the age of 4.EMBARK Study: Encore presentations detailing 3-year outcomes from the Phase 3 EMBARK study.Additional Insights: Cardiac function data, pooled safety analyses, and early-intervention preclinical data from their broader neuromuscular portfolio.

$SRPT Timeline/Dates to keep an eye on over the next 6 or so months. Q4 2026: DM1 & FSHD MAD data, biomarker data, CASI-22 splicing index (DM1), preliminary functional assessments (FSHD), vHOT (DM1) Oct 27th, 2026: Novartis Q3 Earnings. Potential update on if Avidity’s del-brax for FSHD can file a BLA under the Accelerated Approval pathway. Nov 22nd, 2026: Capricor’s Deramiocel PDUFA date Oct 31st, 2026: Exondys MIS51ON trial estimated primary completion date. Topline data would be expected 4-6 weeks later possibly in December, but could be longer. Previously Sarepta guided for end of 2026 readout. Q1 2027: ENDEAVOR Cohort 8 Sirolimus data Q1 2027: SRP-9003 LGMD2E/R4, Meet with FDA with Sirolimus data and align on path forward for lifting clinical hold and filing BLA Jan 21st, 2027: DYNE-251 Dyne’s Exon 51 skipper/Exondys competitor PDUFA date. Feb 28th, 2027: Amondys & Vyondys PDUFA date 1H 2027: SRP-1005 Huntington’s disease proof of biology data

$SRPT ton of important data this afternoon and next week and most of you don’t seem to care because the macro sucks

A deeper look at that 0/11 sirolimus cohort in the $SRPT ENDURE data: while management is right to point out the sample is still small, Bayesian math actually gives a roughly 70% probability that the true underlying liver injury rate is actually under <10% with preventive sirolimus (compared to 20% without at 9/45). Sure, small numbers mean wide confidence intervals, but the mathematical and biological probability that this real-world mitigation is actually working is heavily skewed in our favor.

$SRPT sarepta struck the deal with $ARWR in 2024 and it was pretty significant . Upfront and near term payments totaled well over 1 billion dollar with the total deal potentially worth over 11 billion. More than two years later , can you imagine what that same deal would look like .? I asked chat GPT just for fun . Know what you own , NFA.

$SRPT DM1 MAD will be “Wow!!” FSHD MAD will validate platform and delivery mechanism are real. Huntington’s proof of biology will be: “Holy shit. They’re sitting on a platform capable of producing multiple blockbuster drugs.” That’s the progression. Each successful dataset validates and reprices the entire pipeline.

$SRPT SRP-1003: 8.5x more siRNA in the muscle than in Avidity therapy and 20 percentage points higher nuclear DMPK knockdown

$SRPT Should’ve been up 15-20% on today’s news. Early DM1 data and Arrowhead validation for HD

$SRPT Srapeutics announced at 8:30 a.m. ET today, September 25, that it will present new ELEVIDYS data at the World Muscle Society meeting next week. Most notably, Sarepta has a late-breaking presentation examining ELEVIDYS efficacy and safety in older ambulatory Duchenne patients who were 8–12 years old when treated.

$SRPT A year ago today, this stock was $18.90. Hard to believe the crazy volatility during this past year. I have owned this stock for over 20-years and it used to be my largest personal holding. Today, it’s a very small holding. However, today I’ll be adding for the first time in a long time.