Toward the end of the October 5 lightning round of Mad Money, a caller asked Jim Cramer about Sarepta Therapeutics, Inc. (NASDAQ:SRPT). He replied:
This is a very controversial stock… I want everything to work for people who have this and the families who have it, but I cannot opine on it because it’s just too difficult for me to understand. I’ve just got to own that.
New Follow-Up Data Provides a Clinical Development
On September 30, Sarepta Therapeutics, Inc. reported two-year results involving 25 ambulatory patients aged 8 – 12 who received Elevidys, compared with 99 patients in an external control group. The company reported functional benefits relative to those controls. Because this comparison used an external control rather than a randomized concurrent control group, the study design matters when interpreting the findings.
Commercially, the company generated $328.7 million in second-quarter product revenue, including $230.6 million from its PMO therapies and $98.1 million from Elevidys. It reported GAAP operating income of $13.3 million and approximately $945 million in cash, restricted cash and investments. Sarepta’s cash and investments were also central to earlier coverage of its place among Renaissance Technologies’ holdings, which examined how management planned to fund its next stage of development.
The shares traded at approximately 0.9x trailing sales, compared with 3.2x for rare-disease drugmaker BioMarin Pharmaceutical. Sales multiples avoid some of the distortions in Sarepta’s earnings figures, but the discount must be considered along with its declining revenue and product-specific risks.
Safety Restrictions Remain Central to the Outlook
Elevidys carries an FDA boxed warning for serious liver injury and acute liver failure, including fatal outcomes. In November 2025, the FDA restricted its indication to ambulatory patients aged four and older with a confirmed mutation in the DMD gene, following reports of fatal liver failure in nonambulatory patients. The September follow-up findings do not remove those restrictions or the warning. They add information about a particular patient group, while the approved population and safety requirements continue to shape the commercial opportunity. Even after the label restrictions, management outlined a path back to growth for Elevidys. The assumptions behind that outlook featured in earlier coverage of Sarepta’s place among gene therapy stocks to watch.
Sarepta’s total second-quarter revenue declined to $401.3 million from $611.1 million a year earlier, showing lower Elevidys revenue and changes in collaboration revenue. It highlights that a low trailing-sales multiple can be misleading if investors assume the historical revenue base will remain intact.
Heavy Short Interest Accompanies Fewer Fund Holders
As per Insider Monkey’s data, there were 37 hedge funds holding Sarepta Therapeutics, Inc. in the second quarter, down from 41 in the first. AQR Capital Management was the company’s largest hedge fund holder with 4.3 million shares. Additionally, Marshall Wace LLP increased its position in the stock by 205% to 2.2 million shares. Short interest stood at 28.86% of the float. That is substantial bearish positioning and can contribute to sharp swings around clinical or regulatory news. It does not, by itself, establish that the shares are undervalued or that a short squeeze will occur.
Heavy short selling was already part of Sarepta’s story when it returned to profitability earlier this year. Its inclusion among heavily shorted small- and mid-cap stocks examined what was behind that earnings recovery. Sarepta has commercial products, cash resources and new follow-up data to discuss. Investors should also remember that it has serious safety restrictions and a smaller revenue base than a year earlier.
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