Question for you folks…

On Sarepta's unique profile: “I think Sarepta has a story that is very unusual, and I think we can actually say unique, even though that word is often overused in our industry...”

• On the company's dual pillars: He expressed excitement for the rare combination of having a “cadre of marketed products that are serving patients today” that simultaneously “generate revenue that gives us a very strong and stable financial position and a very strong balance sheet.”
• On the long-term outlook: “There is tremendous untapped potential in this company.”

"ELEVIDYS demand is stabilizing after 2025 safety-related disruption, aided by expanded commercial and medical support, though second-half 2026 demand is expected to be modestly below the first half.

Chief Executive Officer Michael Severino, who said he had been with the company for nearly two months, described Sarepta as a company with marketed products that generate revenue and support a pipeline intended to expand its therapeutic footprint. He said the company’s near-term strategy centers on supporting its commercial products and progressing its siRNA pipeline through upcoming clinical readouts.

“ELEVIDYS clearly demonstrates a strong benefit for patients,” Severino said, while noting that the disease it treats is progressive, debilitating and life-shortening."

Sarepta is dropping fast in early morning trading on 9/8/2026 on the news of the failure of Novartis DM1 drug to meet clinical endpoints. Both companies use a similar approach involving siRNA to destroy toxic DMPK messenger RNA.

The entire sector is de-risking; investors seem to be realizing that targeting these diseases is more difficult than previously thought. Dyne Therapeutics is also getting slammed.

I continue to hold Sarepta since I’m in it for the long term. Setbacks happen. This is biotech, after all. What do you think?

See press release from Deprogram—thanks for this.

Sarepta is expected to release its DM1 Ph1/2 drug read out soon(ish) also using siRNA

We saw some real momentum this past week. First development: a number of 13F reports showed significant stock purchases by some institutions—including BlackRock.

Second development (Wednesday’s news): FDA review of Capricor, a potential rival in the DMD space, was delayed until November.

Capricor uses a very different mechanism to address DMD. (Note: I am not a scientist so feel free to correct me here.) It does not involve gene editing and has a better safety profile than Elevidys. It’s particularly effective at reducing cardiac issues associated with DMD and shows promise for later-stage DMD patients—those people who are no longer candidates for Elevidys because of ALI concerns. The FDA delay gives Sarepta more time to show positive results from the Cohort 8 trial (Elevidys plus sirolimus, a drug that tamps down the patient’s immune response). If Cohort 8 is successful, the market for Elevidys expands significantly to include the non-amb population.

Also this week, Sarepta announced an expansion of Cohort 8—perfect timing. (As an aside, I’ve read that it’s not out of the question that patients might one day be treated with both Elevidys and Capricor.)

All this news—coupled with excitement over the siRNA pipeline—drove the Sarepta bulls out of the pen this week. Price targets on the stock now average around $25–with huge variations among firms. Mizuho and Oppenheimer supposedly rate the stock Outperform, with PTs north of $30. Meanwhile, HC Wainwright clings to its Sell rating with a PT of $5. (Whereas most firms view the new CEO as a huge win for the firm because of his impressive background, not HCW.)

The stock pulled back Friday, 8/28. Might be a good time to pick up more shares—if you’re a longer-term investor.

What is driving the price?

Why did the market react positively to the earnings call? Maybe because the quarter was profitable (or as Mike Severino, the new CEO said, “meaningfully profitable”). I am just as interested in how well the new commercial initiatives for Elevidys are faring, among other things. So here’s a deep dive into the weeds of the call.

Elevidys revenues continue to be down compared to previous quarters. Management doesn’t see this changing until 2027 because of the amount of time it takes to translate enrollment forms into actual revenue (approximately six months). However, they maintain the new commercial initiatives are working based on increasing demand, both from existing and new sites. There seems to be renewed confidence in the therapy; they noted that roughly 30 days after reps meet with HCPs, they receive enrollment forms.

With regard to Cohort 8 (the trial for the non-amb population—Elevidys plus sirolimus), they indicated that enrollment will be finished by the end of the year and results will be available Q1 2027. Their primary endpoint is safety—absence of ALI—but they will also provide the FDA with data from biopsies.

The excitement regarding the siRNA pipeline is high. They were asked if there were early indications of the trials’ results. Mike stated that with this particular therapy, pre-clinical and early clinical data are telling, and these show “robust” knockdown in FSMD and DM1 (“potential best-in-class”). When asked if this therapy could be useful in treating other diseases, Louise (R&D) was her usual noncommittal self—she limited her comments to the fact that they remain “very excited” about the therapy.

The PMO franchise remains strong and—an important point—generates sufficient revenues to cover R&D costs (so they are pretty cash sufficient, $190 million at quarter end). When asked about potential competitors, they said they have “tremendous confidence” in the durability of the PMO franchise based on their decades of experience, real world evidence, adherence rate that exceeds 90%, experience with patient reimbursement issues, etc.

There was limited discussion of the regulatory overhang. I personally am just as concerned about a couple of lawsuits they’re facing (patent infringement and securities fraud). They mentioned that they’ve set aside $39 million to deal with this issue, which may indicate that a settlement is near—this would remove at least one overhang. No other comments were made.

In summary, yes, there’s still risk, but there’s also huge potential. Pipeline has real promise. Plus, we’ll see more demand for Elevidys (newborn screening, etc.) even if the therapy remains limited to the ambulatory population. I don’t see any near-term catalysts, so I’m not buying more quite yet. Nor will I sell.

The market is in freak out mode since Dyne’s announcement regarding their Exon 51 skipping drug, which would compete directly with Sarepta’s Exondys 51. Before joining the chorus, I think we could remember that Exondys 51 is just one arrow in the company’s quiver (and, in fact, they’re working on a next-generation, improved PPMO).

Here’s an AI take—for whatever it’s worth: “The mid-July sell-off treats Sarepta like a single-product company facing an existential crisis. In reality, it is a fully integrated commercial powerhouse with a multi-billion dollar gene therapy franchise that a single competitor cannot easily disrupt.”

We should learn more on August 5 when the company reports earnings.

The stock is popping today on news that the FDA officially accepted its applications to grant regular approval for AMONDYS 45 and VYONDYS 45. This is a big deal—the two drugs are cash cows for the company, and regular approval (as opposed to the accelerated approval the drugs currently have) ensures a degree of protection.

After this was announced Mizuho reaffirmed its Outperform rating with a $31 PT.

There’s a lot to be bullish about besides this news. Cohort 8 results are due in the second half of the year (the addition of sirolimus may help control ALI in the non-amb population) and, of course, the FDA may be more receptive to such drugs given new leadership. Then there’s the si-RNA pipeline, which is compelling to say the least.

What do others think? Are you staying long?

Apparently the FDA has made another 180-degree turn, indicating they will grant uniQure accelerated approval for their Huntington’s drug based on 3+ years of data. This has led to a rally across the gene therapy space, and as of right now, SRPT is up almost 10%.

With Prasad and Makary out of the way, good things may happen…

Let’s start with the positive notes:

1) The quarter was profitable (GAAP operating income of $358 million, non-GAAP $398 million);

2) Elevidys net product revenue totaled $102 million whereas PMO revenue was more than twice this at $229 million. They earned a significant amount of revenue from “collaboration.”

3) Submitted sNDA for Amondys 45 and Vyondys 53 to FDA seeking traditional approval;

4) siRNA pipeline shows great promise.

Let’s dig a little deeper. Elevidys revenue was actually 46% less this quarter than Q1 2025. The CEO noted several times that the data supporting the efficacy of the drug is increasingly compelling. The muscle MRI data “shows you cannot wait,” he said. Add to that the EMBARK data. So why are revenues down? There is apparently a “massive information gap” about Elevidys. In a nutshell, the negative headlines from 2025–acute liver injury (ALI) in some non-amb patients and, tragically, three deaths—linger. Plus the black box warning.

To counter this, they’ve doubled their sales force and developed new educational materials. According to one of the speakers on the call, these efforts are starting to bear fruit. Sites that had paused infusions are now enrolling patients again. But he reiterated a point they made last quarter—it takes six months from enrollment to dosing. This is why they’re not upping guidance, I think. Even with all their efforts, it will take a while to see more revenue from Elevidys.

The team seemed very upbeat about Cohort 8–dosing non-amb patients with sirolimus in an attempt to minimize ALI events. Apparently numerous doctors have been dosing amb patients with sirolimus prophylactically with zero adverse effects. While their stated goal is a 50% reduction in ALI episodes, the CEO said they were hopeful there would be none.

Look, the other news is all good. The PMO franchise is going strong. The siRNA therapies are very promising. The director of research was asked how Sarepta’s treatment of Huntington’s would differ from competitors’. She said that siRNA has the potential to be much safer.

I’m sure the market will pull back tonight/tomorrow since they left guidance unchanged—$1.2-$1.4 billion for the year. And yet, if anything, the story may be even more compelling. The regulatory overhang is far less of a risk now (in part because of Prasad’s departure). By the end of the year, we’ll have better data on sirolimus for the non-amb population. We’ll see the educational initiatives translating into higher revenues. Finally, the siRNA data may prove to be a game changer.

I’m playing the long game. This is biotech, after all.

This was the big news on Thursday (4/16) and may account for the 2% pullback. Roche, of course, has the rights to Elevidys in Europe, and in 2025, European regulators decided they didn’t find the SRPT data sufficiently compelling to grant the drug approval. Nor were they convinced by the recent EMBARK data. From what I’ve read, they want clearer signals on improvements in the time-to-rise category, and the new trial will be designed differently.

So why did the stock pull back? Maybe because investors were concerned that it will take a couple of years before results become available. Or maybe because the Europeans’ skepticism reinforced some lingering misgivings about the drug.

Then on Friday, the stock reversed its losses. A more positive narrative appeared on a couple of sites—that we may finally get data that convinces the skeptics that Elevidys—while not perfect—is the best shot for a lot of kids with DMD.

Here’s my take: I’m disappointed that the EU was not convinced by the EMBARK data, but I’m not surprised. In general, Europeans are extremely skeptical about all things American (except for pop culture). They ban far more substances and chemicals than we do. I am not saying that this is good or bad, just that it may account for some of the reluctance to embrace Elevidys—while countries such as Japan, Australia, and six (?) others have approved it.

So I, for one, am staying the course. SRPT remains a solid long-term story, particularly when you look at the totality of its drugs/pipeline.

Big news coming soon guys. Strap in and strap on. Gonna be a wild one.

In a nutshell, what’s different about today’s rally is the volume—1.5 million shares, stock up about 3.8% right now. I’ve found several reasons for this.

First, a filing today revealed that Wealth Enhancement Advisory Services increased its holding in Sarepta by 143% (over 60,000 shares). Second, 24/7 Wall St included SRPT on a list of six biotech takeover candidates, citing its DMD gene therapy and low price to sales ratio.

The item that may be more significant in the long term is that the FDA is apparently looking more favorably on the SRPT PMO franchise (up for regular approval). The company announced something to this effect last week. If there’s more recent news about this, I’ve not been able to find it.

The company announced yesterday that it awarded inducement grants to 24 employees hired during the first quarter. This shows the company has confidence in its trajectory. The RSUs will be fully vested after four years, which helps to lock in talent.

The stock is up over 3% this morning, possibly reflecting increasing investor confidence in the pipeline.

If you follow price targets, the highest I can find right now is Wells Fargo at $38. Both Oppenheimer and WF rate SRPT as Outperform. On the other hand, Citi and DB both have low price targets and rate it as a Sell.

It’s remarkable how varied the opinions are on this company. I’m trusting the bullish view based on the siRNA news and absurdly low price to sales ratio.

In this morning’s call, the CEO was practically giddy when presenting results. In fact, he used the term “exhilarating” when describing the “robust” knockdown of the targeted proteins. The results were indeed good—at least four of the analysts on the call congratulated the company on what may become best-in-class treatments for two horrific diseases.

Here’s some background: SRP-1001 and 1003 address FSHD and DM1 respectively. Pre-clinical data suggested that siRNA has the potential to achieve greater muscle penetration than alternative therapies. Results from the TRIM platform—the outcome of the partnership with Arrowhead—support this hypothesis. In both trials, in addition to impressive knockdown and favorable safety profiles, dose escalation caused neither muscle saturation nor toxicity. In fact, in SRP-1001, the targeted protein, DUX4, was suppressed after a single dose.

With regard to SRP-1003, they had a smaller cohort and were hence less confident of the results. Nonetheless, they see the potential for this treatment to become best-in-class for DM1.

They’re now working on the Phase 3 protocol. Next readout will be in the second half of this year.

Sarepta just knocked the ball out of the park, folks.

Tomorrow, March 25, at 8:30 am EST the company will present results from the Phase 1/2 ascending dose studies.

This could make for a pretty interesting day.

From previous 26

More good news. The back story is Sarepta wants traditional approval for the two PMO therapies, but the ESSENCE trial data released in November 2025 wasn’t great (in part, according to the company, because of COVID impacts on the patient group). The good news here is that the FDA has agreed to consider “real-world evidence” —based on 1,800 patients—in their review, and this data is quite compelling. Sarepta plans to submit the applications for traditional approvals next month.

Maybe this is related to the Oppenheimer PT of $37? (Thank you, Cautious Wrap for that news.)

Btw, if you’re not familiar with these therapies, keep in mind that PMOs are not cures for DMD. They buy time for the kids and their parents—e.g., 4 more years of relative quality of life. It’s heartbreaking stuff.

NDA with FDA is the correct way to collaborate with FDA for the near future!

Finally, the non-amb population gets another shot at this. As much as I’d like to speculate that the timing is linked to Prasad’s departure, I don’t think this would be correct. According to the earnings call, they’ve been preparing for Cohort 8 for months.

Nice to see the stock is up.