@bradloncar Funny how you never see the videos of kids who can't walk up stairs or get up from the floor after taking a gene therapy. Sure, videos like the one shared by $SLDB and previously by $SRPT are heartwarming and encouraging, but they are not data.
Roche to launch another Elevidys trial, with eyes on European approval https://t.co/eAANNiihpt via @DrewQJoseph $SRPT
Sarepta Therapeutics $SRPT shares rise on early promise for rare disease drugs. New results from small studies could support the beleaguered company’s pivot. https://t.co/fRuvcTmt0i via @damiangarde
Let's look at how the FDA is treating $SRPT and $QURE differently. The FDA grants accelerated approval to Amondys and Vyondys to treat Duchenne muscular dystrophy. Sarepta conducts a post-marketing confirmatory study that fails to show a benefit for either drug. There is no evidence that patients are benefiting from either drug, based the outcome of a randomized, controlled study. Still, Sarepta pushes forward, seeking to convert accelerated approval to final approval. The FDA allows Sarepta to submit an application. "The adequacy of the data to support conversion to traditional approval will be a matter of review," the FDA concludes, according to Sarepta. Amondys and Vyondys should be withdrawn from the market. My opinion. I've said that repeatedly. There's a process, of course, so FDA should review the data. That's what regulators are supposed to do. And then, there's UniQure and AMT-130, its treatment for Huntington's disease.... The FDA won't even allow UniQure to submit an application for accelerated approval. Bar the door, the FDA says, not even a review. https://t.co/CmDs13aVry
This week's Biotech Scorecard: Catching up on non-Prasad work. -- $ALDX is complaining about short sellers in the run-up to an FDA approval decision. Red flag! -- The $VRTX era of frictionless commerce is coming to an end. IgAN competition. -- $SRPT at a CEO crossroads. https://t.co/QIgn9e15Hd
$SRPT Sarepta Therapeutics CEO Doug Ingram will retire after a tumultuous decade https://t.co/EBnOUKSLJg via @Jasonmmast
$SRPT CEO Doug Ingram is out https://t.co/0IAMBkji3d
In this morning's press release, this is all that $SRPT discloses about Elevidys safety, one year after liver toxicity, liver-injury related hospitalizations and deaths due to acute liver failure nearly ended the gene therapy. "No new treatment-related safety signals were observed, reinforcing the consistent and manageable safety profile seen in ambulatory patients treated with ELEVIDYS to date."
Thank you! I’m particularly proud of my $SRPT work last year. Accountability journalism at its best. I’m glad you recognize it.
@CloisterRes I hope $SRPT tells how many Elevidys patients have been hospitalized for liver injury. That's really the only relevant data left to be told.
$SRPT Elevidys Q4 sales $110M vs consensus $120M At his #JPM26 presentation, CEO Doug Ingram notes 80% of addressable ambulatory DMD patients remain untreated. He sees this a huge opportunity for Elevidys, but I will note it's also the same for $SLDB and $RGNX and others developing next-gen gene therapies.
The Worst Biopharma CEO of 2025 made decisions with tragic consequences Doug Ingram, Sarepta Therapeutics. $SRPT Is anyone surprised? Does anyone disagree? https://t.co/1I7VrJFzpC
$DYN Dyne Therapeutics plans to file for approval for next-generation Duchenne drug. The medicine could be a more effective version of controversial Sarepta $SRPT drug https://t.co/BBGe0cju3l via @Jasonmmast
This week's Biotech Scorecard newsletter: Richard Pazdur surely has many items on his to-do list now that he’s the top drug regulator at the Food and Drug Administration. One of the decisions he will soon help make — how to deal with the dangling accelerated approvals given to Sarepta Therapeutics $SRPT for its Duchenne muscular dystrophy drugs — will tell us a lot about how much independence and scientific rigor Pazdur intends to bring to the new job...
FDA’s stronger warning on Sarepta $SRPT gene therapy raises new questions about heart risk https://t.co/xY4146JVTI via @Jasonmmast and me
$SRPT New Elevidys label https://t.co/N0eknOP1aR
Lastly, I revisited $SRPT and the absurd claim of a "positive trend" for Amondys and Vyondys. The treatment difference between Amondys and Vyondys, Sarepta’s exon-skipping drugs, and a placebo was six-hundredths of a step per second on a timed test that asked patients to walk up a flight of four stairs. 0.06 steps/second is a meaningful improvement in velocity if you’re an elite swimmer or runner training for the Olympics. For boys with Duchenne — or for any person going about their day — it’s imperceptible and totally meaningless.
Sarepta’s $SRPT dismal quarter marred by drug failures and a long list of excuses https://t.co/FZor1xE0Ap
$SRPT changed the primary endpoint of the ESSENCE study bc they believed the new endpoint was more sensitive -- and it still failed.
$SRPT now -35% after hours.
$SRPT said Q4 Elevidys infusion volumes would be flat or lower than Q3.
$SRPT - CEO Doug Ingram is very good at making excuses for why ph3/confirmatory studies fail.
What's the bigger shock? That $SRPT failed a confirmatory trial, or that it ran one?
$SRPT still has never conducted a successful Phase 3 or confirmatory study.
$SRPT Amondys/Vyondys confirmatory study failed, company will file for full approval anyway
Sarepta $SRPT to seek approval for gene therapy in rare form of muscular dystrophy “The company has said it plans to file for approval in the disease, known as limb girdle muscular dystrophy (LGMD) 2E. That would make it the first approved treatment in LGMD, a broad collection of highly rare diseases that can deprive patients of the ability to walk and in some case shorten life. But it is likely to face a significant uphill battle. “ https://t.co/BVIUZUfeuF via @Jasonmmast
@tmulpuri @JenniferHandt @Sarepta The NY State vote on $SRPT Elevidys was last Friday. Technically, “last week” but it took my colleague a day plus to get clarity on what the decision meant and its implications. That’s called reporting. It takes time. More than a tweet. The suggestion that we delayed a story for any reason is false and absurd.
Adam, why are you not reporting on the $SRPT Elevidys 3-yr data being presented today at WMS? Because these very same data were already presented at conferences in May and March. The data ARE NOT NEW.
$SRPT Sarepta suffers a setback as N.Y. panel recommends state Medicaid pause coverage of Duchenne gene therapy Elevidys https://t.co/8f1N0ugfLf via @pharmalot
$AUPH down on this Tidmarsh comment. $SRPT is not.
$SRPT recovery stalled.
@Alpha_bro1 @MattB79569101 @commonsenseplay @Sanctuary_Bio The FDA approved Elevidys despite the failure of its clinical trial. Secondary endpoints can’t “succeed” if the primary endpoint fails. The FDA did not set the primary endpoint of the Elevidys study. $SRPT did. Sarepta was confident NSAA at 1 year would be enough to prove Elevidys benefit based on lessons learned from the prior studies. Sarepta was wrong. Do you honestly not know any of this, or are you just shit posting?
@MattB79569101 @commonsenseplay @Sanctuary_Bio No. The $SRPT EMBARK study failed to demonstrate Elevidys’ benefit. The negative results were reported in 2023 and published. What you’re referring to is an open label, post hoc comparison against an external control at two years. By definition, these data cannot be assessed for statistical significance, and have limited clinical relevance, if any.
@commonsenseplay @Sanctuary_Bio During the last reporting reporting period (June 30), Avoro, Farallon, Ecor1, Alkeon, Camber and many other funds all closed out their $SRPT long positions. Janus reduced its stake by 94%. T. Rowe, Deep Track also sold down their positions significantly. What makes you think "whales" are buying back in?
@matthewherper @BiotechObserver @john_hersc79276 @Biohazard3737 More excuses and gaslighting from the $SRPT cult. 6MWT was the best endpoint, until it wasn't. NSAA was the best endpoint, until it wasn't. Now, time to rise is the best endpoint?
@Biohazard3737 Couple of responses: "If Elevidys is disease modifying..." This gets to the root of all $SRPT criticism, legitimately. The company has never conducted a successful clinical trial - meaning demonstrably proven any of its drugs/gene therapies are effective or beneficial for patients. That's a tremendous problem that should never be ignored. What is the acceptable death rate? That's a great question. I don't know. I'd like $SRPT or those who think deaths don't matter to provide an answer. There will be more Elevidys-related deaths, including in ambulatory patients. I agree. Tragically. So, what will the FDA do when it happens?
Read the story here: As Sarepta Therapeutics $SRPT fights for Duchenne therapy, a group of patients gets left behind Families with limb-girdle muscular dystrophy hoped for gene therapy but ‘overnight, it’s gone’ https://t.co/3hkMeGfWIq
From Jason's $SRPT story: Even as Sarepta fought to keep its Duchenne treatment Elevidys on the market, despite the deaths of two teenagers, it pulled out of a nearly decade-long commitment to develop gene therapies for limb-girdle muscular dystrophy, a lesser known collection of over 30 ultra-rare and debilitating muscle disorders. The news devastated patients and families, who for years watched their programs inch along while Sarepta devoted most of its resources to Duchenne, the most well known and universally fatal form of muscular dystrophy. The company had only just started a clinical trial for Jacob’s subtype, LGMD 2D, in January, one of at least four LGMD studies it planned to launch or file this year. “Families in our community are left asking hard questions right now in rare disease drug development,” Kathryn Bryant Knudson, head of the Speak Foundation, the largest LGMD advocacy group, said in an email. “Families want their children to have the right to try these treatments.”
$SRPT refinanced debt while abandoning patients with limb girdle muscular dystrophy and their families. CEO Doug Ingram talks about "tranforming the lives of patients with rare diseases," but that doesn't include kids like Jacob, 4, Sammy, 17 and others. My colleague @Jasonmmast tells their stories today. Link below.
Sarepta $SRPT hired a Trump-connected firm to lobby on Duchenne treatment https://t.co/mHKwoEEzAK via @john_wilkerson
Are we sure Vinay Prasad’s ouster is positive for biotech? Some musings in this week's newsletter. A commonly held view of his three-month tenure running the FDA’s biologics division was that he was more obstructionist and inflexible toward cell and gene therapies than one might have predicted, even accounting for his reputation as a conservative data nitpicker. The FDA rejected $REPL $RARE and $CAPR. Prasad went to war against $SRPT His departure means the FDA will revert to its more permissive, flexible ways, the thinking goes, and that’s good for developers of cell and gene therapies, good for makers of drugs for rare diseases, and a tailwind for biotech overall. I’m not so sure. You know me by now. I have a hard time fully buying into the rosy story. What if Prasad’s replacement as CBER chief is worse? By that, I mean a person who is more politically motivated and more in the MAHA, anti-pharma and anti-vaccine orbit of Health and Human Services Secretary Robert F. Kennedy Jr. Read more at link below, plus some thoughts on the road ahead for $SRPT
@JPZaragoza1 You come at the king, you best not miss $SRPT
Another twist... FDA permits use of Sarepta Therapeutics’ $SRPT Duchenne therapy in younger patients after short-lived halt https://t.co/AAZpW30smm
$SRPT news https://t.co/ANZFDSu9Zq
$SRPT The death referred to in the FDA statement may be the boy from Brazil who was disclosed earlier today by Brazilian authorities. Details in that announcement were scant, but the timing of that death and age described in the FDA statement fit what STAT had learned about that case. Will let you know if I confirm.
$SRPT FDA Investigating Death of 8-Year-Old Boy Who Received Elevidys So sad. https://t.co/TSLPxoXv0J
Amazing to watch anonymous cowardly trolls (not you, @PCM_bio ) criticize STAT for negative bias against $SRPT and then when we bring a Duchenne mother’s voice DIRECTLY to listeners, we’re also scheming somehow. The case exemplified by Kaylen, the mom we spoke with on the podcast, is not an anomaly. I’ll correct you here, Thomas. Yes, liver problems are a known risk factor, but the rate and severity in the real world setting are under-reported. Sarepta will not disclose hospitalization rates, despite being asked repeatedly. Regulators are very concerned, and it’s a key reason behind the actions taken over the past 2 weeks.
$SRPT Fully updated story w/ Brazil, Japan suspensions, Elevidys patient death in Brazil said to be due to influenza Europe moves to reject embattled Duchenne muscular dystrophy gene therapy https://t.co/jrKJVuY9ck via @DrewQJoseph
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy https://t.co/jrKJVuY9ck via @DrewQJoseph $RHHBY $SRPT
For many Duchenne families, halt to Sarepta $SRPT gene therapy treatments is heartbreak upon heartbreak https://t.co/wdq8Hmd55r My colleague @Jasonmmast with more reporting on the patient/physician perspective.
The podcast interview I mentioned earlier: A mother’s perspective on Sarepta’s $SRPT gene therapy halt https://t.co/R1QLcBCIxI via @statnews
This week's Biotech Scorecard digs deeper into the Sarepta $SRPT #Duchenne gene therapy debacle. 1. Sarepta may not survive. If Elevidys cannot return to the market (and STAT's reporting this week suggests it will not) the company is facing a severe cash flow and debt crisis. I dig deeper into the numbers. 2. Putting business and stock prices aside, this week has been wrenching for Duchenne families. On this week's Readout LOUD podcast, we spoke with the mother of a boy who was treated with Elevidys. She wants Sarepta and the FDA to reach an agreement that returns Elevidys to the market. Listen when the episode drops tonight. 3. What happens to Doug Ingram? His future as CEO hinges on two of the eight Sarepta board members, including investor Rick Barry. Remember him? Yes, he's the $SAVA guy, too. I take a closer look at some weird Sarepta board dynamics. Want to read more? The web edition of this week's newsletter can be read at the link below.
$SRPT Bloomberg story just published confirmed our reporting from yesterday.
Because it's always scientifically sound to run stats on a dataset designed to show something completely different $SRPT
Read more about $SRPT and the FDA officials comments here. https://t.co/wpvzowDcdj
To recap what we are reporting on $SRPT and its Duchenne gene therapy. A senior FDA official told us that Elevidys faces an “arduous and treacherous path” to try to get it back onto the market. It would be difficult for Sarepta to prove that any new safety protocols could eliminate the risk of liver injuries tied to Elevidys, the FDA official told us. “How do you show something is safe when it’s already proven to be not safe?” the official said. Story linked below, with more comments, background and detailed explanation of how this week went down inside the FDA.
Sarepta Therapeutics, $SRPT the maker of a gene therapy for Duchenne muscular dystrophy that is being temporarily shelved because of safety concerns, faces an “arduous and treacherous path” to try to get it back onto the market, a senior official at the Food and Drug Administration told STAT, suggesting the treatment’s license could be revoked.
EXCLUSIVE: Sarepta Therapeutics’ $SRPT Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says https://t.co/wpvzowDK2R via @Jasonmmast and me.
$REPL rejection follows the rejection of $CAPR and $RARE drugs, and the FDA's actions against $SRPT. CBER Director Vinay Prasad is setting a different tone than his predecessor.
$SRPT With Elevidys paused, risk to balance sheet and solvency is an issue again. Obviously, will depend on how long Elevidys remains off the market, and whether Sarepta can restructure debt if necessary.
Why did $SRPT reverse course? Sarepta says this "proactive" step will give it time to confer with FDA and remove Elevidys from its regulatory limbo (meaning, hopefully, keeping the drug on the market for ambulatory patients.) But Sarepta was also forced into this reversal bc hospitals were pausing use of Elevidys out of caution and safety/liability concerns, STAT learned. https://t.co/L76E4Ee8EJ
BREAKING: In surprise reversal, Sarepta $SRPT says it will pause shipments of Duchenne gene therapy https://t.co/cJW1ofyOUF via @Jasonmmast and me
$SRPT statement here https://t.co/0QiohT4XdV
$SRPT just announced pause to all Elevidys shipments in the US.
The crisis over Sarepta’s $SRPT Duchenne therapy is a mess. But the lesson is clear https://t.co/hA8382IL8I via @matthewherper
Meanwhile $SRPT https://t.co/4p3YDAriym
$SRPT pre market -10% to $12.61
$SRPT It’s always my fault. Or, the media’s fault. 🙄 Well, folks… I didn’t cause Sarepta’s clinical trials to fail. I didn’t decide to approve Elevidys for a broad swath of DMD patients. I don’t treat patients, nor influence whether they benefit or not. I’m not responsible for liver failure, hospitalizations or deaths. I didn’t set Sarepta’s material disclosure policy. The FDA didn’t ask for my advice on whether to ask Sarepta to halt shipments, and Sarepta didn’t consult with me when it refused. I don’t work for Sarepta. I don’t work for the FDA. I have no financial ties to Sarepta. No financial disclosures, no conflicts, no personal or familial stake in whether the stocks goes up or down. If you want to blame someone for whatever you’re mad about, look elsewhere.
@Biohazard3737 I thought we were debating $SRPT and the downstream impact on biotech?
@rabmanduky I was thinking the same. And if you recall, Biogen publicly reported Tysabri PML cases every month. It was a transparent process. That's not something Sarepta $SRPT has done with Elevidys-related liver toxicity, or seems willing to do based on its responses to questions. Maybe it will reverse course.
I'm just seeing this now, but Joe Schwartz at Leerink is doing hero's work today on $SRPT. https://t.co/obOQCcuv6B
UPDATED:. Sarepta Therapeutics $SRPT crisis is huge blow to Duchenne families, company Company has refused FDA request that it pull gene therapy from the market following patient deaths https://t.co/TH7rjarWHv
Let’s also note $SRPT CEO Doug Ingram insisted the company was the “most transparent” yet said nothing about any of this…
More $SRPT news: FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths Sarepta refused. https://t.co/OzGG9bEnw6
NEW: Sarepta Therapeutics $SRPT crisis is huge blow to Duchenne families, company Our attempt to put the past 20-ish insane hours into perspective. https://t.co/TH7rjarWHv via @Jasonmmast and me.
Needless to say, the last 12 hours have been stunning for $SRPT. Shocking. Frustrating. Aggravating. Maddening. The fallout will impact gene therapy and all of biotech.
We have confirmed the earlier Reuters report. The FDA is requesting $SRPT halt ALL shipments of Elevidys. This includes ambulatory Duchenne patients.
CONFIRMED: FDA to request Sarepta Therapeutics $SRPT halt shipments of Duchenne gene therapy https://t.co/TH7rjarWHv
$SRPT FDA will request Sarepta Therapeutics voluntarily stop all shipments of Elevidys - Reuters, citing a source familiar with the matter Stock halted.
Folks, note that the FDA said in June that it was investigating $SRPT Elevidys. If Makary today is referring to considering a withdrawal of Elevidys for ambulatory Duchenne patients, then that's new and significant, but I did not see him say that specifically. https://t.co/JHETJyi7p9
$SRPT CEO Doug Ingram concludes today's conference call by saying, "We are a very transparent organization."
$SRPT conference call at 10:30 am EDT
@Sanctuary_Bio That's why I find the latest attempts to divert attention away from management and on to Richard Marks a bit lame. They're all culpable $SRPT
This is from the Q&A portion of $SRPT investor call on Wednesday. Sarepta CEO Doug Ingram is asked if safety played any role in the decision to cut the Limb-Girdle gene therapy programs: h/t AlphaSense https://t.co/ZwI5YfjAjF
Risk-adjusted NPV calculation didn’t justify the costs. But remember, folks, IT'S ABOUT THE BOYS! $SRPT
Also: Asked on a Wednesday investor call if the decision was driven by any safety concerns, CEO Doug Ingram said the company had made “a risk-adjusted net present value” calculation and determined the financial opportunity didn’t justify the costs of developing the limb girdle treatments. A Sarepta $SRPT spokesperson said Thursday the company “prioritized disclosing to clinicians, regulators, and the community.” Sarepta informed the patient community about the fatal case earlier Thursday, weeks after the man’s death. It’s not clear when regulators and investigators were informed.
Important point from our $SRPT story: Although they deliver different genes, Elevidys and the company’s experimental limb girdle gene therapies rely on the same engineered virus to ferry the gene to patients’ muscles. In all three cases, patients suffered acute liver failure after treatment.
Patient dies in Sarepta $SRPT gene therapy trial, adding to safety concerns https://t.co/cCnhL8CkGb via @Jasonmmast
$SRPT More confirmation here from @Jared_Whitlock of what we heard. The patient death occurred one month ago. Let that sink in when you think back to yesterday.
This is a story my colleague @Jasonmmast was chasing as well. A third patient death from liver failure caused by $SRPT gene therapy, confirmed. The patient had limb-girdle disease. Sarepta knew about the death yesterday when it announced the restructuring and layoffs, did not disclose.
$SRPT now basically a debt vs equity story, but I guess it's always good news https://t.co/kB8tRFJ3bo
$SRPT 8K is where the promotions and salary bumps were disclosed, by the way. https://t.co/0ZCNPrKQek
$SRPT After firing 500 employees, cutting programs and decimating the stock price, all after two wheelchair-bound boys died bc of Elevidys. CEO Doug Ingram keeps his job Ian Estepan promoted to President / COO with a salary bump to $800K Louise Rodino-Klapac promoted to President, Research and Development and Technical Operations with a salary bump to $800K Ryan Wong promoted to CFO with a salary bump.
$SRPT exec team got promotion and raises.
Now official and then some... Sarepta Therapeutics $SRPT lays off one-third of workforce in drastic cost-cutting move https://t.co/Bv9otdq2b9 w/ @Jasonmmast
Sarepta $SRPT employees are dealing with a lot today. My sympathies to you all. If you want or need to vent, I can be reached on Signal at stataf.54 My DMs here are also open.
$SRPT - As @Sanctuary_Bio mentioned last night, a significant restructuring, including employee layoffs, appears to be underway. This, of course, follows the deaths of two patients treated with Elevidys. No company announcement yet, but affected employees were posting on Reddit last night. Sad day for Sarepta employees impacted by the layoffs. These business decisions, while necessary, carry a very human cost, and that's unfortunate. From an investor perspective, restructuring and cost reductions were expected given the sharp drop in its gene therapy business and inability to forecast future revenue. We'll see how the stock reacts later today, with shares now trading at a five-year low.
