$RGNX Duchenne gene therapy study results are out: microdystrophin expression reached an average of 71% of normal across all 31 boys at 12 wks. Functional improvements in a subset of boys followed for one year also seen.
The big question remains: Will the FDA still follow the $SRPT precedent and grant accelerated approval to the Regenxbio gene therapy?
CEO Curran Simpson told me the company is holding off on submitting to the FDA until early next year.
“I think our data checks every single box that you would want for accelerated approval,” he told me. "We’re not in a rush to go to the FDA at this point. We want to give time for the leadership changes to occur. But that’s OK, because we can still meet our timeline of securing an approval in 2027."
More details in our story, written with @Jasonmmast in the following post.
@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Amondys and Vyondys should be withdrawn from the market.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.
$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.
Duchenne gene therapy stocks today:
$SLDB +22%
$RGNX +14%
$SRPT +1%
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?
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.”
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
$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
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.
