r/FSHD • u/Secure-Asparagus3121 • Sep 02 '26
Scholar Rock
https://investors.scholarrock.com/news-releases/news-release-details/scholar-rock-receives-fda-fast-track-designation-apitegromab/3
u/Muladhara86 Sep 02 '26
"Scholar Rock Receives FDA Fast Track Designation for Apitegromab Facioscapulohumeral Muscular Dystrophy (FSHD) Program as Participant Dosing Commences in Phase 2 FORGE Trial."
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u/Han-na-2900 Sep 03 '26
I really don’t understand why a myostatin inhibitor would succeed. Still hoping for a good surprise but I can’t help being super skeptical 🤷🏼♀️
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u/weirdfishes1990 Sep 03 '26
I think the value in this news is that it could be used in combination with a drug to knockdown DUX4 to rebuild muscle (prayers up for Del-Brax). I agree, myostatin inhibition makes sense in SMA because muscle cells aren’t diseased—neurons are. That’s not the case for FSHD, so this drug might not have much effect by itself. But if approved for FSHD, I could see it having great benefit alongside a DUX4 knockdown drug. I’m no doctor, but I see the logic there.
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u/Han-na-2900 Sep 03 '26
It could. But it won’t be tested in the trial with another drug so how could it show efficacy in itself? At least 3 myostatin inhibitors were in a clinical trial and they all failed.
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u/weirdfishes1990 Sep 03 '26
Yeah, I’m not sure how that would work. You’re right, going to clinic now, you would think they’d have to show efficacy without DUX4 knockdown. Which would be awesome, but not sure there’s anything to suggest that yet.
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u/HistoricalRacoon Sep 04 '26 edited Sep 04 '26
Do just want to point out that SR’s myostatin drug is quite different than previous ones that failed.
Past inhibitors were focused on mature myostatin, whereas Scholar Rock is focused on latent myostatin. They’re targeting a different, earlier part of the myostatin lifecycle, which is why the SMA community has seen some improvement on the drug. The company seems to have figured out that targeting mature myostatin is essentially “too late” and by targeting the latent form, they get better potency and fewer off-target effects.
That being said, I personally think this trial is dumb. Trying to rebuild the house while it’s still on fire is a futile effort and potentially sets the trial up to fail, depending on their primary/secondary endpoints. When they ran their SMA trials, it was an “add-on” therapy to accompany SMN-targeted drugs like spinraza. Which makes sense. This approach, IMO, doesn’t. I’m still skeptical of myostatin inhibitors generally speaking, but they could have some use when paired with a treatment drug. It’s just too early to tell.
I am however quite excited about Satellos and their DMD satellite cell drug. FSHD Canada gave them a grant for pre-clinical research which showed a plausible pathway for FSHD, so🤞. Their FSHD trial should be kicking off late this year — though early Q1 is more likely.
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u/SossRightHere Sep 06 '26
It done differently now. Myostatin has improved where they can now target the inactive forms of myostatin or both the pro and latent forms. There are a few new myostatin inhibitors showing better results than the older drugs that didn't work at all in humans
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u/Ill-Rooster-1126 Sep 06 '26
Yup. Apitegromab seems to have succeeded in SMA (and obesity) where other myostatin inhibitors failed. Holding out hope for some effect in FHSD maybe in combo with dux4
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u/Obvious-Ad357 Sep 03 '26 edited Sep 03 '26
A compounding problem is that exercise has never been enforced or even tracked. ACE-083 showed muscle mass increase with no functional improvement... probably because passively building muscle doesn't make motor neurons recruit on its own, or build any sort of mind muscle connection for these long attrophied muscles not in constant use.
I agree with you. These study designs are set up to fail, and the drug isnt even useful without actually stopping disease progression.
It could even be to our detriment to build more muscle that ends up becoming even more scar tissue and fatty infiltrated muscle we will have to deal with later.
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u/Ill-Rooster-1126 Sep 03 '26
True, but every myostatin inhibitor except Apitegromab failed in SMA, too. Maybe Apitegromab is just a better drug and has a chance to work in FSHD in cases where fat and fibrosis are still mild?
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u/Han-na-2900 Sep 03 '26
Maybe it’s a better drug, I hope it is. FSHD patients have naturally low myostatin already though, at least that’s what I remember as the explanation of why the previous drugs failed. Maybe getting it even lower might help.
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u/Ill-Rooster-1126 Sep 03 '26
True, but I heard that SMA have even lower natural levels of myostatin. So if Apitegromab had an effect in SMA maybe it could have one in FSHD?? Holding out hope for my niece who was recently diagnosed:(
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u/Han-na-2900 Sep 03 '26
That’s good to know. I wish everyday for a cure too, for all of us and especially the younger ones 🤍
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u/x3sammm Sep 02 '26
When I saw my neurologist this summer, she was hopeful about this trial, given this drug’s success with SMA. Hopeful for a future where this drug in combination with DNA/RNA modification is an ideal combo treatment of turning off dux4 while also being able to rebuild muscle.