SS-31 (brand name, Forzinity) was just granted US FDA accelerated approval for the rare mitochondrial disease, Barth Syndrome. Physicians have been prescribing SS-31 off-label for anti-aging and enhanced athletic performance in the US for some time. The implications of FDA-approval for compounding pharmacies and biohackers are unclear.
It does seem surprising to see a phase 3 with such a small n, but Barth is very rare. The subject ages seem to be mid-to-late tens through early adults. Quite a lot of people with Barth die in infancy and more don't make it to adulthood. Looks like there are only 100-200 adults w/ Barth in the US. So that explains why the study n was so small.
Nice job reviewing some of the other trials in which the therapy failed, but I think you are being too one-sided: You could also have pointed out some of the other ongoing trials in which it's still being evaluated for other indications, at least one of which is in phase 3 so presumably had positive ph2 data. That's for dryAMD (ReNew trial). And yes, I see the positive ph2 trial record there too.
For mito myopathy it looks like the story isn't as simple as just failure: It looks like the failed ph3 MMPOWER-3 trial, rather than leading to conceding defeat instead lead to another phase 3 for mito myopathy, the NuPower trial (NCT05162768) for which I don't see results yet. (And this is after presumably positive ph2 results.)
Also, looks like the heart failure trial failure you mention was a phase 2, so you could presumably also try to comprehensively review other positive phase 2s it had. I haven't done that so not sure if there are any for conditions not yet mentioned here.
Good points! I didn't thoroughly review the ongoing trials and these represent more shots on goal for SS-31. I suspect that this approval is just the beginning of the story and am hopeful that we will see it move across indications with approvals.
It does seem surprising to see a phase 3 with such a small n, but Barth is very rare. The subject ages seem to be mid-to-late tens through early adults. Quite a lot of people with Barth die in infancy and more don't make it to adulthood. Looks like there are only 100-200 adults w/ Barth in the US. So that explains why the study n was so small.
Nice job reviewing some of the other trials in which the therapy failed, but I think you are being too one-sided: You could also have pointed out some of the other ongoing trials in which it's still being evaluated for other indications, at least one of which is in phase 3 so presumably had positive ph2 data. That's for dryAMD (ReNew trial). And yes, I see the positive ph2 trial record there too.
For mito myopathy it looks like the story isn't as simple as just failure: It looks like the failed ph3 MMPOWER-3 trial, rather than leading to conceding defeat instead lead to another phase 3 for mito myopathy, the NuPower trial (NCT05162768) for which I don't see results yet. (And this is after presumably positive ph2 results.)
Also, looks like the heart failure trial failure you mention was a phase 2, so you could presumably also try to comprehensively review other positive phase 2s it had. I haven't done that so not sure if there are any for conditions not yet mentioned here.
Good points! I didn't thoroughly review the ongoing trials and these represent more shots on goal for SS-31. I suspect that this approval is just the beginning of the story and am hopeful that we will see it move across indications with approvals.
Great summary. Thank you!!