Has everyone here heard about the ALD drug PXL 770? It is undergoing phase II trials and will soon be available for use.poxelpharma.com/en_us/news-...
PXL 770: Has everyone here heard about the ALD... - AMN EASIER
PXL 770


It looks like a superb contender.
pmc.ncbi.nlm.nih.gov/articl...
Though, plenty of substances work in mice, but fail to repeat their efficacy in humans.
Still, I like the look of this drug.
That press release is from October 2022. The clinical trial (clinicaltrials.gov/study/NC... ) has been posted for years but is still not yet recruiting. And based on the stock history of POXEL (live.euronext.com/en/produc... ), I would be wary of starting a trial with them, with the hope that they stick around.
As of December 2024, the US PXL770 adrenomyeloneuropathy trial is withdrawn due to lack of funding (clinicaltrials.gov/study/NC... ):
> Withdrawn
> Lack of funding to initiate the study
The US PXL065 AMN trial was also withdrawn in December 2024 for the same reason (clinicaltrials.gov/study/NC... ):
> Withdrawn
> Funding unavailable
The EU PXL770 trial's recruitment status is still "Not Recruiting", the same as it was before (trialsearch.who.int/Trial2.... ).
In Poxel's February 2025 press release, they still mention PXL770 in their company description (poxelpharma.com/en_us/inves... ):
> In rare diseases, development of PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, is focused on the treatment of adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD).
Maybe Poxel will find more funding for a US PXL770 AMN clinical trial.