Early-stage US clinical holds spread

It might be going too far to suggest that the recent spate of clinical holds is evidence of the FDA suddenly getting tough, but the fact that all of these concern very early-stage projects should give pause. The agency today put on hold a phase 1 X-linked adrenoleukodystrophy trial of Viking’s VK0214, after stymying no fewer than three IND applications, from Denali, Dyne and Yumanity, over the previous 10 days. And neither do these holds affect cutting-edge gene therapies, which have faced their own toxicity questions; the four assets in question comprise two small molecules, an antibody and a morpholino oligomer (two of these are using a TfR1-based transporter). At least Viking’s setback appears to be the result of a technicality; the FDA recently designated VL0214’s phase 1 trial a phase 2, something that requires a rodent genotoxicity study to be carried out first, Viking says. And it has not been all bad news of late: the FDA this month lifted two clinical holds, one on Spero’s nontuberculous mycobacterial disease project SPR720 and the other on Allogene’s entire pipeline. The Spero hold had been in place for 11 months, but Allogene’s took just three months to resolve.

Selected US clinical holds
Hold issued Hold announced Company Project Status/detail Company timeline
ND 24 Jan Viking VK0214 Ph1 (now ph2) X-linked adrenoleukodystrophy trial on hold; rodent genotoxicity trial needed Expects to fulfil request Q2 2022
ND 19 Jan Yumanity YTX-7739 Preclinical (IND application on hold for multiple dosing in Parkinson's disease) None (no official hold letter yet)
14 Jan 18 Jan Dyne DYNE-251 Preclinical (IND application on hold for exon 51 skipper in Duchenne muscular dystrophy; more preclinical & clinical data requested) Expects to fulfil request Q2 2022
12 Jan 13 Jan Denali DNL919 Preclinical (IND application on hold for anti-Trem 2 MAb in Alzheimer's) None (no official hold letter yet)
Source: company announcements. ND=not disclosed.

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