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Valerion’s trial of therapy for Pompe stopped.

A trial by Valerion Therapeutics for a potential new treatment for GSD2, was halted last week. VAL-1221 was delivered intravenously in ambulatory and ventilator-free participants with late-onset Pompe disease.

Trial at NHNN, London

Initially four people in the UK started the trial at the Charles Dent Metabolic Unit at the National Hospital for Neurology and Neurosurgery, one had to leave half way through, so there were three remaining.

Stopped on 24 January

On Friday 24 January they were informed the trial was being stopped.

It is a blow for everyone involved. In 2016, as some people will remember, the same happened with the BioMarin 701 trial for a potential new enzyme treatment for Pompe disease.

Trial participants invest a huge amount of passion and commitment when joining, not least because of the effort made in travelling to the trial centre fortnightly for years. It also has an impact on medical teams who, likewise, make a huge commitment and join patients in their hopes for a new and effective therapy.

An update will be provided once more is known about the reasons for the closure of the trial.

Further reading

VAL-1221 trial information on

Challenges in treating Pompe disease: an industry perspective. July 2019 by Do, Khanna & Gotschall.