Orphazyme CEO disappointed with failed ALS trial – pins hope on other disease area

Christophe Bourdon, Orphazyme's new CEO, is candid about the group's main candidate against ALS, arimoclomol, came out of its phase-3 trial with weak results.

Photo: Orphazyme / PR

It's disappointing that Orphazyme's main treatment candidate,against amyotrophic lateral sclerosis, arimoclomol, did not meet expectations in its phase III trial, Chief Executive Christophe Bourdon tells MedWatch after the news emerged Friday that the product has not been able to fulfill neither the primary nor secondary criteria in the crucial phase III trial.

"It's a setback. We were hoping to see a benefit of arimoclomol for ALS patients, and the data is very clear that there is no efficacy signal on the primary and secondary endpoints," says Bourdon, who emphasizes that no safety-related issues were reported in the study.

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