Ultragenyx Reports Positive Interim Data From Phase 1/2 Study Of RhGUS In MPS 7

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Wednesday announced the presentation of positive interim data from the Phase 1/2 study of recombinant human beta-glucuronidase or rhGUS, an investigational therapy for the treatment of mucopolysaccharidosis 7 or MPS 7, Sly syndrome.

The data are being presented at the Society for the Study of Inborn Errors of Metabolism Annual Symposium in Innsbruck, Austria.

The Phase 1/2 open-label clinical study is assessing the safety, efficacy, and dose of rhGUS administered every other week via intravenous infusion in three patients.

A 12-week primary analysis phase evaluating 2 mg/kg of rhGUS every other week is being followed by dose-exploration and long-term extension.

Results from the primary analysis phase show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan excretion and the reduction in liver size.

The change in urinary glycosaminoglycan excretion was observed by two weeks after the first dose of rhGUS and declined by 40-50 percent from baseline after 12 weeks of treatment.

Decreases in liver size were observed in the two patients who had enlarged livers at baseline.

No serious adverse events were observed in the 12-week primary analysis phase and through up to 28 total weeks of treatment.

The most common adverse events reported were infections and gastrointestinal disorders.

No infusion-associated reactions were observed after 38 infusions to date in the three subjects.

Emil Kakkis, Chief Executive Officer and President of Ultragenyx, said, "Based on the reduction of lysosomal storage shown in all patients in the Phase 1/2 study, we plan to move into Phase 3 testing."