Jefferies Highlights Clinically Meaningful Data From Ultragenyx On KRN23

Following the presentation of KRN23 data by Ultragenyx Pharmaceutical Inc RARE Monday, Jefferies noted the longer follow-up data is clinically meaningful, supporting filing for a EU conditional approval by 2016.

Elaborating on the data, equity analyst Gena Wang noted that data from the Phase 2 40-week and 64-week trial showed the magnitude of improvement in rickets and growth velocity from bi-weekly dosing were largely in line with her expectations.

According to the analyst, continued improvement in all patients and magnitude of improvement in rickets at ~2 (in RGI-C) in severe patients is clinically meaningful. Jefferies also noted that positive trend in growth at 64 weeks supports clinical benefit for KRN23. The serum phosphate levels achieved slightly above lower-limited norm and additional functional measurements also point to the right direction, according to the firm.

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Wang also pointed out that safety and tolerability profile gives it an edge over the current standard of care. The analyst also noted that incremental update from adult XLH Phase 2b extension study suggested improvement in serum Pi and that pain scores showed acceptable safety profile.

Although the company sees possibility for an accelerated approval, given breakthrough designation and updated data, it indicated that it would initiate a Phase 3 trial in 2016, as the FDA would need a confirmatory trial.

Jefferies has a Buy rating and $109 price target on the shares of the company.

At time of writing, Ultragenyx was down 1.61 percent on Tuesday, trading at $75.35.

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