“The limited benefit seen with UX007 (triheptanoin) in a Ph 2 Glut1 DS (glucose transporter type-1 deficiency syndrome) seizure study increases the risk for the Ph 3 in patients with a different disease phenotype,” analysts at Wedbush said in a note.
The analyst reiterated an Outperform rating on Ultragenyx Pharmaceutical Inc RARE, while lowering the price target from $88 to $80.
However, the analysts continue to recommend Ultragenyx Pharmaceutical shares ahead of the XLH Ph 3 data, expected later in the first half, “as we have confidence of a positive readout that should support a BLA (biologics license application) filing in 2H17.”
Primary Endpoint Failed
The primary endpoint of the study, a reduction in the frequency of motor and non-motor seizures during the treatment period, failed to meet statistical significance, with only 13.4 percent patients on UX007 showing a decrease in overall seizure frequency.
On the other hand, patients with non-motor seizures saw stronger benefit, with 43.7 percent decrease in frequency of seizures.
Secondary Endpoint Failed
The study also failed to meet a key secondary endpoint of cognitive function, as compared to placebo, while Ultragenyx Pharmaceutical did not provide results regarding other key secondary endpoints, such as walking capacity and time to onset of paroxysmal exertional dyskinesia.
In addition, a higher rate of treatment-related adverse effects (AEs) were observed with UX007, as compared to placebo, with several patients discontinuing treatment due to the AEs.
Phase 3
While no Phase 3 was planned for UX007 in this phenotype, the company recently initiated at Phase 3 for the drug candidate in Glut1 DS patients with movement disorders. Data from the study are expected in 2018.
“We are adjusting our model to account for increased risk and lower our penetration and compliance estimates for UX007 program,” the analysts added.
At last check, shares of Ultragenyx were down 7.84 percent at $72.22.
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