Leerink Raises Ultragenyx Pharma's Price Target On Confidence In MPS 7 Treatment

Leerink analysts Joseph Schwartz and Dae Gon Ha are confident that Ultragenyx Pharmaceutical Inc RARE will gain from MPS 7 treatment ahead of pivotal data. The two analysts boosted their price target to $85 from $80 while rating the stock at Outperform. The brokerage listed four main reasons to be more optimistic about the pivotal data.

"We are raising our probability of success (POS) and peak probability weighted (p(w)) sales estimates to 80 percent and $80 million from 60 percent and $60 million respectively. Although the market opportunity for rhGUS is on the low end, the opportunity should be durable and positive rhGUS data would be a significant indicator that the co. is on the cusp of becoming a commercial organization," said the analysts in a research note to clients.

Leerink thinks Ultragenyx Pharmaceutical Inc "also expects CHMP opinion for UX001 in GNE myopathy" in the second half of the current year, which the brokerage continues to handicap at 60 percent POS. The two analysts outlined an extensive natural history study published earlier this year, which cataloged records from 56 patients in 11 countries and suggested patient identification is likely to be less challenging than it assumed earlier.

Furthermore, the brokerage believes Ultragenyx Pharmaceutical Inc is adept at exceeding the bushes and could gain from the efforts of companies that have fostered a global MPS disorder "net" for MPS7 patients.

Leerink concluded, "We believe rhGUS has an opportunity to enhance several features: pulmonary function, through shrinking of visceral organs such as liver and spleen, which restrict the diaphragm and thus ventilation."