At the 14th International Symposium on MPS and Related Diseases, Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) presented positive pivotal Phase 3 trial of recombinant human beta-glucuronidase (rhGUS) in replacement therapy in development for Mucopolysaccharidosis 7 (MPS 7).

In reaction, Cowen analyst Eric Schmidt reiterated an Outperform rating on the stock, without providing a price target.

Schmidt commented, “Despite MPS7 being a progressive disease and a trial size of just 12 patients, rhGUS nearly provided a statistically significant improvement at 24-weeks relative to baseline in patient function as measured by the MDRI. We find this to be convincing evidence that rhGUS has met the admittedly subjective FDA hurdle of altering the expected disease progression. Therefore, we think today’s data is likely supportive of FDA approval (although there may be a bit more risk to FDA than EMA approval). Moreover, longer-term followup data on enzyme replacement therapies often shows continued improvement on clinical endpoints. If this is the case for the 48+ week rhGUS data, it would further bolster confidence in U.S. approval. On the safety/tolerability front, as with all enzyme replacement therapies rhGUS is not a trivial drug to administer. However, its AE profile appears consistent with if not superior to other enzyme replacement therapies for LSDs.”

According to, which measures analysts’ and bloggers’ success rate based on how their calls perform, analyst Eric Schmidt has a yearly average return of 13.6% and a 47% success rate. Schmidt has a 40.7% average return when recommending RARE, and is ranked #201 out of 4060 analysts.

Out of the 13 analysts polled by TipRanks, 11 rate Ultragenyx Pharmaceutical stock a Buy, 1 rates the stock a Hold and 1 recommends Sell. With a return potential of 82.3%, the stock’s consensus target price stands at $95.50.