Ultragenyx Pharmaceutical Inc (NASDAQ;RARE) is rated 'outperform' by broker Wedbush, which has a target price of US$64, after it released clinical data.
That's a fair chalk from the current price of US$52.95 each.
Ultragenyx is developing for market novel products for the treatment of rare and ultra-rare diseases and today announced positive longer-term safety and efficacy data from the first dose cohort of a phase 1/2 study of DTX301.
The gene therapy is for the treatment of ornithine transcarbamylase (OTC) deficiency - a urea cycle disorder, which in severe cases, can cause death.
Wedbush said today's update showed the benefit in first patient was durable, supporting discontinuation of alternate medicines. It added that there was no change in the status of other two patients treated with low-dose DTX301 and that enrolment in mid-dose cohort was set to begin.
Analyst David Nierengarten added that he believed RARE’s gene therapy programmes were an overlooked long-term value driver.
"In addition to DTX301, we expect progress this year with other key gene therapy assets gained from Dimension, including DTX401 for glycogen storage disease type 1a (GSD1a) and DTX201 for hemophilia A," he noted.
US approval for Crysvita, for X-linked hypophosphataemia, is expected on time next month, with a slow rollout expected but long-term commercial potential not priced into the stock, he added.