Could Regenxbio Shares Have 320% Upside?

Shares of Regenxbio Inc RGNX could have 320 percent upside, according to Piper Jaffray, which has a price target of $33 on the stock.

The $33 target price represents a potential upside of 320 percent from Wednesday's close of $7.86.

"Regenxbio is a leader in the AAV gene therapy world and holds an extensive intellectual property portfolio of next-generation AAV vectors that forms the basis for the company's partnerships and internal programs," analyst Joshua Schimmer explained.

Drug Updates

The company recently provided an update on its gene therapy pipeline, mentioning "a modification to the RGNX-111 inclusion criteria to facilitate enrollment and updated timelines for IND filings for its other pre-clinical stage programs."

Related Link: Biogen And Regenexbio To Collaborate With University Of Pennsylvania On Gene Therapy Programs

"Recent successes in the field, most notably exciting clinical updates by NAV vector licensee AVXS's for SMA, highlight RGNX' central position in this rapidly evolving field," Schimmer elaborated.

The Phase 1 study of RGX-501 for HoFH opened in the first half of 2016 and Regenxbio expects the first patient enrollment to happen in the second half of 2016. Homozygous Familial Hypercholesterolemia, or HoFH, is a rare genetic lipid disorder caused by defects in the low-density lipoprotein, or LDL, receptor genes. Untreated, HoFH often causes heart diseases in early teens.

Meanwhile, an IND for RGX-111 gene therapy for MPS I is now expected to be filed in the first half of 2017 versus prior mid-2016. "This was shifted in response to findings from a dog model which showed ability to have a fuller impact on the disease at higher doses," Schimmer explained.

Mucopolysaccharidosis type I (MPS I), which is also been called Hurler, Hurler-Scheie and Scheie Syndrome, is a condition that affects many parts of the body.

"Nonhuman primate studies have now begun to show similar benefit at higher doses, and the company wants the starting human dose to have a greater chance of delivering a full treatment effect," said Schimmer.

Schimmer reiterated his Overweight rating on the stock, which closed Wednesday's regular trading at $7.86.