October 2, 2015 7:00am

RGNX closed DOWN -$3.02 and has a mixed performance in its 17 days of public status. RGNX priced (9/16/15) 6.3 M shares <above previous plans to offer 5.6 M shares> at $22.00 a share <was at $17 to $19.  The shares had popped to $30.45 but, has steadily declined during the recent depreciating period. Headwinds are questionable! BUY


 

The U.S. FDA has granted Orphan Drug Designation to REGENXBIO's investigational gene therapy product candidate RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I).

 

The Bottom Line: RGNX intends to file an IND with the FDA in the first half of 2016 to support the initiation of a dose-escalation P1/2 clinical trial of RGX-111.

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene. Over 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. RGX-111 uses an AAV9 vector to deliver the IDUA gene to the central nervous system.