Ultragenyx's genetic disease treatment front-runner among rivals - Truist

Metalert

24/12/2020 14:14

** Brokerage Truist raises PT of drug developer  RARE.O  to
$185 from $110 on prospects of its treatment for a rare,
neurogenetic disorder 
    ** Says GTX-102, co's treatment for Angelman syndrome, is
now the front-runner in the race and well ahead of competition 
    ** Data reported to date show "impressive efficacy" of
therapy - brokerage
    ** Says while further dosing in early-stage trials currently
on hold due to safety events at the higher doses, co has
proposed capping the dose at 14mg and gradual dose escalation 
    ** Brokerage expects an update as early as Q1 2021, after
FDA meeting
    ** Says inclusion of GTX-102 in valuation timely, given 
void created by failure of rival Ovid Therapeutics  OVID.O  
candidate in a late-stage study for Angelman syndrome
    ** People with Angelman syndrome have developmental delay,
balance issues and debilitating seizures, with some of them
unable to walk and most unable to speak
    ** U.S. FDA has earlier granted GTX-102 orphan drug, rare
pediatric disease and fast track tags 
    ** Median PT $110; stock up ~315% YTD, as of last close

 (Reporting by Vishwadha Chander in Bengaluru)
 ((Vishwadha.Chander@thomsonreuters.com; within U.S. +1 646 223
8780, outside U.S. +91 80 6749 6132;))