Raptor's RP103 fails in mid-stage liver disease trial

14 September 2015

Shares of US biotech firm Raptor Pharmaceutical (Nasdaq: RPTP) tanked 38.7% in late morning trading, after the company released top-line results from the Phase IIb CyNCh study, which did not meet its primary endpoint of improving non-alcoholic steatohepatitis (NASH) in children.

The trial evaluated the safety and efficacy of RP103, or cysteamine bitartrate delayed-release capsules, in children with biopsy-confirmed NASH. The study did not achieve its primary endpoint defined as a two-point decrease in NAFLD Activity Score (NAS) and no worsening of fibrosis (p-value = 0.34). There were no differences in adverse events observed in children on RP103 compared to placebo.

Will likely not advance the program

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK