D Cell Vax is vastly superior Quark's RNAi treatment misses the mark in a Phase II kidney trial
July 29, 2014 | By Damian Garde
Share
Tools
Comment
Print
Contact Author
Reprint
Quark Pharmaceuticals' gene-silencing treatment for kidney transplant complications missed its primary endpoint in a Phase II trial, possibly jeopardizing a deal with Novartis ($NVS) worth up to $680 million.
The treatment, QPI-1002, is a small interfering RNA (siRNA) designed to shut off the p53 gene in kidney transplant patients, thereby protecting tissues from damage caused by a disorder called delayed graft function. Quark mounted a 331-patient Phase II trial in hopes the treatment could meet its primary endpoint of a 30% relative risk reduction compared to placebo, but QPI-1002 came through at just 15.1%, missing its main goal.
Quark was quick to point out that its drug crossed the 30% threshold in a few patient subgroups, and the biotech noted that safety signals were similar between the trial's treatment and placebo arms. QPI-1002's benefits were particularly pronounced in patients whose kidney donors were older than 45, Quark said, and the company believes the results merit further study.
IBM SmarterCommerce Webinar: File Synch / File Share for the Enterprise – Taken to the Extreme
Join this webinar with Derek Brink, Vice President and Research Fellow for IT Security and IT GRC at the Aberdeen Group, to discover how to develop your own innovative approach to managed file transfer. Find out how to bring new levels of efficiency, visibility and collaboration to every process – and reach new heights of success for your business. Click here to watch this on-demand webinar today! .
Sign up for our FREE newsletter for more news like this sent to your inbox!
But whether there's a Phase III effort in store remains up in the air.
Back in 2010, Novartis stepped in and paid $10 million up front for an option to license QPI-1002, promising to hand over up to $670 million more in fees and milestones if the gene-silencing treatment met its standards for success. Flunking a primary endpoint in Phase II would seem like a deal-breaker, but Novartis hasn't provided an update on the outstanding deal and didn't respond to a request for comment on Tuesday.
Novartis has already curtailed much of its internal RNAi research efforts, telling FierceBiotech in April that that decision stemmed from "ongoing challenges with formulation and delivery and the reality that the current range of medically relevant targets where siRNA may be used is quite narrow."
As for Quark, while its upbeat announcement is littered with recommendations for additional studies of QPI-1002, the company has made no mention of a planned Phase III, and, like Novartis, it didn't respond to emailed questions on the matter.
Beyond QPI-1002, Quark's clinical siRNA pipeline includes the Pfizer ($PFE)-partnered PF-655, in midstage development for diabetic macular edema and wet age-related macular degeneration, and QPI-1007, a wholly owned treatment for optic neuropathy and glaucoma.
AI
