- An investigational formulation of Shire plc's Elaprase has failed a Phase 2/3 trial testing it in children with a rare genetic disorder.
- Elaprase is an intravenous enzyme replacement therapy that the Food and Drug Administration first approved in 2006 to treat patients with Hunter syndrome, a disorder that harms physical and mental development. Shire's new formulation, known as SHP609 or HGT-2310, is largely the same, only it's administered directly into a patient's cerebrospinal fluid through an intrathecal drug delivery device.
- After a year of monthly treatments, participants in the trial's experimental arm didn't show significant improvement in cognition compared to control groups, according to topline results released Tuesday. SHP609 also didn't meet the trial's key secondary endpoint.
Elaprase (idursulfase) is one of Shire's most profitable neuroscience products, falling just behind its hereditary angioedema drug Firazyr (icatibant injection). Between January and the end of September, Elaprase raked in $455 million, up more than 7% from the same period a year prior.
It's unclear, though, how much longer Elaprase will contribute significantly to Shire's bottom line. The Irish pharma, now hyper-focused on hedging its bets in the rare disease market, announced earlier this year that it had begun a strategic review of its neuroscience business. Nothing official has come from the review so far, but Shire has noted a potential public listing is one route under consideration.
If Shire ends up holding onto the business, SHP609 could come in handy.
The company says its new formulation is meant to be used with Elaprase for the treatment for Hunter syndrome. SHP609 entered Phase 2/3 testing in late 2013, and about a year later the FDA granted it Fast Track Designation. The formulation also holds an Orphan Drug Designation in the U.S.
The latest data are undoubtedly a setback for SHP609, stalling its path toward regulatory filing. Shire plans to analyze the results further and present additional findings and an upcoming congress, according to a Howard Mayer, the company's global head of R&D.