- Ipsen, a Paris-based pharmaceutical company, has paused two studies of a drug it acquired for $1 billion. The decision stems from an analysis that, according to independent reviewers, suggests an ongoing late-stage trial is unlikely to succeed.
- Researchers are testing the drug in patients with an ultra-rare disorder that causes bone to grow in muscle tissue. Called FOP for short, the disorder disables patients over time and can increase risk of death, with median life expectancy commonly cited at 40 years.
- While dosing has paused, Ipsen hasn't discontinued its Phase 2 and Phase 3 studies because post-hoc analyses of the latter showed "signals of encouraging therapeutic activity." The independent committee acknowledged these findings, according to Ipsen, and recommended against stopping the trials outright. A partial clinical hold issued in December for patients in these trials under 14 years of age is still in effect.
Ipsen's trial pauses come two weeks after Regeneron posted data on its own experimental treatment for FOP, or fibrodysplasia ossificans progressiva.
That mid-stage study showed patients treated with Regeneron's drug had 90% fewer new lesions than patients given placebo. Investigators also found a 25% relative decrease in the number of bone lesions, both new and existing, while disease flare-ups were seen in 10% of garetosmab-treated patients versus 42% of placebo-treated patients.
Regeneron's data are the culmination of more than two decades of work that could land it the first-ever approved treatment for FOP. The big biotech plans on discussing an approval filing with regulators as well as initiating a study of its drug in children.
While Regeneron's drug is an antibody, Ipsen's is a small molecule thought to regulate a protein involved in skeletal growth.
Ipsen acquired the drug, known as palovarotene, through a $1 billion acquisition of Clementia Pharmaceuticals last year. At the time, Ipsen said palovarotene was on track to be filed for approval in the second half of 2019 as a treatment for episodic flare-up in FOP patients, with a corresponding launch expected in mid-2020.
That timeline is now unlikely. In December, the Food and Drug Administration issued a partial clinical hold for all patients under 14 participating in studies palovarotene. The hold included both FOP patients as well as those multiple osteochondromas, another uncommon condition for which Ipsen is testing its drug. Ipsen noted the hold came after safety reports detailed early growth plate closure in pediatric FOP patients treated with palovarotene.
Ipsen said that, with dosing paused, it will further analyze data and talk with regulators about the findings and next steps.
The company also said that the independent reviewers highlighted how "the protocol-prespecified model may have negatively affected the efficacy analysis and shifted the statistical conclusion from significant therapeutic benefit to showing futility of the treatment."
Shares of Ipsen, which trades on the over-the-counter stock market, were down nearly 25% Friday morning to trade at $17.43 apiece.