Palovarotene is a retinoic acid receptor γ (RARγ) agonist intended to address rare and debilitating bone diseases, such as FOP and multiple osteochondromas (MO).
Ipsen gained the drug during the acquisition of Clementia Pharmaceuticals in April last year.
The Phase II PVO-1A-202/204 extension and Phase III PVO-1A-301 studies involved chronic and episodic dosing of the drug. In these studies, dosing was paused based on interim analysis data.
A futility analysis by the Independent Data Monitoring Committee (IDMC) found that the Phase III trial may not achieve the primary efficacy endpoint of annualised change in new HO volume.
However, preliminary post-hoc analyses of the Phase III trial indicated encouraging therapeutic activity and this data was shared with the IDMC. As a result, IDMC recommended the study continue.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below formBy GlobalData
The committee noted the possibility of a negative effect of the protocol-pre-specified model on the efficacy analysis.
The company will pause dosing in the trials and further analyse the complete data set. It will discuss the findings with regulators to decide the path forward for the palovarotene FOP programme.
In addition, the company is analysing the financial effects of these developments.
Ipsen CEO Aymeric Le Chatelier said: “While the study has met prespecified statistical futility, we are encouraged by the results observed in the preliminary post-hoc analyses and look forward to discussing these with regulators as quickly as possible to determine the next steps for the palovarotene programme.”
The partial clinical hold placed last month on paediatric patients at all global sites of the Phase II PVO-1A-202/204, PVO-2A-201, and Phase III PVO-1A-301 trials assessing palovarotene is still in effect.