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Palovarotene for the Potential Treatment of MO

The goal of our clinical development program in MO is to obtain regulatory approval for the use of palovarotene for the treatment of MO in medical needs worldwide. There are currently no approved therapies for MO. Surgical excision is currently the only treatment available for symptomatic OCs and can be associated with serious complications. Following proof-of-concept animal studies, and as a result of our extensive clinical experience with palovarotene, we are planning to initiate a Phase 2/3 clinical trial in MO.

Planned Phase 2/3 Trial

We intend to use the learnings from our development program in FOP to design the most efficient path to approval for MO. Also, because of our extensive studies (including juvenile toxicity studies) and experience with oral palovarotene in the clinic, we do not believe we will need to conduct any additional pre-clinical or clinical studies prior to initiating our clinical trials in MO.

The objective of our planned Phase 2/3 study will be to evaluate the efficacy and safety of palovarotene in MO in patients with confirmed Ext1 or Ext2 mutations. A protocol for a multi-center, randomized placebo-controlled study is being finalized. We hope to commence this study in 2017 and report data in 2020, with a potential interim read-out in 2019.



Efficacy of Palovarotene Oral Treatment on Prevention of Osteochondroma Formation in the Fsp1-Ext1CKO Mouse Model of Multiple Osteochondromas [Oral Presentation at 2017 American Society for Bone and Mineral Research (ASBMR) Annual Meeting]

Efficacy of palovarotene on prevention of osteochondroma formation in the Fsp1-Ext1 conditional knockout mouse model of Multiple Osteochondromas (MO) [Poster presentation at the 13th meeting of ISDS, 2017]