Endocrine Abstracts

Objectives: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder caused by activin receptor-like kinase-2/activin A receptor type 1 (ALK2/ACVR1) mutation and characterized by heterotopic ossification (HO) inducing progressive restriction of mobility. IPN60130 is a selective ALK2/ACVR1 inhibitor being investigated for the treatment of FOP.¹ Here, we describe methodology of the FALKON trial (NCT05039515) designed to compare efficacy and safety of IPN60130 with placebo in patients with FOP.

Methods: Patients will be randomized to oral placebo, or low or high dose IPN60130 for 12 months; patients receiving placebo will then transition to IPN60130 for 12 months. Enrollment criteria include: ≧5 years old, clinical FOP diagnosis with disease-causing mutation, and either a flare-up, new HO or joint ankylosis, or increase in Cumulative Analogue Joint Involvement Scale (CAJIS) score in the prior year. Recruitment is ongoing to enroll 90 patients. The primary efficacy outcome will be annualized change from Baseline in HO volume to Month 12, assessed by low-dose whole-body computed tomography (CT). Secondary efficacy outcomes are presented in the Table. Safety will be assessed via adverse event (AE) and serious AE incidence over 25 months. Patients aged ≧15 years will be eligible for a sub-study assessing HO by [¹⁸F]NaF positron emission tomography-CTT.

Summary: Results from FALKON, estimated to complete in August 2025, will allow evaluation of the efficacy and safety of IPN60130 in patients with FOP.

References: 1. Davis A et al. J Bone Miner Res 2019;34(Suppl 1):290

Funding: Sponsored by Ipsen