Janssen Discontinues Late Stage Development of Treatment for CTEPH

Rare Daily Staff

The Janssen Pharmaceutical Companies of Johnson & Johnson said it decided to stop the phase 3 study evaluating a high dose of its pulmonary hypertension drug macitentan in patients with chronic thromboembolic pulmonary hypertension due to futility.

Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare condition where there is elevated blood pressure in the pulmonary arteries caused by chronic blood clots, which obstruct the free flow of blood through the lungs.

Macitentan, dosed at 10 mg daily, was approved in 2013 under the brand name Opsumit for treating pulmonary arterial hypertension. Janssen was testing it at a 75 mg dose in the phase 3 MACiTEPH study, a prospective, multi-center, double-blind, randomized, placebo-controlled, parallel-group in patients with CTEPH to evaluate the efficacy and safety of macitentan 75 mg compared to placebo on exercise capacity, the primary efficacy endpoint, in a broad population including inoperable patients and those who underwent mechanical treatment, including pulmonary endarterectomy or balloon pulmonary angioplasty.

The decision to stop the trial was made in accordance with a recommendation by the study’s independent data monitoring committee following a pre-planned interim analysis. No new safety signals were observed.

The interim results of the study do not impact any of the company’s commercialized pulmonary hypertension medicines. Additionally, the pivotal phase 3 UNISUS study, which aims to establish the superiority of a 75 mg dose of macitentan over the currently available 10 mg dose in patients with pulmonary arterial hypertension, is continuing as planned.