Actelion initiates Phase III study to evaluate macitentan (Opsumit) in children with PAH - Seite 2

Dunbar Ivy, Director of the Pediatric Pulmonary Hypertension Program at Children's Hospital Colorado, commented: "As there is no globally approved treatment for PAH in children, it was not possible to define a single unique treatment as reference drug or standard background. The design of the TOMORROW study will ensure that children receive the best available standard of care therapy or macitentan, a treatment that is well supported by long-term efficacy and safety data in adults. This will render the study ethical, informative, and feasible and I welcome Actelion's effort to broaden the pediatric knowledge base, which will help both physicians and their young PAH patients in the long term."

Jean-Paul Clozel, MD and CEO of Actelion, commented: "We take our leadership in PAH as a responsibility and have now committed to the first long-term, event-driven study in children with PAH using a pediatric formulation of macitentan. The design of the TOMORROW clinical trial allows the safe and comprehensive assessment of children over a long period of time. This is another example of our long-term commitment to the PAH community and our intention to broaden the use of macitentan to new patient populations."

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ABOUT THE TOMORROW STUDY
TOMORROW (pediaTric use Of Macitentan tO delay disease pRogRessiOn in PAH Worldwide) is a multicenter, open-label, randomized, event-driven study to assess the efficacy, safety and pharmacokinetics of macitentan versus standard of care in children with pulmonary arterial hypertension. The study will enroll patients with an age range from 1 month to 18 years in more than 20 countries. The patients will be randomized in a 1:1 ratio into two treatment groups - macitentan or standard of care as per local practice. The trial is expected to last up to 6 years, with patients remaining in the study until the target number of primary efficacy endpoints is met. The primary efficacy endpoint is defined as time to the first Clinical Event Committee (CEC) confirmed disease progression event, comprising:

• Death (all causes), or
• Atrial septostomy or Pott's anastomosis, or registration on lung transplant list, or
• Hospitalization due to PAH, or
• Clinical worsening of PAH

Due to the open-label nature of the study, the management of investigational centers as well as data management, data analysis and coordination of the CEC will be conducted by a CRO. The primary endpoints of the study will be adjudicated by a blinded CEC, in a similar approach to that used in the Phase III SERAPHIN study, where macitentan was studied in adult patients with PAH. An interim analysis for early efficacy or futility is planned when at least 131 CEC-confirmed first disease progression events (70% information fraction) have occurred. If Actelion completes the study as outlined, the company can apply for the extension of the marketing exclusivity for Opsumit both in the US and the European Union.