ALLSCHWIL/BASEL, SWITZERLAND – 07 November 2016 – Actelion Ltd (SIX: ATLN) announced that the company will provide an update on its cardiovascular activities today (Monday, 07 November) in an investor conference call and webcast at 14:00 hrs CET.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “Actelion has changed the way pulmonary arterial hypertension (PAH) is treated – with therapies across the continuum of care that have improved the long-term outcome for patients suffering from this devastating disease. We are fully committed to PAH and take our leadership role with great responsibility as we optimize the way our drugs are used as well as look for potential new PAH therapeutic targets.”

Jean-Paul Clozel continued: “I am pleased to provide an update on the strong cardiovascular pipeline that we have built beyond PAH. The excellent MERIT results announced today highlight one of the ways patients with different forms of pulmonary hypertension (PH) might benefit from Opsumit. With cardiovascular disease remaining the number one cause of death in many countries, as a cardiologist, I am proud that Actelion is committed to finding new cardiovascular therapies.”

The investor conference call and webcast will be hosted by Actelion’s CEO Jean-Paul Clozel, Head of Global Clinical Development, Guy Braunstein and Chief Scientific Officer, Martine Clozel, who will discuss the following topics:

The company will present its efforts to develop macitentan in new PAH patient populations (WHO Classification Group 1) with label-enabling studies in children (TOMORROW), in patients with Eisenmenger Syndrome (MAESTRO), and in patients with portopulmonary hypertension (PoPH) (PORTICO).

The company will also discuss its commitment to post-launch characterization and safety activities in PAH with an observational registry (OPUS study), studies (SYMPHONY & ORCHESTRA) to validate the patient-reported outcome instrument PAH-SYMPACT®, a study (REPAIR) to evaluate the effect of macitentan on right ventricular remodeling and hemodynamic properties in patients with symptomatic PAH.

Actelion’s efforts to expand the clinical utility of macitentan to new pulmonary hypertension (PH) indications will also be outlined. A study (SOPRANO) to assess the efficacy and safety of macitentan in patients with pulmonary hypertension after Left Ventricular Assist Device Implantation will be presented. In addition, the positive results from the MERIT study in patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH; WHO Classification Group 4) are now available and will be presented.

As part of investigating macitentan in new cardiovascular indications beyond PH, the company will introduce a Phase III study, RUBATO. This study will assess the efficacy and safety of macitentan in stable Fontan-palliated adolescent and adult patients. In addition, following full evaluation of the pilot MELODY study in patients with combined pre- and post-capillary pulmonary hypertension (CpcPH), the company believes it has identified a heart failure patient population that could most likely benefit from treatment with an endothelin receptor antagonist (ERA). A study is currently being discussed with health authorities.

Following the outstanding launch momentum of Uptravi, the company will present its measures to expand the clinical utility of this important asset.

Actelion is conducting a study (TRANSIT) to assess the tolerability and safety of the transition from inhaled treprostinil to oral selexipag in adult patients with PAH.

To further advance standard of care in PAH, Actelion is conducting a study (TRITON) to compare the efficacy and safety of an initial triple oral treatment regimen of macitentan together with tadalafil and selexipag versus an initial dual oral treatment regimen in newly diagnosed, treatment-naïve patients with PAH.

Working closely with health authorities, the company is in the process of developing a strategy for investigating the use of Uptravi in children with PAH.

In addition, an intravenous (i.v.) formulation of selexipag is being developed for the treatment of patients with PAH who are prescribed oral selexipag and who are temporarily unable to take oral medication.

As there is no therapy approved for children with PAH in the US, in August of this year, Actelion submitted an NDA for the pediatric appropriate dispersible tablet formulation of Tracleer to the FDA. The company looks forward to further discussions with FDA on this important program and if approved, will make the pediatric formulation available to treat these young patients.

The company is currently investigating a new potent dual ERA, ACT-132577, the main active metabolite of macitentan, in a dose-finding study in essential hypertension. Once the optimal dose has been identified, the company will investigate this dual ERA in resistant hypertension as a first indication.

Actelion also has a new chemical entity for cardiovascular indications which is currently in Phase I development.

In addition to the cardiovascular pipeline which Actelion presents today, the company is also pursuing development of in-house innovation in other therapeutic areas. The programs are progressing as communicated with the exception of ponesimod in graft versus host disease, where the company has decided to stop the current study due to difficulty in enrollment.

  Compound Indication Study Status
Phase III Cadazolid Clostridium difficile-associated diarrhea IMPACT Ongoing
Macitentan Eisenmenger syndrome MAESTRO Ongoing
Macitentan Pediatric PAH TOMORROW Initiating
Macitentan Portopulmonary hypertension (PoPH) PORTICO Ongoing
Macitentan Fontan-palliated RUBATO Initiating



Multiple sclerosis OPTIMUM Ongoing
Ponesimod Multiple sclerosis POINT Initiating
Phase II Cenerimod Systemic lupus erythematosus - Ongoing
Clazosentan Reversal of vasospasm associated with aneurysmal subarachnoid hemorrhage REVERSE Ongoing
Dual Orexin Receptor Antagonist Insomnia - Ongoing
Endothelin Receptor Antagonist Specialty cardiovascular disorders - Ongoing
Macitentan Chronic thromboembolic pulmonary hypertension MERIT Complete
Phase Ib Lucerastat Fabry disease - Complete
Phase I New Chemical Entity Cardiovascular disorders - Ongoing
New Chemical Entity Inflammatory disorders - Ongoing
Selective Orexin 1 Receptor Antagonist Neurological disorders - Ongoing
T-type Calcium Channel Blocker Neurological disorders - Ongoing