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Macitentan for use in treating portopulmonary hypertension

A technology of pulmonary arterial hypertension and macitentan, applied in organic active ingredients, cardiovascular system diseases, medical preparations containing active ingredients, etc., can solve problems such as no treatment

Pending Publication Date: 2021-12-14
ACTELION PHARM LTD
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0015] Despite current standard of care, there is no treatment for subjects with PoPH; instead, such patients have been managed with commercially available PAH drugs, including SRA

Method used

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  • Macitentan for use in treating portopulmonary hypertension
  • Macitentan for use in treating portopulmonary hypertension
  • Macitentan for use in treating portopulmonary hypertension

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0127] Example 1: The PORTICO Study

[0128] The study was conducted in seven countries, including Brazil, Czech Republic, France, Germany, Spain, the United Kingdom and the United States (52 sites initiated and patients enrolled and randomized at 36 sites).

[0129] Research objectives

[0130] main target

[0131] To assess the effect of macitentan versus placebo on PVR in patients with PoPH.

[0132] secondary goal

[0133] To evaluate the effect of macitentan compared with placebo on cardiopulmonary hemodynamics, hepatic portal pressure, disease severity and exercise capacity in patients with PoPH.

[0134] To evaluate the safety and tolerability of macitentan in patients with PoPH.

[0135] To assess the PK of macitentan and its active metabolite ACT-132577 in patients with PoPH (PK subgroup).

[0136] research plan

[0137] A. Overall Study Design and Plan

[0138] A1. Main research

[0139]The study was designed as a randomized, double-blind (DB), ...

Embodiment 2

[0662] Example 2: Analysis of Liver Transplantation

[0663] A. Purpose

[0664] The purpose of the analysis includes :

[0665] - To evaluate the effect of macitentan compared to placebo on N-terminal pro-b-type natriuretic peptide (NT-proBNP) relative to a threshold of 300 ng / L in patients with PoPH.

[0666] - To assess the effect of macitentan compared with placebo on perioperative risk stratification of liver transplantation in patients with PoPH.

[0667] - To evaluate the effect of macitentan compared to placebo on MELD exception eligibility in patients with PoPH.

[0668] - To assess the effect of macitentan compared with placebo on the risk of liver transplant waitlist mortality in patients with PoPH.

[0669] B. method

[0670] (i) Diagnosis and main inclusion criteria :

[0671] The study enrolled adult male or female patients (≥18 years) with confirmed symptomatic PoPH and baseline PVR≥4WU (≥320dyn.s.cm -5 ), and be able to perform a 6-minute walk t...

Embodiment 3

[0724] Example 3 - Post hoc analysis

[0725] This example provides a post hoc analysis of the study conducted in Example 1. These analyzes include:

[0726] • To assess the effect of macitentan on mean pulmonary arterial pressure (mPAP) relative to pretreatment mPAP.

[0727] • To assess the effect of macitentan on pulmonary vascular resistance (PVR) relative to pretreatment PVR.

[0728] For the post hoc analysis the following exploratory endpoints were defined:

[0729] • Changes in mPAP relative to pre-treatment mPAP.

[0730] Changes in PVR relative to pretreatment PVR

[0731] A. Statistical methods

[0732] All analyzes were performed on the full analysis set (FAS, see Randomization, N=85) and no data imputation was performed. FAS included all randomized patients who received at least one dose of study treatment during the double-blind treatment period and had a baseline value for the primary endpoint, PVR.

[0733] Risk categories are recorded in Table 38. Fo...

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Abstract

The present invention provides methods for treating portopulmonary hypertension, comprising administering to a patient in need thereof, a therapeutically effective amount of macitentan. Preferably, the methods are clinically proven safe and / or effective. Also provided are methods of improving liver transplant perioperative mortality risk category, improving MELD exception eligibility, and reducing the risk of removal from a liver transplant waitlist in a patient with portopulmonary hypertension and liver disease, comprising administering to a patient in need thereof a therapeutically effective amount of macitentan.

Description

[0001] Cross References to Related Applications [0002] This application claims priority to U.S. Provisional Patent Application 62 / 830,008, filed April 5, 2019, the disclosure of which is incorporated herein by reference in its entirety. technical field [0003] The present invention relates to methods for treating a subject diagnosed with portopulmonary hypertension. Background technique [0004] Portopulmonary hypertension (PoPH) is defined as pulmonary arterial hypertension (PAH) associated with portal hypertension, with or without underlying liver disease. PoPH belongs to group I (PAH) of the current WHO classification of pulmonary hypertension (PH). PoPH diagnosis is based on pulmonary hemodynamic criteria for PAH obtained via right heart catheterization (RHC), including mean pulmonary artery pressure (mPAP) ≥25 mmHg at rest and mean pulmonary artery wedge pressure (PAWP) in the case of portal hypertension ≤15mmHg. [0005] The development of PoPH appears to be ind...

Claims

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Application Information

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IPC IPC(8): A61K31/506A61K31/4965A61K31/4985A61K31/519A61K31/5575A61K31/5578A61K45/06A61P1/16A61P11/00
CPCA61K31/506A61K45/06A61P11/00A61P1/16A61K31/519A61K31/4985A61K31/5575A61K31/5578A61K31/4965A61K2300/00A61P9/12
Inventor 洛伊克·佩舍内艾曼纽·科特雷尔
Owner ACTELION PHARM LTD
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