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Treatment of cardiac dysfunction and heart failure with reduced ejection fraction with compound (r)-4-(1-((3-(difluoromethyl)-1-methyl-1h-pyrazol-4-yl) sulfonyl)-1-fluoroethyl)-n-(isoxazol-3-yl) piperidine-1-carboxamide

A heart failure, dysfunction technique applied to the use of the compound (R)-4-(1-((3-(difluoromethyl)-1-methyl-1H-pyrazol-4-yl)sulfonyl )-1-fluoroethyl)-N-(isoxazol-3-yl)piperidine-1-carboxamide in the field of treatment of systolic dysfunction and heart failure with reduced ejection fraction, capable of addressing non-approved cardiac Exhaustion and other issues

Pending Publication Date: 2022-03-11
MYOKARDIA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0010] There are currently no approved therapies for heart failure by directly targeting the contracting organ

Method used

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  • Treatment of cardiac dysfunction and heart failure with reduced ejection fraction with compound (r)-4-(1-((3-(difluoromethyl)-1-methyl-1h-pyrazol-4-yl) sulfonyl)-1-fluoroethyl)-n-(isoxazol-3-yl) piperidine-1-carboxamide
  • Treatment of cardiac dysfunction and heart failure with reduced ejection fraction with compound (r)-4-(1-((3-(difluoromethyl)-1-methyl-1h-pyrazol-4-yl) sulfonyl)-1-fluoroethyl)-n-(isoxazol-3-yl) piperidine-1-carboxamide
  • Treatment of cardiac dysfunction and heart failure with reduced ejection fraction with compound (r)-4-(1-((3-(difluoromethyl)-1-methyl-1h-pyrazol-4-yl) sulfonyl)-1-fluoroethyl)-n-(isoxazol-3-yl) piperidine-1-carboxamide

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0189] Example 1: Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of Compound I in Healthy Adult Volunteers

[0190] This example illustrates the first study of Compound I in humans. Based on the mechanism of action of Compound I, Compound I may provide targeted therapy for patients suffering from DCM caused by genetic or non-genetic mechanisms. The study was a randomized, double-blind, placebo-controlled, sequential group, single ascending (oral) dose study in healthy subjects aged 18-55 years. Eight dosing cohorts were enrolled, each containing 8 healthy subjects. Within each cohort, subjects were randomized 6:2 to Compound 1:placebo.

[0191] Materials and methods

[0192] Research design

[0193] Subjects stayed at the clinical site for up to 5 days and 4 nights, from Day -1 (the day before dosing) to Day 4, and received a single dose of Compound I or placebo on Day 1. ECG t...

Embodiment 2

[0318] Example 2: An open-label, pilot, randomized two-phase crossover study evaluating the effect of food on a 25 mg tablet formulation of Compound I at a dose of 200 mg in healthy adult volunteers

[0319] This example illustrates the clinical study used to establish the effect of a high fat, high calorie meal on the PK profile of Compound I in healthy volunteers compared to drug administered in the fasted state. The study also sought to determine the safety and tolerability following a single oral dose of Compound I in healthy volunteers in the fed and fasted state. Measurements of PK, PD and other clinical parameters were performed as described in Example 1 above.

[0320] Materials and methods

[0321] Research design

[0322] This study was an open-label, randomized, two-period crossover study in healthy volunteers aged 18-55 years. Subjects were screened up to 28 days prior to the first treatment period. Subjects were admitted to the clinical site on Day -1 of Perio...

Embodiment 3

[0369] Example 3: Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single and Multiple Ascending Oral Doses of Compound I in Patients with Stable HFrEF Randomized, Double-Blind, Placebo-Controlled, Two-Part, Adaptive Design Study

[0370] This example illustrates a preliminary safety and tolerability study establishing single and multiple ascending oral doses of Compound I in ambulatory patients with stable heart failure with reduced ejection fraction (HFrEF). Key eligibility criteria included stable HFrEF of ischemic or non-ischemic origin treated with guideline-directed medical therapy (EF at screening initially required 20% to 45%, and subsequently changed to 15% to 35% upon revision). Subjects with active ischemia or severe or valvular heart disease were excluded. The study also aimed to (1) establish the preliminary human PK of Compound I after single and multiple ascending oral doses of Compound I in patients with HFrEF; Changes in left ventri...

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Abstract

Provided herein are methods, uses, and compositions for treating systolic dysfunction, such as heart failure with reduced ejection fraction.

Description

[0001] 【CROSS-REFERENCE TO RELATED APPLICATIONS】 [0002] This application claims priority to U.S. Provisional Patent Application 62 / 849,936, filed May 19, 2019, and U.S. Provisional Patent Application 62 / 852,739, filed May 24, 2019. The disclosures of these priority applications are hereby incorporated by reference in their entirety. 【Background technique】 [0003] Heart failure (HF) is a global epidemic that affects approximately 26 million people worldwide. It is the fastest growing cardiovascular disease worldwide with a very high morbidity, mortality and cost burden on the health care system (Ponikowski et al., ESC Heart Fail. (2014) 1(1):4-25; Savarese and Lund, Card Fail Rev. (2017) 3(1):7-11). HF is the most common cause of hospitalization in patients over 65 years of age (Ponikowski, supra; Savarese and Lund, supra; and Shah et al., J Am Coll Cardiol. (2017) 70(20):2476-86). The five-year mortality rate after HF hospitalization is approximately 42%, comparable to m...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/454A61K45/06A61K9/20A61K9/48A61P9/00A61P9/04A61P9/02A61P9/10A61P9/12A61P11/00A61P7/10A61P31/12A61P3/00A61P3/10
CPCA61K31/454A61K45/06A61K9/20A61K9/48A61P9/00A61P9/04A61P9/02A61P9/10A61P9/12A61P11/00A61P7/10A61P31/12A61P3/00A61P3/10A61K2300/00A61K9/0095A61K9/0053A61K9/14
Inventor J-F·坦比杨春T·卡尔森
Owner MYOKARDIA
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