New drugs in preclinical and early stage clinical development in the treatment of heart failure

Expert Opin Investig Drugs. 2019 Jan;28(1):51-71. doi: 10.1080/13543784.2019.1551357. Epub 2018 Dec 7.

Abstract

Introduction: Heart failure (HF) represents a major health problem because of its high prevalence, high rates of hospitalizations, and mortality and significant healthcare costs. HF comprises a heterogeneous group of syndromes with different pathophysiology, clinical presentations, and response to therapy. Basic research has identified multiple putative therapeutic targets involved in the pathogenesis of HF and many promising drugs are under development.

Areas covered: This review summarizes the main drugs currently in preclinical and clinical development for HF treatment. Drugs were identified by search of MEDLINE and PubMed and trials from registries (clinicaltrials.gov, clinicaltrialsregister.eu, pubmed.gov) from 2010.

Expert opinion: Despite significant advances in the pharmacological treatment of HF with reduced ejection fraction, no treatment has yet convincingly shown to improve outcomes in patients with HF with preserved ejection fraction and acute HF. Many promising drugs emerged in preclinical models and small phase 2 trials, but with a few exceptions, they fail to meet the primary endpoints in larger adequately powered phase 3 trials. Which new drugs will prove valuable remains uncertain. Improving clinical outcomes, delaying or preventing HF worsening and reducing hospital admissions should be prioritized in future drug development programs.

Keywords: Heart failure; acute heart failure; clinical trials; novel investigational drugs; preserved ejection fraction; reduced ejection fraction.

Publication types

  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Drug Development / methods*
  • Drug Evaluation, Preclinical / methods
  • Health Care Costs
  • Heart Failure / drug therapy*
  • Heart Failure / epidemiology
  • Heart Failure / physiopathology
  • Hospitalization / statistics & numerical data
  • Humans
  • Molecular Targeted Therapy*