Cytokinetics Ends ALS Pipeline Drug Trial

San Francisco-based biotech firm, Cytokinetics, has discontinued its Phase III trial of reldesemtiv, its amyotrophic lateral sclerosis (ALS) pipeline drug. The company took the decision as there was no evidence of its effectiveness compared to placebo on primary or secondary endpoints.

This news comes after Cytokinetics announced that the FDA had rejected its heart failure drug, omecamtiv. The failure of reldesemtiv and omecamtiv adds to the pressure on the success of Cytokinetics's final Phase III pipeline candidate, aficamten, which has the potential to generate up to $1.37 billion by 2031, according to GlobalData.

Aficamten is a myosin inhibitor that targets patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM). It received Breakthrough Therapy Designation by the FDA in December 2021. Its Phase III trial, titled SEQUOIA-HCM, is set to be completed by September 2023.

Cardiomyopathy is a collection of diseases characterised by impaired heart muscle, with HCM being one of several subtypes. Aficamten is one of two Phase III pipeline therapies for cardiomyopathies. The other, caficrestat, is in clinical trial development by Applied Therapeutics. Caficrestat also targets patients with myocardial infarction, COVID-19, congestive heart failure, diabetic peripheral neuropathy, and pulmonary inflammation.

Sarah Bundra, Pharma Analyst at GlobalData, commented that aficamten will need to have a superior efficacy or safety profile compared to Bristol Myers Squibb's Camzyos (mavacamten) to succeed, as it is the only marketed cardiomyopathies therapy with a unique mechanism of action. Camzyos is a first-in-class myosin inhibitor that received FDA approval in October 2022.