Promising Results on Gene Therapy for Congestive Heart Failure


California researchers report good results in a phase II study of a gene therapy for congestive heart failure. The news comes five months after Celladon's Mydicar gene therapy for CHF failed to meet its trial's endpoints.

San Diego-based Renova Therapeutics reported that its RT-100 gene therapy treatment for patients with reduced left-ventricular ejection fraction congestive heart failure (CHF) is proving safe and effective.

The treatment involves delivering a therapeutic gene encoding adenylyl cyclase type 6 (AC6) directly into the heart via a modified adenovirus.

It is a single-dose procedure done in an outpatient setting.

Reporting on the results of a multi-center, placebo-controlled Phase 2 trial, company co-founder Kirk Hammond, MD, said the therapy showed a strong safety profile and clear dose response.

The study assessed the safety of various doses of RT-100 in people with CHF.

Preliminary results showed no specific safety concerns and no unexpected changes in blood pressure or heart rate, the company said.

The researchers also found "statisically meaningful improvements from baseline" at 12 weeks in key primary and secondary endpoints. Those included left ventricular ejection fraction and patients' reports of quality of life.

Both results were found in two groups of subjects getting the highest doses of the product.

These patients also showed significant improvements over placebo in negative dP/dt, the company said.

The study involved 56 patients in seven US medical centers.

The trial was funded in part by the National Institutes of Health.

The positive news on the trial was in marked contrast to that about the last gene therapy to be touted as a breakthrough in treating CHF.

In April, 2015, San Diego-based Celladon announced that its potential gene therapy Mydicar had failed to reach its primary and secondary endpoints and that researchers could not prove efficacy.

That trial involved a different gene, SERCA2A. Its trial was being conducted under a breakthrough therapy protocol and was funded in part with a $53 million infusion of capital from three big pharma companies.

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