Celladon Starts Clinical Trial Of Mydicar in Heart Failure Patients
By Cyndi Root
Celladon announced in a press release that it has dosed its first heart failure patient supported with a Left Ventricular Assist Device (LVAD) in a trial of Mydicar. The genetically targeted enzyme replacement therapy restores the enzyme SERCA2a to normal levels. The trial, partially funded by the British Heart Foundation and the Imperial College London, is a proof-of-concept trial to furnish data for future investigations.
Krisztina Zsebo, CEO of Celladon, said, "The initiation of this trial highlights the potential for Mydicar to positively impact a wide range of cardiovascular conditions. The LVAD patient population has exhausted existing pharmaceutical and device treatment options.”
The trial's lead investigator, Dr. Alex Lyon from the Imperial College London's National Heart and Lung Institute, said this is the first time that this patient group has received cardiac gene therapy. He said, “We will be taking a heart muscle biopsy from each patient to measure the levels of treatment gene DNA delivered to their heart, and whether the presence of AAV neutralizing antibodies interferes with the efficiency of gene delivery.”
Mydicar Trial
The new Celladon clinical trial is titled "Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients with Heart Failure and a Left Ventricular Assist Device (LVAD).” Investigators intend to assess Mydicar in patients supported by the LVAD device. The study will enroll 24 patients and evaluate safety and feasibility. Half of the participants will be treated with Mydicar and the other half will receive a placebo.
Researchers will measure the effects using Doppler echocardiography, six-minute walk tests, cardiopulmonary exercise, and metabolic stress tests. All patients will undergo a heart biopsy to determine whether the SERCA2a gene is still present at six months. In a sub-study, researchers will assess if the treatment is effective enough to wean or remove the LVAD device from the patient’s treatment regime.
Mydicar
Mydicar targets and restores SERCA2a enzyme levels, thereby improving the heart’s pumping ability. The agent is infused into the coronary arteries in a one-time outpatient cardiac catheterization procedure. An adeno-associated virus (AAV) transfers the SERCA2a gene into the cell nucleus, improving heart muscle contractions. In a Phase 2a study of 39 patients, Mydicar-treated patients showed that the treatment was safe and well tolerated. The agent improved symptoms, reduced hospitalizations, and showed improvements in markers predictive of survival.