Capricor Therapeutics Inc., a biotech company focuses on the identification, development and marketing of premiere therapeutic drugs, announced a positive clinical trial results for DYNAMIC or Dilated cardiomYopathy, iNtervention with Allogeneic MyocardIally-Regenerative Cells. This experimental trial evaluated their lead candidate drug – CAP-1002 in patients with advanced heart failure. CAP-1002 is an investigational allogeneic cardiosphere-derived cell or CDC therapy.
In the DYNAMIC trial, 14 patients which are classified as NYHA or New York Heart Association Class III heart failure had undergone a multi-vessel intracoronary infusion of Capricor’s CAP-1002 in four increasing dose cohorts. All patients received a triple-vessel infusion, and one time dose of CAP-1002. The doses range from 37.5 to 75 million cells. Patients had undergone treatment for 12 months. The echocardiographic studies were read in a core lab.
At the six months of the clinical trial, directional improvements were observed based from the baseline of the key efficacy measures. These include quality of life, cardiac dimensions and function and assessments of functional status.
There were 12 patients that were available for follow-up after a year. These patients had clinical assessments; there were improvements from the baseline in the dimensional indices and key cardiac function. More importantly, the change in the median left ventricular ejection fraction from baseline levels to 12 months has maintained statistically significant levels at six months. On the absolute basis, the patients continued to have improvements from 6 to 12 months of treatment.
Based from the result, five NYHA class III subject received the highest dosage of CAP-1002. There were two subjects who improved by two Classes, from Class III they were moved to Class I. There were two Class II subjects that demonstrate further improvement which indicates the durability effect of the drug. The CAP-1002 has a prolonged effect on the improvement of heart failure status which can be maintained for as long as 1 year following the administration of the drug.
The 75 million cell dosage is also presently being assessed in HOPE-Duchenne Clinical Trial. This experimental trial is initiated by Capricor to help boys suffering from DMD or Duchenne muscular dystrophy (DMD) associated with cardiomyopathy.
The infusion of CAP-1002 was well-tolerated by the participants in the DYNAMIC Study. Two out of 14 patients, who were given the lower two doses out of the four dose cohorts, died due to progressive heart failure. The patient died approximately one and three months before the study ended.
Raj R. Makkar, M.D., the lead investigator of the trial and the Director of the Interventional Cardiology and Cardiac Catheterization Laboratory of Cedars-Sinai Medical Center said in an interview that they are very impressed with the results of the preliminary data. The results show consistent and important improvements in cardiac function and structure of the patients. He also noted that the Class III patients are known to follow a foreseeable course of clinical deterioration due to the progression of heart failure. Thus, to see a sustained improvement for 1 year of treatment proves to be a milestone of the study. This may herald novel treatment paradigms for over one million patients suffering from advanced heart failure.
Dr. Linda Marban, CEO and President of Capricor, said that they are pleased with the preliminary results of CAP-1002 in Class III advanced heart failure patients. The ventricular volume and ejection fraction were reduced which implies that CAP-1002 is effective in reverse remodeling. Marban noted that, the most important benefits of CAP-1002 are its durability for 12 months. The clinical data supports the potential of CDC to alter the potential prognosis of patients with advanced and chronic heart failure for which the current treatment options are limited.
Saving the Failing Heart
Capricorn’s CAP-1002 has promising initial trial results. If granted an approval, it will be able to help millions of patients suffering from advanced heart failure. Heart failure is one of the deadly diseases, thus, having a new treatment that promises an effective and long-term efficacy will be welcomed by patients and the medical community.