Capricor Therapeutics (CAPR) Announces Statistically Significant Data from CAP-1002 DYNAMIC Trial in Adv. Heart Failure

October 31, 2016 9:04 AM EDT

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Capricor Therapeutics, Inc. (Nasdaq: CAPR) announced detailed results from its DYNAMIC clinical trial of CAP-1002 in patients with advanced heart failure. The results were featured at a poster session held on Saturday, October 29th at the 28th Transcatheter Cardiovascular Therapeutics (TCT), the annual scientific symposium of the Cardiovascular Research Foundation, in Washington, D.C. The poster may be accessed at www.capricor.com. Capricor had announced top-line 12-month data from DYNAMIC in June. CAP-1002 is Capricor's investigational allogeneic cardiosphere-derived cell (CDC) therapy.

In DYNAMIC, 14 patients with New York Heart Association (NYHA) Class III heart failure secondary to dilated cardiomyopathy, and who demonstrated a left ventricular ejection fraction (LVEF) ≤35% despite maximal medical and device-based therapy, were treated with a single administration of CAP-1002. The study medication was given at one of four escalating dose levels (37.5 million, 50.0 million, 62.5 million, or 75 million cells) and was delivered directly to the three major coronary arteries via intracoronary infusion. Patients were then followed for 12 months for periodic safety and efficacy assessments.

Efficacy was evaluated at six and 12 months following CAP-1002 infusion. In pooled-dose analyses (N=10-13), measures of functional status and capacity, cardiac function and dimension, and quality-of-life broadly showed trends of improvement from baseline at both time points. At six months, statistically-significant improvements in NYHA Class (p=0.01) and in left ventricular ejection fraction (LVEF) (p=0.02), well as in the Minnesota Living with Heart Failure Questionnaire (MLHFQ) score (p=0.01), were demonstrated. The level of significance for LVEF improvement was maintained at twelve months (p=0.02).

Left Ventricular Measure

Change* (SE)at Month 6

p value†

Change* (SE)at Month 12

p value†

Ejection Fraction (EF)

14.3 (6.9, 24.8)

0.02

17.5 (6.1, 33.4)

0.02

Fractional Shortening (FS)

14.4 (-5.2, 60.8)

0.08

28.5 (-13.7, 61.4)

0.11

End-Diastolic Volume (EDV)

-15.2 (-30.9, 6.1)

0.15

-4.2 (-38.4, 8.1)

0.28

End-Systolic Volume (ESV)

-20.4 (-29.9, 2.2)

0.09

-14.3 (-45.9, 8.0)

0.27

All echocardiographic studies were read by a core lab.

* Median (interquartile range) percent change.

† Signed rank test of the null hypothesis that change from baseline = 0.

Eleven of twelve (92%) patients assessed improved by at least one NYHA Class at six months (p=0.01). All five patients who received the highest dose of CAP-1002 improved. Among this group, at six months three improved by one class to Class II and two improved by two classes to Class I; at 12 months, two were at Class II and three were at Class I, demonstrating further improvement and indicating durability of the benefit of CAP-1002 on heart failure status for as long as 12 months following administration.

High Dose Group (N=5):

NYHA Class

Baseline

N (%)

Month 6

N (%)

Month 12

N (%)

I

2 (40%)

3 (60%)

II

3 (60%)

2 (40%)

III

5 (100%)

IV

CAP-1002 was well-tolerated in DYNAMIC. There were no events recorded for the primary safety endpoint, which was a composite of five cardiac event types.

Event

N (%)

TIMI Flow Score 0-2

0 (0%)

Acute Myocarditis Within One Month of Infusion

0 (0%)

Ventricular Tachycardia or Ventricular Fibrillation Within 72 Hours of Infusion

0 (0%)

Sudden Unexpected Death Within 72 Hours of Infusion

0 (0%)

MACE Within 72 Hours of Infusion

0 (0%)

"Patients with NYHA Class III heart failure experience marked limitations in their physical activity, and suffer fatigue and shortness of breath even at less than ordinary levels of exertion. Although current options enable us to provide some support for their condition, the ability to therapeutically alter their progressive decline over the long-term is still lacking. Therefore, it is very encouraging to see evidence that CAP-1002 may be capable of modifying the underlying disease process in heart failure, the leading cause of hospitalization among U.S. citizens over 65 years of age," said Raj R. Makkar, M.D., Director, Interventional Cardiology and Cardiac Catheterization Laboratory, Cedars-Sinai Medical Center.

Dr. Linda Marbán, president and chief executive officer of Capricor, added, "We are very encouraged by both the safety as well as the signs of efficacy shown by CAP-1002 in DYNAMIC. This trial indicates, for the first time, that the cells can work in patients with longstanding heart failure, potentially providing entry into the large and poorly-met heart failure market. It also delineates a dose-response relationship that will inform the design of future trials in this and other indications. In fact, as in DYNAMIC, CAP-1002 at a dose level of 75 million cells was given by triple-coronary infusion in our ongoing HOPE trial in Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. We expect to report top-line six-month data from HOPE in the first quarter of 2017."



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