Celladon Corporation has secured a $43m equity financing to advance its lead investigational product candidate MYDICAR®, designed for the treatment of heart failure.

MYDICAR is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling and contractility.

The financing round was led by Pfizer Venture Investments and also included new investors Lundbeckfond Ventures, Novartis Venture Funds, H&Q Healthcare/Life Sciences Investors, and GBS Venture Partners.

Previous investors also participated in this round, including Enterprise Partners Venture Capital, Johnson & Johnson Development Corporation and Venrock Associates.

In an earlier Phase 2 Cupid trial, high-dose MYDICAR met its primary safety and efficacy endpoints at six months compared to placebo.

At the 12-month period, patients treated with the highest dose of MYDICAR showed an 88% risk reduction of major cardiovascular events, such as death, need for left ventricular assist device (LVAD) or cardiac transplant, episodes of worsening heart failure and a number of heart failure-related hospitalisations.

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The mean duration of hospitalisation in the MYDICAR high-dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group.

The study also showed that heart failure, which is a progressive disease, became stabilised in high-dose MYDICAR-treated patients.

Celladon president and CEO Krisztina Zsebo Ph.D., said: “The positive results of the phase 2 CUPID trial demonstrated the potential of MYDICAR to become an important treatment for patients with chronic, advanced heart failure.”

MYDICAR is delivered using a recombinant adeno-associated virus (AAV) in a single dose directly to the heart during a routine outpatient cardiac catheterisation procedure.

The common symptoms of heart failure include shortness of breath, feeling tired and swelling in the ankles, feet, legs, and sometimes the abdomen.

In December 2011, MYDICAR was granted fast-track designation by the US Food and Drug Administration.

Celladon is a privately held biotechnology company that specialises in developing molecular therapies for the treatment of heart failure.