About Carfostin®

Carfostin® is NanoCor’s lead candidate.

 

    Compared to existing treatments, Carfostin addresses heart disease at the molecular level, which allows for four primary benefits to CHF patients:

  1. Increased heart contractility
  2. Halting of the remodeling and disease progression process
  3. Minimally invasive treatment
  4. Long-term, sustained therapeutic benefits with a one-time treatment

Carfostin® Target

Carfostin® targets a key enzyme that regulates calcium cycling and contractility in heart muscle cells and is deficient in advanced heart failure. Carfostin takes advantage of recent advances in the understanding of the molecular basis of myocardial dysfunction, and the evolution of safe and efficient gene transfer technology. Carfostin consists of a therapeutic gene, Protein Phosphatase-1 Inhibitor-1 (“I-1”), delivered by two proprietary delivery technologies:

Biological NanoParticles (“BNPs”)
AND
Self-Complementary Vectors.

Intellectual Property

NanoCor has an exclusive license to these two technologies for the delivery of certain genes and other reagents for the treatment of heart failure and other heart diseases. Carfostin® is ready to enter human clinical trials. The company has demonstrated efficacy of I-1 gene transfer in large animal (porcine) heart failure models (ischemic & non-ischemic) and recently filed an IND application.

Market Opportunity

There are 5.8 million heart failure patients in the United States and about 15 million worldwide. The initial target market for Carfostin® is stage III and IV CHF patients with systolic heart failure. Assuming exclusion of about 25% of patients due to neutralizing antibodies against AAV, the initial addressable population for Carfostin® is approximately 435,000 late-stage heart failure patients. Based on competitive price assumptions and 5-10% market penetration, the Company believes that Carfostin® can generate worldwide annual revenues ranging from $650 million to $1.3 billion. Porcine studies indicating efficacy of Carfostin®  in the treatment of diastolic heart failure could result in a second label indication for the product. As there are very few treatment options available for diastolic heart failure, this is a significant market opportunity for Carfostin®.

Future Products

NanoCor expects to develop other therapeutics for the treatment of heart disease, following the achievement of proof of principle with Carfostin®. Once the vector technology has been validated in human clinical studies, the I-1 gene can be removed and replaced with other therapeutic genes or RNAi for the treatment of other forms of heart disease. NanoCor has licensed a variety of genes and RNAi which have been proven to be effective in reputable academic studies for a variety of heart diseases.