Innovative Therapeutic Development for Duchenne Muscular Dystrophy Moves Niche Stocks Up 300% to 400%: An Expert …

67 WALL STREET, New York – May 16, 2013 – The Wall Street Transcript has just published its Investing in Health Care Report offering a timely review of the sector to serious investors and industry executives. This special feature contains expert industry commentary through in-depth interviews with highly experienced Money Managers. The full issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online.

Topics covered: Health Care – Electronic Health Record Adoption – Biotechnology and Pharmaceutical Investing – Biotechnology and Pharmaceuticals – Biotechnology and Pharmaceutical Companies Valuation – Platform Interoperability and Data Analytics – HITECH Act Incentives – Affordable Care Act

Companies include: Dendreon Corp. (DNDN), Pharmacyclics Inc. (PCYC), Johnson Johnson (JNJ), Celgene Corporation (CELG), Biogen Idec Inc. (BIIB), Pfizer Inc. (PFE), Merck Co. Inc. (MRK) and many more.

In the following excerpt from the Investing in Health Care Report, an experienced portfolio manager discusses his asset allocation philosophy for investors:

TWST: Would you tell us about some of your favorite stocks right now? Tell us a little bit about what you like about them and how they meet your investment criteria.

Dr. Kuruvilla: I can mention a couple of ideas, some of which are becoming more well-known, some of which are still obscure. I’ll start with one that is, I would say, moderate in terms of awareness. We’ve owned it for almost a year, since last summer, but I still don’t think the market has properly valued it. It’s Sarepta (SRPT), which we still own.

It became well-known last year, because it had one of these massive moves up; something like 300% to 400%, maybe more. It’s a company that is developing therapeutics for Duchenne muscular dystrophy, or DMD. DMD is a catastrophic disease. It’s an X-linked disease that afflicts males. They tend to die in their 20s or so, a very steady, debilitating, progressive weakness in the limbs as well as the diaphragm that leads to typical respiratory failure and pneumonia and death.

One of the things that’s really interesting about this company that I don’t think the market has fully digested is the fact that the way they treat Duchenne muscular dystrophy is by manufacturing a particular type of oligonucleotide that has an unusual morpholino backbone, and these morpholinos have thus far been remarkably safe and they’re known to penetrate into cells, and particularly cells that have a leaky membrane, which is exactly the type of cell that a DMD patient suffers from.

DMD patients have a mutation in a gene called dystrophin, and their muscle cells tend to rupture and lyse. They have these leaky muscle cells throughout their periphery, and these morpholinos can go into the muscle cells and they induce, without getting too technical, an exon-skipping approach that essentially leads to a slightly repaired, sometimes fully repaired, version of dystrophin. That enables the patient to be able to make normal dystrophin like you and I have, unlike what they’ve had before…

For more of this interview and many others visit the Wall Street Transcript – a unique service for investors and industry researchers – providing fresh commentary and insight through verbatim interviews with CEOs, portfolio managers and research analysts. This special issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online.