This week Jim Cramer has been highlighting the hottest biotech stocks for 2015 as a part of his series “Biotech, the Next Generation.”
One of the best performing biotechs for 2014 was the orphan drug company PTC Therapeutics, which rallied 205 percent last year. Shire PLC recently acquired NPS Pharmaceuticals at a 51 percent premium to where it was trading before.
This acquisition is a signal to Cramer that big pharma has finally figured out the enormous earning potential for orphan drug companies, which could mean there is plenty more upside for PTC.
PTC focuses on rare genetic disorders, especially those caused by nonsense mutations in DNA. Its main drug is Translarna, approved in the EU for duchenne muscular dystrophy (DMD) and could launch in the U.S. next year.
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What makes Translarna unique from other treatments is that PTC is developed for a small subset of patients for a particular genetic mutation. Cramer thinks it could potentially be worth more than a billion dollars.
To find out if this company can keep pulling in profits for 2015, Cramer sat down with PTC Therapeutics CFO Shane Kovacs.
“We’ve got a different and unique approach to RNA biology and to rare diseases. Unlike some of the enzyme replacement therapies where you make the protein in a lab and then infuse it, our approach is to enable the patients to produce their own protein using a small molecule approach,” said Kovacs.
The CFO emphasized that DMD is a gene disorder specific to boys, and the earlier the treatment in life the better. In fact, the treatment might be able to save lives if started between the ages of 5 and 6.
“In Duchenne this is a muscle wasting disorder. What we are trying to do is stabilize the boys, to keep their healthy muscle healthy so that it isn’t so easily damaged. So you want to treat these boys as young as possible as a chronic therapy that they will take for the rest of their lives,” he added.
Cramer pointed out that it is a rare circumstance where the EU likes a drug, but the FDA does not. This could mean potentially that the drug could be also be approved in the U.S. Either way; the EU approval indicates that PTC will have plenty of revenue for the year.
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“Last year was a monumental year for us, because the European authorities approved our drug. This is the first approval ever in this disease for the underlying cause,” said Kovacs.
As the only approved treatment for patients of DMD, that makes PTC more valuable. However, before buying the stock the “Mad Money” host wants investors to read the most recent presentation from PTC and see the value of this stock with their own eyes.
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