Duchenne Muscular Dystrophy Drug Could Unlock Huge Potential For This Pharmaceutical

By Jordo Bivona - January 31, 2013 | Tickers: CELG, GILD, SRPT | 0 Comments

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Presentingat the 31st Annual JPMorgan Healthcare Conference, Sarepta Therapeutics (NASDAQ: SRPT) CEO Christopher Garabedian focused primarily on the company's Duchenne muscular dystrophy program.

Duchenne Muscular Dystrophy is a disease that is characterized by patients being unable to produce the essential protein Dystrophin. Because of this lack of Dystrophin, patients end up on a progressive track towards losing the ability to walk by their pre-teen years, and have a life expectancy into their 20s, rarely living beyond the age of 30.

Dystrophin is the shock absorber of the muscles and allows people to maintain good muscle function into their adult lives. The company's drug, Eteplirsen, is an anti-sense Oligonucleotide with a very different backbone structure than seen in any other RNA therapeutics. The drug is made by manipulating RNA, or ribonucleic acid, which controls protein synthesis. The company is working on using its technology to direct alternative splicing to repair the RNA mutation and restore the translation to produce the protein. The mechanism of action is what can be described as Exxon skipping; the Duchenne genotype is defined an out of frame deletion in the dystrophin gene. This renders them unable to translate to the protein. If translation can be restored by turning this out of frame deletion into an in-frame deletion, then the translation of the protein can be restored.

Market Opportunity

From a market opportunity point of view, there are many drugs that have been approved. The various price points are, on average, between $300,000 and $500,000 per patient per year. If you consider this in terms of the target population, the company estimates there would be about 2,000 patients in the U.S. that would be amenable to Eteplirsen. This translates into a $600 million to $1 billion market opportunity in the U.S. alone. The EU could add 9,000 additional patients as well.

Financials

Sarepta has a strong cash position, with approximately $187 million on the balance sheet. 2012 guidance showed an operating loss of $25 million to $30 million. Significant additional cash burn is not expected in 2013. Any increases would come from a manufacturing scale up, and this depends on whether or not large scale production and accelerated approval is pursued.

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Duchenne Muscular Dystrophy Drug Could Unlock Huge Potential For This Pharmaceutical