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UPDATE - Summit receives cornerstone patent in US for muscular dystrophy drug

Published: 08:17 28 Aug 2013 EDT

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The drug development group Summit (LON:SUMM) has been issued a composition of matter patent that covers its discovery SMT C1100 for use in the treatment of the fatal muscle wasting disease Duchenne Muscular Dystrophy.

This “cornerstone” patent provides a period of exclusivity for the small molecule utrophin modulator to at least November 2028.

Earlier in the month the US issued a patent that protects the use of C1100 in combination with existing treatments for DMD, such as medical steroids prednisolone or deflazacort.

C1100 is a utrophin modulator, which means it has the potential to maintain healthy muscle tone. This offers a potential breakthrough in treating DMD, a rare wasting disease that affects boys only (and 1,500 here in the UK).

It is caused by faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of muscles.   There is no cure and life expectancy is around the mid-twenties.

Utrophin protein is the functional equivalent of dystrophin and non-clinical studies have shown it can substitute for dystrophin.

Chief executive Glyn Edwards said: "The grant of this cornerstone patent for SMT C1100 in a key commercial market represents a critical component in our strategy for advancing this promising utrophin modulator in the treatment of DMD.

"Together with the recently issued US patent covering the use of SMT C1100 in combination with steroids, the intellectual property estate protecting this potential life-changing drug has been significantly strengthened as we prepare to start patient clinical trials during the second half of this year."

Shares in the group, which have almost doubled in value in the past month, advanced a further 7% to 8.84p.

That values the group at almost £40mln, which is a long way behind its closest comparator company, NASDAQ-listed Sarepta Therapeutics (NASDAQ:SRPT), which is worth US$1.1bn (£711mln). It too is developing a treatment for DMD.

In the US, the NASDAQ biotechnology index has been on something of a roll, doubling in value in two years.

Last September it exceeded its all-time high (posted in 2000) and has continued to rise as the larger, traditional drugs giants have weighed in and begun to snap up some of the more promising bio-pharma firms.

This process of consolidation shows no signs of slowing as deep-pocketed big pharma resorts to buying in new products as its R&D departments run out of ideas.

AstraZeneca's (LON:AZN) US$500mln deal to acquire privately-owned Amplimmune, which hasn't a single drug in the clinic, would appear to be further evidence of this trend.

Here in the UK, there is no NASDAQ-style index charting the ups and downs of the sector, but we are seeing signs of a revival. A bellwether for the health of the sector this side of the Atlantic is the willingness of investors, particularly the institutions, to fund the ongoing development of drugs.

Summit is one of the companies to secure significant funding, alongside e-Therapeutics (LON:ETX) and Silence Therapeutics (LON:SLN).

Summit is also developing a promising antibiotic for the c.difficile.

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