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Sarepta surges on new hopes for its DMD drug

Last updated: 15:08 07 Jun 2016 BST, First published: 09:20 07 Jun 2016 BST

A clinical research environment
The FDA, has requested additional data from an ongoing study as it decides whether to approve the drug or not.

Shares in drug developer Sarepta Therapeutics Inc (NASDAQ:SRPT) skyrocketed in pre-market as there was renewed hope for approval  its compound to treat fatal muscle wasting disease Duchenne Muscular Dystrophy.

The all important body, the FDA, has requested additional data from an ongoing study as it decides whether to approve the drug or not.

It means it could lead to an approval, and shares In Sarepta rose over 40%.

The latest twist comes after news in April this year, the drug, which is called eteplirsen was given short shrift after an advisory panel to the FDA, determined that the treatment was not effective, sending Sarepta shares down by a third.

But now the FDA has requested that Sarepta provide dystrophin data from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing evaluation of the new drug application (NDA) for eteplirsen New Drug Application.

Sarepta plans to submit data from 13 patient biopsy samples to the FDA over the coming weeks to facilitate a prompt decision on the NDA by the Agency.

Eteplirsen is designed to address the underlying cause of DMD by restoring the dystrophin messenger RNA (mRNA) reading frame, thus enabling the production of a shorter, functional form of the dystrophin protein.

Wedbush upgrades the share

Broker Wedbush lifted its rating on Sarepta to 'outperform' from 'neutral'

"In our view, FDA has found middle ground and given Sarepta a rapid path towards accelerated approval by requesting dystrophin data that the company has in hand."

It added: "We believe there is a good chance these data will demonstrate required dystrophin production and recommend shares ahead of a regulatory decision, which could come in 2016."

Summit Therapeutics shares nudge higher

Today's news will be closely watched by London and Nasdaq -quoted  Summit Therapeutics PLC (NASDAQ:SMMT, LON:SUMM), whose shares nudged 1.5% higher in London.

It is developing a treatment called Ezutromid for the disease - still in the formative stages.

Last week said it expects to start enrolling and dosing patients in what's called the PhaseOut DMD at trial sites in the UK during the second quarter of 2016 and muscle data from this Phase 2 clinical trial  is expected in January 2017.

In May, the US FDA cleared ezutromid as an investigational new drug and enrolment of patients into PhaseOut DMD at trial sites in the USA is expected during the third quarter of 2016.

DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world, generally leading to death by the late twenties.

The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles.

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