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Sarepta's Golodirsen Positive in DMD Study, Shares Soar

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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced positive results from a phase I/II study – 4053-101 – evaluating its Exon 53 skipping candidate, golodirsen, for the treatment of Duchenne Muscular Dystrophy (DMD) patients.

DMD is a genetic disorder in which the absence of a protein called dystrophin progressively weakens the muscles.

Golodirsen achieved a 100% response rate with 10.7 times increase in mean dystrophin protein from baseline.

We remind investors that Sarepta’s Exondys 51, the only approved treatment for DMD, is an Exon 51 skipping DMD treatment and had achieved nearly three times increase in mean dystrophin protein from baseline. Golodirsen’s better efficacy in terms of dystrophin production than Exondys 51 raised investor skeptism that golodirsen could prove to be a new and better treatment option for DMD patients

It is also anticipated that golodirsen may receive an earlier-than-expected approval, further strengthening Sarepta’s DMD franchise. Shares of Sarepta surged 13.75% on Wednesday on this optimism. In fact shares of the company have increased 70.4%, significantly outperforming the industry’s gain of 15.7% so far this year.

The data from the 4053-101 study showed that after 48-weeks of treatment with golodirsen, mean dystrophin protein increased to 1.019% of the amount found in normal human from the baseline of 0.095% of the normal level. In its clinical study, Exondys 51 had increased the pre-treatment dystrophin level of 0.16% of normal to 0.44%.

The phase I/II study will be continued for 144 weeks.

Exondys 51 was approved in September 2016 and it generated sales of more than $51 million in the first half of 2017. Full-year sales are expected to be in the range of $125 to $130 million.

Sarepta has seven other potential DMD therapies under investigation.

Sarepta has multiple collaborations agreements for developing treatment for DMD. These include a deal with Summit Therapeutics plc (SMMT - Free Report) for developing the latter’s ezutromid and other utrophin modulator pipeline and Catabasis Pharmaceuticals, Inc. (CATB - Free Report) for combination drug treatment.

Moreover, in July 2017, Sarepta signed a settlement agreement with BioMarin Pharmaceutical Inc. (BMRN - Free Report) for resolving global patent proceedings related to the sale of Exondys 51 and development of future DMD exon-skipping products.

Sarepta has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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