LONDON (ShareCast) - (Sharecast News) - Switzerland's Roche has bought the rights to launch and sell one of Sarepta Therapeutics's gene therapy treatments for Duchenne muscular dystrophy outside of the States, sending shares in the US-based biotechnology outfit flying in premarket trading.
At closing, Roche will pay $750.0m upfront, in cash, and another $400.0m in shares for Sarepta's SRP-9001 micro-dystrophin gene therapy.

Under the agreement, which was expected to close in the first quarter of 2020, Sarepta would also be eligible to receive as much as $1.7bn in regulatory and sales milestones, in addition to royalties on net sales.

According to Sarepta, pre-clinical trials with mice had revealed that after four weeks of treatment, SRP-9001 was capable of triggering at least a 70.0% increase in dystrophin production within muscle fibers, together with a marked improved in muscle function.

DMD patients are missing the gene responsible for the creation of dystrophin by the human body, a protein key for maintaining muscle fibre strength and avoiding injury, the absence of which, most typically in males, can lead to death by the time of early adulthood.

Making use of a non-harmful virus 'vector', researchers can introduce 'transgenes' into skeletal and cardiac muscle that have many of the same properties as regular dystrophin.

Sarepta was due to deliver a business update at 1330 GMT on Monday.

According to the US National Institutes of Health, the estimated primary completion date for the ongoing clinical trial for SRP 9001, which began on 22 December 2018, was 10 October 2022.

That trial had two parts, with the first consisting of a 48-week of a randomized, double-blinded, placebo-controlled period, while the second consisted of a 96-week, double-blinded extension period.

As of 0950 GMT, shares of Sarepta were climbing by 9.77% to $138.50, while those of Roche were up by 0.41% to 314.90 Swiss francs.

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