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Sarepta soars on positive results from early-stage DMD clinical trial

The phase I/II study of Golodirsen hit all of its primary and secondary endpoints, with patients seeing a significant rise in dystrophin which is vital for muscle function
young boy
Duchenne muscular dystrophy is a rare muscle wasting disease that predominantly affects young boys

Shares in Sarepta Therapeutics Inc (NASDAQ:SRPT) surged in pre-market trade on Wednesday after the drug developer reported successful results from an early stage trial of its Duchenne muscular dystrophy treatment.

Sarepta said the phase I/II study of Golodirsen met all primary and secondary endpoints.

DMD is a rare progressive disease muscle disease found only in young boys who lack the protein dystrophin which is critical to muscle function.

All of the 25 boys who took part in the European trial responded to the treatment which Sarepta said demonstrated “proof of mechanism”.

On average, the levels of dystrophin in each patient increased almost eleven-fold from baseline.

Sarepta already has a drug that received accelerated approval from the FDA last year and is approved for treatment in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

That accounts for around 13% of the DMD market. Golodirsen is testing patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping, which accounts for around 8% of patients.

“This is now the second exon-skipping agent to have shown a statistically significant increase in dystrophin production, validating the exon-skipping approach to treating DMD boys with amenable mutations,” said principal investigator Francesco Muntoni.

Sarepta shares added 14.3% tin pre-market trading to US$46.94. In the regular session they added over 14% to $46.93 each.

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