Sarepta rated outperform by Baird on positive biomarker data

 

• Baird has issued Sarepta Therapeutics (NASDAQ:SRPT) an Outperform rating citing the positive topline data from the Phase 3 study conducted in partnership with Lysogene (OTCPK:LYSGF) for LYS-SAF302. The price target of $192 implies an 11.7% upside to today’s close.
• In the ongoing phase 2/3 clinical trial, the two companies evaluate LYS-SAF302 in mucopolysaccharidosis type IIIA. The changes in heparan sulfate concentration in cerebrospinal fluid are being monitored in patients for evidence of in vivo biological activity of the drug and demonstrate proof of concept.
• Yesterday, Lysogene reported positive biomarker data from the study noting statistically significant average reductions in the concentration of HS in the CSF of all nine patients analyzed so far, at 6 and 12 months, relative to pre-treatment values.
• The company said there were no statistically significant changes in serum HS concentrations proving the hypothesis that LYS-SAF302 leads to a reduction of HS entering the CSF from the brain parenchyma, with little or no effect on HS-derived oligosaccharides entering the CSF from extra-parenchymal sources like blood.
• Looking forward to additional data in early 2021, the analysts led by Brian P. Skorney note that they are ‘encouraged by these early biomarker results,’ even though safety concerns remain.
• Citing that the trial was paused in May due to MRI findings, and a patient death was reported in November, the analysts argue there was less impact of safety concerns on Sarepta, which has full commercial rights to LYS-SAF302 in the US and markets outside Europe.
• https://seekingalpha.com/news/3647788-sarepta-therapeutics-rated-outperform-baird-on-positive-biomarker-data