Neuren Pharma (ASX:NEU) has seen its shares jump over +11% as the company wins a patent to treat Pitt Hopkins Syndrome (PTHS) with its drug NNZ-2591.
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During clinical trials treating PTHS with the drug over 13 weeks, Neuren reported that neuropsychological “improvements were seen.”
PTHS is typically marked by pronounced intellectual disability and can also produce breathing problems among myriad afflictions. While the syndrome itself is not life-threatening, its complications can be.
“There are currently no approved treatments for PTHS, which has a severely debilitating impact on the lives of patients, as well as their parents and siblings,” Neuren wrote on Thursday.
It fits the company’s overall profile. It already focuses on drugs treating rare conditions in children, the best known being DAYBUE.
Still, 1Y returns for Neuren shareholders are down -33%, though that isn’t related to the short seller report.
Stocks appear to have begun a downward trajectory last August when the company’s Phase 2 results in a trial treating Angelman Syndrome failed to impress.
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