Raptor Pharmaceutical Corp said it plans to advance development of its brain disorder drug to a registration study, despite it failing to show statistical significance in a trial.

The drug, RP103, is being evaluated in patients with Huntington’s Disease, a type of neurodegenerative genetic disorder that affects muscle coordination and leads to mental decline and behavioral symptoms.

The drug showed a slowing rate in total motor score, compared with a placebo, according to data from the 36-month larger study.

Though not statistically significant, the 25 percent treatment effect in the motor score rate in patients treated with RP103 is regarded as “clinically meaningful”, Raptor said on Thursday.

Total motor score is a rating system to quantify the severity of Huntington’s Disease.

There is no currently approved drug that slows disease progression, the company said.

Up to Wednesday’s close of $5.60, the company’s shares had fallen nearly 47 percent this year.


(Reporting by Shailesh Kuber and Rosmi Shaji in Bengaluru; Editing by Sriraj Kalluvila)

Source: Reuters Health