Rafael Stock Slides as Niemann-Pick Disease Drug Fails to Meet Statistical Significance in Trial
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Yesterday
Shares of Rafael Holdings declined after its treatment for Niemann-Pick Disease Type C did not show a statistically significant slowdown in disease progression in a recent trial.
The stock fell 35% to $1.31 Wednesday. Shares are up 10% year to date.
Rafael said its Trappsol Cyclo treatment demonstrated a directionally favorable treatment effect compared with placebo, slowing disease progression by 64% at week 96, but the result did not achieve statistical significance.
The company said it remains on track to submit a new drug application Trappsol Cyclo in the fourth quarter.
"We believe the totality of the data shared today provide a compelling rationale for the continued advancement of Trappsol Cyclo," Chief Medical Officer Karen Mullen said.
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