Alcobra acknowledged today that its attention-deficit/hyperactivity disorder (ADHD) candidate metadoxine extended release (MDX) has failed a Phase III trial.
MDX did not meet the primary endpoint of the MEASURE clinical trial, namely demonstrating a statistically significant difference from placebo in the change from baseline as measured using the Conners’ Adult ADHD Rating Scales (CAARS).
“We are exceedingly disappointed with these topline results,” Alcobra president and CEO Yaron Daniely, Ph.D., MBA, said in a statement. “In the coming weeks, the Company intends to review the full dataset from MEASURE. Consequently, we will evaluate our options and communicate our strategic plan to investors.”
Alcobra said its topline data analysis was conducted on MEASURE’s full analysis set, which included all of the study’s 283 randomized subjects with at least one post-baseline efficacy assessment.
The failure announcement came a week after Alcobra won FDA agreement to review data collected to date in the MEASURE study and consider it in a future NDA submission of MDX for ADHD. The FDA also agreed to change a full clinical hold for the trial to a partial clinical hold pending review and approval of the company’s proposed protocol for a 6-month, Phase I study to assess the potential relevance of adverse findings observed in long-term animal studies of metadoxine in relation to human exposure, Alcobra said.
MDX is an extended-release oral formulation of metadoxine (pyridoxol L-2-pyrrolidone-5-carboxylate), an inhibitor of the gamma-aminobutyric acid (GABA) transaminase enzyme responsible for the degradation of GABA. In addition to ADHD, Alcobra is developing MDX as a treatment for Fragile X syndrome and cognitive impairment.
Investors responded to the failure announcement with a stock sell-off that sent the price of Alcobra shares tumbling nearly 52% in premarket trading as of 8:55 a.m., to 92 cents from the Friday close of $1.90.