- In a 62-week, phase II trial, MDX was tested for treatment ADHD associated with fragile X syndrome, a genetic disease linked with intellectual disabilities and behavioral problems, and failed to meet primary endpoints.
- In the fragile X trial, placebo outperformed MDX in terms of meeting primary endpoints.
- In a previous phase III trial focused on treatment of ADHD, MDX also failed to meet primary endpoints.
Even in the face of spectacular failure, Alcobra's executives are determined to move this drug forward through trials. The company has cited the success of MDX in meeting two tertiary endpoints in the phase II trials, as well as demonstrating efficacy in other areas of improving cognition, specifically performance on the Vineland Adaptive Behavioral Scale. Alcobra claims that the results of the trial are distorted because patients in the placebo arm may have been taking other ADHD medications, and also because patients with fragile X syndrome have varying degrees of intellectual ability.
Even in the area of creative trial design, Alcobra is pushing the envelope and relying on strenuous analysis to make MDX a contender for approval. However, the market is sending a different signal. The stock was down 20% on the news yesterday, and is currently trading at $7.13 as of the open of the market---down roughly 2.3%.