On to Phase 3 for ADHD clinical trial

Alcobra’s MDX meets primary endpoint in Phase 2 trial in adolescents with attention deficit hyperactivity disorder

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TEL AVIV—Alcobra Ltd., an emerging pharmaceutical company focused on the development of new medications to help patients with cognitive disorders, including attention deficit hyperactivity disorder (ADHD) and fragile X syndrome, has announced that its Phase 2 safety and tolerability study of a single administration of MDX in adolescent patients with ADHD achieved its primary endpoint. In the study, MDX showed good tolerability and no safety concerns were identified. The profile of adverse events in the MDX treated group was not different from the placebo comparator group. There were no reported cases of discontinuation from the study due to adverse events.
A total of 83 patients enrolled in the Phase 2 trial, which was a multicenter, randomized, double-blind, placebo-controlled, fixed-dose study designed to evaluate the safety and tolerability of a single administration of MDX in adolescents (aged 13 to 17) with predominantly inattentive ADHD. Following a screening period, patients were exposed to either placebo or one of three MDX dose options determined by their weight and followed for several hours that day. Patients returned several days after this treatment visit for a safety follow-up visit.
Patients’ baseline characteristics were similar between MDX and placebo groups. The most common side effects were headache (8 percent of patients in the MDX group and 12 percent in the placebo group), nausea (3 percent vs. 5 percent) and fatigue (3 percent vs. 5 percent). No clinically significant safety findings in laboratory values, vital sign measurements, ECG parameters, cardiovascular parameters or findings during clinical examination were observed. Analyses of secondary cognitive measures, after a single administration of MDX compared to placebo, did not produce statistically significant findings, yet resulted in an efficacy signal on the Test of Variables of Attention (TOVA) assessments Response Time and Errors of Omission.
“The successful completion of this safety study is an important milestone in the development of MDX and supports our ability to advance into efficacy trials of MDX for pediatric ADHD,” said Dr. Yaron Daniely, president and CEO of Alcobra. “I would like to thank all the patients who participated in the trial and their parents, as well as our investigators who quickly and rigorously conducted this trial over the last few months.”
The study is the first clinical trial using MDX in a pediatric ADHD population. A separate study evaluating MDX in adolescents and adults with fragile X syndrome is currently underway and actively enrolling patients. Results of the fragile X study are expected to be reported by the end of the second quarter of this year.
MDX (Metadoxine Extended Release [MG01CI]) is a proprietary investigational new drug candidate being developed by Alcobra for the potential treatment of ADHD and fragile X syndrome. MDX is not a stimulant and acts as a monoamine-independent modulator of GABA (gamma-aminobutyric acid) transmission. This novel mechanism of action does not directly affect dopamine or norepinephrine. In studies to date, metadoxine has shown no potential for abuse or addiction. MDX is currently in Phase 3 development for adults with ADHD and Phase 2 development for pediatric ADHD. A study of MDX is also ongoing in adolescents and adults with fragile X syndrome.
ADHD is a common and impairing neuropsychiatric condition. Once believed to only affect children, ADHD is now known to persist into adolescence and adulthood in a sizeable number of cases. Key symptoms of ADHD include inattention, hyperactivity and impulsivity. According to the Centers for Disease Control and Prevention, about 9 percent of children in the United States meet criteria for ADHD with similar numbers reported in other countries. Although boys are more commonly diagnosed, ADHD is also common in girls, who often go undiagnosed. Approximately 4 to 5 percent of adults worldwide are affected with ADHD, yet most adults with ADHD remain undiagnosed and untreated. There is no known cause of ADHD, however studies suggest that genetics may play a role.
Alcobra Ltd. was founded in 2008 and is a relative newcomer to the ADHD space, which is dominated by Shire, with strong product entries from Johnson & Johnson, according to an analysis by The Motley Fool, noting that Alcobra is focused on the nonstimulant ADHD drug market and that its experimental drug MDX is a nonstimulant treatment being tested for predominantly inattentive ADHD, which accounts for 40 percent of all ADHD cases, and fragile X syndrome, a genetic syndrome that causes autism in boys. MDX was shown to be effective and well tolerated in two Phase 2 studies of adults with ADHD. It has begun enrolling patients in a Phase 3 trial, with completion expected in the second half of this year, The Motley Fool notes.
“Certainly, Alcobra is an interesting stock to watch,” the analyst firm states. “Nonetheless, this is a small, clinical-stage biotech, so an investment here is comparatively risky.”

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