Ovid Pharmaceutics announced their novel delta (δ)-selective GABAA receptor agonist, gaboxadol (OV101), was shown to have a significant improvement in behaviour of young adults with Fragile X syndrome.

Fragile X syndrome is an inherited form of intellectual disability and autism that affects 1 in 3,600 – 4,000 males and 1 in 4,000 – 6,000 females. Common symptoms may include cognitive impairment, anxiety, mood swings, hyperactivity, attention deficit, poor sleep, self-injury and heightened sensitivity to various stimuli, such as sound.

Persons with Fragile X syndrome are also susceptible to comorbid medical issues including seizures and sleep disturbance. Fragile X syndrome is due to mutations in the FMR1 gene that disrupts the expression of the Fragile X Mental Retardation Protein (FMRP). FMRP is associated with the synthesis of GABA.  Gaboxadol is a novel GABA-A agonist.

The 12-week Phase 2 trial, known as the ROCKET trial, was a randomized, double-blind, parallel-group trial that evaluated the safety and efficacy of gaboxadol in young men (13 – 22 years; N=23). The primary outcome measure was safety and tolerability while a key secondary outcome measure was efficacy, as measured by various behavioural tests [e.g., Aberrant Behavior Checklist-Community for Fragile X syndrome (ABC-CFXS); Anxiety, Depression and Mood Scale (ADAMS); Clinical Global Impressions Severity scale (CGI-S)]. Three doses were assessed in the trial; 5 mg once-daily (QD), 5 mg twice-daily (BID), and 5 mg three-times-daily (TID).

At the end of the 12-week study, the primary endpoint was met and the drug was well tolerated with no serious adverse events reported across all three dose cohorts. The most common adverse events were  diarrhea (9%), irritability (9%), headaches (13%), and upper respiratory infections (18%). Regarding efficacy, gaboxadoldemonstrated a statistically significant effect on secondary behavioral endpoints in the three combined study groups as follows: 26.2% mean improvement in the ABC-CFXS total score (P =.002), a 21.6% mean improvement in the ADAMS total score (P =.004), and a mean reduction of 0.4 in the CGI-S total score (P=.002).

More detailed efficacy data are provided in the tables below.

Table 1: Mean ABC-CFXS total scores

Baseline Week 12 % Improvement

(p value)

Study Population 65.7


52.3 26.2%

(P = .0017)

QD 80.5
60.5 21.7%

(P = .1763)

BID 60.0
52.6 24.5%

(P = .0402)

TID 62.1
46.7 30.7%

(P = .0402)


Table 2: Mean ADAMS scores

Baseline Week 12 % Change (p value)
Study Population 24.7


18.3 -21.6%

(P = ..0039)

QD 28.4
20.7 -22.6%

(P = .0897)

BID 20.1
15.4 -18.9%

(P = .1000)

TID 26.1
19.0 -23.3%

(P = .1341)


In a news release, Elizabeth Berry-Kravis, MD, Professor of Pediatrics and Neurological Sciences and Co-Director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush University Medical Center said, “The data from the ROCKET trial demonstrate that OV101 may have a meaningful effect on improving the lives of some individuals living with Fragile X syndrome.”  Dr. Berry-Kravis added, “Fragile X is a challenging neurodevelopmental genetic condition, with complex symptomatology that significantly impacts patients and their families. I am encouraged by the safety results of this Phase 2 study of OV101 and am excited to further investigate its therapeutic potential in patients living with Fragile X syndrome.”in baseline population due to patients who discontinue later in the

Based on the results of the Phase 2 study, the developers of the drug, plans to request meetings with regulatory authorities to discuss the development path and registration pathway for gaboxadol (OV101) for the treatment of Fragile X syndrome.


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