Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) saw its shares rise in pre-market deals after the group unveiled "compelling" top-line results from a Phase 2 clinical trial of its Zygel product to treat autism spectrum disorder (ASD).
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Autism Spectrum Disorder (ASD) affects communication and behavior in around 1 million children and adolescent patients aged between five and 17 in the USA.
In the study named BRIGHT, Zygel was given to 37 patients aged between 3 and 17 with moderate-to-severe symptoms of ASD as an add-on therapy to their standard of care using a variety of efficacy assessments.
"We are very encouraged by the compelling top-line results of the BRIGHT trial and we expect to meet with the FDA to discuss the clinical pathway for developing Zygel for the treatment of behavioral symptoms of ASD in the second half of this year,” Armando Anido, Zynerba’s chairman and CEO said in a statement.
"I am very impressed with the improvements my patients made over the 14-week treatment period while receiving Zygel; the reduction in irritability, communication deficits, and repetitive movements were especially noteworthy since some of these are core autistic behaviors," added Helen Heussler, the principal investigator in the BRIGHT trial.
“The magnitude of effect on autistic behaviors in this trial is significant, including hyperactivity and stereotypy, which are among the most difficult behaviors to improve with therapeutic intervention. The results of this study strongly suggest the potential of this drug as an important treatment for ASD and I look forward to participating in future clinical studies with Zygel.”
Key findings of the trial, said the firm, included that Zygel was well tolerated, with no serious or severe adverse events.
In addition, "all five subscales of the Aberrant Behavior Checklist – Community (ABC-C) as well as the Parent Rated Anxiety Scale - Autism Spectrum Disorder (PRAS-ASD) showed both statistically significant and clinically meaningful improvements at 14 weeks of treatment from baseline," the pharma firm added.
Zynerba is also working on developing Zygel for patients with Fragile X syndrome, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies.
Zynerba shares in pre-market trade in New York added 7.4% to $6.65.
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