Highland Therapeutics subsidiary Ironshore Pharmaceuticals & Development (Ironshore) has found positive clinical data from the first of two phase III trials of its investigational drug, Benjorna, which is a delayed-release and extended-release methylphenidate capsule.
Formerly known as HLD-200, Benjorna is currently under development as a potential new option for physicians treating patients with attention deficit hyperactivity disorder (ADHD).
The clinical study (HLD200-107) started in July 2015 and involved 153 pediatric patients. Those who were randomised to receive Benjorna showed a statistical improvement compared with those patients who received placebo (p=0.01), based on a composite measure from 8am through to 8pm on the Swanson, Kotkin, Agler, M-Flynn and Pelham (SKAMP) rating scale, the study's primary endpoint.
Patients randomised to the Benjorna treatment also showed better functioning during the morning routine as measured by the parent rating of evening and morning behaviour-revised (PREMB-R) morning subscale, which was the study's key secondary endpoint.
Benjorna's potential to enhance its functioning during the morning routine will be evaluated more in a second study (HLD200-108), initiated last August and currently ongoing with results expected in the second quarter of this year.
In the HLD200-107 trial, the Benjorna treatment group also indicated statistically significant improvement, compared with placebo, in functioning in the evening as measured by the PREMB-R evening (PM) subscale (p<0.001).
The extended duration of clinical effect was earlier observed in a phase III study, conducted in 2014, where the patients receiving evening-dosed Benjorna showed improved functioning through to 8pm the following day, the last time point measured.
Ironshore research and development executive vice-president Bev Incledon said: "The positive results from this pivotal trial are just the latest evidence that the company has taken another step towards its stated goal of bringing to patients and physicians a new medicine that, if approved by the FDA, could become the standard of care for the treatment of ADHD.
"We currently believe that there is something unique happening at the dopamine neurotransmission level that may be attributed to a unique absorption profile, which will be the subject of an upcoming study led by a team of thought leaders.
"This work will be important for the medical community to consider and will surely deepen our appreciation of the potentially critical role that pharmacokinetics have on the effectiveness of stimulant medications."
The company aims to advance two new drug candidates into trials in the second half of this year. HLD-900 is an amphetamine-based product for the treatment of binge eating disorder (BED), which was the subject of an end of phase 2 (EOP2) meeting with the US Food and Drug Administration in February, in addition to HLD-100, which is under development as an amphetamine-based treatment for patients with ADHD.