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Positive pivotal ADHD trial results announced for its investigational drug candidate Benjornatm

Highland Therapeutics Inc.’s wholly owned subsidiary, Ironshore Pharmaceuticals & Development, Inc., has announced positive clinical data from the first of two Phase III pivotal trials of its investigational drug product, BenjornaTM (delayed-release and extended-release methylphenidate capsules). BenjornaTM, formerly referred to as HLD-200, is under development as a potential new option for physicians treating patients with Attention Deficit Hyperactivity Disorder (ADHD). In the clinical study (HLD200- 107), which included 153 pediatric patients, those who were randomized to receive BenjornaTM demonstrated a statistically significant improvement compared to 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 randomized to the BenjornaTM treatment arm also demonstrated improved functioning during the morning routine as measured by the PREMB-R (Parent Rating of Evening and Morning Behavior-Revised) morning (AM) subscale, which was the study’s key secondary endpoint (p<0.001). The potential for BenjornaTM to improve functioning during the morning routine will be evaluated further in a second pivotal study (HLD200-108), which is currently ongoing with results expected in the second quarter of 2016.

In the HLD200-107 study, the BenjornaTM treatment group also achieved 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 previously observed in a prior Phase III study, conducted in 2014, in which patients receiving evening-dosed BenjornaTM showed improved functioning through to 8pm the following day, the last time point measured.

Commenting on the HLD200-107 results, Dr. Bev Incledon, Ironshore’s Executive Vice President of Research and Development 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.”

Dr. Randy Sallee, Chief Medical Officer stated, “As a practicing child and adolescent psychiatrist for more than 35 years, I believe the most impressive result from the clinical trial is the robust and consistent effect reported across the entire day; from wakening, through the academic day and into the evening period. Importantly, in the HLD200-107 study, there was no difference in the number of sleep-related side effects between the BenjornaTM and placebo groups.”

David Lickrish, President and Chief Executive Officer stated, “The last 12 months have been transformational for Highland, which is emerging as a fully integrated pharmaceutical company and a future leader in neuroscience, including ADHD. With these positive results in hand, we will not only continue to invest in our business as we become more vertically integrated, but we will accelerate that pace of activity so that the requisite teams are in place to support the launch of this product upon FDA approval. In addition to these milestones, we expect to make several other near-term announcements regarding our rapidly expanding pipeline.”

Pipeline Developments

Ironshore intends to advance two new drug candidates into pivotal trials in the second half of 2016: HLD-900, an amphetamine-based product for the treatment of Binge Eating Disorder (BED) which was the subject of an End of Phase 2 (EOP2) meeting held with the U.S. Food and Drug Administration in February 2016, and HLD-100, which is under development as an amphetamine-based treatment for patients with ADHD.

About the HLD200-107 Study

Initiated in July 2015, the HLD200-107 study was a Phase III, multicenter, open-label, treatment-optimized, double-blind, randomized, placebo-controlled, forced-withdrawal, parallel group study to evaluate the safety and efficacy of evening-dosed BenjornaTM in children aged 6- 12 with ADHD in a laboratory classroom setting. A total of 153 patients were randomized across seven sites in the US.

About the HLD200-108 Study

Initiated in August 2015, trial HLD200-108 is a Phase III, multicenter, double-blind, randomized, placebo-controlled, parallel group study to evaluate the safety and efficacy of evening-dosed BenjornaTM on post-waking, early morning function in children aged 6-12 with ADHD. The study will randomize approximately 150 patients at 22 centers across the US.