THX-110

Research and Development

THX-110: A Proprietary Combination of THC and PEA

THX-110 is a drug candi­date platform for the treat­ment of symptoms related to Tourette syndrome (TS), as well as Obstructive Sleep Apnea (OSA) and chronic pain.

Tourette Syndrome

TS is a neuropsy­chi­atric disorder starting in child­hood. Symptoms include multiple physical and at least one vocal tic that can be disabling and associ­ated with hyper­ac­tivity, anger control problems, sleep disor­ders, and obsessive-compulsive behavior. Some of the most dramatic and disabling tics include motor movements that result in self-harm or vocal tics including copro­lalia or echolalia.  Current pharma­co­log­ical treat­ments have provided very limited efficacy and are almost always accom­pa­nied by severe side effects. To date only 3 drugs are approved by the FDA for the treat­ment of TS patients. However, exposure to these drugs is associ­ated to severe side effects, which may limit their toler­a­bility and use in many TS patients.

Therapix completed an Investigator Initiated Phase IIa study at Yale University, suggesting that THX-110, which is a combi­na­tion of THC and PEA, signif­i­cantly improved symptoms over time in adult Subjects with Tourette syndrome.

The study was a single-arm, open-label trial, in which each subject both received one daily treat­ment of the drug via oral admin­is­tra­tion and was followed-up for a period of 12 weeks. Sixteen subjects partic­i­pated in the study and received THX-110 at the Yale University Child Study Center at Yale University, USA. The primary endpoint of the study was to assess the perfor­mance of THX-110 in the treat­ment of adult patients suffering from symptoms of Tourette syndrome, as measured by the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS), the gold-standard and customary index for assessing symptom severity. Treatment was given in a dose titra­tion regimen with a maximum dose of THX-110 consisting of 10mg Dronabinol and 800mg PEA.

The study showed that these 16 subjects with medication-refractory TS had an average tic reduc­tion of 21% across the entire sample. Six of the 16 medication-refractory TS subjects experi­enced a response to treat­ment as defined by a reduc­tion in YGTSS-TTS of greater than 25%. The medica­tion was gener­ally well-tolerated.

The study was investigator-initiated and led by Michael H. Bloch, M.D., Associate Professor at the Yale University Child Study Center, and James F. Leckman, M.D., Ph.D., of the Neison Harris Professor in the Child Study Center and Professor of Pediatrics Child Psychiatry at Yale University.

Therapix is initi­ating a Phase IIb clinical trial in two centers in Germany, Hanover and Munich, which will evaluate the safety and efficacy of THX-110 for the treat­ment of symptoms of Tourette syndrome. This study is intended to be double-blind, random­ized, and placebo controlled.

Therapix is seeking orphan drug desig­na­tion for this compound.

Obstructive Sleep Apnea

OSA is a poten­tially serious sleep disorder.  It causes breathing to repeat­edly stop and start during sleep.  There are several types of sleep apnea, but the most common is obstruc­tive sleep apnea.  This type of apnea occurs when the throat muscles inter­mit­tently relax and block the airway during sleep.  A notice­able sign of obstruc­tive sleep apnea is snoring. OSA affects 29.4 million American men and women, which repre­sents 12 percent of the U.S. adult popula­tion. There are currently no approved pharma­ceu­tical approaches. The standard treat­ments for obstruc­tive sleep apnea (OSA) are nonin­va­sive venti­la­tion devices (CPAP) and oral appli­ances that are very incon­ve­nient to the patient, thus result in low compli­ance.

Therapix initi­ated a Phase IIa sponsor-initiated clinical trial using the Company’s cannabinoid-based technology THX-110 for Obstructive Sleep Apnea (OSA), at Assuta Medical Center, the largest private hospital network in Israel.

The clinical trial is a single-arm, open-label trial, in which 30 subjects receive one daily treat­ment of the drug via oral admin­is­tra­tion and is followed-up for one month with the primary efficacy endpoint evalu­ating a signif­i­cant change in the AHI (apnea-hypopnea index – which refers to the severity of sleep apnea as measured during a sleep test), which assesses the quality of sleep before and after treat­ment. The clinical trial is designed to prove the safety, toler­ance and efficacy of the drug’s treat­ment, and to assess its perfor­mance in adult patients suffering from symptoms of Obstructive Sleep Apnea.

Chronic Pain

Pain is a distressing feeling often caused by intense or damaging stimuli. Pain is the primary reason why people seek medical care; more than 40% of the US popula­tion is affected by chronic pain. There are two main classi­fi­ca­tions of pain: the common sensical sort that arises from damaged tissue (nocicep­tive pain), and the more exotic kind that comes from damage to the system that reports and inter­prets damage, the nervous system (neuro­pathic pain). Pain can also be classi­fied as acute or chronic. Acute pain is provoked by a specific disease or injury, is associ­ated with skeletal muscle spasm and sympa­thetic nervous system activa­tion. Chronic pain, in contrast, may be consid­ered a disease state. It is pain that outlasts the normal time of healing, if associ­ated with a disease or injury. When treating moderate to severe pain, the type of the pain, acute or chronic, needs to be consid­ered. The type of pain can result in different medica­tions being prescribed.

The Phase IIa Clinical Trial titled “Examining the Efficacy of THX-110 [Dronabinol (synthetic Δ9-tetrahydracannabinol) and Palmitoylethanolamide (PEA)] for Chronic Low Back Pain” is a single-arm, open-label trial, in which each subject receives one daily treat­ment of the drug via oral admin­is­tra­tion and is followed-up for a period of 6 weeks with an additional 2 weeks follow up after the last study treat­ment. Approximately 20 subjects are expected to partic­i­pate and receive the drug in the context of the Clinical Trial. The primary endpoint of the Clinical Trial is to prove the safety, toler­ance and efficacy of THX-110 in the treat­ment of adult patients suffering from chronic low back pain, with efficacy measured by the improve­ment of quality of life outcomes.