The Essential 3 Study 2 was one of two studies conducted in parallel through Praxis Pharmaceuticals. And what was interesting about these studies is that they were a decentralized study, meaning that there was no brick-and-mortar site for the patients. The patients were seen in their home with a study nurse that went out to the house and then evaluated by investigators through a telemedicine visit at the same time as the nurse being there...
The Essential 3 Study 2 was one of two studies conducted in parallel through Praxis Pharmaceuticals. And what was interesting about these studies is that they were a decentralized study, meaning that there was no brick-and-mortar site for the patients. The patients were seen in their home with a study nurse that went out to the house and then evaluated by investigators through a telemedicine visit at the same time as the nurse being there. It was unique in that we had participants from all over the country, all 50 states, and we had about 200,000 patients self-register out of interest for the study, which goes to show the need for treatment for essential tremor in the United States. And of those 200,000 patients, through inclusion criteria and rigorous evaluation, they were whittled down to about 698 patients. Those 698 patients were then consented to both studies. They didn’t know if they were going to be enrolled in study one or study two. They were randomized in a two-to-one fashion. And then in study one, it was your typical study. They had a placebo and drug, and they were just followed over the course of 14 weeks. Study two was the same study essentially from the first eight weeks, except everyone received the drug. And then at the eight-week mark, they were randomized in either continuing on study drug or withdrawing from study drug. And the main focus was looking to see if their symptoms returned as the study drug was removed. And the symptoms did, so it met its endpoint. I think it has a huge potential for impact. And the reason is, in the United States, we only have one FDA-approved medication for essential tremor, and it’s not even a neurological medication. It’s borrowed from cardiology. It’s a beta blocker called propranolol. And we do treat with other things, but all of those other things are borrowed from other aspects of neurology. There’s anti-seizure medicines and anti-headache medicines and neuropathic pain medicines that we try to use for essential tremor treatment. And unfortunately, patients don’t stick with our current treatments available because efficacy is lacking and tolerability is also a problem. Side effects of, you know, making people feel dizzy, lightheaded, cognitively, you know, medicine heady sort of a thing. So this is the first drug that is really looking at the pathophysiology behind essential tremor. And it’s focusing centrally in the brain on the circuit that drives tremor. And it’s blocking the receptors in the brain that drive the tremor, hoping to sort of reset the circuit so that there’s less tremor and smoother movement wherever the tremor happens to be on the body, head, arms, legs, neck, that sort of thing. So since there’s nothing readily available, to have something that is pharmacologically mechanistically appropriate that has been shown to be effective is very exciting. It’s also been shown to be well-tolerated in the studies, so it has the potential for very strong clinical utility.
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