From 'party drug' to PTSD treatment: ATAI launches EmpathBio to develop MDMA-based therapy

Treatment for post-traumatic stress disorder (PTSD) varies from patient to patient and can combine different kinds of therapy and drugs, including anti-anxiety meds and antidepressants. Problem is, the only drugs approved for PTSD don’t work for everyone, and even when they do, they don’t work perfectly and come with side effects.

EmpathBio, ATAI Life Sciences’ latest company, is looking to fill that gap with treatments based on 3,4-methylenedioxymethamphetamine, better known as the “party drug” MDMA. The hope is that MDMA derivatives will work better as add-ons for patients with PTSD who are undergoing psychotherapy than antidepressants, such as Zoloft and Paxil, do.

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MDMA is a psychedelic, but it's not a traditional one like N,N-dimethyltryptamine (DMT)—the active ingredient in ayahuasca—or psilocybin, which is found in magic mushrooms. Instead, it’s thought of as an entactogen, meaning it can reduce anxiety, evoke feelings of empathy and sympathy and promote a sense of psychological safety. This puts patients in a place where they can process the intrusive thoughts and memories that characterize PTSD.

“The idea is that this compound and compounds like it allow you to get some degree of comfort and also give you a better bond [with a therapist] to allow you to express these feelings and thus work through them,” said Srinivas Rao, M.D., Ph.D., chief scientific officer of ATAI Life Sciences.

Compare that approach with using with Zoloft and Paxil, which work by boosting levels of serotonin—sometimes called the “feel-good” hormone—in the brain. They can rein in the emotional ups and downs that come with PTSD, but they can also hinder patients’ progress when undergoing psychotherapy.

“When it comes to medically assisted therapy, they are not particularly great. They don’t really allow for opening up and facilitating a patient’s alliance with their therapist,” Rao said.

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The Multidisciplinary Association of Psychedelic Research (MAPS) is already testing the MDMA approach in PTSD. Phase 2 data published in June showed that after two months of MDMA-assisted psychotherapy, more than half of 100 patients no longer met the diagnostic criteria for PTSD. And just last week, the nonprofit announced it had raised $30 million to bankroll phase 3 studies and an FDA submission for MDMA-assisted therapy as a treatment for PTSD.

Unlike MAPS, which is running with MDMA itself, EmpathBio will develop derivatives of the compound with the goal of delivering its benefits without its downsides, such as increases in blood pressure or heart rate.

“There are other side effects associated with MDMA and those are very mild to moderate, including anxiety, dizziness, fatigue and headache. Jaw clenching is a big one,” Glenn Short, the early development lead at ATAI, said.

Creating a safer, more tolerable version of MDMA could open up the treatment to more patients, such as those who suffer from hypertension and could do without a drug that elevates blood pressure, he added.

Throw in a digital therapeutic that can help doctors monitor patients and tailor their treatment, and MDMA-assisted therapy could reach patients who live far away from treatment centers. That would be an improvement over MDMA-based approaches in development, which would require patients to stay overnight while they are undergoing treatment.

EmpathBio is in the lead selection stage of discovery and aims to move an MDMA derivative into human trials as quickly as possible.

“We are obviously a number of years behind MAPS—they’re in phase 3 and that’s OK. We view this as a second-generation program,” Rao said.