atai Life Sciences (NASDAQ: ATAI), a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders, reported fourth quarter and full year 2022 financial results and provided clinical pipeline highlights.

“With multiple clinical-stage programs aimed at addressing significant unmet patient needs in mental health and the capital to fund us into the first half of 2026, we believe we are in a strong position to advance our clinical candidates towards proof of concept in patients. Recently we announced dosing the first patient in our Phase 2 study of RL-007 in CIAS and the modification of the GRX-917 clinical development plan to advance directly into a proof-of-concept study in patients with anxiety.”

Florian Brand, CEO and Co-Founder of atai

Clinical Pipeline Highlights

RL-007 (Pro-Cognitive Neuromodulator for Cognitive Impairment Associated with Schizophrenia (CIAS))

  • The first patient was recently dosed in the phase 2b study of RL-007 in patients with CIAS and topline results from this study are expected in the 2nd half of 2024.
  • The phase 2b US-based study is a randomized, placebo-controlled, double-blind, 3-arm study evaluating 20mg and 40mg of RL-007 vs placebo in approximately 230 patients with CIAS. The primary endpoint of the study is the change from baseline in the MATRICS Consensus Cognitive Battery (MCCB) neurocognitive composite score, a well-established regulatory endpoint, at week 6.
  • RL-007 is an orally available compound that modulates cholinergic, glutamatergic and GABA-B receptors, thereby putatively altering the excitatory/inhibitory balance in the brain to produce pro-cognitive effects. It has previously been evaluated in 10 clinical studies with over 500 unique participants dosed to-date and in which it was well tolerated at all doses tested.
  • Notably, in four clinical studies that assessed cognition, including one in patients with CIAS, the compound consistently demonstrated pro-cognitive effects. In atai’s previous Phase 2a proof-of-mechanism study in CIAS of RL-007, the compound showed a large effect size on Symbol Coding, a sub-component of the MCCB that correlates with the overall composite.

GRX-917 (Deuterated Etifoxine for Anxiety Disorders)

  • The clinical development plan was modified to proceed with a phase 2 study in patients, which is intended to generate the robust clinical data needed to best inform a future registrational program. More details of the phase 2 clinical development plan will be provided upon initiation of the study.
  • In January 2022, positive results were announced from the phase 1 single and multiple ascending dose study of GRX-917. In this study, GRX-917 was well-tolerated. Additionally, GRX-917 had an improved pharmacokinetic profile relative to etifoxine and provided pharmacodynamic evidence of GABA receptor target engagement through qEEG.
  • GRX-917 is a deuterated version of etifoxine, a drug used for anxiety and first approved in France in 1979. Etifoxine has a rapid onset and efficacy comparable to benzodiazepines, which are currently considered standard of care. In contrast to benzodiazepines, etifoxine appears to be non-addictive, less sedating, and better tolerated. It is believed that etifoxine achieves its anxiolytic activity by increasing the endogenous production of brain neurosteroids like allopregnanolone.

COMP360 (Psilocybin Therapy for Treatment-Resistant Depression (TRD))

  • COMPASS Pathways (“COMPASS”) recently announced an acceleration of the Pivotal Trial 1 (COMP 005) part of the phase 3 program in TRD, with top line data from Pivotal Trial 1 now expected in the summer 2024.
  • The on-going phase 3 program is composed of two pivotal trials, each of which will have a long-term follow-up component. The primary endpoint in both pivotal trials is the change from baseline in MADRS total score at week 6.
  • COMP360 is a proprietary formulation of synthetic psilocybin that is administered in conjunction with psychological support. Previously, COMPASS completed a phase 2b study with top line data showing a statistically significant (p<0.001) and clinically relevant improvement in depressive symptom severity after three weeks for patients who received a single high dose of COMP360 psilocybin with psychological support.

VLS-01 (N,N-dimethyltryptamine (DMT) for TRD)

  • In the fourth quarter of 2022, the first subject was dosed in a Phase 1 study of VLS-01. Initial results from the study are expected in the 1st half of 2023.
  • The phase 1 study is a two-part single-ascending dose study designed to evaluate the safety, tolerability and relative bioavailability of oral transmucosal film (OTF) versus intravenous (IV) formulations of VLS-01.
  • The study includes atai’s IDEA-1 companion digital therapeutic for psychological support to be used in combination with VLS-01. The app-based support comprises “(mind)set-and-setting” prior to dosing and limited post-dose integration appropriate for healthy volunteers.
  • VLS-01 is an OTF formulation of DMT. DMT is a partial agonist of the 5-HT 1A/2A/2C receptors, characterized by an intrinsically short duration of psychedelic effect, with a serum half-life estimated at less than 10 minutes. DMT results in rapid-acting antidepressant effects when administered via IV infusion in patients with major depressive disorder (MDD). VLS-01 is formulated for oral delivery, potentially eliminating the need for IV infusion.

DMX-1002 (Ibogaine for Opioid Use Disorder)

  • In the third quarter of 2022, the first subject was dosed in a phase 1/2 study of Ibogaine. Initial results from the Phase 1 portion of the study are expected in the 1st half of 2023.

EMP-01 (MDMA Derivative for PTSD)

  • In the third quarter of 2022, a phase 1 study of EMP-01 was initiated. Initial results from this study are expected in the 2nd half of 2023.

Consolidated Financial Results

Cash, Cash Equivalents, and Short-term investments: Cash and cash equivalents and short-term investments were $273.1 million as of December 31, 2022, as compared to $362.3 million as of December 31, 2021. The decrease of $89.2 million was primarily driven by net cash used in operating activities of $104.5 million and $3.6 million additional investment in platform companies, partially offset by $15.0 million draw on the Hercules debt, $4.6 million of proceeds from the conversion of notes and $2.9 million of proceeds from stock sales and stock option exercises. The Company expects its cash position, combined with access to up to $160M in additional capital from its term loan facility with Hercules Capital, Inc., will be sufficient to fund operations into 1H 2026.

Research and development (R&D) expenses: R&D expenses were $21.9 million and $74.3 million for the three and twelve months ended December 31, 2022, respectively, as compared to $13.0 million and $48.0 million for the same prior year periods. The year-over-year full-year increase of $26.3 million was primarily attributable to an increase of $22.2 million of contract research organization expenses related to advancements of R&D programs and $3.5 million increase in R&D personnel costs.

General and administrative (G&A) expenses: G&A expenses for the three and twelve months ended December 31, 2022 were $15.7 million and $70.4 million, respectively, as compared to $25.9 million and $92.7 million in the same prior year periods. The year-over-year decrease of $22.3 million was primarily attributable to a decrease of $18.0 million in non-cash stock compensation expense, $9.8 million decrease in value added tax expense, $4.0 million decrease in professional consulting services. These decreases were partially offset by an increase of $7.3 million in personnel expenses and $1.9 million increase in insurance expense.

Net loss: Net loss attributable to shareholders for the three and twelve months ended December 31, 2022 was $45.0 million and $152.4 million, respectively, as compared to $88.9 million and $167.8 million for the comparable prior year periods.

About atai Life Sciences

atai Life Sciences is a clinical-stage biopharmaceutical company aiming to transform the treatment of mental health disorders. Founded in 2018 as a response to the significant unmet need and lack of innovation in the mental health treatment landscape, atai is dedicated to acquiring, incubating, and efficiently developing innovative therapeutics to treat depression, anxiety, addiction, and other mental health disorders.

By pooling resources and best practices, atai aims to responsibly accelerate the development of new medicines across its companies to achieve clinically meaningful and sustained behavioral change in mental health patients.

atai’s vision is to heal mental health disorders so that everyone, everywhere can live a more fulfilled life.