New Phase 2 Signal Detection Results: HLP004 for Moderate-to-Severe Generalized Anxiety Disorder

Helus Pharma today announced topline results from a phase 2 signal detection study evaluating HLP004 as a potential treatment for adults with moderate-to-severe generalized anxiety disorder (GAD) who remained symptomatic despite ongoing standard of care antidepressant therapy, including selective serotonin reuptake inhibitors and related agents.¹

As no adjunctive pharmacologic treatments for GAD have ever been approved, and the last new monotherapy was approved almost 2 decades ago, any new treatment in this area has potential to transform the GAD treatment space.

In the phase 2 signal detection study, 36 participants were randomized 2-to-1 active-to-placebo to HLP004 20 mg or 2 mg and received 2 intramuscular doses 3 weeks apart. Participants were followed through week 12, with continued observational follow-up extending up to 1 year. Participants had an average baseline Hamilton Anxiety Rating Scale (HAM-A) score of 22 and a General Anxiety Disorder-7 score of greater than or equal to 10 at screening. All study participants were already being treated, and continued treatment throughout the trial, with standard of care medications for generalized anxiety disorder. The 10-point improvement in anxiety symptoms is above and beyond what was already being seen with standard of care treatment.

Patients that received 20 mg HLP004 adjunctive to standard of care therapy achieved mean reduction of 10.4-points (P<0.0001) in the HAM-A from baseline at 6 weeks. Participants randomized to both 20 mg and 2 mg dosing arms experienced meaningful subjective effects and showed clinically significant responses over standard of care, with 59% meeting the criteria for response and 32% for remission in the 20 mg arm and a 30% responder and remitter rate in the 2 mg arm at week 6. At 6 months, the pooled study population showed 67% responders and 39% remitters. Participants also experienced short in-clinic treatment time with acute drug effects lasting approximately 90 minutes and discharge readiness within approximately 3 hours. HLP004 also has a favorable tolerability profile with no drug-related serious adverse events or suicidality-related safety signals.

“These phase 2 results support the continued development of HLP004, and I am encouraged by the magnitude of improvement observed over standard of care treatments, together with the rapid onset and short in-clinic treatment experience for this patient population with limited options,” said Andrew J. Cutler, MD, a clinical professor of psychiatry at SUNY Upstate Medical University and senior advisor to Helus Pharma.

“Patients living with generalized anxiety disorder remain significantly underserved, with many continuing to struggle despite currently available treatments. We are encouraged by these data and the potential for HLP004 to bring hope to GAD patients,” said Michael Cola, the chief executive officer of Helus Pharma. “The Company’s broad intellectual property portfolio has been leveraged once again to create what we feel are best in class products and we are further excited to release the data on HLP003 targeted at major depressive disorder in the fourth quarter of 2026.”

Helus Pharma also recently shared phase 2a results on SPL026 for the treatment of major depressive disorder, which met its primary endpoint, showing clinically significant reduction in depression symptoms. SPL026 is a form of intravenous dimethyltryptamine psychedelic, which showed antidepressant effects within a week of treatment, and sustained effects for up to 3 months.²

References

1. Helus Pharma announces topline results in phase 2 signal detection study for HLP004 in patients with generalized anxiety disorder. News release. March 5, 2026. Accessed March 5, 2026. https://www.globenewswire.com/news-release/2026/03/05/3250048/0/en/Helus-Pharma-Announces-Topline-Results-in-Phase-2-Signal-Detection-Study-for-HLP004-in-Patients-with-Generalized-Anxiety-Disorder.html

2. Walters J. Phase 2a trial meets endpoints for psychedelic SPL026 to treat major depressive disorder. Psychiatric Times. February 17, 2026. https://www.psychiatrictimes.com/view/phase-2a-trial-meets-endpoints-for-psychedelic-spl026-to-treat-major-depressive-disorder