Helus Pharma (HELP) investor relations material

Conference highlights

• Over 25 companies presented at the summit, with sessions available on demand and a full day of panels and fireside chats focused on CNS drug development, including depression, epilepsy, and BBB delivery.

• Key opinion leaders participated in discussions on advances and challenges in neuropsychiatric drug development.

Pipeline and clinical program updates

• Two clinical-stage programs: HLP003 for major depressive disorder (MDD) in phase III with FDA breakthrough therapy designation, and HLP004 for generalized anxiety disorder (GAD).

• Both programs use deuterated analogs of naturally occurring compounds, aiming for improved properties and strong intellectual property protection through at least 2041.

• HLP003 phase III PARADIGM program includes APPROACH, EMBRACE, and EXTEND trials, focusing on efficacy, durability, and safety in adjunctive MDD treatment.

• APPROACH and EMBRACE are double-blind, placebo-controlled studies, with top-line data for APPROACH expected by year-end.

Clinical trial design and regulatory strategy

• Trials address functional unblinding and expectancy effects using FDA guidance, remote blinded raters, and safeguards to maintain study integrity.

• Efficacy bar for APPROACH is set by phase II results, with even half the effect size considered class-leading for adjunctive MDD.

• Durability of response and safety, including cardiovascular, psychological, and drug-drug interaction data, are key endpoints.

• Regular FDA engagement under breakthrough therapy designation supports expedited development and rolling review, targeting NDA submission in 2028.