Helus Pharma (HELP) Status Update summary

Clinical program updates

• 12-month Phase 2 data for CYB003 in MDD showed a 100% response rate and 71% remission at the 16mg dose, with a mean MADRS score reduction of 23 points.

• 60% response and 50% remission were observed at 12 months for two 12mg doses.

• CYB003 is administered as adjunctive therapy, allowing patients to remain on existing antidepressants.

• CYB004, targeting generalized anxiety disorder, is in Phase 2 with top-line data expected in Q1 2025.

• Phase 3 Paradigm program for CYB003 in MDD has been initiated, including two pivotal trials and a long-term extension, with top-line results expected in 2026.

Safety and tolerability

• CYB003 demonstrated a favorable safety profile, with only mild, transient adverse events such as headache, nausea, and dizziness, resolving on dosing day.

• No long-term adverse effects or suicidality were observed at 12 months.

• CYB003 continues to be well-tolerated in clinical studies.

• Treatment experience at 16mg was described as intense but not unpleasant.

Market and regulatory positioning

• CYB003 received FDA Breakthrough Therapy Designation for MDD, expediting development and review.

• The target indications, MDD and GAD, represent large, underserved markets with high unmet need.

• Engagement with the FDA has ensured Phase 3 trial designs align with regulatory guidance, including measures to address functional unblinding and expectancy bias.

• The company is well-capitalized with CAD 154 million in cash as of September 30, 2024, sufficient to fund Phase 3 trials.