Pharmather preparing phase 3 FDA package for ketamine

Executive Summary

• Pharmather Holdings Ltd. is preparing a pre-phase 3 (Type B) FDA briefing package to finalize the design for a Phase 3 registrational study of ketamine for levodopa-induced dyskinesia in Parkinson's disease (LID-PD).
• The company is actively engaging with prospective pharmaceutical partners to evaluate collaboration structures for co-development and commercialization of the asset.
• The Phase 3 program leverages the 505(b)(2) regulatory pathway, building on existing clinical data showing significant reductions in dyskinesia severity and an FDA-reviewed Chemistry, Manufacturing, and Controls (CMC) foundation.

Key Details

• Regulatory Strategy: Submission of a pre-phase 3 (Type B) FDA meeting request and briefing package planned for Q4 2025 and Q1 2026 to secure alignment on the Phase 3 protocol.
• Study Design: The Phase 3 trial is designed as a single, well-controlled study under the 505(b)(2) pathway, leveraging existing clinical evidence and FDA-reviewed CMC data.
• Clinical Data (Phase 1/2):
  • 51% reduction in dyskinesias during infusion 2 (Unified Dyskinesia Rating Scale).
  • 49% reduction at three weeks.
  • 41% reduction at three months.
  • Ketamine was reported as generally safe and well-tolerated.
• Intellectual Property: Patent coverage extends through 2036 (U.S. Patent No. 11,426,366).
• Market Opportunity: Estimated U.S. treated market opportunity of $750 million to $2.2 billion for an effective LID-PD therapy.
• Milestones:
  • Q4 2025 / Q1 2026: Submit pre-phase 3 meeting request and briefing package; hold FDA meeting.
  • Q1 / Q2 2026: Finalize Phase 3 protocol (endpoints, inclusion/exclusion, statistical plan) based on FDA feedback.
  • H1 2026 Onward: Initiate Phase 3 activities, contingent on FDA alignment and securing partnering/financing.
• Efficacy Endpoints: Primary focus on dyskinesia severity and impact (Unified Dyskinesia Rating Scale); pain and mood assessed as exploratory domains.

Notable Quotes

• "We're moving from planning to execution -- securing FDA alignment for a phase 3 path while also advancing active discussions with multiple partners who recognize the potential for ketamine in LID-Parkinson's," said Fabio Chianelli, chief executive officer of Pharmather. "Our strategy builds on the 505(b)(2) pathway, strong clinical signals and an FDA-reviewed CMC foundation. We believe a well-structured partnership can accelerate timelines, optimize commercial reach and unlock meaningful value for shareholders."