top of page
Higher Medicine is developing transformative small molecule therapies to functionally cure Friedreich’s ataxia (FA) through a multipronged pipeline targeting iron-sulfur-cluster biogenesis. Leveraging a novel epigenetic discovery platform and clinically validated p38 MAPK inhibitors, the company is accelerating toward Phase 2 readiness with a strategic focus on rapid value inflection and long-term pipeline expansion.

Lead Program & Pipeline

  • Lead candidate: Clinically validated p38 MAP Kinase inhibitor targeting the root biochemical defect in FA

  • Rapid development path leveraging existing safety data for accelerated Phase 2 entry

  • Pipeline includes:

    • Ferroptosis inhibitors for additive therapeutic impact

    • Novel epigenetic platform to discover future targets and hit compounds

  • Global TAM: ~21,000 FA patients; potential $1B+ in peak revenue per program

Technology Differentiators

  • Targets Iron-Sulfur Cluster Biogenesis (ISCB), a core defect in FA pathology

  • Small molecule approach enables oral delivery and scalable access

  • Strategic use of licensed, de-risked compounds for faster development

  • Epigenetic platform opens the door to first-in-class disease-modifying therapies

  • Supported by IP from Penn, Johns Hopkins, and internal discoveries

Stage & Investment

  • Stage: Preclinical (p38 program poised for Phase 2 entry)

  • Funding Raised: <$500K

  • IP: Mix of licensed and internally developed patents from Penn and Johns Hopkins

  • Regulatory: Type C FDA meeting in planning; IND preparation underway

  • Collaborations: University of Pennsylvania, Johns Hopkins University

What's Next

  • Secure funding for IND clearance and launch of Phase 2 trial

  • Advance development of ferroptosis inhibitors and validate new epigenetic hits

  • Build out internal R&D capabilities while maintaining lean external partnerships

  • Position p38 MAPK inhibitor for clinical proof-of-concept and follow-on indication expansion

Leadership

Leo-Kim.jpg
Leo Kim, PhD
Founder & CEO
bottom of page