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
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Lead candidate: Clinically validated p38 MAP Kinase inhibitor targeting the root biochemical defect in FA
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Rapid development path leveraging existing safety data for accelerated Phase 2 entry
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Pipeline includes:
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Ferroptosis inhibitors for additive therapeutic impact
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Novel epigenetic platform to discover future targets and hit compounds
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Global TAM: ~21,000 FA patients; potential $1B+ in peak revenue per program
Technology Differentiators
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Targets Iron-Sulfur Cluster Biogenesis (ISCB), a core defect in FA pathology
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Small molecule approach enables oral delivery and scalable access
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Strategic use of licensed, de-risked compounds for faster development
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Epigenetic platform opens the door to first-in-class disease-modifying therapies
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Supported by IP from Penn, Johns Hopkins, and internal discoveries
Stage & Investment
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Stage: Preclinical (p38 program poised for Phase 2 entry)
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Funding Raised: <$500K
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IP: Mix of licensed and internally developed patents from Penn and Johns Hopkins
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Regulatory: Type C FDA meeting in planning; IND preparation underway
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Collaborations: University of Pennsylvania, Johns Hopkins University
What's Next
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Secure funding for IND clearance and launch of Phase 2 trial
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Advance development of ferroptosis inhibitors and validate new epigenetic hits
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Build out internal R&D capabilities while maintaining lean external partnerships
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Position p38 MAPK inhibitor for clinical proof-of-concept and follow-on indication expansion
Leadership
Leo Kim, PhD
Founder & CEO
