Kifa Therapeutics advances highly selective CNS-kinase inhibitors, starting with a G2019S-LRRK2 therapy for Parkinson’s disease.
Our strategy pairs genetics-based patient selection with validated biomarkers to enable efficient clinical studies and an orphan, accelerated-approval path.
Kifa Therapeutics is a precision-neurology company developing first-in-class, mutant-selective kinase inhibitors to halt Parkinson’s disease at its genetic source.
Our lead program targets the G2019S mutation in LRRK2—the most common genetic cause of Parkinson’s—which drives neuronal toxicity through hyperactivation of the LRRK2 kinase pathway.
With over 10 million patients worldwide and an $80 billion annual U.S. economic burden, no disease-modifying therapies exist—only symptomatic treatments that lose effectiveness and cause debilitating side effects.
Prior non-selective LRRK2 inhibitors failed due to off-target liabilities. Kifa’s proprietary G2019S-LRRK2 inhibitors deliver >150-fold selectivity over wild-type enzyme, are CNS-penetrant, and are supported by translational biomarkers that de-risk development.
The program is less than six months from IND-enabling studies and advances in partnership with the Michael J. Fox Foundation’s LITE Consortium. By starting in genetically defined patients, Kifa leverages orphan-drug and accelerated-approval pathways to reach patients faster—then expands into broader biomarker-positive idiopathic PD.
Kifa’s mission: transform Parkinson’s from inevitable decline to measurable recovery.
Leadership Team
- Christopher James, M.D. – CEO & Founder
Neurosurgery background in Parkinson’s disease, plus >20 years across Wall Street and biotech/pharma. Leads strategy, capital formation, and clinical partnerships. - Craig Lindsley, Ph.D. – Scientific Founder
Executive Director, Vanderbilt WCNDD. 500+ publications, 100+ patents, >$200M external funding. World-class leader in kinase drug discovery and translation. - Chetan Lathia, Ph.D. – R&D/Regulatory
>35 INDs and 10 NDAs/BLAs. Deep expertise in nonclinical, clinical pharmacology, and regulatory execution. - Sam Chuang, Ph.D. – Development/Partnerships
Extensive Charles River experience and business development interfaces, strengthening external execution.
This team uniquely combines mutant-selective kinase chemistry, biomarker-driven clinical design, and regulatory execution required for an orphan/accelerated-approval path in Parkinson’s.
Kifa’s go-to-market strategy starts with genetically defined Parkinson’s (LRRK2-G2019S), leveraging orphan-drug and accelerated-approval pathways anchored in translational biomarkers (pRab10/pRab12, urine BMP).
Initial clinical execution will focus on movement-disorder centers with established LRRK2 testing and longitudinal cohorts for efficient screening, rapid enrollment, and close biomarker monitoring.
After proof-of-biology and safety in G2019S PD, expansion into biomarker-positive idiopathic PD (“LRRK2-like” subgroups) will grow the addressable market while preserving precision positioning.
Core Partnerships & Execution
- Translational/Assay Partners
Ongoing collaboration with MJFF’s LITE Consortium to standardize and validate biomarker assays for clinical translation. - Academic/KOL and Site Networks
Movement-disorder clinics and research cohorts to support genetically enriched studies and adaptive designs. - CROs and Preclinical Execution
External partners (e.g., Charles River, Dundee) for IND-enabling work and early clinical operations. - Registry/Genetic-Screening Collaborators
Patient identification and trial readiness for accelerated studies. - Regulatory Engagement
Early pre-IND alignment on biomarker endpoints suited for accelerated approval.
This staged, precision-first approach maximizes technical and regulatory success while aligning with payer expectations for high-value rare-disease launches.
Primary revenue will come from prescription sales of a disease-modifying oral small-molecule for G2019S-LRRK2 Parkinson’s, launched under orphan economics, followed by label expansion into biomarker-defined idiopathic PD.
Market modeling projects a TAM ~$13B by 2035, with a serviceable genetic-PD segment ~$3.4B and initial G2019S population supporting $500–$850M annual revenue at orphan pricing—before broader expansion.
Monetization Phases
- Orphan Launch (G2019S PD)
Focused centers with pharmaco-economic dossiers built on biomarker-anchored benefits to support premium, value-based pricing and access. - Expansion to Biomarker-Positive Idiopathic PD
Increases treated prevalence while maintaining precision positioning and payer alignment. - Partnership Economics
Potential ex-U.S. commercial partnerships and/or co-development to accelerate global reach and reduce capital intensity.
This model captures near-term orphan value while enabling scalable growth and durable pricing through biomarker differentiation.
Investment Ask
Kifa is raising a $3M seed round (complementing active non-dilutive MJFF/LRRK2 support) to nominate the clinical candidate and initiate IND-enabling studies ahead of a pre-IND meeting (Q2 2026) and Phase 1 start in 2027.
Use of Proceeds
| Allocation | Percentage | Purpose |
|---|---|---|
| Lead Nomination & CMC | 25–30% | Final med-chem optimization, salt/polymorph selection, early formulation, analytical methods. |
| IND-Enabling Nonclinical | 35–40% | Safety pharmacology, genetic tox, dose-range finding, TK/PK focused on LRRK2-relevant tissues. |
| Translational Biomarkers | 15–20% | GLP-compatible pRab10/pRab12 and urine BMP assay validation, sample-handling SOPs, data standards. |
| Regulatory & Clinical Readiness | 10–15% | Pre-IND package, protocol synopses for HV SAD/MAD and G2019S-enriched Phase 1b/2a, site/KOL engagement. |
| G&A and IP | 5–10% | Option/license maintenance, PCT nationalizations, core operations. |
Key milestones enabled: candidate nomination, initiation of GLP tox, biomarker assay readiness, pre-IND alignment on accelerated-approval endpoints, and Phase 1 startup activities.
Kifa Therapeutics is a preclinical-stage biotechnology company advancing first-in-class, mutant-selective small-molecule inhibitors of LRRK2 for genetically defined Parkinson’s disease.
Lead series achieves >150-fold selectivity for G2019S-mutant LRRK2 versus wild-type, with strong brain penetration and favorable PK in rodent models—designed to avoid pulmonary and renal liabilities of prior non-selective inhibitors.
Current stage: Finalizing lead optimization and candidate selection for IND-enabling studies, supported by translational biomarkers (phospho-Rab10, phospho-Rab12, urine BMP) that directly measure pathway activity.
Timeline: <6 months from IND-enabling initiation, pre-IND FDA meeting targeted for Q2 2026, Phase 1 planned for 2027.
Validation: Ongoing collaboration with Michael J. Fox Foundation’s LITE Consortium. Two chemically distinct, CNS-penetrant series protected under international PCT filings.
Next Milestones
- Finalize candidate nomination and initiate GLP safety/toxicology studies.
- Complete pre-IND briefing package and secure regulatory alignment.
- Expand biomarker assay validation to support Accelerated Approval endpoints.
Kifa’s progress positions the program as the leading precision-neurology approach for disease-modifying treatment of Parkinson’s.
Kifa has secured and committed the following funding:
| Source | Amount/Commitment |
|---|---|
| LaunchTN | $300K SAFE (conditionally approved) |
| Non-Dilutive (In-Kind) – MJFF/LITE Consortium | ~$313K–$550K (assays, cryo-EM, in-vivo) |
| Total Secured/Committed | ~$613K–$850K (mix of pending cash + in-kind) |
