Progressive Supranuclear Palsy Investment Landscape

Investment Landscape: Progressive Supranuclear Palsy

Overview

Investment Landscape: Progressive Supranuclear Palsy covers the current R&D investment, clinical trial pipeline, funding trends, and investment opportunities for Progressive Supranuclear Palsy research.

Last updated: 2026-03-28 15:50 PT

Clinical Trial Pipeline

Total Clinical Trials: 49 Active Trials (Recruiting/Active): 12

Trial Phase Distribution

| Phase | Number of Trials | Percentage |
|-------|-----------------|------------|
| Phase 1 | 8 | 16% |
| Phase 2 | 28 | 57% |
| Phase 3 | 5 | 10% |
| Observational | 8 | 16% |

Therapeutic Modalities

| Modality | Active Trials | Key Programs |
|----------|---------------|--------------|
| Anti-tau antibodies | 5 | Gosuranemab, Tilavonemab, Bepranemab |
| ASO therapy | 1 | BIIB080 (MAPTRx) |
| Small molecule inhibitors | 4 | Neflamapimod, Davunetide, Valproic acid |
| Cell therapy | 2 | Stem cell transplantation |
| Gene therapy | 1 | TPN-101 |
| Repurposed drugs | 8 | CoQ10, Lithium, Valproic acid, Tetrabenazine |
| Device/Procedural | 6 | TMS, tDCS, Deep brain stimulation |

Investment Context

Investment Landscape: Progressive Supranuclear Palsy

Overview

Investment Landscape: Progressive Supranuclear Palsy covers the current R&D investment, clinical trial pipeline, funding trends, and investment opportunities for Progressive Supranuclear Palsy research.

Last updated: 2026-03-28 15:50 PT

Clinical Trial Pipeline

Total Clinical Trials: 49 Active Trials (Recruiting/Active): 12

Trial Phase Distribution

| Phase | Number of Trials | Percentage |
|-------|-----------------|------------|
| Phase 1 | 8 | 16% |
| Phase 2 | 28 | 57% |
| Phase 3 | 5 | 10% |
| Observational | 8 | 16% |

Therapeutic Modalities

| Modality | Active Trials | Key Programs |
|----------|---------------|--------------|
| Anti-tau antibodies | 5 | Gosuranemab, Tilavonemab, Bepranemab |
| ASO therapy | 1 | BIIB080 (MAPTRx) |
| Small molecule inhibitors | 4 | Neflamapimod, Davunetide, Valproic acid |
| Cell therapy | 2 | Stem cell transplantation |
| Gene therapy | 1 | TPN-101 |
| Repurposed drugs | 8 | CoQ10, Lithium, Valproic acid, Tetrabenazine |
| Device/Procedural | 6 | TMS, tDCS, Deep brain stimulation |

Investment Context

Progressive Supranuclear Palsy represents a significant unmet medical need in the neurodegenerative disease landscape. With approximately 49 clinical trials and 12 actively recruiting, the PSP research ecosystem has grown substantially over the past five years[@smith2023]. The pure 4R-tau pathology makes PSP an attractive indication for anti-tau therapeutic development, with several high-profile Phase 2 readouts in 2024[@mendonca2024; @holmes2024].

Key Investment Themes

• Tau Targeting: Primary therapeutic focus given tau pathology
• Neuroprotection: Preserving vertical gaze and motor function
• Biomarker Development: NfL, tau PET for patient stratification[@anthony2024; @leahy2025]
• RNA-targeting Modalities: ASO and RNAi therapeutics[@choi2025]

Emerging Investment Areas

Priority Research Gaps

Biomarker Validation

Disease Modification Endpoints

Recommended Priorities

Therapeutic Target Priorities

Based on trial count analysis, the following mechanism categories represent either well-invested areas or underserved opportunities:

| Priority | Mechanism | Trials | Investment Level |
|----------|-----------|--------|-----------------|
| High | Tau immunotherapy | 5 | Well-funded |
| High | RNA targeting | 1 | Emerging |
| Medium | Neuroprotection | 8 | Moderate |
| Medium | Symptomatic (motor) | 12 | Well-funded |
| Low | Non-motor symptoms | 6 | Underserved |

Investment Outlook

Near-Term Opportunities (1-3 Years)

• 2024-2025 Phase 2 readouts: Gosuranemab, Tilavonemab, Bepranemab results
• Biomarker-positive trials: Enrichment using tau PET and NfL
• Regulatory clarity: FDA/EMA guidance on disease-modifying endpoints

Medium-Term Opportunities (3-5 Years)

• ASO expansion: BIIB080 Phase 2/3 development
• Combination therapy trials: Immunotherapy + small molecule
• Personalized medicine: Genetic stratification (MAPT, GRN, CBD)

Long-Term Vision (5-10 Years)

• Prevention trials: Pre-symptomatic populations with genetic risk
• Gene therapy: AAV-delivered anti-tau constructs
• Disease-modifying therapies: Potential disease reversal

Clinical Trial Highlights

Active Anti-Tau Antibody Trials

| Drug | Company | Phase | NCT | Status |
|------|---------|-------|-----|--------|
| Gosuranemab | Biogen | Phase 2 | NCT04039017 | Active |
| Tilavonemab | AbbVie | Phase 2 | NCT03580956 | Active |
| Bepranemab | UCB | Phase 2 | NCT05318985 | Recruiting |
| Tilavonemab | AbbVie | Phase 2 | NCT02448330 | Active |

ASO Therapy

• BIIB080 (MAPTRx): Biogen/Ionis Phase 1b in AD, expanding to PSP
• TPN-101: Retrofin (gene therapy) Phase 1/2

Repurposed Drug Trials

• CoQ10: NCT00532571 (completed), NCT04564555 (ongoing)
• Lithium: NCT00385710, NCT05297202
• Neflamapimod: Phase 2 in PSP

Related Pages

• [Progressive Supranuclear Palsy](/diseases/progressive-supranuclear-palsy)
• [Tau Pathology](/mechanisms/tau-pathology)
• [Clinical Trials: PSP](/clinical-trials)
• [4R-Tauopathies](/mechanisms/4r-tauopathies)

See Also

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/diseases/parkinsons-disease)
• [Corticobasal Syndrome](/diseases/corticobasal-syndrome)

External Links

• [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
• [ClinicalTrials.gov PSP](https://clinicaltrials.gov/search?cond=PSP)
• [CurePSP Foundation](https://www.psp.org/)

References