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

Anti-tau Immunotherapy: Gosuranemab (Biogen), Tilavonemab (AbbVie), Bepranemab (UCB)
Tau PET Imaging: PI-2620, other 4R-tau selective ligands
RNA Therapeutics: BIIB080 (MAPTRx) ASO showing promise
Biomarker-Driven Trials: NfL as endpoint, tau PET for enrollment

Priority Research Gaps

Biomarker Validation

While NfL shows promise as a progression biomarker, additional validation is needed for clinical trial endpoints[@anthony2024]. Tau PET ligands specific for 4R-tau are in development but not yet validated for PSP[@leahy2025].

Disease Modification Endpoints

The field needs better understanding of clinically meaningful endpoints beyond traditional scale-based measures.

Recommended Priorities

Biomarker standardization: Harmonize NfL and tau PET methodologies
Trial design innovation: Adaptive trials and platform protocols
Patient recruitment: Investment in trial-ready cohorts and registry infrastructure

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

Progressive Supranuclear Palsy Investment Landscape