Parkinson's Disease Research Shifts Focus to Disease-Modifying Therapies
- Clinical research in Parkinson's disease (PD) is increasingly focused on disease-modifying therapies (DMTs) to address unmet needs beyond motor symptom management.
- 66% of the 93 products in Phase I-III development target key mechanisms like alpha-synuclein aggregation and neuroinflammation to slow disease progression.
- Late-stage DMTs include Annovis Bio’s Posiphen and BioVie’s Triolex, targeting alpha-synuclein inhibition and inflammatory mediators, respectively.
- Research also emphasizes non-motor symptoms, with agents like Cerevance’s solengepras and IRLAB’s Pirepemat targeting postural instability and PD-dementia.
The Parkinson’s disease (PD) therapeutic landscape is evolving, with a notable shift towards neuroprotective and disease-modifying therapies (DMTs) to address the limitations of current treatments that primarily focus on motor symptoms. A recent analysis by GlobalData highlights a growing emphasis on DMTs and therapies targeting non-motor symptoms within the seven major markets (7MM), signaling a commitment to addressing the full spectrum of patient challenges.
According to Lorraine Palmer, Pharma Analyst at GlobalData, 66% of the 93 products currently in Phase I-III development within the 7MM for PD are prospective neuroprotective agents or DMTs. These investigational agents target key mechanisms implicated in the pathophysiology of PD, such as alpha-synuclein aggregation and neuroinflammation, with the goal of slowing disease progression.
Several neuroprotective/DMT agents are in late-stage development. Annovis Bio’s Posiphen (buntanetap tartrate), is in Phase III development (NCT05357989) in the US and 5EU, seeks to inhibit alpha-synuclein aggregation. BioVie’s Triolex (bezisterim), an inhibitor of inflammatory mediators, has a Phase III trial planned in the US due to its neuroprotective potential.
Notably, 26% of the pipeline DMTs target alpha-synuclein aggregation. However, this mechanism of action remains divisive, with some key opinion leaders (KOLs) expressing skepticism due to the failure of Prothena and Roche’s prasinezumab in the Phase II PASADENA trial (NCT03100149) and safety concerns related to alpha-synuclein's role in healthy functioning.
KOLs interviewed by GlobalData emphasized the need for research to focus on DMT development, while also highlighting that the pathogenesis of PD can differ across patients. They expressed enthusiasm for therapies addressing falls and non-motor PD symptoms, which remain a significant unmet need. 18% of the pipeline is classified as 'other antiparkinsonian agents,' emphasizing symptom management beyond core motor symptoms. Examples include Cerevance’s solengepras (Phase III; NCT06553027), which aims to improve postural instability in PD patients; IRLAB’s Pirepemat (Phase IIb; NCT05258071), under investigation for both postural instability and PD-dementia; and Silo Pharma’s psilocybin (Phase II), targeting depression and anxiety in PD patients.
The research focus on improving non-motor symptoms is further highlighted by the fact that 3% of the pipeline specifically targets PD-dementia and PD-psychosis. This comprehensive research outlook aims to improve both motor and non-motor symptoms, addressing the broader spectrum of challenges faced by PD patients.
The distribution of the 93 pipeline agents underscores a strong emphasis on neuroprotective and disease-modifying treatments, demonstrating an industry-wide focus toward therapies that could potentially slow the progression of PD.

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Highlighted Clinical Trials
Hoffmann-La Roche
Posted 6/27/2017
Annovis Bio Inc.
Posted 8/3/2022
Integrative Research Laboratories AB
Posted 6/15/2022
Cerevance
Posted 9/20/2024
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Reference News
[1]
Parkinson's disease clinical research in seven major markets focuses on disease-modifying agents
financialexpress.com · Nov 11, 2024
Parkinson’s disease (PD) therapeutic landscape lacks neuroprotective and disease-modifying therapies (DMTs). Current tre...