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Parkinson's Disease Research Focuses on Disease-Modifying Therapies

  • The Parkinson's disease (PD) therapeutic market faces unmet needs, notably the absence of neuroprotective/disease-modifying therapies (DMTs) and treatments for non-motor symptoms.
  • A significant portion of the PD drug development pipeline is dedicated to neuroprotective and disease-modifying agents, targeting mechanisms like alpha-synuclein aggregation and neuroinflammation.
  • Clinical trials are underway for therapies addressing postural instability, PD-dementia, and cognitive/emotional impairments, reflecting a comprehensive approach to managing PD's complexities.
While current Parkinson's disease (PD) treatments primarily address motor symptoms, a significant shift in research is focusing on disease-modifying therapies (DMTs) to alter the course of the disease. Key opinion leaders (KOLs) have highlighted the urgent need for therapies that can slow or halt disease progression and manage non-motor symptoms, which greatly impact patients' quality of life.

Emphasis on Neuroprotection and Disease Modification

According to GlobalData's drug database, a substantial 66% of the 93 pipeline products in Phase I to Phase III development across the seven major markets (7MM: France, Germany, Italy, Japan, Spain, the UK, and the US) are being investigated for their neuroprotective or disease-modifying properties. These investigational agents target key mechanisms implicated in the pathophysiology of PD, such as alpha-synuclein aggregation and neuroinflammation, to slow disease progression.

Targeting Alpha-Synuclein Aggregation

Notably, therapies targeting alpha-synuclein aggregation constitute 26% of the DMT pipeline. Annovis Bio’s Posiphen (buntanetap tartrate), currently in Phase III development (NCT05357989) in the US and five major European markets (5EU: France, Germany, Italy, Spain, and the UK), seeks to inhibit alpha-synuclein. However, KOLs have expressed divided opinions on the likely efficacy of anti-alpha-synuclein therapies, with some citing safety concerns and the failure of Prothena/Roche’s prasinezumab in the Phase II PASADENA trial (NCT03100149).

Addressing Non-Motor Symptoms

There is a growing recognition of the importance of addressing non-motor symptoms in PD, such as falls, cognitive impairment, and emotional disturbances. Approximately 18% of the pipeline is classified as "other anti-Parkinsonian agents," emphasizing symptom management beyond core motor symptoms. Cerevance’s solengepras, currently under Phase III (NCT06553027) investigation in the US, aims to improve postural instability in PD patients. Additionally, IRLAB’s Pirepemat is under investigation at Phase IIb (NCT05258071) and Phase II for both postural instability and PD-dementia, and Silo Pharma’s psilocybin (Phase II) targets cognitive and emotional impairment (particularly depression and anxiety) in patients with PD.
The focus on improving non-motor symptoms in PD patients is further highlighted by the fact that 3% of the pipeline is specifically targeting PD-dementia and PD-psychosis. This comprehensive approach aims to improve both motor and non-motor symptoms, addressing the broader spectrum of challenges faced by PD patients.
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Reference News

[1]
Parkinson's disease clinical research in 7MM focuses on disease-modifying agents
clinicaltrialsarena.com · Nov 7, 2024

Parkinson’s disease (PD) lacks neuroprotective/disease-modifying therapies (DMTs) and non-motor symptom treatments, with...

[2]
7 Most Promising Drugs in Parkinson's Disease Treatment Pipeline - BioSpace
biospace.com · Dec 10, 2024

The Parkinson's disease market is expected to grow significantly, reaching USD 6.2 billion by 2034, driven by advancemen...

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