Parkinson’s Disease: Molecular Landscape & Future Disease Modifying Targeted Therapies

Neurological disorders are becoming the leading cause of disability worldwide.  Among these neurological disorders Parkinson’s Disease (PD) growth is expected to surpass that of Alzheimer disease! It is projected that the number of people with PD will double from 6.9 million in 2015 to 14.2 million in 2040. Action requires improved disease modifying drugs, targeted treatment to reduce side effects and improve efficacy, early diagnosis and of course affordable medicines so that all patients have access to the medicines worldwide.

It has been 200 years since Dr. James Parkinson published an essay on the Shaking Palsy describing the signs and symptoms of Parkinson’s disease (PD).  However, treatment to date is only symptomatic and there are no disease modifying therapies available yet.  We foresee this scenario to change considering a better understanding of the biology and genetics of the disease. This report focuses on the new developments in the understanding of the disease that could lay the foundation for targeted therapies in PD.

Advances in the understanding of the biological pathway and genetics involved in the pathogenesis of the disease has identified triggers of neurodegeneration and factors that contribute to the onset and progression of PD.  A targeted approach for developing molecular therapeutics for PD can emerge just as it happened in the case of Migraine. Three drugs targeting Calcitonin-Gene Related Peptide (CGRP) for migraine treatment (Amgen/ Novartis’ Aimovig/ erenumab, launched; Lilly’s Emgality/ Galcanezumab, filed; and Teva’s Ajovy/ fremanezumab, filed) are likely to change the treatment paradigm for this disease.  We expect more targeted therapies to follow in other Neurodegenerative diseases including PD as new science and improvements in drug delivery technologies identifies druggable targets.

In this report we highlight the several years of genetic research in PD that has led to the identification of several monogenic forms of the disorder and the numerous genetic factors that increase the risk to develop PD. 

Drugs in the research and development pipeline are discussed in the report and the pipeline includes:

  •  
  • Drugs that targets receptors/gene products associated or mutated in PD disease.
  • New delivery mechanisms/formulations of approved treatments for PD.
  • Repurposed Drugs approved for other indications being pursued in PD.
  • Other approaches

Author Bio Subita Srimal

ProGrow Pharma (PGP) Partners caters the full spectrum of services to Pharma companies (Pharma/ Biotech, Generic, CRAMS, CROs, Start-ups) and its Investors (Private Equity, Fund Managers, HNI).  With decades of experience in strategic advisory, regulatory aspects, technical publications, understanding complex drugs, identifying market potential and all related services, the team is widely approached by pharmaceutical companies and experts.  Our team can support you from defining strategic programmes to providing expert guidance for tactical operations to transform your operational or market based challenges into growth aligned opportunities.

  1. Executive Summary
  2. Disease Overview
    • Pathogenesis and Types
    • Prevalence
    • Current Treatment Paradigm
    • Approved Drugs
    • Unmet Needs
  3. Molecular Landscape: New Insights and Genetic Profiling
    • Key Genes being Targeted by New Drugs in Development
      • SNCA (PARK1–4)
      • The GBA1 Gene and the GCase Protein
      • Parkin (PARK2)
      • PINK1 (PARK6)
      • DJ-1 (PARK7)
      • LRRK2: Leucine-Rich Repeat Kinase-2 Gene (PARK8)
      • ATP13A2 (PARK9))
  4. Therapeutics in Development
    • Targeting Specific Genes/proteins
    • Improved Formulation of Approved Drugs for PD
    • Repurposed Drugs
    • Others
  5. Market Opportunity
  • AffiRis
  • Biogen
  • Prothena/Roche
  • Denali
  • GSK
  • Neuraly
  • Impax/Amneal
  • Acorda Therapeutics
  • SynAgile
  • Abbvie
  • Neuroderm/Tanabe
  • Luye America Pharmaceuticals
  • PharmaTwoB
  • UniQure
  • Voyager Therapeutics/Sanofi
  • Orion
  • BIAL /Neurocrine
  • Genervon
  • Kyowa Hakko Kirin Pharma
  • BioElectron Technology
  • Metabolic solutions
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