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The essence of innovative drug research and development is to solve unmet clinical needs, the evolution of human disease spectrum guides the direction of pharmaceutical research and development investment, along the change trend of disease spectrum, in advance layout, in order to be expected to grasp the general trend
of industry development.
The global spectrum of diseases has changed, and the era of neuropsychiatric diseases has arrived
The global spectrum of diseases has changed, and the era of neuropsychiatric diseases has arrived In the past three decades, with the development of society and the advancement of medical technology, most infectious diseases have been controlled by humans, the smallpox virus that once ravaged the world has been eliminated globally, and hepatitis C virus has been conquered
with the widespread use of direct antiviral drugs.
Since the 70s of the 20th century, with the development and marketing of a series of antihypertensive and lipid-lowering drugs, most cardiovascular and cerebrovascular diseases can be effectively controlled
.
In recent years, in addition to malignant tumors, neuropsychiatric diseases are threatening the health of
all mankind.
According to IHME data, in 2019, the global disability-adjusted life years for mental illness reached 130 million years, and the disability-adjusted life years for neurological diseases could reach 100 million years
.
▲Source: IHME Remarks: Red labels indicate infectious diseases, neonatal diseases, and nutrition-related diseases, blue labels indicate non-communicable diseases, and gray labels indicate injuries
.
DAYLs stand for Disability Adjusted Life Year
Disease diversity, large patient size, unmet clinical needs are huge
Disease diversity, large patient size, unmet clinical needs are huge At present, there are many types of neuropsychiatric diseases, involving more than 1,000 indications, mainly including: neurodegenerative diseases, which include many "well-known" diseases: including Alzheimer's disease and other types of dementia, Parkinson's disease, multiple sclerosis, Huntington's disease, amyotrophic lateral sclerosis, etc.
; Psychiatric disorders, such as depression, schizophrenia, bipolar disorder, autism, various substance addictions, etc.
; Pain; Epilepsy and infectious diseases of the nervous system
.
The number of patients with neuropsychiatric disorders is large and growing
.
For example, 53 million people worldwide suffer from Alzheimer's disease and other dementias, about 200 million suffer from depression, and headaches are one of the most common neurological conditions, affecting 2.
7 billion people worldwide.
Source: World Bank, IHME
With the further aging of the population, the accelerated pace of modern people's life, the combined influence of social factors such as high work pressure, will also lead to the continuous increase
in the prevalence of neuropsychiatric diseases.
According to the prediction of relevant research institutions, in 2033, China's population over 65 years old will account for 20%, which means that China will enter a super-aging society, and under accelerated aging, the prevalence of neurodegenerative diseases and other diseases will continue to rise
.
However, compared with the continuous progress in other medical fields, human research on neuropsychiatric diseases seems to be stagnant, there is a huge unmet need in the field of neuropsychiatric diseases, many diseases currently have no effective treatment drugs approved for marketing, and the mechanism research of some diseases has made little
progress.
▲Source: Diagnostic guide, compiled by the author
R&D and trading are active, and the neuropsychiatric field has attracted much attention
R&D and trading are active, and the neuropsychiatric field has attracted much attention In recent years, despite the multiple challenges of innovative drug research and development, such as high R&D costs and low success rates in late-stage clinical trials, the field of neuropsychiatric diseases has attracted a large number of companies to develop a deep understanding of the biological mechanisms of diseases and try to transform the possibilities
of clinical practice of neuropsychiatric diseases.
A total of 1,211 innovative drugs have been marketed for neuropsychiatric diseases worldwide, involving more than 300 targets, dominated by neurotransmitter receptors, such as GABAAR, dopamine receptor D2, opioid receptor, mAChRs, and 5-HT2A; Cyclooxygenase is the most marketed drug target
.
According to relevant data, there are a total of 1,631 drugs under clinical research for neuropsychiatric diseases worldwide, involving nearly 600 targets, of which 374 have not yet been successfully marketed, and 129 have entered the late-stage research and development stage (clinical phase III and declared for marketing) of 129 targets
.
In the cold capital winter environment, transactions in the neuropsychiatric field are still hot, and there are a number of high-priced M&A transactions related
to this disease field in 2021.
For example, the biggest deal in 2021 was a $3 billion
collaboration between Roche and Shape Therapeutics to develop gene therapies for Alzheimer's, Parkinson's and rare diseases.
Through the collaboration, the two companies plan to use Shape Therapeutics' novel RNA editing platform, RNAfix™, and AAVid™ technology platform for clinical research to develop next-generation tissue-specific adeno-associated viruses (AAVs).
Source: IQVIA
Breakthrough technologies continue to advance the field of neuropsychiatry
Breakthrough technologies continue to advance the field of neuropsychiatry In recent years, emerging technologies such as gene therapy, cell therapy, and digital therapy have brought breakthroughs to the treatment of neuropsychiatric diseases and injected new impetus
into the development of the industry.
For example, the gene therapy AXO-Lenti-PD developed by Oxford Biomedica and Axovant increases dopamine synthesis by constructing lentiviral vectors to simultaneously transduce the key enzyme genes of AADC, GCH1 and TH to achieve the treatment of
Parkinson's disease.
At present, the drug is in the second phase of clinical practice, and its effectiveness is still to be verified
.
NurOwn®, developed by BrainStorm Cell Therapeutics, is an autologous stem cell therapy that is derived from the patient's own bone marrow mesenchymal stem cells that are directed to differentiate into neurotrophic factor (NTF) in vitro called MSC-NTF cells
.
Modified MSC-NTF cells are then infused back into the patient, effectively delivering a variety of NTF and immunomodulatory cytokines directly to the site of injury, causing the desired biological effects and ultimately slowing or stabilizing disease progression
.
This cell therapy can be used for the potential treatment
of amyotrophic lateral sclerosis, autism, Parkinson's disease, spinal cord injury, multiple sclerosis, sciatica, and Huntington's disease.
In recent years, digital health technology is developing rapidly, and digital innovation is expected to provide new health solutions
for patients with neuropsychiatric disorders.
For example, Boehringer Ingelheim is collaborating to develop the CT-155 mobile application as an adjunct to drug therapy, which aims to provide psychotherapeutic interventions
for people with schizophrenia.
CT-155 is guided by the basic science of cognitive behavioral therapy and combines multiple therapeutic interventions to help patients change behavior and train new skills
.
Boehringer Ingelheim is currently conducting clinical trials to verify the effectiveness of
this digital therapeutic.
▲Source: Compiled by the author
epilogue
epilogue The field of neuropsychiatric diseases is full of unknown explorations and challenges, and the huge patient size and a large number of unmet clinical needs attract pharmaceutical companies to invest in new technologies and innovative drug research and development
in this field.
In the future, the booming business prospects in the neuropsychiatric field are bound to attract many market players to participate in it, resulting in more fierce market competition and increasingly concentrated research and development
.
For future innovators, if they want to seize the wave of innovation in the neuropsychiatric field, they need to target the needs of the currently unmet patient population and differentiate themselves, and the value proposition of new drugs is based on the precision of biomarker research
.
【Reference】
【Reference】【Reference】
1.
"In the face of capital winter, breakthrough technologies continue to drive the CNS trading boom", IQVIA, 2022
"In the face of capital winter, breakthrough technologies continue to drive the CNS trading boom", IQVIA, 2022
2.
"Riding the Wind and Waves| Gene Therapy: Ushering in an "Age of Healing," BCG, 2020
"Riding the Wind and Waves| Gene Therapy: Ushering in an "Age of Healing," BCG, 2020
3.
Advances in Global Neuropsychiatric Research, 2021
Advances in Global Neuropsychiatric Research, 2021
4.
"The field of neuropsychiatric diseases is hot! AbbVie reaches new cooperation, multi-enterprise investment layout", 2022
"The field of neuropsychiatric diseases is hot! AbbVie reaches new cooperation, multi-enterprise investment layout", 2022
5.
Parkinson's disease gene therapy ushered in a new cooperation! Oxford and Axovant form 3-year clinical supply partnership, Ek, 2020
Parkinson's disease gene therapy ushered in a new cooperation! Oxford and Axovant form 3-year clinical supply partnership, Ek, 2020
6.
"From the Evolution of Human Disease Spectrum to Explore the Ultimate Route of Drug R&D", 2021
"From the Evolution of Human Disease Spectrum to Explore the Ultimate Route of Drug R&D", 2021
7.
How Digital Therapeutics Benefits Patients, Healthcare Organizations, and the Healthcare Ecosystem, IBM Institute for Business Value, 2019
How Digital Therapeutics Benefits Patients, Healthcare Organizations, and the Healthcare Ecosystem, IBM Institute for Business Value, 2019
8.
Company official website
Company official website
9.
Source: World Bank, Institute for Health Indicators and Evaluation, University of Washington, USA
Source: World Bank, Institute for Health Indicators and Evaluation, University of Washington, USA