Therapeutics
Table 1: RNA-based therapeutics in clinical development. RNA drugs in five or more clinical trials as of July 2018 are tabulated. Data provided by GlobalData Plc
Class mRNA Company
Argos Therapeutics Inc
eTheRNA Immunotherapies NV
Boehringer Ingelheim GmbH
Antisense RNA
Biogen Inc
Akcea Therapeutics Inc
Sarepta Therapeutics Inc
Antisense Therapeutics Ltd
Pharmaxis Ltd siRNA
The Medicines Company
Alnylam Pharmaceuticals Inc
RXi Pharmaceuticals Corp
Alnylam Pharmaceuticals Inc
Drug (Brand Name)
Rocapuldencel-T AGS-004
TriMix-based immunotherapy
BI-1361849
Nusinersen (Spinraza)
Volanesorsen sodium
Eteplirsen (Exondys 51) ATL1102
ASM-8 Inclisiran
Patisiran (Onpattro)
RXI-109 Fitusiran Cemdisiran
Therapeutic Area
Oncology
Infectious Disease
Oncology Oncology
Central Nervous System
Metabolic Disorders
Genetic Disorders
Central Nervous System
Respiratory Cardiovascular
Metabolic Disorders
Metabolic Disorders
Dermatology Ophthalmology
Haematological Disorders
Gastrointestina
Haematological Disorders
Quark Pharmaceuticals Inc
Bristol-Myers Squibb Co Pfizer Inc
QPI-1002
Genito Urinary System And Sex Hormones
Immunology
BMS-986263 PF-655
Metabolic Disorders
Ophthalmology
RNA Aptamer
Pfizer/Valeant Pharmaceuticals International Inc Ophthotech Corp
Noxxon Pharma AG
Pegaptanib (Macugen)
Avacincaptad pegol sodium (Zimura)
Olaptesed pegol Emapticap pegol
Indication
Muscle Invasive Bladder Cancer (MIBC); Non- Small Cell Lung Cancer; Renal Cell Carcinoma
Human Immunodeficiency Virus (HIV) Infections (AIDS)
Melanoma; Multiple Myeloma (Kahler Disease) Non-Small Cell Lung Cancer Spinal Muscular Atrophy (SMA)
Familial Chylomicronemia (Type I Hyperlipoproteinemia)
Duchenne Muscular Dystrophy
Relapsing RemittingMultiple Sclerosis (RRMS); Secondary Progressive Multiple Sclerosis (SPMS)
Allergic Asthma
Atherosclerosis; Cardiovascular Disease Homozygous Familial
Hereditary transthyretin-mediated amyloidosis (hATTR)
Hypertrophic Scars
Wet (Neovascular/Exudative) Macular Degeneration
Hemophilia A; Hemophilia B
Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) Paroxysmal Nocturnal Haemoglobinuria
Acute Renal Failure (ARF) (Acute Kidney Injury)
Kidney Transplant Rejection Gastrointestinal Liver Fibrosis Diabetic Macular Edema
Wet (Neovascular/Exudative) Macular Degeneration
Ophthalmology Age-Related Macular Degeneration (AMD) Ophthalmology
Oncology Oncology
Metabolic Disorders
Dry (Atrophic)Macular Degeneration B-Cell Chronic Lymphocytic Leukemia
Sold Tumour Diabetic Nephropathy
38 11
11 5
Marketed Phase III
Phase II Phase II
6 5
Phase II Phase II
7 7
Phase III Phase II
Clinical Trials (! 5)
9 6 6
5
13 13 12 8
6 11 8 8
Development Stage
Phase II Phase II Phase II
Phase II Marketed
Pre-registration Marketed Phase II
Phase II Phase III Marketed Phase II
6
Phase III
modalities to target RNA are also being devel- oped including the application of CRISPR-Cas9 genome editing technology and the development of selective small-molecule modulators of RNA or RNA-modifying enzymes. The global RNA drugs market is forecast to exceed $10 billion by 2024 (based on an analysis carried out using the GlobalData Plc database), highlighting the signif- icant commercial potential of this emerging class of therapeutics.
! Drug Discovery World Fall 2018
!
Development hurdles Despite the potential of RNA therapeutics, efficient and safe delivery remains a significant challenge. There are a number of significant issues that need to be overcome in their development: instability and immunogenicity; rapid clearance from the blood by the kidneys and liver scavenger receptors; cellular uptake and endosomal escape3. These hurdles can be overcome by chemically modifying RNA and by using improved synthetic delivery carriers4.
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