R&D Trends: Multiple Sclerosis - Progressive subtypes remain underserved in expansive pipeline

Introduction

In recent years, there has been a paradigm shift in the pipeline for new MS therapies, with a growing number of candidates progressing through trials that have the potential for higher efficacy and greater convenience. Despite a relatively small population, MS is an attractive target for drug developers due to its chronic nature, inadequacies of treatments, and the potential for premium pricing.

Features and benefits

* Understand the key trends for the R&D pipeline for new MS therapies
* Benchmark novel and existing therapies against a target product profile
* Support R&D decision making by evaluating MS clinical trial designs that have set a precedent
* Access Datamonitor's opinion regarding how the treatment landscape will change in the next 20 years

Highlights

The launch of the first oral drug in marks a new wave of novel compounds ready to enter the MS market in the near future. With over 70 candidates in development, the MS drug pipeline is rich with a variety of candidates that have the potential to improve treatment outcomes for patients.
Relapse remitting MS is a less risky and more appealing route to market compared to progressive subtypes and this is reflected by the high proportion of RRMS candidates. The small number of progressive candidates highlights a large opportunity for developers willing to target this hard to treat subpopulation.
Looking beyond the next decade, exciting new therapeutic approaches will emerge for MS potentially including RNA, gene, and stem cell therapies. However, given the likely high cost, these innovative approaches will need to show excellent safety and efficacy if they are to find a place in the increasingly diverse treatment algorithm.

Your key questions answered

* What are the key trends in the multiple sclerosis pipeline?
* What is the clinical gold standard and how do new candidates have to compare to this to successfully penetrate the market?
* How will new MS treatments evolve in the next 20 years?
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Executive Summary
OVERVIEW
Catalyst
Summary
CLINICAL PIPELINE OVERVIEW
Multiple sclerosis pipeline overview
Multiple sclerosis has one of the largest clinical pipelines in the neurology field
Pipeline subtype analysis
Over 40% of the clinical pipeline is in development for "unspecified" multiple sclerosisThe majority (43%) of the multiple sclerosis pipeline candidates are in development for "unspecified" multiple sclerosis. However, despite this large number, only one of these 27 candidates is in Phase III development, while five are in Phase II trials and 21 are in Phase I development.
Only five drugs are in the primary progressive multiple sclerosis pipeline
No secondary progressive candidates in late-stage development
Many different modes of action are being explored in multiple sclerosis, especially at early-stage development
TARGET PRODUCT PROFILE
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Avonex remains the gold standard in multiple sclerosis
Target product profile versus current level of attainment
CLINICAL TRIAL DESIGN IN MULTIPLE SCLEROSIS
Clinical trial endpoints in multiple sclerosis
Relapse rate
Disability/progression measures
Health-related quality-of-life assessments
MRI measures
Typical multiple sclerosis clinical trial design
Future developments in clinical trial designApproved multiple sclerosis disease-modifying drugs set the standard for future trial design
The revised McDonald criteria allow trials to include patients earlier in the course of their disease
Placebo-controlled multiple sclerosis trials might be considered unethical
A placebo control arm is essential in Phase III trials but less of an issue in Phase II trials
The SENTINEL and BEYOND studies provide alternative trial designs
INNOVATIVE EARLY-STAGE APPROACHES
Therapeutic vaccines
BHT-3009 (Bayhill Therapeutics)According to Correale and Fiol (2009), preclinical studies in different models of autoimmunity have demonstrated that injecting plasmid DNA encoding a self-antigen in mice restores self-tolerance, leaving immunity against infectious and tumor antigens intact. Based on this evidence, the first DNA vaccine for multiple sclerosis was created.
Tovaxin (Opexa Therapeutics)
RNA therapy
Cell therapy
Gene therapy
Candidates in preclinical development
THE FUTURE OF TREATMENT IN MULTIPLE SCLEROSIS
Increased disease etiology understanding will provide the next disease breakthroughThe advent of genome-wide association studies has allowed investigators to identify numerous susceptible alleles, enhancing the understanding of the genetic architecture of multiple sclerosis, the eventual understanding of which will allow for the identification of numerous therapeutic targets for developers to investigate.
Therapies treating progressive forms of multiple sclerosis will be the next therapeutic goalDatamonitor believes that the continued development of novel therapies will help drive market growth over the medium term, however, the development of drugs addressing the wider range of multiple sclerosis disease severities is regarded as the next hurdle for developers. Datamonitor also believes that treating progressive forms of multiple sclerosis will be the next breakthrough for developers, with the greater understanding of the progressive multiple sclerosis etiology in the future leading to more precise targeting of drug treatments.
Remyelination will become an achievable therapeutic goal in the future
Biomarker research will lead to the advent of personalized medicine
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Improved screening and diagnosis will aid preventative therapies
BIBLIOGRAPHY
Journal papers
Websites
Datamonitor reports
Other
APPENDIX
Contributing experts
Report methodology
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