CFSAC Lucinda Bateman Silvana Borges Andrew Breeden - - PowerPoint PPT Presentation

MEMBERS (ex-officios)

• Lucinda Bateman
• Silvana Borges
• Andrew

Breeden

• Gustavo Ceinos
• Mary Dimmock
• Gunnar Gottschalk
• Rochelle Joslyn
• Jin-Mann

(Sally) Lin

• Nancy McGrory
• Courtney Miller
• Amrit Shahzad
• Vicky Whittemore
• Elizabeth Unger

CFSAC

Clinical Trials & Treatment Working Group

(CT&T WG) June 2018

CT&T Working Group

MISSION

• Identify barriers and challenges to developing drug treatments in

ME/CFS and define ways to address them

• Engage biopharmaceutical companies with drugs which can be tested

in, or repurposed for, ME/CFS

• Promote and facilitate clinical development of new and repurposed

drugs TEAM

• Uniquely positioned with ME/CFS knowledge, expertise, drug

development experience, and agency representation

• Strategic plan developed with members taking on tasks per their

expertise and experience

• Meeting held via conference call 2x every month

CT&T Working Group 2

Strategic Plan Designed to Achieve Goals

• Identify biopharmaceutical companies to engage
• Drugs used in clinical trials for ME/CFS
• Drugs used to treat adjacent diseases
• Drugs for targets identified in clinical and preclinical work in ME/CFS
• Identify and address hurdles to clinical development
• Clinical trial challenges
• Diagnostic criteria, standard of care, diseaseprogression
• Patient subgroups, disease severity, inclusion/exclusion criteria, patient monitoring
• Regulatory challenges
• Primary and secondary end points for efficacy
• Regulatory guidance document: Out reach to patient advocacy groups for other diseases

and non profit companies regarding

• Conference for industry executives, ME/CFS experts, experts from

adjacent disease areas, and federal agency representatives

• Cross pollination of ideas between Industry and research community in ME/CFS andother

disease areas

• Consider treatment subgroup meetings where appropriate

CT&T Working Group 3

Identify Industry Partners: Progress

✓Drugs used in clinical trials for ME/CFS

• Literature review
• Trials listed on Clinicaltrials.gov
• Findings
• Increase in published literature with a peak in 2015
• Largest category on published literature is neuromodulators with

serotonergic drugs leading the category

• Drugs of interest
• Ampligen: Small working group to understand issues related to clinical trial

requirements for physician initiated trials by Dr. Peterson

• Rituxan: Team will engage with PIs running Rituxan clinical trial to understand

reason for lack of efficacy in clinical trial. Start up company in CA with a modified CD20 molecule approached for possibility of clinical trials in ME/CFS

• Anakinra: Lack of efficacy in small trial. Anakinra requires daily administration,

more potent anti-IL-1 agents can be explored.

CT&T Working Group 4

Identify Industry Partners: Work in Progress

• Anecdotal use of drugs by clinicians and patients
• Data compiled from social media and other websites, analysis and follow

up is in progress

• Separately looking to extract data from treating physicians
• Drugs used to treat adjacent diseases
• Rationale based on disease symptoms and/or pathophysiology
• Based on preliminary work:-
• Multiple Sclerosis:

✓ Tecfidera/Dimethylfumarate (Biogen) ✓ Ampyra/Fampridine (Acorda) ✓ Amantidine (Endo)

• Myasthenia Gravis

✓ Mestinon/Pyridostigmine (generic)

• Drugs for targets identified in clinical and preclinical work in ME/CFS
• Early stage of data collection

CT&T Working Group 5

Few Significant Hurdles for Clinical Development Identified

• Heterogeneity of disease symptoms
• Identify ways to form patient subgroups
• Sparse data on natural history of disease progression
• Team is gathering data from MCAM and other studies
• Work with agencies to understand the viability and need for an

epidemiological study for ME/CFS

• Clinical trial inconsistencies
• Diagnostic criteria used
• I/E criteria
• Disease severity
• Duration of treatment
• Primary endpoint / patient outcome assessment
• Patient monitoring (signs and symptoms, and labs)
• Shrinking pool of ME/CFS treating clinicians

CT&T Working Group 6

Regulatory Challenges to be Addressed

• Regulatory guidance document requirements
• Patient advocacy groups for other diseases e.g. Duchenne’s Muscular

Dystrophy

• Non profit companies who can help with regulatory strategy
• Work with ex officios to define primary and secondary endpoints for

efficacy

• CDE vs. PROs (which can be used as approvable endpoints)
• Get updates from research community for surrogate markers and/or

biomarkers for efficacy

• Validation required for monitoring devices
• Understand CPET and it’s use in clinical trial
• Engage expert clinicians and researchers for clinical trial and

regulatory challenges

CT&T Working Group 7

Conclusions

• Capable and informed team with patients, patient advocates,

researchers, treating physician and ex-officios at the table

• Progress made in identifying potential treatment options, and

some clinical trial challenges

• A lot of work remains to be done to achieve goals set for the

working group

• Funding sources to be identified
• Industry Engagement Summit in 4Q2018-1Q2019
• ~100 - 150K
• Include
• Industry partners
• Physicians treating ME/CFS
• Researchers and clinicians in adjacent disease areas
• Federal agency representatives
• Aim to have new clinical development in ME/CFS over next

few years

CT&T Working Group 8