Repurposing Research Clinical Trials

HX Refactored 2016 Boston

Dr. Bruce E. BloomPresident and Chief Science Officer

Cures Within Reach

W W W & H?• Why I am here• Who we are• What types/phases of clinical trials• How repurposing clinical trials arise• What are our success stories• What are clinical trial issues/challenges• What are current/future trials changes

Cures Within Reach is a non-profit that works to catalyze repurposing research to quickly and affordably improve patient lives. Repurposing research seeks to create new treatments for serious unmet needs using existing drugs, devices and nutriceuticals.

We accomplish this through collaborations that connect researchers and funding, by facilitating crowd sourcing, building community and by identifying alternative financing models and incentives.

Repurposing vs. de novo research

Over 40 Academic research institution partners

23 projects currently in process Focus on late pre-clinical or

pilot clinical proof of concept 13 CWR repurposed treatments

in use by patients today.

Phases of clinical trials•Phase I-safety•Phase II-controlled population efficacy•Phase III-wider population efficacy•Phase IV-post marketing surveillance

Off label Open labelRandomized

Blinded

Controlled

Blinded

Types of Trials

Controlled

Crossover

PlaceboNatural History v test

SOC v testSOC v SOC + test

Origin of Repurposing Clinical Trials•Discovery about a disease•Discovery about a therapy•Clinical observation/co-morbidity•Off-label use/patient insight•Systematic screening

Generic Drug

DevicesChange in Therapy

Nutriceuticals

Successful CWR repurposing clinical trials

Generic drug saves lives of kids with rare auto-immune disease

6 patients clinical trial delivers life-saving treatment

Total $250K3 years

Endpoints Always Matter

Size DOESN’T Always Matter

Led to 5 additional clinical trials repurposing

same drug

Change in therapy improves success of ASCT by 40% in rare

blood cancersCould affect 600,000 patients

worldwide each year

Total $600K grant over5 years

Clinical Trials to Validate

Clinical Observations

Serendipity plus curiosity plus persistence

Started with NHL-Then applied to MM

and other blood cancers

$1M 7 years

6 repurposed nutriceuticals create 100% protein levels

Supplement combo “cures”

Familial Dysautonomia

Nature’s“drugs”

The fastest, cheapest way to a new treatment

No clinical trial required!

Laser device repurposed for prostate cancer treatment

eliminates side effects

$50K CWR pilot $500K NIH grant for Phase III trial. Device moving

into SOC at major institutions

Device+PT restores lost function-MS

$50K CWR pilot $4M for 510K FDA approval

Don’t Overlook Devices

Faster path to commercialization

Current CWR repurposing clinical trials

Phase III clinical trial repurposing

BCG

Common disease

Rare disease repurposing with CIHR

Cross border co-PIs

Generics Repurposing

England

New SIB economic engine-pay for success model

Open Source Pharma

Foundation

Repurposing for neglected disease

of poverty

Promoting RepurposingCollaboration

Created by Cures Within

Reach

Launched inJune 2015

Platform built with a grant

from the Robert Wood Johnson

Foundation

The world’s first online, interactive platform dedicated

to repurposing research, testing new treatments for unsolved diseases using

existing drugs and devices

Since November 2015

Over 120 projects submitted

From 52 Institutions

In7 Countries

11 RFPs

Oncology-45 Neuro-20Rare diseases-23 Infectious-6 Autoimmune-6 Pediatric-5 Hematology-4 Cardiology 4Ophthalmology-3 Pulmonary-2GI-1 Nephrology-1

Multiple Stakeholder Participation

Future of repurposing clinical trials•Virtual clinical research• “Big Data” retrospective studies•Prospective observational studies•Clinical trial crowd research-tech driven

Join us

1. Register for CureAccelerator™ https://app.cureaccelerator.org/registration

2. Log in and start a digital discussion, post an RFP or post a project

3. Encourage your research institution, biotech or disease specific non-profit to become a CureAccelerator partner

To learn more

Dr. Bruce Bloom Bruce@cureswithinreach.org 847-745-1245

www.cureswithinreach.org

www.cureaccelerator.org

Cures Within Reach is a 501(c)3 non-profit organization, tax ID 20-3620169